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FDA Revokes Emergency Use Authorization for Monoclonal Antibody Bamlanivimab

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FDA Revokes Emergency Use Authorization for Monoclonal Antibody Bamlanivimab

April 16, 2021: “The U.S. FDA revoked the emergency use authorization (EUA) that allowed for the investigational monoclonal antibody therapy bamlanivimab, when administered alone, to be used for the treatment of mild-to-moderate COVID-19 in adults and certain pediatric patients.

Based on its ongoing analysis of emerging scientific data, specifically the sustained increase of SARS-CoV-2 viral variants that are resistant to bamlanivimab alone resulting in the increased risk for treatment failure, the FDA has determined that the known and potential benefits of bamlanivimab, when administered alone, no longer outweigh the known and potential risks for its authorized use.

Therefore, the agency determined that the criteria for issuance of an authorization are no longer met and has revoked the EUA.

On Nov. 9, 2020, based on the totality of scientific evidence available at the time, the FDA issued an EUA to Eli Lilly and Co. authorizing the emergency use of bamlanivimab alone for the treatment of mild to moderate COVID-19 in adults and pediatric patients (12 years of age and older weighing at least 40 kg) with positive results of direct SARS-CoV-2 viral testing, and who are at high risk for progressing to severe COVID-19 and/or hospitalization. Importantly, although the FDA is now revoking this EUA, alternative monoclonal antibody therapies remain available under EUA, including REGEN-COV (casirivimab and imdevimab, administered together), and bamlanivimab and etesevimab, administered together, for the same uses as previously authorized for bamlanivimab alone.

The FDA believes that these alternative monoclonal antibody therapies remain appropriate to treat patients with COVID-19 when used in accordance with the authorized labeling based on information available at this time.


Related News: EMA issues advice on Lilly’s bamlanivimab with etesevimab for confirmed COVID-19 in EU
Lilly’s bamlanivimab administered with etesevimab receives FDA emergency use authorization for COVID-19

“While the risk-benefit assessment for using bamlanivimab alone is no longer favorable due to the increased frequency of resistant variants, other monoclonal antibody therapies authorized for emergency use remain appropriate treatment choices when used in accordance with the authorized labeling and can help keep high risk patients with COVID-19 out of the hospital,” said Patrizia Cavazzoni, M.D., director of the FDA’s Center for Drug Evaluation and Research.

“We urge the American public to seek out these therapies when needed while we continue to use the best data available to provide patients with safe and effective treatments during this pandemic.” 

Monoclonal antibodies are laboratory-made proteins that mimic the immune system’s ability to fight off harmful pathogens such as viruses, like SARS-CoV-2. Like other infectious organisms, SARS-CoV-2 can mutate over time, resulting in genetic variation in the population of circulating viral strains.

The FDA has a responsibility to regularly review the appropriateness of an EUA, and as such, the agency will review emerging information associated with the emergency uses for the authorized products.

Recent data from the U.S. Centers for Disease Control and Prevention’s (CDC) national genomic surveillance program show an increased frequency of SARS-CoV-2 variants that are expected to be resistant to bamlanivimab administered alone.

As of mid-March 2021, approximately 20% of viruses sequenced in the U.S. were reported as variants expected to be resistant to bamlanivimab alone, increasing from approximately 5% in mid-January 2021.

Additionally, there are currently no testing technologies available that enable health care providers to test individual patients for SARS-CoV-2 viral variants prior to start of treatment with monoclonal antibodies.

Therefore, empiric treatment with monoclonal antibody therapies that are expected to work broadly against all variants across the nation should be used to reduce the likelihood of treatment failure. 

The FDA will continue working closely with other federal governmental agencies, including the CDC and the National Institutes of Health, on the surveillance of variants that may impact the monoclonal antibody therapies authorized for emergency use.

The agency remains committed to providing timely and transparent communication as additional information becomes available.”

https://www.fda.gov/news-events/press-announcements/coronavirus-covid-19-update-fda-revokes-emergency-use-authorization-monoclonal-antibody-bamlanivimab

FDA Approves First Immunotherapy for Initial Treatment of Gastric Cancer

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FDA Approves First Immunotherapy for Initial Treatment of Gastric Cancer

April 16, 2021:The U.S. Food and Drug Administration approved Opdivo (nivolumab), in combination with certain types of chemotherapy, for the initial treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer and esophageal adenocarcinoma.

This is the first FDA-approved immunotherapy for the first-line treatment of gastric cancer.

“Today’s approval is the first treatment in more than a decade to show a survival benefit for patients with advanced or metastatic gastric cancer who are being treated for the first time,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research.

“The FDA is committed to bringing new safe and effective treatment options like Opdivo to patients with advanced cancer.”

There are approximately 28,000 new diagnoses of gastric cancer each year in the U.S. With currently available therapy, overall survival is generally poor; the rate of cure with resection is very low and the survival rate for all stages is 32%. The 5-year survival rate for advanced or metastatic gastric cancer is 5%.

Opdivo is a monoclonal antibody that inhibits tumor growth by enhancing T-cell function. Its efficacy was evaluated in a randomized, multicenter, open-label trial of 1,581 patients with previously untreated advanced or metastatic gastric cancer, gastroesophageal junction cancer and esophageal adenocarcinoma.

The 789 patients who received Opdivo in combination with chemotherapy, on average, lived longer than the 792 patients who received chemotherapy alone.

