Archive for May, 2020

The Covid-19 (coronavirus) pandemic is impacting society and the overall economy across the world. The impact of this pandemic is growing day by day as well as affecting the supply chain. The COVID-19 crisis is creating uncertainty in the stock market, massive slowing of supply chain, falling business confidence, and increasing panic among the customer segments. The overall effect of the pandemic is impacting the production process of several industries including medical devices, pharmaceuticals, healthcare, biotechnology, and many more. Trade barriers are further restraining the demand- supply outlook. As government of different regions have already announced total lockdown and temporarily shutdown of industries, the overall production process being adversely affected; thus, hinder the overall Stem Cell Therapy Market globally. This report on Stem Cell Therapy Market provides the analysis on impact on Covid-19 on various business segments and country markets. The report also showcases market trends and forecast to 2027, factoring the impact of Covid -19 Situation.

The stem cell therapy marketwas valued at US$ 1,534.55 million in 2019 and is expected to grow at a CAGR of 16.7% from 2020to 2027 to reach US$ 5,129.66 million by 2027.

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What is Stem Cell Therapy Market?

Stem cells are preliminary body cells from which all other cells with specialized functions are generated. Under controlled environment in the body or a clinical laboratory, these cells divide to form more cells called daughter cells. Due to the advent of modern health science, these cells play a major role in understanding the occurrence of diseases, generation of advanced regenerative medicines, and drug discovery. There are certain sources such as embryo, bone marrow, body fats, and umbilical cord blood amongst others, where stem cells are generated. The global stem cell therapy market is driven by factors such asincreasing awareness related to the stem cells therapy in effective disease management and growing demand for regenerative medicines. However, high cost related with stem cell therapy is likely to obstruct the growth of the stem cell therapymarket during the forecast period. The growing research and development activities in Asia Pacific region is expected to offer huge growth opportunity for stem cell therapy market.

Researchers are further investigating stem cell therapy in autoimmune disorder. Other adult stem cells based treatments are under clinical trials. Hematopoietic stem cells are currently used for treating more than 80 medical diseases, which include diseases of the immune system, blood disorders, neurological disorders, metabolic disorders, genetic disorders, and several types of cancers like leukemia, lymphoma, etc.Emerging Players in the Stem Cell Therapy Market Research include:

A factor which can be a restraint for Stem Cell Therapy Market can be some companies do not collaborate with service providers or they dont take advantage of digitization as they dont have awareness for the same. Nevertheless, digitization in services is opting by an online company to know more exactly about consumer behavior plus it makes business policies flexible to adopt changes as per the market condition on which success and growth of an organization depend which will give more growth opportunities in coming years.

This report will help you determine and analyze your portfolio of key market players with information such as company profile, components and services offered, financial information from the past three years, and key developments it helps you to develop a strategy to gain a competitive edge in the past 5 years. The market payers from Stem Cell Therapy Market are anticipated to lucrative growth opportunities in the future with the rising demand for Stem Cell Therapy Market in the global market.

Key questions answered by this report:

Global Stem Cell Therapy Market By Type

Global Stem Cell Therapy Market By Treatment

Global Stem Cell Therapy Market ByApplication

Global Stem Cell Therapy Market By End User

Global Stem Cell TherapyMarket By Geography

Stem Cell Therapy Market Table of Contents:

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Stem Cell Therapy Market To Boom In Near Future By 2027 Scrutinized In New Research - Cole of Duty

The adaptable FLASH-CAR platform can be used to create personalized cell therapy from a patients own cells, as well as off-the-shelf cell therapy from a universal donor

(NASDAQ: AVCO) announced Friday that it is advancing its next-generation immune cell therapy to treat blood cancers using FLASH-CAR technology co-developed with strategic partner Arbele Limited.

The adaptable FLASH-CAR platform can be used to create personalized cell therapy from a patients own cells, as well as off-the-shelf cell therapy from a universal donor.

Currently, the Chimeric Antigen Receptor T (CAR-T) cellular immunotherapy platform is available. It involves a patients own T-cells a type of white blood cell that protects against infections and other diseases including cancer that are turned into personalized cancer-fighting cells.

The T-cells are removed from the patient, reprogrammed in the lab using a viral vector to target cancer cells, and infused back into the patient as a cancer immunotherapy.

But in contrast to these existing therapies, Avalon said its FLASH-CAR platform uses next-generation CAR technology to modify patients T-cells and natural killer (NK) cells using a ribonucleic acid (RNA)-based platform rather than a viral vector.

Similar to T-cells, NK cells are a type of white blood cell, also able to attack cancer cells, but utilize different mechanisms. By using RNA molecules rather than a viral vector, Avalons RNA-based CAR technology is designed to rapidly create personalized CAR therapies in 1 to 2 days compared to the 10- to 14-day bio-manufacturing time necessary to generate currently available CAR-T cellular immunotherapy.

Avalon said its FLASH-CAR technology is also designed to reprogram the immune cells to hone in on multiple crucial cancer cell targets, called tumor antigens, to potentially achieve superior therapeutic effect. Avoiding the use of viral vectors and complicated bio-processing procedures significantly reduces manufacturing costs, resulting in a more affordable and potentially breakthrough therapy for cancer patients.

The FLASH-CAR technology can also be used to generate off-the-shelf,universal cell therapy that has the potential to reach even more patients.

Avalons first FLASH-CAR platform candidate, AVA-011, targets both CD19 and CD22 tumor antigens on cancer cells.

Pre-clinical research on AVA-011, including tumor cytotoxicity studies, has been successfully completed and Avalon said it is immediately entering the process development stage to generate clinical-grade CAR-T and CAR-NK cells for use in human clinical trials.

Avalon and Arbele have jointly filed for US patents for this RNA-based CAR platform cellular therapy and for other applications.

Avalon expects to begin a first-in-human clinical trial with AVA-011 for the treatment of relapsed or refractory B-cell lymphoblastic leukemia (B-ALL) and non-Hodgkin lymphoma in the first quarter of 2021. The goal is to use AVA-011 as a bridge to bone marrow stem cell transplant therapy, currently the only curative approach for patients with these blood cancers.

Avalon GloboCare is committed to decreasing the time it takes to deliver cellular immunotherapies to cancer patients, as well as lowering the cost of manufacturing by building on our unique RNA-based CAR platform that does not require using a viral vector, said CEO Dr David Jin.

We are accelerating our innovative discovery and development plan, as well as delivering precise clinical execution and leadership in cellular immunotherapy. Our pre-clinical studies are encouraging and we are excited for AVA-011 to enter the clinical development stage, including multi-center clinical trials following completion of process development to generate the cell therapy.

Arbele CEO John Luk added: Through this strategic partnership with Avalon GloboCare, we envision an accelerated scientific and clinical development of the RNA-based FLASH-CAR technology platform with great potential to generate 'off-the-shelf'immune effector cell therapies to treat both hematologic and solid malignancies.

Avalon, based in Freehold, New Jersey, specializes in developing cell-based technologies and is involved in the management of stem-cell banks and clinical laboratories.

The companys stock recently traded up 10% to $1.99a share in New York.

--UPDATES stock price--

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Avalon GloboCare advancing immune cell therapy to treat blood cancers using FLASH-CAR technology - Proactive Investors USA & Canada

Thalassemia is a type of inherited blood disorder. It is passed from parents to children through genes. This disorder involves lack of oxygen-carrying protein called hemoglobin (an important part of red blood cells). When there is insufficiency of hemoglobin in the body, the red blood cells dont function properly. It also reduces the life of RBC, which means fewer healthy RBC travel in the blood.

RBC carries oxygen to all the cells of the body. Oxygen acts as food, which is used by cells to function. Shortage of healthy RBC means shortage in supply of oxygen to all other cells of the body. This may lead to lethargy in a person. The person may feel tired, weak or short of breath. This condition is termed as Anaemia.

People with thalassemia may suffer from mild or acute Anaemia. Acute Anaemia can be very severe and can lead to damage of major organs. It can even cause death.

Thalassemia major babies are born to parents who are carriers of thalassemia gene. According to rough estimates, each year some 10000 babies are born in India with thalassemia. Best way to prevent or eliminate thalassemia is screening of all pregnant women between 9 to 12 weeks.

Thalassemia is diagnosed through blood tests which include doing a complete blood count (CBC) and special hemoglobin tests. Through a sample of blood, CBC measures the amount of hemoglobin and the different kind of blood cells, such as red blood cells. Hemoglobin tests measure the types of hemoglobin in blood.

Moderate and acute thalassemia is usually diagnosed in childhood. This is because signs and symptoms, such as acute Anemia usually occur at an early age of 2 years. People who have mild form of thalassemia may get diagnosed after a routine blood test, as it will detect if they have anemia.

Here's Dr. Rahul Bhargava, Director and Head, Hematology, Haemato- Oncology and Bone Marrow Transplant, Fortis Memorial Research Institute, Gurugram has to say about the treatments:

Blood Transfusion

Treatment of thalassemia major relies on regular blood transfusion at regular intervals, to keep Hb above 9 gm. percent. It will help prevent form short stature and other skeletal and facial deformities. Recurrent lifelong blood transfusion since 6 months of birth is necessary.

Iron Chelation Therapy

With transfusion comes the problem of iron deposition, as each blood transfusion lead to incremental iron deposition in various tissues like pituitary gland liver and heart leading to early death. So along with transfusion patient also needs iron chelation therapy. It can be either oral (defriprone and defreseirox) or IV desferoxmine. Serum ferritin is one of the surrogate markers of iron overload in thalassemia patients. It needs to be done every 3 months. Gov.s efforts of providing free blood products and iron chelators is bearing fruits as life expectancy has shown an upward trend.

Bone Marrow or Stem Cell Transplant

As it is commonly known, bone marrow or stem cell transplant is the only curative modality for thalassemia. If done at an early age, 80 percent patients can be cured. Source of stem cell could be either brother or sister whose HLA is a complete match. Otherwise fully matched HLA donor can be tried in various international registries. This process is called as match unrelated donor transplant.

Gene Therapy

Gene therapy is gaining lot of traction in field of hemoglobinopathies. It has shown remarkable result with minimum toxicities and sustained haemoglobin production in various trials. There has been no major risk of cancer or other late effects.

We have come a long way and probably this decade will bring the much awaited cheers to thalassaemics. Till then in India, prevention is the only strategy to reduce the burden on already stretched health care system.

Better rate of blood transfusion

Regular Blood screening has significantly impacted reduction of infections due to blood transfusion

Significant improvement in treatment

Bone Marrow Transplant and Stem cell transplantation has led to patients having a good quality of life

Read more| 10 things to keep in mind while travelling with Asthma

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World Thalassemia Day: All you need to know from the expert - India Today

A Washington based biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announcedthe availability of thepre-print version of the manuscriptdescribing the immunological mechanism by whichleronlimabrestores immune function and impacts disease in COVID-19 patients.

This manuscript,Disruption of the CCL5/RANTES-CCR5 Pathway Restores Immune Homeostasis and Reduces Plasma Viral Load in Critical COVID-19, has been shared with the World Health Organization and is currently under peer review, said CytoDyn, Inc., as of May 6, 2020.

As described in thepre-print, in a cohort of 10critically ill patients, after treatment with leronlimab, these critically ill patients experienced reversed hyperimmune activation and inflammation, as well as reversed immunosuppression, therebyfacilitating a more effective immune responsecorrelated with decreases in SARS-CoV-2 level in blood.

These results demonstrate a novel approach to resolving unchecked inflammation whilerestoring immunologic deficiencies.

This is an important finding since according to various studies, a major driver of severe COVID-19 disease is excessive inflammation.

Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn said in a press statement, We are now most hopeful the entire medical community will understand the potential benefit leronlimab can provide critically ill COVID-19 patients."

"Moreover, this discovery by Dr. Bruce Patterson that leronlimab decreases plasma viral load may have tremendous long-term positive ramifications to bring this pandemic under control. We are grateful that we are able to release this research at such a critical time for patients throughout the world.

Leronlimab (PRO 140) is being used as a treatment for severe COVID-19 under the emergency Investigational New Drug (IND) recently granted by the U.S. Food and Drug Administration (FDA).

Leronlimab is a drug candidate that is aCCR5 antagonist with the potential for multiple therapeutic indications.Leronlimab belongs to a group of HIV drugs calledCCR5antagonists.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions.

Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells.

Previously, CytoDyn announced it is seeking a compassionate use designation for leronlimabfor the treatment of COVID-19 patients who are ineligible for participation in its two existing clinical trials. If this request is granted by the FDA, it will significantly expand the pool of patients who would be eligible to receive leronlimabtherapy.

CytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor.

Precision Vaccinations publishes developing COVID-19 therapeutic drug news.

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CCR5 Receptor Appears Effective in Modulating Inflammation - Precision Vaccinations

Sapelo Skin Care is one of those brands you'll love for a lifetime. Not only is it luxe, the products are also wrapped in the loveliest packaging, with designs that make me think of a beautifulobjetfound in a well-appointed Southern mansion. So it should be no surprise to learn that the brand is handmade in Savannah. Founders Stephanie Duttenhaver and Cindy Edward took note of the city's natural beauty, especially the rivers and creeks that swell with high tides twice each day, and followed those cues to craft a small-batch, non-toxic product. The twice-daily tinctures soothe and replenish the skin, boosting collagen, elastin and skin-cell restoration to give us the healthy glow we all want (especially as we spend more time indoors).

I gave four Sapelo products a go: the Gentle Seaweed Cleanser ($40), Milk & Honey Hydrating Mask ($75), Restoring Eye Serum ($95) and Spring Tide Serum ($195). I loved them all, and I will be returning to Saks Fifth Avenue at the Mall at University Town Centerwhich reopens with limited hours on May 12for a moisturizer to complete my regimen.

The cleanser sets the foundation for the serums, leaving the skin feeling clean and oh-so velvety. And the serums are heavenly, with bottles dispense the perfect droplet of product. They're clearly designed by women who know we do not like to waste a good thing.

Then there's the Milk & Honey Hydrating Mask, another winner. After all the ups and downs of our Sarasota temperatures these last months, my skin needed a little love and felt re-textured and re-hydrated after using the mask.

Now, it may just be me, a woman from New York, who feels that Southern woman knowthings. Such as how to deliver the hard truth with a smile (and you may even thank them for it). Or how to set a proper table for tea. Or in this case, the secret to aging gracefully.

So I had to know why Sapelo is so wonderful. Well, first, it is created in a pristine lab under careful supervision. There, Duttenhaver and Edward marry bio-active, Southern-inspired ingredients with a scientific system that mimics our body's natural healing process. It's my favorite kind of skincare: nature and science combined with cruelty-free ingredients, formulated without fillers, silicone, parabens, sulfates, GMOs, triclosan or petrochemicals.

What's more, Sapelo's formulations are a botanical profusion of gardenia stem cells, magnolia oil, oyster shell calcium, seaweed, essential oils and Georgia honey. Sigh. Now I'm craving a cold glass of sweet tea and a front porch.

Let's turn to Duttenhaver and Cindy Edwards for a quick Q&A.

Cindy Edwards: Sapelo Skin Care is named after one of the beautiful, largely undisturbed, and under-developed barrier islands off of the coast of Savannah, Georgia. It is a nod to our inspiration of nature and heeding lessons from generations of southern women to be gentle with our skin.

Stephanie Duttenhaver: Cindy and I both were looking for an authentic anti-aging strategy for our skin. We were seeing the first signs of aging, partly due to the effects of decades of southern sun exposure and to our unavoidable hormonal milestones! We were frustrated by the mediocre results of potions and lotions sold in luxury stores but were also wary of the quick-fix, invasive solutions, like dermabrasion, acid peels, and laser and light therapies.

My husband, a radiation oncologist, challenged me once with this comment: "When a radiation oncologist creates a laser burn on a cancer patient's skin, it's called a complication. But when skin is burned in a medical spa, clients pay for it. Does that make sense?"

The penny droppedmost mainstream dermatological procedures create damage to the skin; likewise, so do prestigious skincare brands containing retinoids and other acids. We realized that these strategies had one thing in common: creating havoc in the skin through damage and injury, then relying on the injury to recruit the body's immune system response to initiate repair and renewal.

When your skin is injured, your body releases a cascade of healing peptides, proteins, vitamins, and minerals to rebuild the damaged cells. We began to look at that cascade and wondered if we could replicate the restorative cascade in a skin care regimen that topically infused the same cascade but without the damage. It was at that moment we started to formulate the philosophy behind Sapelo Skin Care.

After three years of research, counsel with physicians and chemists, and the unerring support of friends and family, we launched our Three-Step Skin Recovery System. It is a gentle, stand-alone regimen that mimics our body's natural immune system response to injury but without the injury.

CE: We spent almost three years developing a product that nurtures and rejuvenates the skin gently and naturally by using Southern-inspired ingredients including gardenia stem cells, magnolia oil, oyster shells, Atlantic seaweed, bio-active peptides and micro-nutrients. We committed ourselves to only using the highest-quality ingredients in the highest quantity in our products.

For example: In our Renewing Serum, we use more than 4 percent hylauronic acid (HA) and a combination of three separate HAs at different molecular weights in order to flood the skin and really get down to all of the layers of the dermis, where new cells are produced.

SD: Relying on skin-damaging procedures and retinols as your anti-aging strategy. Our skin has an amazing capacity to heal, but that capacity is not unlimited. With repeated injury to your skin, year after year, your skin will thin we believe it leads to accelerated aging.

SD: Don't use acids to exfoliate your skin; use enzymes. Acids burn the skin, whether they are dead surface cells or healthy ones. But enzymes "eat" dead skin and leave healthy skin alone. Once you have removed the dead skin, that encourages a healthy environment for your complexion. I also do not like face scrubs. Use a soft cotton washcloth (I love baby washcloths!) to gently cleanse and remove dead skin.

SD: I love breakfast smoothies. Most mornings I will blend my favorite fruits, yogurt, cranberry juice, chia seeds and a collagen powder, which is full of peptides and amino acids, to make my daily concoction. My favorite collagen supplement is Beauty Collagen by Vital Proteins. It was developed to support collagen and hyaluronic acid production in the skin and hair. And it is manufactured in the U.S.that means a lot to me- especially in today's environment.

SD: Never turn down an opportunity to introduce or present your company's philosophy to an individual or group. As women, we are multitaskers and always juggling work, home, family and social connections. Cindy and I are no exceptionwe wonder how can we squeeze one more engagement into our day. The extra preparation and time you spend presenting your business at a small gathering or a grand occasion can often feel unwelcome or inconsequential. However, we have learned over and over again that every time we put energy out into the world, something of value always comes back to us, and that keeps us motivated and moving forward.

Sapelo Skin Care is available at Saks Fifth Avenue, 120 University Town Center Drive, Sarasota. The store is offering curbside service for purchases, returns, exchanges and alterations between 12-4 p.m., and plans to reopen to the public with limited hours12-6 p.m.on May 12. Call (941) 364-5300 for more information.

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Sapelo Skin Care Combines High-Tech Ingredients and Southern Charm - Sarasota

Youve probably seen those before-and-after quarantine memes, the ones featuring faces or more specifically, lips that have undergone a drastic change in shape in the absence of access to an aesthetician. Exaggerated they might be, but it is true that lockdown has left an injectable-shaped hole in some of our lives, and whether youre into them or not, our newly facial-free (and less-than-active) lifestyles are wreaking a certain amount of havoc on our skin anyway. For those who have regular in-clinic treatments, lockdown can mean that lines start to show, full lips diminish, cheeks depress and sunken eye sockets and dark circles re-appear, says aesthetic doctor Dr Sophie Shotter, who has temporarily shuttered her clinic and returned to the NHS frontline to join the fight against Covid-19. And, while the issue might seem trivial in light of everything else going on in the world, it is possible to harness the power of intelligent skincare to help mimic the effects of filler and Botox in lieu of the real thing. Dr Shotter shares her tips here.

Filler is routinely administered to add volume to the skin, usually in the face, says Shotter, who explains that its made from hyaluronic acid, a molecule we should all be utilising in our skincare routines to help soften fine lines. One of the best serums for the job is SkinCeuticals HA Intensifier, which boosts hyaluronic acid levels in the skin, making it look and feel firm, plump and hydrated. Alternatively, try LOrals Revitalift Filler Hyaluronic Acid for a more affordable option.

To smooth skin, Shotter also recommends BioEffect EGF Serum, which boosts skin thickness and collagen levels in the skin, meaning fewer wrinkles and a healthy glow. And finally, look to SkinBetter Science AlphaRet Overnight Cream to reap the benefits of prescription strength vitamin A (otherwise known as retinol): Retinol is the ultimate age management molecule that increases collagen levels, she says.

If youve had Botox before, you will know that its a toxin injected where there are dynamic lines to relax them and remove creases within the skin, says Shotter. There are products that mimic its effects: Meder Skincare Myo-Fix Concentrate, is full of muscle-relaxing peptides that work to block the nerve communicating with the muscle. Although it might sound daunting, its totally safe to use, and Shotter recommends regular and sustained use for the best results. Another great product to use if you usually have Botox administered superficially to regulate oil production is Institut Esthederms Pure System Pore Refiner Concentrate it is silicone based so refines their appearance beautifully.

Used religiously, there are products that can help to lift and plump the lips, although, like all of the products listed here, they wont have effects on a par with the treatment itself. One of the best to try is Fillerinas Lip Volume Grade 3, which contains six different hyaluronic acid molecules of varying weights and sizes, to deeply hydrate lips and give a fuller effect with continual use. Shotter also recommends a brilliant lip balm. Medik8s Mutiny Squalane Lip Balm helps to nourish and heal the lips, drawing moisture to the area to give them a plumper, more hydrated look, she says, adding that its important to use SPF to protect them from collagen-depleting UV rays. Protecting the health of the lips will help them stay plump and full.

Filler is often strategically placed to fill the hollows under the eyes, to refresh and hydrate and to lift dark circles, says Shotter. A (plant-based) stem cell serum, like Dr Levy Intense Stem Cell Eye Booster Concentrate, will boost the thickness of the delicate under eye skin, plus it also contains retinol and antioxidants to smooth fine lines and brighten the area. MGC Dermas CBD Stem Cells and Peptides Eye Cream is a brilliant option too, thanks to firming peptides, hyaluronic acid and calming CBD.

More from British Vogue:

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11 Skincare Products To Try Now Injectables Are Off The Cards - British Vogue

Marky Jaquez, 19, has recessive dystrophic epidermolysis bullosa, a condition found in fewer than one in one million newborns. (Picture: Melissa Jaquez/Metro.co.uk)

A teenager whose rare skin disorder means that even the slightest touch leaves him in extreme pain has defied doctors predictions to live to adulthood. Marky Jaquez, from Wichita, Kansas, cannot hug his parents, wear clothes or even walk because any friction causes layers of skin to peel away from his body.

Wheelchair-bound, Marky, 19, has never been able to walk because the ground would shred his feet like a cheese grater and must be covered head-to-toe in bandages to protect his fragile skin from tearing. He has recessive dystrophic epidermolysis bullosa, a condition found in an estimated one in one million newborns that often leads to life-threatening skin infections and even cancer.

The illness is caused by a faulty gene, passed onto children by parents. Tragically, Markys brother Carlos was born with it too, and died aged just 14. Doctors warned Markys parents he was unlikely to live beyond the age of 11, but he defied that grim prediction and will celebrate his 20th birthday later this year.

His mom, Melissa Jaquez, 40, said: Marky only has one layer of skin and because of his genetic makeup, the skin has no way of anchoring itself the body. Any form of touch or friction means his skins just tears away.

Its world shattering to think I will lose both of my boys to this and I dont want to think about what life will be like afterwards. I feel like my purpose in life is to give Mary the best life I can for the time that I have him.

I am not able to hug my son because any friction just causes his skin to come away and tear right off. It causes him excruciating pain and I feel so much guilt, but theres nothing I can do to help him.

But despite all of this he is a very happy and joyful boy and he amazes me every single day. Hes an inspiration and proves to everyone that you can keep going with whatever youre going through, no matter how bad it is.

Marky is the youngest of three brothers born to healthcare worker, Melissa. Eldest son Michael is 24, with Carlos dying of heart failure caused by the skin condition in 2015. Marky and Carlos, who would be 21 this year, were both born with the same condition after inheriting a faulty gene from their parents.

