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MedNess: bite-size biopharma and medtech news

10th March
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MedNess This Week
HIGHLIGHTS
COVID Special
FDA issues Emergency Use Authorization for the distribution of a third COVID-19 vaccine 
FDA recently issued the Emergency Use Authorization (EUA) for the first single shot COVID-19 vaccine, developed by the Janssen Pharmaceutical Companies of Johnson and Johnson, to prevent COVID-19 in individuals 18 years and older. The FDA’s Vaccines and Related Biological Products Advisory Committee unanimously voted in favor of the EUA. A pharmacovigilance plan has been submitted on behalf of the company to the FDA where they include initiatives to look into the complete long-term safety of patients who are currently enrolled in the clinical trials. While more data is being gathered and analyzed, the EUA grants the use of the vaccine in the meantime for active immunization to prevent COVID-19. The company aims to file a Biologics License Application (BLA) later in 2021. It is the first single dose vaccine to be developed. Till now, the two other vaccines approved from Pfizer and BioNTech, and Moderna are both double-shot vaccines which are administered 21 and 28 days apart respectively. Janssen’s single shot vaccine can remain stable at -4 C for two years. It can be stored for a maximum of three months in normal refrigerating conditions of 2 to 8 C. The company will be shipping the vaccine using the same cold chain technologies that it uses to ship other medicines and immunological products. The company plans to deliver 100 million vaccines during the first half of 2021 in the US.
 