Median survival was 13.8 months for patients who received Opdivo plus chemotherapy compared to 11.6 months for patients who received chemotherapy alone.


Related News: BMS Announces Opdivo plus Yervoy in Esophageal Squamous Cell Carcinoma

EMA Validates BMS Opdivo as Adjuvant Treatment for Muscle-Invasive Urothelial Carcinoma

EMA launches review of BMS’ Opdivo(nivolumab) for oesophageal cancer

The most common side effects of Opdivo in combination with chemotherapy include peripheral neuropathy (damage to the nerves outside of the brain and spinal cord), nausea, fatigue, diarrhea, vomiting, decreased appetite, abdominal pain, constipation and musculoskeletal pain.

Opdivo can cause serious conditions known as immune-mediated side effects, including inflammation of healthy organs such as the lungs (pneumonitis), colon (colitis), liver (hepatitis), endocrine glands (endocrinopathies) and kidneys (nephritis).

Patients should tell their healthcare providers if they have immune system problems, lung or breathing problems, liver problems, have had an organ transplant, or are pregnant or plan to become pregnant before starting treatment.

Opdivo received Priority Review and Orphan Drug designations for this indication.

Priority Review designation directs overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis or prevention of serious conditions when compared to standard applications.

Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).

The FDA granted approval to Bristol-Myers Squibb Company.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners.

For this review, the FDA collaborated with the Australian Therapeutic Goods Administration, the Brazilian Health Regulatory Agency, Health Canada and Switzerland’s Swissmedic. The application reviews are ongoing at the other regulatory agencies.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices.

The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

https://www.fda.gov/news-events/press-announcements/fda-approves-first-immunotherapy-initial-treatment-gastric-cancer

Alchemab raises £60M A financing to advance novel platform

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Alchemab raises £60M A financing to advance novel platform

Apr 15, 2021: “Alchemab Therapeutics, a biotech company developing novel products for patients with hard-to-treat diseases by harnessing the power of naturally protective antibodies announces the completion of a £60 million ($82 million) Series A financing round.

The international investment syndicate is led by RA Capital Management, with participation from Lightstone Ventures, Data Collective VC (DCVC), DHVC, SV Health Investors and the Dementia Discovery Fund.

The company was created by SV Health Investors who led the Seed round in 2019.

The proceeds will be used to advance Alchemab’s unique target-agnostic drug discovery platform.

The approach interrogates the entire antibody repertoires of individuals from well-defined groups who show unexpected resistance to disease despite genetic disposition or other risk factors predicative of a poor prognosis.

Using insights gained, Alchemab can identify naturally protective antibodies with therapeutic potential.

Alchemab’s primary focus is the development of novel therapeutics for hard-to-treat neurodegenerative diseases and cancers, and the Company currently has several programs at the preclinical stage.

Alchemab’s approach was developed with support from scientific founders at Oxford University, Johns Hopkins University and Mount Sinai Hospital. Alchemab is amongst the first commercial group to access Illumina’s Cambridge, UK Accelerator.

“Our aim is to become a major player in the identification of novel targets and antibodies in the areas of neurodegeneration and cancer,” notedAlex Leech, CEO at Alchemab.

“The substantial financial commitment by this high-caliber group of US and European investors is a strong endorsement of our science and team.”

His colleague and co-founder, Dr Jane Osbourn OBE, CSO at Alchemab, added: “Our approach to understanding the natural immunological response to disease and why some people are able to stay well has huge potential to identify antibody therapies across a range of indications.

The Series A financing offers a great opportunity to accelerate our efforts to positively impact the lives of patients.

Dr Houman Ashrafian, Managing Partner at SV Health Investors, commented: “Alchemab is a brilliant company driven by science.

The Company combines elements of traditional drug discovery techniques with advanced analytics, and in doing so turns the conventional biotech model upside down.”

Dr Andrew Levin, Managing Director at RA Capital, added:“It is a privilege to work with this exceptional team.

Alex, Jane and the scientists at Alchemab are offering a truly innovative, disease-agnostic approach that we believe has great potential for the development of novel therapeutics.”

Last month, Alchemab announced the award of an Innovate UK grant to support the development of a novel disease-modifying antibody therapy for Huntington’s disease, in collaboration with the Medicines Discovery Catapult.”

Alchemab raises £60 million ($82 million) in Series A financing to advance novel platform for identifying disease-modifying antibody therapeutics

Amylyx Pharmaceuticals provides global regulatory update on amx0035 for ALS

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Amylyx Pharmaceuticals provides global regulatory update on amx0035 for ALS

April 14, 2021: Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s Disease and other neurodegenerative diseases provided an update on its plans to advance AMX0035 through the clinical development process for the treatment of ALS.

The company intends to submit a Marketing Authorization Application (MAA) for AMX0035 to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) by the end of 2021.

As previously reported, the company plans to submit a marketing application with Health Canada Q2 2021.

The FDA has expressed an interest in seeing data from an additional placebo-controlled clinical trial prior to receiving a New Drug Application (NDA), the vehicle through which a pharmaceutical sponsor formally proposes that the FDA approve an investigational product.

To fulfill the FDA’s request and to continue to build upon the growing body of evidence supporting AMX0035 for the potential treatment of ALS, Amylyx plans to initiate a Phase 3 clinical trial in Europe and the United States. The trial is expected to begin enrollment in Q3 2021.