Melissa said doctors suspect both she and the boys father carry the faulty gene that causes the genetic mutation. Although both are unaffected themselves, they have an estimated 25% chance of having a child born with recessive dystrophic epidermolysis bullosa. Melissa has since split up with Marky and Carlos father neither of whom knew they carried the faulty gene until Carlos was born, she said.

Melissa said she became aware of the risks involved with having a child after Carlos was born, but claims she was told by a clinician that the chance of her having a second child with the same condition would be less than 1%.

Melissa said: I lost Carlos to the condition because his heart just gave up. His body was in a constant battle to repair his wounds and that took its toll eventually.

His condition was much more progressive than Markys. He got real bad, real quick and by the end could not even breathe on his own.

My pregnancy with Marky was unplanned, but when I got pregnant I was told the chance of me having another baby with the same condition is 1%. They said having two children with it is unheard of.

Marky was born with the most severe type of epidermolysis bullosa meaning he was delivered without skin on his hands, feet and chest because because of the friction of childbirth.

Ever since Marky was little, Melissa, who is now married to Marcos, 30, has had to spend up to three hours a day covering his skin in protective bandages and cleaning open wounds to prevent infection.

She has said every day of motherhood so far has felt like a battle and admits she has struggled to have a normal mother and son relationship with Marky, because of his condition.

Every day is such a massive challenge for us. I struggle with the grief of knowing that when I walk into my sons room in a morning, we cant have fun. I dont get to do the normal things that a mom and a son does.

Instead when I go into his room I know I have to cause him pain and spend two hours doing his bandages. That does give me a lot of anxiety and guilt.

Taking his bandages off does cause excruciating pain. We have to soak his skin every day to soothe his wounds and give him pain medication. Its incredibly upsetting for me.

Whilst there is no cure for epidermolysis bullosa and Marky is highly likely to sadly one day lose his life to the condition, Melissa is hopefully he will survive for several more years.

He has been selected to take part in experimental treatment to create genetically modified skin stem cells that can slow down the effects of the progressive disease. The family plan to travel to Stamford Hospital, Connecticut, later this year to undergo the treatment.

Melissa and Marcos work opposite shifts and have a carer who comes to help look after Marky for three hours each day. Melissa has paid tribute to her amazing son, who she believes can go on to live for many happy years, despite the predictions of doctors.

She said: The treatment at Stamford could add several years to his life. It will reduce the pain and make it easier for his body to cope.

We have tried for that treatment for years, so were thrilled.

Marky has always been an incredibly happy boy and he has just got on with it, without ever moaning or saying why me. Ive had messages from people all over the world to say Marky has inspired them.

Hes an amazing human being and he makes me so proud.

Go here to read the rest:
Terminally-ill teen with agonizingly-sensitive skin defies doctors to live to 19 - Metro.co.uk

This story was initially printed 2020/03/19 9:55am PDTon Mar 19, 2020 and final up to date 2020/05/08 11:34am PDTCould eight, 2020.

Again at F8 2019, Fb promised a redesign for its cellular apps and web site. The Android app began receiving the brand new interface nearly instantly, however the change was solely promised on the net in the following few months. Now, it is beginning to roll out and together with it comes a brand new darkish mode possibility.

For the previous few days, many users have reported that they received prompted by Fb to check out the brand new interface. After accepting, they get requested in the event that theyd like to make use of the white or darkish structure. The selection can also be out there underneath the fast settings accessible from the highest proper avatar.

Picture credit score: @teepusahab, @kimsantoshansen

After all, the brand new design isnt universally liked, and it even has issues with text readability in a few dark mode screens. However that is the worth of any early launch.

If you arent getting prompted by Fbs web site to attempt the brand new look, there does not appear to be any workaround to force-trigger it simply but.

Rolling out to more users

The new Facebook web UI with the dark theme seems to be rolling out widely. Thanks, Anurag Dalmia!

Facebook announcement

Based on the reports we received, it was clear that Facebooks new look was already being widely deployed, but today the company has put the final pin in this story by formally announcing the UI overhaul.

In a news post, Fb discusses its planning and analysis efforts that went in to designing the brand new interface. Not everybodys been a fan of those modifications, however at the very least we are able to hear Fb out on why it thinks this new look is superior.

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Top 10 Best The Body Shop Anti Aging Body Creams 2020 - Best gaming pro

You should read the following discussion and analysis of our financial conditionand results of operations together with the condensed consolidated financialstatements and related notes that are included elsewhere in this QuarterlyReport on Form 10-Q and our Annual Report on Form 10-K for the fiscal year endedDecember 31, 2019 filed with the U.S. Securities and Exchange Commission, or theSEC, on March 6, 2020, or our 2019 Form 10-K. This discussion containsforward-looking statements based upon current plans, expectations and beliefsthat involve risks and uncertainties. Our actual results may differ materiallyfrom those anticipated in these forward-looking statements as a result ofvarious factors, including, but not limited to, those discussed in the sectionentitled "Risk Factors" and elsewhere in this Quarterly Report on Form 10-Q. Inpreparing this MD&A, we presume that readers have access to and have read theMD&A in our 2019 Form 10-K, pursuant to Instruction 2 to paragraph (b) of Item303 of Regulation S-K. Unless stated otherwise, references in this QuarterlyReport on Form 10-Q to "us," "we," "our," or our "Company" and similar termsrefer to Rocket Pharmaceuticals, Inc.

We are a clinical-stage, multi-platform biotechnology company focused on thedevelopment of first, only and best-in-class gene therapies, with directon-target mechanism of action and clear clinical endpoints, for rare anddevastating diseases. We currently have three clinical-stage ex vivo lentiviralvector ("LVV") programs currently enrolling patients in the US and EU forFanconi Anemia ("FA"), a genetic defect in the bone marrow that reducesproduction of blood cells or promotes the production of faulty blood cells,Leukocyte Adhesion Deficiency-I ("LAD-I"), a genetic disorder that causes theimmune system to malfunction and Pyruvate Kinase Deficiency ("PKD"), a rare redblood cell autosomal recessive disorder that results in chronic non-spherocytichemolytic anemia. Of these, both the Phase 2 FA program and the Phase 1/2 LAD-Iprogram are in registration-enabling studies in the US and EU. In addition, inthe US we have a clinical stage in vivo adeno-associated virus ("AAV") programfor Danon disease, a multi-organ lysosomal-associated disorder leading to earlydeath due to heart failure. Finally, we have a pre-clinical stage LVV programfor Infantile Malignant Osteopetrosis ("IMO"), a genetic disorder characterizedby increased bone density and bone mass secondary to impaired bone resorption -this program is anticipated to enter the clinic in 2020. We have globalcommercialization and development rights to all of these product candidatesunder royalty-bearing license agreements. Additional work in the discovery stagefor an FA CRISPR/CAS9 program as well as a gene therapy program for the lesscommon FA subtypes C and G is ongoing.

Recent Developments

On February 20, 2020, we entered into separate, privately negotiated exchangeagreements (the "Exchange Agreements") with certain holders of our outstanding5.75% Convertible Senior Notes due 2021 (the "2021 Convertible Notes") to extendthe maturity date by one year. Pursuant to the Exchange Agreements, we exchangedapproximately $39.35 million aggregate principal amount of the 2021 ConvertibleNotes (which represents approximately 76% of the aggregate outstanding principalamount of the 2021 Convertible Notes) for (a) approximately $39.35 millionaggregate principal amount of 6.25% Convertible Senior Notes due August 2022(the "2022 Convertible Notes") (an exchange ratio equal to 1.00 2022 ConvertibleNote per exchanged 2021 Convertible Note) and (b) $119,416 in cash to pay theaccrued and unpaid interest on the exchanged 2021 Convertible Notes from, andincluding, February 1, 2020 to February 20, 2020. The 2022 Convertible Noteswere issued in private placements exempt from registration in reliance onSection 4(a) (2) of the Securities Act of 1933, as amended (the "SecuritiesAct"). Upon completion of the exchange transactions, approximately $12.65million aggregate principal amount of 2021 Convertible Notes remainedoutstanding.

Gene Therapy Overview

Genes are composed of sequences of deoxyribonucleic acid ("DNA"), which code forproteins that perform a broad range of physiologic functions in all livingorganisms. Although genes are passed on from generation to generation, geneticchanges, also known as mutations, can occur in this process. These changes canresult in the lack of production of proteins or the production of alteredproteins with reduced or abnormal function, which can in turn result in disease.

Gene therapy is a therapeutic approach in which an isolated gene sequence orsegment of DNA is administered to a patient, most commonly for the purpose oftreating a genetic disease that is caused by genetic mutations. Currentlyavailable therapies for many genetic diseases focus on administration of largeproteins or enzymes and typically address only the symptoms of the disease. Genetherapy aims to address the disease-causing effects of absent or dysfunctionalgenes by delivering functional copies of the gene sequence directly into thepatient's cells, offering the potential for curing the genetic disease, ratherthan simply addressing symptoms.

We are using modified non-pathogenic viruses for the development of our genetherapy treatments. Viruses are particularly well suited as delivery vehiclesbecause they are adept at penetrating cells and delivering genetic materialinside a cell. In creating our viral delivery vehicles, the viral (pathogenic)genes are removed and are replaced with a functional form of the missing ormutant gene that is the cause of the patient's genetic disease. The functionalform of a missing or mutant gene is called a therapeutic gene, or the"transgene." The process of inserting the transgene is called "transduction."Once a virus is modified by replacement of the viral genes with a transgene, themodified virus is called a "viral vector." The viral vector delivers thetransgene into the targeted tissue or organ (such as the cells inside apatient's bone marrow). We have two types of viral vectors in development, LVVand AAV. We believe that our LVV and AAV-based programs have the potential tooffer a long-lasting and significant therapeutic benefit to patients.

Gene therapies can be delivered either (1) ex vivo (outside the body), in whichcase the patient's cells are extracted and the vector is delivered to thesecells in a controlled, safe laboratory setting, with the modified cells thenbeing reinserted into the patient, or (2) in vivo (inside the body), in whichcase the vector is injected directly into the patient, either intravenously("IV") or directly into a specific tissue at a targeted site, with the aim ofthe vector delivering the transgene to the targeted cells.

We believe that scientific advances, clinical progress, and the greaterregulatory acceptance of gene therapy have created a promising environment toadvance gene therapy products as these products are being designed to restorecell function and improve clinical outcomes, which in many cases includeprevention of death at an early age.

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The chart below shows the current phases of development of Rocket's programs andproduct candidates:

LVV Programs. Rocket's LVV-based programs utilize third-generation,self-inactivating lentiviral vectors to target selected rare diseases.Currently, Rocket is developing LVV programs to treat FA, LAD-I, PKD, and IMO.

Fanconi Anemia Complementation Group A (FANCA):

FA, a rare and life-threatening DNA-repair disorder, generally arises from amutation in a single FA gene. An estimated 60 to 70% of cases arise frommutations in the Fanconi-A ("FANCA") gene, which is the focus of our program. FAresults in bone marrow failure, developmental abnormalities, myeloid leukemiaand other malignancies, often during the early years and decades of life. Bonemarrow aplasia, which is bone marrow that no longer produces any or very few redand white blood cells and platelets leading to infections and bleeding, is themost frequent cause of early morbidity and mortality in FA, with a median onsetbefore 10 years of age. Leukemia is the next most common cause of mortality,ultimately occurring in about 20% of patients later in life. Solid organmalignancies, such as head and neck cancers, can also occur, although at lowerrates during the first two to three decades of life.

Although improvements in allogeneic (donor-mediated) hematopoietic stem celltransplant ("HSCT"), currently the most frequently utilized therapy for FA, haveresulted in more frequent hematologic correction of the disorder, HSCT isassociated with both acute and long-term risks, including transplant-relatedmortality, graft versus host disease ("GVHD"), a sometimes fatal side effect ofallogeneic transplant characterized by painful ulcers in the GI tract, livertoxicity and skin rashes, as well as increased risk of subsequent cancers. Ourgene therapy program in FA is designed to enable a minimally toxic hematologiccorrection using a patient's own stem cells during the early years of life. Webelieve that the development of a broadly applicable autologous gene therapy canbe transformative for these patients.

Each of our LVV-based programs utilize third-generation, self-inactivatinglentiviral vectors to correct defects in patients' HSCs, which are the cellsfound in bone marrow that are capable of generating blood cells over a patient'slifetime. Defects in the genetic coding of HSCs can result in severe, andpotentially life-threatening anemia, which is when a patient's blood lacksenough properly functioning red blood cells to carry oxygen throughout the body.Stem cell defects can also result in severe and potentially life-threateningdecreases in white blood cells resulting in susceptibility to infections, and inplatelets responsible for blood clotting, which may result in severe andpotentially life-threatening bleeding episodes. Patients with FA have a geneticdefect that prevents the normal repair of genes and chromosomes within bloodcells in the bone marrow, which frequently results in the development of acutemyeloid leukemia ("AML"), a type of blood cancer, as well as bone marrow failureand congenital defects. The average lifespan of an FA patient is estimated to be30 to 40 years. The prevalence of FA in the US and EU is estimated to be about4,000, and given the efficacy seen in non-conditioned patients, the addressableannual market opportunity is now thought to be in the 400 to 500 range.

We currently have one LVV-based program targeting FA, RP-L102. RP-L102 is ourlead lentiviral vector based program that we in-licensed from Centro deInvestigaciones Energticas, Medioambientales y Tecnolgicas ("CIEMAT"), whichis a leading research institute in Madrid, Spain. RP-L102 is currently beingstudied in our sponsored Phase 2 registrational enabling clinical trialstreating FA patients initially at the Center for Definitive and CurativeMedicine at Stanford University School of Medicine ("Stanford") and HospitalInfantil de Nino Jesus ("HNJ") in Spain. The Phase 2 portion of the trial isexpected to enroll ten patients total from the U.S. and EU. Patients willreceive a single IV infusion of RP-L102 that utilizes fresh cells and "ProcessB" which incorporates a modified stem cell enrichment process, transductionenhancers, as well as commercial-grade vector and final drug product.

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Table of ContentsIn October 2019, at the European Society of Cell and Gene Therapy ("ESGCT") 2019Annual Congress, long-term Phase 1/2 clinical data of RP-L102, from the clinicaltrial sponsored by CIEMAT, for FA "Process A", without the use of myeloablativeconditioning was presented demonstrating evidence of increasing and durableengraftment leading to bone marrow restoration exceeding the 10% thresholdagreed to by the FDA and EMA for the ongoing registration-enabling Phase 2trial. In patient 02002, who received what we consider adequate drug product,hemoglobin levels are now similar to those in the first year after birth,suggesting hematologic correction over the long term.

During the third quarter of 2019, we received alignment from the FDA on thetrial design and the primary endpoint. This alignment was similar to thatpreviously received from the European Medicines Agency ("EMA"). Resistance tomitomycin-C, a DNA damaging agent, in bone marrow stem cells at a minimum timepoint of one year to serve as the primary endpoint for our Phase II study. InDecember 2019, we announced that the first patient of the global Phase 2 studyfor RP-L102 "Process B" for FA received investigational therapy. There will betotal of 10 patients enrolled in the global Phase 2 studies.

In December 2019, we also announced preliminary results from two pediatricpatients treated with "Process B" RP-L102 prior to development of severe bonemarrow failure in our Phase 1 trial of RP-L102 for FA. To evaluate transductionefficiency, an analysis of the proportion of the MMC-resistant colony formingcells was conducted and both patients have thus far exhibited early signs ofengraftment, including increases in blood cell lineages in one patient. Nodrug-related safety or tolerability issues have been reported.

Leukocyte Adhesion Deficiency-I (LAD-I):

LAD-I is a rare autosomal recessive disorder of white blood cell adhesion andmigration, resulting from mutations in the ITGB2 gene encoding for the Beta-2Integrin component, CD18. Deficiencies in CD18 result in an impaired ability forneutrophils (a subset of infection-fighting white blood cells) to leave bloodvessels and enter into tissues where these cells are needed to combatinfections. As is the case with many rare diseases, true estimates of incidenceare difficult; however, several hundred cases have been reported to date.

Most LAD-I patients are believed to have the severe form of the disease. SevereLAD-I is notable for recurrent, life-threatening infections and substantialinfant mortality in patients who do not receive an allogeneic HSCT. Mortalityfor severe LAD-I has been reported as 60 to 75% by age two in the absence ofallogeneic HCST.

We currently have one program targeting LAD-I, RP-L201. RP-L201 is a clinicalprogram that we in-licensed from CIEMAT. We have partnered with UCLA to leadU.S. clinical development efforts for the LAD-I program. UCLA and its Eli andEdythe Broad Center of Regenerative Medicine and Stem Cell Research is servingas the lead U.S. clinical research center for the registrational clinical trialfor LAD-I, and HNJ is serving as the lead clinical site in Spain.

The ongoing open-label, single-arm, Phase 1/2 registration enabling clinicaltrial of RP-L201 has dosed one severe LAD-I patient in the U.S. to assess thesafety and tolerability of RP-L201. The first patient was treated with RP-L201in third quarter 2019. This study has received $6.5 million CLIN2 grant awardfrom the California Institute for Regenerative Medicine ("CIRM") to support theclinical development of gene therapy for LAD-I.

In December 2019, we announced initial results from the first pediatric patienttreated with RP-L201, demonstrating early evidence of safety. Analyses ofperipheral vector copy number ("VCN"), and CD18-expressing neutrophils wereperformed through three months after infusion of RP-L201 to evaluate engraftmentand phenotypic correction. The patient exhibited early signs of engraftment withVCN myeloid levels at 1.5 at three months and CD-18 expression of 45%. No safetyor tolerability issues related to RP-L201 administration (or investigationalproduct) had been identified as of that date. The study is expected to enrollnine patients globally.

Pyruvate Kinase Deficiency (PKD):

Red blood cell PKD is a rare autosomal recessive disorder resulting frommutations in the pyruvate kinase L/R ("PKLR") gene encoding for a component ofthe red blood cell ("RBC") glycolytic pathway. PKD is characterized by chronicnon-spherocytic hemolytic anemia, a disorder in which RBCs do not assume anormal spherical shape and are broken down, leading to decreased ability tocarry oxygen to cells, with anemia severity that can range from mild(asymptomatic) to severe forms that may result in childhood mortality or arequirement for frequent, lifelong RBC transfusions. The pediatric population isthe most commonly and severely affected subgroup of patients with PKD, and PKDoften results in splenomegaly (abnormal enlargement of the spleen), jaundice andchronic iron overload which is likely the result of both chronic hemolysis andthe RBC transfusions used to treat the disease. The variability in anemiaseverity is believed to arise in part from the large number of diverse mutationsthat may affect the PKLR gene. Estimates of disease incidence have rangedbetween 3.2 and 51 cases per million in the white U.S. and EU population.Industry estimates suggest at least 2,500 cases in the U.S. and EU have alreadybeen diagnosed despite the lack of FDA-approved molecularly targeted therapies.Enrollment is currently ongoing and we anticipate treating the first patient inthe third quarter of 2020.

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Table of ContentsWe currently have one LVV-based program targeting PKD, RP-L301. RP-L301 is aclinical stage program that we in-licensed from CIEMAT. The IND for RP-L301 toinitiate a global Phase 1 study was cleared by the FDA in October 2019. Thisprogram has been granted EMA orphan drug disease designation and FDA orphan drugdisease designation ("ODD").

This global Phase 1 open-label, single-arm, clinical trial is expected to enrollsix adult and pediatric transfusion-dependent PKD patients in the U.S. andEurope. Lucile Packard Children's Hospital Stanford will serve as the lead sitein the U.S. for adult and pediatric patients, and Hospital InfantilUniversitario Nio Jess will serve as the lead site in Europe for pediatricsand Hospital Universitario Fundacin Jimnez Daz will serve as the lead site inEurope for adult patients.

Infantile Malignant Osteopetrosis (IMO):

IMO is a genetic disorder characterized by increased bone density and bone masssecondary to impaired bone resorption. Normally, small areas of bone areconstantly being broken down by special cells called osteoclasts, then madeagain by cells called osteoblasts. In IMO, the cells that break down bone(osteoclasts) do not work properly, which leads to the bones becoming thickerand not as healthy. Untreated IMO patients may suffer from a compression of thebone-marrow space, which results in bone marrow failure, anemia and increasedinfection risk due to the lack of production of white blood cells. Untreated IMOpatients may also suffer from a compression of cranial nerves, which transmitsignals between vital organs and the brain, resulting in blindness, hearing lossand other neurologic deficits.

We currently have one LVV-based program targeting IMO, RP-L401. RP-L401 is apreclinical program that we in-licensed from Lund University, Sweden. Thisprogram has been granted ODD and Rare Pediatric Disease designation from theFDA. The FDA defines a "rare pediatric disease" as a serious andlife-threatening disease that affects less than 200,000 people in the U.S. thatare aged between birth to 18 years. The Rare Pediatric Disease designationprogram allows for a sponsor who receives an approval for a product topotentially qualify for a voucher that can be redeemed to receive a priorityreview of a subsequent marketing application for a different product. We havepartnered with UCLA to lead U.S. clinical development efforts for the IMOprogram and anticipate that UCLA will serve as the lead U.S. clinical site forIMO. We intend to file an IND for IMO and commence our clinical trial in thefourth quarter of 2020.

Danon disease is a multi-organ lysosomal-associated disorder leading to earlydeath due to heart failure. Danon disease is caused by mutations in the geneencoding lysosome-associated membrane protein 2 ("LAMP-2"), a mediator ofautophagy. This mutation results in the accumulation of autophagic vacuoles,predominantly in cardiac and skeletal muscle. Male patients often require hearttransplantation and typically die in their teens or twenties from progressiveheart failure. Along with severe cardiomyopathy, other Danon disease symptomscan include skeletal muscle weakness, liver disease, and intellectualimpairment. There are no specific therapies available for the treatment of Danondisease. RP-A501 is in clinical trials as an in vivo therapy for Danon disease,which is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S.and the EU, however new market research is being performed and the prevalence ofpatients may be updated in the future.

In January 2019, we announced the clearance of our IND application by the FDAfor RP-A501, and in February 2019, we were notified by the FDA that we weregranted Fast Track designation for RP-A501. University of California San DiegoHealth is the initial and lead center for our Phase 1 clinical trial.

On May 2, 2019, we presented additional preclinical data at the ASCGT annualmeeting, indicating that high VCN, in Danon disease-relevant organs in both miceand non-human primates ("NHN's"), with high concentrations in heart and livertissue (for NHP, cardiac VCN was approximately 10 times higher on average thanin skeletal muscle and central nervous system), which is consistent withreported results in several studies of heart tissue across different species.There were no treatment-related adverse events or safety issues up to thehighest dose. We have dosed three patients in the RP-A501 phase 1 clinicaltrial. We will continue further enrollment with clinical data read-outs in thefourth quarter of 2020.

As of March 2020, we have dosed three patients in the RP-A501 phase 1 clinicaltrial. This completes the first low dose cohort of the Phase 1 study. Based onthe preliminary safety and efficacy data review of this completed cohort, boththe FDA and IDMC has provided clearance to advance to a higher dose cohort inPhase 1 Trial of RP-A501 for Danon Disease. We will continue further enrollmentwith clinical data read-outs in the second half of 2020.

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In addition to its LVV and AAV programs, we also have a program evaluatingCRISPR/Cas9-based gene editing for FA. This program is currently in thediscovery phase. CRISPR/Cas9-based gene editing is a different method ofcorrecting the defective genes in a patient, where the editing is very specificand targeted to a particular gene sequence. "CRISPR/Cas9" stands for Clustered,Regularly Interspaced Short Palindromic Repeats ("CRISPR") Associated protein-9.The CRISPR/Cas9 technology can be used to make "cuts" in DNA at specific sitesof targeted genes, making it potentially more precise in delivering genetherapies than traditional vector-based delivery approaches. CRISPR/Cas9 canalso be adapted to regulate the activity of an existing gene without modifyingthe actual DNA sequence, which is referred to as gene regulation.

