Archive for March, 2021

MONTPELLIER, France--(BUSINESS WIRE)--Regulatory News:

Sensorion (FR0012596468 ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, announces today its full-year 2020 financial results and provides an update on its business activities and outlook for 2021.

We are pleased with the progress made in 2020. We raised 36 million in equity financing to help advance our otoprotective small molecule SENS-401 and our pipeline of promising preclinical gene therapies from our strategic partnership with Institut Pasteur. We expanded our unique technology platform in gene therapy by further building our internal preclinical, process development and analytical capabilities. In Q4 2021, we expect the topline data readout from the Phase 2 trial of SENS-401 to treat sudden sensorineural hearing loss (SSNHL). In H2 2021, we also expect to initiate a clinical trial of SENS-401 to treat cisplatin-induced ototoxicity, a large indication with a significant unmet medical need said Nawal Ouzren, CEO of Sensorion.

Key developments in 2020: science and operational

Gene therapy collaboration with Institut Pasteur on hearing loss

On June 9, 2020, Sensorion announced positive preliminary preclinical data in non-human primates for its gene therapy program targeting Otoferlin deficiency (OTOF-GT), showing that it could deliver an intracellular marker to the inner hair cells at levels that reflect the future clinical requirements. The company plans to discuss its OTOF-GT program with regulatory authorities in H1 2021.

Additionally, Sensorion announced an agreement with Novasep on October 27, 2020, for the process development and manufacturing of adeno-associated viruses (AAV) gene therapy products for the Companys OTOF-GT program.

Technology platform expanded

Sensorion has built a unique R&D technology platform over the years to deepen the understanding of the pathophysiology and etiology of inner ear related diseases. The platform is being actively deployed to select targets, identify biomarkers and to optimize small molecules and gene therapy candidates.

The strengthened platform includes in-vitro assays encompassing the key cochlear cell types, systems to investigate explant tissue and advanced electrophysiological methods for assessing neuronal activity.

On the in-vivo side, Sensorion is developing a suite of validated preclinical models reflecting specific pathologies and inner ear lesions, as well as advancing the use of techniques such as auditory brainstem response (ABR) and distortion product oto-acoustic emission (DPOAE) audiometry for measuring and analysing hearing parameters in those models.

The last pillar of the Companys technology platform focuses on developing biomarkers to improve diagnosis and guide treatment in the key areas of unmet medical need in hearing loss.

Drug candidate SENS-401

Our clinical program with small molecule SENS-401 continued to progress in 2020.

Sensorion is conducting a Phase 2 clinical trial of SENS-401 in the treatment of SSNHL in adults. This Phase 2, randomized, double-blinded, placebo-controlled study is being conducted in multiple countries in Europe and Canada.

On February 17, 2020, Sensorion received Ethics Committee approval to include new military sites in this study, allowing clinical investigators to recruit volunteer military personnel who have suffered from acute hearing loss.

On March 13, 2020, Sensorion provided an update on the recruitment schedule for the ongoing SENS-401 Phase 2 study for the treatment of SSNHL. Due in part to the impact of COVID-19 on patient enrolment, the availability of topline data were expected to be delayed to Q4 2021 (see below in 2021 announcements).

On June 5, 2020, the independent Data Safety Monitoring Board (DSMB) confirmed the absence of safety concerns and recommended continuation of the Phase 2 trial as scheduled.

Having demonstrated otoprotective activity in several preclinical models, SENS-401 is being studied as a potential option to preserve residual hearing in people with cochlear implants under a collaboration with Cochlear, the world leader in implantable hearing solutions. Positive preclinical data were announced on January 5, 2021 (see below in 2021 announcements). Next steps are being discussed with Cochlear.

On December 15, 2020, Sensorion and Sonova Holding AG, a leading provider of hearing solutions, announced that Sonova had acquired a 3.7% equity stake in Sensorion via an investment of 5 million. At the same time, the two companies signed a letter of intent to exclusively explore potential plans for a strategic collaboration in the field of innovative diagnostic and therapeutic solutions for certain types of hearing loss. Discussions between Sensorion and Sonova are ongoing.

Strengthened scientific and medical leadership

As part of Sensorions strategic move into gene therapy for hearing restoration, on February 19, 2020 the Company announced the appointment of Dr. Graldine Honnet as Chief Medical Officer. Dr. Honnet has extensive expertise both in gene therapy and small molecule clinical development across numerous disease areas.

During 2020, Sensorion also increased its R&D headcount by 30% strengthening its team with experienced scientists and researchers in gene therapy, the inner ear and neurosciences. In addition to Dr Honnet, Sensorion also appointed a preclinical head in gene therapy and a new CMC gene therapy lead with more than 20 years of experience in the field.

On July 29, 2020, Sensorion announced the appointment of five distinguished experts to its Scientific Advisory Board (SAB); Prof. Alain Fischer, Dr. Robert Dow, Prof. Paul Avan, Dr. Diane Lazard and Dr. Hernn Lpez-Schier. The SAB is chaired by Prof. Christine Petit, Founding Director of the French Hearing Institute and a world-renowned geneticist and neurobiologist in hearing and hearing disorders.

Scientific communications

Sensorion presented at various scientific congresses and hosted two Key Opinion Leaders (KOLs) calls in 2020, including:

Governance: appointment of a new Chairman of the Board of Directors

On July 6, 2020, Sensorions Board of Directors was strengthened by the appointment of Edwin Moses as Chairman of the Board of Directors. Dr. Moses has more than 25 years of executive experience as both CEO and Chairman of numerous life science companies, including Ablynx, where he led its rapid growth from a small research-focused organization to one of Europes leading biotechnology companies, prior to its $4.8 billion acquisition by Sanofi in 2018.

2021 announcements

Since the end of the fiscal year, the key business updates are as follows:

On January 5, 2021, Sensorion provided an update on plans and progress made in the development of SENS-401 for the prevention of hearing loss. After encouraging efficacy data in preclinical models, the Company expects to begin a proof-of-concept clinical trial with SENS-401 to treat patients suffering from cisplatin-induced ototoxicity (CIO) in the second half of 2021. A natural history study of CIO in adult cancer patients is expected to start in the first half of 2021. Successful clinical findings would significantly expand Sensorions target market for SENS-401: approximately 500,000 cancer patients are treated annually with cisplatin in the USA and EU5, a significant proportion of whom will experience severe hearing loss.

At that time, Sensorion also announced a delay in the availability of the topline results from the Phase 2 study of SENS-401 in SSNHL to Q4 2021, due to the impact of COVID-19. Additionally, Sensorion indicated that it planned to review the study design and consider opportunities to aid its successful on-time completion. Following this review, an amendment to the statistical analysis plan (SAP), which would significantly reduce the sample size without compromising on the quality and potential outcome of the trial, has been submitted to the regulatory authorities. The responses from regulators so far have been encouraging and this increases the Companys confidence in being able to generate topline data this year.

On January 19, 2021, Sensorion announced positive preclinical data demonstrating the potential of SENS-401 to preserve residual hearing after cochlear implantation, in a collaboration with Cochlear, the global leader in implantable hearing solutions. Cochlear implants are very effective in treating severe to profound hearing loss, but preserving acoustic hearing in patients with residual hearing who receive cochlear implants could provide substantial benefit. Sensorion and Cochlear are making progress in the discussion around potential clinical study designs. Next steps are being discussed with Cochlear.

On February 15, 2021, Sensorion announced a third gene therapy collaboration with Institut Pasteur around the GJB2 gene targeting important pediatric and adult deafness markets. GJB2 mutations had already been widely recognized as the most prevalent cause of congenital deafness and now, new findings from Institut Pasteur have now demonstrated that GBJ2 mutations also underlie a wide range of other instances of severe hearing loss in the adult population. Sensorion will pursue three initial GBJ2-related indications: congenital deafness, progressive childhood hearing loss and early onset of severe presbycusis in adults.

2021 strategy and prospects

As of 31st December 2020, the Company had 62 million in cash, boosted by a successful 31 million Reserved Offering conducted in September and the December investment of 5 million by Sonova. Sensorion intends to use the new funds to develop its current gene therapy programs (OTOF-GT, GJB2-GT and USHER-GT), to support its pharmacology and clinical studies of SENS-401 and for general corporate purposes.

Nawal Ouzren, CEO of Sensorion commented: We are working hard to secure further approvals of the protocol amendment to reduce the sample size for the SENS-401 Phase 2 SSNHL trial which will help ensure that we could complete this study on time. Sensorion will also engage with regulatory authorities in Europe and the US to discuss our potential Otorferlin gene therapy clinical trial design. We recently added a promising third program to the Sensorion gene therapy pipeline centered around the GJB2 gene, the most prevalent cause of congenital deafness. This program will focus on major new markets with an estimated patient population of 300,000 children and adults in Europe and the United States. We remain very focused on delivering on our ambitious goals this year and we will stay true to our vision to serve patients with hearing loss.

Expected future milestones and estimated timelines:

2020 financial results

The annual accounts at 31 December 2020, drawn up according to IFRS standards and approved by the Board of Directors on 17 March 2021, have been duly reviewed by statutory auditors.

The simplified income statement as of 31 December 2020 is as follows:

In Euros IFRS standards

31.12.2020

31.12.2019

Operating income

2,421,267

2,522,717

Research & Development expenses

-7,679,365

-10,208,520

General & Administrative expenses

-3,631,123

-3,128,236

Total operating expenses

-11,310,488

-13,336,756

Operating profit/loss

-8,889,220

-10,814,039

Financial charges

-88,869

-1,282,141

Net profit/loss

-8,978,089

-12,096,181

For the year ended 31st December 2020, Sensorion reported operating income of 2.4 million, which included 1.8 million in research tax credit and 0.6 million in grants from Audinnove (RHU) and Patriot (PSPC) collaborations. The decrease of -0.1 million compared to 2019 is explained by a decrease of -0.7 million in research tax credit partly offset by an increase of +0.6 million in grants.