The FDA also expressed that it would continue to discuss with Amylyx how regulatory requirements may be met in the most expeditious way possible.

Amylyx is also continuing to discuss AMX0035 with additional regulatory bodies worldwide to determine the most appropriate path forward.

“People living with ALS need clinical answers and solutions quickly. We have been in close contact with physicians and global health authorities to rapidly evaluate AMX0035 for the treatment of ALS,” said Joshua Cohen, Co-CEO, Chairman and Co-Founder of Amylyx.

“We are thrilled to plan our submissions in Europe and Canada and will continue working closely with regulators and the ALS community worldwide to determine the most expeditious and responsible pathways to advance AMX0035 through the clinical development process.

We appreciate all of the advice and guidance from the regulators worldwide and will continue to act with haste and to keep the community updated.”

CENTAUR was a 24-week placebo-controlled study of 137 participants with ALS that evaluated the safety and efficacy of AMX0035.

The trial was led by investigators at the Healey & AMG Center for ALS at Massachusetts General Hospital in collaboration with the Northeast ALS Consortium.

As published in the New England Journal of Medicine (NEJM), the study met its primary efficacy endpoint of slowing ALS progression as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R).

The rates of adverse events were similar between the AMX0035 and placebo arms of the study. However, discontinuations related to adverse events occurred more frequently in the AMX0035 arm than in the placebo arm.

Participants who completed CENTAUR were eligible to enroll in an open-label extension (OLE) and receive AMX0035.

In a nearly three-year overall survival analysis of all randomized participants from CENTAUR that was published in Muscle & Nerve, those originally randomized to AMX0035 had a 44% lower risk of death compared to those originally randomized to placebo.

“With the results from CENTAUR, we showed that AMX0035 may provide people living with ALS hope and the chance to function better and live longer lives,” said Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR trial, investigator at the Healey & AMG Center for ALS at Massachusetts General Hospital and Assistant Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital.

“We are very excited to see AMX0035 advancing on multiple regulatory fronts and remain optimistic that it can potentially help people living with ALS around the world.”

“ALS is a devastating progressive disease that impacts patients and their families not only physically, but also mentally and emotionally,” said Leonard H. van den Berg, M.D., Ph.D., Professor of Neurology at UMC Utrecht in the Netherlands and Chairman of the Treatment Research Initiative to Cure ALS (TRICALS), a large European trial network dedicated to finding a treatment for ALS.

“After decades of ALS trial failures, AMX0035 has given us the hope that a new potential treatment option may be on the horizon for those living with ALS.

We remain highly encouraged as AMX0035 continues to move through the regulatory review process, and are excited for the Phase 3 clinical trial.”

“The pivotal Phase 3 clinical trial will catalyze a global collaboration between Amylyx, European and US ALS experts, advocacy groups, and clinical trial networks,” said Merit Cudkowicz, MD, co-principal investigator of the CENTAUR trial and co-founder of the Northeast ALS Consortium, Director of the Healey & AMG Center for ALS and Chair of Neurology at Massachusetts General Hospital and the Julieanne Dorn Professor of Neurology at Harvard Medical School.

“We hope this is just the beginning of providing new options to people living with ALS.”

“People with ALS are fighting against this disease every day,” said Evy Reviers, Chairwoman of European Organization for Professionals and Patients with ALS (EUpALS) and CEO of ALS Liga Belgium.

“As a caregiver of a loved one with ALS, I know how critical it is to make potential treatment options available to the ALS community. On behalf of EUpALS, I look forward to working with industry, academia and health authorities to make that a reality.”

“We are pleased to share our plans to initiate regulatory filing in Europe,” said Justin Klee, Co-CEO and Co-Founder of Amylyx.

“Every single day matters for people living with ALS and we will continue to work with the U.S. FDA and global regulatory agencies to meet their requests so that we can advance AMX0035 through the clinical development process as quickly as possible.

We will share updates on our progress with the community as we have them.”

https://www.amylyx.com/2021/04/14/amylyx-pharmaceuticals-provides-global-regulatory-update-on-amx0035-for-als/

Novartis signs initial agreement with Roche to reserve capacity and implement the technology transfer for the production of Roche’s Actemra/RoActemra®

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Novartis signs initial agreement with Roche to reserve capacity and implement the technology transfer for the production of Roche’s Actemra/RoActemra®

April 15, 2021: Novartis has signed an initial agreement with Roche to reserve capacity and implement the technology transfer for the production of the active pharmaceutical ingredient (API) for Roche’s Actemra/RoActemra® (tocilizumab), a treatment for rheumatoid arthritis which is also being tested in various clinical trials investigating the safety and efficacy in COVID-19 associated pneumonia.

Under the terms of the initial agreement, the manufacturing process expertise of Roche will be transferred to the Novartis Drug Substance Singapore site during the second quarter this year. The initial agreement covers the technology transfer and the process validation.

“Novartis is fully committed to collaborating with Roche in offering our proven biologics production capabilities,” said Steffen Lang, Head of Novartis Technical Operations and member of the Novartis Executive Committee.

“As one of the world’s largest producers of medicines, Novartis can mobilize its manufacturing capabilities on multiple fronts.”

Actemra/RoActemra® is also approved for the treatment of paediatric juvenile idiopathic arthritis, systemic juvenile idiopathic arthritis, giant cell arteritis and CAR-T cell-induced cytokine release syndrome. Actemra/RoActemra® is available in both subcutaneous and intravenous formulations.