Strategy

We seek to bring hope and relief to patients with devastating, undertreated,rare pediatric diseases through the development and commercialization ofpotentially curative first-in-class gene therapies. To achieve these objectives,we intend to develop into a fully-integrated biotechnology company. In the near-and medium-term, we intend to develop our first-in-class product candidates,which are targeting devastating diseases with substantial unmet need, developproprietary in-house analytics and manufacturing capabilities and continue tocommence registration trials for our currently planned programs. In the mediumand long-term, we expect to submit our first biologics license applications("BLAs"), and establish our gene therapy platform and expand our pipeline totarget additional indications that we believe to be potentially compatible withour gene therapy technologies. In addition, during that time, we believe thatour currently planned programs will become eligible for priority review vouchersfrom the FDA that provide for expedited review. We have assembled a leadershipand research team with expertise in cell and gene therapy, rare disease drugdevelopment and commercialization.

We believe that our competitive advantage lies in our disease-based selectionapproach, a rigorous process with defined criteria to identify target diseases.We believe that this approach to asset development differentiates us as a genetherapy company and potentially provides us with a first-mover advantage.

Financial Overview

Since our inception, we have devoted substantially all of our resources toorganizing and staffing the Company, business planning, raising capital,acquiring or discovering product candidates and securing related intellectualproperty rights, conducting discovery, research and development activities forthe programs and planning for potential commercialization. We do not have anyproducts approved for sale and have not generated revenue from product sales.From inception through March 31, 2020, we raised net cash proceeds ofapproximately $373.1 million from investors through both equity and convertibledebt financing to fund operating activities. As of March 31, 2020, we had cash,cash equivalents and investments of $275.9 million.

Since inception, we have incurred significant operating losses. Our ability togenerate product revenue sufficient to achieve profitability will depend heavilyon the successful development and eventual commercialization of one or more ofthe current or future product candidates and programs. We had net losses of$24.7 million for the three months ended March 31, 2020 and $77.3 million forthe year ended December 31, 2019. As of March 31, 2020, we had an accumulateddeficit of $207.8 million. We expect to continue to incur significant expensesand higher operating losses for the foreseeable future as we advance our currentproduct candidates from discovery through preclinical development and clinicaltrials and seek regulatory approval of our product candidates. In addition, ifwe obtain marketing approval for any of their product candidates, we expect toincur significant commercialization expenses related to product manufacturing,marketing, sales and distribution. Furthermore, we expect to incur additionalcosts as a public company. Accordingly, we will need additional financing tosupport continuing operations and potential acquisitions of licensing or otherrights for product candidates.

Until such a time as we can generate significant revenue from product sales, ifever, we will seek to fund our operations through public or private equity ordebt financings or other sources, which may include collaborations with thirdparties and government programs or grants. Adequate additional financing may notbe available to us on acceptable terms, or at all. We can make no assurancesthat we will be able to raise the cash needed to fund our operations and, if wefail to raise capital when needed, we may have to significantly delay, scaleback or discontinue the development and commercialization of one or more productcandidates or delay pursuit of potential in-licenses or acquisitions.

Because of the numerous risks and uncertainties associated with productdevelopment, we are unable to predict the timing or amount of increased expensesor when or if we will be able to achieve or maintain profitability. Even if weare able to generate product sales, we may not become profitable. If we fail tobecome profitable or are unable to sustain profitability on a continuing basis,then we may be unable to continue our operations at planned levels and be forcedto reduce or terminate our operations.

Revenue

To date, we have not generated any revenue from any sources, including fromproduct sales, and we do not expect to generate any revenue from the sale ofproducts in the near future. If our development efforts for product candidatesare successful and result in regulatory approval or license agreements withthird parties, we may generate revenue in the future from product sales.

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Research and Development Expenses

Our research and development program ("R&D") expenses consist primarily ofexternal costs incurred for the development of our product candidates. Theseexpenses include:

expenses incurred under agreements with research institutions that conduct

research and development activities including, process development,

preclinical, and clinical activities on Rocket's behalf;

costs related to process development, production of preclinical and clinical

materials, including fees paid to contract manufacturers and manufacturing

input costs for use in internal manufacturing processes;

consultants supporting process development and regulatory activities; and

costs related to in-licensing of rights to develop and commercialize our

product candidate portfolio.

We recognize external development costs based on contractual payment schedulesaligned with program activities, invoices for work incurred, and milestoneswhich correspond with costs incurred by the third parties. Nonrefundable advancepayments for goods or services to be received in the future for use in researchand development activities are recorded as prepaid expenses.

Our direct research and development expenses are tracked on a program-by-programbasis for product candidates and consist primarily of external costs, such asresearch collaborations and third party manufacturing agreements associated withour preclinical research, process development, manufacturing, and clinicaldevelopment activities. Our direct research and development expenses by programalso include fees incurred under license agreements. Our personnel, non-programand unallocated program expenses include costs associated with activitiesperformed by our internal research and development organization and generallybenefit multiple programs. These costs are not separately allocated by productcandidate and consist primarily of:

Our research and development activities are central to our business model.Product candidates in later stages of clinical development generally have higherdevelopment costs than those in earlier stages of clinical development. As aresult, we expect that research and development expenses will increasesubstantially over the next several years as we increase personnel costs,including stock-based compensation, support ongoing clinical studies, seek toachieve proof-of-concept in one or more product candidates, advance preclinicalprograms to clinical programs, and prepare regulatory filings for productcandidates.

We cannot determine with certainty the duration and costs to complete current orfuture clinical studies of product candidates or if, when, or to what extent wewill generate revenues from the commercialization and sale of any of our productcandidates that obtain regulatory approval. We may never succeed in achievingregulatory approval for any of our product candidates. The duration, costs, andtiming of clinical studies and development of product candidates will depend ona variety of factors, including:

the scope, rate of progress, and expense of ongoing as well as any future

clinical studies and other research and development activities that we

undertake;

future clinical trial results;

uncertainties in clinical trial enrollment rates;

changing standards for regulatory approval; and

the timing and receipt of any regulatory approvals.

We expect research and development expenses to increase for the foreseeablefuture as we continue to invest in research and development activities relatedto developing product candidates, including investments in manufacturing, as ourprograms advance into later stages of development and as we conduct additionalclinical trials. The process of conducting the necessary clinical research toobtain regulatory approval is costly and time-consuming, and the successfuldevelopment of product candidates is highly uncertain. As a result, we areunable to determine the duration and completion costs of research anddevelopment projects or when and to what extent we will generate revenue fromthe commercialization and sale of any of our product candidates.

Our future research and development expenses will depend on the clinical successof our product candidates, as well as ongoing assessments of the commercialpotential of such product candidates. In addition, we cannot forecast with anydegree of certainty which product candidates may be subject to futurecollaborations, when such arrangements will be secured, if at all, and to whatdegree such arrangements would affect our development plans and capitalrequirements. We expect our research and development expenses to increase infuture periods for the foreseeable future as we seek to complete development ofour product candidates.

The successful development and commercialization of our product candidates ishighly uncertain. This is due to the numerous risks and uncertainties associatedwith product development and commercialization, including the uncertainty of:

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Table of Contents

the scope, progress, outcome and costs of our clinical trials and other

research and development activities;

the efficacy and potential advantages of our product candidates compared to

alternative treatments, including any standard of care;

the market acceptance of our product candidates;

obtaining, maintaining, defending and enforcing patent claims and other

intellectual property rights;

significant and changing government regulation; and

the timing, receipt and terms of any marketing approvals.

A change in the outcome of any of these variables with respect to thedevelopment of our product candidates that we may develop could mean asignificant change in the costs and timing associated with the development ofour product candidates. For example, if the FDA or another regulatory authoritywere to require us to conduct clinical trials or other testing beyond those thatwe currently contemplate for the completion of clinical development of any ofour product candidates that we may develop or if we experience significantdelays in enrollment in any of our clinical trials, we could be required toexpend significant additional financial resources and time on the completion ofclinical development of that product candidate.

General and Administrative Expenses

General and administrative ("G&A") expenses consist primarily of salaries andrelated benefit costs for personnel, including stock-based compensation andtravel expenses for our employees in executive, operational, finance, legal,business development, and human resource functions. In addition, othersignificant general and administrative expenses include professional fees forlegal, patents, consulting, investor and public relations, auditing and taxservices as well as other expenses for rent and maintenance of facilities,insurance and other supplies used in general and administrative activities. Weexpect general and administrative expenses to increase for the foreseeablefuture due to anticipated increases in headcount to support the continuedadvancement of our product candidates. We also anticipate that we will incurincreased accounting, audit, legal, regulatory, compliance and director andofficer insurance costs as well as investor and public relations expenses.

Interest Expense

Interest expense is related to the 2021 Convertible Notes, which mature inAugust 2021, and the 2022 Convertible Notes, which mature in August 2022.

Interest Income

Interest income is related to interest earned from investments.

Critical Accounting Policies and Significant Judgments and Estimates

Our consolidated financial statements are prepared in accordance with generallyaccepted accounting principles in the U.S. The preparation of our financialstatements and related disclosures requires us to make estimates and judgmentsthat affect the reported amounts of assets, liabilities, costs and expenses, andthe disclosure of contingent assets and liabilities in our financial statements.We base our estimates on historical experience, known trends and events andvarious other factors that we believe are reasonable under the circumstances,the results of which form the basis for making judgments about the carryingvalues of assets and liabilities that are not readily apparent from othersources. We evaluate estimates and assumptions on an ongoing basis. Actualresults may differ from these estimates under different assumptions orconditions.

Our significant accounting policies are described in more detail in our 2019Form 10-K, except as otherwise described below.

Results of Operations

Originally posted here:
ROCKET PHARMACEUTICALS : Management's Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) - marketscreener.com

KEY POINTS

Health experts have been baffled as to why there are people infected with COVID-19 and yet barely feel the infection while others suffer life-threatening symptoms even if healthy and young. Scientists are looking for answers in the genes of patients, trying to discover mutations that affect the immune response, hoping that it could help in coming up with new treatments.

Profile Of A Severe Case

During the early days of the pandemic, a general profile of a severe case of coronavirus infection started to emerge. They are older adults with pre-existing medical conditions and are likely to be male. As the virus continued to infect more people, a small fraction started to deviate from the general profile.

Health experts are starting to see around 5% of those infected are under the age of 50 and do not have any underlying health conditions. These are the group of patients that interest Dr. Jean-Laurent Casanova, a geneticist and head of the St. Giles Laboratory of Human Genetics of Infectious Diseases.

Dr. Casanova told the AFP it is possible for someone who joined a marathon in October 2019 to find himself in intensive care, ventilated and intubated in April 2020. He revealed his desire to know if these types of patients have rare genetic mutations that have been triggered by the coronavirus infection. The assumption is that these patients have genetic variations that are silent until the virus is encountered, the doctor said. coronavirus silent mutation in human body may be the one exploited by the virus Photo: TPHeinz - Pixabay

A Huge Global Effort

The geneticist co-founded the COVID Human Genetics Effort, a collaborative work that seeks to know more about the genome of severely-ill young patients in several countries worldwide. These include patients in Europe, Japan, Iran, China, and the United States.

Dr. Casanovas group is also studying those who did not get infected despite being exposed many times. He said their main goal is to know why some are sicker than others, a knowledge that the geneticist said might help them in their quest to develop anti-viral therapies.

Gene Mutations Have A Long History

Scientists have long known that gene mutations can make people more susceptible to an array of infectious diseases, ranging from influenza to viral encephalitis. These gene mutations can also offer protection sometimes.

In the 1990s, a group of researchers found out that some rare mutations of a single gene successfully protected people against HIV infection. This discovery led to a betterunderstanding of how the virus worked and eventually paved the way for scientists to develop new treatments.

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Its In The Genes? Scientists Think Coronavirus Exploits Silent Hidden Mutations In The Body - International Business Times

The approval was based on a biomarker subgroup analysis of 387 patients with HRD-positive tumors from the Phase 3 PAOLA-1 trial, which showed that LYNPARZA in combination with bevacizumab reduced the risk of disease progression or death by 67% (HR 0.33 [95% CI, 0.25-0.45]). It improved progression-free survival (PFS) to a median of 37.2 months vs. 17.7 months with bevacizumab alone in patients with HRD-positive advanced ovarian cancer.

The most common adverse reactions (ARs) 10% in the overall trial population for PAOLA-1 when treated with LYNPARZA in combination with bevacizumab (N=535) and at a 5% frequency compared to bevacizumab alone (N=267) were fatigue (53% vs. 32%), nausea (53% vs. 22%), anemia (41% vs. 10%), lymphopenia (24% vs. 9%), vomiting (22% vs. 11%) and leukopenia (18% vs. 10%). Grade 3 or above ARs were anemia (17% vs. <1%), lymphopenia (7% vs. 1%), fatigue (5% vs. 2%), nausea (2% vs. 1%), leukopenia (2% vs. 2%) and vomiting (2% vs. 2%). Additional adverse reactions that occurred in 10% of patients receiving LYNPARZA in combination with bevacizumab irrespective of the frequency compared to bevacizumab alone were diarrhea (18%), neutropenia (18%), urinary tract infection (15%) and headache (14%). Fatal adverse reactions occurred in one patient due to concurrent pneumonia and aplastic anemia. Serious adverse reactions occurred in 31% of patients who received LYNPARZA in combination with bevacizumab. Serious adverse reactions in >5% of patients included hypertension (19%) and anemia (17%).

In addition, venous thromboembolic events occurred more commonly in patients receiving LYNPARZA in combination with bevacizumab (5%) than in those receiving bevacizumab alone (1.9%). ARs led to dose interruption in 54% of patients on LYNPARZA in combination with bevacizumab, while 41% of patients on LYNPARZA in combination with bevacizumab had a dose reduction. Discontinuation of treatment due to ARs occurred in 20% of patients on LYNPARZA in combination with bevacizumab.

Approximately one in two women with advanced ovarian cancer has an HRD-positive tumor. For patients with advanced ovarian cancer, the primary aim of first-line maintenance treatment is to delay disease progression for as long as possible.

Isabelle Ray-Coquard, principal investigator of the PAOLA-1 trial and medical oncologist, Centre Lon Brard and President of the GINECO group, said, Ovarian cancer is a devastating disease. The magnitude of benefit in HRD-positive patients in the PAOLA-1 trial is impactful. I look forward to seeing this translate into clinical practice.

Dave Frederickson, executive vice president, head of the oncology business unit, AstraZeneca, said, This approval represents another milestone for LYNPARZA in patients with ovarian cancer. The median progression-free survival of more than three years offers new hope for women to delay relapse in this difficult-to-treat disease. These results further establish that HRD-positive is a distinct subset of ovarian cancer and HRD testing is now a critical component of diagnosis and tailoring of treatment for women with advanced ovarian cancer.

Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories, said, Advances in understanding the role of biomarkers and PARP inhibition have fundamentally changed how physicians treat this aggressive type of cancer. Todays approval based on the PAOLA-1 trial highlights the importance of HRD testing at diagnosis to identify those who may benefit from LYNPARZA in combination with bevacizumab as a first-line maintenance treatment.

The full results from the Phase 3 PAOLA-1 trial were published inThe New England Journal of Medicine.

Regulatory reviews are currently underway in the European Union, Japan and other countries for LYNPARZA in combination with bevacizumab as a first-line maintenance treatment for patients with advanced ovarian cancer. As part of a broad development program, LYNPARZA is being assessed as a monotherapy and in combination across multiple tumor types.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

There are no contraindications for LYNPARZA.

WARNINGS AND PRECAUTIONS

Myelodysplastic Syndrome/Acute Myeloid Leukemia (MDS/AML): Occurred in <1.5% of patients exposed to LYNPARZA monotherapy, and the majority of events had a fatal outcome. The duration of therapy in patients who developed secondary MDS/AML varied from <6 months to >2 years. All of these patients had previous chemotherapy with platinum agents and/or other DNA-damaging agents, including radiotherapy, and some also had a history of more than one primary malignancy or of bone marrow dysplasia.

Do not start LYNPARZA until patients have recovered from hematological toxicity caused by previous chemotherapy (Grade 1). Monitor complete blood count for cytopenia at baseline and monthly thereafter for clinically significant changes during treatment. For prolonged hematological toxicities, interrupt LYNPARZA and monitor blood count weekly until recovery.

If the levels have not recovered to Grade 1 or less after 4 weeks, refer the patient to a hematologist for further investigations, including bone marrow analysis and blood sample for cytogenetics. Discontinue LYNPARZA if MDS/AML is confirmed.

Pneumonitis: Occurred in <1% of patients exposed to LYNPARZA, and some cases were fatal. If patients present with new or worsening respiratory symptoms such as dyspnea, cough, and fever, or a radiological abnormality occurs, interrupt LYNPARZA treatment and initiate prompt investigation. Discontinue LYNPARZA if pneumonitis is confirmed and treat patient appropriately.

Embryo-Fetal Toxicity: Based on its mechanism of action and findings in animals, LYNPARZA can cause fetal harm. A pregnancy test is recommended for females of reproductive potential prior to initiating treatment.

Females

Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for 6 months following the last dose.

Males

Advise male patients with female partners of reproductive potential or who are pregnant to use effective contraception during treatment and for 3 months following the last dose of LYNPARZA and to not donate sperm during this time.

ADVERSE REACTIONSFirst-Line Maintenance BRCAm Advanced Ovarian Cancer

Most common adverse reactions (Grades 1-4) in 10% of patients in clinical trials of LYNPARZA in the first-line maintenance setting for SOLO-1 were: nausea (77%), fatigue (67%), abdominal pain (45%), vomiting (40%), anemia (38%), diarrhea (37%), constipation (28%), upper respiratory tract infection/influenza/nasopharyngitis/bronchitis (28%), dysgeusia (26%), decreased appetite (20%), dizziness (20%), neutropenia (17%), dyspepsia (17%), dyspnea (15%), leukopenia (13%), UTI (13%), thrombocytopenia (11%), and stomatitis (11%).

Most common laboratory abnormalities (Grades 1-4) in 25% of patients in clinical trials of LYNPARZA in the first-line maintenance setting for SOLO-1 were: decrease in hemoglobin (87%), increase in mean corpuscular volume (87%), decrease in leukocytes (70%), decrease in lymphocytes (67%), decrease in absolute neutrophil count (51%), decrease in platelets (35%), and increase in serum creatinine (34%).

ADVERSE REACTIONSFirst-Line Maintenance Advanced Ovarian Cancer in Combination with Bevacizumab

Most common adverse reactions (Grades 1-4) in 10% of patients treated with LYNPARZA/bevacizumab compared to a 5% frequency for placebo/bevacizumab in the first-line maintenance setting for PAOLA-1 were: nausea (53%), fatigue (including asthenia) (53%), anemia (41%), lymphopenia (24%), vomiting (22%) and leukopenia (18%). In addition, the most common adverse reactions (10%) for patients receiving LYNPARZA/bevacizumab irrespective of the frequency compared with the placebo/bevacizumab arm were: diarrhea (18%), neutropenia (18%), urinary tract infection (15%), and headache (14%).

In addition, venous thromboembolic events occurred more commonly in patients receiving LYNPARZA/bevacizumab (5%) than in those receiving placebo/bevacizumab (1.9%).

Most common laboratory abnormalities (Grades 1-4) in 25% of patients for LYNPARZA in combination with bevacizumab in the first-line maintenance setting for PAOLA-1 were: decrease in hemoglobin (79%), decrease in lymphocytes (63%), increase in serum creatinine (61%), decrease in leukocytes (59%), decrease in absolute neutrophil count (35%), and decrease in platelets (35%).

ADVERSE REACTIONSMaintenance Recurrent Ovarian Cancer

Most common adverse reactions (Grades 1-4) in 20% of patients in clinical trials of LYNPARZA in the maintenance setting for SOLO-2 were: nausea (76%), fatigue (including asthenia) (66%), anemia (44%), vomiting (37%), nasopharyngitis/upper respiratory tract infection (URI)/influenza (36%), diarrhea (33%), arthralgia/myalgia (30%), dysgeusia (27%), headache (26%), decreased appetite (22%), and stomatitis (20%).

Study 19: nausea (71%), fatigue (including asthenia) (63%), vomiting (35%), diarrhea (28%), anemia (23%), respiratory tract infection (22%), constipation (22%), headache (21%), decreased appetite (21%), and dyspepsia (20%).

Most common laboratory abnormalities (Grades 1-4) in 25% of patients in clinical trials of LYNPARZA in the maintenance setting (SOLO-2/Study 19) were: increase in mean corpuscular volume (89%/82%), decrease in hemoglobin (83%/82%), decrease in leukocytes (69%/58%), decrease in lymphocytes (67%/52%), decrease in absolute neutrophil count (51%/47%), increase in serum creatinine (44%/45%), and decrease in platelets (42%/36%).

ADVERSE REACTIONSAdvanced gBRCAm Ovarian Cancer

Most common adverse reactions (Grades 1-4) in 20% of patients in clinical trials of LYNPARZA for advanced gBRCAm ovarian cancer after 3 or more lines of chemotherapy (pooled from 6 studies) were: fatigue/asthenia (66%), nausea (64%), vomiting (43%), anemia (34%), diarrhea (31%), nasopharyngitis/upper respiratory tract infection (URI) (26%), dyspepsia (25%), myalgia (22%), decreased appetite (22%), and arthralgia/musculoskeletal pain (21%).

Most common laboratory abnormalities (Grades 1-4) in 25% of patients in clinical trials of LYNPARZA for advanced gBRCAm ovarian cancer (pooled from 6 studies) were: decrease in hemoglobin (90%), mean corpuscular volume elevation (57%), decrease in lymphocytes (56%), increase in serum creatinine (30%), decrease in platelets (30%), and decrease in absolute neutrophil count (25%).

ADVERSE REACTIONSgBRCAm, HER2-Negative Metastatic Breast Cancer

Most common adverse reactions (Grades 1-4) in 20% of patients in OlympiAD were: nausea (58%), anemia (40%), fatigue (including asthenia) (37%), vomiting (30%), neutropenia (27%), respiratory tract infection (27%), leukopenia (25%), diarrhea (21%), and headache (20%).

Most common laboratory abnormalities (Grades 1-4) in 25% of patients in OlympiAD were: decrease in hemoglobin (82%), decrease in lymphocytes (73%), decrease in leukocytes (71%), increase in mean corpuscular volume (71%), decrease in absolute neutrophil count (46%), and decrease in platelets (33%).

ADVERSE REACTIONSFirst-Line Maintenance gBRCAm Metastatic Pancreatic Adenocarcinoma

Most common adverse reactions (Grades 1-4) in 10% of patients in clinical trials of LYNPARZA in the first-line maintenance setting for POLO were: fatigue (60%), nausea (45%), abdominal pain (34%), diarrhea (29%), anemia (27%), decreased appetite (25%), constipation (23%), vomiting (20%), back pain (19%), arthralgia (15%), rash (15%), thrombocytopenia (14%), dyspnea (13%), neutropenia (12%), nasopharyngitis (12%), dysgeusia (11%), and stomatitis (10%).

Most common laboratory abnormalities (Grades 1-4) in 25% of patients in clinical trials of LYNPARZA in the first-line maintenance setting for POLO were: increase in serum creatinine (99%), decrease in hemoglobin (86%), increase in mean corpuscular volume (71%), decrease in lymphocytes (61%), decrease in platelets (56%), decrease in leukocytes (50%), and decrease in absolute neutrophil count (25%).

DRUG INTERACTIONS

Anticancer Agents: Clinical studies of LYNPARZA in combination with other myelosuppressive anticancer agents, including DNA-damaging agents, indicate a potentiation and prolongation of myelosuppressive toxicity.

CYP3A Inhibitors: Avoid concomitant use of strong or moderate CYP3A inhibitors. If a strong or moderate CYP3A inhibitor must be co-administered, reduce the dose of LYNPARZA. Advise patients to avoid grapefruit, grapefruit juice, Seville oranges, and Seville orange juice during LYNPARZA treatment.

CYP3A Inducers: Avoid concomitant use of strong or moderate CYP3A inducers when using LYNPARZA. If a moderate inducer cannot be avoided, there is a potential for decreased efficacy of LYNPARZA.

