Archive for May, 2020

Getting your period during a pandemic is like a movie scene where characters acknowledge their plight and say, At least things cant get worse. Getting a yeast infection and period double-whammy right now, though? Thats the moment that comes right after that scene, when it starts to rain.

As unpleasant as it may be to experience the irritation of a yeast infection alongside period cramps and other symptoms, your period shouldnt have too much of an effect on how you treat and get rid of your yeast infection. Hopefully, this is particularly comforting to hear when heading to your health care providers office might not be as simple as it used to be, and stressing over anything can feel more intense than normal. If you suspect youre suddenly dealing with both a yeast infection and your period, keep reading for some insight on why this might be happening and what you can do about it.

The fungus that most often causes yeast infections is known as Candida albicans, and it occurs naturally in the body, including in the vagina. Usually, bacteria called Lactobacillus prevent this fungus from growing out of control. But sometimes this yeast has a chance to grow unchecked and cause a yeast infection, which is usually characterized by itching, irritation, and a cottage cheese-like discharge.

This overgrowth can happen for a few reasons, including using antibiotics that throw off your vaginal flora, hormone fluctuations, health conditions like uncontrolled diabetes, or even lifestyle habits like regularly wearing your sweaty workout clothes for way too long, according to the Mayo Clinic. In any case, rest assured that yeast infections are incredibly common. In fact, 75 percent of women report getting a yeast infection at some point, with many experiencing at least two infections in their lifetimes, the Mayo Clinic notes. Basically, youre in great (and probably also very annoyed) company.

While most people dont regularly get yeast infections simultaneously with their periods, its definitely possible to have both at once, Taraneh Shirazian, M.D., a gynecologist and assistant professor in the Department of Obstetrics and Gynecology at NYU Langone Health, tells SELF. This is because hormones, vaginal pH, and bacteria levels can all fluctuate in the time leading up to your period, making it easier for yeast to grow too much, H. Frank Andersen, M.D., a clinical education director and ob/gyn in the Department of Medical Education and Clinical Sciences at Washington State University, tells SELF.

More specifically, an increase in estrogen in the days before your period could predispose you to a yeast infection/menstruation combo. A significant enough uptick in estrogen is a known risk factor for yeast infections; high levels of estrogen appear to lower vaginal pH, causing it to become more acidic in a way that makes it easier for yeast to overgrow to the point of infection.

Even if youre on a combined hormonal contraceptive that suppresses this kind of natural hormonal fluctuation, the estrogen in your birth control itself can also increase your risk of a yeast infection, the Mayo Clinic says. There is also research to indicate that levels of Lactobacillus drop during your period, which causes vaginal pH to become more acidic.

Luckily, Dr. Andersen notes that theres no reason to worry that your period will make the yeast infection any worse, symptomatically speaking. You might be more annoyed, of course (see the aforementioned rain metaphor), but the fact that youre menstruating shouldnt extend or exacerbate the infection. With treatment and time, your yeast infection should conclude as usual, Dr. Shirazian says.

Normally, when you notice the first signs of a yeast infection, youre probably tempted to run to the drug store, pick up an antifungal suppository, and call it a day. But you should consider at least calling your ob/gyn before trying to treat the infection at home, especially given our current reality.

Continued here:
How to Manage a Yeast Infection and Period Symptoms at the Same Time - Self

The Latest Growth Report on Endometriosis Therapies Market size | Industry Segment by Applications (Hospital,Clinic andOther), by Type (Hormonal Contraceptives,Gonadotropin-releasing Hormone (Gn-RH) Agonists ,Progestin Therapy andAromatase Inhibitors), Regional Outlook, Industry Demand, Latest Trends, Endometriosis Therapies Industry Share, Research Growth Forecast & Revenue by Manufacturers, The Leading Company Profiles, Growth Forecasts 2026.

The Endometriosis Therapies market study offers a thorough examination of the industry space primarily by evaluating the major parameters that define the market dynamics production and consumption. In term of production, the report incorporates information pertaining the product manufacturing, its revenue, along with gross margins of the manufacturers. The unit costs offered by the producers across various geographies are also provided in the report.

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With respect to the consumption facet, the research report details the product consumption share and volume while listing out the individual sale prices. It also lays out the import and export graphs across various geographies and concludes by predicting the production and consumption patterns of the Endometriosis Therapies market during over the projection period.

A brief outline of the regional outlook:

A gist of the product spectrum:

An outline of the application spectrum:

Brief summary of the competitive landscape:

To sum it up, assessment on the upstream raw materials, downstream buyers, and distribution channels has been documented in the Endometriosis Therapies market report.

Table of Contents:

Executive Summary: It includes key trends of the Endometriosis Therapies market related to products, applications, and other crucial factors. It also provides analysis of the competitive landscape and CAGR and market size of the Endometriosis Therapies market based on production and revenue.

Production and Consumption by Region: It covers all regional markets to which the research study relates. Prices and key players in addition to production and consumption in each regional market are discussed.

Key Players: Here, the report throws light on financial ratios, pricing structure, production cost, gross profit, sales volume, revenue, and gross margin of leading and prominent companies competing in the Endometriosis Therapies market.

Market Segments: This part of the report discusses about product type and application segments of the Endometriosis Therapies market based on market share, CAGR, market size, and various other factors.

Research Methodology: This section discusses about the research methodology and approach used to prepare the report. It covers data triangulation, market breakdown, market size estimation, and research design and/or programs.

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Endometriosis Therapies Market Growth Is Skyrocketing Beyond Predictions - News by aeresearch

With his narrow face, prominent teeth and large eyes, Joey Hoofdman always looked very different from his siblings.

As well as that, his strong work ethic and keen sense of order was greatly at odds with the rest of his chaotic family.

Feeling like such an outsider made Joey miserable throughout his life. But when he began digging around for answers about his identity, little did he anticipate the scale of the genetic and ethical minefield he was about to step into.

While he was growing up in Rotterdam, Holland, Joeys parents never hid the fact they had needed help conceiving him. His dad who already had two kids from a previous marriage had gone through a vasectomy reversal for Joeys mum.

They told me they went to a doctor because of my father, explains Joey, 33. They undid the procedure because my mum really wanted to have children of their own.

Joeys parents insisted the fertility treatment theyd received had been straightforward his mother had been artificially inseminated with his fathers sperm. But Joey was unconvinced by this explanation: I was just so different from my brother and sister.

While Joeys father drank, argued, and was constantly in debt selling the kids belongings for cash, Joey by contrast was sensible, calm and early on began saving for a better life.

Eventually, sick of feeling so isolated, at just 15 Joey met his first boyfriend, moved in with him, and left his family home for ever. It was the best decision I ever made, he admits.

By the time Joey was 19, he was a successful gym owner and in a stable, loving relationship. But even though hed been able to escape the chaos of his family home, he was dogged with unease about his identity.

It nagged away at him until his father died in 2012 and believing hed never get to the bottom of it Joeys mental health spiralled.

Close friends saw it was really bothering me. It became a depression when my father passed away. I thought it was grief, but it never stopped. I felt, Maybe Im never going to know what happened?

'After three or four years, I was still in a state of depression and I didnt want to get out of bed. It was the worst feeling Id ever had.

When Joey considered suicide, his psychiatrist urged him to confront his mother. So, in March 2017, after making her a coffee and sitting her in the garden, Joey begged her for answers.

Is my father my biological father? Did something happen to you? Or am I from a neighbour or somebody else? Mum was quite upset when I asked her that. She couldnt believe I thought she might have been with another man. It distressed her, me thinking she had been unfaithful, but I just said, Mum, please. I have to know for sure.

Joey asked her to take him through exactly how he was conceived, and she said shed seen the vials of sperm labelled with the name of Joeys father.

When pressed on the name of the clinic and doctor, his mums patchy recollections led Joey to do an online search, which brought up a suburban clinic near where he grew up and the doctor Jan Karbaat.

Joey was shocked to see many complaints about missing paperwork and lost vials, suspicions that women had been given the wrong sperm, and also a court case. The government had shut the clinic in 2009. It was a horror story. Joeys mother was so upset by these findings, she threw her son out of the house there and then.

Continuing to Google Jan Karbaat sitting in his car, Joey spotted a photo of the fertility doctor behind the scandal, and got goosebumps, certain he was looking at his biological father.

It was like an electric shock. I couldnt see his face, only myself. I had the same smile, the same eyes. I sent the picture to friends, they said, Is that you, Joey?

Desperate for answers, Joey tracked down Karbaat, who was living locally. But when he knocked on his door, he was told the doctor, then 89, was too sick for visitors. A week later, he died.

Robbed of the opportunity to confront him, Joey searched for answers elsewhere. He discovered a Facebook group whose members suspected they were all Karbaats children.

Joey quickly built a rapport with Moniek Wassenaar, and noticed her Amsterdam home was strikingly similar in taste to his own. We decided to see if we were really related and did a test we were indeed half-siblings. That was the first match. It was very emotional.

Then, in June 2017, Joey put his results on a genealogy site that matches DNA results with others around the world. It paired him with Inge Herlaar, 39, who he rang, saying, I think Im your half-brother. Then came a match with Marsha Elvers, 38, another half-sister.

At the time of Karbaats death in 2017, he was thought to have had 22 recognised children from several marriages, and was suspected of secretly fathering 49 more with his patients without their permission.

In early 2019 a court decision was ruled allowing Karbaats DNA to be released for testing. Receiving the confirmation he was Karbaats son was momentous for Joey. When I saw it written down, I broke, he admits. It was almost too much to process.

Joey has at least 61 known siblings and the family keeps growing. With dark humour theyve called themselves the Karbastards. Now, Im no longer angry but I am curious, says Joey. I have the puzzle in my head, but I cant put the pieces together.

Maybe he did it because of the money, maybe he had a God complex. Thats what everyone says. But I think, at the start, he just wanted to help mothers have children.

Last month, Joey visited Karbaats grave in Rotterdam. It was the closest I could get to him. That was the most important thing so I could have peace of mind. Im not responsible for his wrongdoings and his actions, but I need to forgive him to move on. I deserve that.

Shockingly, Jan Karbaat is not the only doctor to have committed fertility fraud but no fertility doctor has ever been put behind bars for using their own sperm.

Donald Cline

In May 2015 it emerged that physician Donald Cline, whod featured in People magazine, had used his own sperm in the 1970s and 1980s while treating women in Indianapolis.

Cline was exposed after one of the people conceived at his clinic took a 23andMe DNA test, only to discover several unexpected half-siblings, whose mothers had all been Clines patients.

Norman Barwin

A pillar of the Jewish community in Ottawa, Barwin was head of the Canadian Fertility Society and had received the Queens Golden Jubilee Medal. Like Karbaat, he was first accused of chaotic record keeping, which led to women being given the wrong sperm.

But in 2016 it emerged hed been using his own sperm. Hes currently being sued by a group of his biological children, and their legal mothers and fathers, his former patients.

Cecil Jacobson

This twisted doc nicknamed The Sperminator by a TV doc about him fathered over 70 children in Virginia. His case inspired a 1993 book, Babymaker.

He also injected women with the pregnancy hormone HCG, so they produced symptoms and believed themselves pregnant.

He would give them ultrasounds and tell them that bowels or faecal matter were foetuses, before later telling them their baby had died.

The Immaculate Deception podcast is available weekly on Apple, Spotify and all podcast providers

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Doctor secretly fathered at least 60 children with patients without their permission - Mirror Online

Henry Budzynski, a 65-year-old airplane mechanic, is one of a small segment of society that is the most vulnerable to the coronavirus: those whose immune systems are compromised. He is undergoing cancer treatment and says, I cant afford the virus right now."

He stays home as much as he can, but every Wednesday, Henry Budzynski drives to a doctors office in Mechanicsville, where an inch-long needle is stuck into his skin just above his belly button, and a shot of chemotherapy is injected into the fatty tissue around his stomach.

Because of the cancer called amyloidosis that inhabits his body, his immune system is 40% to 50% the strength of a healthy persons. Later this month, after he checks himself into the 10th floor of the Massey Cancer Center for a high dose of chemotherapy and a stem cell transplant, his immune system temporarily will cease to exist altogether.

The timing undergoing such a procedure during a pandemic isnt ideal, but he has no other choice.

Budzynski, a 65-year-old airplane mechanic, is one of a small segment of society that is the most vulnerable to the coronavirus: those whose immune systems are compromised. Should he contract COVID-19, his body would be less prepared to fight off the virus, and he would be less likely to survive. Chemotherapy, certain types of cancer and organ transplants can cause ones immune system to be weakened.

I cant afford the virus right now, Budzynski said.

But the immunocompromised are also, in many ways, the most prepared to combat the pandemic. They were comfortable with social distancing before there was a name for it. Theyre adept at keeping their hands clean and watching what they touch.

Its been months since Budzynski stepped foot into a Lowes, Home Depot or Food Lion. When its time to buy groceries, he drives to the store with his wife, Rosemary, and waits in the car while she shops. Riding in his vehicle, which he designated a clean space, is off limits to everyone but Rosemary and their daughter.

Wednesday is a milestone in Budsynzkis treatment. After a year of weekly chemo shots to different points on his abdomen, this will be the final injection. His upcoming treatment, he said, should extend his life another 20 years.

While Budzynski cant afford to wait out the pandemic, other cancer patients have decided that COVID-19 poses a greater threat than their cancer. Surgery often means entering the same building where the virus is treated and interacting with potential vectors.

Even if COVID patients are corded off, the virus can still spread. One study by the U.S. Centers for Disease Control and Prevention found that the coronavirus could be found on half of the shoes of medical staffers. The virus was even discovered on hospital floors not walked by COVID patients.

Thats all my patients need to hear, said Dr. Kelly Hagan, a hematologist-oncologist with the Virginia Cancer Institute.

One of Hagans patients, an 88-year-old woman, was recently diagnosed with breast cancer. In a normal world, her surgery would have been scheduled immediately. Instead, she asked to delay it until July or August, and Hagan accepted. Six months ago, the doctor said, such hesitancy never would have been tolerated.

Hagan has found herself asking questions shes never asked in her career: Are some patients better off without immediate treatment? She examines the cancer, and how aggressively it spreads. She asks herself if the patient can survive two months without intervention. She studies the comorbidities, or risk factors, of the patient.

These questions, she said, involve the art of medicine more than its science.

Dr. Eric Douglas, with his dog, Jayden, could not visit hospitals even before the pandemic. He has a disease that causes cancerous cells to replace his disease-fighting white blood cells. A cold can spiral into pneumonia

Dr. Eric Douglas couldnt visit hospitals even before the pandemic. The cancer inside him, Waldenstroms macroglobulinemia, causes cancerous cells to replace the disease-fighting white blood cells that make up his bone marrow. A simple cold can spiral into pneumonia.

Disease can overtake his body without any explanation for its origin. In 2015, he nearly died of septic shock, and he doesnt know why.

His cancer forced him to leave his career in anesthesiology, but Douglas, 64, accepted the transition as an opportunity to remake himself. He went back to school and earned masters degrees in theology and religion. He now teaches part time at Randolph-Macon College and is on staff at New Hanover Presbyterian Church.

When the pandemic reached Virginia, he pulled down the stairs to his attic and retrieved two boxes of surgical masks he had stored in his home. He had purchased them during the 2009 swine flu epidemic, and he strapped one over his ears recently when he drove to UPS to mail a package a sweater knitted by his wife for his 1-week-old grandson he had not yet met. He sat in his Toyota RAV4 and waited until every customer left the building.

When he returned to his car, he wiped down his hands with sanitizer and drove home. But his children were upset when they found out he had taken a risk by leaving the house, so he hasnt gone anywhere since. Once a week, hell sit in his car, turn the key and let the engine idle for five minutes just to keep it running.

Among those who cannot delay his next procedure is Jerry Deans, 71, who was diagnosed with prostate cancer in 1999 and has undergone a litany of treatments since.

Later this month, hell lie on a table for 30 minutes at the Sarah Cannon Cancer Institute in Henrico County while a focused beam of radiation is projected into the T12 vertebra of his spine and his left hip. If all goes well, he wont feel a thing, and he wont experience a single side effect.

But waiting was never an option. Putting off radiation would mean risking his bones fracturing and his spine collapsing.

It needs to be done, Deans said. Its not an emergency, but its pretty urgent.

Because the cancer institute is in a separate building from the hospital, he wont be treated under the same roof as COVID patients.

When the pandemic struck, Deans was undergoing chemotherapy treatments. He began taking precautions in January, wearing gloves and no longer hugging others, long before they became common practices.

When the radiation therapy is behind him, he and his wife, Patsi, and his dog, Zoey, will take their camper north to the Potomac River for a small vacation. Patsi and Zoey, he said, are his hugging partners.

Isolation might be the hardest part for Angela Bowman, 61. She misses having physical human contact, and theres a new baby in the family she hasnt met yet. Before the pandemic, shed walk the malls at Short Pump or Virginia Center Commons and window-shop. She enjoyed going to the grocery store and finding a good sale.

Her friends will bring her groceries and leave them on the front stoop, or she might sneak into a grocery store on the way home from her chemotherapy treatment for non-Hodgkins lymphoma. Her friends have reliably called and visited in creative ways.

Two friends parked their car in her driveway, and called on the phone so they could speak to her and see her at the same time. Another came for coffee and sat on the back deck, a good distance away.

For Jim Price, 65, life in lockdown is familiar. He learned to social distance in 2013, when he had a liver transplant. Thats when he realized the importance of washing his hands and avoiding touching public doorknobs.

For Jim Price, 65, life in lockdown feels familiar. Its similar to the summer of 2013, when he received a liver transplant and spent the next eight weeks at home. Few visitors were allowed to enter his home, and if they did, they were all asked to scrub their hands in Purell, which had been purchased in bulk from Costco.

In the years since, he has become adept at washing his hands longer and more often. He keeps small containers of hand sanitizer in his car. He avoids touching public doorknobs whenever possible.

To help his body accept its new organ, Price takes an anti-rejection medicine that suppresses his immune system. For the past two months, he has lived life the way he did after his transplant, staying home, watching Bonanza or The Andy Griffith Show on television and reading fiction by authors like Dean Koontz. He also works full time as a lumber salesman.

You get this gift, this opportunity to stay alive, Price said. So you want to limit the number of opportunities to screw up.