Janssen’s COVID-19 vaccine utilizes adenovirus type 26 to deliver a piece of the genetic material that makes the spike protein of the SARS-CoV-2 virus. The Ad26 can cause cold symptoms and pink eye and therefore has been modified for the vaccine so that it cannot cause illness in the body by replicating. The body will temporarily produce these spike proteins after vaccination. These spike proteins will not cause any disease but trigger the immune system to generate an immune response against the virus. The EUA was based on the Phase 3 ENSEBLE study. It is a randomized, double-blind, placebo-controlled study that was conducted in subjects 18 years and older. The aim of the study was to determine the safety and efficacy of the vaccine in providing protection against the moderate and severe form of the disease, with day 14 and day 28 being the co-primary endpoints. A total of 43,783 subjects were enrolled in the study. In the US, a total of 44% of subjects were enrolled in the study. Among those enrolled in the study, 41% of the subjects had comorbidities associated with an increased risk of progression to severe COVID-19. The effectiveness of the vaccine was analyzed in 39,321 subjects. Among these 19,630 received the vaccine and 19,691 received the placebo. The vaccine was found to be 67% effective in preventing moderate and severe COVID-19 occurring at least 14 days after vaccination and 66% effective in preventing the same at least 28 days after vaccination. It was 77% effective in preventing severe/critical COVID-19 occurring at least 14 days after vaccination and 85% effective in preventing the same at least 28 days after vaccination. At this time, there isn’t sufficient data to determine how long the effectiveness of the vaccine will last or if the vaccine prevents transmission from person to person.
Sanofi and Merck enter into collaboration with Janssen Pharmaceutical Companies of Johnson and Johnson to support the manufacture of the latter’s COVID-19 vaccine 
Sanofi and Merck have entered into a collaboration with the Janssen Pharmaceutical Companies of Johnson and Johnson to help support them in the manufacturing process for the latter’s single-shot COVID-19 vaccine. Merck will utilize its facilities in the United States to produce, formulate and fill vials of the vaccine. Additionally, Merck will receive a funding up to $268.8 million from BARDA, to adapt and make available a number of their existing facilities for the production of the COVID-19 vaccine. Sanofi has also entered into an agreement that will support manufacturing of the COVID-19 vaccine developed by Janssen. Sanofi will provide access to their vaccine manufacturing facility in France to formulate and fill vials of the COVID-19 vaccine at a rate of 12 million doses approximately per month.
“At Merck, we have a rich legacy in vaccine manufacturing and look forward to combining our expertise with Johnson & Johnson to help increase supply and expand access to authorized SARS-CoV-2/COVID-19 vaccines,” said Sanat Chattopadhyay, executive vice president and president, Merck Manufacturing Division. “We are steadfast in our commitment to contribute to the global response to the pandemic as part of the remarkable efforts of the entire medical and scientific community,” said Mike Nally, executive vice president, Human Health at Merck. “This funding from BARDA will allow us to accelerate our efforts to scale up our manufacturing capacity to enable timely delivery of much needed medicines and vaccines for the pandemic.” “Today’s agreement is the second of its kind and demonstrates Sanofi’s ongoing commitment to the collective effort to ending this crisis as quickly as possible,” said Paul Hudson, Chief Executive Officer, Sanofi. “While our priority remains advancing our two COVID-19 vaccine programs, we recognize there are opportunities to increase supply and expand access to COVID-19 vaccines. As such, without compromising other essential medicines and vaccines, and where we have the right manufacturing capabilities, we are stepping forward to show solidarity in the industry and continue doing our part in the fight against COVID-19.”
Click here for more COVID news
Collated by : Esha Sehanobish, PhD
    Drug Approvals
U.S. FDA Approves Yescarta® for R/R Follicular Lymphoma After Two or More Lines of Systemic Therapy
“Once a follicular lymphoma patient’s disease relapses, the duration of response to care shortens with each round of therapy,” said Caron A. Jacobson, MD, MMSc, Medical Director, Immune Effector Cell Therapy Program, Dana-Farber Cancer Institute and Assistant Professor of Medicine, Harvard Medical School. “Additionally, for follicular patients in the third line of therapy, the five-year survival rate is only 20 percent, highlighting the urgent need for treatments that offer a real chance for durable remission. Impressively, 91 percent of follicular lymphoma patients in the ZUMA-5 study responded to a single infusion of axicabtagene ciloleucel, including an estimated 74 percent of patients in a continued remission at 18 months, giving these patients much-needed hope and oncologists an important addition to the treatment armamentarium.”
FDA approves PEPAXTO® (melphalan flufenamide) for patients with R/R multiple myeloma
“The accelerated approval of PEPAXTO in the US is an important milestone for Oncopeptides, and a major step ahead in fulfilling our mission, to bring hope to patients with difficult-to-treat hematological diseases, through innovative science”, says Marty J Duvall, Chief Executive Officer at Oncopeptides AB. “Moving ahead, our focus is to further advance PEPAXTO. We look forward to receiving top line data from the phase 3 OCEAN-study in relapsed refractory multiple myeloma, in the second quarter. The comparative study with pomalidomide, is designed to support a future supplementary New Drug Application to expand the label”.
  Click here for more Drug Approvals
Regulatory News
sNDA submitted to FDA for TIBSOVO® (ivosidenib tablets) for Patients with Previously Treated IDH1-Mutant Cholangiocarcinoma
“Cholangiocarcinoma is a rare, aggressive cancer with limited effective therapies, and patients are in desperate need of new treatment options – particularly those who experience disease progression after chemotherapy,” said Chris Bowden, M.D., chief medical officer at Agios. “We are proud of the work we have done on behalf of these patients and look forward to working closely with the FDA during the review of the first oral therapy targeting an IDH1 mutation for patients with previously treated IDH1-mutated cholangiocarcinoma.”
Rolling Submission of BLA for Toripalimab to the U.S. FDA initiated for the Treatment of NPC
“There has been limited development of treatment approaches for patients with advanced NPC in the U.S.," said Dr. Patricia Keegan, Chief Medical Officer of Junshi Biosciences. “We are determined to advance effective treatments in the U.S. by leveraging the successful experience with toripalimab, a safe and effective treatment for previously treated NPC that is now approved for marketing in China. We appreciate the FDA’s recognition of this potentially important new treatment through its Breakthrough Therapy Designation, which enables the acceleration of the review process. We will work closely with the FDA to facilitate the review of the U.S. marketing application in order to make toripalimab available for patients in the U.S. as soon as possible.”
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Trial Results
BLINCYTO® (Blinatumomab) Demonstrated Significantly Prolonged EFS Compared With Consolidation Chemotherapy In Pediatric Patients With Relapsed ALL
"Acute lymphoblastic leukemia is the most common type of cancer in children. Unfortunately, approximately 15% of children with high-risk B-ALL relapse after frontline chemotherapy," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "There remains an urgent need for novel treatment options for these patients, and the study results support BLINCYTO as a new standard of care consolidation therapy for patients with this aggressive disease."
 