Operating expenses declined by 15% from 13.3 million in 2019 to 11.3 million for fiscal year 2020.

R&D expenses decreased by 2.5 million mainly due to the completion of the SENS-111 clinical trial in 2019 and a slowdown of SENS-401 activities linked to the global Covid-19 pandemic situation, and partly offset by an increase in headcounts to strengthen the R&D team with Gene Therapy expertise.

G&A expenses increased by 0.5 million, mainly driven by an increase in headcount.

Operating loss at 31 December 2020 was -8.9 million compared with -10.8 million at 31 December 2019.

The net financial charges decreased by 1.3 million compared to 2019 which was mainly due to the costs related to the convertible bond operations.

Net loss was -9.0 million at 31 December 2020 compared with -12.1 million at 31 December 2019.

As of 31 December 2020, the company employed 28 persons.

Financial structure

The simplified balance sheet at 31 December 2020 is as follows:

In Euros IFRS standards

31.12.2020

31.12.2019

Non-current Assets

1,474,117

1,724,348

Other Current Assets

4,254,909

5,946,864

Cash & cash equivalent

62,174,948

30,428,319

Total Assets

67,903,976

38,099,532

Equity

58,379,653

13,218,525

Non-current Liabilities

5,246,408

2,036,933

Current Liabilities

4,277,915

22,844,074

Total Liabilities

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Sensorion Reports Full-Year 2020 Financial Results and Provides Business Highlights - Business Wire

BOONE, Iowa, March 19, 2021 /PRNewswire/ -- Gene therapy company ENDSULIN has expanded to a second study site at Boone Veterinary Hospital, where it is looking for diabetic dogs to participate in a study to evaluate the effectiveness of a treatment that could reduce or even eliminate insulin injections.

The study could be a critical step in advancing a one-time gene therapy treatment for diabetes in both dogs and humans. The treatment, which has proven efficacy in hundreds of small animals, is based on more than 25 years of research at the University of WisconsinHospitals and Clinics in Madison, where the company is based.

"We hope to free families who are caring for their diabetic pets around the clock," said Hans Sollinger, ENDSULIN founder. "Giving dogs and their families their independence back is a step in our mission to do the same for millions of people suffering with diabetes."

ENDSULIN covers the cost of the procedure, which takes about 30 minutes total and is administered by a certified veterinarian. After treatment, the ENDSULIN team will periodically monitor dogs' health to observe the long-term effects.

While this particular gene therapy is novel, dogs treated with gene therapies in Barcelona, Spain have been followed for up to 8 years, with no evidence of adverse events.

Ideal dogs are small, and have been recently diagnosed with diabetes. Families must be able to bring their pet to either the Boone or Waunakee, WI, clinics for the one-time treatment and five follow-up visits, and also provide follow-up information to the ENDSULIN research team.

People with diabetic dogs can learn more about the study or ask about enrollment at endsulin.com/pilot-study, or contact ENDSULIN directly at [emailprotected].

ABOUT ENDSULINENDSULIN is reshaping the way we approach a cure for diabetes. They are working to free patients from daily injections and 24/7 management using the most cutting-edge gene therapy technology, developed from decades of research by noted diabetes leader Hans Sollinger, MD, PhD, Dr hc, at the University of Wisconsin Hospitals and Clinics. Their sole focus is to get a durable, one-time treatment to the millions of people who need it.

SOURCE ENDSULIN

https://www.endsulin.com

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Diabetic Dogs Needed for Study that Could Reduce or Eliminate Insulin Injections - PRNewswire

NEW YORK, March 24, 2021 /PRNewswire/ --Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium" or the "Company") today announced that its collaborator, Memorial Sloan Kettering Cancer Center ("MSK"), has commenced patient enrollment in the Phase 1 study evaluating Iomab-ACT for targeted conditioning prior to treatment with MSK's CD19 targeted CAR T-cell 19-28z. Iomab-ACT is a low dose version of Actinium's Phase 3 drug candidate Iomab-B, a CD45 targeting antibody radiation conjugate ("ARC"). Actinium and MSK were jointly awarded National Institutes of Health Small Business Technology Transfer grant funding for this first ever trial to evaluate ARC-based targeted conditioning prior to CAR-T therapy. The scientific rationale for this trial builds on preclinical data published in 2020 and is further supported by clinical observations from the SIERRA trial to justify combining MSK's 19-28z CAR T-cell therapy with Iomab-ACT. Manufacturing of patient CAR T-cells has commenced and patient conditioning with Iomab-ACT followed by 19-28z CAR T-cell infusion is expected early in the second quarter of 2021, with proof-of-concept data expected in the second half of 2021.

Results of a Phase 2 trial in 53 patients with relapsed and refractory B-cell acute lymphoblastic leukemia with MSK's 19-28z CAR-T published in the New England Journal of Medicine reported complete remissions in 83% (44/53) of patients, median event-free survival (EFS) of 6.1 months and median overall survival (OS) was 12.9 months at a median follow up period of 29 months (range 1 65 months). There was a 26% (14/53) rate of Grade 3 of greater cytokine release syndrome (CRS), with 1 patient death as a result, and 42% of patients experienced Grade 3-4 immune effector cell-associated neurotoxicity syndrome (ICANS).

Dr. Dale Ludwig, Actinium's Chief Scientific and Technology Officer, said "MSK's 19-28z CAR T-cell therapy has produced high response rates in patients with relapsed or refractory B-ALL who have previously undergone several lines of standard therapy. However, toxicities such as cytokine release syndrome and neurologic toxicity, as well as durability of response, remain a significant challenge. The ARC technology Iomab-ACT employs enables the delivery of targeted radiation that selective and specifically targets immune cells including those implicated in the CAR-T-associated toxicities of neurotoxicity and cytokine release syndrome. We are eager to begin treating patients in this first of its kind pilot study to explore the potential of Iomab-ACT targeted lymphodepletion to modulate the immune system and improve the safety profile of CAR T-cell therapy. We are hopeful this technology may ultimately enhance our ability to deliver CAR T-cell therapies more safely. Positive results from pilot study could have a meaningful impact on the way we condition patients for CAR-T and other adoptive cell therapies, which have transformed the treatment of patients with blood cancers."

Sandesh Seth, Actinium's Chairman and CEO, said "This is a major milestone for Actinium and one we are very excited by. Adoptive cell therapies like CAR-T and gene therapies have emerged as some of the most promising areas in medicine and hold tremendous promise for patients, many of whom have limited or no treatment options remaining. This promise has led to a large and growing field of therapies where we intend to establish Iomab-ACT as the universal solution for targeted, non-chemotherapy conditioning, that harnesses the power of radiation. With Iomab-B nearing complete enrollment in the Phase 3 SIERRA trial for bone marrow transplant conditioning, starting to treat patients in this Iomab-ACT trial for CAR-T conditioning could not come at a better time. We look forward to continuing to build out our strategic business unit in targeted conditioning to best serve patients seeking potentially curative bone marrow transplant, adoptive cell therapy and gene therapy to improve patient access and outcomes."

About Iomab-ACT

Iomab-ACT targets cells that express CD45, an antigen found on immune cells such as lymphocytes and macrophages as well as leukemia and lymphoma cancer cells and delivers the radioisotope warhead iodine-131 to achieve cell depletion. Iomab-ACT is intended to deplete CD45+ immune cells such as macrophages that are implicated in CAR-T related toxicities and may also have an anti-tumor effect on chemo-refractory cancers. Iomab-ACT is a low dose extension of Actinium's lead program, Iomab-B, which is being studied in a pivotal Phase 3 trial for targeted conditioning prior to a bone marrow transplant. Preclinical data supporting Iomab-ACT's application in targeted lymphodepletion prior to ACT such as CAR-T was recently published in the journalOncotarget(https://www.oncotarget.com/archive/v11/i39/).

In addition, clinical data with trace doses of Iomab-B has shown transient, reversible lymphodepletion in patients and drug clearance pharmacokinetics that fit within the vein-to-vein time of CAR-T manufacturing and administration.

About Actinium Pharmaceuticals, Inc. (NYSE: ATNM)

Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, I-131 apamistamab (Iomab-B) is being studied in the ongoing pivotal Phase 3 Study of Iomab-B in Elderly Relapsed or Refractory Acute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over seventy-five percent enrolled and positive single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. Iomab-ACT (low dose I-131 apamistamab) is also be studied as a targeted conditioning agent in a Phase 1 study with a CD19 CAR T-cell Therapy with Memorial Sloan Kettering Cancer Center and is intended to be studied for conditioning prior to gene therapy. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 140 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc. Website:https://www.actiniumpharma.com/

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Hans Vitzthum LifeSci Advisors, LLC[emailprotected](617) 430-7578

SOURCE Actinium Pharmaceuticals, Inc.

http://www.actiniumpharma.com/

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Actinium Announces Initiation of Patient Enrollment in Iomab-ACT Trial for Targeted Conditioning Prior to CD19 CAR T-Cell Therapy - PRNewswire

CAMBRIDGE, Mass.--(BUSINESS WIRE)--ElevateBio, a cell and gene therapy technology company focused on powering transformative cell and gene therapies, today announced that the companys Chief Scientific Officer of Regenerative Medicine, Dr. Melissa Carpenter, has been appointed to the International Society for Stem Cell Research (ISSCR) Board of Directors. In this role, Dr. Carpenter will work with the ISSCR officers and board to advance the organizations mission of bringing together researchers, clinicians, academics, and industry to promote excellence in stem cell science and applications to human health.