Thanks to the coronavirus, healthy people are seeing the world from the perspective of someone whos immunocompromised, said Faith Jones, 38, who was diagnosed with lupus when she was in college.

Because of her condition, shes prone to developing pneumonia. When she felt feverish and stopped breathing normally last month, she went to the University of Virginia to be tested for COVID. The test came back negative.

She wants people to know that when they stay at home, they arent just protecting themselves. They are protecting people like her, and they are protecting the elderly.

Its not just about you, she said.

The residents of Deltaville in Middlesex County have taken the pandemic seriously, said Anne Cooper, 68. The customers at the local market generally wear masks, and at the hardware store theres a Purell station and a sign that says Use it or leave. Campgrounds have remained closed to keep outsiders away. The average age in Middlesex, she said, is 58.

Cooper administers chemotherapy pills at home for her breast cancer, twice every morning and twice every evening. She visits the market once a week, but its easy to avoid crowds in Deltaville, she said.

Twice a week, she receives physical therapy for the sciatica in her spine that causes hip and leg pain. She waits in the parking lot until her name is called, walks directly to the treatment room and interacts only with the therapist attending to her.

Still, in the back of her mind, she wonders who the therapist has interacted with and what germs he or she might have caught.

Sometimes, it scares me that Ive survived cancer for 17 years, and this might kill me in four months, she said. I leave it in the hands of the Lord.

Originally posted here:
Those with compromised immune systems are the most vulnerable to COVID-19. They are also the most prepared. - Richmond.com

Megan Dunn takes a long look at the art in the Auckland Hospital collection and finds out that yes, there is art, even in intensive care but the price of seeing it is everything.

I got the pamphlet in the mail back in January. It says after someone close to you dies it is natural to:

The day the pamphlet arrived I splayed it open and looked at the photo inside: a network of hands holding other hands. Cropped close. Just linked hands held in support of one another. Beneath the photo: we grieve as deeply as we love.

I am unable to return to normal services.

Painting from collection donated by the Art Komiti of Aucklands Paremoremo Prison

I had noticed art in waiting rooms before, not art with a capital A, not the kind of art that an art writer such as myself would bother to write about, because art like so much else in our society has its hierarchy. Contemporary art is a high-stakes game; who you write about is as important as what you say. Who cares about the art in the waiting room? Lets abbreviate: who cares?

Me. Ive had cause to think about it because last year Mums cancer returned from outer space. Shed been in remission for 17 years, which might be a national record for multiple myeloma, a cancer of the blood. Ive never really understood what her cancer is, just that she beat it first time round, and, last July, the cancer cell count was up. Then she started chemo.

Im not coping, Im just not coping, Mum called after the first round, in tears.

Have you eaten anything?

No. I dont feel like eating.

It was the dex. The specialist, a blonde in a miniskirt and long black boots, said, Thatll be the dex. Short for dexamethasone, a steroid. Another bitter pill to swallow. At that point Mum had to take up to 10 pills a day. I looked at the specialist. I was angry. Mums not coping. I had flown to Auckland to join Mum for her next haematology appointment.

But what art could I prescribe? Optimism matters, but art isnt always soothing or kind.

Im lucky I have a chance of getting better, Mum said. True. Her prognosis was good.

We went upstairs for her second round of chemo. The nurse in blue gown, gloves and a shower cap wheeled it around in a mauve container, hooked it on a drip, and inserted the port into Mums vein.

Other patients stretched out on beds like La-Z-Boys, skin drained. They too were waiting, but looked like they didnt have a chance of getting better.

I stared into the little office next to Mums chair. Harried papers on the desk; a PC lit up like a bright face, ready to supply details, cheer, whatever counts most. On the wall, an original painting of a phutukawa tree. I call it original but the concept wasnt. The sea peeked out of the background, the sky bluer than the sea. The oils had been used with little mixing, but I didnt mind that painting of the beach in summer rudely lit up by the phutukawa tree, the needles burst open in the leaves, red as fireworks.

The nurses are very nice here, Mum said.

Niceness counts like stem cells it has a tally.

That was quick, I said.

It really doesnt take long.

I wondered how long the painting took the artist. Guessing from the paint application, not long, but longer than it takes a dose of chemo to run into your veins.

I wanted to ask the nurse: who bought that painting? Who is the artist? Who put that there? But I was too shy. Why stop this real raw moment, for an intellectual aside, a detour into the art we look at it when were waiting to get better or to see how much worse it can get?

The painting of the phutukawa tree in summer yields its bouquet. Just be.

***

Mum and I got lost en route to blood work. The hospital has an art collection in its corridors that must be maintained by someone, however irregularly. We passed a Claudia Pond Eyley print black lines of plants, bright colours, a Pacific infusion abandoned along a corridor. A trio of photographs of flowers. Where are we? Is it this the way? They need to give better directions. Then, in a gangway, a pair of large, textured, brown abstracts, neither good nor bad, just out of time, like photos of your parents when they were young, in the fashions of the day. Those sideburns. What chops! Mums perm. Dads flares. A big Pat Hanly painting called Vacation was by the escalator. I meant to come back, retrace my steps, and find all this art again. And I did come back, but by then everything had changed.

I dont want to write what comes next, because I dont want the wait to be over. Waiting is a comfort in its own sad way. Just wait and see.

The sea. Waves lap in and out. I see it whenever I attend my doctors surgery above Courtenay Place. Piha is by the Korean-born photographer Jae Hoon Lee. Its moved around the surgery over the years. Now its in the waiting room above a line of plastic chairs. Whoever bought it must have thought it was soothing and anaesthetic, a balm for a worried soul like mine.

What do you want to see when you go for a smear test, or hold a baby that wont go the fuck to sleep, or wait with your partner for test results, a prognosis, a new vaccine?

I thought Ill wait to write about the art at the doctors surgery or the hospital. Or the dentist, though I have not been to the dentist in ages, no cash, so Ill wait. How much art is in your life? How many fillings? Do you have art at home? If so, what? Where did you get it? At what price?

In the waiting room, I dont blame you if you dont want to look at something difficult and ugly or think about something hard. If youre just after a good view, Arent we all?

At my doctors surgery Pihahangs near a plastic container for pamphlets, ruffled in waves.

For every problem there is a pamphlet.

The pamphlet that arrived in the mail in January is titled Department of Critical Care Medicine Bereavement Follow-up Service.

Piha in situ at Courtenay Medical, Wellington

The waiting room isnt just literal, but it is literal too. I know because the last time I was there I got a hot chocolate from the machine. Warm, syrupy. No art, just a TV on the wall.

At the haematology department, I watched a dad sitting with his young daughter on his lap. Her mind looked far away. She waited with the patience of one who has waited before. Then her mum appeared in a turban. I looked at this young mother, I had no idea what cancer of the blood she had, but I really hoped she had a chance of getting better.

Two in 100 people die, Mum said. Back in December she was waiting for her stem-cell transplant. She would be in hospital for two to three weeks. The transplant would take her immunity to zero that was where the risk of infection crept in but then the white cells would ingraft and her count would go back up. Shed signed the forms, accepting the 2% chance she would die. If theres not a bed on Thursday, it will be next week.

I wondered: Will there be any art in her room? And if so, what will it say?

The phutukawa painting in the office of the haematology department says shush shush, that lulling noise of waves from the beach. Dont worry, relax. Its OK. Look at the view. But the Jae Hoon Lee photograph in my doctors surgery says to me your hurt is timeless, the sea will exist whether we do or not, release your grip, whatever happens next will be surgically safe, emotions are never still, time is an inlet, the sea runs in and out.

***

I always knew that waiting was part of the problem. What if you wait too long? Then you cant get around to what you were going to say because you are:

***

Outside Ward 82, ICU (acute surgery). On the intercom, the number one has been buzzed off, pushed too many times. All other numbers present and correct. My mother is in bed 17, I tell the intercom, after Ive pressed the number one that isnt there and waited. Then I walked through vaguely yellow corridors lined with three framed prints, each composition a rectangle yellow, blue, orange lined with holes down the middle. They reminded me of paeans to the common household sponge. I stop at the hand-gel pump and sanitised. The art at the hospital is sanitised too but Im beyond caring. Too much caring and you move though it and pass out on the other side somewhere in the vicinity of bed 17.

At the end of the corridor past the hand gel, the Chen family have donated a small square print, red with black scribble, in honour of the ICU team. I clocked the gold engraved plaque, their appreciation registered on the wall. The painting not unlike the size of a fire alarm, but there is no glass to break open, the call has already been raised.

I got the call from the registrar on December 22 to say that Mums stem-cell plant was not going as expected and she had been admitted to ICU.

Ive been thinking about what I will do when I get better, Mum said, the night I arrived. She sat propped up in bed, on oxygen. Her face flushed, swollen, but superficially OK.

I sat next to her bed. Oh yes, what do you think?

Im going to come to your book launch.

I smiled. My book of personal essays about art and life, already way behind schedule.

What else? I asked.

I might meet someone new. I might travel.

Where would you like to go? I asked.

She paused. Maybe Africa, she said. I could go on a safari.

I nodded. Mum found it hard to walk up the small pronounced hill to our house in Wellington, sweated easily, mopped her forehead. Then shed wait, slightly panting, for her breath to right itself again, restart.

That night, I slept in the hospital. They say you can stay in my room at the Motutapu Ward, Mum said. There was no art in her room, but a wall-to-ceiling poster of a forest, kauri trees, dense, shady, green.

In the morning I got buzzed back into ICU. Mums arms and legs, twitched, calling out; face red, body puffed up; trying to unpick the PICC line from her arm, in among the beeping and the rising heart monitor, the oxygen exhaling. She doesnt recognise me, is raving to the charge nurse, who held her hand and looked at me and explained, Im just going some gentle reorientation work.

I must have seemed stunned.

Do you want to speak outside the room? the charge nurse asked.

I nodded. In the room next door to bed 17, I wailed, What is happening? Oh, what is happening? The charge nurse held me in place and comforted me, when there was no comfort to give.

Next the intensivist arrived in her blue scrubs, removed her surgical mask, wiped her hands with gel, introduced herself as Kylie. She wanted a family meeting.

The whnau room: the painting was donated by the Art Komiti of Aucklands Paremoremo Prison

The whnau room at ICU contains another Claudia Pond Eyley print on one wall and a multi-panel painting of a New Zealand landscape on the other. The mountains, a lake, smooth and even and still. A perennial view of nature, so calm, so undisturbing to see. I cant blame the hospital for containing so many paintings of the view sky, sand, sea, soothing, stretching, somehow infinite. A vista of comfort, comprehended.

I sat down on one side of the big meeting table, Kylie on the other. What is your understanding of your mothers condition? she asked. I rattled off the facts while an accompanying nurse took notes.

Mum had come in for a stem-cell transplant. She had caught pneumonia at a point in the process when she had no natural immunity. She had developed delirium. Her pre-existing heart condition had been set off: arterial fibrillation. She needed oxygen support to breathe.

This is what the 2% of risk looks like? I asked.

Yes. Kylie explained that they couldnt provide sedatives like morphine as that would risk compromising Mums breathing further. They could put Mum on a ventilator if she deteriorated but that would not be a good sign and would come with an extra level of risk. What helped patients with delirium was familiar voices.

I asked her, Are you holding anything back?

Just that this is very life threatening. Her eyes, an expression, I can now only call grave.

Well, it seemed apt. I liked Kylie. The intensivist understood how much intensity was required.

The job title intensivist seemed funny to me because in high school I was always told I was too intense. We have to find a happy medium, a teenage girlfriend once told me. So I got into art because it seemed like a place where intensity went and didnt have to turn down a notch. But art isnt the only place for intensity. At the intensive care unit (acute) on the eighth floor of Auckland Hospital the intensivists are also at work.

All day the nurse at bed 17 and I spooned jelly or orange juice into her mouth, helped her not frantically unpick her PICC line, I just want to go for a walk! No, no dear, you cant get up. Mum, you just need to rest. Rest. A familiar voice on replay. Youll feel so much better if you rest.

In the night I noticed the print of a lone bugle boy down some New Orleans alley, presumably playing jazz. No plaque. Who bought it? What family? Who was lost?

In ICU the beeping was persistent, insistent. The constant sound of inhaling, exhaling. Poke your tongue out. Ahhhh. Good girl.

Were concerned about the delirium. In ICU, delirium can be intensified, especially in older people, by the strange sounds, lights, faces.

I forgot the ducks! I keep meaning to mention the duck painting, a watercolour, a good one too, of some ducks paddling around their pond, giving no quacks in ICU. The duck pond was donated by another family, with a brass plaque. I should have jotted down the name.

The eyeball is so moveable, up and over, it can even see things that arent there. Mum said her family prayer, over and over, eyes roving. We consecrate to Thee, O Jesus of Love My aunt clutched her hands and said the prayer with her.

One night in ICU I passed a large rectangular collage of brightly coloured red and pink buttons like some budget Damien Hirst pill painting. Never passed it again. Beep, beep.

Her oxygen levels are saturating nicely.

How much?

Fifty-five percent, then down to 48%.

Dont obsess about the blood count.

Look at the patient. Look at the face.

The face her not her.

My daughter in the car on the way to the hospital singing: Pop bang crack goes the Christmas cracker, pop bang, crack goes the Christmas cracker, we will pull it off POP.

Eyes popped, snapped.

Shes been restless.

You need to sleep.

Sleep.

I stop and start, keep typing the next line, then deleting it. I dont want to get to the end of this. I dont want to remember all the family meetings in the whnau room waiting. I dont want to chart the order of those disordered days.

What was I going to say about art?

***

I spent the first weekend after Mums stem-cell transplant in her room at the Motutapu Ward. Motutapu means sacred or sanctuary. Shed just had the big dose of chemo and was quiet, but not yet unwell. My aunt was going to join her on Monday Mums 69th birthday for what would be the worst week of the process. I sat on the bed, Mum on the La-Z-Boy. We said not a whole lot.

At one point, I think I can manage a walk.

We passed the two nurses stations. En route I pointed out the art. We stopped by a faded print of sunflowers, beneath glass, but the artist was no Van Gogh. The first time Id passed the sunflowers, I hadnt rated them at all. But that was before Id read that they were by Chris Corlett, a 17-year-old who died of acute lymphoblastic leukaemia 20 years ago. Sunflowers of Hope does look like it was painted by a teenage boy. Theres something gnarly about them. Large and abundant. Full of life. Except for the leaves Chris paid special attention to the leaves stippled with decay, a bit heavy metal.

Sunflowers of Hope, Chris Cortlett, Auckland Hospital, 2019.

We stood reading the accompanying framed text about the foundation Chris had started to build up a database of 100,000 bone-marrow donors.

Courage, charisma, strength of character, sincerity whatever it is that makes some people inspirational and very special, Chris Corlett had it.

Another Claudia Pond Eyley print near the kitchen and on the door a sign that read: Dont use if youre come from a red room. I was confused by it when Id made Mum a cup of tea earlier and had to ask the nurse, Is this a red room? (Mums room was not a red room.)

I felt the light weight of Mums hand on my arm as we looped around to reception. I showed her my favourite painting, tucked in a corner. Its colours were so bold that from the corner of my eye I first suspected Matisse. Then was embarrassed when I realised the artist is Harriet, aged six, who donated Flowers for the Leukaemia Ward in memory of her father, Ned.

Harriet may not be Matisse but for a six-year-old her vase of flowers is a masterpiece of colour and compression.

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Art in the waiting room - The Spinoff

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Approach may lead to new treatment approach for osteoarthritis, obesity

Researchers at Washington University School of Medicine in St. Louis found that gene therapy in mice helped build strength and significant muscle mass quickly, while reducing the severity of osteoarthritis. The gene therapy also prevented obesity, even when the mice were fed a high-fat diet.

Exercise and physical therapy often are recommended to help people who have arthritis. Both can strengthen muscle a benefit that also can reduce joint pain. But building muscle mass and strength can take many months and be difficult in the face of joint pain from osteoarthritis, particularly for older people who are overweight. A new study in mice at Washington University School of Medicine in St. Louis, however, suggests gene therapy one day may help those patients.

The research shows that gene therapy helped build significant muscle mass quickly and reduced the severity of osteoarthritis in the mice, even though they didnt exercise more. The therapy also staved off obesity, even when the mice ate an extremely high-fat diet.

The study is published online May 8 in the journal Science Advances.

Obesity is the most common risk factor for osteoarthritis, said senior investigator Farshid Guilak, PhD, the Mildred B. Simon Research Professor of Orthopaedic Surgery and director of research at Shriners Hospitals for Children St. Louis. Being overweight can hinder a persons ability to exercise and benefit fully from physical therapy. Weve identified here a way to use gene therapy to build muscle quickly. It had a profound effect in the mice and kept their weight in check, suggesting a similar approach may be effective against arthritis, particularly in cases of morbid obesity.

With the papers first author, Ruhang Tang, PhD, a senior scientist in Guilaks laboratory, Guilak and his research team gave 8-week-old mice a single injection each of a virus carrying a gene called follistatin. The gene works to block the activity of a protein in muscle that keeps muscle growth in check. This enabled the mice to gain significant muscle mass without exercising more than usual.

Even without additional exercise, and while continuing to eat a high-fat diet, the muscle mass of these super mice more than doubled, and their strength nearly doubled, too. The mice also had less cartilage damage related to osteoarthritis, lower numbers of inflammatory cells and proteins in their joints, fewer metabolic problems, and healthier hearts and blood vessels than littermates that did not receive the gene therapy. The mice also were significantly less sensitive to pain.

One worry was that some of the muscle growth prompted by the gene therapy might turn out to be harmful. The heart, for example, is a muscle, and a condition called cardiac hypertrophy, in which the hearts walls thicken, is not a good thing. But in these mice, heart function actually improved, as did cardiovascular health in general.

Longer-term studies will be needed to determine the safety of this type of gene therapy. But, if safe, the strategy could be particularly beneficial for patients with conditions such as muscular dystrophy that make it difficult to build new muscle.

In the meantime, Guilak, who also co-directs the Washington University Center for Regenerative Medicine and is a professor of biomedical engineering and of developmental biology, said more traditional methods of muscle strengthening, such as lifting weights or physical therapy, remain the first line of treatment for patients with osteoarthritis.