USE IN SPECIFIC POPULATIONS

Lactation: No data are available regarding the presence of olaparib in human milk, its effects on the breastfed infant or on milk production. Because of the potential for serious adverse reactions in the breastfed infant, advise a lactating woman not to breastfeed during treatment with LYNPARZA and for 1 month after receiving the final dose.

Pediatric Use: The safety and efficacy of LYNPARZA have not been established in pediatric patients.

Hepatic Impairment: No adjustment to the starting dose is required in patients with mild or moderate hepatic impairment (Child-Pugh classification A and B). There are no data in patients with severe hepatic impairment (Child-Pugh classification C).

Renal Impairment: No dosage modification is recommended in patients with mild renal impairment (CLcr 51-80 mL/min estimated by Cockcroft-Gault). In patients with moderate renal impairment (CLcr 31-50 mL/min), reduce the dose of LYNPARZA to 200 mg twice daily. There are no data in patients with severe renal impairment or end-stage renal disease (CLcr 30 mL/min).

INDICATIONS

LYNPARZA is a poly (ADP-ribose) polymerase (PARP) inhibitor indicated:

First-Line Maintenance BRCAm Advanced Ovarian Cancer

For the maintenance treatment of adult patients with deleterious or suspected deleterious germline or somatic BRCA-mutated (gBRCAm or sBRCAm) advanced epithelial ovarian, fallopian tube or primary peritoneal cancer who are in complete or partial response to first-line platinum-based chemotherapy. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

First-Line Maintenance HRD Positive Advanced Ovarian Cancer in Combination with Bevacizumab

In combination with bevacizumab for the maintenance treatment of adult patients with advanced epithelial ovarian, fallopian tube or primary peritoneal cancer who are in complete or partial response to first-line platinum-based chemotherapy and whose cancer is associated with homologous recombination deficiency (HRD) positive status defined by either:

Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

Maintenance Recurrent Ovarian Cancer

For the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer, who are in complete or partial response to platinum-based chemotherapy.

Advanced gBRCAm Ovarian Cancer

For the treatment of adult patients with deleterious or suspected deleterious germline BRCA-mutated (gBRCAm) advanced ovarian cancer who have been treated with 3 or more prior lines of chemotherapy. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

gBRCAm HER2-Negative Metastatic Breast Cancer

For the treatment of adult patients with deleterious or suspected deleterious gBRCAm, human epidermal growth factor receptor 2 (HER2)-negative metastatic breast cancer who have been treated with chemotherapy in the neoadjuvant, adjuvant, or metastatic setting. Patients with hormone receptor (HR)-positive breast cancer should have been treated with a prior endocrine therapy or be considered inappropriate for endocrine therapy. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

First-Line Maintenance gBRCAm Metastatic Pancreatic Cancer

For the maintenance treatment of adult patients with deleterious or suspected deleterious gBRCAm metastatic pancreatic adenocarcinoma whose disease has not progressed on at least 16 weeks of a first-line platinum-based chemotherapy regimen. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

Please click here for complete Prescribing Information, including Patient Information (Medication Guide).

About PAOLA-1

PAOLA-1 is a double-blind Phase 3 trial evaluating the efficacy and safety of LYNPARZA in combination with standard-of-care bevacizumab vs. bevacizumab alone, as a first-line maintenance treatment for advanced FIGO Stage III-IV high grade serous or endometroid ovarian, fallopian tube, or peritoneal cancer patients who had a complete or partial response to first-line treatment with platinum-based chemotherapy and bevacizumab.

PAOLA-1 is an ENGOT (European Network of Gynaecological Oncological Trial groups) trial, sponsored by ARCAGY Research (Association de Recherche sur les CAncers dont GYncologiques) on behalf of GINECO (Groupe dInvestigateurs National des Etudes des Cancers Ovariens et du sein). ARCAGY-GINECO is an academic group specializing in clinical and translational research in patients cancers and a member of the GCIG (Gynecologic Cancer InterGroup).

In the U.S., eligible advanced ovarian cancer patients will be selected for therapy based on the FDA-approved myChoice HRD Plus, an HRD test designed to detect when a tumor has lost the ability to repair double-stranded DNA breaks. Myriad Genetics, Inc. owns and commercializes myChoice HRD Plus.

About LYNPARZA (olaparib)

LYNPARZA is a first-in-class PARP inhibitor and the first targeted treatment to potentially exploit DNA damage response (DDR) pathway deficiencies, such as BRCA mutations, to preferentially kill cancer cells. Inhibition of PARP with LYNPARZA leads to the trapping of PARP bound to DNA single-strand breaks, stalling of replication forks, their collapse and the generation of DNA double-strand breaks and cancer cell death. LYNPARZA is being tested in a range of tumor types with defects and dependencies in the DDR.

LYNPARZA, which is being jointly developed and commercialized by AstraZeneca and Merck, has a broad and advanced clinical trial development program, and AstraZeneca and Merck are working together to understand how it may affect multiple PARP-dependent tumors as a monotherapy and in combination across multiple cancer types.

About Ovarian Cancer

Ovarian cancer is the fifth most common cause of death from cancer in women in the United States. This year, it is estimated that more than 21,000 women will be diagnosed with ovarian cancer and nearly 14,000 women will die of this disease.

Women with ovarian cancer are often diagnosed with advanced disease, which has a five-year survival rate of about 48%. For newly diagnosed advanced ovarian cancer, the primary aim of treatment is to delay progression of the disease for as long as possible. BRCA1/2 mutations are found in approximately 22% of all ovarian cancers and approximately 50% of ovarian cancers are HRD-positive.

About Homologous Recombination Deficiency

HRD encompass a wide range of genetic abnormalities, including BRCA mutations, that can be detected using tests. As the BRCA gene drives DNA repair via homologous recombination, mutation of this gene leads to homologous recombination deficiency thereby interfering with normal cell DNA repair mechanisms. BRCA mutations are just one of many HRDs which confer sensitivity to PARP inhibitors including LYNPARZA.

About the AstraZeneca and Merck Strategic Oncology Collaboration

In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the United States and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialize certain oncology products, including LYNPARZA, the worlds first PARP inhibitor, for multiple cancer types. Working together, the companies will develop these products in combination with other potential new medicines and as monotherapies. Independently, the companies will develop these oncology products in combination with their respective PD-L1 and PD-1 medicines.

Mercks Focus on Cancer

Our goal is to translate breakthrough science into innovative oncology medicines to help people with cancer worldwide. At Merck, the potential to bring new hope to people with cancer drives our purpose and supporting accessibility to our cancer medicines is our commitment. As part of our focus on cancer, Merck is committed to exploring the potential of immuno-oncology with one of the largest development programs in the industry across more than 30 tumor types. We also continue to strengthen our portfolio through strategic acquisitions and are prioritizing the development of several promising oncology candidates with the potential to improve the treatment of advanced cancers. For more information about our oncology clinical trials, visit http://www.merck.com/clinicaltrials.

About Merck

For more than 125 years, Merck, known as MSD outside of the United States and Canada, has been inventing for life, bringing forward medicines and vaccines for many of the worlds most challenging diseases in pursuit of our mission to save and improve lives. We demonstrate our commitment to patients and population health by increasing access to health care through far-reaching policies, programs and partnerships. Today, Merck continues to be at the forefront of research to prevent and treat diseases that threaten people and animals including cancer, infectious diseases such as HIV and Ebola, and emerging animal diseases as we aspire to be the premier research-intensive biopharmaceutical company in the world. For more information, visit http://www.merck.com and connect with us on Twitter, Facebook, Instagram, YouTube and LinkedIn.

Forward-Looking Statement of Merck & Co., Inc., Kenilworth, N.J., USA

This news release of Merck & Co., Inc., Kenilworth, N.J., USA (the company) includes forward-looking statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. These statements are based upon the current beliefs and expectations of the companys management and are subject to significant risks and uncertainties. There can be no guarantees with respect to pipeline products that the products will receive the necessary regulatory approvals or that they will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements.

Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of the recent global outbreak of novel coronavirus disease (COVID-19); the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the companys ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the companys patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions.

The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the companys 2019 Annual Report on Form 10-K and the companys other filings with the Securities and Exchange Commission (SEC) available at the SECs Internet site (www.sec.gov).

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LYNPARZA (olaparib) Approved by FDA as First-Line Maintenance Treatment with Bevacizumab for HRD-Positive Advanced Ovarian Cancer - BioSpace

LONDON When the first cases of a new coronavirus started to appear in China last December, the disease seemed to be a particularly aggressive respiratory infection. An "urgent notice" that month from the Wuhan health commission warned of "successive cases of unknown pneumonia".

Respiratory symptoms are still the first signs that doctors look for in suspected Covid-19 cases: cough, shortness of breath and fever.

But, less than five months after it was first identified, this new coronavirus is managing to throw up a series of medical mysteries - from blood clots and strokes to digestive problems - that are confounding the scientific community.

From head to foot, Covid-19 causes a fiendish variety of symptoms. Some are relatively mild, such as loss of smell and taste or chilblain-like sores on toes. But others may be fatal, such as when what doctors call an immune storm destroys vital organs. The more this virus is studied, the more complex it appears to be. "Every day we're learning of new tricks that the virus plays," Imperial College London's professor of experimental medicine Peter Openshaw says. "It is remarkable to see a disease unfolding in front of our eyes with so many twists and turns."

The proliferation of complex symptoms is not just a challenge for doctors treating the disease, but also for health systems trying to adapt to the pandemic. In the early months, the focus was on getting hold of ventilators that could help patients with severe respiratory problems. But now hospitals are also scrambling for more kidney dialysis machines and anticoagulant drugs.

A single individual can suffer the disease in more than one form, Prof Openshaw adds. "There are accounts of people experiencing one symptom, for example coughing, appearing to recover or go into remission and then returning with a more serious systemic disease."

With the worldwide death toll from Covid-19 already nearing 260,000 and confirmed cases close to exceeding 3.7 million, according to Johns Hopkins University, scientists have mobilised at a speed and on a scale unprecedented in the history of medicine, in an effort to understand the myriad ways in which the virus affects the human body. They hope that their research will not only improve clinical care of patients but also help the development of drugs and vaccines.

The initial diagnosis was that it was a respiratory infection, like its sister diseases Sars and Mers which are also caused by coronaviruses.

Respiratory symptoms remain the most common manifestations of Covid-19 in patients who go to hospital, according to a study of almost 17,000 people admitted to 166 UK hospitals carried out by a research consortium from Imperial College and Liverpool and Edinburgh universities. About two-thirds of patients in the study - the largest of Covid-19 hospital patients outside China - were admitted suffering from respiratory symptoms, says Dr Annemarie Docherty of Edinburgh, the lead author of the paper. But that proportion may have been raised by the fact that they reflect the official case definition of Covid-19.

But two other clusters of symptoms also dominate hospital admissions: systemic musculoskeletal symptoms (muscle and joint pain and fatigue) and enteric symptoms (abdominal pain, vomiting and diarrhoea). Many patients suffer from several symptoms simultaneously.

How the immune system reacts to Covid-19 is key to the course of the disease in adults. People who have been suffering with mild to moderate symptoms for a week or so often seem to hit a critical point: usually their immune system gets the virus under full control and sets them on a path to full recovery - but sometimes it goes into overdrive, triggering systemic inflammation and in severe cases a "cytokine storm" that destroys tissues and whole organs.

Inflammation also helps to explain why obesity makes people more susceptible to severe Covid-19. Seventy-three per cent of coronavirus patients in UK intensive care units are overweight or obese, with a body mass index above 25. "Fat cells secrete chemicals that increase the body's inflammatory response," says Liverpool University's professor of child health Calum Semple.

Kidney damage has emerged as another of the most frequent serious consequences of Covid-19, with 23 per cent of patients in intensive care requiring renal support. As with other organs, it is uncertain to what extent the virus is directly attacking the kidneys or whether the harm results more from generalised overactivity of the immune system and consequent changes in the patient's blood circulation.

Cardiovascular disease is the most common pre-existing health condition in people who die of Covid-19, ahead of lung and respiratory disorders such as asthma and chronic obstructive pulmonary disease. And many patients without a previous history of heart trouble develop severe cardiac symptoms while they are in hospital.

"When we first heard about the coronavirus we expected people with lung and breathing problems to be most at risk but that has not been the case," says the British Heart Foundation's medical director Nilesh Samani. "We need to understand why the virus is causing so many problems outside the lungs - and cardiovascular complications in particular."

The exaggerated immune response to the virus sometimes causes abnormal blood clotting. If this thrombosis happens in the brain, it may trigger a stroke. Neurologists at University College London (UCL) studied six Covid-19 patients who suffered acute stroke as a result of a large arterial blockage - in five of the cases more than a week after suffering headache, cough and fever and in one patient before other symptoms appeared.

The UCL researchers found all six patients had markedly raised blood levels of a protein fragment called D-dimer associated with abnormal clotting. The findings suggest that early testing for D-dimer could enable doctors to prescribe blood-thinning drugs to people at risk, reducing the chance of stroke or harmful clotting elsewhere in the body. "Early use of anticoagulant drugs might be helpful but this needs to be balanced against their brain bleeding risk," says study leader David Werring.

STRAITS TIMES GRAPHICS

"This study is consistent with the growing evidence that people hospitalised with Covid-19 are at risk from blood clots in multiple locations: the lungs (causing pulmonary embolus), the brain (causing stroke) and the veins (causing DVT)," says professor of cardiovascular medicine Tim Chico at Sheffield University. "The risk of blood clots with Covid-19 appears to be even greater than the increased risk of blood clots seen in other severe illnesses."

The coronavirus also seems capable of attacking the brain and nervous system directly, as well as indirectly through abnormal blood clotting, though the evidence for acute symptoms of neural infection is limited. The effects may show up in the longer term as post-viral fatigue.

Neurons in the olfactory bulb, which transmits information from the nose to the brain, are apparently infected by the virus. Indeed, anosmia - loss of the sense of smell - is one of the most frequently reported symptoms of mild infection, affecting about half of patients and lasting for several weeks in some cases.

The good news for those who develop anosmia is that they are much less likely to become seriously ill with Covid-19. Dr Carol Yan and colleagues at the University of California San Diego (UCSD) reported last week that patients reporting loss of smell were 10 times less likely to be admitted to hospital for Covid-19 than those without loss of smell.

The UCSD researchers suggest that a relatively small dose of virus delivered to the upper airway, where it causes anosmia, may be less likely to overwhelm the host immune response. "This hypothesis is in essence the concept underlying live vaccinations, where low dosage and a distant site of inoculation generates an immune response without provoking a severe infection," they say.

The declining strength of the immune system with age is a partial explanation for the increasing incidence of Covid-19 in older people. PHOTO: AFP

Besides anosmia, the most frequently seen minor symptoms are rashes, pustules and blisters on the skin - including lesions like chilblains that dermatologists are calling "Covid toe".

The results from the study led by Imperial College, Liverpool and Edinburgh universities echo other findings that the disease is much more common in men - who make up 60 per cent of UK Covid-19 hospital admissions - and its severity rises markedly with advancing years (the median age of patients is 72). The strong associations with the male sex and old age are a particular feature of Covid-19 compared with other infectious illnesses.

Data from the UK Intensive Care National Audit & Research Centre shows that men make up 71.5 per cent of patients whose disease becomes severe enough to require intensive care treatment. A comparable control group of patients critically ill with non-Covid viral pneumonia was just 54.3 per cent male.

"The reason behind this difference in Covid risk is unknown," says Dr James Gill, honorary clinical lecturer at Warwick Medical School. "There are several schools of thought on the matter, from the assumption that simply men don't look after their bodies as well, with higher levels of smoking, alcohol use, obesity and other deleterious health behaviours, through to immunological variations in genders. Women may have a more aggressive immune system, meaning a greater resilience to infections."

University of Oxford's professor of immunology Philip Goulder points out that several critical immune genes are located on the X chromosome - of which women have two copies and men one. "The immune response to coronavirus is therefore amplified in females," he says.

The declining strength of the immune system with age is also a partial explanation for the increasing incidence of the disease in older people, though it is not clear why this trend is more pronounced in Covid-19 than in many other viral infections.

Children are remarkably - but not completely - resistant to the disease. Just 3 per cent of UK hospital patients are under 18. Again no one knows quite why. But one answer may lie in the "keyhole" through which coronavirus enters human cells, known as the ACE2 receptor. In children these receptors have not developed to their full adult stage and therefore may not fit the "spike protein" that the virus uses to enter cells.

It is also possible that ACE2 develops more quickly in children's upper airways than their lower respiratory tract, allowing them to become infected - and thus able to transmit Covid-19 - without showing the same progression to severe symptoms.

The National Health System in London and the UK Paediatric Intensive Care Society recently alerted doctors to a rise in the number of children suffering from "a multi-system inflammatory state" similar to toxic shock, which might result from the immune system overreacting to viral infection. Italian and US paediatricians have noticed a similar body-wide inflammatory syndrome in children.

This paediatric condition is rare but researchers are investigating, says Prof Semple. "Some respiratory viruses are associated with a systemic inflammatory response, typically two weeks after infection. But this could be a phenomenon of heightened awareness."

Research also shows that children are remarkably - but not completely - resistant to the disease. PHOTO: AFP

For Prof Openshaw, the mysteries of Covid-19 recall the early days of the HIV/Aids outbreak in the 1980s - except that this time, they are unfolding much more quickly. "We need the answers also to appear far faster than they did with HIV," he says.

A global research effort is on to discover human genetic factors that would help to explain why Covid-19 infection varies so much in its symptoms.

Although much of the variation results from environmental and lifestyle factors, scientists are convinced that genetics play a significant role too.

"Experience with other viruses shows that genetics can explain some of the different responses to infection," says Dr Mark Daly, director of the Institute for Molecular Medicine Finland in Helsinki, who is coordinating the global response through the Covid-19 Host Genetics Initiative.

For example, genetic mutations on the CCR5 protein, which HIV uses to enter human cells, make rare individuals resistant to Aids. Researchers may find comparable variations in the human ACE2 protein, entry point of the coronavirus, designated as Sars-Cov-2, that causes Covid-19.

The Covid programme has two overlapping components. One uses human genomes already obtained for other research purposes from volunteers through bodies such as UK Biobank and Genomics England - and looks for differences in DNA between participants who become ill with Covid-19 and those who do not.

The other part obtains the fresh genomes from Covid-19 patients, looking for variations that might explain why some experience only mild symptoms while others become severely ill.

Genomics England, a public body owned by the UK Department of Health and Social Care, is involved in both approaches. Dr Mark Caulfield, its chief scientist, says it is too early to have obtained any results. "But I am confident that reading whole genomes will help to identify variation that affects response to Covid-19 and to discover new therapies."

Prof Daly hopes the initiative will have tens of thousands of human genomes to analyse. "We particularly want to identify a subset of younger individuals with no comorbidities who have a severe response to Sars-Cov-2 infection," he says.

FINANCIAL TIMES

See the original post:
From blood clots to 'Covid toe': Experts confounded by series of medical mysteries - The Straits Times

If youre looking for a natural way to increase your energy and feel younger, you may be curious about Human Growth Hormone. While its sometimes painted in a negative light, due to its abuse by athletes and bodybuilders, when used properly, growth hormone replacement therapy is a safe and scientific way to regain energy and improve your overall quality of life.

What is Human Growth Hormone?

Human Growth Hormone (or HGH) is a protein that is secreted in spurts by thepituitary gland. HGH is secreted after exercise, trauma and right before falling asleep. Under normal conditions, levels alternate throughout the day, with the highest amount appearing at night. Human Growth Hormone secretion rises during childhood, peaks at puberty and starts to decline in middle age.

What Does Human Growth Hormone Do?

HGH is responsible for promoting vertical growth in kids and teens, as well as a few key metabolic responsibilities throughout adulthood.

Functions include: stimulating the growth of bone and cartilage raising the production of protein in the body utilizing fat and regulating blood sugar

Our bodies natural production of this vital hormone starts to declines in our thirties.The results can lead to:

But isnt this just a normal part of aging? Dont we simply have to embrace that, as we get older, we are going to put on a few pounds and need to double our espresso order? Not necessarily.

Replacement Therapy is a safe, medically supervised treatment for growth hormone deficiency in adults. Studies have proven that the replacement of growth hormone can lead to: increased lean muscle mass decreased fat mass

Where Can I Access Hormone Replacement Therapy?

The following clinics in Florida are certified in the administration of hormone replacement therapy:

With locations in Jacksonville, Miami, Orlando, Tampa and West Palm Beach, Ehormones MD offers a convenient way to explore hormone replacement therapy. They have a team of medical doctors who will design a unique protocol for you based on your lab work. Medications and supplies are shipped directly to your home. You can administer the hormones by yourself via a small needle into your lower belly every night, before bed. Follow-up lab work is scheduled five to eight weeks following the start of treatment, with additional labs every six months thereafter. Additional services include fitness programs, nutrition plans, stress reduction plans and ongoing evaluations.

Health Gains clinics are located in Aventura and Madeira Beach, Florida. The clinics offer white-glove concierge service with no waiting. Their highly trained medical team includes doctors, nurse practitioners and bi-lingual medical assistants. The latest technology is combined with a spa-like environment to help patients overcome the symptoms of aging and improve their quality of life. Services also include Peptide Therapy, Thyroid Hormone Therapy and Testosterone Therapy for men.

Located in Miami,HGH Therapy Clinicwas founded in 2009 as a free information center for endocrine problems. Since then, it has evolved into state-of-the-art facility featuring the best and newest technologies of hormone treatment. Their team of doctors includes specialists in endocrinology, diabetes, metabolism and internal medicine. They specialize in Growth Hormone Deficiency Treatment for both men and women.

The goal of the Rejuvenation Clinic is to promote vitality through the early detection of hormonal imbalances. Their patient advocate support team will empower you to understand your treatment from start to finish. The process starts by filling out a confidential medical history form online. Within minutes, you will be assigned to a wellness advisor who will help you to set up a physical exam and a lab test. Additional treatments include Testosterone Therapy, Intracavernous Injection Therapy (ICI), supplements and amino acids.

AtTampa Rejuvenation, a team of doctors and nurse practitioners are there to help you achieve optimal health through weight loss and bioidentical hormone replacement therapy. With three convenient locations in Tampa and one in Brandon, FL, accessing the treatment you need is easy. Services include hormone therapy for both men and women, as well as treatments for adrenal fatigue, thyroid issues and hair restoration.

Go here to read the rest:
HGH Replacement Clinics of Florida: Choose Only the Best - South Florida Reporter

According to The International Dermal Institute, 55% of women over 25 have adult acne. And chin acne lets call it ch-acne is the second most common kind of adult acne (67%) falling shortly behind cheek acne (81%). But why do spots congregate in this relatively small area of the face? Weve spoken to Dr Nick Lowe, Consultant Dermatologist at The Cranley Clinic and co-author of Perfectly Clear, to find out why the chin has become a sebum battleground.

Acne tends to occur more in areas with a lot of oil-producing glands, AKA sebaceous glands, explains Lowe. Pores can become blocked by excess oil from the glands, and acne bacteria builds up below the skin, producing inflamed spots. One of the highest concentrations of these glands is, you guessed it, around your chin area.

But theres more to it. For starters, particular irritants specific to the chin can cause breakouts that look just like, but actually arent, acne (more on this later). Also, as much as we all hate to admit this, it looks like those old wives tales were right factors like touching your face, as well as eating sugary food, play a big part too.

Read on to discover the main causes of chin acne, or ch-acne, and how to tackle it.

Acne is caused when a pesky group of hormones caused androgens are released often as a response to your contraception. This type of acne is characterised by smaller inflamed spots around the mouth and chin although you can also get larger, cystic acne spots on the chin too.

Sound familiar? 'It tends to be contraceptives with high progesterone compared to oestrogen that aggravates acne,' says Nick. So if youre on something like the progesterone mini-pill or the hormonal implant (which releases progestogen, a hormone similar to progesterone) and youre getting acne flare-ups around your chin and mouth then, well, it adds up. Instead, you could switch to a low progesterone contraceptive pill that actually helps your skin, like Yasmin.

Your chin also acts as a landing pad for your toothpaste, which, it turns out, is chock-a-block with irritants especially one ubiquitous ingredient: fluoride. This condition, where acne-like symptoms occur around the mouth and chin area, looks like acne, but its actually peri-oral dermatitis, explains Nick.