I am honored to have been elected to, and serve on the Board of, the ISSCR and foster the continued progress in advancing stem cell science alongside this impressive leadership and fellow board members, said Melissa Carpenter, PhD, Chief Scientific Officer of Regenerative Medicine at ElevateBio. Collaboration across the stem cell professional community is critical to our ability to translate promising stem cell research and regenerative medicine science into treatments that can have dramatic benefit for global human health globally.

Dr. Carpenter served on the ISSCR Task Force to revise the Guidelines for Stem Cell Research and Clinical Translation that will be released in May and advocated in support of the value of stem cell research as part of the Societys 2019 Advocacy Day, meeting with members of the U.S. Congress. She has also served on the Clinical Translation Committee.

We are delighted to welcome Melissa Carpenter to the ISSCR Board of Directors, said Christine Mummery, ISSCR President. Melissas dedication to supporting the translation of stem cell discoveries into therapeutics and her leadership has been crucial for advancing the clinical development of multiple therapies. Her experience will be an asset to the Board as the field of stem cell science continues to rapidly evolve.

The International Society for Stem Cell Research is the preeminent global, cross-disciplinary, science-based organization dedicated to stem cell research and its translation to the clinic. With nearly 4,000 members from more than 60 countries, the ISSCR mission is to promote excellence in stem cell science and applications to human health.

About ElevateBio:

ElevateBio is a cell and gene therapy technology company built to power the development of transformative cell and gene therapies today and for many decades to come. The company has assembled industry-leading talent, built world-class facilities, and integrated diverse technology platforms necessary for rapid innovation and commercialization of cell, gene, and regenerative therapies. The company has built an initial technology stack, including gene editing, induced pluripotent stem cells, and protein, viral, and cellular engineering. At the center of the business model is ElevateBio BaseCamp, a centralized R&D and manufacturing company that offers research and development (R&D), process development (PD), and Current Good Manufacturing Practice (CGMP) manufacturing capabilities. The company is focused on increasing long-term collaborations with industry partners while also continuing to develop its own highly innovative cell and gene therapies. ElevateBio's team of scientists, drug developers, and company builders are redefining what it means to be a technology company in the world of drug development, blurring the line between technology and healthcare.

ElevateBio is headquartered in Cambridge, Mass, with ElevateBio BaseCamp located in Waltham, Mass. For more information, visit us at http://www.elevate.bio, or follow Elevate on LinkedIn, Twitter, or Instagram.

*As of the date of this press release, SoftBank Group Corp. has made capital contributions to allow investments by SoftBank Vision Fund 2 ("SVF 2") in certain portfolio companies. The information included herein is made for informational purposes only and does not constitute an offer to sell or a solicitation of an offer to buy limited partnership interests in any fund, including SVF 2. SVF 2 has yet to have an external close, and any potential third-party investors shall receive additional information related to any SVF 2 investments prior to closing.

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ElevateBio Announces Chief Scientific Officer of Regenerative Medicine, Melissa Carpenter, PhD, Elected to the International Society for Stem Cell...

Dive Brief:

Sarepta has three marketed products treating Duchenne, plus a beefy pipeline of 39 experimental RNA-based programs and genetic medicines for rare diseases. One towers above them all, however: SRP-9001. Its clinical trial setback in January cut the company's valuation in half as a rival treatment from Pfizer appeared to be taking a lead.

With the path to market looking longer for SRP-9001, other pipeline projects may figure more prominently in Sarepta's outlook, and the data from the follow-up project, known as SRP-9003, may be reassuring to investors.

SRP-9003 treats a type of muscular dystrophy called Limb-girdle that particularly affects the arms and legs. To do so, it helps replenish the deficiency of a protein called beta-sarcoglycan, the lack of which is thought to trigger the disease.

The first three patients in Sarepta's early-stage trial were infused with a low dose of the gene therapy, and after 18 months of treatment had improved their score by 5.7 points on a 20-item test that measures their ability to do such tasks as stand up from a chair or stand briefly on one foot. That improvement was sustained at two years, Sarepta reported Thursday.

In a research note, SVB Leerink's Schwartz wrote that untreated patients would have been expected to decline by 4.6 points over the same time period. Biological measures, such as expression of the beta-sarcoglycan protein, also showed positive signs, although they were short of expression seen in the healthy population.

Three higher-dose patients also showed improvements at one year. Their four-point increase on the functional test was short of the six points seen in the lower-dose group at 12 months, although Schwartz noted that biomarkers indicated the high-dose group may have been less disabled when they received the gene therapy.

The company hasn't seen any new safety signals. One adverse event was reported in an earlier data release, a patient who was dehydrated from vomiting.

Schwartz wrote that the data could set the stage for a study that could be submitted to regulators to support approval. There are no treatments for limb-girdle muscular dystrophy, so a regulatory pathway will need to be discussed with the Food and Drug Administration, as well as a quality assessment of the batch of gene therapy to be used in that trial. That could make the path forward for limb-girdle gene therapy different than the one Sarepta's traversing in Duchenne, where there are at least some marketed drugs for the condition.

The next study should begin this year, Schwartz wrote.

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With new results, Sarepta's 2nd gene therapy holds steady - BioPharma Dive

GERMANTOWN, Md., March 18, 2021 (GLOBE NEWSWIRE) -- Orgenesis Inc. (NASDAQ: ORGS) (Orgenesis or the Company), a global biotech company working to unlock the full potential of celland gene therapies, announces that it has entered a collaboration with MIDA Biotech B.V. (MIDA). Together, the companies will work to establish point-of-care centers at hospitals and other medical institutions across western Europe.

Orgenesis and MIDA plan to deploy Orgenesis Mobile Processing Units and Labs (OMPULs) at leading hospitals for the onsite development of promising cell and gene therapies and immunotherapies from MIDA. The OMPULs are multi-purpose, mobile, autonomous good manufacturing practice (GMP) facilities used to develop, optimize, and manufacture cell and gene therapies at the point of care. Via the collaboration, the teams will align with various hospitals with projects focused on scaling the therapies through to commercialization with regulatory compliance and governmental approval standards as guiding principles.

In connection with the Agreement, Orgenesis and MIDA entered into agreements to establish point-of-care centers and deploy OMPULs within leading hospitals in Italy, Germany, Spain and Benelux, as well as other activities including joint research, development and validation activities related to the development of cell and gene therapies.

These agreements mark a major milestone towards the commercial launch of our OMPULs across Europe, and we believe provide further validation of our platform, as each of the respective hospitals conducted extensive due diligence and verification around our technology and capabilities prior to signing the agreements, stated Vered Caplan, CEO of Orgenesis. We see MIDA as an ideal partner based on their established relationships with the leading medical centers across western Europe and their promising science. Our teams are aligned in the desire to help lower costs and eliminate the logistical nightmares of building a centralized production facility or cleanrooms in the hospitals, which can stand in the way of getting a therapy to market effectively.

The additional sites will significantly expand the Orgenesis POCare Network capacity, which already includes active development centers in the United States, Belgium, Israel, and South Korea, as well as a growing number of joint venture agreements with regional partners and ongoing therapeutic development programs.

About OrgenesisOrgenesis is a global biotech company working to unlock the full potential of celland gene therapies (CGTs) in an affordable and accessible format at the point of care. The Orgenesis POCarePlatform is comprised of three enabling components: a pipeline of licensedPOCare Therapeuticsthat are processed and produced in closed, automatedPOCare Technologysystems across a collaborativePOCare Network. Orgenesisidentifies promising new therapies and leverages its POCare Platform to provide a rapid, globally harmonized pathway for these therapies to reach and treat large numbers of patients at lowered costs through efficient, scalable, and decentralized production. The POCare Network brings together patients, doctors, industry partners, research institutes and hospitals worldwide to achieve harmonized, regulated clinical development and production of the therapies. Learn more about the work Orgenesis is doing atwww.orgenesis.com.

Notice Regarding Forward-Looking StatementsThis press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities and Exchange Act of 1934, as amended. These forward-looking statements involve substantial uncertainties and risks and are based upon our current expectations, estimates and projections and reflect our beliefs and assumptions based upon information available to us at the date of this release. We caution readers that forward-looking statements are predictions based on our current expectations about future events. These forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and assumptions that are difficult to predict. Our actual results, performance or achievements could differ materially from those expressed or implied by the forward-looking statements as a result of a number of factors, including, but not limited to, our reliance on, and our ability to grow, our point-of-care cell therapy platform, our ability to achieve and maintain overall profitability, our ability to manage our research and development programs that are based on novel technologies, our ability to control key elements relating to the development and commercialization of therapeutic product candidates with third parties, the timing of completion of clinical trials and studies, the availability of additional data, outcomes of clinical trials of our product candidates, the potential uses and benefits of our product candidates, our ability to manage potential disruptions as a result of the coronavirus outbreak, the sufficiency of working capital to realize our business plans, the development of our POCare strategy, our trans differentiation technology as therapeutic treatment for diabetes, the technology behind our in-licensed ATMPs not functioning as expected, our ability to further our CGT development projects, either directly or through our JV partner agreements, and to fulfill our obligations under such agreements, our license agreements with other institutions, our ability to retain key employees, our competitors developing better or cheaper alternatives to our products and the risks and uncertainties discussed under the heading "RISK FACTORS" in Item 1A of our Annual Report on Form 10-K for the fiscal year ended December 31, 2020, and in our other filings with the Securities and Exchange Commission. We undertake no obligation to revise or update any forward-looking statement for any reason.