Something like this could take years to develop, but were excited about its prospects for reducing joint damage related to osteoarthritis, as well as possibly being useful in extreme cases of obesity, he said.

Tang R, Harasymowicz NS, Wu CL, Collins KH, Choi YR, Oswald SJ, Guilak F. Gene therapy for follistatin mitigates systemic metabolic inflammation and post-traumatic arthritis in high-fat diet-induced obesity. Science Advances, published online May 8, 2020.

This work was supported by the Shriners Hospitals for Children, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute on Aging and the Office of the Director of the National Institutes of Health (NIH). Grant numbers AR50245, AR48852, AG15768, AR48182, AG 46927, AR073752, OD10707, AR060719, AR057235. Additional funding was provided by the Arthritis Foundation and the Nancy Taylor Foundation for Chronic Diseases.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Gene therapy in mice builds muscle, reduces fat Washington University School of Medicine in St. Louis - Washington University School of Medicine in...

PHILADELPHIA, May 07, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders and the Gene Therapy Program (GTP) at the University of Pennsylvania (UPenn) today announced the expansion of their collaboration agreement to include an additional five programs and extending Passage Bios period to exercise new programs for an additional three years (through 2025). Additionally, Passage Bio will fund discovery research at GTP and will receive exclusive rights, subject to certain limitations, to technologies resulting from the discovery program for Passage Bio products developed with GTP, such as novel capsids, toxicity reduction technologies and delivery and formulation improvements.

Our collaboration with the GTP gives us access not only to the best discovery, technology, and research available but also to pioneering expertise in the field of gene therapy, including pre-clinical development and manufacturing experience that will help guide our programs as we move into clinical development, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. Expanding this collaboration provides us with the opportunity to not only deepen our pipeline but also strengthen our own expertise and capabilities as we strive to develop transformative gene therapies for patients. We are tremendously proud of the progress we have accomplished to date through this partnership and look forward to continuing this momentum in the years to come.

This expansion builds upon the original collaboration, which successfully established a strong partnership between Passage and GTP. Under the expanded agreement, Passage will pay $5 million annually to Penn to fund research across numerous technology applications for gene therapy. In addition to five additional program options and an extension of the relationship through 2025, Passage will receive exclusive rights, subject to certain limitations, to IP arising from this research and related indications that are applicable to the products it develops with GTP.

The partnership between GTP and Passage Bio continues to be extremely strong and productive as we collaborate to bring our gene therapy products to patients. We are extremely excited to expand the reach of our CNS products and discovery research through this continued collaboration, said James Wilson, M.D., Ph.D. director of the Gene Therapy Program at the University of Pennsylvania and chief scientific advisor of Passage Bio. As a co-founder of the company, I am also deeply committed to the growth and success of Passage. I believe that the expansion of this strong collaboration further establishes Passage Bios leadership in gene therapy and I look forward to continuing to work with our dedicated teams to reach these shared goals of helping patients with rare, monogenic CNS disorders.

About Passage Bio Passage Bio is a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders with limited or no approved treatment options. The company is based in Philadelphia, PA and has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program (GTP). The GTP conducts discovery and IND-enabling preclinical work and Passage Bio conducts all clinical development, regulatory strategy and commercialization activities under the agreement. The company has a development portfolio of six product candidates, with the option to license eleven more, with lead programs in GM1 gangliosidosis, frontotemporal dementia and Krabbe disease.

Forward Looking StatementThis press release contains forward-looking statements within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about our collaborators and partners ability to execute key initiatives and the benefits and obligations associated with our arrangements with our collaborators and partners; and the ability of our lead product candidates to treat the underlying causes of their respective target monogenic CNS disorders. These forward-looking statements may be accompanied by such words as aim, anticipate, believe, could, estimate, expect, forecast, goal, intend, may, might, plan, potential, possible, will, would, and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop, obtain regulatory approval for and commercialize our product candidates; the timing and results of preclinical studies and clinical trials; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; failure to protect and enforce our intellectual property, and other proprietary rights; failure to successfully execute or realize the anticipated benefits of our strategic and growth initiatives; risks relating to technology failures or breaches; our dependence on collaborators and other third parties for the development of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; risks associated with current and potential future healthcare reforms; risks relating to attracting and retaining key personnel; failure to comply with legal and regulatory requirements; risks relating to access to capital and credit markets; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with theSecurities and Exchange Commission(SEC), and other reports as filed with theSEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

For further information, please contact:

Investors:Sarah McCabeStern Investor Relations, Inc.212-362-1200sarah.mccabe@sternir.com

Media:Emily MaxwellHDMZ312-506-5220emily.maxwell@hdmz.com

Financial Disclosure: The University of Pennsylvania and Dr. James Wilson are both co-founders of Passage Bio and hold equity interests in the company. Dr. Wilson is also the chief scientific advisor of the Company. Penn and GTP are the recipients of significant sponsored research support from the Company under research programs directed by Dr. Wilson. Penn has licensed or optioned numerous technologies to Passage Bio under an existing license and these ongoing sponsored research activities, and both Penn and Dr. Wilson stand to receive additional financial gains in the future under these licensing arrangements.

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Passage Bio Announces Expansion of Gene Therapy Collaboration with University of Pennsylvania - GlobeNewswire

Scientists at the Washington University School of Medicine in St. Louis, believe that gene therapy could one day be used as fat reducing therapy.

In a new study conducted on mice, scientists found that gene therapy helped build significant muscle mass quickly and reduced the severity of osteoarthritis in the mice without exercising more. Surprisingly, the therapy also staved off obesity, even when the mice ate an extremely high-fat diet.

Senior investigator Farshid Guilak, Ph.D., the Mildred B. Simon Research Professor of Orthopaedic Surgery and director of research at Shriners Hospitals for ChildrenSt. Louis said,Obesity is the most common risk factor for osteoarthritis. Being overweight can hinder a persons ability to exercise and benefit fully from physical therapy. Weve identified here a way to use gene therapy to build muscle quickly. It had a profound effect on the mice and kept their weight in check, suggesting a similar approach may be effective against arthritis, particularly in cases of morbid obesity.

Scientists gave 8-week-old mice a single injection of a virus conveying a gene called follistatin. The gene works to obstruct the action of a protein in muscle that keeps muscle growth in check. This empowered the mice to gain significant muscle mass without exercising more than usual.

Even without additional exercise, and while continuing to eat a high-fat diet, the muscle mass of these super mice more than doubled, and their strength nearly doubled, too. The mice also had less cartilage damage related to osteoarthritis, lower numbers of inflammatory cells and proteins in their joints, fewer metabolic problems, and healthier hearts and blood vessels than littermates that did not receive the gene therapy. The mice also were significantly less sensitive to pain.

During the study, scientists were concerned that some of the muscle growth might lead to being harmful. But, they found that heart function improved, as did cardiovascular health in general.

Although scientists think that long-term studies are required to determine the safety of this type of gene therapy, but, if safe, the strategy could be particularly beneficial for patients with conditions such as muscular dystrophy that make it challenging to build new muscle.

Guilak said,More traditional methods of muscle strengthening, such as lifting weights or physical therapy, remain the first line of treatment for patients with osteoarthritis. Something like this could take years to develop. Still, were excited about its prospects for reducing joint damage related to osteoarthritis, as well as possibly being useful in extreme cases of obesity.

Link:
Study suggests effective fat-reducing therapy - Tech Explorist

A new business intelligence report released by CMI with the title Global Gene Therapy for Rare Disease Market Research Report 2020-2027 is designed covering micro level of analysis by manufacturers and key business segments. The Global Gene Therapy for Rare Disease Market survey analysis offers energetic visions to conclude and study market size, market hopes, and competitive surroundings. The research is derived through primary and secondary statistics sources and it comprises both qualitative and quantitative detailing.

Whats keeping Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., uniQure N.V, Orchard Therapeutics Plc., PTC Therapeutics, Inc., and BioMarin Pharmaceutical Inc. Ahead in the Market? Benchmark yourself with the strategic moves and findings recently released by CMI.

Get Sample Copy Report @ https://www.coherentmarketinsights.com/insight/request-sample/2321

This report sample includesBrief Introduction to the research report.Table of Contents (Scope covered as a part of the study)Top players in the marketResearch framework (presentation)Research methodology adopted by Coherent Market Insights

Market Overview of Global Gene Therapy for Rare Disease

If you are involved in the Global Gene Therapy for Rare Disease industry or aim to be, then this study will provide you inclusive point of view. Its vital you keep your market knowledge up to date segmented by Applications and major players. If you have a different set of players/manufacturers according to geography or needs regional or country segmented reports we can provide customization according to your requirement.

This study mainly helps understand which market segments or Region or Country they should focus in coming years to channelize their efforts and investments to maximize growth and profitability. The report presents the market competitive landscape and a consistent in depth analysis of the major vendor/key players in the market.

Furthermore, the years considered for the study are as follows:

Historical year 2015 2019

Base year 2019

Forecast period** 2020 to 2027 [** unless otherwise stated]

**Moreover, it will also include the opportunities available in micro markets for stakeholders to invest, detailed analysis of competitive landscape and product services of key players.

Regions included:

North America (United States, Canada, and Mexico)

Europe (Germany, France, UK, Russia, and Italy)

Asia-Pacific (China, Japan, Korea, India, and Southeast Asia)

South America (Brazil, Argentina, Colombia)

Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

Read Detailed Index of full Research Study at https://www.coherentmarketinsights.com/market-insight/gene-therapy-for-rare-disease-market-2321

Important Features that are under offering & key highlights of the report:

Detailed overview of Gene Therapy for Rare Disease market

Changing market dynamics of the industry

In-depth market segmentation by Type, Application etc

Historical, current and projected market size in terms of volume and value

Recent industry trends and developments

Competitive landscape of Gene Therapy for Rare Disease market

Strategies of key players and product offerings

Potential and niche segments/regions exhibiting promising growth

A neutral perspective towards Gene Therapy for Rare Disease market performance

Market players information to sustain and enhance their footprint

Buy This Complete A Business Report: https://www.coherentmarketinsights.com/insight/buy-now/2321

Major Highlights of TOC:

Chapter One: Global Gene Therapy for Rare Disease Market Industry Overview

1.1 Gene Therapy for Rare Disease Industry

1.1.1 Overview

1.1.2 Products of Major Companies

1.2 Gene Therapy for Rare Disease Market Segment

1.2.1 Industry Chain

1.2.2 Consumer Distribution

1.3 Price & Cost Overview

Chapter Two: Global Gene Therapy for Rare Disease Market Demand

2.1 Segment Overview

2.1.1 APPLICATION 1

2.1.2 APPLICATION 2

2.1.3 Other

2.2 Global Gene Therapy for Rare Disease Market Size by Demand

2.3 Global Gene Therapy for Rare Disease Market Forecast by Demand

Chapter Three: Global Gene Therapy for Rare Disease Market by Type

3.1 By Type

3.1.1 TYPE 1

3.1.2 TYPE 2

3.2 Gene Therapy for Rare Disease Market Size by Type

3.3 Gene Therapy for Rare Disease Market Forecast by Type

Chapter Four: Major Region of Gene Therapy for Rare Disease Market

4.1 Global Gene Therapy for Rare Disease Sales

4.2 Global Gene Therapy for Rare Disease Revenue & market share

Chapter Five: Major Companies List

Chapter Six: Conclusion

Get PDF Brochure of this Business Report @ https://www.coherentmarketinsights.com/insight/request-pdf/2321

Key questions answered

o Who are the Leading key players and what are their Key Business plans in the Global Gene Therapy for Rare Disease market?

o What are the key concerns of the five forces analysis of the Global Gene Therapy for Rare Disease market?

o What are different prospects and threats faced by the dealers in the Global Gene Therapy for Rare Disease market?

o What are the strengths and weaknesses of the key vendors?

Thanks for reading this article; you can also get individual chapter wise section or region wise report version like North America, Europe or Asia.

About the Author:

Coherent Market Insights is a prominent market research and consulting firm offering action-ready syndicated research reports, custom market analysis, consulting services, and competitive analysis through various recommendations related to emerging market trends, technologies, and potential absolute dollar opportunity.

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Gene Therapy for Rare Disease Market 2020 Coronavirus (Covid-19) Business Impact 2026 Growth Trends by Manufacturers, Regions, Type and Application,...

The smid-cap biotech earnings deluge hit Wall Street in the week ended May 9, positively impacting the stocks in the sector. The iShares NASDAQ Biotechnology Index (NASDAQ: IBB) gained about 6% for the week.

Large-cap pharma names AstraZeneca plc (NYSE: AZN) and Novartis AG (NYSE: NVS) received FDA nods for their heart failure and lung cancer therapies, respectively. After receiving emergency use authorization from the FDA in the U.S., Gilead Sciences, Inc.'s (NASDAQ: GILD) remdesivir obtained full regulatory approval in Japan.

Moderna Inc (NASDAQ: MRNA) received the OK toproceedwith the Phase 2 trial of its mRNA coronavirus vaccine candidate mRNA-1273.

The following are the key events and catalysts that biotech investors need to watch in the coming week.

Clovis Oncology Inc (NASDAQ: CLVS) awaits the FDA nod for an expanded indication for its cancer therapy Rubraca. The sNDA seeks approval of Rubraca as a monotherapy treatment for patients with BRCA1/2-mutant recurrent, metastatic castrate-resistant prostate cancer.

Abeona Therapeutics Inc (NASDAQ: ABEO) is due to present updated interim results from the Transpher A and Transpher B studies, Phase 1/2 trials of ABO-102 and ABO-101, respectively, in mucopolysaccharidosis type IIIA, aka as Sanfilippo syndrome type.

Rocket Pharmaceuticals Inc (NASDAQ: RCKT will present updated data from Phase 1/2 FANCOLEN-I study, which is evaluating the safety and efficacy of infusion of autologous CD34 + cells transduced with a lentiviral vector carrying the FANCA gene in patients with Fanconi anemia subtype A, and updates from the Phase 1 LAD-I study that is evaluating its investigational gene therapy RP-L201 to treat severe Leukocyte Adhesion Deficiency-I.

Avrobio Inc (NASDAQ: AVRO) is scheduled to make an oral presentation on new data from the Phase 2 trial of AVR-RD-01 for Fabry (Wednesday). The company will also make an oral presentation of new data from the collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04 in cystinosis. Another oral presentation on new data from a preclinical research program for a gene therapy for Pompe disease is also scheduled for Wednesday.

Ultragenyx Pharmaceutical Inc (NASDAQ: RARE) is due to present updated data from the first three cohorts of a Phase 1/2 study of DTX301 in treating ornithine transcarbamylase deficiency (Wednesday). The company will also present updated data from the confirmatory cohort from a Phase 1/2 study of DTX401 in glycogen storage disease Type 1a (Friday).

Pfizer Inc. (NYSE: PFE) will present Phase 1b data for PF-06939926 in Duchenne muscular dystrophy on Friday.

Krystal Biotech Inc (NASDAQ: KRYS) will present a poster on KB407, an HSV-1 based gene therapy vector, for the treatment of cystic fibrosis.

See Also: Gilead Works To 'Maximize Global Supply' Of Coronavirus Candidate Remdesivir Amid Threat Of Patent Loss

Genocea Biosciences Inc (NASDAQ: GNCA) will host a KOL symposium with a live Q&A for analysts and investors to reflect on the progress of the T cell therapy landscape and provide an in-depth profile of GEN-011 Genocea's neoantigen cell therapy. (Tuesday)

Krystal Biotech is due to present at the SID meeting withresults froma Phase 1/2 study of in vivo gene therapy KB105 for treating autosomal recessive congenital ichthyosis as well as results of a Phase 1/2 trial that is evaluating in vivo correction of dystrophic epidermolysis bullosa by direct cutaneous COL7A1 gene replacement.

Caladrius Biosciences Inc (NASDAQ: CLBS) will present at the SCAI meeting Thursday with full data from the ESCaPE-CMD study of CLBS16 for the treatment of coronary microvascular dysfunction.

Constellation Pharmaceuticals Inc (NASDAQ: CNST) said abstracts of a presentation due at the June 11-14 European Hematology Association meeting will be made available Thursday. The abstract pertains to an interim update from the MANIFEST Phase 2 study that is evaluating CPI-0610 in myelofibrosis.

Auris Medical Holding Ltd (NASDAQ: EARS) is due to release top-line data in early May from the Phase 1b trial that is evaluating AM-201 in healthy volunteers. AM-201 is the company's investigational drug for the prevention of antipsychotic-induced weight gain and somnolence.

Monday

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ORIC Pharmaceuticals Inc (NASDAQ: ORIC)

2020 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

Excerpt from:
The Week Ahead In Biotech: Virtual Conference Presentations, Clovis PDUFA Date In The Spotlight - Benzinga

PTC Therapeutics is acquiring Censa Pharmaceuticals in a cash and stock deal. PTC, based in South Plainfield, New Jersey, focuses on rare diseases. Censa, based in Wellesley, Massachusetts, is developing CNSA-001 for phenylketonuria and other metabolic diseases.

Under the terms of the deal, PTC is paying Censa $10 million in cash and up to 850,000 shares of PTC common stock, which as of Tuesday, May 5, was trading at $48 per share, totaling about $41 million. In addition, there are development and regulatory milestones for the two most advanced programs that can reach $217.5 million. There is also receipt of a priority review voucher and $30 million to be paid in either cash or PTC common stock after completing the enrollment of a Phase III clinical trial for CNSA-001, $109 million in development and regulatory milestones for each extra indication for CNSA-001, net sales milestones up to about $160 million and a contingent value payment of a percentage of annual net sales in the single to low double digits.

Results from a Phase II clinical trial of CNSA-001 demonstrated significant and clinically relevant reductions in phenylalanine levels compared to current first-line treatment, said Stuart W. Peltz, PTCs chief executive officer. We believe that CNSA-001 has the potential to address the majority of PKU patients whose condition is not adequately managed by current treatments. We look forward to initiating a Phase III study in PKU so that patients diagnosed with this devastating condition can have a new oral treatment option as soon as possible.

Phenylketonuria (PKU) is an inborn metabolic disorder that results in decreased metabolism of the amino acid phenylalanine. If untreated, it can cause intellectual disability, seizures, behavioral issues, and mental disorders. It is caused by a mutation in the gene that codes for the enzyme needed to metabolize phenylalanine. Patients with PKU must eat a diet that limits phenylalanine, found mostly in protein.