A secondary culprit is sodium lauryl sulphate (SLS), which is present in toothpaste brands as well as many other cosmetics such as shampoo. However, this will only irritate skin, rather than single-handedly cause something which resembles acne, explains Nick.

While we're not suggesting for a minute you ditch toothpaste in the name of good skin, there are some great irritant-free toothpastes that do the job just as well as your go-to brand. Try JASONs brilliant range of SLS-free, fluoride-free toothpastes.

Sadly, when tackling acne, your diet could be the cause. There are two main food culprits to avoid if you get chin acne, according to Nick. First off, its high GI foods like chocolate, white bread and sugary breakfast cereals (check the sugar content of your granola). The body produces insulin-like hormones to deal with the high amount of carbohydrate youre absorbing in the bowel, and for some those hormones cause acne.

Milk can also be a trigger. In people susceptible to acne, drinking milk including skimmed can make the condition worse, because it contains androgenic hormones. Nick recommends swapping to soy or almond milk over a six to eight-week trial. It takes that long to see an improvement. The good news is, yoghurt doesnt seem to have the same effect, thanks to the fermenting process removing some of the hormones; so your mid-morning Fage habit is safe.

Yes, you're mobile phone can cause spots...and anything else coming into contact with your chinny chin chin, basically. If you rest your chin on your hands or phone, the friction can block the pores, causing acne bacteria to build up, says Nick. Same story if you wear a cycling helmet with a chin strap. If you have existing acne, friction aggravates the inflamed spots so snogging your stubbly boyfriend wont help either.

As for me, my acne struggle had a happy ending. A course of antibiotics (to kill the acne bacteria) and a new contraception pill (Yasmin, my old friend) got rid of most of it. Then, just three months ago, the last nail in the coffin was changing my toothpaste who knew fluoride was the cause of my remaining acne-that-isnt-actually-acne?

For those still battling acne, on the chin or otherwise and believe me, I know how crap it is Nicks advice is to stick with finding a solution: If you have acne that is continuing, look at all your habits: your toothpaste, your diet and your contraception. Start off with topical medicine, then look into these other factors and find what works for you.

Shop the best products for acne-prone skin:

Apply a thin layer of this serum meets moisturiser all over the affected area and let the five acids (including Salicylic and Glycolic acid) get to work to minimise the appearance of spots.

Keep this handy stick in your handbag and apply it at the first sign of a spot - so, whenever that sore, swollen bump appears. Formulated with 2% salicylic acid to exfoliate the skin and tea-tree oil to calm the area - you'll notice the benefits in no time.

It might sound counter intuitive to add more oil to oily skin but it can in fact help to balance the skin's mantle. This blemish oil calms irritated skin, helps to heal spots and will also treat scars.

La Roche-Posay's anti-blemish cream is formulated to target oily, blemish-prone skin. It has become a cult product for beauty buffs who have raved about its magical ability to unclog pores, minimise imperfections and give the surface of the skin a smooth velvety finish.

The Body Shop's Tea Tree Oil range is proven to contain healing and antibacterial properties for every type of skin. The Body Shop claim that the range can create clearer skin in 3-days without over-stripping the skin. We love the Tea Tree Squeaky Clean Scrub (with biodegradable micro-beads) and Tea Tree Night Lotion to combat shine and impurities day and night.

This highly coveted skin elixir is not only surprisingly affordable but also promises to refresh and repair your complexion by clearing up and soothing whiteheads overnight. All Mario Badescu asks is that you dip a cotton swab into the pink sediment, dab on a pimple and wash off in the morning. Do not shake the bottle.

Enriched with cocoa butter, rosemary, eucalyptus and chamomile this indulgent cream cleanses, soothes and moisturises. Used alongside Liz Earle's pure cotton cloth, dead skin cells and daily grime are lifted away to reveal brighter looking skin.

Aesop is fast becoming the favorite beauty brand of the year because of the visible results. The parsley seed extract cleanses deep within the pores - exactly what you need for troublesome skin. Frequent use of this product will prevent future breakouts, and using this overnight won't leave you feeling oily or clogged up the next morning.

Dermalogica's Clay Cleanser treats spots when used as a wash and as a face mask. It concentrates on the affected area and uses refreshing cucumber to leave you feeling rejuvenated.

The most common places for acne and spots, particularly in the winter months, are your cheeks and jawline. To prevent this your skin must be well moisturized, and this Origins product is enriched with boosting ingredients to brighten your complexion. And it's completely oil-free.

Apply this serum onto your acne scars or discolouration after you've washed your face and before you moisturise, and overtime it will even your complexion.

READ MORE: Breakout Breakdown: What Is Actually Causing Your Spots?

READ MORE: Everything You Need To Know About Pimple Patches Do They Really Keep Spots At Bay?

View post:
What Causes Chin Acne And How Should You Treat It? - Grazia

MILPITAS May 9, 2020 (Thomson StreetEvents) -- Edited Transcript of Protagonist Therapeutics Inc earnings conference call or presentation Thursday, May 7, 2020 at 9:00:00pm GMT

* Dinesh V. Patel

Protagonist Therapeutics, Inc. - CEO, President, Interim PAO, Secretary & Director

* Donald A. Kalkofen

Protagonist Therapeutics, Inc. - CFO

* Samuel R. Saks

Protagonist Therapeutics, Inc. - Chief Medical Officer

* Christopher N. Marai

Nomura Securities Co. Ltd., Research Division - MD & Senior Analyst of Biotechnology

SVB Leerink LLC, Research Division - MD of Rare Diseases & Senior Research Analyst

Good day, and welcome to the Protagonist Therapeutics PTG-300 Development Update Call. Please note that today's conference is being recorded. At this time, I would like to introduce Don Kalkofen, Protagonist's Chief Financial Officer. Please go ahead.

Donald A. Kalkofen, Protagonist Therapeutics, Inc. - CFO [2]

Thank you, operator. Good afternoon, everyone. Please note that a replay of today's call will be available at the Investors section of our website at protagonist-inc.com.

Before we begin, I'd like to remind you that today's discussion will include statements about the company's future expectations, plans and prospects that constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in the Risk Factors section of our quarterly and annual reports on forms 10-Q and 10-K, which are on file with the SEC. While we may elect to update these forward-looking statements at some point in the future, we specifically disclaim any obligation to do so even if our view should change.

With that, I will now turn the call over to Dinesh Patel, President and CEO, to provide you an update on the company's progress to date.

Dinesh V. Patel, Protagonist Therapeutics, Inc. - CEO, President, Interim PAO, Secretary & Director [3]

Thanks, Don. Good afternoon, everyone, and thank you all for joining us today. On today's call, we'll also hear from Samuel Saks, our Chief Medical Officer; and Dr. Ronald Hoffman, Professor of Medicine in Hematology and Medical Oncology at the Mount Sinai Hospital in New York City. Dr. Hoffman has been involved in the Phase II clinical study of PTG-300 in polycythemia vera, and he will provide a brief overview of the disease and his perspectives on PTG-300 and its potential benefits as the novel treatment for polycythemia vera or PV.

Also present for the call today are David Liu, our Chief Scientific Officer and Head of R&D; and Suneel Gupta, our Chief Development Officer. We are pleased to have all these individuals available to address questions during the Q&A session of today's call.

So let's start with Slide #3. As you are well aware, Protagonist started the year 2020 with 3 clinical assets, all of which have been discovered through the use of our peptide technology platform in 6 different clinical proof-of-concept studies. The 3 assets fall in 2 broad categories: PTG-300 for various blood disorders, most of which are rare disease; and oral GI-restricted targeted peptide PTG-200 and PN-943 for inflammatory bowel disease, or IBD. All of these assets have a multibillion-dollar potential in multiple indications.

PTG-300 peptide mimetic of the natural hormone hepcidin that serves as a master regulator of iron homeostasis storage and distribution in the body was being pursued in Phase II open-label proof-of-concept studies in beta-thalassemia, or beta-thal; polycythemia vera, or PV; hereditary hemochromatosis, or HH; and an investigator-sponsored study in myelodysplastic syndrome, or MDS. As you may recall, a major objective for 2020 was to pick our first clinical indication for PTG-300 that we can progress towards a pivotal study in 2021.

Today, we are pleased to announce initial but yet very robust clinical data from our polycythemia vera program. And the selection and prioritization of polycythemia vera as the first indication for a pivotal study with 300. This decision is based on 3 criteria: strength and consistency of the clinical data, favorable regulatory path forward and the commercial opportunity. This is our major announcement today. And while most of our discussion will be around 300 and polycythemia vera, let me also take this opportunity to talk briefly about our oral gut-restricted IBD assets, largely in the context of corporate update and cash runway.

The enrollment rate of Phase II studies of these agents, namely the oral alpha-4-beta-7 integrin antagonist PN-943 for ulcerative colitis and the oral interleukin-23 receptor antagonist PTG-200 for Crohn's disease are understandably going to be influenced by the current COVID-19 situation. Therefore, while these studies are continuing, we believe it is appropriate to remove future guidance on the timelines for top line data from these studies.

In addition to the decision to focus our efforts towards rapidly advancing 300 in polycythemia vera indication, we are continuing the ongoing proof-of-concept study of 300 in hereditary hemochromatosis as a potential second indication and are also deciding to now discontinue further development of 300 for beta-thalassemia as well as for myelodysplastic syndrome.

On the financial side, we ended the first quarter of this year with about $117 million in cash and investments. By factoring in the delay in enrollment and the associated clinical development costs, coupled with a reduction in operational needs and expenditures, we are now extending our cash runway by an additional 6 months through the middle of 2022.

Now let's go to Slide #4, and let me turn to PTG-300, a major highlight of today's call and to provide rationale for why we are pursuing polycythemia vera as our first indication. First and foremost, it's the robust and consistent clinical responses we have seen in our ongoing Phase II PV trial. Although small, the data set is very compelling and consistent. On 6 out of 6, dose compliant patients treated for up to 28 weeks with 300 are phlebotomy free as of today. These are very noteworthy results. Keeping in mind that prior to entering our study, these subjects were receiving frequent phlebotomies in the weeks leading up to enrollment. We are obviously very encouraged by these results, and the enrollment in the study continues with 8 patients enrolled to date. And we have also decided to now expand the current study from 30 patients to 50 patients.

The second factor is the regulatory path forward for the development of 300 in polycythemia vera indication. As you know, approved drugs for polycythemia vera fall into the orphan drug designation category by virtue of it being a rare disease in the U.S. and Europe. And orphan status provide certain benefits to the drug developer, including 7 years of market exclusivity upon FDA approval, prescription drug use of fee waivers and tax credits for qualified clinical trial.

The third factor besides the data and the regulatory path is the very significant commercial opportunity that lies ahead of us based on the significant unmet need that exists today for these patients. And specifically, by virtue of PTG-300 being a novel mechanism-based, first-in-class non-cytoreductive natural hormone mimetic agent. While PV is a rare disease, there are approximately 100,000 people in the U.S. living with this disease, and this number is unfortunately expected to grow as the population ages. The majority of PV patients are treated with phlebotomies, cytoreductive therapies or a combination of those treatments.

Jakafi is the only FDA-approved cytoreductive therapy in PV and despite having a limited indication in patients who have had an inadequate response to current treatment, Jakafi is expected to reach almost $2 billion in sales in 2020, and the largest percentage growth is in the PV population, 19% year-over-year based on their most recently quarterly update. PTG-300 has the potential to address a significant unmet need in this category where there is a lack of new cytoreductive agents in development. 300 offers the potential of keeping patients phlebotomy-free without causing iron deficiency.

So to summarize, we are incredibly encouraged by our preliminary but strong and consistent data and, therefore, are electing to focus our resources on expedited development of 300 for polycythemia vera.

To speak more about this, I would now like to introduce our Chief Medical Officer, Dr. Samuel Saks. Sam?

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Samuel R. Saks, Protagonist Therapeutics, Inc. - Chief Medical Officer [4]

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Thanks, Dinesh. As Dinesh mentioned, we are really encouraged by the results we have seen in the ongoing Phase II study of PTG-300 in patients with polycythemia vera. Dr. Hoffman is going to walk you through the study results in detail, but I wanted to highlight why I believe this data has prompted us to focus on PV as the first indication for 300.

The majority of PV patients today are treated with phlebotomy, or phlebotomy in combination with cytoreductive therapies to control erythrocytosis and keep the hematocrit levels below 45%, as indicated in many treatment guidelines. There is a large body of evidence that shows that managing hematocrit is challenging and regardless of best available therapy, they're a substantial portion of patient segments that have poorly controlled hematocrit and therefore, a higher mortality rate compared to age match controls. The treatment paradigm of PV allows patients to enter what I call the danger zone before they get the next treatment. So even those people who are considered well-controlled have increased hematocrit leading up to their phlebotomy. Self-administered 300 may reduce doctor visits and the anxiety associated with the uncertainty of phlebotomy at that time. Phlebotomy is those of donated blood know, is associated with acute symptoms and difficulties. But the real problem in these patients is the potential for chronic symptoms related to iron deficiency in phlebotomized patients.

PTG-300 offers the possibility of putting the control of the hematocrit in the patient's hands with a weekly self-injected mimetic of an endogenous hormone mimetic. With a clear decision to focus PTG-300 development in PV, we are expanding the current study to include additional patients, and we'll be also hosting a scientific planning meeting with leaders in the field of myeloproliferative neoplasms. We will also be working with patient advocates to discuss pivotal and future studies in polycythemia vera.

I will close by saying that while further follow-up and data from additional patients will be needed to confirm the continuity of the robust clinical responses observed to date, we believe this study provides a compelling rationale to initiate planning for a pivotal program in PV. In the near term, we are expanding the current study to include additional patients as we focus on these encouraging results.

Finally, I want to express how proud I am of the entire clinical development team of Protagonist and all the Protagonist employees that helped us to get where we are today with this program.

With that, I would like to turn it over to Dr. Hoffman, who needs no introduction. He's a recognized expert in the field of myeloproliferative disorders and is Director of the Myeloproliferative Disease Program at The Icahn School of Medicine at Mount Sinai and an investigator in the 300 study. Dr. Hoffman?

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Ronald Hoffman, [5]

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Yes. Thank you very much, Sam. Thanks for the opportunity to present this really exciting data. So if we can go to Slide 7, I can educate the audience on what polycythemia vera is so they can have a better understanding of the potential for 300 and put this in context.

So as you can see here, this slide is entitled "What is Polycythemia Vera?" Well, polycythemia vera is a member of a group of chronic hematologic malignancies termed myeloproliferative neoplasms. And these neoplasms are unusual in that patients frequently live several decades. And these neoplasms occur at the level of the hematopoietic stem cell. And unlike leukemia, they are characterized by increased production of mature blood cells. In polycythemia vera, the cell that's most destructive to the patients are really the increased production of red blood cells. And that's the term erythrocytosis that you've heard previously and we'll hear subsequently.

Now the interesting thing about this disease is that there are 100,000 cases of polycythemia vera in the United States. This disease occurs across races and age groups. And the critical diagnostic warning sign is that of an elevated hematocrit, which is the hallmark of the disease. I think the importance of this drug is that although there's 100,000 cases of these patients, these patients, as I mentioned, live for several decades, so the usage of this drug would be quite prolonged over that period of time.

The disease occurs primarily in individuals who are 50 to 70 years of age. There is, however, a higher incidence in young females who present with life-threatening thrombotic events. The thrombotic events that occur in patients with polycythemia vera include strokes, as well as life-threatening thrombosis of arteries or veins within the venus -- within the abdominal cavity. There's a very high incidence of acute myocardial infarction, cerebral vascular incidents and strokes. The women that we've talked about, the young women, they are particularly prone to develop thrombosis within the abdominal cavity, especially the hepatic vein, and that leads to a life-threatening condition called Budd-Chiari syndrome. In its most severe forms, it requires liver transplantation.

Over the last 15 years, there's been tremendous understanding -- new understanding of this group of myeloproliferative neoplasms. And they've been shown to be characterized by mutations in genes that are associated with myeloid blood cell production. So the myeloproliferative neoplasms are associated with 3 driver mutations: one involving JAK2, the other gene called calreticulin and the other MPL, which is a receptor for the hormone thrombopoietin. Virtually 99% of patients with polycythemia vera have a mutation in JAK2. Most commonly, it is a mutation that occurs at exon 14, which is the JAK2V617F mutation. A smaller group of patients have a mutation in exon 12, and those are referred to as exon 12 patients, and they have a similar phenotype, again, elevated hematocrits, increased thrombosis and shorter lifespan due to these thrombotic events and severe erythrocytosis.

Next slide, please. We're going to talk now about the diagnosis, symptoms and treatment of polycythemia vera, so that you can get an understanding of the role of -- the potential role of PTG-300. The diagnosis of polycythemia vera is triggered by finding an elevated hematocrit serendipitously or by a thrombotic event, and that initiates the physician looking for the diagnosis. So for instance, a patient could present within an acute myocardial infarction and be found to have hematocrit. These warning signs, then prompt, the blood tests to be performed that look for the presence of the JAK2V617F mutation or the exon 12 mutation of JAK2V617F is not present. And sometimes, a bone marrow examination is required to perform histopathologic confirmation of the diagnosis.

This disease is not only complicated by thrombotic episodes, but it's also complicated by burdensome systemic symptoms, including fatigue, headache, visual symptoms, night sweats and itchiness. And this itchiness characteristically occurs on exposure to water and has termed aquagenic pruritus. This might seem like a trivial symptom but aquagenic pruritus can be so severe that patients are unable to shower for months or can lead to such stress that occasionally, we've unfortunately had patients who have succumbed to suicide. Thrombotic events is the greatest risk to patients with polycythemia vera. These thrombotic events, as I mentioned, include heart attacks, strokes, deep vein thrombosis or pulmonary embolism or the Budd-Chiari syndrome. The cardinal feature of treatment and reduction of this thrombotic risk is to reduce the hematocrit below 45%. Again, the hematocrit is just the measure that we use of the number of red blood cells and the degree of erythrocytosis. And that assures that essentially the viscosity of the blood or the stickiness of the book is reduced, thereby reducing the incidence of thrombosis.

So normalization of the hematocrit using therapeutic phlebotomy is very common, and it is really the first treatment in newly diagnosed patients with polycythemia vera. Unfortunately, therapeutic phlebotomy leads to the removal of red blood cells, which contain iron and it leads to more severe iron deficiency contributing to many of the exacerbation, of many of the systemic symptoms that I referred to previously. And those patients that require frequent phlebotomies or at their advanced stage that is over 60 years of age or have thrombosis, a prior thrombosis, it is the recommendation of the standard of care that those patients all be treated with hydroxyurea, interferon or Jakafi. It is in this situation that 300 is especially attractive drug because it can be used to treat patients who are not requiring myelosuppressive agents or it could be a replacement for those myelosuppressive agents. Again, the site of the reductive agents are hydroxy and interferon, which are used in combination with phlebotomy, with supplemental phlebotomy, or an alternative drug is Jakafi or ruxolitinib, which is the only U.S. FDA-approved product for phlebotomy. Each of these drugs, and I can go into that in detail during the question-and-answer period are associated with significant adverse effects and have a question -- and have a questionable surrounding their tolerability. Significant evidence, as I've mentioned, shows that controlling the hematocrit below 45% is the most important factor in minimizing thrombosis, cardiovascular events and death due to thrombosis.

Please let's go to the next slide. So we're going to talk a little bit about phlebotomy, which many of you have probably experienced when you donate blood. So most patients receive phlebotomy initially. Phlebotomy is basically the removal of a unit of blood to reduce the hematocrit below 45%. So our target is to keep those patients always below 45% because it's been well demonstrated in the literature that, that reduces significantly the incidence of thrombosis. And the incidence of thrombosis has been shown to be reduced when the degree of time with an adequate of 45% is maximized. In addition, these would patients receive low-dose aspirin to paralyze their platelets and their -- or further augment this antithrombogenesis in patients with polycythemia vera. Patients who undergo phlebotomies will often report feeling tired or dizzy after the phlebotomy. And especially in the elderly, this is a major factor. Many of these patients become hypertensive, require long stays in our outpatient facility or require fluid infusions because -- or going to congestive heart failure because of their poor fluid balance. Furthermore, regular phlebotomy results in iron deficiency that makes -- the augmentation of iron deficiency that may have a debilitating -- that may have debilitating symptoms. As I mentioned, particularly severe fatigue, weakness and cognitive impairments. This cognitive impairment is a really big deal because many of the patients complain of having a fuzzy brain syndrome, and they cannot concentrate and do their jobs. In addition, some of the patients have craving for unusual substances such as ice or clay, which is termed pica. And then, unfortunately, some of the patients have a restless leg syndrome, which prevents them from going to sleep. Three or more phlebotomies in a year in those patients receiving hydroxyurea is associated with an inferior prognosis and a higher incidence of thrombotic events. So you can also think that in those -- in that situation, even in a patient who is receiving hydroxyurea, their combination therapy with 300 could eliminate those frequent phlebotomies and further improve the treatment regimen for these patients.

I think the most important part that you'll see about this drug are the real high-quality aspect of it is that we see these patients, let's say, a 3- or 4-month intervals. At that 3- or 4-month interval, we see that they have an elevated amount of hematocrit and that triggers us to do a phlebotomy. But in reality, we have no way to monitor them during the interval between their visits, and it's likely that for significant periods of time, they have an elevated hematocrit that's only detected at the time of their visit to our clinic. So as you'll see, the really cool part about PTG-300 is that there's a sustained hematocrit control, which we've really never been able to achieve with any of the other agents or but -- or with phlebotomy alone.

Let's go now on to Slide #10, which is PTG-300 and non-cytoreductive hepcidin hormone mimetic, its mechanism of action. So how does this drug really work? Well, hepcidin hormone, has been established recently in the last couple of decades, is playing a primary role in promoting the sequestration of iron and macrophages and decreasing iron availability for the production of red blood cells. So macrophages are a type of white blood cell, and they are present not only in the bone marrow, but they're also present in all other body tissues. But the macrophages in the marrow are termed nursing cells because their activity is essentially to feed iron to red blood cells in the marrow and to alter -- to allow them to produce additional red cells. This process is disregulated by the JAK2 mutation leading to erythrocytosis. So PTG-300 is believed to limit the excess production red cells in polycythemia vera by essentially allowing that iron to remain in the macrophages. Therefore, preventing its transfer into the erythroid precursors. In addition, it blocks the iron that's present in the macrophages in the tissues outside of the marrow. And therefore, we believe alleviates many of the systemic symptoms that are associated with polycythemia vera.

Let's now go on to Slide 11, which is the Phase II study design. This is a really interesting manner in which this study has been constructed. You can see there are 3 phases. Part one is the dose-finding period, which is over 28 days. And there are 2 components to that. I believe a dose -- effective dose-finding phase, where we essentially dose-escalate patients to meet the inadequate requirements. Then a 12- to 14-week period where we essentially have efficacy evaluation that is to maintain the patient's hematocrit below 45%. And then there's part 2 as a blinded withdrawal where the patients are randomized to receive either the fixed active dose or placebo dose for up to 12 weeks. And then I'm sure the patients will be happy about that, those that go on placebo, they will then allow them -- we will be allowed to put them on to an open-label extension phase up to 52 weeks where they will again be able to receive 300. If the patient's hematocrits exceed 45%, they get supplemental phlebotomies.

Let's go on to Slide #12. This is essentially the responses that we've seen in the 7 patients that have been described, that have been evaluated. The red triangles indicate the phlebotomies, and you can see a lot of them to the left of the solid line. And that's the 32 weeks prior to treatment. And you can see that each of these patients, and these were entry criteria, had at least 3 phlebotomies during that period. And then you can see this dotted line, and that's the time at which the doses -- the drug was started. And the first thing that strikes you is that there are a lot of red triangles to the left and only one red triangle to the right, and that's one of our patients who missed one dose due to the -- unfortunately, due to the pandemic. And the numbers indicate the doses that were administered over that period of time. So for instance, you can see in patient 150201, there was a dose escalation and then de-escalation, which I'll discuss on the subsequent slide. So this really -- this slide really summarizes and probably is our most important slide. And it basically shows that with the administration of this drug and careful titration that phlebotomy is almost virtually eliminated.