Contact for Orgenesis:Crescendo Communications, LLCTel: 212-671-1021Orgs@crescendo-ir.com

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Orgenesis Enters into Collaboration Agreement with MIDA to Deploy OMPULs for Point-of-Care Research and Development of Promising Cell and Gene...

SparingVision Announces Upcoming Presentations at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting

Paris, March 19, 2021 SparingVision (the Company), a genomic medicine company developing vision saving treatments for ocular diseases, announces today that three abstracts highlighting the companys recent research into ocular diseases and its lead gene therapy treatment SPVN06 have been accepted for the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting, to be held virtually from 1-7 May. The three abstracts will be given as poster presentations for which the details can be found below.

Title: SPVN06, a Novel Mutation-Independent AAV-based Gene Therapy, Protects Cone Degeneration in a Pig Model of Retinitis PigmentosaDate and Time: May 3, 2021 from 11:15 AM to 1:00 PM EDT

Presenter: Dr. Jennifer Noel, University of LouisvilleSession Title: Drug delivery and Gene Therapy

Title: Correlations between progression markers in rod-cone dystrophy due to mutations in RHO, PDE6A, or PDE6BDate and Time: May 3, 2021 from 4:30 PM to 6:15 PM EDT

Presenter: Dr. Daniel Chung, Chief Medical Officer, SparingVisionSession Title: Visual Impairment - Assessment and Measurement

Title: A 1-Month Toxicology and Biodistribution NHP Pilot Study Evaluating a Single Subretinal Bilateral Administration of SPVN06 - A Novel AAV-Based Gene Therapy for the Treatment of Rod-Cone Dystrophies Agnostic of the Causative Mutation Date and Time: May 5, 2021 from 2:45 PM to 4:30 PM EDT

Presenter: Dr. Melanie Marie, SparingVisionSession Title: AMD and retinal physiology

**ENDS**

Contacts:

NOTES TO EDITORS:

About SparingVision:SparingVision is a genomic medicines company, translating pioneering science into vision saving treatments. Founded to advance over 20 years of world-leading ophthalmic research from its scientific founders, SparingVision is leading a step shift in how ocular diseases are treated, moving beyond single gene correction therapies. At the heart of this is SPVN06, a gene independent treatment for retinitis pigmentosa (RP), the most common inherited retinal disease affecting two million people worldwide. SPVN06 could form the basis of a suite of new sight saving treatments as it could be applicable to many other retinal diseases, regardless of genetic cause.

The Company is supported by a strong, internationally renowned team who aim to harness the potential of genomic medicine to deliver new treatments to all ocular disease patients as quickly as possible. SparingVision has raised 60 million to date and its investors include 4BIO Capital, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, UPMC Enterprises, Jeito Capital and Ysios Capital. For more information, please visit http://www.sparingvision.com.

About SPVN06:SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy approach comprised of one neurotrophic factor and one enzyme reducing oxidative stress which, acting synergistically, aim at slowing or stopping the degeneration of cone photoreceptors, which inevitably leads to blindness in patients with rod-cone dystrophies (RCD). SparingVisions primary disease target is Retinitis Pigmentosa (RP), one of the most common inherited retinal diseases that affects two million patients worldwide. There is currently no treatment approved to treat RP patients independently of their genetic background. This approach is potentially applicable to many more diseases where the loss of rods is known to be an early signal of the disease. First-in-man trials, with SPVN06 in patients with RP, will be commencing in H2 2021.

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SparingVision Announces Upcoming Presentations at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting - GlobeNewswire

24 Mar 2021

FPIs raison detre is to get effective, international clinical trials on the road (see Part 6of this series). At ICFTD, FPIs co-leader, Adam Boxer of the University of California, San Francisco, summarized what trials exist already, and offered a glimpse of the future.

Currently, the field lacks biomarkers for the underlying neuropathology of FTD, so there is no concrete way to know whether tau fibrils or TDP-43 inclusions lurk within the brain of a patient who has no known autosomal-dominant mutation. Hence, present-day FTD trials are limited to carriers of pathogenic C9ORF72, GRN, or MAPT mutations, and to people whose clinical syndromes are known to be tauopathies, such as progressive supranuclear palsy.

Trials for carriers of GRN mutations are the farthest along. At centers that are part of FPI, Alector is conducting Phase 2 and Phase 3 trials evaluating AL001, an anti-sortilin antibody meant to slow progranulins degradation. Early trials of AL001 indicated that it restores CSF progranulin levels in mutation carriers. The Phase 3 trial is the first in the field to include presymptomatic mutation carriers. They can enroll if their plasma NfL is elevated, suggesting they are nearing symptom onset. The trial aims for 180 participants total, and also includes symptomatic carriers. It uses the CDR-NACC-FTLD as a clinical primary endpoint, and is slated to run through 2023.

Two companiesPrevail Therapeutics (recently acquired by Eli Lilly & Company) and Passage Bioare pursuing a gene-therapy approach. Both are hoping to replenish progranulin levels by injecting an adeno-associated virus bearing the progranulin gene directly into the cisterna magna of GRN mutation carriers with FTD. Prevails trial of PR006started in July 2020, while Passage Bios trial of PBFT02 is slated to start this month.

Julio Rojas of the University of California, San Francisco, said the combination of sensitive disease biomarkers (CSF progranulin, plasma NfL, and others) and progranulin-targeted treatment in trials bodes well for this form of familial FTD. Its likely well see a treatment for GRN mutation carriers with FTD before we see one for AD, Rojas said.

Carriers of hexanucleotide expansions in the C9ORF72 gene are also being included within the progranulin umbrella. Owing to the lysosomal dysfunction wrought in both familial forms of the disease, C9ORF72 carriers are included in Alectors Phase 2 study of AL001.

Other trials are targeting the C9ORF72 mutation specifically. They enroll people with ALS and/or FTD. The furthest along is Ionis/Biogens antisense oligonucleotide BIIB078, in Phase 1. The multiple-ascending-dose trial includes 114 people with ALS; results are expected later this year. Participants in the randomized portion of the trial are being enrolled in a two-year, open-label extension. WaveLife Sciences, Cambridge, Massachusetts, is planning to start a Phase 1 trial of its C9-ASO, called WVE-004, in people with ALS and FTD due to C9ORF72 expansion this year (see press release).

Yet another C9-ASO, called afinersen, was designed in Robert Browns group at the University of Massachusetts in Worcester. At ICFTD, Boxer showed data from a man with ALS who was treated with afinersen for over a year. During this time, his CSF levels of poly-GP, a dipeptide translated from transcripts of the hexanucleotide expansion, plummeted, and his disease stayed stable during treatment.

The diabetes drug metformin is being tested at the University of Florida, Gainesville, in a Phase 1 clinical trial for C9ORF72 hexanucleotide expansion carriers with ALS or FTD. Besides its better-known effects on glucose levels and insulin sensitivity, metformin has been reported to squelch repeat-associated non-AUG (RAN) translation, the mechanism responsible for translating toxic dipeptides from the C9 repeats (Zu et al., 2020). This open-label study includes 18 participants, and is expected to finish in August 2022.

What about trials for FTLD-tau? So far, most trials have focused on people with clinical syndromes, such as progressive supranuclear palsy, which is known to have underlying tau pathology, but not necessarily a pathogenic tau mutation. Two different anti-tau monoclonal antibodies failed in trials for PSP last year (Jul 2019 news; Dec 2019 news). This prompted cancellation of a basket trial that had also included people with non-fluent primary progressive aphasia, corticobasal syndrome, and MAPT mutation carriers with FTD.

Why did these trials fail? A definitive answer is not in, but Boxer noted that CSF studies from the trials indicated that both antibodies, which target taus N-terminus, did not rid the brain of all forms of tau pathology. Boxer said the field eagerly awaits results of Biogens trial of BIIB080, a tau-targeting ASO in people with mild AD, which could provide proof-of-concept data for primary tauopathies. That Phase 2 trial will finish in May of 2022.

One hurdle to treating primary tauopathies is the lack of fluid biomarkers or PET imaging tracers for many non-AD forms of tau. While some tracers have been found to bind to the 4R-tauopathies PSP and CBD, so far none reliably tag tau in people with bvFTD or PPA, who could have underlying tau or TDP-43 pathology (Jul 2020 news). Once the field develops a marker for either one, people with sporadic FTD will be able to join clinical trials targeting their specific pathology.

MAPT mutations are the rarest cause of familial FTD, i.e., patients with these mutations are scarce. To ensure that trials in this far-flung group of patients are run with the most power possible, the FPI is working to coordinate international platform trials for them, which will test multiple therapies simultaneously against a shared placebo group, Boxer said.Jessica Shugart

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FTD Trials: The Now and the Future | ALZFORUM - Alzforum

Guest editorial: Technology to meet the challenge of efficiency in the regulated AAV vector space

In this guest editorial, based on a blog post by Gyros Protein Technologies, learn how scientists at AstraZeneca developed a reliable method for adeno-associated virus (AAV) capsid titer quantification.

Vectors based on AAV are used widely in gene therapy for in vivo gene delivery. The success of AAV-based therapeutic production depends on the availability of laboratory tools that can efficiently deliver reliable data, including the determination of virus particle titer (physical titer). To achieve this, a team at AstraZeneca searched for an immunoassay platform that could improve on ELISA in terms of dynamic range, sample volume, and productivity. Its evaluation showed that Gyrolab systemmet its needs for AAV process development, with a more reliable measurement of AAV capsid titer for in-process and purified samples.