In March, Novartis announced an initial agreement to manufacture the mRNA and bulk drug product for the COVID-19 vaccine candidate CVnCoV from CureVac to aid in the fight against the COVID-19 pandemic in a new high-tech production facility at the Novartis site in Kundl, Austria.

And in January, an initial agreement with BioNtech was also signed to provide manufacturing capacity for a COVID-19 vaccination at the Novartis site in Stein, Switzerland.

https://www.novartis.com/news/media-releases/novartis-signs-initial-agreement-reserve-capacity-and-implement-technology-transfer-production-active-pharmaceutical-ingredient-roche%27s-actemraroactemra

FDA Provides Guidance on Remote Interactive Evaluations for Oversight of Drug Facilities During COVID-19

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FDA Provides Guidance on Remote Interactive Evaluations for Oversight of Drug Facilities During COVID-19

April 14, 2021: “During this worldwide public health emergency, the FDA has used a variety of tools to oversee facilities that manufacture FDA-regulated products.

These tools include record requests in advance of or in lieu of a drug facility inspection, relying on information from trusted regulatory partners, and remote interactive evaluations (such as remote livestreaming video of operations, teleconferences and screen sharing).

We have used some or all of these approaches to evaluate facilities for human and animal medical products during the public health emergency when inspections of drug facilities were not possible due to travel or quarantine restrictions.

Inspections are an important tool to keep Americans safe, and are part of a set of tools used for regulatory oversight.

As part of the wide variety of tools we have deployed during the COVID-19 pandemic, remote interactive evaluations have informed the FDA’s regulatory decision-making, contributed to ensuring drug quality and helped determine the scope, depth and timing of future inspections.

By necessity, we have adapted by conducting more remote interactive evaluations throughout the public health emergency and are continuing to expand their use as appropriate.

The purpose of this new guidance is to provide further clarity for regulated facilities on how the FDA will request and conduct these remote interactive evaluations during the COVID-19 public health emergency.

We recognize that remote interactive evaluations do not replace inspections, and that there are situations where only an inspection is appropriate based on risk and history of compliance with FDA regulations.

Within the exceptional context of a global pandemic, we see remote interactive evaluations as part of a necessary strategy to evaluate medical product facilities by using all available approaches to ensure the medical products we regulate are safe, effective and of high quality.”

https://www.fda.gov/news-events/press-announcements/fda-provides-guidance-remote-interactive-evaluations-oversight-drug-facilities-during-covid-19

U.S. FDA Approves Trodelvy® for Treatment of Metastatic Urothelial Cancer

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U.S. FDA Approves Trodelvy® for Treatment of Metastatic Urothelial Cancer

April 13, 2021: Gilead Sciences, Inc. announced that the U.S. FDA has granted accelerated approval of Trodelvy® (sacituzumab govitecan-hziy) for use in adult patients with locally advanced or metastatic urothelial cancer (UC) who have previously received a platinum-containing chemotherapy and either a programmed death receptor-1 (PD-1) or a programmed death-ligand 1 (PD-L1) inhibitor.

The accelerated approval was based on data from the international Phase 2, single-arm TROPHY study.

Of the 112 patients who were evaluable for efficacy, 27.7% of those treated with Trodelvy responded to treatment, with 5.4% experiencing a complete response and 22.3% experiencing a partial response.

The median duration of response was 7.2 months (95% CI: 4.7-8.6). The Trodelvy U.S. Prescribing Information has a BOXED WARNING for severe or life-threatening neutropenia and severe diarrhea; see below for Important Safety Information.

The FDA’s accelerated approval mechanism enables drugs that treat serious diseases with unmet medical need to be approved based on a surrogate or intermediate clinical endpoint. Continued approval is contingent upon verification and description of clinical benefit in a confirmatory trial.

“Only a fraction of patients derives long-term benefit from previously approved cytotoxic therapy or immunotherapy, leaving a great unmet need for treatment options for patients with advanced urothelial cancer who have progressed on first- and second-line therapies,” said Scott T. Tagawa, MD, MS, FACP, Professor of Medicine and Urology at Weill Cornell Medicine, an oncologist at New York-Presbyterian/Weill Cornell Medical Center and principal investigator of the TROPHY study.

“The response rate and tolerability seen with sacituzumab govitecan-hziy may provide physicians an effective new treatment option for patients whose cancer continues to progress even after multiple therapies.”

UC is the most common type of bladder cancer and occurs when the urothelial cells that line the inside of the bladder and other parts of the urinary tract grow unusually or uncontrollably.

An estimated 83,000 Americans will be diagnosed with bladder cancer in 2021, and almost 90% of those diagnoses will be UC. The relative five-year survival rate for patients with metastatic UC is 5.5%.

“Cases of urothelial cancer continue to rise in the U.S., yet prognosis remains the same for the vast majority of patients,” said Andrea Maddox-Smith, CEO of the Bladder Cancer Advocacy Network (BCAN).

“Bladder cancer patients need as many treatment options as possible, and we are pleased that Trodelvy can be a potentially viable treatment for them.”

Trodelvy’s safety profile in the TROPHY study is consistent with previous observations in metastatic UC and other tumor types.

Among all evaluable treated metastatic UC patients (n=113), the most common (≥25%) adverse reactions were diarrhea (72%), anemia (71%), fatigue (68%), neutropenia (67%), nausea (66%), alopecia (49%), decreased appetite (41%), constipation (34%), vomiting (34%) and abdominal pain (31%).