There are two drugs, both commercialized by BioMarin Pharmaceutical, approved for PKU. Sapropterin hydrochloride (Kuvan), an oral formulation, was approved in 2007 with a diet low in phenylalanine. In a Phase II trial, Censas drug was superior to Kuvan.

BioMarins Palynziq (pegvaliase-pqpz) was approved in 2018 by the U.S. Food and Drug Administration (FDA). It is an injectable enzyme therapy approved for adults with PKU whose blood phenylalanine concentrations remain high on current treatment. BioMarin planned to begin clinical testing of a gene therapy for the disease this year, but the COVID-19 pandemic has delayed the launch, although the company indicates it hopes to begin in the second half of 2020.

CNSA-001 is an oral formulation of synthetic sepiapterin, a precursor to intracellular tetrahydrobiopterin. Intracellular tetrahydrobiopterin is a critical enzymatic cofactor involved in the metabolism and synthesis of various metabolic products.

PTCs pipeline includes early research in gene therapy, nonsense mutation, splicing and oncology. The therapies are focused on Huntingtons disease, Angelman syndrome, relapsed/refractory acute leukemias, ovarian cancer and others. The company markets Emflaza, a treatment for Duchenne muscular dystrophy (DMD) in the U.S., and markets another DMD drug, Translarna, in Europe. It also markets Tegsedi for polyneuropathy of hereditary transthyretin-mediated amyloidosis in Latin America.

PTC, with Roche, is awaiting an approval by the FDA this year for risdiplam for spinal muscular atrophy (SMA).

I am proud of the team at Censa and its achievements to date demonstrating the potential role of CNSA-001 in treating diseases of the BH4 pathway, said Jonathan Reis, president and chief executive officer of Censa. It is the right time to have an excellent fully integrated, patient-focused biotechnology organization like PTC Therapeutics take over the late-stage development of CNSA-001 so that this promising compound becomes available to patients in the near future.

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PTC Therapeutics to Acquire Censa for $10 Million in Cash, $41 Million in Stock and More - BioSpace

SHANGHAI, China, May 10, 2020 (GLOBE NEWSWIRE) -- Burning Rock and Illumina (NASDAQ: ILMN), a global leader in gene sequencing and array-based technologies, jointly announced today that they are joining forces to promote the development and standardization of NGS-based cancer therapy selection in China based on Illuminas NextSeqTM 550Dx system.

In 2015, Illuminas sequencing technology and Burning Rocks development and commercial capabilities were for the first time combined, providing Chinas precision oncology market with advanced NGS-based cancer therapy selection solutions in the past five years. In 2020, as the first genetic testing company in China to achieve development of in vitro diagnostic (IVD) tests for both circulating tumor DNA (ctDNA) and tissue based on the NextSeqTM 550Dx system agreement with Illumina, Burning Rock will further expand and deepen the application of NGS technologies in the field of cancer therapy selection.

The value of NGS application in precision medicine and companion diagnostics has been widely recognized by clinical experts and cancer patients. Compared with traditional genetic testing methods, NGS-based cancer therapy selection allows patients to understand the mutation of multiple genes related to cancer treatment, providing doctors and patients with one-stop targeted therapy and immunotherapy drugs testing solutions, ultimately saving time and preserving samples.

In July 2018, Burning Rocks innovative product "Human EGFR / ALK / BRAF / KRAS gene mutation detection kit (reversible end termination sequencing method)" based on Illumina sequencing system became the first NGS-based reagent kit to be approved by National Medication Products Administration (NMPA). Since then, tumor NGS testing can be officially used in Chinese hospitals. In the future, Burning Rock will continue to seek NMPA approvals for its IVD products based on NextSeqTM 550Dx and other sequencing systems to promote the implementation of tumor NGS products in hospitals and benefit more cancer patients.

Through our long-term, close and pleasant cooperation with Illumina, we have delivered the world's leading NGS-based therapy selection solutions for clinical oncology, and improved the development and application of NGS-based cancer therapy selection in China. said Mr. Han Yusheng, founder and CEO of Burning Rock. Today we are delighted to announce that Burning Rock and Illumina will further deepen cooperation based on the NextSeqTM 550Dx system, to provide more high-quality molecular diagnostic solutions for clinical oncology treatment and promote the standardization of NGS-based cancer therapy selection in China.

Burning Rock is one of the leading precision oncology companies in China, said Joydeep Goswami, Senior Vice President of Corporate Development and Strategic Planning at Illumina, said. I am pleased to see that during the close cooperation with Illumina in the past five years, Burning Rock has continuously developed tumor diagnosis solutions that meet the needs of the local market. The new agreement is a testament to our strong cooperation in the past, and also the beginning of a more in-depth cooperation.

Li Qing, General Manager of Greater China at Illumina, said: Burning Rock has brought hope to countless Chinese patients by providing a series of tumor molecular diagnostic solutions. And we are very happy to be involved. In the future, I firmly believe that genetic testing technology will further change the current treatment paradigm for cancer and provide critical support to conquer this disease at an early date.

About Burning RockBurning Rock, whose mission is to Guard Life via Science, focuses on the application of next generation sequencing (NGS) technology in the field of precision oncology. Its business consists of i) NGS-based therapy selection testing for late-stage cancer patients, with the leading market share in China and over 185,000 tissue and liquid-based tests completed cumulatively, and ii) NGS-based cancer early detection, which has moved beyond proof-of-concept R&D into the clinical validation stage.

About IlluminaIllumina is improving human health by unlocking the power of the genome. Our focus on innovation has established us as the global leader in DNA sequencing and array-based technologies, serving customers in the research, clinical and applied markets. Our products are used for applications in the life sciences, oncology, reproductive health, agriculture and other emerging segments. To learn more, visit http://www.illumina.comand follow @illumina.

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Research report on Viral Vector Manufacturing Market size | Industry Segment by Applications, by Type, Regional Outlook, Market Demand, Latest Trends, Viral Vector Manufacturing Industry Share & Revenue by Manufacturers, Company Profiles, Growth Forecasts 2025. Analyzes current market size and upcoming 5 years growth of this industry.

Report Covers Global Industry Analysis, Size, Share, CAGR, Trends, Forecast And Business Opportunity.

Download Premium Sample Copy Of This Report: https://brandessenceresearch.biz/Request/Sample?ResearchPostId=473&RequestType=Sample

Global Viral Vector Manufacturing Market to reach USD 1.24 billion by 2025.

Global Viral Vector Manufacturing Market valued approximately USD 225.44 million in 2016 is forecasted to grow with a healthy growth rate of more than 20.84% over the forecast period 2018-2025. The major factors speculated to augment the markets are availability of funding for the advancement of gene therapy, and increasing frequency of cancer, genetic disorders & infectious diseases. The risk of undesirable outcomes including mutagenesis are major challenges for the global market. Viral vectors are tools commonly used by molecular biologists to deliver genetic material into cells. This process can be performed inside a living organism (in vivo) or in cell culture (in vitro). Molecular biologists first harnessed this machinery in the 1970s.Global Viral Vector Manufacturing Market is segmented based on Type, Disease, Application, and Industry. The Adeno-associated subsegment segment of Type segment is forecasted to grow with highest CAGR while the Cancers subsegment is expected to dominate in terms of market share. Gene Therapy subsegment is also expected to achieve highest growth rate whereas Biopharmaceutical & Pharmaceutical Companies subsegment would remain dominant in market share size.The regional analysis of Global Viral Vector Manufacturing Market is considered for the key regions such as Asia Pacific, North America, Europe, Latin America and Rest of the World. North America is the leading region across the world. Whereas, owing to rising no. of research activities in countries such as China, India, and Japan, Asia Pacific region is also expected to exhibit higher growth rate / CAGR over the forecast period 2018-2025. The objective of the study is to define market sizes of different segments & countries in recent years and to forecast the values to the coming eight years. The report is designed to incorporate both qualitative and quantitative aspects of the industry within each of the regions and countries involved in the study. Furthermore, the report also caters the detailed information about the crucial aspects such as driving factors & challenges which will define the future growth of the market. Additionally, the report shall also incorporate available opportunities in micro markets for stakeholders to invest along with the detailed analysis of competitive landscape and product offerings of key players. The detailed segments and sub-segment of the market are explained below:

By Type:

oAdenoviraloRetroviraloAdeno-associatedoOthers

By Disease:

oGenetic DisordersoInfectious diseasesoCancersoOthers

By Application:

oVaccinologyoGene Therapy

By End-Use:

oResearch InstitutesoBiopharmaceutical & Pharmaceutical CompaniesoOthers

By Regions:oNorth AmericaoU.S.oCanadaoEuropeoUKoGermanyoAsia PacificoChinaoIndiaoJapanoLatin AmericaoBraziloMexicooRest of the World

Furthermore, years considered for the study are as follows:

Historical year 2015, 2016Base year 2017Forecast period 2018 to 2025

The industry is seeming to be fairly competitive. Some of the leading market players CGT Catapult, Lonza, uniQure, Merck, Cobra Biologics, Oxford BioMedica, FUJIFILM Diosynth Biotechnologies, Novasep, Spark Therapeutics, Kaneka Eurogentec, Brammer Bio, Massbiologics, Finvector Vision Therapies, Regenxbio, Thermo Fisher Scientific, Inc., Sanofi, Shenzhen SiBiono GeneTech Co., Ltd., and so on. The fierce competitiveness has made these players spend in product developments to improve the customers requirements.

Target Audience of the Viral Vector Manufacturing Market Study:

oKey Consulting Companies & AdvisorsoLarge, medium-sized, and small enterprisesoVenture capitalistsoValue-Added Resellers (VARs)oThird-party knowledge providersoInvestment bankersoInvestors

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Table of Content:

Market Overview:The report begins with this section where product overview and highlights of product and application segments of the Global Viral Vector Manufacturing Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Competition by Company:Here, the competition in the Worldwide Global Viral Vector Manufacturing Market is analyzed, By price, revenue, sales, and market share by company, market rate, competitive situations Landscape, and latest trends, merger, expansion, acquisition, and market shares of top companies.

Company Profiles and Sales Data:As the name suggests, this section gives the sales data of key players of the Global Viral Vector Manufacturing Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the Global Viral Vector Manufacturing Market.

Market Status and Outlook by Region:In this section, the report discusses about gross margin, sales, revenue, production, market share, CAGR, and market size by region. Here, the Global Viral Vector Manufacturing Market is deeply analyzed on the basis of regions and countries such as North America, Europe, China, India, Japan, and the MEA.

Application or End User:This section of the research study shows how different end-user/application segments contribute to the Global Viral Vector Manufacturing Market.

Market Forecast:Here, the report offers a complete forecast of the Global Viral Vector Manufacturing Market by product, application, and region. It also offers global sales and revenue forecast for all years of the forecast period.

Research Findings and Conclusion:This is one of the last sections of the report where the findings of the analysts and the conclusion of the research study are provided.

About Us:

We publish market research reports & business insights produced by highly qualified and experienced industry analysts. Our research reports are available in a wide range of industry verticals including aviation, food & beverage, healthcare, ICT, Construction, Chemicals and lot more. Brand Essence Market Research report will be best fit for senior executives, business development managers, marketing managers, consultants, CEOs, CIOs, COOs, and Directors, governments, agencies, organizations and Ph.D. Students.

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COVID-19 pandemic Viral Vector Manufacturing Market Size Share Analysis and System Production (2020-2025) - Cole of Duty

For people with rare diseases, a single gene therapy treatment could restore normal function and alleviate the burden of ongoing care, as Dr Ian Winburn tells Kennas Fitzsimons

Dr Ian Winburn

Gene therapy is the next generation of medicine that targets the underlying cause of genetic diseases. It has the potential to offer patients a really transformational clinical benefit and improve quality of life.

Thats according to Dr Ian Winburn, Global Medical Lead, Haemophilia, Endocrine and Inborn Errors of Metabolism (IEM), Rare Diseases, Pfizer Biopharmaceuticals Group. Dr Ian Winburn, Global Medical Lead, Haemophilia, Endocrine and IEM, Pfizer Biopharmaceuticals Group, pictured right during his presentation on gene therapy at BioPharma Ambition held in Dublin Castle on March 4. Pic: Conor McCabe Photography.

Formerly a clinician in the UK National Health Service (NHS), Dr Winburn trained in general surgery and completed a PhD on novel drug discovery in renal transplantation before moving into industry 10 years ago, where he worked in the area of inflammation and immunology before leading the European haemophilia team.

Dr Winburn is now working to develop innovative gene therapies with the potential to restore normal function to patients with rare diseases, possibly with just a single treatment, changing the way people manage their disease.

Gene therapy: What is it?Gene therapy uses genes as medicine. It works by introducing functioning copies of missing or defective genes into the body and can target the underlying cause of a disease at the cellular level.

There are various types of gene therapies, such as the gene editing technique, CRISPR (clustered regularly interspaced short palindromic repeats), as well as epigenetic approaches that look at ways in which genes may be turned on or turned off.

Pfizer Rare Disease is focusing on an in-vivo approach that utilises a recombinant adeno-associated virus (AAV) to deliver the gene therapy.

This approach works by targeting the missing or non-functional gene in an individuals DNA and adding a copy of it with a functioning gene that, in turn, produces a functioning protein.

The functioning gene serves as a blueprint for the tissue to create the missing or non-functioning protein that is causing a disease.

Dr Winburn said: Gene therapy is in the branch of genetic medicine, where you can think about approaches that look to add a gene to a host cell, and that gene goes on and codes for a protein. That protein its coding for can replace a missing protein. So, in the example of haemophilia, where theres a missing factor VIII or factor IX clotting factor, that protein that is either missing or is faulty could be essentially administered through a gene therapy. A gene is added to a host cell that codes for the factor VIII or factor IX and therefore replaces that protein.

The functioning gene is delivered directly to the targeted cells by means of a highly specialised viral vector. This vector, effectively, is the package that contains the gene. In simple terms, it can be likened to the cardboard boxes that online retailers use to ship products.

The manufactured vectors are protein shells modelled after viruses in which all infectious viral components have been removed, and a functioning gene is added. Different viral vectors are used to reach specific tissues in the body, such as the liver or muscle.

VectorVector is a great word because vector describes a direction, by definition, and the other way we can think about vector is a vector often carries something. There are a few approaches you can use to develop a vector. We have embraced an AAV vector that has the capacity to deliver the transgene, the gene that is going to be added to the host somatic cell. In the case of haemophilia, it is targeted at the liver, Dr Winburn says.

Rare diseases focusAbout 280 million people worldwide live with a genetic disease, and more than 80 per cent of rare diseases are genetic in origin, according to Pfizer. For people born with rare diseases, the burden of disease management can be huge. Treatment is often ongoing and may be lifelong. Gene therapy could enable patients to live without the need for ongoing treatment. This raises the prospect of relief not only from symptoms but also from the burden of disease management.

Dr Winburn elaborates on the reasons why gene therapy approaches are currently focused specifically on rare diseases that have single-gene alterations.

It tends to be rare genetic diseases that are monogenic in nature Some of the more common diseases are very much multifactorial in origin: there may be a genetic component but there are other aspects to their aetiology rather than these single, monogenic conditions that gene therapy really lends itself to.

The other aspect is that these are areas of huge unmet medical need. Often, there isnt a high standard of care with either medicines or clinical interventions that are ultimately influencing the progression and the symptoms of the disease, he says.

A lot of rare diseases often affect children by the very nature of their being of genetic origin. In some cases, children dont get the opportunity to grow up into adulthood because of these rare diseases. Having the opportunity to develop medicines where there is such a high level of unmet need and, ultimately, impact in a positive way the lives of families and their carers is a huge motivation.

Dr Winburn adds that rare diseases, collectively, are common. There are approximately 7,000 rare diseases, and the majority of these are of genetic origin. Gene therapy offers a groundbreaking technology to address these genetic diseases that have historically not had particularly strong standards of care or clinical treatment paradigms offered to them.

Gene therapy for haemophiliaPeople with the genetic disorder of haemophilia have insufficient levels of a clotting factor that helps to stop bleeding. Consequently, they bleed for longer than other people. The disease is typically treated through infusions of the missing clotting factor, with patients undergoing regular replacement therapy. Gene therapy could revolutionise this treatment model.

Its really important to put yourself in the position of a parent who has a young child who has haemophilia, Dr Winburn says.

Often, this disorder of coagulation that results in spontaneous bleeding due to the lack of functioning clotting factor first presents as early as the age of two, classically when children are becoming toddlers, when they start bumping into things and they develop bruising and the likes.

That alerts their parents attention to the possibility that there is something wrong with their clotting system and they [undergo] clinical tests and a diagnosis is made. Or, because its a genetic condition, it may run in families and parents are aware of the possibility of their newborn having haemophilia.

But if you are diagnosed, for example, at the age of two, it means that the mainstay of treatment is factor replacement. So, that commonly is an intravenous infusion possibly two to three times a week, possibly once a week, or once every other week, depending on whether its haemophilia A or B and what type of medicine is being prescribed. But its certainly frequent treatments. Again, if you put that back to a parent wholl be doing those infusions from the age of two or three that lifelong need and burden is huge.

While factor replacement enables children to live a full and active life to a degree, children with haemophilia may not necessarily get the opportunity to engage in all the activities children typically partake in as they are growing up, such as contact sports, Dr Winburn says.

There is this ongoing, lifelong burden of treatment. As those boys transition into adulthood, they often take responsibility for that and if they dont get their treatment then they will bleed spontaneously into their joints, they get problems with haemarthropathy, causing damage.

Ultimately, the incidence of joint damage and joint replacement surgeries is incredibly significant in the haemophilia population. And that is often despite optimal prophylaxis, where its being prescribed.

So, when you think about gene therapy, this is a single, one-off treatment with the potential to alleviate the need for regular infusion for a patient.

Not all patients will be eligible for a gene therapy or are being studied in gene therapy trials. This is not going to be something thats available for everybody. But for those that are eligible, and ultimately in disease where a gene therapy has been licensed by the regulator, this really does have the potential to massively impact their lives and give them a sense of normality that they havent necessarily had up until that point in time.