Pirtobrutinib (LOXO-305) Ph 1/2 Data published in The Lancet
"We are extremely pleased to see the pirtobrutinib data from the ongoing Phase 1/2 BRUIN study published in The Lancet and shared with the broader clinical community", said David Hyman, M.D., chief medical officer of Loxo Oncology at Lilly. "The data to date from this study have continued to strengthen our conviction that pirtobrutinib has the potential to meaningfully improve the inadequate treatment options available to CLL and MCL patients who have been previously treated with the main treatment classes of today's standard of care. We are focused on quickly advancing the pirtobrutinib development program, including through a series of Phase 3 studies that will be initiated over the course of 2021."
Trial/Program Status
U.S. indication for KEYTRUDA (pembrolizumab) in mSCLC patients with disease progression on or after platinum-based chemotherapy and at least one other prior line of therapy withdrawn
“The accelerated pathways created by the FDA have been integral to the remarkable progress in oncology care over the past five years and have helped many cancer patients with advanced disease, including small cell lung cancer, access new treatments,” said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories. “KEYTRUDA remains a foundational treatment for certain patients with metastatic non-small cell lung cancer. We will continue to rigorously evaluate the benefits of KEYTRUDA in small cell lung cancer and other types of cancer, in pursuit of Merck’s mission to save and improve lives.”
First patient dosed in Ph 2 trial of MT-401 in AML patients following allogeneic stem cell transplant in both the adjuvant and active disease settings
“We are pleased to have dosed the first patient with MT-401 in our Company-sponsored clinical trial, particularly in a patient population in which there remains a critical unmet need,” said Mythili Koneru, M.D., Ph.D., Chief Medical Officer of Marker Therapeutics. “Today, adult patients with post-transplant AML have a 25 percent chance of 5-year survival. In various investigator-sponsored Phase 1 trials at the Baylor College of Medicine, our MultiTAA-specific T cell therapies have been generally well-tolerated and demonstrated durable anti-cancer responses across a broad range of cancers—including post-transplant AML. Based on these results, we believe that MT-401 has the potential to become a meaningful treatment option for patients suffering from this disease.”
Click here for more Trial Statuses
Collated by : Richa Tewari, PhD
MedNess Plus
FDA approves Nulibry injection to reduce the risk of death due to Molybdenum cofactor Deficiency Type A 
FDA approves the use of Nulibry (fosdenopterin) to reduce the risk of death in a rare, genetic, metabolic disorder known as Molybdenum Cofactor Deficiency Type A. The application from Origin Bioscience Inc. was granted Priority Review and Breakthrough Therapy along with an Orphan Drug designation. They shall also receive a Rare Pediatric Disease Priority Review Voucher that encourages the development of drugs and biologics for the prevention and the treatment of rare pediatric diseases.
 
Molybdenum Cofactor Deficiency Type A is a rare, genetic disease that occurs in the early stages of one’s life. It is characterized by brain encephalopathy which deteriorates over time. Even though babies may appear normal at birth, they develop difficulty in feeding and have intractable seizures which worsen with time causing brain damage. The disorder is caused mainly due to molybdenum cofactor deficiency and the condition can be so severe that those with this disorder may not survive beyond childhood. Those with this condition are unable to produce cyclic pyranopterin monophosphate (cPMP). Nulibry functions by replacing this protein and the mode of administration is intravenous. The approval was based on a study that investigated the effectiveness of Nulibry in 13 patients compared to 18 unmatched and untreated patients. Those who were on Nulibry had a survival rate of 84% in three years compared to 55% in those who were untreated. The side effects associated with the disease include intravenous line, respiratory infections, fever, diarrhea and vomiting. Animals investigated with Nulibry exhibited phototoxicity. Therefore, patients on Nulibry should avoid exposure to sunlight and wear proper garments and take proper protection against direct sunlight.
 
FDA approves Amondys 45 injection for the treatment of Duchenne muscular dystrophy in patients with a confirmed mutation 
FDA recently granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular dystrophy (DMD) characterized by a confirmed mutation in the DMD gene that can involve skipping of exon 45. Around eight percent of patients with DMD are diagnosed with a mutation that is amenable to exon 45 skipping and this approval targets the first therapy of its kind for such DMD patients. The approval was granted to Sarepta Therapeutics Inc. FDA granted this application a Priority Review and a Fast-Track designation along with an Orphan Drug designation. It was approved using the Accelerated Approval, in which the FDA grants approval for treatment for serious conditions which have unmet needs and the application indicated likely benefit for patients using the drug in the question.
 
Duchenne muscular dystrophy is usually a genetic disorder that is caused due changes in the protein dystrophin. The function of dystrophin is to keep the muscle cells intact. The disorder is characterized by progressive muscle degeneration and weakness. It can begin as early as two to three years of age and can start by affecting the proximal muscles and then affecting the distal muscles. At a later stage the heart and respiratory muscles are also affected. DMD has an X-linked recessive inheritance pattern and females are the usual carriers who have a normal dystrophin gene on one X-chromosome and an abnormal one on another. The approval was based on data from a study that evaluated Amondys 45. It was a placebo-controlled, double-blind study. A total of 43 patients were involved in the study and they were randomized in a 2:1 ratio to receive either 30 mg/kg of Amondys 45 or placebo. The patients enrolled in the study were between seven and 20 years of age. All of the patients had a genetically confirmed mutation on the DMD gene that was amenable to skipping exon 45. Those on Amondys 45 exhibited a significant increase in the dystrophin protein levels from baseline to week 48 of treatment, compared to those on placebo. The common side effects observed in DMD patients treated with Amondys 45 include cough, fever, upper respiratory tract infections, throat and joint pains.
 