Gene therapy has seen a massive explosion in activity since the first clinical trial in 1989, and, by the latter half of 2020, had engaged 536 advanced therapy developers, with 373 clinical trials ongoing1. There are currently three gene therapy products approved by the US Food and Drug Administration (FDA), and the field continues to expand with more than 900 investigational new drug (IND) applications for ongoing gene therapy clinical studies2.

Recombinant vectors based on the non-pathogenic AAV are currently one of the most widely used vehicles for delivery in gene therapy. Different serotypes of AAV can be used to specifically target certain tissues and organs and are the subject of intensive research to improve their properties. AAV has also been used in a vaccine against COVID-19.

Regulatory authorities are increasing their support for initiatives in cell and gene therapy, resulting in new guiding documents on gene therapy manufacturing and clinical development of products3, but there are issues with inconsistent directives that are confounding manufacturing efforts4.

Some of the biggest challenges in manufacturing AAV therapeutics concern chemistry, manufacturing, and control (CMC). Gene therapy drugs often target rare diseases with trials involving only 1020 participants, and yet organizations must conduct all the CMC operations normally required for product commercialization in much larger manufacturing batches.

Advances in AAV-based therapeutics range from vector engineering to increasing transduction efficiency, to fine-tuning tissue tropism, and the avoidance of host immune response activation, as well as optimization of the small- and large-scale vector production. Support of bioprocess development relies on the availability of fit-for-purpose analytical tools to assess potential critical quality attributes (pCQAs) that are relevant to characterization and lot release testing. In the case of AAV-based therapeutics, this includes the ability to determine the concentrations of viral genomes (genome titer) and viral particles (capsid titer). Genome titer can be determined by PCR, but determining the capsid titer is more of a challenge.

In a recent webinar on capsid titer quantification for AAV-based therapeutics, AstraZeneca scientist Dr. Tomasz Witkos talks about how miniaturized immunoassays can be used to measure purified AAV vector as well as in-process samples and how the immunoassay experimental setup can be modified to measure several AAV serotypes.

He describes the need for a method to measure titer that could meet several criteria:

The drive to engineer recombinant AAVs to improve their properties can make capsid titer determination a challenge. The few commercial kits available for measuring capsid titer did not meet AstraZenecas needs in terms of dynamic range and accuracy. The team also needed a kit that could handle small sample volumes, preferably on an automated platform to save time.

In the webinar, Witkos presents an immunoassay developed on Gyrolab platform with AAV serotype-specific antibodies that can reliably measure AAV capsid titer in purified and in-process samples, delivering accurate and precise data suitable for AAV process development. The assay is versatile, with an antibody pair comprising a capture antibody that recognizes a broad range of AAV serotypes and another that is serotype-specific.

This assay setup can be readily adapted to titer measurements of various AAV serotypes and has a broader quantification range (3 3.5 logs) compared to a commercially available capsid ELISA kit (1 1.5 logs). The Gyrolab assay could be used to test neat samples and at low dilutions, which increases reproducibility compared to ELISA, which requires greater than 1,000x dilutions. Throughput was estimated at 50 samples in four dilution series in a 5-CD run, with reproducibility in the range of 1015 % CV. The team also appreciates Gyrolab Viewer as a very valuable tool to spot any issues with AAV particle aggregation.

Find out more about how Gyrolab system is improving the productivity of gene therapy efforts at AstraZeneca, by watching the Gyros Protein Technologies webinar, Capsid titer quantification for AAV-based therapeutics.

Discover the new Gyrolab AAVX Titer Kit when used in combination with Gyrolab system, this kit is designed to quickly deliver high-quality titer data for AAV serotypes 18 and AAVrh10 using smaller sample volumes compared to ELISA.

References

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Capsid titer quantification for AAV-based therapeutics - SelectScience

PUNE, India, March 22, 2021 /PRNewswire/ --Genetic Testing Market:Global Size, Trends, Competitive, Historical & Forecast Analysis, 2021-2027", Rising incidence of blood cancer, genetic disorders, cardiovascular diseases and surge in availability of new tests are some major factors driving the growth of the global genetic testing market.

Genetic Testing Market is valued at USD 10801.98 Million in 2020 and expected to reach USD 23143.42 Million by 2027 with the CAGR of 11.5% over the forecast period.

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Genetic testing is a type of medical test that detects changes in chromosomes, genes, or proteins. The results of a genetic test can confirm a suspected genetic condition and helps to control a person's chance of developing or passing on a genetic disorder. Genetic testing, also called as DNA testing. Genetic testing can also contain measuring the results of genetic changes such as RNA analysis as an output of gene expression, or through biochemical analysis to measure specific protein output. There are various type of genetic test are available to determine disorders such as predictive testing, carrier testing, prenatal and newborn testing, diagnostic testing, pharmacogenomic testing, nutrigenomics and others. Among all, newborn testing can identify genetic disorders early in life so treatment can be started as early as possible.

Global genetic testing market has turn out to be an important topic during COVID-19 pandemic outbreak. Genetic testing market gaining huge attention due to pandemic outbreak.

Genetic testing market report is segmented on the basis of test type, application and by regional & country level. Based upon test type, genetic testing market is classified into predictive testing, carrier testing, prenatal and newborn testing, diagnostic testing, pharmacogenomic testing, nutrigenomics and others. Based upon application, genetic testing market is classified into cancer, genetic diseases, cardiovascular diseases and others.

Genetic Testing MarketSegmentation

By Test Type:

By Application:

Primary Key Players who are working in Genetic Testing Market:

Genetic testing market report covers prominent players are Ambry Genetics, Illumina, Inc., IntegraGen, BGI, Roche Diagnostics, Biocartis Group NV, Bio-Helix Co. Ltd., 23andMe, Inc., bioMerieux SA, Abbott, Blueprint Genetics, Quest Diagnostics, Sequenom, Inc., Cepheid, Counsyl, Inc., deCODE Genetics, GeneDx, Genomic Health, Inc., Genomictree, Inc., LabCorp (Laboratory Corporation of America Holdings), Invitae Corporation, Luminex Corporation, Molecular MD, Myriad Genetics, Inc., Natera, Inc., Pacific Biosciences of California, Inc., Pathway Genomics Corporation, Qiagen, Siemens Healthineers AG, HTG Molecular Diagnostics, Inc. and Others.

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BGI has Launched NGS-Based HPV Test to the Global Market

News: April 20, 2018, BGI launched NGS-based HPV genotyping product, SentisTM-HPVseq, to the global market. This product provides detection of both high-risk and low-risk HPV types, which is used to screen for cervical cancer. It isrecognized as an effective method for primary cervical cancer screening. This HPV testing initiative has helped BGI expand its genetic testing portfolio and market share.

Genetic Testing Market Dynamics:

Rising incidence of blood cancer, genetic disorders, cardiovascular diseases and technological advancements and availability of new tests are some major factors driving the growth of the global genetic testing market. According to the American Heart Association, about 2,200 Americans die of disorder every day, a mean of 1 death every 40 seconds. According to Leukemia Research Foundation, every three minutes, someone is diagnosed with blood cancer more than 175,000 new cases are expected in the United States in 2019. Additionally, Leukemia is diagnosed 10 times more often in adults than children. New cases of leukemia, lymphoma and myeloma are expected to account for 10 percent of the estimated 1,762,450 new cancer cases diagnosed in the US in 2019.

According to American Cancer Society's, estimates for leukemia in the United States for 2020 are about 60,530 new cases of leukemia and 23,100 deaths from leukemia in all ages as well as about 19,940 new cases of acute myeloid leukemia in adults only. Moreover, due to the advancement in cancer research, more patients with cancer are being successfully treated will propel growth of markets. However, high costs of genetic testing and lack of skilled professionals will hamper the development of genetic testing market. According to the National Institutes of Health, the cost of genetic testing can range from under USD 100 to more than USD 2,000, depending on the nature and complexity of the test. However, innovations and advance development in genetic test and the increasing investment in cancer research is expected to boost the opportunity for the growth of genetic testing market.

Genetic Testing Market Regional Analysis:

North America is dominating the genetic testing market with the highest share due to rising awareness among the people about advanced treatment for healthcare. Presence of key players, established healthcare infrastructure, and availability of branded drugs are a number of the factors liable for its large share. Besides, favorable government initiatives and increase in number of research collaborations are some of the drivers expected to accelerate the regional market growth. The growth of genetic testing market in this region is primarily driven by the increasing blood cancer patient. In 2016, National Cancer Institutes stated that, there were an estimated 414,773 people living with leukemia in the United States. Cancer is one among the leading causes of death and disease within the U.S. The American Cancer Society (ACS) estimates that roughly 1.7 million new cases of cancer are going to be diagnosed within the U.S. in 2017.

According to The Leukemia & Lymphoma Society, an estimated combined total of 176,200 people in the US are expected to be diagnosed with leukemia, lymphoma or myeloma in 2019. Cancer usually develops in older people; 87% of all cancers in the United States are diagnosed in people 50 years of age or older. U.S. Department of Health and Human Services, the number of new cases of leukemia was 14.1 per 100,000 men and women per year and the number of deaths was 6.5 per 100,000 men and women per year.

The Asia Pacific is expected to emerge as the fastest-growing regional market over the forecast period with due to the increase in healthcare expenditure, large population rate, growing prevalence of cardiac diseases, rising awareness about early diagnosis, developing healthcare infrastructure, and availability of effective treatment in emerging countries, such as China and India. According to American Heart Association, in 2016, among NH Asians, CVD caused the deaths of 11,023 males and 10,672 females. In the Asia-Pacific region, rising disposable income, adoption of western lifestyle, living more sedentary lives and consuming junk foods with higher energy and fat. Asia Pacific has leading innovation in the treatment of blood cancers by developing and providing transformational treatments that extend and enhance lives. These are the major factors increase the growth of genetic testing markets. According to leukemia study report, in 2017, there were 11,923 combined new cases of leukemia, lymphoma and myeloma in Australia. India has the third highest number of blood cancer patients in the world after the US and China. Blood cancer contributes to 7% of all cancer cases in India.