Future expectationsWhile gene therapy holds promise for many people with genetic diseases, it will not be an appropriate solution for every patient. The potential risks and benefits of gene therapy will be fully established through clinical trial programmes and with continued research and evaluation.

Patient safety and suitability are always primary considerations in the development of new treatments as they progress from preclinical and clinical testing through regulatory approval to potential commercial distribution. Dr Winburn stresses that, as regards the development of new gene therapies, patient safety is paramount.

Safety is always at the forefront of our thinking, it is the heart of our clinical trial programmes, it is the heart of all our regulatory work.

It is an ongoing process around evaluating safety, and particularly long-term safety, and there is a critical importance for all patients that ultimately receive a gene therapy to be followed up long-term within registries, within clinical databases, so that we can monitor and evaluate long-term safety. All our trials are designed so that safety outcomes are critically part of it and its something that we are ever watchful of.

For some patients, gene therapy is already a reality. There are currently a few rare diseases for which gene therapies are available as therapeutic options in Europe, and Dr Winburn anticipates that there may be up to 30 approved by 2023.

There is a rare congenital cause of blindness that currently has a gene therapy available and similarly a rare neurological condition that affects children also has a gene therapy available, thats spinal muscular atrophy (SMA). There is also a gene therapy for beta thalassaemia that has recently been approved and is available within Europe. In terms of haemophilia, the first gene therapy is currently under review in Europe.

It may be premature to imagine a scenario whereby gene therapy is used to treat chronic diseases more generally, but in terms of future applications for these emerging technologies it is a case of watch this space.

This is an area where we are definitely in breakthrough technology. At this moment in time, our focus has been on rare diseases. There is, of course, an interest in understanding what is possible with gene therapy in terms of where it could be utilised, Dr Winburn says.

I dont want to provide any false hopes, but I think aspirationally, there is a hope that this could certainly impact many patients and their families in a positive way.

In association with Pfizer Biopharmaceuticals Group.

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Gene therapy: The 'next generation' of medicine - Irish Medical Times

publication date: May. 8, 2020

FDA has granted accelerated approval to Tabrecta (capmatinib) for adult patients with metastatic non-small cell lung cancer whose tumors have a mutation that leads to mesenchymal-epithelial transition exon 14 skipping as detected by an FDA-approved test.

Tabrecta is the first FDA-approved therapy to treat NSCLC with specific mutations (those that lead to mesenchymal-epithelial transition or MET exon 14 skipping).

Tabrecta is sponsored by Novartis.

FDA also approved the FoundationOne CDx assay (Foundation Medicine, Inc.) as a companion diagnostic for Tabrecta. Most patients had tumor samples that were tested for mutations that lead to MET exon 14 skipping using local tests and confirmed with the F1CDx, which is a next-generation sequencing based in vitro diagnostic device capable of detecting several mutations, including mutations that lead to MET exon 14 skipping.

Lung cancer is increasingly being divided into multiple subsets of molecularly defined populations with drugs being developed to target these specific groups, Richard Pazdur, director of the FDA Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDAs Center for Drug Evaluation and Research, said in a statement. Tabrecta is the first approval specifically for the treatment of patients with non-small cell lung cancer whose tumors have mutations that lead to MET exon 14 skipping. This patient population now has an option for a targeted therapy, which they didnt have prior to today.

Efficacy was demonstrated in the GEOMETRY mono-1 trial (NCT02414139), a multicenter, non-randomized, open-label, multicohort study enrolling 97 patients with metastatic NSCLC with confirmed MET exon 14 skipping. Patients received Tabrecta 400 mg orally twice daily until disease progression or unacceptable toxicity.

The main efficacy outcome measures were overall response rate (ORR) determined by a blinded independent review committee using RECIST 1.1 and response duration. Among the 28 treatment-nave patients, the ORR was 68% (95% CI: 48, 84) with a response duration of 12.6 months (95% CI: 5.5, 25.3). Among the 69 previously treated patients, the ORR was 41% (95% CI: 29, 53) with a response duration of 9.7 months (95% CI: 5.5, 13.0).

FDA approves daratumumab and hyaluronidase-fihj for multiple myeloma

FDA has approved daratumumab and hyaluronidase-fihj (Darzalex Faspro) for adult patients with newly diagnosed or relapsed/refractory multiple myeloma. This new product allows for subcutaneous dosing of daratumumab.

Darzalex Faspro is sponsored by Janssen Biotech Inc.

Daratumumab and hyaluronidase-fihj is approved for the following indications that intravenous daratumumab had previously received:

in combination with bortezomib, melphalan and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant,

in combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy,

in combination with bortezomib and dexamethasone in patients who have received at least one prior therapy,

as monotherapy, in patients who have received at least three prior lines of therapy including a proteasome inhibitor and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent.

Efficacy of daratumumab and hyaluronidase-fihji (monotherapy) was evaluated in COLUMBA (NCT03277105), an open-label non-inferiority trial randomizing 263 patients to daratumumab and hyaluronidase-fihj and 259 to intravenous daratumumab (daratumumab IV). The trials co-primary endpoints were overall response rate and pharmacokinetic endpoint of the maximum Ctrough on cycle 3, day 1 pre-dose. Daratumumab and hyaluronidase-fihj was non-inferior to daratumumab IV in evaluating these two endpoints.

The ORR was 41.1% for daratumumab and hyaluronidase-fihj and 37.1% for daratumumab IV with a risk ratio of 1.11 (95% CI: 0.89, 1.37). The geometric mean ratio comparing daratumumab and hyaluronidase-fihj to daratumumab IV for maximum Ctrough was 108% (90% CI: 96,122).

Efficacy of daratumumab and hyaluronidase-fihj in combination with VMP (D-VMP) was evaluated in a single-arm cohort of PLEIADES (NCT03412565), a multi-cohort, openlabel trial. Eligible patients were required to have newly diagnosed multiple myeloma and were ineligible for transplant. The major efficacy outcome measure, ORR, was 88.1% (95% CI: 77.8, 94.7).

Efficacy of daratumumab and hyaluronidase-fihj in combination with Rd (D-Rd) was evaluated in a single-arm cohort of this trial. Eligible patients had received at least one prior line of therapy. ORR was 90.8% (95% CI: 81.0, 96.5).

FDA accepts NDA for CC-486 in AML indication

FDA has accepted a New Drug Application for CC-486, an investigational oral hypomethylating agent, for the maintenance treatment of adult patients with acute myeloid leukemia who achieved complete remission, or CR with incomplete blood count recovery, following induction therapy with or without consolidation treatment, and who are not candidates for, or who choose not to proceed to, hematopoietic stem cell transplantation.

CC-486 is sponsored by Bristol Myers Squibb. FDA granted the application Priority Review and set a Prescription Drug User Fee Act goal date of Sept. 3, 2020.

The NDA submission was based on the efficacy and safety results of the phase III QUAZAR AML-001 study, which met the primary endpoint of improved overall survival for patients receiving AML maintenance treatment with CC-486 versus placebo.

Often, newly diagnosed adult patients with AML achieve a complete response with induction therapy, however many patients will relapse and experience a poor outcome. Patients in remission are seeking treatment options that decrease the likelihood of relapse and extend overall survival, Noah Berkowitz, senior vice president of Global Clinical Development, Hematology, at Bristol Myers Squibb, said in a statement.

CC-486 is an investigational therapy that is not approved for any use in any country.

Caris Life Sciences submits two PMA applications to FDA for whole exome and whole transcriptome sequencing

Caris Life Sciences has submitted two Pre-Market Approval applications for MI Exome CDx and MI Transcriptome CDx to FDA.

MI Exome CDx, whole exome sequencing (DNA), and MI Transcriptome CDx, whole transcriptome sequencing (RNA), are precision medicine assays that include key companion diagnostic biomarkers with therapy claims, and detect all classes of alterations including genomic signatures for microsatellite instability, tumor mutation burden, and loss of heterozygosity.

MI Exome CDx is a next-generation sequencing-based test utilizing DNA isolated from formalin-fixed paraffin embedded tumor tissue specimens for the qualitative detection of genomic alterations. MI Exome CDx can identify genetic variants (single nucleotide variants, insertions and deletions), copy number alterations, MSI, TMB and LOH.

MI Transcriptome CDx is a next-generation sequencing-based test that utilizes RNA isolated from formalin-fixed paraffin embedded tumor tissue specimens for the qualitative detection of genomic and transcriptomic alterations. MI Transcriptome CDx is a broad, multi-gene panel utilized to identify gene fusions, transcript variants, genetic variants (single nucleotide variants, insertions and deletions), and gene expression changes. FDA granted MI Transcriptome CDx received Breakthrough Device designation in 2019.

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FDA approves Tabrecta, first targeted therapy to treat metastatic NSCLC - The Cancer Letter

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Orchard Therapeutics(NASDAQ:ORTX)Q12020 Earnings CallMay 9, 2020, 8:30 p.m. ET

Operator

Ladies and gentlemen, thank you for standing by, and welcome to the Orchard Therapeutics First Quarter 2020 Investor Conference Call. [Operator Instructions]

I would now like to hand off the conference over to your speaker today, Renee Leck, Director of Investor Relations. Please go ahead, ma'am.

Renee T. Leck -- Director, Investor Relations

Thanks, Sonia. Good morning, everyone, and welcome to Orchard's First Quarter 2020 Investor Call. You can access the slides for today's call by going to the Investors section of our website, orchardtx.com.

Before we get started, I'd like to remind everyone that statements we make on this call will include forward-looking statements. Actual events and results could differ materially from those expressed or implied by any forward-looking statements as a result of various risk factors and uncertainties, and including those set forth in our annual 10-K filed with the SEC and any other filings we may make. In addition, any forward-looking statements made on this call represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update or revise any forward-looking statements.

And with that, I'll turn the call over to our CEO, Bobby Gaspar.

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Thanks, Renee. Hello, everyone, and welcome. I'd like to start by first acknowledging the tremendous efforts of our organization and our partners in the healthcare field to ensure patients in need continue to receive care during this difficult time. Thank you, everyone. The last few weeks have been an important period for Orchard. Since taking on the leadership, Frank and I, together with the executive team, have thought very carefully about what the new Orchard can become, how we can ensure that Orchard can fulfill its true potential and what we need to do to make that happen.

When we think about our strategic vision as a company, it's really all based on the potential of the hematopoietic stem cell gene therapy platform, where it can take us and the benefit it can provide for many patient populations even beyond our current portfolio of ultra-rare diseases. We have taken some bold and decisive actions that we believe will allow Orchard to achieve long-term growth and focus the company on sustainable value creation. This vision is supported by a new strategic plan that we have developed and which is built around four pillars. Each of these forms a chapter in our remarks this morning.

First, operating efficiencies. We have made a series of important changes to our operations that will enable us to sharpen our focus and more efficiently execute our strategy, which I will detail in a moment. Second is our commercial build. We are focused on establishing the right model for the diagnosis and treatment of patients undergoing HSC gene therapy, and see the true value of this approach over a series of ultra-rare products. Third, one of the most exciting areas in gene therapy right now is the innovation taking place in manufacturing technologies that have the potential to deliver economies of scale. We want to be leaders and invest in this space, knowing that our near-term capacity needs are covered by our experienced CDMO network.

Finally, central to this strategy is prioritizing our portfolio to enable the expansion of Orchard's pipeline beyond ultra-rare to less-rare indications. We are disclosing two new research programs for the first time today, and these are a genetic subset of frontotemporal dementia or FTD, and a genetic subset of Crohn's disease. We believe that the biological and clinical validation that has already been shown in our ultra-rare indications allow us to expand with confidence to these larger indications.

Turning to the first chapter in our new strategic plan. We are focused on improving the operational efficiency throughout the organization. This started with an extensive evaluation over the past six weeks of each program in our portfolio using several criteria that are shown here on the left-hand side of slide five. We undertook an objective analysis that involved both financial metrics and strategic considerations in identifying those programs where there was high need for patients and high-value creation for shareholders. As you can imagine, these were difficult decisions given the potentially transformative nature of many of these programs. Each has value, and we intend to realize that in different ways and over different time horizons.

Today, however, we believe our resources are best focused on Metachromatic Leukodystrophy, Wiskott-Aldrich syndrome, the MPS programs and our research programs. This also means that we have a balanced portfolio with late, mid and early stage programs. The programs I haven't mentioned such as OTL-101 and ADA-SCID and the transfusion-dependent, beta-thalassemia program, OTL-300, will have a reduced investment moving forward. We will look for alternative ways to realize value with those programs, including through partnerships.

So slide six brings together a summary of the operational changes that we've announced today. We believe these changes were important and necessary to enable Orchard to execute its mission and objectives at the highest level by matching our attention and resources to a set of core imperatives for the business. As summarized here, we expect to realize cash savings of approximately $15 million from the prioritization of our portfolio. Another $60 million in savings results from the decision to consolidate our R&D teams to one site and defer the investment in the manufacturing facility. Finally, the more staged approach to the commercial build-out and 25% reduction to our existing workforce and future headcount planning will each yield another $25 million in savings.

All of these cash savings are expected to be realized over 2020 and 2021, and result in total expected savings of $125 million over that period. With the revised plan, we now have cash runway into 2022 and no near term need to finance. It's worth briefly mentioning that this $125 million savings is after making investments in the following key areas to support our new strategy, shown on slide seven. In commercial, diagnostic and screening initiatives, including no-charge testing programs to help identify patients with MLD and other neurodegenerative conditions in time for treatment. In manufacturing, the technology, process innovations and efficiencies to drive scalability.

In R&D, initiatives in less-rare diseases that have the potential to fuel the company's future growth in a substantial way. This wasn't just an exercise to reduce expenses, but important decision-making to ensure our capital is deployed in a disciplined manner, while building a pipeline that can leverage our success across all phases of our business.

Now let me turn the call over to Frank to discuss additional key elements of the new plan.

Frank Thomas -- President and Chief Operating Officer

Thanks, Bobby, and good morning, everyone. As you can tell from this morning's press release, we have carefully examined each aspect of our business. You heard that a moment ago from Bobby, with the way we are creating operational efficiencies, and I think you will see additional evidence in the next two sections as we summarize our latest thinking around commercial deployment and manufacturing. Starting with commercial. We understand the importance of developing a commercial model that will demonstrate our ability to execute and bring these therapies to the market successfully. This model and the infrastructure that we build will also be leveraged for any future product launches.

As you'll note from the bottom of slide nine, each rare disease has certain dynamics that will impact the launch trajectory and speed with which we can penetrate the market. In fact, we anticipate our first two potential launches in WAS and MLD having distinct but complementary launch curves, as you can see from the illustrative diagrams. Let me start with MLD on the left, where we expect to launch first in the EU, followed by the U.S. and then other countries around the world. We think an important inflection point on the revenue curve with MLD will come later when newborn screening is established, providing an opportunity for an acceleration in growth rate. Disease progression is a second important dynamic that will affect market penetration. Because MLD advances so rapidly, it will be important to diagnose patients early and get them treated.

For Wiskott-Aldrich syndrome, the dynamics are very different, and it's reflected in the shape of the curve on the right. Unlike MLD, this disease is slower progressing and more readily diagnosed. We believe that WAS will provide an opportunity to treat a number of prevalent patients from the outset and also give us additional long-term revenue stream. This program, the BLA and MAA filings are on track for 2021. Turning back to MLD for an update on the regulatory time line. We are on track to get a decision from the European Medicines Agency later this year, and if approved, launch in the EU in the first half of 2021.

In the U.S., we recently engaged with the FDA on our planned BLA submission of OTL-200 for the treatment of MLD. The FDA has provided written feedback on the sufficiency of the company's data package, including the clinical endpoint, the natural history comparator and the CMC data package. As a result of this feedback, we intend to file an IND later this year and also seek RMAT designation, both of which we believe will facilitate a more comprehensive dialogue to discuss the data more fully and resolve the open matters before submitting a BLA. We are committed to working closely with the agency, and we'll provide updated guidance on the new filing time lines for the BLA after further regulatory interaction.

On slide 10, you can see that we're tracking nicely for the launch of OTL-200 in the EU in the first half of 2021, if approved, with Germany being the first country where we expect to treat commercial patients. Many of the prelaunch activities are under way, and the team has been able to keep up momentum during the pandemic to work with key centers and progress with site qualifications. We intend to set up a network of treatment centers where MLD patients are often referred and who also have transplant expertise. These same centers can be leveraged in future launches, especially for programs in the neurometabolic franchise.

I previously mentioned the importance of diagnosis in MLD to identify patients at early stages of disease, and we are taking the necessary steps to achieve long-term success. Beyond typical disease awareness efforts, we are also looking at initiatives such as no-charge diagnostic testing with partners such as Invitae, and we are looking to facilitate newborn screening for MLD with funding of upcoming pilots in New York State and Italy that are designed to validate the assay and provide the data for wider implementation. Success in these key initiatives will support early MLD patient identification.

Coming up quickly behind MLD and the neurometabolic franchise, our two proof-of-concept programs in MPS disorders, where we have made recent progress even during this challenging period with COVID-19. For MPS-I, over the past year, we've shown promising preliminary proof-of-concept data with positive engraftment, biomarker correction and encouraging early clinical outcomes, and we are excited to announce our plan to begin a registrational trial next year, bringing this program one step closer to commercialization.

For MPS-IIIA, we announced late last month that the first patient was treated in a proof-of-concept trial at Royal Manchester Children's Hospital, with enrollment planned to continue this year and interim data to be released in 2021. You can see graphically on slide 12 how the aggregation of these commercial markets lead to sustainable revenue growth. In addition, the infrastructure build is designed to provide the necessary commercial capabilities to realize the potential of the portfolio. On this slide, we've included the incidence figures for MLD and the incidence and prevalence figures for WAS to help you understand each opportunity as we see it today.

Given the dynamics at play for MLD that I described on slide nine, we believe this opportunity should largely be tied to the incident patient population, which we believe ranges from 200 to 600 patients per year in countries where rare diseases are often reimbursed. We've taken a more conservative view than previously on the addressable patient and market opportunity in countries such as those in the Middle East and Turkey, where the literature has a wide range of differing incident figures. Also, over time, with improved disease awareness, there may be prevalent patients identified who also could benefit from therapy. Our commercial strategy has always been and continues to be based not only on one product, but rather the aggregation of multiple potential products launching off one HSC gene therapy platform and infrastructure.