Next slide. Let's focus on 2 patients. One is the top patient, which is the blue line. And you can see that this patient -- this is the individual who missed the dose because of the COVID infection. And you can see that he was started at 10 milligrams, then went up to 20 milligrams. And on 20 milligrams, he had a hematocrit 47.5, he was unable to come in at that time because of the COVID infection. So we increased his dose to 40 milligrams, his hematocrit dropped, but it was still above 45. He came in, we phlebotomized him, and we've kept him now on 40 milligrams, and you would have seen on Wednesday, and he did not require a phlebotomy and his hematocrit was below 45.

If you look at the bottom patient who is in bright red, you can see that, that patient started at time 0 on 10, then was increased to 20 and then on week 7 was -- got 40 milligrams and then on week 12 got 80 milligrams. And you can see that, that led to a hematocrit dropping to 37.5. We cut our dose to 40 milligrams, and you can see that over that period of time, she has remained phlebotomy-free. We saw her on Wednesday, and she again remains below hematocrit of 45. So there's a dose adjustment that you can easily do. The drugs are being administered at home by the patients, giving them a lot of freedom and also empowering them to control their own disease.

If we go to Slide 14. This addresses how do we know that PTG-300 is really hitting its target. And what we're measuring here is serum ferritin. And if you can recall, ferritin is a molecule that reflects storage iron. And you can see these numbers in the vertical column, and if you're below 25, that indicates that one is iron-deficient. And you can see it with the administration of the drug at subsequent doses over time, the ferritin doses, the ferritin levels increased in each of these patients except the bottom line where a patient received an extremely hematocrit control with an extremely low dose of 300. So these data indicate that the drug is acting on target.

What about the adverse defense? Because this is a chronic disease, one must anticipate that patients will be treated over decades. This is a small sample set but you can see that we had no serious adverse events and the adverse events that were recorded were essentially just irritations, essentially at the injection site that slowly resolved and one of the patients had a bruise at a 10-milligram dose.

Let's go on now to Page 16, which is our summary. So just to start this off. I think phlebotomy is used -- has probably been used for almost 100 years. And for at least, since I've been in this field, which is from the 70s, there's been great debate about what is the optimal myelosuppressive agent. There's great concern about chemotherapy being used in these patients, perhaps increasing the incidence of evolution to acute myeloid leukemia. We never really anticipated that a hormone therapy might be used for this type of disease. So I think this really represents -- this trial really represents a paradigm shift and it has great importance for patients with polycythemia vera. So I think we can easily conclude that the additional data demonstrates the potential of this drug to almost entirely avoid the need for bottoming the treatment of polycythemia vera. All dose compliant patients were phlebotomy-free. Persistent control of hematocrit levels, I think that's really one of the most important issues is that the patients are -- have a sustained control of their hematocrit, which we anticipate will lead to sustained alleviation of their systemic symptoms and elimination of thrombotic episodes. And again, as I tried to indicate to you these elevated hematocrit levels are associated with significant cardiovascular events such as heart attack and stroke. This drug also offers the possibility of weekly administration without the up and down excursions inherent in phlebotomy therapy or any of the toxicities associated with phlebotomy therapy that I discussed with you.

Also, this reduction in phlebotomy may allow sufficient iron to be available systemically to avoid symptoms that we know that are related to iron deficiency. So PTG-300 has the potential as the first non-cytoreductive therapy for PV. This drug is well tolerated and has a safety profile similar with results in prior studies. So as you can see from my presentation, I'm extremely enthusiastic about this drug and its future. Thank you for your time.

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Dinesh V. Patel, Protagonist Therapeutics, Inc. - CEO, President, Interim PAO, Secretary & Director [6]

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Thank you, Dr. Hoffman. I would like to note that the results we have discussed today address the study data as of May 1, and the study continues to enroll. And our plan will be to submit to present additional data at upcoming medical meetings. So at Protagonist, we are now working on 4 clinical programs with our 3 candidates, all discovered through our technology platform.

Our priorities are: one, advancing PTG-300 in polycythemia vera as well as in hereditary hemochromatosis; two, advancing PN-943 to a Phase II study in ulcerative colitis; and three, working closely with our partner, Janssen, to continue Phase II development of PTG-200 in Crohn's disease. As a result of our ability to limit our focus with 300 to PV and HH, we have organized our efforts to extend our cash runway for an additional 6 months. We have taken steps to manage and lower our operating costs and align our resources around our current studies. As a consequence, we now have adequate financial resources to fund planned operating and capital expenditures through the middle of 2022.

Finally, we are mindful of the impact that the current coronavirus outbreak may have on our local operations and global activities. As we disclosed in our financial results released today, we are suspending guidance for PN-943 Phase II initiation and PTG-200 Phase II data, and we are actively working to minimize impact on timelines and move as quickly as conditions would permit. Our priority is maintaining the health and safety of our employees and those who are participating in our studies. We will continue to monitor changing conditions carefully and provide updates as appropriate.

With that, we would like to now open up the call for questions. Operator?

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from the line of Chris from Nomura.

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Christopher N. Marai, Nomura Securities Co. Ltd., Research Division - MD & Senior Analyst of Biotechnology [2]

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This is Chris Marai from Nomura Instinet. Maybe the first one is for the physician on the line. Thank you so much for the overview of PV. I was curious how you look at a therapeutic like PTG-300 in terms of clinical practice on sort of an everyday basis as you see PV patients? And how do you think it may fit, assuming this profile remains consistent in sort of the treatment lines of PV, how you may see its usage, in particular relative to drugs like Jakafi?

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Ronald Hoffman, [3]

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Okay. I think that's a really terrific question. I think its use would be quite wise -- wide. I think virtually all patients with PV would be candidates for this drug. We've -- as you can see, we have treated young patients and also older patients with this drug. So it's been well tolerated. I think the advantage, again, is that, especially with young patients, those individuals, we do not want to give life-long myelosuppressive therapy or interferon or Jakafi because of unanticipated -- potential for unanticipated adverse effects. So if they have an increased phlebotomy requirement, this would be an ideal treatment for those folks because basically, it would free them up from returning to the clinic as frequently as they do and requiring repeated phlebotomies. When we risk-stratify these patients, those patients that go on the 3 treatments that I've discussed before, frequently, they still require phlebotomies, and it's been shown by European investigators, if you have additional phlebotomies during your administration of these drugs, that you are an increased risk of thrombosis. So in that setting, this drug could be an adjunctive agent that would eliminate the needs for phlebotomy.

Alternatively, and I could see this developing quite quickly, patients are frequently unwilling to take any of these agents, especially hydroxyurea and interferon because of the potential of hydroxyurea and the concern about interferon essentially having systemic symptoms, problems with individuals about immune disease and also its effect to exacerbate depression in patients. So in those individuals, I could see and even in those high-risk patients, if we can control their hematocrits with this drug, which I think we can easily do, and we've demonstrated that. That this drug would be a real competitor for any of those agents. So I think it could be essentially utilized potentially for the broad swath of patients with polycythemia vera. So I think -- and for long periods of time, that's the issue that I'd like to really emphasize to you. For instance, like young females who have Budd-Chiari syndrome, and we usually put those young females on myelosuppressive therapy and interferon for life-long periods of time, and they can live several decades. They would far prefer being on a non-chemotherapy, non-biologic agent like interferon, it would really liberate them, especially from the adverse effects.

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Christopher N. Marai, Nomura Securities Co. Ltd., Research Division - MD & Senior Analyst of Biotechnology [4]

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Okay. That's very helpful. And then I was wondering if we could touch upon the data for a minute. Dr. Hoffman, could you elaborate on some of the effects of spleen size that you may have observed in the trial, if any? What you would hope to see there on an endpoint like that in a patient population like this?

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Ronald Hoffman, [5]

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Yes. So in this situation, what you have to understand is this is not myelofibrosis. So in reality, most patients with -- or virtually all patients -- I'll say most, the overwhelming majority of patients with patients with polycythemia that don't have symptomatic splenomegaly. So the endpoints that have been used in the past, let's say, for Jakafi for reduction of spleen size are of minimal importance. So these patients, even though some of them have high-risk disease, really didn't have -- they had minimal splenomegaly at all, only 40% of patients with polycythemia vera have splenomegaly and only for our advanced disease it's symptomatic. So the sample size is too small. In animal models, when hepcidin mimetics were given, those are polycythemia vera animal models, the administration of a different hepcidin mimetic did lead to reduction in splenomegaly but we really were not able to assess this because none of these patients really had significant splenomegaly. So we await treating patients like that to see if the data in the mice is recapitulated in humans as it relates to spleen size.

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Christopher N. Marai, Nomura Securities Co. Ltd., Research Division - MD & Senior Analyst of Biotechnology [6]

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Very helpful.

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Operator [7]

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Our next question comes from the line of George Farmer of BMO.

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Read more:
Edited Transcript of PTGX earnings conference call or presentation 7-May-20 9:00pm GMT - Yahoo Finance

In 2018, the floor fell out beneath Protagonist Therapeutics. Months ahead of an expected readout, the companys first major trial, a Phase IIb testing their lead drug in ulcerative colitis, failed a futility analysis. Investors sold off en masse. Protagonist searched for answers.

Two years later, CEO Dinesh Patel maintains the drug had efficacy pointing to a blinded re-read conducted later that year but the company has moved on. Now, armed with the results from a small proof-of-concept, they are readying to put their resources behind a new lead program and another attempt at a pivotal trial in an entirely different disease.

We couldnt have asked for better data, Patel toldEndpoints News.

The drug is an artificial protein called PTG300. Unlike the drug in the 2018 trial or a compound licensed in 2017 to J&J, PTG-300 is designed to treat blood disorders. Since August, the company has been testing it in a series of tiny proof-of-concept trials to see how well it works in different diseases, including beta thalassemia, hereditary hemochromatosis and myelodysplastic syndromes.

The latest study, announced today, is in people with polycythemia vera, a chronic blood cancer in which patients produce an overabundance of red blood cells. Complications can range from itchiness to blood clots. Normally, patients are treated with phlebotomies every few weeks or months. The problem, though, is that their red blood cell counts can rise in-between and continually removing a persons blood is not without consequence. It can lead to weakness and iron deficiency, among other complications.

In the part of the trial that is now evaluable, investigators gave different doses of PTG-300 to 7 different patients who had at least three phlebotomies in the 24 weeks prior. Six patients did not need a phlebotomy over the 4 to 28 weeks they took it. A 7th had an unintended interruption to his dosing, received a single phlebotomy and continued therapy.

Lead investigator Ron Hoffman, called it a potential paradigm shift. Unlike phlebotomies or the other drugs used to treat the disease, it could allow patients to maintain good red blood cell counts over long stretches without severe side effects.

What has been done for 100 years is that patients undergo blood letting essentially, Hoffman told Endpoints. We didnt anticipate using this kind of approach. Its really pretty cool actually, Im excited.

The drug mimics the effect of hepcidin, a natural hormone that regulates iron levels. Because the body needs iron to produce red blood cells, limiting those levels can in theory limit overproduction in these patients.

The results convinced Protagonist to pick their lead indication ahead of schedule, Patel said. They will now expand their current study from a goal of 30 patients to 50 patients and they plan to start a pivotal trial next year. That will move it significantly ahead of their remaining irritable bowel disease asset, PTG-943. Development of that drug has been pushed back significantly during the Covid-19 pandemic; because it targets IL-23, it could make patients more susceptible to infection.

These are immuno-suppressive agents at the end of the day, Patel said of PTG973. Basically, for now the focus is on blood disorders.

Its a marked shift for Patel and Protagonist. The company spent a decade building its peptide-based platform, and its earliest assets were for IBD. They raised investors confidence around PTG100 and quickly brought it into a Phase IIb study. J&J offered $990 million in a deal centered on a second IBD drug, PTG-200. These drugs appealed particularly because they were oral, unlike the injectables that have dominated the inflammatory disorders market.

The J&J drug is still in the clinic, but in March of 2018, Protagonist announced that PTG-100 failed a futility analysis; an independent review board determined it had no chance of beating placebo. The stock fell 58%, from $20.43 to $8.59, roughly where it sits today.

PTG-100 has since been retired, but Patel claimed the review decision was actually wrong. The company noted a high placebo response in the initial assessment and sent it for a blinded review to a third party and CROs subcontractor, who determined some of the initial endoscopes had been initially misread and the trial should have continued.

Within two days we figured out the endoscopy readouts were errant, he said. The outcome should not have been futile.

The company went back to the FDA with the third party review, Patel said, but the FDA said they had to start a new trial. By that point, though, the company had already come up with PTG-943, which they believed worked better and was wiser to advance if they were going to start a new trial.

Now with Covid-19 pushing back IBD further, blood disorders have become the companys main strategy, despite having only a tiny, open-label data set. Patel is confident, though, that it can be a fruitful one. He said there was a clear regulatory path with a 150-person trial, good effects in those patients, and a significant potential market.

It fits all of the three category: strong data, a good regulatory path forward, and a very large commercial opportunity, he said. So thats where Protagonist will be focused.

Read more:
Two years after a wrenching setback, Protagonist touts a slice of data and sets sights on pivotal trial in a surprising place: the blood - Endpoints...

SOUTH SAN FRANCISCO May 8, 2020 (Thomson StreetEvents) -- Edited Transcript of CytomX Therapeutics Inc earnings conference call or presentation Thursday, May 7, 2020 at 9:00:00pm GMT

CytomX Therapeutics, Inc. - Senior VP & CFO

* Christopher S. Keenan

CytomX Therapeutics, Inc. - VP of IR & Corporate Communications

* Sean A. McCarthy

CytomX Therapeutics, Inc. - Chairman, CEO & President

* Biren N. Amin

* Christopher N. Marai

Nomura Securities Co. Ltd., Research Division - MD & Senior Analyst of Biotechnology

H.C. Wainwright & Co, LLC, Research Division - Associate

* Terence C. Flynn

Good day, ladies and gentlemen, and welcome to the CytomX Therapeutics First Quarter 2020 Financial Conference Call. (Operator Instructions)

As reminder, this call may be recorded. I would now like to introduce your host for today's conference, Christopher Keenan, Vice President of Investor Relations. Chris, you may begin.

Christopher S. Keenan, CytomX Therapeutics, Inc. - VP of IR & Corporate Communications [2]

Good afternoon, and thank you for joining us. Earlier today, we issued a press release that includes a summary of our recent progress and our first quarter 2020 financial results. This press release and a recording of this call can be found under the Investors and News section of our website at cytomx.com.

With me today are CytomX President, Chief Executive Officer and Chairman, Dr. Sean McCarthy; and CytomX's newly appointed Chief Financial Officer, Carlos Campoy.

During today's call, we will be making forward-looking statements. Because forward-looking statements relate to the future, they are subject to inherent uncertainties and risks that are difficult to predict and many of which are outside of our control. Important risks and uncertainties are set forth in our most recent public filings with the SEC at sec.gov, including our Form 10-Q filed today. We undertake no obligation to update any forward-looking statements, whether as a result of new information, future developments or otherwise.

I will now turn the call over to Sean.

Sean A. McCarthy, CytomX Therapeutics, Inc. - Chairman, CEO & President [3]

Great. Thanks, Chris, and good afternoon, everybody. Thanks for your patience as we were getting the call up and running. I gather there's quite a lot of call volume at the moment.

Anyhow, once again, good afternoon, and thanks for joining us. It's a pleasure to be here to provide an update on our progress during the first quarter of 2020. I'll begin today's call with a brief overview of our business operations against the backdrop of COVID-19. And then, we'll review first quarter highlights across the pipeline. I'll then turn the call over to Carlos, our new CFO, you just heard off from Chris, and Carlos will review our first quarter and financial results, and I'll wrap up. And then open the call up for questions.

Let me start by saying that all of us at CytomX hope that you and your families are well and keeping safe during the ongoing pandemic. The situation is impacting all of us in different ways, and it looks like it will continue to do so for some time. We are committed to ensuring the continued well-being of those involved with the conduct of our business. This includes the patients, the staff, physicians engaged in our clinical trials, the vendors and partners who support these trials and, of course, our dedicated employees who continue to impress with their drive and focus during these challenging times.

Despite the operational challenges presented by COVID-19, our team remains highly focused and very mindful that as the American Cancer Society has recently reminded us all, cancer is not waiting, and so neither are we.

At CytomX, we see a major opportunity to more effectively target therapeutic antibodies into disease tissues, generating new classes of anti-cancer therapies. We believe our unique Probody therapeutic platform represents a fundamental advance in the field of antibody engineering, and we are highly focused on using our platform to discover and develop a broad clinical pipeline of novel drug candidates to make a meaningful difference in the treatment of cancer. Our unique science offers the potential for new and highly effective anticancer therapies, including first-in-class molecules against novel undruggable targets, potentially best-in-class molecules against validated targets and new combination therapies.

Probody is our fully recombinant antibody prodrugs comprised of a therapeutic antibody and a mask designed to block the binding of the antibody to its target until the mask is removed. Mask removal is achieved specifically and selectively within cancer tissue by certain disease associated proteins called proteases that we know are present and active in most cancers. Proteases are in effect molecular scissors, which in the context of tumor progression, function to cut a path for invading and metastasizing cancer cells. Our Probody strategy is to leverage tumor proteases to localize antibody activity into cancer tissue, thereby decreasing target engagement in normal tissues and broadening or even creating a therapeutic window.

We have pioneered this new approach, that we believe has the potential to improve and optimize a range of antibody formats, including cancer immunotherapies, antibody drug conjugates and T-cell engaging bispecific antibodies.

Despite the emergence of COVID-19, we had a very productive and important first quarter towards the ongoing advancement of our strategy of developing innovative cancer therapies in areas of significant unmet medical need and with a particular emphasis on undruggable targets.

I'd like to start today's update with CX-2009, our wholly-owned Probody drug conjugate that targets the previously undruggable target CD166. CD166 is a tumor antigen that's expressed a high levels on most solid tumors, but it's also present on most normal tissues, ruling it out as a target for a conventional antibody drug conjugate. Our previously presented data from Phase I dose escalation in various solid cancers has shown CX-2009 to be well tolerated and clinically active as monotherapy at doses of 4 mgs per kg and above. This dose is the threshold of which drug conjugates comprising the DM4 payload, the warhead on CX-2009 have been shown by others to be active in the clinic.

Clinical activity was observed in breast, head and neck and ovarian cancers with CX-2009, and we will be presenting updated data from Phase I dose escalation for this agent at ASCO in a couple of weeks.

In the fourth quarter of 2019, we announced the initiation of a Phase II expansion study of CX-2009 monotherapy in patients with hormone receptor positive HER2-negative breast cancer at a dose of 7 mgs per kg administered every 3 weeks, with the objective of enrolling up to 40 patients. Enrollment was initiated and patients were treated during Q1, but regressively, the COVID-19 situation led us to temporarily pause new patient enrollment and new site activation in this study. Our team continues to closely monitor emerging health authority guidance at IRB/Ethics Committee recommendations and our goal is to resume the CX-2009 clinical program as soon as practical.

Now staying with the theme of undruggable targets, I'd now like to turn to CX-2029,a CD71 targeting Probody drug conjugate that we're developing in partnership with AbbVie, and for which, in Q1, we announced the achievement of a major collaboration milestone.

Long considered a high potential but undruggable antibody drug conjugate target, CD71 is known as a professional internalizer, given its role of moving iron from the extracellular space into intracellular compartments. And it does this in all dividing cells. In fact, many consider CD71 to be the gold standard internalizer to assess the in vitro activity of antibody drug conjugates.

But the presence of CD71 on normal cells has been an impediment to its use as a drug target. CX-2029 is a Probody against CD71, conjugated to the cytotoxic payload MMAE. We recently announced the achievement of prespecified dose escalation success criteria for the CX-2029 Phase I dose escalation study, resulting in a $40 million milestone payment from AbbVie to CytomX. Data from this Phase I study will be the subject of an oral presentation at ASCO 2020.

The CytomX and AbbVie teams are now actually finalizing plans for the initiation of Phase II expansions as soon as possible. CytomX has the responsibility for advancing this program through initial proof of concept, whereupon, if successful, the program will transition to AbbVie for registrational studies and ultimate commercialization. CytomX retained significant U.S. commercial rights to this asset and is also eligible to receive double-digit ex U.S. royalties, should the product reach the market.

I'd now like to move to another unique R&D strategy that we're pursuing at CytomX, which is to use our Probody technology to generate first-in-class agents against undruggable targets in the context of T-cell engaging bispecific antibodies, which I will refer to, going forward as, TCBs. TCBs are highly potent therapeutics, which directs the activity of cytotoxic T-cells to tumors. This approach has the potential to take immunologically cold tumors and make them hot, opening many new avenues for cancer treatment. While clinical advances have been made with this approach in hematologic malignancies, notably with Amgen's CD19 CD3 bispecific Blincyto. Its application in solid tumors has been challenging. The reason for this is that the high potency of TCBs can target normal tissues with low antigen expression, resulting in significant toxicities.

For several years, we've been working at CytomX to research and optimize the Probody or masked versions of TCBs with an initial focus on the EGFR-CD3 target pair. Data published by others has shown that EGFR, whilst the well-validated oncology target is undruggable in the context of a CD3 bispecific, a conventional CD3 bispecific.

After this preclinical findings have shown that Probody TCBs against EGFR could induce tumor regressions and create a therapeutic window for this cancer target. These important findings served as the foundation for our ongoing collaboration with Amgen, which I'll speak about in just a few moments.

Building on our successful research on Probody TCBs, during Q1, we announced a major strategic collaboration in this area with Astellas. Under this new agreement, CytomX and Astellas will collaborate on 4 initial programs focused on the discovery, research, development and commercialization of Probody TCBs targeting undisclosed tumor antigens for the treatment of cancer. CytomX will lead early drug discovery activities with Astellas leading preclinical and clinical development and commercialization activities. Under the terms of the agreement, CytomX received an $80 million upfront payment and is eligible to receive future preclinical, clinical and commercial milestones of over $1. 6 billion, together with tiered royalties on product sales that range from high-single digits into the mid-teens.

For certain targets, CytomX may co-fund a predetermined portion of product development costs and become eligible to receive a prespecified portion of profits in the United States. CytomX may also later elect to co-commercialize products directed towards such targets in the U.S. Research work and the collaboration is underway, and we are thrilled to have Astellas as our newest partner.

Returning now to our Amgen partnership. I'm also delighted to report that we have recently advanced a lead Probody TCB candidate against EGFR that we call CX-904 into IND-enabling studies. This is the first pro TCB from our platform to reach this important landmark. CytomX is responsible for IND filing, which is targeted for late 2021 and for early clinical development. We're very pleased with this excellent scientific progress with our Amgen alliance and with the growing excitement around the potential of the Probody TCB space, as also evidenced by our new partnership with Astellas.

Moving now to our potential best-in-class programs, CX-072, our wholly-owned anti-PD-L1 Probody and BMS-986249, the anti-CTLA-4 Probody partnered with Bristol Myers Squibb.

The CX-072 was the first Probody we advanced into the clinic, and it has provided us with crucial insights and the first clinical proof-of-concept for our platform. We'll be presenting long-term follow-up data from the CX-072 Phase I/II study as an oral presentation at ASCO.

In Q1, as part of our portfolio reprioritization, we announced the termination of the Phase II program combining CX-072 with ipilimumab, the anti-CTLA-4 antibody in patients with relapsed or refractory melanoma. This decision followed a reevaluation of the evolving clinical competitive and commercial landscape in immuno-oncology, taken together with the impact of the COVID-19 pandemic. We continue to evaluate opportunities for the further advancement of the CX-072 program and we plan to initiate combination studies with our second wholly-owned program CX-2009 later this year.

During Q1, we also announced an important pipeline milestone in our foundational oncology collaboration with BMS. The leading edge of this alliance is the anti-CTLA-4 Probody BMS-986249.

CTLA-4, the target of ipilimumab, is the prototypical checkpoint target, and blocking this mechanism has proven highly effective in the treatment of patients with melanoma and other cancer types, both as monotherapy and in combination with PD-pathway inhibitors. While a very important advance, CTLA-4 blockade can cause severe immune-related toxicities, creating a clear opportunity for a Probody version of this agent to improve tolerability, increased duration of treatment and potentially improved activity. BMS and CytomX have previously presented preclinical proof-of-concept for CTLA-4 Probodies at several major research conferences and Phase I clinical data for this Probody will be presented at ASCO.