Continued Genetic TestingMarket Report

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At 11.5% CAGR, Genetic Testing Market Size Expected to Reach 23143.42 Mn by 2027 Says Brandessence Market Research - PRNewswire

Hemophilia is a genetic bleeding disorder in which an individual lacks or has low levels of a protein called a clotting factor. There are about 13 types of clotting factors that act on platelets, which are needed to initiate the clotting process. There are three forms of hemophilia: A, B, and C. Hemophilia A is the most common form and is caused by a deficiency of clotting factor VIII. Hemophilia B is caused by a deficiency of clotting factor IX, and hemophilia C is caused by a deficiency of clotting factor XI. Hemophilia cannot be cured with current treatment options, which not only reduces symptoms such as spontaneous bleeding in muscles and joints, but increases the risk of intracranial bleeding.

The Research Insights has added a new market aptitude report to its extensive collection of research. The report is titled as Hemophilia Gene Therapy Market which emphases in describing the primary prospects and outlines in the market. Moreover, it gives a broad overview of the global market including the cataloguing, descriptions and executions. Additionally, it also converses the growth strategies along with the cost structures and production processes.

Hemophilia Gene Therapy market is expected to exhibit robust growth rate during the forecast period. Growing inclination towards digital payments has significantly driven the overall online payment gateways market. Various national governments as well banking organizations are now encouraging digital payment in order to reduce their operating costs and better visibility of transactions.

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Top Key Vendors in Market: Spark Therapeutics, Ultragenyx, Sangamo Therapeutics, Bioverativ, Shire PLC, Freeline Therapeutics, BioMarin, uniQure

The report has been put together in a chapter-wise arrangement, by separating required illustrations transversely. This report is an expedient tool to get responses to some of the queries that hold significance for the growth of the Hemophilia Gene Therapy market during the forecast period. The evidence in the report was congregated from qualified organizations & dependable sources and was further authenticated by industry specialists for increased integrity.

The report also uses feedbacks given by industry experts to support the present and new players in enclosing effective business policies in the upcoming years. The report has been accumulated by taking the aid of info graphics, charts and tables to present the historical data and appraised figures of the Hemophilia Gene Therapy Market.

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This report is a thorough piece of work and assembled by primary as well as secondary research. The top segments in the Hemophilia Gene Therapy market have been emphasized clearly in the report for the readers to comprehend in a condensed manner. These sectors have been presented by giving information on their existing and anticipated state by the end of the forecast period.

The major stratagems approved by the well-known players for a better diffusion in the Hemophilia Gene Therapy market also forms a key section. The global market has also been analyzed in terms of revenue and also determines the regional outlook. The market crescendos such as market drivers, challenges, opportunities, and trends have been also presented.

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Table of Content:

Global Hemophilia Gene Therapy Market Research Report 2020-2026

Chapter 1: Industry Overview

Chapter 2: Hemophilia Gene Therapy Market International and China Market Analysis

Chapter 3: Environment Analysis of Hemophilia Gene Therapy.

Chapter 4: Analysis of Revenue by Classifications

Chapter 5: Analysis of Revenue by Regions and Applications

Chapter 6: Analysis of Hemophilia Gene Therapy Market Revenue Market Status.

Chapter 7: Analysis of Hemophilia Gene Therapy Industry Key Manufacturers

Chapter 8: Sales Price and Gross Margin Analysis

Chapter 9: Marketing Trader or Distributor Analysis of Hemophilia Gene Therapy.

Chapter 10: Development Trend of Hemophilia Gene Therapy Market 2020-2026.

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Hemophilia Gene Therapy Market is Thriving by World during Upcoming Year | Top Companies: Spark Therapeutics, Ultragenyx, Sangamo Therapeutics,...

NEW YORK, March 24, 2021 /PRNewswire/ --The push for psychedelic medicine is surging across North America and around the world. Scientific evidence supports the life-changing impact this safe and natural alternative has in treating mental illness of all types. New research from a number of organizations, including Johns Hopkinsin Baltimore and Imperial Collegein London, which revealed that patients with depressive disorders had a clinically significant positive response to psilocybin-assisted therapy may represent a therapeutic breakthrough in treating multiple neurological disorders. As research expands, so does support for the revolutionary treatment, which has actually been around for centuries. As seen before, anecdotal opinions often trigger scientific research which then, with validation, drives growth in the industry as well investor interest. That's exactly what's occurring in the psychedelic therapeutics space, major upticks in both new companies entering the space as well as investors excited about the possibilities. Potential stakeholders in this game-changing new market are looking for companies that can weather the complex and expensive process of running clinical trials and bringing a drug to market. A leader in psychedelic therapeutic research and drug development, Cybin Inc. (NEO: CYBN) (OTCQB: CLXPF)(Profile)has a strong clinical pipeline with phase 2 trials underway, holds ten provisional patents,and boasts a proven leadership and a time-tested tradition. Other companies vying for position in the psychedelics medicine space include COMPASS Pathways Plc (NASDAQ: CMPS), Mind Medicine Inc (OTCQB: MMEDF) (NEO: MMED), Numinus Wellness Inc. (OTCPK: LKYSF) (NEO: NUMI) (TSX.V: NUMI) and Field Trip Health Ltd.(OTCQX: FTRPF) (CSE: FTRP). Each of these companies is focused on providing better treatments for the millions suffering from mental health issues.

Click hereto view the custom infographic of theCybin editorial.

The Psychedelic Answers

More than 700 million people worldwide struggle with some form of mental illness, be it depression, addiction or post-traumatic stress disorder and the people counted are just those who seek help. The actual number is likely much higher, given the high frequency of nondiagnosis as well as stigmas and lack of effective treatments. One in four people will face a mental or neurological disorder at some point in their lives, yet "current treatments and the dominant model of mental health care do not adequately address the complex challenges of mental illness, which accounts for roughly one-third of adult disability globally."

More and more people are looking outside conventional protocols for solutions, and they're finding those solutions in the world of psychedelic medicine, specifically psilocybins. Psilocybins are a hallucinogenic substance found in certain types of mushroom, dubbed magic mushrooms, and used for centuries by indigenous cultures for religious, spiritual and health-related purposes. As is often the case, the modern world is learning from the ancient as mounting evidence points to these prolific fungi as a source for long-sought-for help in mental health and neurological disorders.

A Legacy of Success

Cybin Inc. (NEO: CYBN) (OTCQB: CLXPF)is intent on becoming the leader in this exciting breakthrough for mental well-being. The company is at the forefront of the revolution in mental health therapeutics and is developing a new class of psychedelic medicines and treatment protocols. Driving its commitment to excellence is the company's impressive leadership team of experienced professionals with a combined 80-plus years in the pharmaceutical industry.

CEO Doug Drysdale has three decades in the health-care sector, successfully building and turning around three pharmaceutical companies, and co-founder Eric So has raised hundreds of millions for various companies, directing value creation and strategic exits. Cybin's executive team has the experience and critical insights to navigate through the complexities the promising psychedelics industry offers.

Staffed with luminaries, the company's scientific team,helped develop widely used drugs such as Allegra, Sabril, Anzemet and Vaniqa, and is the only scientific team to have successfully commercialized a psychedelic drug to date.In addition, the team has facilitated more than $2 billion in pharmaceutical sales alongside being collectively involved in 37 exits across the biotech sector and various other verticals. Cybin's leadership team has a rich legacy of success and has the proven ability to both guide the company and maximize opportunity.

Tested, Proven Fundraising Ability

The importance of being well funded can't be overlooked and is especially important for research and drug development. Cybin and its leadership team have demonstrated ability to raise funds for key, strategic steps necessary for success. Most recently, Cybin raised C$45 million in the largest go-public capital raise in the Canadian psychedelic sector and a total of C$88 million across Seed, Series-A and Series-B financing rounds.

Part of that capital was used in the company's strategic acquisition of Adelia Therapeutics Inc., whose novel psychedelic molecules allowed Cybin to diversity its portfolio and provided access to multiple future indications. The acquisition also resulted in Cybin obtaining a range of technologies related to novel therapeutic delivery methods and therapeutic regimens, along with six patent applications. The acquisition also brought with it an expanding library of psychedelic derivative drug development candidates, with the first lead compounds expected to enter clinical studies this year.

A Three-Pillar Pipeline

Cybin is committed to a strong, well-established IP portfolioand clinical pipeline, as indicated by the Adelia acquisition. The company leads the industry in therapeutic development programs and innovative drug delivery systems using a three-pillar strategy with its novel drug-discovery platform, optimal novel and proprietary drug-delivery systems, and an innovative treatment regimen.

The company's IP model is diverse, covering chemically synthesized molecules, delivery mechanisms, screeners, protocols and new drug formulations alongside a merger and acquisition strategy focused on acquiring proprietary technologies and novel compounds and molecules. In addition, Cybin holds worldwide exclusive rights to sublingual film delivery technology from Intelgenx for the delivery of psilocybin and other psychedelic molecules.

In addition, the company has key partnerships in place. Cybin recently partnered with neurotech pioneer Kernelto leverage its Kernel Flow, an innovative technology designed to detect hemodynamic changes in the brain that pulses light through the skull and into the bloodstream to measure how much oxygen the blood is carrying at any given time. Cybin anticipates that the quantitative measurements enabled by Flow may improve the development, delivery and scaling of its psychedelic therapeutics.