Turning to manufacturing. We've also made some key changes to our approach in manufacturing and how we allocate capital in the short and mid-term. On slide 14, you'll see the main tenets of our new manufacturing strategy. First, in the near term, we plan to focus on innovative technologies to enable commercial scalability.

Second, to ensure the appropriate focus on those technologies, we've made a decision to consolidate R&D to a single site in London, which brings together our organization in a more efficient way. This will allow efforts made to improve our manufacturing processes to be quickly and easily shared and then scaled commercially to transfer to our third party manufacturers, all of whom are currently located in Europe. As part of this consolidation, we will close our California site, including the termination of the Fremont project and associated capital spend.

Third, we have strong relationships with CDMOs that will ensure supply of clinical and commercial product to satisfy near-term requirements. And longer term, we intend to identify a new site in the U.S. to eventually bring manufacturing capabilities in-house with a facility that is appropriately sized and fitted for future techniques and operations.

Slide 15 shows the three phases of our approach in manufacturing: invest, partner and build. Today, we are investing, and we'll continue to invest in technologies such as transduction enhancers, stable producer cell line and closed automated processing of the drug product. This will potentially reduce the amount of vector needed, drive down COGS and potentially change the way products are manufactured, making it less labor-intensive, less expensive and more consistent. In the near and mid-term, we will continue to rely on our manufacturing partners for the early planned launches in MLD and WAS. For example, MolMed has been with these programs since the beginning, and they've been a reliable commercial partner with Strimvelis.

In addition to our existing CDMO network, we have begun to search for a drug product partner in the U.S. to complete a tech transfer and serve the U.S. market, thereby reducing scheduling challenges and creating some redundancy. And finally, over time, we plan to build in-house manufacturing capabilities closer to when there is a need for additional capacity. This enables us to explore options that are more aligned with our business in terms of scale and timing.

And with that, I'll turn the call back over to Bobby.

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Thanks, Frank. In this section, I'm going to briefly highlight the potential of HSC gene therapy to correct not only blood lineage cells, but also how through natural mechanisms, specific cell types may allow correction of disease in specific organ systems and enable expansion of our portfolio into new research indications. As many of you know, and as shown on slide 17, through HSC gene therapy, we are able to insert a working copy of the gene permanently into the genome of HSCs, and these genetically modified cells can lead to multiple corrected cell types in the bloodstream, including immune cells, red blood cells and platelets.

In addition, HSCs can differentiate into cells of the monocyte macrophage lineage that naturally migrate into various organ systems, and thus gives us an opportunity to deliver genes and proteins directly to those organs, including the brain and the GI tract. Within the neurometabolic space, in particular, we have understood through our preclinical and clinical programs in MLD, MPS-I and MPS-IIIA how HSC gene therapy can deliver genes and proteins to the CNS to correct neurodegeneration. Here is an example of this natural mechanism at work in slide 18.

Data shows that there are a population of gene-modified HSCs that can naturally cross the blood-brain barrier, distribute throughout the brain, engraft as microglia and express enzyme that is taken up by neurons. We have seen this approach results in clinical benefits for patients with MLD, and we are also using the same approach for MPS-I and MPS-IIIA. Beyond this, we see that the HSC gene therapy approach could be used to deliver specific genes and proteins for other larger neurodegenerative conditions which have high unmet need.

One of the conditions we are disclosing today, and shown on slide 19, is a specific genetic subset of frontotemporal dementia, where the underlying pathogenesis has a number of parallels with the neurometabolic conditions that we are already addressing. This program involves a broad strategic alliance with Dr. Alessandra Biffi, Boston Children's Hospital and Padua University in Italy, to further explore the potential of ex vivo HSC gene therapy in neurometabolic and neurodegenerative conditions. In other organ systems, such as the GI tract, there are similar mechanisms at work which are illustrated on slide 20. Tissue resident macrophages in the gut wall are required to respond to bacterial invasion from the gut lumen and prevent infection. In certain disorders, such as X-linked chronic granulomatous disease or XCGD, defects in macrophage function results in an abnormal immune response and severe colitis.

Moving on to slide 21. We have already seen in our XCGD program the modification of HSCs and migration of gene-modified cells into the gut can lead to resolution of colitis through presumed reconstitution of the immune response. Certain subsets of Crohn's disease are also associated with mutations in genes that affect the response of macrophages to infection, and so our clinical observations that HSC gene therapy for XCGD suggest that the same approach may be applicable to this genetic subset of Crohn's disease. This preclinical work is ongoing in our Orchard research laboratories.

As we advance our work in FTD and Crohn's disease, and assuming we show preclinical proof-of-concept, these will become exciting opportunities for us to expand and address larger patient populations, either alone or in partnership. We believe we have truly just begun to explore the potential for HSC gene therapy in diseases such as these and others, and are excited to share more about the preclinical development of these programs later this year.

So to summarize our path forward on slide 22, the next 12 to 18 months offers many important milestones as we continue our evolution to a commercial stage company and advance our next wave of clinical stage therapies. We anticipate approval and launch of OTL-200 for MLD in the EU, additional regulatory filings in Wiskott-Aldrich syndrome and MLD, a new registrational study next year in MPS-I, multiple clinical data readouts from our neurometabolic franchise and further detail and progress on our research programs in FTD and Crohn's disease.

To wrap up our prepared remarks, we are confident that our new strategic plan and operational decisions announced today will set us on the right path to achieve long-term growth, build sustainable value and serve an even larger number of patients who could benefit from hematopoietic stem cell gene therapy.

Thank you very much. And now we'll use the rest of the time to answer your questions. So let's have the operator open up the line.

Operator

Thank you. [Operator Instructions] And our first question comes from Whitney Ijem from Guggenheim. Your line is now open. Please go ahead.

Whitney Ijem -- Guggenheim -- Analyst

Hey guys, thanks for the question. So first, just wondering, can you give us some more color maybe on the discussions you're having with the FDA in MLD? Kind of what are they looking for? And I guess is the IND just sort of a tool to get RMAT? Or is there additional kind of clinical work you plan you think you'll need to do?

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Hi. Whitney, Bobby here. Thanks for that. In general, we can't go into all of the details, obviously, of the discussions with the FDA. But I think in the release and in the script, we've talked about the fact that they've commented on certain endpoints, the natural history, the CMC package, etc. Now I think I'd just like to say this is a and obviously, a very complex disease, a very ultra-rare population, we have extensive data set, and we have already filed with the EMA. Now for historical reasons, there hasn't been an IND in the U.S., and so we haven't had the opportunity to discuss that data in full with the FDA.

What I can say is that we do have an extensive body of data. We want to be able to talk to the FDA and have a comprehensive dialogue to be able to explain that full data set. We feel confident that we have the endpoints that they are looking for and the data that they are asking for. But we need to have that conversation with them in order to be explain to be able to do that fully. So that's why we're filing an IND filing, filing the RMAT, so we can have that dialogue. And once we can clarify those issues, then we can go ahead with submission of the BLA.

Whitney Ijem -- Guggenheim -- Analyst

Okay. Got it. And then just one quick follow-up on MLD. Can you remind us where you are with newborn screening, I guess, both in Europe and then in the U.S.?

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Yes, sure. So newborn screening for MLD, I think, is an important, a very important issue, because, obviously, that means that we'll be able to get earlier diagnosis and have more patients be able to access therapy. So it's a very important part of our kind of diagnostic initiatives in this disease. What we have so far is that we have worked with a key scientist, where an assay has been developed, that's been published to show that there is an assay that we've done on a dry blood spot to understand the decrease in the enzyme activity and also the increase in the sulfur-type levels.

And that assay is now going to be put into pilots, and we are funding a pilot in New York State, and that will start later this year. And we're also looking at pilots in other states as well. We're also transferring that assay to Italy and that and we're funding a program in Tuscany and in Italy where that will be rolled out. And we're also looking for opportunities in other EU states as well. So I'd say, there are already two that are going to start, we are looking to fund other pilots as well.

And together, that data will allow us to validate the assay but also allow wider implementation of newborn screening, and also for nomination, for example, onto the WAS panel for implementation in states in the U.S. So I say there's a lot of work going on in order to make sure that happens.

Whitney Ijem -- Guggenheim -- Analyst

Great, thanks.

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Thank you.

Operator

And your next question comes from Esther Rajavelu from Oppenheimer. Your line is now open. Please go ahead.

Esther Rajavelu -- Oppenheimer -- Analyst

Hey guys. Congrats on all the changes. I guess, my first question again on MLD is I'm trying to understand the duration between EU approval and NBS. I don't know if that math or if that graph was drawn to scale, but it looks like it's almost a four-year lag from first approval to newborn screening. Can you help us understand the time line there?

Frank Thomas -- President and Chief Operating Officer

You mean between EU and U.S. or around newborn screening or both?

Esther Rajavelu -- Oppenheimer -- Analyst

Around newborn screening, generally, between EU approval and newborn screening.

Frank Thomas -- President and Chief Operating Officer

Yes, sure. As Bobby mentioned, there's a pretty active program planned around newborn screening that I think we will expect will come over time in order to even apply for the Ross Panel, there are certain requirements that need to be met in terms of the number of patients or a number of children that have to be screened, identifying the positive patients and then you can apply on the Ross Panel. And then from there, there's a process that you go through in the U.S., at least, on a state-by-state basis to get it added.

So I think there are a number of steps along the way. We haven't guided specifically on the time line, but I think there are other precedents out there that suggest that this could take years. Once we screen the once we apply for the Ross Panel to get sort of full reimbursement, but obviously, we'll focus on states initially after that approval that have the largest populations.

Esther Rajavelu -- Oppenheimer -- Analyst

And my Yes, go ahead.

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Esther for I was just going to say for the EU, obviously, we're looking for approval for MLD later this year. As far as people screening in the EU is concerned, that's on a country-by-country basis, and sometimes it's even certain states. But I've worked on newborn screening for SCID, for example, in the EU. And now there are numerous countries in the EU that are screening for SCID with a number of pilots also in the pipeline as well. And so with that kind of experience, and we would be looking to kind of really facilitate that uptake in the EU and as in and in the U.S., as Frank has already mentioned.

Esther Rajavelu -- Oppenheimer -- Analyst

Understood. And then the decision to defer capex, is that related to some of the time lines for U.S. versus EU approvals and the newborn screening? Or what really kind of went into that delay, given you already have some cost into that facility?

Frank Thomas -- President and Chief Operating Officer

Yes. I can start, and Bobby can add on that again. I think, obviously, we continue to believe in-house manufacturing is an important capability that we're going to want to have over some period of time. It really comes down to sort of when is the need for that capacity and capability relative to the various programs we have. Working with the CDMOs that we have today, we know that we have capacity for the MLD and WAS launches and for a period beyond the launch. So there's not an imminent need to secure the capacity today, and we think that deferring it makes the most sense. We'll continue to work with CDMOs on those launches.

We will look at bringing on a U.S. supplier for drug product to be able to more easily service the U.S. market. And then longer term, look at, potentially, in-house manufacturing at a site and location that we think is more fitted to what the capacity needs will be. So I wouldn't say it's tied to any sort of launch time lines because the plan always was to utilize CDMOs for WAS and MLD. But certainly, as those launches roll out and demand grows, our capacity needs will grow and that will be the appropriate time, we think, to make the investment.

Esther Rajavelu -- Oppenheimer -- Analyst

Understood. Thank you very much.

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Thank you.

Operator

And your next question comes from Anupam Rama from JPMorgan. Your line is now open. Please go ahead.

Tessa Romero -- JPMorgan -- Analyst

Good morning, guys. This is Tessa on the call this morning for Anupam. You pointed out that updated interim data from the proof-of-concept trial for OTL-203 and MPS-I is expected at ASGCT upcoming here next week. Can you remind us of what will be the size and scope of data that we will see at the conference? And maybe can you just clarify if there is any other newly updated clinical data we should be thinking about for other programs at ASGCT?

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Okay, fine. Bobby here, and I'll take this question. On MPS-I, so just to remind you, the proof-of-concept study has enrolled all eight patients, so that's been fully recruited into. What we've shared with you previously is biochemical data showing the overexpression of IDUA activity, the decrease in the heparan and dermatan sulfate, the engraftment of gene-modified cells and some early clinical data on patients who have got beyond the one-year time frame after gene therapy. There was only previously one patient who had reached that time point.

So there's been further follow-up on those eight patients. We'll be able to show you longer-term engraftment of the gene-modified cells, more consistent overexpression of enzymatic activity, longer follow-up, decrease in GAG levels and also more clinical data on patients who have got to longer endpoints as well. So we'll be able to show data assay on clinical data on patients after longer follow-up. And this will be both on their cognitive outcome, but we also will have data on, for example, growth parameters as well, which is again a big issue in MPS-I. So that is for MPS-I.

We will also be sharing data on OTL-101 as well for ADA-SCID. There will be further follow-up on patients who have undergone treatment for transfusion-dependent beta-thalassemia, so longer follow-up on the patients who have been treated so far. So there's really quite, as well as other programs. So there's really quite an extensive body of data, and it just showcases the potential of Orchard's platform across a number of different diseases and how HSC gene therapy can correct the underlying defects in immune deficiencies, neurometabolic deficiencies and hemoglobin opportunities as well. And obviously, we'll give you more detail on those different abstracts next week.

Tessa Romero -- JPMorgan -- Analyst

Great, thank you.

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Thank you.

Operator

Read more:
Orchard Therapeutics (ORTX) Q1 2020 Earnings Call Transcript - Motley Fool

ENGLEWOOD, CO / ACCESSWIRE / May 7, 2020 / Aytu BioScience, Inc. (AYTU) (the "Company"), a specialty pharmaceutical company focused on commercializing novel products that address significant patient needs, announced today that the Company will present its operational results for the quarter ended March 31, 2020 on May 14, 2020, at 4:30 p.m. ET. The Company will review accomplishments from the quarter and provide an overview of its business and growth strategy.

Conference Call Information

1- 877-407-9124 (toll-free)1- 201-689-8584 (international)

The webcast will be accessible live and archived on Aytu BioScience's website, within the Investors section under Events & Presentations, at aytubio.com, for 90 days.

A replay of the call will be available for fourteen days. Access the replay by calling 1-877-481-4010 (toll-free) or 919-882-2331 (international) and using the replay access code 34718.

About Aytu BioScience, Inc.

Aytu BioScience, Inc. is a commercial-stage specialty pharmaceutical company focused on commercializing novel products that address significant patient needs. The Company currently markets a portfolio of prescription products addressing large primary care and pediatric markets. The primary care portfolio includes (i) Natesto, the only FDA-approved nasal formulation of testosterone for men with hypogonadism (low testosterone, or "Low T"), (ii) ZolpiMist, the only FDA-approved oral spray prescription sleep aid, and (iii) Tuzistra XR, the only FDA-approved 12-hour codeine-based antitussive syrup. The pediatric portfolio includes (i) AcipHex Sprinkle, a granule formulation of rabeprazole sodium, a commonly prescribed proton pump inhibitor; (ii) Cefaclor, a second-generation cephalosporin antibiotic suspension; (iii) Karbinal ER, an extended-release carbinoxamine (antihistamine) suspension indicated to treat numerous allergic conditions; and (iv) Poly-Vi-Flor and Tri-Vi-Flor, two complementary prescription fluoride-based supplement product lines containing combinations of fluoride and vitamins in various for infants and children with fluoride deficiency. Aytu recently acquired exclusive U.S. distribution rights to the COVID-19 IgG/IgM Rapid Test. This coronavirus test is a solid phase immunochromatographic assay used in the rapid, qualitative and differential detection of IgG and IgM antibodies to the 2019 Novel Coronavirus in human whole blood, serum or plasma.

Aytu recently acquired Innovus Pharmaceuticals, a specialty pharmaceutical company commercializing, licensing and developing safe and effective consumer healthcare products designed to improve men's and women's health and vitality. Innovus commercializes over thirty-five consumer health products competing in large healthcare categories including diabetes, men's health, sexual wellness and respiratory health. The Innovus product portfolio is commercialized through direct-to-consumer marketing channels utilizing the Company's proprietary Beyond Human marketing and sales platform.

Aytu's strategy is to continue building its portfolio of revenue-generating products, leveraging its focused commercial team and expertise to build leading brands within large therapeutic markets. For more information visit aytubio.com and visit innovuspharma.com to learn about the Company's consumer healthcare products.

Contact for Media and Investors:

James CarbonaraHayden IR(646) 755-7412james@haydenir.com

SOURCE: Aytu BioScience, Inc.

View source version on accesswire.com: https://www.accesswire.com/588844/Aytu-BioScience-to-Report-Fiscal-Q3-FY-2020-Results-and-Business-Update

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Aytu BioScience to Report Fiscal Q3 FY 2020 Results and Business Update - Yahoo Finance

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A 7-year-old boy in Marylandwho suffers from sickle cell anemiais on his way to a full recovery after being hospitalized with a case of the coronavirus, according to reports.

Nasir Striggs was first hospitalized at Sinai Hospital in Baltimore in early April. His mother, Deshannon Striggs, brought him in for an examination after she noticed her son was experiencing trouble breathing.

He tested positive for COVID-19 at the hospital. An X-ray revealed he also had pneumonia in both lungs. The child, diagnosed with sickle cell disease, an inherited red blood cell disorder, underwent several blood transfusions at the hospital before his release.

CLICK HERE FOR FULL CORONAVIRUS COVERAGE

Nasir Striggs, 7, who has sickle cell anemia, is home from the hospital after recovering from the coronavirus. (Courtesy: Deshannon Striggs)

He had to keep getting stuck by the needle because the needle kept coming out, the mother told WBAL. To watch him go through that, it was really scary.

After undergoing treatment for several days, his condition began to improve, Deshannon said. She said prayers and support, as well as the dedicated care from the hospitals medical team, have helped her sons recovery.

Just keep the faith. Thats the message: keep the faith, she said.

CLICK HERE TO GET THE FOX NEWS APP

Deshannon said doctors have been monitoringNasirs conditionvia virtual check-ups since he was discharged from the hospital. Photos she shared with Fox News show the boy at home smiling,his face mask pulled beneath his chin.