Based on these Phase I findings, BMS recently initiated a randomized Phase II expansion study comparing the tolerability and activity of BMS-986249, plus nivolumab, to ipi plus nivo in frontline metastatic melanoma. The advancement of the CTLA-4 Probody into this study triggered a milestone payment of $10 million from BMS to CytomX.

This is an important study, that if positive, has the potential to place the ipilimumab Probody on a registrational path.

Moreover, this work is a terrific example of what we set out to do with our platform when it was first conceived of, and we're excited about its potential for cancer patients.

Additional recent progress within our BMS alliance includes the initiation of the dose escalation phase of another clinical study, a Phase I/II study for a second anti-CTLA-4 Probody. We call this BMS-986288. And this is based on a modified version of ipi.

The second clinical Probody program demonstrates BMS' ongoing commitment to our technology platform as a way to potentially unlock additional value in the CTLA-4 mechanism and across other targets.

Before handing over to Carlos, I want to also note that we continue to strengthen our executive leadership at CytomX and with the appointments in Q1 of Carlos, our CFO; and also Dr. Alison Hannah as Chief Medical Officer. Carlos and Alison bring -- each bring over 30 years of leadership experience from across their respective domains, and we are absolutely delighted to welcome them to the team.

I would now like to turn the call over briefly to Carlos.

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Carlos Campoy, CytomX Therapeutics, Inc. - Senior VP & CFO [4]

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Thank you, Sean. I'm very pleased to be here. I'd like to review the financial highlights for the first quarter ending March 31, 2020. Revenue for the quarter was $50 million compared to $29 million in the corresponding period in 2019. The increase was primarily due to the partial revenue recognition of the $40 million milestone earned from AbbVie associated with the CX-2029 project and $10 million related to the milestone earned from BMS associated with the initiation of the Phase II randomized cohort expansion of BMS-986249.

Research and development expenses were $43 million for the quarter compared to $36 million in the corresponding period in 2019. The increase was largely attributed to license and sublicense fees associated with milestones and upfront payments earned in the first quarter of 2020.

General and administrative expenses were flat compared to the corresponding period in 2019. We ended the quarter with cash, cash equivalents and investments totaling $247.9 million compared to $296.1 million as of December 31, 2019.

Our achievements in existing and new partnerships during Q1 have resulted in $130 million in milestone and upfront payments to CytomX that are not reflected in our end of Q1 cash balance. I would like to underscore the company's continued strong track record of executing strategic business development transactions to broaden our pipeline and access additional non-dilutive operating capital. We expect our strong balance sheet to allow us to comfortably meet projected operating requirements into the second half of 2022, assuming no new collaborations or financings.

With that, I'll turn the call back to Sean.

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Sean A. McCarthy, CytomX Therapeutics, Inc. - Chairman, CEO & President [5]

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Great. Thanks, Carlos.

So to wrap up, CytomX had a very strong first quarter of 2020, with many key achievements across our preclinical and clinical programs in our existing partnerships and in the formation of a major new strategic alliance. We have a strong balance sheet to advance our pipeline and weather market uncertainty. And we're looking forward to ASCO, of which we have multiple presentations that will provide important updates on all of our clinical stage programs. I am very proud of the CytomX team for staying intensely focused in these challenging times, as we drive towards making the biggest difference we can for patients with cancer.

So thanks all for your time today. We wish the very best to you and your families. And Chris, please now open the call up to questions.

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Christopher S. Keenan, CytomX Therapeutics, Inc. - VP of IR & Corporate Communications [6]

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Operator, we'll take our first question.

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Questions and Answers

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Operator [1]

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(Operator Instructions)

Our first question comes from Peter Lawson with Barclays.

Our next question comes from Peter (sic) [Chris] Marai with Nomura Instinet

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Christopher N. Marai, Nomura Securities Co. Ltd., Research Division - MD & Senior Analyst of Biotechnology [2]

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CD166 -- Can you hear me okay?

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Operator [3]

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Yes, sir. You were muted in the beginning. You came on halfway.

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Christopher N. Marai, Nomura Securities Co. Ltd., Research Division - MD & Senior Analyst of Biotechnology [4]

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Okay. I'm sorry about that. So what I mentioned is the toxicity around the CD166 program versus the CD71. I'm just curious about the payloads being used here. And the internalization profiles of the targets, given that CD71 is very efficient in internalizing, does that impact the type of payload that you chose to use? I noticed that you 2 different payloads for these products.

Also for the CD71 PDC, I'm curious about what would some expected on target toxicities might look like versus toxicities due to the payload in general?

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Sean A. McCarthy, CytomX Therapeutics, Inc. - Chairman, CEO & President [5]

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Yes. Thanks for the questions. So first of all, with regards to the payloads, going back several years, when we designed the Probody drug conjugate strategy, we made a very conscious decision that the first 2 Probody drug conjugates to take into the clinic that we would work with, the most established warheads or payloads, if you like. And DM4 was selected for the CD166 program through an alliance with ImmunoGen and MMAE, which at the time was the second most validated payload. We were able to access that through our alliance with AbbVie via their alliance with Seattle Genetics. And so frankly, we could have used -- it could have ended up being the other way around. That's just the way it played out at the time. We considered those 2 payloads to be the most and best validated at that time.

In terms of the toxicities with DM4, we've known for a long time that the principal toxicity with DM4 is ocular tox. That's exactly what we saw at the higher doses in our dose escalation in Phase I, is something that is manageable in the form of ocular prophylaxis. So that's something that we're implementing in the ongoing Phase II study.

With regard to CD71 and MMAE, the principal toxicities with MMAE are a little bit different to DM4. They're more hematologic in nature. And that's what we saw in our preclinical studies and hematologic toxicities that we're looking out for in the clinic.

With regards to CD721 on target toxicity, it's very difficult to say. With CD166, just as an example, the target is expressed on most normal tissues and you could, therefore, infer that you may see toxicity of any kind. In fact, in the clinic, we really didn't see any evidence of on target toxicity with our reported data on CD166. We'll provide the data on CD71 Phase I dose escalation in a couple of weeks at ASCO.

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Operator [6]

Follow this link:
Edited Transcript of CTMX earnings conference call or presentation 7-May-20 9:00pm GMT - Yahoo Finance

Prediabetes is when your blood sugar levels are high, but not high enough to be considered type 2 diabetes.

Since prediabetes usually doesn't come with symptoms, doctors recommend getting screened if you have certain risk factors.

Image Credit: FatCamera/E+/GettyImages

"It's sort of an in-between condition," says Sue Kirkman, MD, professor of medicine in the division of endocrinology and metabolism at UNC Health. "It's considered a precursor to type 2 diabetes."

Blood sugar levels that are between 100 and 127 mg/dL are considered prediabetes, according to the American College of Cardiology. Normal is between 70 and 100 mg/dL, and anything above 127 is considered type 2 diabetes.

About one-third of Americans have prediabetes, according to the Centers for Disease Control and Prevention (CDC), although the majority don't know it.

People who have been diagnosed with the condition have a 50 percent chance of moving on to full-blown type 2 diabetes within the next five to 10 years, according to the National Institute for Diabetes and Digestive and Kidney Diseases (NIDDK).

"If changes aren't made in eating habits and activity, many people with prediabetes will move into the diabetes category," Julie Stefanski, RDN, a dietitian, certified diabetes instructor and spokesperson for the Academy of Nutrition & Dietetics, tells LIVESTRONG.com.

Even if you don't develop diabetes, though, having prediabetes can up your risk of heart and kidney disease, per the Mayo Clinic.

If you know you have prediabetes, you have the opportunity to take measures to get your blood sugar levels down to a healthy range and prevent the condition from progressing.

Being overweight is a risk factor for prediabetes.

Image Credit: Zero Creatives/Image Source/GettyImages

The biological causes of prediabetes (and diabetes) have to do with insulin resistance.

Insulin is the hormone that guides blood sugar molecules (broken down from the food you eat) out of the bloodstream and into cells, where they're used as fuel. In prediabetes and diabetes, your body either loses the ability to respond properly to insulin or doesn't make enough insulin, which means the sugar levels in your blood build up.

"As prediabetes develops, the body struggles more and more to process certain foods," Stefanski explains. "The pancreas must make higher levels of insulin to take sugar out of the blood and put it into the cells of the body. Eventually the body won't be able to keep up with insulin needs and blood sugar levels will rise."

No one really knows what causes insulin resistance in the first place, though it seems to be a combination of factors.

"The risk factors for prediabetes are the same as risk factors for type 2 diabetes," Dr. Kirkman says. According to the Mayo Clinic, those include:

1. Family history and genetics: If you have a parent or sibling with type 2 diabetes, you're more likely to develop prediabetes (and type 2 diabetes).

2. Age: The risk of developing prediabetes rises after the age of 45.

3. Being overweight or obese: The risk is even greater if fat is concentrated around the abdomen.

4. Waist size: Men with waists larger than 40 inches around and women who have waist circumferences larger than 35 inches are at a higher risk for insulin resistance, even if their body mass index (BMI) is normal, according to the NIDDK.

5. Being inactive: Regular exercise is key to reaching and maintaining a healthy weight and helps your body better process sugar and use insulin.

6. Diet: Red and processed meat along with sugary beverages increase the risk of prediabetes. Eating more fruits, vegetables, whole grains, olive oil and nuts is linked to a lower risk.

7. Gestational diabetes: Women with a history of this pregnancy condition are at higher risk for prediabetes and type 2.

8. Race and ethnicity: African Americans, Hispanics, Native Americans, Asian Americans and Pacific Islanders have a higher risk than other groups.

9. Polycystic ovary syndrome: This hormonal disorder in women, commonly referred to as PCOS, is marked by small cysts on the ovaries, according to the Mayo Clinic.

10. Metabolic syndrome: This condition is marked by high blood pressure combined with abnormal cholesterol levels and a large waist size, per the NIDDK.

11. Other risk factors: Obstructive sleep apnea and smoking may increase insulin resistance. Certain medications and hormonal disorders can also put you at higher risk.

Extreme thirst is one sign of prediabetes.

Image Credit: fizkes/iStock/GettyImages

Like the early stages of diabetes, prediabetes usually doesn't have any symptoms at all. That's why some 90 percent of people with the condition don't even know they have it, Dr. Kirkman says.

Some people may have warning signs, which could include:

Because prediabetes usually doesn't have any symptoms, "it's important to get screening tests if you have risk factors," Dr. Kirkman says.

The American Diabetes Association (ADA) recommends testing be considered in adults who have no symptoms but have a BMI of 25 or higher (23 or higher in Asian Americans) and one or more other risk factors.

Testing should begin for all people starting at age 45. If your results are normal, you don't have to be re-tested for three years, Dr. Kirkman says.

The same three tests that can diagnose diabetes are also used to diagnose prediabetes, according to the Endocrine Society. They are:

1. Fasting blood glucose test (FBG): This is a blood test that takes place after you've been fasting for eight hours (usually overnight) and drinking only water. Prediabetes is when your fasting blood sugar is between 100 and 125 mg/dL, says Dr. Kirkman.

2. Hemoglobin A1c test (HbA1C): This test measures your blood sugar levels over the past three months. Prediabetes is indicated when your numbers are between 5.75 and 6.4 percent, says Dr. Kirkman.

3. Oral glucose tolerance test (OGTT): This test is rarely used, says Dr. Kirkman. That's partly because it's difficult to do. You have to have blood drawn two hours before and after drinking a sugary beverage, all after eight hours of fasting. A result over 140 means you are prediabetic, while over 200 indicates diabetes, per the NIDDK.

Typically, doctors will repeat the tests before giving a diagnosis of diabetes or prediabetes, says Dr. Kirkman.

Lifestyle changes like adding more activity to your days may help prevent prediabetes from turning into type 2 diabetes.

Image Credit: monkeybusinessimages/iStock/GettyImages

Treating prediabetes really means preventing type 2 diabetes, and the sooner you get started the better. The American Academy of Family Physicians (AAFP) notes that the longer you have either condition, the higher the likelihood you'll end up with health complications.

"The main goal is to not advance to the point where you have type 2 diabetes," Dr. Kirkman says. "The treatments are primarily losing weight and exercising or certain medication."

If you're overweight, losing just 5 to 7 percent of your body weight (or 10 to 14 pounds for a 200-pound person) can lower your risk for type 2 diabetes, according to the NIDDK. This will help push your blood pressure and cholesterol levels into healthy ranges, per the AAFP, and most importantly, adds Dr. Kirkman, "some people with weight loss will actually revert to having normal blood sugar."

There's debate about whether you can actually "reverse" prediabetes or even type 2 diabetes but, says Dr. Kirkman, with proper lifestyle changes, you can prevent or indefinitely delay progression.

The best way to lose weight is through diet and exercise, each of which bring their own benefits to the table.

When it comes to diet, there's no one right way to eat if you have prediabetes. One place to start, though, is in the timing of your meals.

"Start by cutting back on the portions of foods you're eating at one time," Stefanski says. "Rather than eating the majority of your food during a few hours at night, spread those choices out more so that the body doesn't have to process everything at the same time."

Did you know that keeping a food diary is one of the most effective ways to manage your weight? Download the MyPlate app to easily track calories, stay focused and achieve your goals!

You can also diminish portion sizes gradually, drink water rather than sweetened beverages and opt for whole fruit over fruit juice.

Green vegetables are a great way to feel full without raising your blood sugar, and the AAFP recommends eating more whole grains, lean proteins and low-fat dairy.

Foods to avoid include those that are processed, fried and sugary.

"No matter what your weight, it's important to become more physically active," Stefankski says. "We've worked regular activity out of our daily lives. As humans, we need to move and stay active to keep processes within the body working appropriately."

The CDC recommends aiming for at least 150 minutes of moderate physical activity each week. Brisk walking is a good option, as are swimming and bike riding.

Research shows that people who get support are more likely to achieve their goals. Indeed, those with prediabetes who enacted changes encouraged by the National Diabetes Prevention Program (DPP), which includes a support component, lowered their risk of developing type 2 diabetes by as much as 58 percent.

There's some controversy as to whether medications can or cannot help people with prediabetes. In one study, though, conducted as part of the DPP Outcomes Study, the medication metformin dropped the risk of developing type 2 diabetes by 31 percent. Ask your doctor if medication might be right for you.

See the article here:
What Is Prediabetes? Causes, Symptoms and Treatment - LIVESTRONG.COM

Boulder May 6, 2020 (Thomson StreetEvents) -- Edited Transcript of Clovis Oncology Inc earnings conference call or presentation Tuesday, May 5, 2020 at 8:30:00pm GMT

Clovis Oncology, Inc. - VP of IR

* Daniel W. Muehl

Clovis Oncology, Inc. - Executive VP & CFO

* Patrick J. Mahaffy

Clovis Oncology, Inc. - Co-Founder, CEO, President & Executive Director

SVB Leerink LLC, Research Division - MD of Targeted Oncology & Senior Research Analyst

H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst

* Kennen B. MacKay

RBC Capital Markets, Research Division - MD & Co-Head of US Biotechnology Research

Thank you for standing by, and welcome to the Clovis Oncology First Quarter 2020 Financial Results Conference Call. (Operator Instructions) Please be advised that today's conference is being recorded. (Operator Instructions) I'd now like to hand the conference over to your speaker today, Anna Sussman, Vice President of Investor Relations. Thank you. Please go ahead.

Anna Sussman, Clovis Oncology, Inc. - VP of IR [2]

Thanks, Jessie. Good afternoon, everyone. Welcome to the Clovis Oncology First Quarter 2020 Conference Call. Thank you for joining us. You've likely seen this afternoon's news release. If not, it's available on our website. As a reminder, this conference call is being recorded and webcast. Remarks may be accessed live on our website during the call and will be available in our archive for the next several weeks.

Today's agenda includes the following: Pat Mahaffy, our President and CEO, will discuss the key components and highlights of today's corporate update, including commentary about any potential impact related to COVID-19; then Dan Muehl, Clovis' Chief Financial Officer, will cover the quarter's financial results in greater detail; Pat will make a few closing remarks; and then we'll open the call for Q&A, during which time, Lindsey Rolfe, our Chief Medical Officer, will also be available to answer questions.

Before we begin, please note that during today's conference call, we may make forward-looking statements within the means of the federal securities laws, including statements concerning our financial outlook and expected business plans. All of these statements are subject to risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Our actual results could differ materially due to a number of factors, including the extent and duration of the effects of the COVID-19 pandemic. Please refer to our recent filings with the SEC for a full review of the risks and uncertainties associated with our business. Forward-looking statements speak only as of the date on which they are made, and Clovis undertakes no obligation to update or revise any forward-looking statements.

Now I'll turn the call over to Pat Mahaffy.

Patrick J. Mahaffy, Clovis Oncology, Inc. - Co-Founder, CEO, President & Executive Director [3]

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Thanks, Anna. Welcome, everybody. Appreciate you taking the time today. As we all know, the world has changed so much since our last quarterly update in late February. It's a complicated time for all of you, I know. Health care professionals have been and remain on the front lines of this global pandemic and I'd like to acknowledge the contributions of health care workers around the world, putting their lives on the line to care for those affected by COVID-19.

Closer to home, we'd also like to recognize the tremendous effort being made by our investigators and prescribers to maintain enrollment and safely manage ongoing patients in our clinical trials and for continuing to prescribe and manage Rubraca commercial patients during this period of significant upheaval to their clinics and practices.

I'll use our time today to discuss the highlights of the topics you've come to expect on our quarterly calls and focus on providing additional color on how we are navigating the COVID-19 world here at Clovis, and our view of how COVID-19 may affect oncology treatment going forward, and then we'll open it up for Q&A with Dan, Lindsey and myself.

Let's begin with a commercial update for Rubraca. I'm pleased to report that we had a very encouraging first quarter. Our global net revenue was $42.6 million. This represents an 8% sequential increase from Q4 2019 and 29% increase over Q1 2019. This was our best quarter of sales to date, despite the fact that reps had to begin staying home beginning in mid-March in the United States and could no longer call on health care providers in person for the last few weeks of the quarter. Also, our European launches in Italy, Spain and France are all occurring in an environment in which our field-based personnel have not been allowed to visit hospitals or clinics beginning in late February and are therefore also working from home.

Given these circumstances, we are very pleased with our sales growth in the first quarter. And now I'll share why we believe that Rubraca is well positioned as an oncology treatment option in the current acute COVID-19 era, and in the chronic COVID environment that is sure to follow. This period has been very disruptive for hospitals, clinics and patients as health care professionals are redirected, broadly described elective procedures are delayed and health care facilities are converted to support COVID-19 treatment efforts. We do believe that oncology will be among the first health care specialty to return to some normalcy so that likely means adapting to a new normal in a chronic COVID-19 world, one in which there is a focus on minimizing clinical visits to avoid risk to patients, especially cancer patients and other patients with known comorbidities.

It is also clear that cancer patients will need to be diagnosed and treated, given the evident risk in not actively managing their disease. We believe that Rubraca, a convenient oral therapy has significant advantages as a maintenance option in the recurrent ovarian cancer setting in an environment in, as I described, physicians are trying to reduce patient visits to their clinics. Unlike Avastin as a maintenance option that requires frequent infusions and weekly monitoring for hypertension, a known risk factor for COVID-19, Rubraca is an oral agent and is taken at home and only requires monthly routine monitor. Unlike observation, which on average leads to disease progression and requires a return to immunosuppressive chemotherapy after approximately 5 months, Rubraca has been shown to extend progression-free survival and therefore, subsequent chemotherapy, on average, nearly 14 months by independent assessment, nearly 3x longer than placebo. In fact, observation is an invitation to infusion. And unlike ZEJULA, which requires weekly blood monitoring for the first month, which obviously requires weekly visits to the clinic or a laboratory, Rubraca requires only monthly routine monitoring. As you can see, Rubraca offers numerous potential advantages in a chronic COVID-19 world, and we have already introduced a variety of new digital materials for our now home-based field personnel we use to engage with hospitals, clinics, doctors and pharmacies.

While we may see some near-term impact on revenues as physicians adapt their practices to COVID-19, we believe these advantages will remain over the course of this year and future years and as we all know, COVID-19 is not likely going away in the near term.

In addition to seeking to establish Rubraca as the maintenance treatment option of choice in recurrent ovarian cancer, we also look forward to the potential launch in the United States of Rubraca in advanced mutant BRCA prostate cancer, and that brings us to our most near-term development and regulatory program in this setting.

In November 2019, we submitted our planned supplemental new drug application, or sNDA for Rubraca as a monotherapy treatment of adult patients with BRCA1/2 mutant recurrent, metastatic CRPC. The FDA filing was based on data from the TRITON2 clinical program in advanced prostate cancer. In the U.S., by the way, approximately 12% of men with metastatic CRPC have a mutation of BRCA1/2 in their tumor.

In January 2020, we announced that the FDA accepted our sNDA for Rubraca and granted priority review status to the application with the PDUFA date of May 15, 2020. Based on our interactions with the FDA, we have no reason to expect any delay to our May 15 PDUFA date. We think that Rubraca represents an important hormone-free and chemotherapy-free option for men with metastatic CRPC and a BRCA1/2 mutation. Recall that we've previously reported at ESMO last fall, a confirmed objective response rate of 44% by investigator and a confirmed PSA response of 52%. The safety data for men with CRPC were consistent with prior safety reports for patients with ovarian cancer and other solid tumors.

We've been engaged by -- encouraged by our interactions with both the medical oncology and urology communities about the potential for Rubraca to address the unmet medical need in recurrent metastatic CRPC. We are actively engaged in launch preparations, including sales force training that was completed in early March, and we will be ready to launch upon approval. Obviously, this will be among the first group of oncology launches that we'll incur entirely or almost entirely virtually. And we have taken considerable effort to prepare for this virtual launch. Our field sales team is prepared to initiate Zoom-based sales calls with prescribers, and will leverage learnings accumulated through their virtual selling efforts in the ovarian cancer setting since mid-March. All launch collateral for the sales team has been digitized to ensure they have the ability to utilize resources in virtual interactions. The promotional national broadcast has been fully converted to a virtual streaming program enabling HCPs to watch from any computer or iPad or any device in their office or home. Additional broadcast times have been added to ensure flexibility across all U.S. time zones. Program registration will be aided through targeted online advertising that will commence the day of approval. Media and advertising efforts have been weighted toward digital programming versus print to maximize impact and effectiveness of resources invested.

So to be clear, we will be ready to launch in prostate even in this new environment. Let me turn now to the clinical pipeline for Rubraca and lucitanib as well as our ongoing plans for FAP-2286. To begin, we are adhering to the regulatory guidance that FDA and other agencies have provided regarding clinical trial conduct during COVID-19, and our clinical teams are working closely with investigators to assure the safety of trial participants and investigators while maintaining compliance with good clinical practice and minimizing risk to the integrity of our trials.

While we did not see any material disruption to our clinical trials as a result of COVID-19 during the first quarter, it is possible that near-term effects may begin to emerge across different aspects of our clinical trial programs. For example, new patient recruitment in certain clinical studies may be affected, and the conduct of clinical trials may vary by geography as some regions are more adversely affected. I will note that we continue to anticipate completing enrollment in our largest study, the ATHENA frontline maintenance study before the end of this quarter. The LODESTAR study, our Phase II pan-tumor study to evaluate Rubraca in homologous recombination repair genes across tumor types continues to enroll patients. The study will evaluate Rubraca in patients with recurrent solid tumors associated with the deleterious homologous recombination repair or HRR gene mutation. Based on our interactions with FDA, this study may be registration-enabling for a targeted gene and tumor-agnostic label. If enrollment continues as planned, we could potentially file for approval in 2021.

Next, I'd like to briefly highlight our combination studies with BMS for both Rubraca and lucitanib, and then discuss our newest compound, 2286. We remain enthusiastic about our ongoing clinical collaboration with Bristol-Myers Squibb and I'll take a moment to review certain of our combination studies for both Rubraca and lucitanib with nivolumab. I'll begin with the Rubraca combinations.