Adverse reactions leading to treatment discontinuation occurred in 10% of those receiving Trodelvy, with 4% discontinuing treatment due to neutropenia.

“Today’s accelerated approval is thanks to the patients and healthcare professionals involved in the TROPHY study, and we appreciate their partnership,” said Merdad Parsey, MD, PhD, Chief Medical Officer, Gilead Sciences.

“This achievement, coupled with last week’s full FDA approval in unresectable locally advanced or metastatic triple-negative breast cancer, underscores our commitment toward rapidly delivering Trodelvy to patients facing some of the most difficult-to-treat cancers.”

https://www.gilead.com/news-and-press/press-room/press-releases/2021/4/us-fda-grants-accelerated-approval-to-trodelvy-for-the-treatment-of-metastatic-urothelial-cancer

Myovant, Pfizer strats dosing in Relugolix Contraceptive study

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Myovant, Pfizer strats dosing in Relugolix Contraceptive study

April 12, 2021: “Myovant Sciences and Pfizer Inc. announced that the first participant has been dosed in the Phase 3 SERENE study evaluating the contraceptive efficacy of relugolix combination tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) in healthy women ages 18-35 years who are at risk for pregnancy.

“We are committed to redefining care for women, which means supporting their overall health and quality of life.

Many women with uterine fibroids and endometriosis need to simultaneously manage their symptoms and their reproductive choices – including prevention of pregnancy,” said Juan Camilo Arjona Ferreira, M.D., Chief Medical Officer of Myovant Sciences, Inc.

“The Phase 3 SERENE study is designed to assess the potential of relugolix combination tablet to prevent pregnancy, and will complement data from our Phase 3 LIBERTY and SPIRIT programs which demonstrated the promise of relugolix combination therapy as a potential treatment for uterine fibroids and endometriosis.”

The SERENE study will enroll 900 sexually active, healthy women ages 18-35 years with presumed normal fertility.

The primary efficacy endpoint is the at-risk Pearl Index, defined as the number of on-treatment pregnancies per 100 women-years of treatment.

On-treatment pregnancies are pregnancies with an estimated conception date between the first day of study intervention intake up to and including seven days after the last intake of study medication.

Women will receive once-daily relugolix combination tablet for 13 28-day at-risk cycles. Safety data will also be collected during the study.

“The findings of the Phase 1 study demonstrated that relugolix combination therapy inhibited ovulation in all the study participants and provided the basis for the SERENE study to evaluate whether relugolix combination tablet has the potential to prevent pregnancy in women receiving therapy,” said James Rusnak, M.D., Ph.D., Senior Vice President, Chief Development Officer, Internal Medicine and Hospital, Global Product Development at Pfizer.

“The data from this Phase 3 study will provide important information to patients and healthcare providers when making treatment decisions for women with endometriosis and uterine fibroids.”

In April 2020, Myovant announced results from a Phase 1 single-arm, open-label ovulation inhibition study to assess the effects of relugolix combination therapy (relugolix 40 mg plus estradiol 1.0 mg and norethindrone acetate 0.5 mg) on ovulation inhibition, per the Hoogland-Skouby assessment scale (score < 5).

In 67 healthy women over an 84-day treatment period (three cycles), relugolix combination therapy achieved 100% ovulation inhibition and was generally well tolerated.

Furthermore, 100% of women resumed ovulation or menses upon discontinuation of treatment, with an average time to ovulation of 23.5 days. Data from this study were previously presented at the American Society for Reproductive Medicine 2020 Virtual Congress.

Relugolix combination tablet is under review by the U.S. Food and Drug Administration for the treatment of women with uterine fibroids, with a decision expected by the June 1, 2021 target action date.

The submission of a New Drug Application for the treatment of moderate to severe pain associated with endometriosis is anticipated in the first half of 2021.”

https://www.pfizer.com/news/press-release/press-release-detail/myovant-sciences-and-pfizer-announce-first-participant

AZ’s Tagrisso approved in China in early lung cancer

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AZ’s Tagrisso approved in China in early lung cancer

April 14, 2021: “AstraZeneca’s Tagrisso (osimertinib) has been approved in China for the adjuvant treatment of patients with early-stage (IB, II and IIIA) epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC) after tumour resection with curative intent, with or without adjuvant chemotherapy as recommended by the patient’s physician. 

Tagrisso is indicated for EGFRm patients whose tumours have exon 19 deletions or exon 21 (L858R) mutations.

The approval by China’s National Medical Products Administration (NMPA) was based on positive results from the ADAURA Phase III trial.

In the trial, Tagrisso demonstrated a statistically significant and clinically meaningful improvement in disease-free survival (DFS) in the primary analysis population of patients with Stage II and IIIA EGFRm NSCLC.

The ADAURA trial also showed a statistically significant and clinically meaningful improvement in DFS in the overall trial population of patients with Stage IB-IIIA disease, a key secondary endpoint.

While up to 30% of all patients with NSCLC may be diagnosed early enough to have surgery with curative intent, recurrence is still common in early-stage disease.

Historically, nearly half of patients diagnosed in Stage IB, and over three quarters of patients diagnosed in Stage IIIA, have experienced recurrence within five years.

More than a third of the world’s lung cancer patients are in China and among those with NSCLC, approximately 40% have tumours with an EGFR mutation.

Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: “The expedited approval of Tagrisso in China as part of a curative-intent regimen for early-stage EGFR-mutated lung cancer underscores the high unmet need in this setting and our commitment to improving outcomes in a country with one of the highest rates of EGFR mutations in the world.

This approval reinforces the importance of EGFR testing across all stages of lung cancer, prior to treatment decisions, to ensure as many patients as possible can benefit from targeted therapies like Tagrisso and live cancer-free longer.”


Tagrisso approved in the US for early-stage EGFR-mutated lung cancer

AZ’s Tagrisso scores priority review from FDA with EGFR-mutated lung cancer

AstraZeneca’s Tagrisso reduced risk of disease recurrence in brain in EGFR-mutated lung cancer

In the ADAURA trial, adjuvant treatment with Tagrisso reduced the risk of disease recurrence or death by 83% in patients with Stage II and IIIA disease (hazard ratio [HR] 0.17; 99.06% confidence interval [CI] 0.11-0.26; p<0.001) and by 80% in the overall trial population of patients with Stage IB-IIIA disease (HR 0.20; 99.12% CI 0.14-0.30; p<0.001).

Consistent DFS results were seen regardless of prior adjuvant chemotherapy use and across all prespecified subgroups, including in Asian and non-Asian patients.

The safety and tolerability of Tagrisso in this trial was consistent with previous trials in the metastatic setting.

The ADAURA results were published in The New England Journal of Medicine.

Tagrisso is approved to treat early-stage lung cancer in more than a dozen countries, including the US, and additional global regulatory reviews are ongoing. 

Tagrisso is also approved for the 1st-line treatment of patients with locally advanced or metastatic EGFRm NSCLC and for the treatment of locally advanced or metastatic EGFR T790M mutation-positive NSCLC in China, and in the US, Japan, the EU and many other countries.

This approval follows a priority review designation by the NMPA’s Center for Drug Evaluation.

It marks the third approved indication for Tagrisso in China following previous approvals in 2nd-line T790M and 1st-line EGFRm NSCLC, which are both included on the National Reimbursement Drug List.

ADAURA
ADAURA is a randomised, double-blind, global, placebo-controlled Phase III trial in the adjuvant treatment of 682 patients with Stage IB, II and IIIA EGFRm NSCLC following complete tumour resection and adjuvant chemotherapy as indicated.

Patients were treated with Tagrisso 80mg once-daily oral tablets or placebo for three years or until disease recurrence.

The trial enrolled patients in more than 200 centres across more than 20 countries, including the US, in Europe, South America, Asia and the Middle East.

The primary endpoint was DFS in Stage II and IIIA patients and a key secondary endpoint was DFS in Stage IB, II and IIIA patients.

The data readout was originally anticipated in 2022. In April 2020, an Independent Data Monitoring Committee recommended for the trial to be unblinded two years early based on a determination of overwhelming efficacy.

Investigators and patients continue to participate and remain blinded to treatment. The trial will continue to assess overall survival.

Tagrisso
Tagrisso (osimertinib) is a third-generation, irreversible EGFR-TKI with clinical activity against central nervous system metastases. 

Tagrisso (40mg and 80mg once-daily oral tablets) has been used to treat approximately 250,000 patients across indications worldwide and AstraZeneca continues to explore Tagrisso as a treatment for patients across multiple stages of EGFRm NSCLC.

In Phase III trials, Tagrisso is being tested in the Stage III locally advanced unresectable setting (LAURA), in the neoadjuvant resectable setting (NeoADAURA) and in combination with chemotherapy (FLAURA2).

AstraZeneca is also researching ways to address tumour mechanisms of resistance through the SAVANNAH and ORCHARD Phase II trials, which test Tagrisso given concomitantly with savolitinib, an oral, potent and highly selective MET TKI, as well as other potential new medicines.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2021/tagrisso-approved-in-china-in-early-lung-cancer.html

Sanofi continues to strengthen its vaccines manufacturing capacities in Singapore

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Sanofi continues to strengthen its vaccines manufacturing capacities in Singapore

April 12 2021: Sanofi announced a €400 million investment over five years to create a unique vaccine production center in Singapore, pushing the boundaries of operations through cutting edge manufacturing and digital technologies.

In partnership with the Singapore Economic Development Board (EDB), the new site will mainly supply the Asian region and complement existing manufacturing capacities in Europe and North America.

The investment furthers Sanofi’s commitment to driving the future of vaccines by expanding manufacturing capabilities to ensure quality and scale, while responding to the risk of future pandemics.

The project is expected to create up to 200 local jobs and enhance Singapore’s position as a regional innovation hub for the healthcare industry.

“As a major healthcare player, it’s our responsibility to act and to meet the unprecedented growing demands for vaccines.

By investing in a new production site in Singapore, Sanofi is aiming to strengthen production capacity to meet ever-growing global demands on vaccines, and answer more rapidly to future pandemics,” said Thomas Triomphe, Executive Vice President and Global Head of Sanofi Pasteur. 

“We are very pleased by the strong collaboration with the Singapore Economic Development Board to achieve this exciting milestone.”

“Sanofi’s decision to locate its first-in-Asia digitally-enabled vaccine production center in Singapore, to supply markets in the region and beyond, is an endorsement of Singapore’s position as a leading centre for advanced manufacturing” said Dr Beh Swan Gin, Chairman, EDB. 