"Access to Kernel's innovative Flow technology adds another exciting dimension to the investigative work that Cybin is doing to develop breakthrough treatments for mental health disorders such as depression and addiction," said CEO Doug Drysdale. "Currently, clinical investigators rely on limited subjective information from patients. The ability to collect quantitative data from our sponsored drug development programs is potentially game-changing in terms of our ability to measure where psychedelics work in the brain in real time, and how we ultimately design our future therapeutics. . . . This new cornerstone component of our sponsored clinical programs follows a record-setting capital raise, listing on the NEO Exchange and the acquisition of Adelia Therapeutics Inc., which added significant scientific capabilities, novel molecules, delivery mechanisms and intellectual property."

Just this week Cybin announced that it had signed a drug-development agreement with Catalent Inc., the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products. Cybin will be applying Catalent's proprietary Zydis(R)orally disintegrating tablet technology for the delivery of its novel deuterated tryptamine, or CYB003, a potential therapy for treatment-resistant psychiatric disorders.

"We are excited to partner with the team at Catalent with the aim of developing fast-acting, shorter-duration formulations of CYB003, recently acquired as part of our acquisition of Adelia Therapeutics," Drysdale said. "Our focus on reducing the need for health system resources, such as in-clinic therapist time, is an important part of our goal to create scalable, more accessible treatments for mental health disorders."

There's little doubt about the lack of effective treatments for mental health and neurological disorders. Scientific research is revealing a new, more effective approaches to address the massive unmet medical need. Leaders in this new frontier of medicine will likely be both appreciated by patients and rewarded by the markets.

Making a Profit and a Difference

Cybin isn't alone in the quest to make a difference in the mental health space. Savvy companies see an opportunity in psychedelic therapeutics and are jostling for position in an industry destined to make a mark.

COMPASS Pathways Plc (NASDAQ: CMPS) intends to accelerate patient access to evidence-based innovation in mental health. "We focus our efforts on those who are not helped by current treatments," states the company, which is developing its COMP360 psilocybin therapy designed to offer relief for the millions of people who suffer with treatment-resistant depression (TRD). The company is pioneering the development of this therapy, in which its proprietary formulation of synthetic psilocybin, COMP360, is administered in conjunction with psychological support.

Mind Medicine Inc (OTC: MMEDF) (NEO: MMED)is a psychedelic medicine biotech company that discovers, develops and deploys psychedelic-inspired medicines and therapies to address addiction and mental illness. The company is assembling a compelling drug development pipeline of innovative treatments based on psychedelic substances including psilocybin, LSD, MDMA, DMT and an Ibogaine derivative, 18-MC.

MMED is also actively pursuing the development of LSD-assisted therapies through its Project Lucy, including a Phase2btrial for anxiety disorders planned to be conducted fully through the FDA pathway.

Numinus Wellness Inc. (OTC: LKYSF) (NEO: NUMI) (TSX.V: NUMI)supports access to psychedelic-assisted psychotherapy through ketamine-assisted psychotherapy and special access and compassionate trials. Numinus partners with practitioners in providing clients with access to psychedelic-assisted psychotherapy, and through its recent acquisition of Mindspace, a Quebec-based psychedelic programming leader, Numinus now supports practitioners across three clinic locations.

Field Trip Health Ltd.(OTCQX: FTRPF) (CNX: FTRP), a global leader in the development and delivery of psychedelic therapies, recently partneredwith WHOOP, a human performance company, to measure the biometric effects of Field Trip's psychedelic therapies. Field Trip is opening Field Trip Health centers across North America and Europe for the delivery of psychedelic therapies, which have demonstrated significant efficacy in treating mental health conditions such as depression, anxiety and PTSD.

Surging interest in psychedelic therapeutics has sparked rising involvement from savvy companies interested in making both a profit and a difference as well as investors looking for the next space to strategically make a move. Companies such as Cybin that offer the science, the leadership and the expertise needed to succeed have a good chance of making a real difference in the nascent industry.

For more information about Cybin, please visitCybin Inc.

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Science Drives Surging Interest in Psychedelic Therapeutics - PRNewswire

Chimeric antigen receptor (CAR) T cell therapy is a new type of cancer treatment. During this treatment, healthcare professionals reprogram the immune system to attack cancer cells.

Healthcare professionals currently use CAR T therapy to treat some blood cancers. However, scientists are investigating whether or not it could also work in other cancers.

This article will explain what CAR T cell therapy is and how it works. It will also look at some possible side effects and the recovery process.

T cells are part of the immune system. They are a type of white blood cell with proteins on the surface that act as receptors.

T cells move around the blood, checking for foreign substances, such as viruses or bacteria. These foreign substances also have proteins on their surfaces. Experts call these proteins antigens.

Immune cell receptors and antigens fit together like a lock and key. Each foreign substance and T cell has a differently shaped antigen or receptor. T cells bind to antigens that fit their receptor, destroying the foreign substance.

Cancerous cells also have antigens. However, T cells rarely have the right receptor to bind to them.

CAR T cell therapy is a way of training the immune system to recognize cancerous cells. It is a type of gene or cell therapy.

Scientists add CARs to a persons T cells. These new receptors help the T cells bind and destroy cancerous cells.

Different cancers have different antigens, and scientists must adapt the treatment accordingly.

Success rates vary depending on the type of cancer a healthcare professional is using CAR T cell therapy to treat.

One 2017 review suggests that up to 90% of people with a specific form of leukemia fully recovered following this form of treatment.

However, the treatment is still very new. The Food and Drug Administration (FDA) approved the first CAR T cell therapy in 2017. So, there is still much to learn about how well it works.

Healthcare professionals may use CAR T cell therapy if traditional cancer treatments, such as chemotherapy, are ineffective or if the cancer returns.

The FDA have approved four CAR T cell therapies in the United States. Healthcare professionals can only use them to treat specific blood cancers in certain groups of people, as follows:

However, the U.S. National Library of Medicine list over 600 ongoing CAR T cell therapy clinical trials. Scientists are currently investigating the use of CAR T cell therapy in many types of cancer, including:

According to the American Cancer Society, receiving CAR T cell therapy can take a few weeks.

The process has three steps:

Healthcare professionals will collect the T cells through an intravenous (IV) line. This can take 23 hours.

Blood will flow from the persons body into a machine that will remove the white blood cells. T cells are a type of white blood cell. The machine will then send the rest of the blood back through another IV line.

Healthcare professionals will separate the T cells from the rest of the white cells and send them to a laboratory. Scientists will then add CARs to the cells, creating CAR T cells.

The scientists will wait for the cells to multiply enough to fight cancerous cells before returning them. This part of the process can take a few weeks.

The next stage will be to insert the new CAR T cells into the persons bloodstream through another IV line.

Healthcare professionals may recommend chemotherapy first to prepare the immune system for the new CAR T cells.

CAR T cell therapy can cause some side effects. The most common side effect is cytokine release syndrome (CRS).

Cytokines are chemical messengers in the immune system that support the T cells. Cytokines multiply when the CAR T cells enter the body, and this can lead to an overproduction of cytokines.

CRS can cause mild symptoms, including:

It can also cause some severe symptoms, such as:

Severe CRS can also lead to neurological problems, including:

Serious CRS can be very dangerous. People with severe CRS will need immediate treatment in intensive care. Most of the symptoms are reversible, but CRS can sometimes be fatal.

People will usually have to stay in the hospital for observation after CAR T cell therapy. The observation period varies from hospital to hospital, but it is usually a few weeks.

Side effects can develop 121 days after treatment. People are also at higher risk of infection for 2830 days after the infusion.

CAR T cell therapy is a new cancer treatment that trains the immune system to fight cancer cells. Scientists genetically modify T cells so that they can detect and fight cancerous cells. The treatment tends to be effective, but it also carries a risk of serious side effects.

CAR T cell therapy is a very new treatment that is currently only available for some blood cancers. However, hundreds of studies are currently investigating its use in other cancer types.

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CAR T cell therapy explained: Cancer types, success rate, and more - Medical News Today

Under the terms of the agreement, the CEO of the Cambridge headquartered Axol Bioscience, Liam Taylor, and the Axol senior leadership team, will take over the management of the merged businesses, with the intent to migrate the brand to Axol Bioscience.

Censos interim CEO, Dr Tom Stratford, was also appointed non-executive director of the combined board.

The new entity, according to the parties, will become a leading provider of products and services in iPSC-based neuroscience, immune cell, and cardiac modeling for drug discovery and screening markets, providing customers validated ready-to-use cell lines and a suite of services with broader expertise, robust functional data, and customization capabilities.

They also promise shorter lead times for clients. On that, Liam Taylor, CEO Axol, told BioPharma-Reporter: Doubling the size of the scientific and technical project team and moving into two sites means bandwidth for larger production scale on the product side and ability to run simultaneous projects.

The transaction was also accompanied by a fundraising round in excess of 3.8m (US$5.3m) across shareholders. The funding was led by EIS fund manager, Calculus Capita, and Par Equity, a VC firm, based in Edinburgh. Also involved in the financing of the merged entity were Jonathan Milner, founder and former CEO of Abcam and chair of the Axol Bioscience board, Intuitive Investment group, Scottish Enterprise, and SyndicateRoom.

The investment will be used to enable growth of the business along with new hires to meet customer demand.

The growth plan, said Taylor, is to increase the manufacturing scale of flagship products while keeping a robust R&D pipeline moving to productize new cell lines as well as those owned by Censo.