FRACTION-GC is a BMS-sponsored multi-arm Phase II study evaluating the combinations of each of Opdivo and Yervoy with Rubraca as well as Opdivo, Yervoy and Rubraca in combination for the treatment of advanced gastric cancer. This is the first sponsored study to explore this triplet combination, and it is currently enrolling patients into the safety lead-in portion of the study. The Clovis-sponsored Phase III ATHENA trial in first-line maintenance for advanced ovarian cancer continues to enroll well, despite the COVID-19 environment. And as I noted, we continue to anticipate completing enrollment in this 1,000 patient study in the second quarter of 2020.

With ATHENA, we believe we are uniquely positioned to evaluate Rubraca in terms of 2 outcomes as monotherapy versus placebo in the first-line maintenance setting in the HRD population, inclusive of BRCA and in the all-comers or intent-to-treat population as well as any potential advantage of the combination of Rubraca and Opdivo in the same patient populations. ATHENA is the first frontline switch maintenance study designed to show both PARP monotherapy and PARP/PD-1 combination therapy in one study design. I'll take a moment to remind you of the statistical analysis planned for ATHENA. First, expected in the second half of next year, we will see the results of Rubraca monotherapy versus placebo in all study populations. And then probably a year or more later, we will see the results of Rubraca plus Opdivo versus Rubraca in all study populations. In each of these analyses, we will first evaluate outcomes in the HRD population, including BRCA, and then step down to the entire intent-to-treat population.

To wrap up Rubraca and move to lucitanib, I'll described SEASTAR, our Clovis-sponsored Phase Ib/II study that includes multiple single-arm Rubraca combination studies, including the combination of Rubraca with sacituzumab govitecan, now known as Trodelvy for the treatment of advanced metastatic triple-negative breast cancer, relapsed platinum-resistant ovarian cancer and metastatic urothelial cancers. A separate arm of SEASTAR includes the combination of Rubraca with lucitanib in advanced solid tumors, which is currently in the dose-finding Phase Ib portion of the study.

Lucitanib, of course, is our investigational inhibitor of tyrosine kinases, including vascular endothelial growth factor receptors 1 through 3, platelet-derived growth factor receptors alpha and beta and fibroblast growth factor receptors 1 through 3. In February 2019, we and Bristol-Myers Squibb expanded our clinical collaboration to include planned combinations of Opdivo with lucitanib. The Clovis-sponsored LIO-1 study is a Phase Ib/II study evaluating lucitanib in combination with Opdivo. LIO-1 is now enrolling patients with advanced solid tumors in the Phase Ib portion of the study. We anticipate submitting abstracts for presentations at a medical meeting in the fall of 2020.

Lastly, the BMS-sponsored CheckMate 79X study is a Phase I/II study evaluating multiple combinations with Opdivo including an arm in combination with lucitanib in patients with second-line non-small cell lung cancer. Start-up activities for the CheckMate 79X study are proceeding for regulatory guidelines for clinical trial conduct during COVID-19.

We remain very enthusiastic about our peptide-targeted radiopharmaceutical therapy program, and in particular, our lead compound, FAP-2286. FAP is highly expressed in cancer-associated fibroblast or CAFs, which are found in the majority of cancer types, potentially making it a suitable target across a wide array of solid tumors. It is highly expressed in many epithelial cancers, including more than 90% of breast, lung, colorectal and pancreatic carcinomas. Recent preclinical data in animal models, which we expect will be reported at an upcoming medical meeting, has only increased our optimism around this program.

In addition, we and 3BP are collaborating on a discovery program directed at 3 additional targets for radionuclide therapy, to which we have global rights. We've regone to this program for many reasons, including, of course, the opportunity to be a leader in the emerging field of targeted radiotherapy for the treatment of solid tumors. In this case, we have the opportunity to be the first to clinically develop an FAP-targeted radionuclide, and we are also enthusiastic about the targets of the subject of our planned -- or our ongoing discovery collaboration.

Clovis currently plans to submit an investigational new drug or IND application for FAP-2286 in the second half of 2020, followed by a Phase I study to determine the dose and tolerability of the FAP-targeting therapeutic agent with expansion cohorts planned in multiple tumor types as part of the global development program. Thus far, in radiotherapeutic development, physicians have used an imaging agent to identify patients with the appropriate level of tumor target, in our case, FAP. We are exploring opportunities to generate imaging data for FAP-2286, potentially even before our IND is submitted. Not only would this information be useful to gain additional experience with FAP-2286 and better understand the characteristics of FAP expression in multiple tumor types, but further will allow us to collaborate with other academic institutions eager to explore the potential of FAP-2286 as an imaging and as a treatment modality.

And with that, I'll turn the call over to Dan to discuss first quarter 2020 financial results.

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Daniel W. Muehl, Clovis Oncology, Inc. - Executive VP & CFO [4]

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Thanks, Pat, and hello, everyone. We reported net product revenue for Rubraca of $42.6 million for Q1 2020, which included U.S. net product revenue of $39.3 million and ex-U. S. net product revenue of $3.3 million. This represents a sequential increase of 8% over Q4 2019 net revenue of $39.3 million and a 29% increase over Q1 2019 net product revenue of $33.1 million. U.S. net product revenue was $39.3 million for the first quarter, up 9% from $36.1 million reported in Q4 2019 and up 23% from the $31.9 million reported in Q1 2019. The supply of free drug distributed to eligible patients in the U.S. through the Rubraca Patient Assistance Program for Q1 2020 was 12% of overall commercial supply compared to 18% in Q4 2018. This represented $5.6 million in commercial value for Q1 2020 compared to $8 million in Q4 2019. We can't yet predict the impact of COVID-19 and related unemployment on cap utilization over the remainder of 2020.

Ex-U. S. net product revenue was $3.3 million for the first quarter of 2020, which represents a slight increase over the $3.2 million reported for Q4 2019 and the $1.2 million reported in the first partial quarter of ex-U. S. sales in Q1 2019. We launched Rubraca in France and Spain during March 2020, so we only expected a small contribution in Q1 for those countries. We have now recorded product revenue in each of Germany, United Kingdom, Italy, France and Spain, and we expect to launch into additional smaller European markets over time.

Gross to net adjustments totaled 22.6% in Q1 2020 compared to 17.4% in Q4 2019. The sequential increase in gross to net adjustments reflects primarily an increase in the U.S. contracting and government-related programs and the impact of growing European sales that generally have higher GTN rates. We expect gross to net adjustments to remain in this low 20% range, depending on revenue and distribution mix for the U.S. and Europe. The number of weeks in distributor inventory was flat at the end of Q1 versus Q4, so there was no buildup of inventory as a result -- as a reaction to COVID-19.

At this point in time, we have no issues with either drug supply or distribution of drug to the patient. We have described product supply costs as a meaningful part of our cash spend over the last couple of years as we transition to a new manufacturing facility, so we are in a favorable position for some time to come.

Turning now to a discussion of cash. As of March 31, we had $228.4 million in cash, cash equivalents and available for sale securities. In January 2020, the company repurchased $123.4 million aggregate principal amount of its 4.5% convertible senior notes due 2024 that were initially issued in August 2019. In April 2020, the company exchanged approximately $36 million in aggregate principal amount of its 4.5% convertible senior notes due 2024 in exchange for approximately $32.8 million in aggregate principal of 2021 notes held by such holder. In May 2020, a holder of the 4.5% convertible notes due 2024, converted $24.3 million par value of notes into approximately 3.3 million shares of common stock per the standard terms of the indenture. Following these transactions, approximately $64.4 million aggregate principal amount of these 2021 notes remain outstanding and approximately $150.6 million in aggregate principal amount of these 2024 notes remain outstanding. Additionally, the company has $300 million aggregate principal amount outstanding of its 1.25% convertible notes due 2025.

As a result of the transactions noted above, the company has reduced its total outstanding convertible debt by $145.1 million in outstanding principal amount from December 31, 2019, through May 5, 2020. And as of March 31, we had drawn approximately $50 million under the TPG ATHENA clinical trial financing and had up to $125 million available to draw under the agreement to fund the expenses of the ATHENA trial through Q3 2022.

Based on the company's anticipated revenues, spending, available financing sources and existing cash, cash equivalents and available for sale securities, we believe we have sufficient cash, cash equivalents and available for sale securities to fund our operating plan into the second half of 2021. This does not include any cash repayment that may be required to pay off unless we refinance earlier the remaining $64.4 million aggregate principal amount of the 2.5% convertible notes at their maturity in September 2021. While we did not see an impact in Q1 on our revenues, the effects of COVID-19 on our future sales are difficult to assess or predict, and we may see some near-term impact on revenues related to COVID-19. Net cash used in operating activities was $82.5 million for Q1 2020 compared to $98.5 million for Q1 2019. In addition, borrowings under the TPG ATHENA financing provided $15.6 million in cash in Q1 2020, reducing net cash utilized in operating activities to $66.9 million during the quarter. Net cash used in operating activities for Q1 2020 included product supply costs of $12.4 million and once-a-year annual incentive compensation payment. We expect product supply costs will be significantly reduced from this first quarter level for the remainder of 2020 and at least the first half of 2021. We also expect significantly lower cash burn in the second half of 2020, assuming achievement of our planned revenues over that time frame.

We reported a net loss for Q1 2020 of $99.3 million or $1.39 per share compared to a net loss for the first quarter of 2019 of $86.4 million or $1.63 per share. Net loss for Q1 2020 included share-based compensation expense of $13 million compared to $13.6 million for Q1 2019. Research and development expenses totaled $68.2 million for Q1 2020 compared to $62 million for the first quarter of 2019. The increase is primarily due to higher research and development costs for Rubraca clinical trials. We expect research and development expenses to be lower in the full year 2021 compared to 2020.

Selling, general and administrative expenses totaled $42.6 million for Q1 2020 compared to $47.8 million for the comparable periods in 2019. Selling, general and administrative expenses decreased during the first quarter of 2020, primarily due to decreased commercialization expenses for Rubraca in the U.S. and Europe. We expect savings in selling, general and administrative expenses as a result of the COVID-19 situation globally.

Lastly, we continue to explore ways to improve our balance sheet and capital structure and extend our cash balance beyond the second half of 2021. As noted, we expect our R&D expenses to decrease in 2021 compared to 2020. SG&A expenses should be lower in the upcoming months, and we expect they will be in line with the Q1 2020 levels through 2021. Our inventory purchases and other nonrecurring milestone payment expenses will significantly decrease through 2021, and we anticipate planned revenues to increase with growth in all geographies and with our anticipated prostate indication approval and launch in the U.S. All of these factors should contribute to a reduction in quarterly cash burn into and through 2021. Back to you, Pat.

--------------------------------------------------------------------------------

Patrick J. Mahaffy, Clovis Oncology, Inc. - Co-Founder, CEO, President & Executive Director [5]

--------------------------------------------------------------------------------

Thanks, Dan. In summary, we're pleased with our progress in the first quarter, and we believe that Rubraca is well positioned as a maintenance therapy of choice for recurrent ovarian cancer patients in the acute and in the coming chronic COVID-19 environment. Physicians will continue to seek to reduce patient visits to their clinics, and Rubraca offers certain advantages to achieve this goal. Rubraca is an oral agent delivered to and taken at home. Rubraca has been shown to extend progression-free survival by independent assessment by nearly 14 months on average compared to placebo or observation, which has shown PFS of only 5 months on average. And Rubraca requires only monthly routine monitoring, thus limiting patient visits to the clinic. We believe these equalities offer a compelling argument for clinicians to consider Rubraca in the maintenance setting for recurrent ovarian cancer. And soon, we hope to offer a new therapeutic option for BRCA-mutant recurrent, metastatic castrate-resistant prostate cancer patients in the U.S. as well.

We remain focused on managing our net cash utilized operations and improving our balance sheet through convertible debt and other transactions such as the transactions which occurred in January, April and May of this year. And last, but certainly not least, I'd like to acknowledge our employees, all of whom have been working from home since mid-March, and I am grateful for their ongoing commitment to support patients, health care providers and each other during this challenging and unprecedented time.

And with that, we're happy to answer any questions you may have.

================================================================================

Questions and Answers

--------------------------------------------------------------------------------

Operator [1]

--------------------------------------------------------------------------------

(Operator Instructions) Your first question comes from Kennen MacKay with RBC Capital Markets.

--------------------------------------------------------------------------------

Kennen B. MacKay, RBC Capital Markets, Research Division - MD & Co-Head of US Biotechnology Research [2]

--------------------------------------------------------------------------------

Congrats on the operational progress despite the pandemic. Pat, it seems like maybe you really have had some tailwinds from the COVID pandemic going on, obviously, arising from some of the decreased toxicity on the myeloid department. Can you maybe talk about how this could read through to prostate cancer, given some of the alternative agents and the chemotherapies that are out there have maybe even more toxicity than the PARP class.

--------------------------------------------------------------------------------

Patrick J. Mahaffy, Clovis Oncology, Inc. - Co-Founder, CEO, President & Executive Director [3]

--------------------------------------------------------------------------------

Yes. As you're aware, both ASCO and FDA have encouraged physicians to consider oral therapeutics as they consider treatment options for patients. And obviously, we hope and believe that, that will continue to accrue to our advantages. I discussed not only in the ovarian cancer setting versus certain alternative infusion based products, but versus immunosuppressive chemotherapy in prostate cancer.

--------------------------------------------------------------------------------

Operator [4]

--------------------------------------------------------------------------------

Your next question comes from Gena Wang with Barclays.

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Huidong Wang, Barclays Bank PLC, Research Division - Research Analyst [5]

--------------------------------------------------------------------------------

Maybe first one is, any geographic differences in terms of a COVID-19 impact regarding launch? And also, second question is regarding the prostate cancer. Should we actually expect any revenue in second quarter?

--------------------------------------------------------------------------------

Patrick J. Mahaffy, Clovis Oncology, Inc. - Co-Founder, CEO, President & Executive Director [6]

--------------------------------------------------------------------------------

Yes. So first, as the geographic differences regarding the launch, yes. We -- it's pretty evident that, for instance, the New York metropolitan area has been hit pretty hard. And I think we probably did see an impact on sales, at least new patient starts in New York, maybe even during the quarter. As to the prostate cancer launch, there will likely be some hotspots, where distractions to the health care system occur and could temporarily impact on prescribing. I will say that with a PDUFA date on May 15 and being prepared to launch on or before May 15, we absolutely would expect to see sales in prostate cancer in the second quarter. We'll have 6 weeks of sales.

--------------------------------------------------------------------------------

Huidong Wang, Barclays Bank PLC, Research Division - Research Analyst [7]

--------------------------------------------------------------------------------

Okay. That's very helpful. If I may just squeeze one more question. Any thoughts on ZEJULA approval in the first-line ovarian cancer? And then, how would that impact the competitive landscape and your ATHENA trial readout?

--------------------------------------------------------------------------------

Patrick J. Mahaffy, Clovis Oncology, Inc. - Co-Founder, CEO, President & Executive Director [8]

--------------------------------------------------------------------------------

So one, it was totally expected. And so it came as expected and are approved. It is not going to have any impact on our ATHENA readout. The trial is almost fully enrolled, so it will have no impact on enrollment, obviously. And in fact, we aren't even enrolling in the United States. We've started shutting down country by country, certain areas, and we've already shut down enrollment in the U.S. So it will have no impact on the timing of our readout for ATHENA.

--------------------------------------------------------------------------------

Operator [9]

--------------------------------------------------------------------------------

Your next question comes from Michael Schmidt with Guggenheim Securities.

--------------------------------------------------------------------------------

Yige Guo, Guggenheim Securities, LLC, Research Division - Associate [10]

Excerpt from:
Edited Transcript of CLVS earnings conference call or presentation 5-May-20 8:30pm GMT - Yahoo Finance

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Constipation: How to relieve constipation - Express

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The scope of the Report:

The report analyzes the key opportunities, CAGR, and Y-o-Y growth rates to allow readers to understand all the qualitative and quantitative aspects of the Male Hypogonadism Therapy market. A competition analysis is imperative in the Male Hypogonadism Therapy market and the competition landscape serves this objective. A wide company overview, financials, recent developments, and long and short-term strategies adopted are par for the course. Various parameters have been taken into account while estimating market size. The revenue generated by the leading industry participants in the sales of Male Hypogonadism Therapy across the world has been calculated through primary and secondary research. The Male Hypogonadism Therapy Market analysis is provided for the international markets including development trends, competitive landscape analysis, and key regions development status.

By Regions:

* North America (The US, Canada, and Mexico)

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Chapter 1: Introduction, market driving force product scope, market risk, market overview, and market opportunities of the global Male Hypogonadism Therapy market

Chapter 2: Evaluating the leading manufacturers of the global Male Hypogonadism Therapy market which consists of its revenue, sales, and price of the products

Chapter 3: Displaying the competitive nature among key manufacturers, with market share, revenue, and sales

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Male Hypogonadism Therapy Market 2020 | Growth Drivers, Challenges, Trends, Market Dynamics and Forecast to 2026 - Cole of Duty

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The impact of the coronavirus on the Male Hypogonadism Market Forecasted To Surpass The Value Of US$ XX Mn/Bn By 20582019-2019 - Jewish Life News

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Government efforts to slow the spread of the SARS-CoV-2 virus have had severe negative consequences for the economy. While that includes obvious industries such as restaurants and air travel, the trickle of first-quarter 2020 earnings reported to date have demonstrated that most economic sectors have been affected in at least some way.

Investors should likely expect that to include genetic testing services offered by Invitae (NYSE:NVTA), which reports first-quarter 2020 operating results after the market closes on May 5. The company has pursued a growth-at-all-costs business strategy that hinges on bringing clinical-grade genetic testing to the masses. To succeed, it needs to provide seamless telemedicine capabilities, which are now being tested sooner than expected by the coronavirus pandemic. Will the company get a passing grade and maintain its growth trajectory?

Image source: Getty Images.

Invitae offers genetic testing services to diagnose disease, inform treatment options, screen individuals for health risks, and guide health decisions during pregnancy. Many tests can be purchased online and processed with a saliva sample. The relative simplicity of sample collection suggests a trip to the doctor's office isn't required, even if clinician support is necessary to complete the ordering process.

That could help Invitae to avoid a decline of similar magnitude to NeoGenomics (NASDAQ:NEO). The oncology reference lab processes various types of cancer diagnostics, including genetic tests, at its 11 global laboratories for hospitals and biopharmaceutical companies. It experienced a 25% to 30% decline in test volumes in April 2020 compared to the year-ago period, but many of the samples it processes are more complex than saliva.

But investors can't be sure Invitae's business will avoid severe negative impacts from stay-at-home orders. The ability to do so rests largely on the success of the company's telemedicine capabilities, which also happens to be a key component of the push to make clinical-quality genetic testing part of routine healthcare.

Invitae launched its first telemedicine initiative in June 2019. The goal was to enable a smooth experience for consumers to order medical genetic testing online. To overcome a lack of local genetic testing providers and expert input, the company designed a process guided by expert genetic counseling services that was seamlessly integrated with its state-of-the-art labs. Tests could be ordered and tracked online, with final results sent directly to a patient's personal physician.

Those nascent telemedicine capabilities were bolstered in November 2019 with the acquisition of Clear Genetics, which developed chatbots for providing genetic services at scale. The HIPAA-compliant software product, named Gia, guides doctors and patients through the process for ordering medical genetic testing directly through Invitae.

In early April, Gia received a timely upgrade to make it easier for telehealth providers to determine testing options, order diagnostics, and ensure samples are delivered to laboratories. Results can also be easily accessed and discussed during telemedicine sessions.

Will it be enough to help Invitae avoid a coronavirus slowdown? Perhaps, but investors might want to consider that the bigger obstacle may not be convenience, but awareness. Genetic testing is still a relatively new service in most doctor's offices. The availability of Gia might not matter much if patients and doctors don't know it, or Invitae's growing menu of genetic tests, exists.

Image source: Getty Images.

Given the sudden arrival of the coronavirus pandemic and stay-at-home orders, it's probably unrealistic for investors to expect Invitae to make a seamless transition to telemedicine services. Investors should prepare for the business to be negatively affected -- at least temporarily.

That said, the investment in telemedicine and telehealth capabilities such as Gia give the company a unique advantage in a highly fragmented genetic testing space. The same holds true for the company's relatively unusual focus on providing clinical-grade genetic testing services directly to consumers, which many competitors have mostly resisted to date. Whether it helps Invitae to offset the negative consequences of the coronavirus pandemic or make up for growing losses incurred by the company's growth-at-all costs business model remain to be seen, but investors will get an answer when first-quarter 2020 earnings are released this week.

Read the rest here:
The Coronavirus Pandemic Will Test Invitae's Telemedicine Ambitions - Motley Fool

Market Expertz have recently published a new report on the global At-home Genetic Testing market. The study provides profound insights into updated market events and market trends. This, in turn, helps one in better comprehending the market factors, and strongly they influence the market. Also, the sections related to regions, players, dynamics, and strategies are segmented and sub-segmented to simplify the actual conditions of the industry.

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The report displays all leading market players profiles functioning in the globalAt-home Genetic Testingmarket with their SWOT analysis, fiscal status, present development, acquisitions, and mergers. The research report comprises of extensive study about various market segments and regions, emerging trends, major market drivers, challenges, opportunities, obstructions, and growth limiting factors in the market.

The report also emphasizes the initiatives undertaken by the companies operating in the market including product innovation, product launches, and technological development to help their organization offer more effective products in the market. It also studies notable business events, including corporate deals, mergers and acquisitions, joint ventures, partnerships, product launches, and brand promotions.

Leading At-home Genetic Testing manufacturers/companies operating at both regional and global levels:

23andMeMyHeritageLabCorpMyriad GeneticsAncestry.comQuest DiagnosticsGene By GeneDNA Diagnostics CenterInvitaeIntelliGeneticsAmbry GeneticsLiving DNAEasyDNA

The report also inspects the financial standing of the leading companies, which includes gross profit, revenue generation, sales volume, sales revenue, manufacturing cost, individual growth rate, and other financial ratios.

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Dominant participants of the market analyzed based on:

The competitors are segmented into the size of their individual enterprise, buyers, products, raw material usage, consumer base, etc. Additionally, the raw material chain and the supply chain are described to make the user aware of the prevailing costs in the market. Lastly, their strategies and approaches are elucidated for better comprehension. In short, the market research report classifies the competitive spectrum of this globalAt-home Genetic Testingindustry in elaborate detail.

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Market revenue splits by most promising business segments by type, by application, and any other business segment if applicable within the scope of the globalAt-home Genetic Testingmarket report. The country break-up will help you determine trends and opportunities. The prominent players are examined, and their strategies analyzed.

The Global At-home Genetic Testing Market is segmented:

In market segmentation by types of At-home Genetic Testing, the report covers-

Diagnostic ScreeningPGDRelationship testing

In market segmentation by applications of the At-home Genetic Testing, the report covers the following uses-

OnlineOffline

This At-home Genetic Testing report umbrellas vital elements such as market trends, share, size, and aspects that facilitate the growth of the companies operating in the market to help readers implement profitable strategies to boost the growth of their business. This report also analyses the expansion, market size, key segments, market share, application, key drivers, and restraints.

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Moreover, the report studies the competitive landscape that this industry offers to new entrants. Therefore, it gives a supreme edge to the user over the other competitors in the form of reliable speculations of the market. The key developments in the industry are shown with respect to the current scenario and the approaching advancements. The market report consists of prime information, which could be an efficient read such as investment return analysis, trends analysis, investment feasibility analysis and recommendations for growth.

The data in this report presented is thorough, reliable, and the result of extensive research, both primary and secondary. Moreover, the globalAt-home Genetic Testingmarket report presents the production, and import and export forecast by type, application, and region from 2020 to 2027.

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To summarize, the global At-home Genetic Testing market report studies the contemporary market to forecast the growth prospects, challenges, opportunities, risks, threats, and the trends observed in the market that can either propel or curtail the growth rate of the industry. The market factors impacting the global sector also include provincial trade policies, international trade disputes, entry barriers, and other regulatory restrictions.

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At-home Genetic Testing Market 2020 | In-Depth Study On The Current State Of The Industry And Key Insights Of The Business Scenario By 2027 - Cole of...