“EDB will continue to promote digitalization, automation and innovation to transform the manufacturing sector and create good jobs for Singaporeans.”

This factory will be designed around a central unit housing several fully digitalized modules that allow production of three to four vaccines simultaneously, versus only one in current industrial sites.

In addition, the factory will have the flexibility to leverage multiple vaccine manufacturing technology platforms based on different cell types This modularity and flexibility will allow the production of a specific vaccine to be prioritized in a faster timeframe depending on public health needs.

The project is currently entering its design phase with construction expected to begin in Q3 2021.

The five-year project will see the site fully operational in Q1 2026 once all qualifications and validations of the first manufactured vaccine have been completed.

In the context of Sanofi’s environmental strategy, the site is designed to be carbon-neutral, and to drastically reduce both resource consumption and waste production.”

https://www.sanofi.com/en/media-room/press-releases/2021/2021-04-12-07-00-00-2207870

AZ’s Update on the DARE-19 Phase III trial for Farxiga in COVID-19

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AZ’s Update on the DARE-19 Phase III trial for Farxiga in COVID-19

April 12, 2021: “AstraZeneca and Saint Luke’s Mid America Heart Institute today announced high-level results of the primary analysis from the DARE-19 Phase III trial assessing the potential of Farxiga (dapagliflozin) to treat patients hospitalised with COVID-19 who are at risk of developing serious complications.

The trial did not achieve statistical significance for the primary endpoint of prevention measuring organ dysfunction and all-cause mortality, and the primary endpoint of recovery measuring a change in clinical status (from early recovery to death), at 30 days.

DARE-19 was the first Phase III trial to evaluate the safety and efficacy of a sodium-glucose co-transporter-2 (SGLT2) inhibitor in patients hospitalised with COVID-19 who also have risk factors for developing serious complications, including hypertension (HTN), type-2 diabetes (T2D), atherosclerotic cardiovascular disease (ASCVD), heart failure (HF) or chronic kidney disease (CKD) Stages 3-4.

Cardiac, renal and metabolic comorbidities have been associated with poor outcomes and death in patients hospitalised with COVID-19.

Mikhail N. Kosiborod, M.D., cardiologist at Saint Luke’s Mid America Heart Institute, Vice President of Research at Saint Luke’s Health System, and principal investigator of DARE-19, said: “DARE-19 provided important data on the potential benefits and risks of using SGLT2 inhibitors to treat hospitalised patients with COVID-19.

While the trial did not achieve statistical significance, the findings are very interesting and valuable, and will inform future clinical science.

Also, of importance, we learned that dapagliflozin’s well-established safety profile was consistent in DARE-19.”

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, said: “Prior to the DARE-19 Phase III trial, there was little data on the use of SGLT2 inhibitors in hospitalised patients with COVID-19 and we have now helped to fill this knowledge gap. We look forward to the efficacy and safety data being presented in the coming weeks.”

The safety and tolerability profile for Farxiga at 30 days in the trial was consistent with the well-established safety profile of the medicine.

The full DARE-19 trial results will be presented at the American College of Cardiology Scientific Sessions in May 2021.

DARE-19
DARE-19 was an international, randomised, double-blind, placebo-controlled, investigator-sponsored Phase III trial in 1,250 patients evaluating the efficacy and safety of Farxiga in addition to background local standard of care therapy in adults who are hospitalised with COVID-19 at the time of trial enrolment.

Patients enrolled in DARE-19 also had a medical history of HTN, ASCVD, heart failure, T2D or CKD Stages 3-4 and received Farxiga or placebo for 30 days.

Farxiga
Farxiga (dapagliflozin) is a first-in-class, oral, once-daily SGLT2 inhibitor.

The research for Farxiga is advancing from cardiorenal effects to prevention and organ protection as science continues to identify the underlying links between the heart, kidneys and pancreas. Damage to one of these organs can cause the other organs to fail – contributing to leading causes of death worldwide, including T2D, HF and CKD.

For nearly a decade Farxiga has been an effective monotherapy and part of combination therapy as an adjunct to diet and exercise to improve glycaemic control in adults with T2D.

Following results from the landmark DECLARE-TIMI 58 Phase III cardiovascular (CV) outcomes trial, it is approved in adults with T2D to reduce the risk of hospitalisation for heart failure or CV death when added to standard of care. 

Farxiga is also the first SGLT2 inhibitor approved for the treatment of HFrEF in adults with and without T2D.

In August 2020, results from the DAPA-CKD Phase III trial demonstrated that Farxiga achieved unprecedented reduction in the composite risk of kidney failure and CV or renal death in patients with CKD with and without T2D versus placebo.

It is now the first SGLT2 inhibitor shown to significantly improve overall survival in a renal outcomes trial for this patient population and provide organ protection. Farxiga is not yet approved for the treatment of CKD.


AZ’s Farxiga-Tracked heart failure following acute myocardial infarction

DapaCare is a robust programme of clinical trials to evaluate the potential CV, renal and organ protection benefits of Farxiga.

It includes more than 35 completed and ongoing Phase IIb/III trials in more than 35,000 patients, as well as more than 2.5 million patient-years’ experience.

It is currently being assessed in patients with HFpEF in the DELIVER Phase III trial. 

Farxiga is also being tested in patients without T2D following an acute myocardial infarction (MI) or heart attack in the DAPA-MI Phase III trial – a first of its kind, indication-seeking registry-based randomised controlled trial.