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Collated by : Esha Sehanobish, PhD
MedNess @ HealthIT
 

Medical robots find their way to acceptance among patients, facilitated by COVID

As we are moving towards a world with robots conducting repetitive and strenuous tasks, both on bench and in the clinic, it is critical to gather the satisfaction level of the end users. Researchers from MIT and Brigham and Women’s Hospital performed a survey within 1000 patients asking for their acceptance level for robots performing routing tasks at a doctor’s visit, and tested a doglike robot manufactured by Boston Dynamics – in the emergency department at Brigham and Women's Hospital last spring. The survey revealed that majority of patients accepted the use of medical robots facilitating telehealth interviews, acquiring vital signs and turning a patient in bed. Nearly 50% participants were okay with robots obtaining nasal or oral swabs. About 40% thought it to be useful for placing an intravenous catheter or for performing phlebotomy. In fact, testing a medical robot named Dr. Spot to interview 41 patients revealed a satisfactory experience in 90% of the cases.
Automating medical tasks has become more important in recent times necessitating physical distancing. Medical robots have made appearance in medical facilities such as Bahrain and Singapore. According to Dr. Jameela Al Salman, an infectious and internal diseases consultant at the Salmaniya Medical Complex, "These devices will provide more protection to medical personnel and reduce the transmission of disease, as well as protect sanitation workers from constant exposure to chemicals.” Although the COVID situation escalated the changes, the technology shows the signs of staying on.
NIH launched the next generation of precision medicine for Alzheimer’s patients
The next version of the AMP AD 2.0 (Accelerating Medicines Partnership Alzheimer’s disease program) is being launched by FNIH (Foundation of NIH): a collaboration between NIH, industry, non-profit, and other organizations that utilize open science practices to accelerate the discovery of new drug targets, biomarkers, and disease subtypes. According to NIH Director Francis S. Collins, MD, PhD, “AMP AD 2.0 aims to add greater precision to the molecular maps developed in the first iteration of this program. This will identify biological targets and biomarkers to inform new therapeutic interventions for specific disease subtypes.” The first AMP Alzheimer’s program facilitated to generate a wealth of data from animal and cell-based models and human samples, leading to the identification of 500 unique candidate targets through computational methods, and was made publicly available through a centralized data infrastructure and data sharing platform called the AD Knowledge Portal, and the portal-linked, open-source platform Agora. With 3000+ users for the database in the field of Alzheimer’s and dementia research field, the 2.0 update aims to facilitate precision medicine even further. NIH will spearhead the research as well as contribute an estimated total of $61.4 million over five years, pending availability of funds. The funds aim to fund a data coordinating center at Sage Bionetworks, as well as six multi-institutional, cross-disciplinary academic research teams.
Medness Business
Century Therapeutics Completes $160 Million Series C Financing to Accelerate Development of iPSC-derived Cell Therapy Pipeline
“We are fortunate to be surrounded by such a top-tier group of investors, whose support will enable the acceleration of Century’s technology platform into the clinic,” said Lalo Flores, Chief Executive Officer, Century Therapeutics. “With this new investor partnership, we are well-positioned to capitalize on the tremendous potential of our integrated iPSC, cell engineering and manufacturing capabilities to develop safer, more effective and more affordable next-generation allogeneic cancer therapies.” 
Amgen to Acquire Five Prime Therapeutics
“The acquisition of Five Prime offers a compelling opportunity for Amgen to strengthen our oncology portfolio with a promising late-stage, first-in-class global asset to treat gastric cancer,” said Robert A. Bradway, chairman and chief executive officer at Amgen. “We look forward to welcoming the Five Prime team to Amgen and working with them to leverage our best-in-class monoclonal antibody manufacturing capabilities to supply additional clinical materials, as well as expanded production quantities, to realize the full potential of bemarituzumab for even more patients around the world as quickly as possible.”
Click here for more on mergers, acquisition and business news
Collated by : Richa Tewari, PhD
Editors' Desk
Richa Tewari, PhD
Oncology News
Esha Sehanobish, PhD
MedNess Plus
Arundithi Ananthanarayanan
MedNess Reviews
Divyaanka Iyer
BioPharma News
Shilpa Rawal, PhD
Onco I-Analyse
Debarati Banik
HealthIT
Rinki Saha
Managing Editor
Shalini Roy Choudhury
Managing Editor
Nisha Peter, PhD
Consulting Editor
Abhi Dey
Consulting Editor
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