Sickle cell disease is usually diagnosed shortly after birth. The genetic disorder results in oxygen-carrying red blood cells taking on a C or sickle shape, instead of round,often getting stuck in small blood vessels and clogging blood flow, according to the Centers for Disease Control and Prevention.

Children with the disease are at an increased risk of infection and other health problems. The only known cure is a bone marrow or stem cell transplant.

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Maryland boy, 7, with sickle cell disease recovers from coronavirus that caused pneumonia in both lungs - News Info Park

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Protagonist Therapeutics, Inc. Common Stock(NASDAQ:PTGX)Q12020 Earnings CallMay 07, 2020, 5:00 p.m. ET

Operator

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.

Don Kalkofen -- Chief Financial Officer

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 Patel -- President and Chief Executive Officer

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 No. 3. As you are well aware, Protagonist started the year 2020 with three clinical assets, all of which have been discovered through the use of our peptide technology platform in six different clinical proof-of-concept studies. The three assets fall in two 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 toward 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 three 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 toward 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 six months through the middle of 2022. Now let's go to Slide No. 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 six out of six, 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 eight 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 seven 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?

Sam Saks -- Chief Medical Officer

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?

Ronald Hoffman -- M.D., Director of the Myeloproliferative Diseases Program

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 three 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, 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 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 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 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 three or four-month intervals. At that three or four-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 with phlebotomy alone. Let's go now on to Slide No. 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 three phases. Part one is the dose-finding period, which is over 28 days. And there are two components to that.

I believe 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 two 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 No. 12. This is essentially the responses that we've seen in the seven 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 three 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 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 two 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 seven 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 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 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 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.

Dinesh Patel -- President and Chief Executive Officer

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 four clinical programs with our three 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 six 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?

Operator

[Operator instructions] Our first question comes from the line of Chris from Nomura. Your line is now open.

Chris Marai -- Nomura Instinet -- Analyst

Hi. 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? Thank you.

Ronald Hoffman -- M.D., Director of the Myeloproliferative Diseases Program

OK. I think that's a really terrific question. I think its use would be quite wide. I think virtually all patients with PV would be candidates for this drug.

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 three 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.

Chris Marai -- Nomura Instinet -- Analyst

OK. 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? Thank you.

Ronald Hoffman -- M.D., Director of the Myeloproliferative Diseases Program

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.

Chris Marai -- Nomura Instinet -- Analyst

Very helpful. Thank you.

Operator

Our next question comes from the line of George Farmer of BMO. Your line is now open.

Gobind Singh -- BMO Capital Markets -- Analyst

Hi, everyone. This is Gobind Singh on for George. Congrats on making a decision and moving forward with those two indications. I guess we had two buckets of questions.

The first two, maybe for Dinesh. I know you guys were thinking about once-weekly dosing and possibly twice weekly dosing in beta-thal. I was wondering if maybe going forward, at least with PV, do you think you might be looking at twice weekly dosing at all? And I was wondering since we are moving past beta-thal and MDS, if you'd be willing to comment at all as to what kind of data we're seeing in those indications? And I'll have a follow-up for Dr. Hoffman, if that's OK.

Dinesh Patel -- President and Chief Executive Officer

Yes. Gobind, thanks for the question. And I will have Sam Saks, our CMO, answer the first question.

Sam Saks -- Chief Medical Officer

Sure. So with respect to the use of 300, we think that it's going to be used widely. You were asking me...

Dinesh Patel -- President and Chief Executive Officer

About the once-weekly.

See the rest here:
Protagonist Therapeutics, Inc. Common Stock (PTGX) Q1 2020 Earnings Call Transcript - The Motley Fool

New York City-based home-lab-test startup LetsGetChecked said this morning that it has wrapped up an oversubscribed $71 million Series C round co-led by Illumina Ventures and HLM Venture Partners.

The raise also included new backers Deerfield, CommonFund Capital and Angeles Investments, while investors Transformation Capital, Optum Ventures and Qiming Venture Partners USA returned to further fuel the startup's business. With this, Illumina's Nick Naclerio, founding partner, and HLM's Steven Tolle, partner, will be joining LetsGetChecked's board as members.

WHAT IT DOES

Founded in 2014, LetsGetChecked provides consumer customers with a range of at-home testing kits, such as ones for hormone levels, sexually transmitted infections, liver performance, nutritionand specifically for frontline healthcare workers COVID-19.

Customers receive their kit in the mail, with which they collect and return a personal sample for testing. One of the startup's lab partners analyzes the sample and returns it to a LetsGetChecked physician, who reviews the results and counsels customers over the phone, if necessary. For later reference, test results are saved in a LetsGetChecked patient app that interfaces with Apple Health, Fitbit, Garmin and other consumer-friendly health tools.

As of March, the company has also been offering a COVID-19 "twin-track" antibody and PCR test exclusive to providers working at the point of care. However, the company said it hopes to release a direct-to-consumer version of the testing kit down the road.

"The company's work has never been more important now that the world faces a global pandemic, forcing us all to re-evaluate how we obtain health and wellness services," Illumina's Naclerio said in a statement."LetsGetChecked is already having a profound impact on patient-led at-home healthcare, which is necessary now and will become increasingly common in the future."

WHAT IT'S FOR

The company said these funds will help build up its manufacturing, supply and testing capacity for COVID-19. It will be increasing activity at its California laboratorywhile boosting operations and partnerships across the U.S. and Europe.

"We have been developing our platform for more than five years and have helped hundreds of thousands of individuals by enabling access to high-quality testing and telehealth services in the home, across many health conditions," Peter Foley, CEO and founder of LetsGetChecked, said in a statement. "With the onset of COVID-19, we realigned resources with a view to helping with this global pandemic and are currently delivering tens of thousands of tests per day to critical and frontline healthcare workers"

MARKET SNAPSHOT

Consumer-facing home testing startups were already picking up momentum around the time of LetsGetChecked's $30 million Series B, with competitors like EverlyWell, healthy.io and Scanwell Health all announcing new rounds or services over the course of the last year. However, COVID-19 has ramped up demand across the industry and led to some confusion with the FDA on more than one occasion. But the window for these companies to take their COVID-19 tests directly to consumers appears to be reopening: It was just a few weeks ago that LabCorp's COVID-19 RT-PCR test became the first permitted for at-home sample collection.

ON THE RECORD

"HLM invests in companies that inherently drive adoption due to the need they serve, and companies that leverage important market trends," Tolle said in a statement. "In this case, LetsGetChecked is helping to improve consumer access to care, which is increasingly essential."

Original post:
LetsGetChecked locks in $71M Series C to expand COVID-19 testing capacity - MobiHealthNews

(MENAFN - GetNews)

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Desert Mobile Medical | Concierge Physicians offers services such as: hormone replacement therapy, mobile lab tests, vaccinations, in-home medical dispensing, mobile diagnostic imaging services, direct primary care services, as well as physical and massage therapy services.

Desert Mobile Medical | Concierge Physicians is headquartered at 9300 E Raintree Dr Suite 130 Suite 2, Scottsdale, AZ 85260. Contact their team via phone at (480) 427-0002 or send online inquiries via email to . For additional information regarding their services, visit their website.

Media Contact Company Name: Desert Mobile Medical Contact Person: Dr. Paresh Goel Email: Send Email Phone: (480) 427-0002 Address: 9300 E Raintree Dr Suite 130 Suite 2 City: Scottsdale State: AZ Country: United States Website: http://desertmobilemedical.com/

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Desert Mobile Medical | Concierge Physicians is Now Offering Virtual Appointments to Help Avoid the Spread of COVID-19 - MENAFN.COM

Working from home requires modifications to a traditional workday. The blending of responsibilities from two major areas of our lives, work and home, can be a considerable source of stress, especially during a pandemic.

This is a reality for me, and my husband is a clinical psychologist, so I asked him for some tips. Here is some advice from Dr. Eric W. Bravo on how to stay productive, reduce stress, and manage psychological well-being while working from home:

Cortisol, the main stress hormone in the brain and body, increases as aresult of stress. The blending of work and home obligations is certainly stressful. During a pandemic, everyones cortisol levels are elevated, even if they are not sick.

Chronic exposure to cortisol leads to fatigue, weight gain, sleep disturbance, and an overall negative mood. To reduce the brains exposure to heightened cortisol, several coping skills should be considered.

Maintaining a daily schedule is an important consideration in preventing depression, reducing stress, and increasing energy levels. In the morning, actively construct your day. Think of the tasks that you need to get accomplished.

Having an outline of your responsibilities will guide your actions through the day and leave you with a sense of accomplishment when you are ready to clockout. Think about how to use this time to your advantage. Ask yourself if it is possible to grow the business, make work more organized and efficient, or learn a new skill.

Be aware of limits your productivity and success caused by self-sabotaging. Vices are more accessible than ever. When working from home, it is easier to sleep in, nap during the day, and procrastinate. Hold yourself accountable to the same standard of discipline when you were going into the office.

Most importantly, wake up every day and dress for success. You may not need to wear a suit, but dress to be active. Be sure to wear proper shoes and leave the pajamas for sleeping only.

Changing clothesJanet Loehrke

The body and brain need routines from the most basic of sleeping and eating to other activities, such as work. We are programmed neurochemically to have basic daily cycles. When daily routines are disrupted, our neurological systems shift. Neurochemical imbalances can occur from a shift in daily cycles, leading to depression, anxiety, lethargy, and poor cognitive performance. The basics of a daily routine, including staying consistent with sleep/wake cycles, regular meals, exercise, and activity are essential.

Work-life boundaries are more important now than ever. Boundaries are the psychological barriers between our life at work and life at home. Create a separation between activities that are work-related and those that are not. As much as possible, have a dedicated workspace free from distractions, leisurely activities, and household responsibilities. The distance created by separating the space can let you focus on a productive workday and allow you to leave the office when needed.

To reduce packing on more stress, be careful to manage your expectations. It is common to be less productive when working from home. Develop a hierarchy of work tasks, target the most necessary first then work down your list. If you are unable to complete all of the work, be kind to yourself, take a break.

Think of coping skills as medication to treat an infirmity. We need to practice self-care to stay motivated, productive, and healthy. Immerse your mind and body in activities that are not related to being on duty. Meditation, exercise, cooking, reading, conversations with friends and support systems, and a good sweat all have an effect on lowering cortisol and increasing a positive mood. Furthermore, as important as it is to stay informed, limit your exposure to upsetting news.

Motivation and productivity take work, but, are attainable. The more that a person prepares for the day with a routine, the better off. Control the amount of time that you are not productive. It is fine to relax and watch a good movie, just have limits. Set boundaries for downtime. Getting in the habit of being sedentary will slow your mind, body, and energy. Conversely, consistent exercise and activity maintain and overall sense of motivation.

Hold yourself accountable by limiting the excuses to get work done. Chances are, these are the same rationalizations that are noticeable before working from home. Avoid working from your bed or bedroom. As comfortable as it may seem, a productive day requires being slightly uncomfortable. Our bedrooms have too many associations of relaxation. We do not want to contaminate the drive needed to complete our daily obligations. We also do not want to tarnish the mellow vibe of where we rest.

Engagement with online hangouts and meetings are crucial for perspective, social interaction, and coping with feelings of loneliness. Avoid isolation and make it a priority to reach out to your social network. Take it another step, change the dynamics, and reach out to a friend that you have not heard from in a while.

A new phenomenon, Zoom Fatigue, is leading to higher levels of lethargy and exhaustion during the workday. Video-conferencing requires much more focus and energy than in-person meetings. Paying attention to multiple people at once, figuring out eyecontact, and concerns about our digital appearance is draining. Interestingly, similar fatigue can also occur during social videoconferencing. In order to combat this fatigue, think about the necessity of using video. If a telephone call will suffice for work or social purposes, it is a much better option.

Practice the awareness of gratitude for your employment. Remind yourself of the reason that you do what you do. Think about your journey and accomplishments. Familiarize yourself with the mission of your job. In a time where unemployment rates are at an all-time high, you have employment to report to. Convince yourself that you have a purpose, arise, go forth, and conquer!

It can be difficult to work from home for long periods of time. By practicing these coping strategies, you can increase the likelihood of success, limit stress, and improve your mood. Keep in mind, we are not in control of what happens to us, but we are responsible for how we adapt and respond.

Editor's Note: The author and a source named in this article are married.

Read more here:
Working from home during the coronavirus pandemic: How to cope - USA TODAY

Deepak Chopra, Special to SFGate

By Deepak Chopra, MD, FACP, Rudolph E. Tanzi, PhD, Michelle Williams, ScD, Ryan Castle, William C. Bushell, PhD, Kimberly Brouwer, PhD, and Paul J. Mills, PhD

Although COVID-19 is very easily transmitted from person to person, the risk of subsequent hospitalization and death primarily affects people who are already at risk because of old age, infirmity and/or chronic diseases such as cancer, diabetes, autoimmune illness, obesity, and heart disease. All of these chronic illnesses are associated with measurable low-grade inflammation in the body. The chronic low-grade inflammation that develops with advanced age has become known as inflammaging. Most people with chronic illness unknowingly have low-grade inflammation. Recent research points to a second finding: these same disorders are often accompanied by persistent low-grade anxiety and depression.

All of this as a background increases the danger for a person when acute illness strikes. In addition to the elderly and chronically ill, COVID-19 is causing acute respiratory illness and stroke sometimes leading to death in seemingly otherwise healthy younger individuals. The transition from SARS-CoV-2 infection to diagnosed COVID-19 is typically accompanied by a cytokine storm. Cytokines are proteins that are major drivers of inflammation, and their rapid increase, or "storm is one of the bodys immune responses to acute threat.

In addition, studies have connected pro-inflammatory cytokines to the stress response; they regulate well-known stress hormones such as ACTH and cortisol. Three major systems are involved: the immune system, the central nervous system and the endocrine hormone system.

In the face of these connections, we are coming forward to suggest that complementary practicesdeep breathing, yoga, and meditationcan play an important role during this pandemic. These practices have been confirmed by hundreds of scientific studies to bring down over-activity of the autonomic nervous system, calm the mind from anxiety, reduce the stress response, regularize heartbeat, and lower blood pressure. Together, all of these diverse benefits are associated with reducing the invisible presence of chronic low-grade inflammation, especially if added to good sleep, exercise, and proper diet.

We dont fully understand how the immune response, linked to stress and inflammation, can turn lethal. As a response to cuts, wounds, invading pathogens, and other threats, prior to antibody formation, the body first responds with inflammation as a normal yet crucial healing function. But it has long been known that inflammation is paradoxical. Acute inflammation can over-react, harming or even killing the patient. (Instances of strokes and heart attacks among young COVID-19 patients might be linked to micro-cytokine storms in the brain and heart.)

The threat from low-grade chronic inflammation was not discovered until recently but seems to be widespread. It is unaccompanied by the swelling, burning, and redness of the skin that marks acute inflammation and therefore goes undetected by the patient or physician. Preventing and addressing chronic low-grade inflammation and its significant adverse consequences are urgent issues, even more urgent during a pandemic. There seems to be every reason to make the public aware how deep breathing, meditation, yoga, and other healthy lifestyle practices can help during this crisis and long afterwards.

Deepak Chopra MD, FACP, Clinical Professor of Family Medicine and Public Health at the University of California, San Diego

Rudolph E. Tanzi, PhD, Kennedy Professor of Neurology at Harvard Medical School/MGH

Michelle Williams, SM, ScD, Dean of the Faculty, Harvard T.H. Chan School of Public Health

Kimberly Brouwer, PhD, Professor and Chief, Department of Family Medicine and Public Health, Division of Global Health, Infectious Diseases Epidemiology, at the University of California, San Diego

Ryan Castle, Executive Director of the Chopra Library

William C. Bushell, PhD, medical anthropologist and research director of the Chopra Library

Paul J. Mills, PhD, Professor and Chief, Department of Family Medicine and Public Health, at the University of California, San Diego

Read more from the original source:
The Key to Handling Stress and COVID-19 - SFGate

CAMBRIDGE, Mass., May 5, 2020 /PRNewswire/ -- Akebia Therapeutics, Inc. (Nasdaq: AKBA), today announced positive top-line results from INNO2VATE, its global Phase 3 cardiovascular outcomes program evaluating the efficacy and safety of vadadustat, its investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI), versus darbepoetin alfa for the treatment of anemia due to chronic kidney disease (CKD) in adult patients on dialysis. (Please refer to Akebia's INNO2VATE Data Announcement for the top-line data.) The Company also reported financial results for the first quarter ended March 31, 2020 and will host a conference call with slides today, Tuesday, May 5, 2020, at 8:30 a.m. Eastern Time to discuss INNO2VATE top-line data, its first quarter financial results and recent business highlights.

"We are very excited about the positive top-line results from INNO2VATE and expect to build on this momentum with many potentially transformational near-term milestones," said John P. Butler, President and Chief Executive Officer of Akebia Therapeutics. "We believe we've developed an exciting path forward for vadadustat and although the COVID-19 environment remains unpredictable, we continue to execute and deliver solid progress against these initiatives. In collaboration with our partner, Mitsubishi Tanabe, we are advancing key pre-commercial activities in support of the first regulatory approval of vadadustat expected in Japan this year. We have also significantly advanced PRO2TECT, our global Phase 3 studies evaluating the safety and efficacy of vadadustat in adult patients not on dialysis with anemia due to CKD, and expect to report top-line data from these studies mid-year, as planned. In addition, we reinforced our intellectual property position for vadadustat, confirming the path for Akebia and our collaboration partner, Otsuka, to launch vadadustat in the U.K. and potentially the rest of Europe, upon approval."

"Akebia continues to provide and support our innovative therapies, which are critical to the care of adult patients with CKD, who are among the most at risk during this pandemic. To date, we have not experienced any significant adverse impact from COVID-19 and our fundamentals remain strong with a cash runway that extends well into 2021. However, these are unprecedented times and, due to COVID-19, we do not have clear visibility on how product demand and payer mix may be impacted. As a result, we continue to actively monitor and assess the potential impact of COVID-19 on our business and operations while continuing to support patients." Butler concluded, "We believe we have tremendous value-enhancing opportunities ahead and we remain focused on supporting patients and executing on our plans to position Akebia for long term growth."