AstraZeneca in CVRM
Cardiovascular, Renal and Metabolism (CVRM) together forms one of AstraZeneca’s three therapy areas and is a key growth driver for the Company.

By following the science to understand more clearly the underlying links between the heart, kidneys and pancreas, AstraZeneca is investing in a portfolio of medicines for organ protection and improve outcomes by slowing disease progression, reducing risks and tackling co-morbidities.

The Company’s ambition is to modify or halt the natural course of CVRM diseases and potentially regenerate organs and restore function, by continuing to deliver transformative science that improves treatment practices and cardiovascular health for millions of patients worldwide.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2021/update-on-farxiga-covid-19-dare-19-phase-iii-trial.html

FDA Authorizes Device that Uses AI to Detect Potential Signs of Colon Cancer

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FDA Authorizes Device that Uses AI to Detect Potential Signs of Colon Cancer

April 09, 2021: “The U.S. FDA authorized marketing of the GI Genius, the first device that uses artificial intelligence (AI) based on machine learning to assist clinicians in detecting lesions (such as polyps or suspected tumors) in the colon in real time during a colonoscopy.

“Artificial intelligence has the potential to transform health care to better assist health care providers and improve patient care.

When AI is combined with traditional screenings or surveillance methods, it could help find problems early on, when they may be easier to treat,” said Courtney H. Lias, Ph.D. acting director of the GastroRenal, ObGyn, General Hospital and Urology Devices Office in the FDA’s Center for Devices and Radiological Health.

“Studies show that during colorectal cancer screenings, missed lesions can be a problem even for well-trained clinicians.

With the FDA’s authorization of this device today, clinicians now have a tool that could help improve their ability to detect gastrointestinal lesions they may have missed otherwise.”

According to the National Institutes of Health, colorectal cancer is the third leading cause of death from cancer in the United States.

Colorectal cancer usually starts from polyps or other precancerous growths in the rectum or the colon (large intestine).

As part of a colorectal cancer screening and surveillance plan, clinicians perform colonoscopies to detect changes or abnormalities in the lining of the colon and rectum.

A colonoscopy involves threading an endoscope (thin, flexible tube with a camera at the end), through the rectum and throughout the entire length of the colon, allowing a clinician to see signs of cancer or precancerous lesions.

The GI Genius is composed of hardware and software designed to highlight portions of the colon where the device detects a potential lesion. The software uses artificial intelligence algorithm techniques to identify regions of interest.

During a colonoscopy, the GI Genius system generates markers, which look like green squares and are accompanied by a short, low-volume sound, and superimposes them on the video from the endoscope camera when it identifies a potential lesion.

These signs signal to the clinician that further assessment may be needed, such as a closer visual inspection, tissue sampling, testing or removal, or ablation of (burning) the lesion.

The GI Genius is designed to be compatible with many FDA-cleared standard video endoscopy systems.

The FDA assessed the safety and effectiveness of the GI Genius through a multicenter, prospective, randomized, controlled study in Italy with 700 subjects 40-80 years old who were undergoing a colonoscopy for colorectal cancer screening, surveillance, positive results from a previous fecal immunochemical (fecal occult blood) test for blood in the stool or gastrointestinal symptoms of possible colorectal cancer.

The primary analyses from the study were based on a sub-population of 263 patients who were being screened or surveilled every 3 years or more.

Study subjects underwent either white light standard colonoscopy with the GI Genius (136 patients) or standard white light colonoscopy alone (127 patients).

The primary endpoint of the study compared how often colonoscopy plus GI Genius identified a patient with at least one lab-confirmed adenoma (precancerous tumor) or carcinoma (cancerous tumor) to how often standard colonoscopy made the same identifications.

In the study, colonoscopy plus GI Genius was able to identify lab-confirmed adenomas or carcinomas in 55.1% of patients compared to identifying them in 42.0% of patients with standard colonoscopy, an observed difference of 13%.

While use of this device led to more biopsies being performed, there were no adverse events reported with the additional biopsies, such as perforations, infections or bleeding. However, there was a slight increase in the number of lesions biopsied that were not adenomas.

The GI Genius is not intended to characterize or classify a lesion, nor to replace lab sampling as a means of diagnosis.

The device does not provide any diagnostic assessments of colorectal polyp pathology, nor does it suggest to the clinician how to manage suspicious polyps.

GI Genius only identifies regions of the colon within the endoscope’s field of view where a colorectal polyp might be located, allowing for a more extended examination in real time during colonoscopy.

It is up to the clinician to decide whether the identified region actually contains a suspected lesion, and how the lesion should be managed and processed per standard clinical practice and guidelines.

The FDA reviewed the GI Genius through the De Novo premarket review pathway, a regulatory pathway for some low- to moderate-risk devices that are novel and for which there is no legally marketed predicate device to which the device can claim substantial equivalence.

The FDA Center for Devices and Radiological Health’s Digital Health Center of Excellence  is looking to the future of AI-based technology, including in its Artificial Intelligence and Machine Learning (AI/ML)-Based Software as a Medical Device (SaMD) Action Plan.”

The FDA granted marketing authorization of the GI Genius to Cosmo Artificial Intelligence, Ltd.”

https://www.fda.gov/news-events/press-announcements/fda-authorizes-marketing-first-device-uses-artificial-intelligence-help-detect-potential-signs-colon

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