At the same time, the merged entity will grow through our ability to run simultaneous service projects across a greater [number of] areas. There will be limited recruitment, merely to fill current gaps in field commercial, scientific technicians and quality, he added.

Dr Milner said merging these two players in the iPSC space, which have complementary expertise and offerings, is the most direct and low risk path to gaining a more competitive market position. He said the deal will move both organizations from thriving start-ups to a more polished commercial entity that is able to meet aggressive demand increases.

Originally posted here:
UK deal sees consolidation of two players in the iPSC space - BioPharma-Reporter.com

KENILWORTH, N.J.--(BUSINESS WIRE)--Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has approved KEYTRUDA, Mercks anti-PD-1 therapy, for the treatment of patients with locally advanced or metastatic esophageal or gastroesophageal junction (GEJ) (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation in combination with platinum- and fluoropyrimidine-based chemotherapy. The approval is based on results from the Phase 3 KEYNOTE-590 trial, which demonstrated significant improvements in overall survival (OS), progression-free survival (PFS) and objective response rate (ORR) for KEYTRUDA plus fluorouracil (FU) and cisplatin versus FU and cisplatin alone, regardless of histology or PD-L1 expression status. For OS and PFS, KEYTRUDA plus FU and cisplatin reduced the risk of death by 27% (HR=0.73 [95% CI, 0.62-0.86]; p<0.0001) and reduced the risk of disease progression or death by 35% (HR=0.65 [95% CI, 0.55-0.76]; p<0.0001) versus FU and cisplatin alone. The ORR, an additional efficacy outcome measure, was 45% (95% CI, 40-50) for patients who received KEYTRUDA plus FU and cisplatin and 29% (95% CI, 25-34) for those who received FU and cisplatin alone (p<0.0001).

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue and can affect more than one body system simultaneously. Immune-mediated adverse reactions can occur at any time during or after treatment with KEYTRUDA, including pneumonitis, colitis, hepatitis, endocrinopathies, nephritis, dermatologic reactions, solid organ transplant rejection, and complications of allogeneic hematopoietic stem cell transplantation. Early identification and management of immune-mediated adverse reactions are essential to ensure safe use of KEYTRUDA. Based on the severity of the adverse reaction, KEYTRUDA should be withheld or permanently discontinued and corticosteroids administered if appropriate. KEYTRUDA can also cause severe or life-threatening infusion-related reactions. Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. For more information, see Selected Important Safety Information below.

Because esophageal cancer generally has poor survival rates, new first-line therapies are urgently needed for these patients, said Dr. Peter Enzinger, Director, Center for Esophageal and Gastric Cancer, Dana-Farber/Brigham and Womens Cancer Center. Todays approval of this indication for KEYTRUDA introduces a new option, which has shown a superior survival benefit compared to FU and cisplatin alone, for newly diagnosed patients with locally advanced or metastatic esophageal or GEJ carcinoma that is not amenable to surgical resection or definitive chemoradiation, regardless of PD-L1 expression status and tumor histology.

There have been few advances in improving survival outcomes in the first-line treatment setting for esophageal cancer over the last three decades, said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories. We are committed to putting patients first and continuing our research to help advance new approaches to potentially extend the lives of people with cancer. We thank all of the patients, their caregivers and healthcare professionals who participated in the study.

This approval was reviewed under the FDAs Real-Time Oncology Review (RTOR) pilot program and the FDAs Project Orbis, an initiative of the Oncology Center of Excellence that provides a framework for concurrent review of oncology drugs among its international partners. Under this project, the FDA, Australian Therapeutic Goods Administration, Health Canada and Swissmedic collaboratively reviewed the KEYNOTE-590 application. The application is still under review in Australia, Canada and Switzerland.

Data Supporting the Approval

The approval was based on data from KEYNOTE-590 (ClinicalTrials.gov, NCT03189719), a multicenter, randomized, placebo-controlled trial that enrolled 749 patients with metastatic or locally advanced esophageal or GEJ (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma who were not candidates for surgical resection or definitive chemoradiation. Patients were randomized (1:1) to receive either KEYTRUDA (200 mg on Day 1 every three weeks) or placebo (on Day 1 every three weeks) in combination with cisplatin (80 mg/m2 on Day 1 every three weeks for up to six cycles) plus FU (800 mg/m2 per day on Days 1 to 5 every three weeks, or per local standard for FU administration, for up to 24 months); all study medications were administered via intravenous infusion.

Randomization was stratified by tumor histology (squamous cell carcinoma vs. adenocarcinoma), geographic region (Asia vs. ex-Asia) and Eastern Cooperative Oncology Group (ECOG) performance status (PS) (0 vs. 1).

Treatment with KEYTRUDA or chemotherapy continued until unacceptable toxicity or disease progression. Patients could be treated with KEYTRUDA for up to 24 months in the absence of disease progression. The major efficacy outcome measures were OS and PFS, as assessed by the investigator according to RECIST v1.1 (modified to follow a maximum of 10 target lesions and a maximum of five target lesions per organ). The study pre-specified analyses of OS and PFS based on squamous cell histology, Combined Positive Score (CPS) 10, and in all patients. Additional efficacy outcome measures were ORR and duration of response (DOR), according to modified RECIST v1.1, as assessed by the investigator.

The study population characteristics were median age of 63 years (range: 27 to 94), 43% age 65 or older; 83% male; 37% white, 53% Asian and 1% Black; 40% had an ECOG PS of 0, and 60% had an ECOG PS of 1. Ninety-one percent had M1 disease, and 9% had M0 disease. Seventy-three percent had a tumor histology of squamous cell carcinoma, and 27% had adenocarcinoma.

The trial demonstrated statistically significant improvements in OS and PFS for patients randomized to KEYTRUDA in combination with chemotherapy compared to chemotherapy alone. Efficacy results showed:

Endpoint

KEYTRUDA + Cisplatin + FU(n=373)

Placebo + Cisplatin + FU(n=376)

OS

Number of events (%)

262 (70)

309 (82)

Median in months (95% CI)

12.4 (10.5, 14.0)

9.8 (8.8, 10.8)

Hazard ratio* (95% CI)

0.73 (0.62, 0.86)

p-value

<0.0001

PFS

Number of events (%)

297 (80)

333 (89)

Median in months (95% CI)

6.3 (6.2, 6.9)

5.8 (5.0, 6.0)

Hazard ratio* (95% CI)

0.65 (0.55, 0.76)

p-value

<0.0001

ORR

ORR, % (95% CI)

45 (40, 50)

29 (25, 34)

Number of complete responses (%)

24 (6)

9 (2.4)

Number of partial responses (%)

144 (39)

101 (27)

p-value

<0.0001

DOR

Median in months (range)

8.3 (1.2+, 31.0+)

6.0 (1.5+, 25.0+)

* Based on the stratified Cox proportional hazard model

Based on a stratified log-rank test

Confirmed complete response or partial response

Based on the stratified Miettinen and Nurminen method

In a pre-specified formal test of OS in patients with PD-L1 (CPS 10) (n=383), the median was 13.5 months (95% CI, 11.1-15.6) for the KEYTRUDA arm and 9.4 months (95% CI, 8.0-10.7) for the placebo arm, with a HR of 0.62 (95% CI, 0.49-0.78; p<0.0001). In an exploratory analysis, in patients with PD-L1 (CPS <10) (n=347), the median OS was 10.5 months (95% CI, 9.7-13.5) for the KEYTRUDA arm and 10.6 months (95% CI, 8.8-12.0) for the placebo arm, with a HR of 0.86 (95% CI, 0.68-1.10).

In the study, the median duration of exposure was 5.7 months (range: 1 day to 26 months) in the KEYTRUDA combination arm and 5.1 months (range: 3 days to 27 months) in the chemotherapy arm. KEYTRUDA was discontinued for adverse reactions in 15% of patients. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA (1%) were pneumonitis (1.6%), acute kidney injury (1.1%) and pneumonia (1.1%). Adverse reactions leading to interruption of KEYTRUDA occurred in 67% of patients. The most common adverse reactions leading to interruption of KEYTRUDA (2%) were neutropenia (19%), fatigue/asthenia (8%), decreased white blood cell count (5%), pneumonia (5%), decreased appetite (4.3%), anemia (3.2%), increased blood creatinine (3.2%), stomatitis (3.2%), malaise (3.0%), thrombocytopenia (3%), pneumonitis (2.7%), diarrhea (2.4%), dysphagia (2.2%) and nausea (2.2%). The most common adverse reactions (all grades 20%) for KEYTRUDA plus chemotherapy were nausea (67%), fatigue (57%), decreased appetite (44%), constipation (40%), diarrhea (36%), vomiting (34%), stomatitis (27%) and weight loss (24%).

About Esophageal Cancer

Esophageal cancer begins in the inner layer (mucosa) of the esophagus and grows outward. Esophageal cancer is the eighth most commonly diagnosed cancer and the sixth leading cause of death from cancer worldwide. In the U.S., about 67% of newly diagnosed esophageal cancer cases were adenocarcinoma, and 33% were squamous cell carcinoma. It is estimated there will be approximately 19,260 new cases of esophageal cancer diagnosed and about 15,530 deaths resulting from the disease in the U.S. in 2021.

About KEYTRUDA (pembrolizumab) Injection, 100 mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,400 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications in the U.S.

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).

KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 (CPS 10), as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

Microsatellite Instability-High or Mismatch Repair Deficient Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer

KEYTRUDA is indicated for the first-line treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or gastroesophageal junction (GEJ) (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:

Cervical Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Continued here:
FDA Approves Merck's KEYTRUDA (pembrolizumab) Plus Platinum- and Fluoropyrimidine-Based Chemotherapy for Treatment of Certain Patients With Locally...