Business Highlights

Today, the Company announced positive top-line results from INNO2VATE. Please refer to

for the top-line data.In April, the Patents Court of the United Kingdom issued a

in favor of Akebia and Otsuka Pharmaceutical Co. Ltd., which found that five of FibroGen, Inc.'s six HIF-related patents were invalid, and a sixth patent would not be infringed.In April, the Company announced that Myles Wolf, M.D., M.M.Sc., joined Akebia's Board of Directors. Dr. Wolf is a leading clinical nephrologist and physician-scientist in the fields of disordered mineral metabolism and cardiovascular disease in patients with CKD. Dr. Wolf serves as Chief of the Division of Nephrology and a Professor of Medicine at Duke University School of Medicine.In April, the Company settled Auryxia patent litigation with Teva Pharmaceuticals USA, Inc. (Teva) and its wholly owned, indirect subsidiary, Watson Laboratories, Inc. (Watson), resolving patent litigation brought in response to Abbreviated New Drug Application (ANDA) filings by Teva and Watson. Consistent with the Company's prior ANDA settlement with Par Pharmaceutical, Inc., the settlement allows Teva and Watson to market its generic version of Auryxia in the United States beginning on March 20, 2025 (subject to U.S. FDA approval), or earlier under certain circumstances customary for settlement agreements of this nature.In April, the Company entered into a new supply agreement with STA Pharmaceutical Hong Kong Limited, a subsidiary of Wuxi AppTec (WuXi), under which WuXi will manufacture vadadustat drug substance for commercial use. The WuXi supply agreement is the third commercial supply agreement the Company has entered into for vadadustat. The Company entered into a commercial supply agreement for vadadustat drug substance with Esteve Qumica, S.A. in April 2019, and a commercial supply agreement for vadadustat drug product with Patheon Inc. in March 2020.In April, the United States District Court for the District of Delaware dismissed a putative securities class action brought against the Company's wholly owned subsidiary, Keryx Biopharmaceuticals, Inc. (Keryx), and former members of Keryx's board of directors, relating to the Company's 2018 merger with Keryx in response to a motion to dismiss filed by Keryx and the former directors.In February, the Company entered into a letter agreement with Vifor (International) Ltd. (Vifor Pharma) relating to Vifor Pharma's agreement with a third party to purchase a Priority Review Voucher (PRV) issued by the U.S. Food and Drug Administration (FDA), subject to satisfaction of customary closing conditions. Pursuant to the letter agreement, Akebia paid Vifor Pharma $10 million. Vifor Pharma is obligated to reserve the PRV for the vadadustat NDA for the treatment of anemia due to CKD in dialysis-dependent and non-dialysis dependent patients until Akebia and Vifor Pharma agree on the financial and other terms under which it will assign the PRV to Akebia or make a mutual decision to sell the PRV. A PRV entitles the holder to priority review of an NDA or a Biologics License Application for a new drug, which reduces the target FDA review time to six months after official acceptance of the submission and could lead to expedited approval.

First Quarter Financial Results

Revenues: Total revenue increased 22 percent to $88.5 million for the first quarter of 2020 compared to $72.7 million for the first quarter of 2019.Collaboration revenue was $59.3 million for the first quarter of 2020 compared to $49.6 million in the first quarter of 2019, an increase of 20 percent.Net product revenue was $29.2 million for the first quarter of 2020 compared with $23.1 million in the first quarter of 2019, an increase of 26 percent.COGS: Cost of goods sold was $27.7 million for the first quarter of 2020, which includes non-cash charges, related to the application of purchase accounting as a result of the merger with Keryx, of $11.2 million for inventory step-up and $9.1 million for amortization of intangibles. Cost of goods sold was $31.3 million for the first quarter of 2019, and included non-cash merger-related charges of $14.6 million for inventory step-up and $9.1 million for amortization of intangibles.R&D Expenses: Research and development expenses were $81.2 million for the first quarter of 2020 compared to $82.4 million for the first quarter of 2019.SG&A Expenses: Selling, general and administrative expenses were $38.0 million for the first quarter of 2020 compared to $34.3 million for the first quarter of 2019.Net Loss: Net loss was $60.7 million for the first quarter of 2020 compared to $72.4 million for the first quarter of 2019.Cash Position: Cash, cash equivalents and available-for-sale securities as of March 31, 2020 were $115.4 million. The Company's cash runway extends well into 2021. The Company's cash runway, consistent with previous commentary, includes the receipt of a $15.0 million regulatory milestone from Mitsubishi Tanabe Pharma Corporation, Akebia's development and commercialization collaboration partner in Japan for vadadustat, assuming approval of vadadustat in Japan.

"Consistent with our prior first quarter periods, the decrease in the Company's cash, cash equivalents and available-for-sale securities from year-end 2019 was due to the timing of cash payments from our collaboration partner, Otsuka, for which $49.5 million was received in the current quarter rather than the first quarter of 2020. In addition, during the first quarter, we paid $10 million to Vifor Pharma in connection with Vifor Pharma's purchase of the PRV," stated Jason A. Amello, Chief Financial Officer of Akebia.

COVID-19 Response The current severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) (COVID-19), pandemic has presented a substantial public health and economic challenge around the world and is affecting Akebia's employees, patients, customers, collaboration partners, vendors, communities and business operations. On March 15, 2020, Akebia issued a press release detailing its response to the COVID-19 pandemic. Consistent with that statement, the Company continues to take action to safeguard its employees, patients and customers, ensure business continuity, and support supply of its innovative therapies which are critical to the care of adult CKD patients, who are among the most at risk during this pandemic. Akebia continues to actively monitor and assess the potential impact of the COVID-19 pandemic on its business and operations. Patients with any questions about accessing Akebia's marketed therapy may refer to AkebiaCares, the Company's patient access program, for further information.

Conference Call: Akebia will host a conference call today, Tuesday, May 5, 2020, at 8:30 a.m. Eastern Time to discuss its INNO2VATE top-line data announced earlier this morning and its first quarter financial results. To listen to the conference call, please dial (877) 458-0977 (domestic) or (484) 653-6724 (international) using conference ID number 8464788. The call will also be webcast LIVE with slides and can be accessed via the Investors section of the Company's website at http://ir.akebia.com.

A replay of the conference call and the slides will be available two hours after the completion of the call through May 11, 2020. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference conference ID number 8464788. An online archive of the conference call can be accessed via the Investors section of the Company's website at http://ir.akebia.com.

About Akebia Therapeutics Akebia Therapeutics, Inc. is a fully integrated biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease. The Company was founded in 2007 and is headquartered in Cambridge, Massachusetts. For more information, please visit our website at http://www.akebia.com which does not form a part of this release.

About Vadadustat Vadadustat is an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor currently in global Phase 3 development for the treatment of anemia due to CKD. Vadadustat is designed to mimic the physiologic effect of altitude on oxygen availability. At higher altitudes, the body responds to lower oxygen availability with stabilization of hypoxia-inducible factor, which can lead to increased red blood cell production and improved oxygen delivery to tissues. Vadadustat is an investigational therapy and is not approved by the U.S. Food and Drug Administration (FDA) or any regulatory authority.

About Anemia due to Chronic Kidney Disease (CKD) Anemia is a condition in which a person lacks enough healthy red blood cells to carry adequate oxygen to the body's tissues. It commonly occurs in people with CKD because their kidneys do not produce enough erythropoietin (EPO), a hormone that helps regulate production of red blood cells. Anemia due to CKD can have a profound impact on a person's quality of life as it can cause fatigue, dizziness, shortness of breath and cognitive dysfunction. Left untreated, anemia leads to deterioration in health and is associated with increased morbidity and mortality in people with CKD.

Forward-Looking Statements Statements in this press release regarding Akebia's strategy, plans, prospects, expectations, beliefs, intentions and goals are forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, as amended, including but not limited to statements regarding clinical trial data and results and the anticipated timing of the availability and reporting thereof; Akebia's momentum and potentially transformational near-term milestones; the safety and efficacy of vadadustat, the potential launch of vadadustat, the potential indications for and benefits of vadadustat, and market size, commercial potential, prevalence, and the growth in, and potential demand for, vadadustat; access to a Priority Review Voucher for vadadustat and the agreement relating thereto; potential and anticipated payments from our collaborators, including the timing thereof; expectations regarding financial position, including cash runway and the components thereof; and our intellectual property position. The terms "anticipate," "believe," "expect," "opportunity," "planned," "potential," "target," "will" and similar references are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including the potential direct or indirect impact of the COVID-19 pandemic on our business, operations, and the markets and communities in which we and our partners, collaborators, vendors and customers operate; the risk that existing preclinical and clinical data may not be predictive of the results of ongoing or later clinical trials, including PRO2TECT; the risk that clinical trials may not be successful; manufacturing risks; risks associated with the Priority Review Voucher for vadadustat; risks associated with management and key personnel changes and transitional periods; the actual funding required to develop and commercialize our commercial product, vadadustat and other product candidates and operate the Company, and the actual expenses associated therewith; the actual costs incurred in the clinical studies of vadadustat and the availability of financing to cover such costs; the risk that clinical studies are discontinued or delayed for any reason, including for safety, tolerability, enrollment, manufacturing or economic reasons; market acceptance and coverage and reimbursement of our commercial product and vadadustat, if approved; the risks associated with potential generic entrants for our commercial product and vadadustat, if approved; early termination of any of Akebia's collaborations; Akebia's and its collaborators' ability to satisfy their obligations under Akebia's collaboration agreements; the timing and content of decisions made by regulatory authorities; the timing of any additional studies initiated for vadadustat; the actual time it takes to initiate and complete preclinical and clinical studies; the competitive landscape for our commercial product and vadadustat; the scope, timing, and outcome of any legal, regulatory and administrative proceedings; changes in the economic and financial conditions of the businesses of Akebia and its partners; the risk that we lose, or settle on less favorable terms, other ANDA litigation, or that other ANDA filers enter the market earlier than March 20, 2025, as well as any other potential settlements; and Akebia's ability to obtain, maintain and enforce patent and other intellectual property protection for our commercial product, vadadustat and any other product candidates. Other risks and uncertainties include those identified under the heading "Risk Factors" in Akebia's Annual Report on Form 10-K for the year ended December 31, 2019 and other filings that Akebia may make with the U.S. Securities and Exchange Commission in the future. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release, and Akebia does not undertake, and specifically disclaims, any obligation to update any forward-looking statements contained in this press release.

Investor Contact: Kristen K. Sheppard, Esq. Ir@akebia.com

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Akebia Announced Positive Top-Line Results from Global Phase 3 Program of Vadadustat for Treatment of Anemia Due to Chronic Kidney Disease in Adult...

In the early 1950s, psychiatrists began treating schizophrenia with a new drug called chlorpromazine. Seven decades later, the drug is still used as an anti-psychotic.

But now scientists have discovered that the drug, also known as Thorazine, can do something entirely different. It can stop the new coronavirus that causes Covid-19 from invading cells.

Driven by the pandemics spread, research teams have been screening thousands of drugs to see if they have this unexpected potential to fight the coronavirus. Theyve tested the drugs on dishes of cells, and a few dozen candidates have made the first cut.

Theyre startlingly diverse. Some, like chlorpromazine, have been used for years not for viral infections, but for conditions including cancer, allergies, arthritis, even irregular menstrual periods. Other drugs have not yet been approved by the Food and Drug Administration, but they have already proven safe in clinical trials. Their track records might help them get approved faster than a drug designed from scratch.

As researchers publish findings on these promising drugs, theyre starting tests on animals and people to see how well they perform. No one should try self-medicating with any of the drugs for Covid-19, the researchers warned, since they may have dangerous side effects and have yet to be proven effective in clinical trials.

Im going to be brutally honest with you: 95 to 98 percent of these are going to fail, said Sumit K. Chanda, a virologist at Sanford Burnham Prebys Medical Discovery Institute in La Jolla, Calif. But we only need one or two.

The strategy Dr. Chanda and other researchers are using is known as drug repurposing. It has a history that started decades before Covid-19 appeared. In 1987, for example, the cancer drug zidovudine became the first F.D.A.-approved drug against H.I.V.

The most obvious drugs to repurpose against the new coronavirus are those that work against other viruses. One high-profile antiviral being investigated is remdesivir, which Gilead Sciences previously tested unsuccessfully as an antiviral against Ebola.

But over the years, researchers have found some drugs that originally had nothing to do with viruses turn out to be good antivirals, too. Its just hard to tell in advance which ones have this hidden power.

We dont know a lot about why drugs do what they do, said Matthew Frieman, a virologist at the University of Maryland School of Medicine.

In 2012, another coronavirus disease known as MERS emerged in the Middle East. Dr. Frieman responded by starting a drug-repurposing study. He and his colleagues tested 290 F.D.A.-approved drugs and found that 27 of them blocked the MERS virus from infecting cells. They also proved effective against the related coronavirus that causes SARS.

Dr. Frieman and his colleagues have now tested those drugs against the new coronavirus, and made a preliminary report that 17 of them showed promise. Along with chlorpromazine, they include drugs for disorders as varied as Parkinsons disease and leukemia.

Recently, Dr. Chandas team in California began a mammoth search of their own for drugs to repurpose for Covid-19. They doused infected cells with 13,000 compounds and looked for ones that slowed down the virus. They then narrowed down these candidates by reducing their doses, in order to mimic the levels that would end up in a patients lungs.

On April 17, Dr. Chandas team reported in a preprint, which has not yet been peer-reviewed by a journal, that six drugs showed particular promise, including one for osteoporosis and one thats been investigated as treatment for arthritis.

Yet another team has been trying to find drugs that work against coronavirus and also to learn why they work.

The team, led by Nevan Krogan at the University of California, San Francisco, has focused on how the new coronavirus takes over our cells at the molecular level.

The researchers determined that the virus manipulates our cells by locking onto at least 332 of our own proteins. By manipulating those proteins, the virus gets our cells to make new viruses.

Dr. Krogans team found 69 drugs that target the same proteins in our cells the virus does. They published the list in a preprint last month, suggesting that some might prove effective against Covid-19.

The researchers shipped the compounds to the Icahn School of Medicine at Mount Sinai in New York and at the Pasteur Institute in Paris. Those labs tried them out on infected cells.

Brian Shoichet, a pharmaceutical chemist at U.C.S.F. who helped build the list, was keenly aware of how often drug repurposing fails.

I wasnt that hopeful at all, he said.

It turned out that most of the 69 candidates did fail. But both in Paris and New York, the researchers found that nine drugs drove the virus down.

The things were finding are 10 to a hundred times more potent than remdesivir, Dr. Krogan said. He and his colleagues published their findings Thursday in the journal Nature.

Strikingly, the drugs hit only two targets.

One group temporarily stops the creation of new proteins inside cells. This group includes molecules that are being tested as cancer drugs, such as ternatin-4 and Zotatifin.

Dr. Shoichet speculated that these compounds starve the virus of the proteins it needs to make new copies of itself. This attack may suddenly halt the viral production line.

Viruses are actually delicate beasts, he said.

The other compounds home in on a pair of proteins known as Sigma-1 and Sigma-2 receptors. These receptors are part of the cells communication network, helping the cell withstand stress in its environment.

Why does the new coronavirus need to manipulate Sigma receptors? We dont really know, Dr. Shoichet said.

One possibility is that the virus uses Sigma receptors to make a cell produce more of the oily molecules that form membranes for new viruses.

Among the substances that act on Sigma receptors and block the virus, the researchers found, are the hormone progesterone and the drugs clemastine and cloperastine, both used against allergies.

In addition, Dr. Krogan said that all of Dr. Friemans candidates, including chlorpromazine, target Sigma receptors. A third of Dr. Chandas candidates do too, he said.

The researchers also tested dextromethorphan, a Sigma-receptor-targeting drug in many brands of cough syrup. They were surprised to find that, at least in their cell samples, it actually made infections of this coronavirus worse.

In their paper, the researchers raised the possibility that Covid-19 patients may want to avoid dextromethorphan. Dr. Krogan emphasized that more study would be needed to see if it actually increases coronavirus infection in humans. But if it was me, he said, to be cautious, I would not be taking these cough syrups.

The anti-malaria drugs chloroquine and hydroxychloroquine act on the Sigma receptor. Dr. Krogans team found that they also fought the virus in cells. Those compounds were extolled by President Trump for weeks despite no firm evidence they actually helped cure Covid-19.

Dr. Frieman and Dr. Chanda also found that chloroquine-related drugs worked fairly well in slowing the virus in cell cultures. But Dr. Chanda found they didnt work as well as the six compounds at the top of his list.

Dr. Chanda expressed skepticism about the chloroquine drugs, noting their failure against other viruses.

Weve been down this road multiple times, he said. I would happy to be wrong about this.

Last week, the F.D.A. issued a warning against using hydroxychloroquine or chloroquine for Covid-19 outside the hospital setting or a clinical trial. Thats because the drug has a well-known risk for causing irregular heart rhythms.

In their new study, Dr. Krogan and his colleagues ran an experiment that might explain this risk at the molecular level.

They found that chloroquine and hydroxychloroquine bind not just to Sigma receptors, but to a heart protein called hERG, which helps control heartbeats.

I think its a rational argument, said Dr. Frieman, who was not involved in the Nature study. Chloroquine does a lot of things in the cell.

Dr. Krogan and his colleagues found that other compounds target Sigma proteins in a more promising way.

An experimental anticancer compound called PB28 is 20 times more potent than hydroxychloroquine against the coronavirus, for example. But its far less likely to grab onto the hERG protein.

Dr. Chanda said that PB28 in particular looks really fantastic.

Dr. Krogan said that studies are underway to test the drug in hamsters to see if that promise holds. Dr. Frieman and his colleagues are starting animal studies of their own, as well as testing drugs on a chip lined with human lung cells.

Timothy Sheahan, a virologist at the University of North Carolina who was not involved in the new studies cautioned that it will take more testing to make sure these promising drugs are safe to give to patients ravaged by Covid-19.

Cancer drugs, for example, can be like a sledgehammer to your body, he noted. Are you going to want to do that when someone is really sick?

In addition to animal tests and clinical trials, researchers are now planning to tweak the structure of these drugs to see if they can work even more effectively against the virus.

Originally posted here:
Old Drugs May Find a New Purpose: Fighting the Coronavirus - The New York Times