BioSpace

In this edition of The Weekly, Managing Editor

Also, on Tuesday, Eli Lilly announced that an advisory committee meeting will be held for its Alzheimer’s drug donanemab on June 10. Lilly is aiming for full approval of the anti-amyloid antibody after accelerated approval was denied in January 2023. And the race between Vertex and bluebird bio’s gene therapies Casgevy and Lyfgenia is heating up.

In this special edition of The Weekly, we discuss the much-anticipated annual meeting of the American Society of Gene & Cell Therapy in Baltimore. It starts Tuesday and will hit on wide-ranging issues facing the cell and gene therapy (CGT) space, from new therapeutic advances to safety concerns and regulatory considerations to manufacturing and commercialization.

Big hitters in the CGT space will be there. Sarepta, whose gene therapy for Duchenne muscular dystrophy Elevidys received accelerated approval in June 2023, will be presenting, as will CRISPR Therapeutics, which in the last few months brought to market Casgevy, its Vertex-partnered CRISPR-based therapy, for sickle-cell disease and beta thalassemia.

There will also be plenty of big names, including Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research. Marks will be speaking in two sessions at ASGCT 2024, one on global regulatory convergence and the other on the regulation of CGTs, from IND to BLA.

Stay tuned for this week’s regular episode on Wednesday when we discuss what we’re seeing and hearing in Baltimore.

J&J and BMS’ challenges to Medicare drug price negotiations shut down in federal court less than a week after BMS announced it was laying off more than 2,000 employees. 

Earlier this week, a federal judge in New Jersey added fuel to the ongoing legal battle between Big Pharma and the government over the Inflation Reduction Act. The judge on Monday dismissed claims from Johnson & Johnson and Bristol Myers Squibb that Medicare’s drug price negotiations program is unconstitutional.  

The big news last week also involved BMS, which announced it will let go 2,200 people by the end of 2024 in what the company is calling a “strategic productivity initiative.” Layoffs are already underway in New Jersey, California and elsewhere, and may be related to the pharma giant’s recent M&A spending spree.  

Elsewhere around the industry, Q1 earnings season rolls on, with Sanofi and AstraZeneca also announcing cuts to their pipeline, while Gilead, Merck, and Lilly all touted numbers that exceeded expectations. 

Finally, on the regulatory front, X4 Pharmaceuticals got a win this week with the approval of its first commercial drug—and the first therapy for an ultra-rare disease called WHIM. 

The potential impacts on clinical trial outcomes warrant a discussion surrounding the current definition of women’s health.

In the second episode on our series on women's health, we discuss what may happen to future generations if women, the custodians of generational health, are not comfortable sharing complete medical histories. Additionally, we discuss how broadening the definition will open funding to address women’s health.

Host 

⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, ⁠BioSpace⁠  

Guests

⁠Sans Thakur⁠, Founder and Chairwoman of ⁠Tower Capital⁠

⁠Chia Chia Sun⁠, Chief Commercial Officer at ⁠Fab Biopharma⁠

⁠Elisa Cascade⁠, Chief Product Officer of at ⁠Advarra⁠

Shawana Moore, Medical Advisor on the ⁠Advisory Council for Healthy Women⁠

⁠Katie Schubert⁠,  President and Chief Executive Officer of the ⁠Society for Women’s Health

Sanofi will cut an undisclosed number of jobs as it revamps its U.S. vaccines commercial operations, as well as 99 employees from its Belgian sites—moves that are part of a full pipeline reprioritization. Meanwhile, the company got a win this week, with its investigational BTK inhibitor rilzabrutinib generating positive results in a Phase III study for adult patients with persistent or chronic immune thrombocytopenia. Sanofi will present its Q1 earnings tomorrow.

Meanwhile, the FDA announced an update to the boxed warnings it now requires for CAR T cell therapies regarding the heightened risk of secondary T cell malignancies. Additional requirements involve various aspects of the label, including the warnings and precautions, post-marketing experience, patient counseling information and medication guide sections.

And finally, GLP-1s are once again making headlines, as Eli Lilly’s Zepbound produced positive Phase III results that could earn the weight-loss drug a label expansion into sleep apnea and a new company, New York–based Metsera emerged from stealth with $290 million in financing. The startup’s pipeline includes multiple GLP-1 agonists in early-stage development.

The past two weeks have seen Amylyx lose its only marketed product but receive numerous accolades for its decision to voluntarily withdraw Relyvrio after a failed Phase III trial in amyotrophic lateral sclerosis. Analysts and insiders spoke with BioSpace this week about the company's future and a possible regulatory precedent for other drugs approved based on a single trial.

Elsewhere, antibody-drug conjugates continue to steal the show in oncology. At the American Association for Cancer Research’s annual meeting, Merck and Kelun Biotech presented Phase I/II data showing that their TROP2-directed ADC elicited promising disease control and a potential survival benefit in gastric cancer patients, and Bristol Myers Squibb–backed TORL netted $158 million in an oversubscribed Series B to advance its pipeline of novel ADC candidates.

Finally, drug shortages in the U.S. have reached an all-time high, according to the latest data from the American Society of Health-System Pharmacists, with both basic and life-saving products on the list.

Last month, President Biden signed an Executive Order on Advancing Women’s Health Research and Innovation, signalling the significance of women's health to the government and consequently investors.

While women’s clinical trial participation has increased, clinical trials still largely are not designed for women. In this episode, our guests discuss the many areas where trials can be improved, such as human and historical diagnosis and screening biases, accessibility, data capturing, and more. They further discuss the importance of women’s health for the future.

Host 

⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace  

Guests

Sans Thakur, Founder and Chairwoman of Tower Capital

Chia Chia Sun, Chief Commercial Officer at Fab Biopharma

Elisa Cascade, Chief Product Officer of at Advarra

Shawana Moore, Medical Advisor on the Advisory Council for Healthy Women

Katie Schubert,  President and Chief Executive Officer of the Society for Women’s Health

Conference season is in full swing, with the American Association for Cancer Research and American College of Cardiology concurrently holding their annual meetings in San Diego and Atlanta, respectively. From opposite coasts, biopharma companies are presenting their latest data in the cancer and cardiology spaces, both hits and misses alike.

Oncology continues to be a hot area for investment, with several highly anticipated readouts coming down the pike. In addition to all the data coming out of AACR, conference organizers this year put together the meeting’s first-ever industry event, bringing together investigators, investors and Big Pharma.

Over at ACC, one exciting race to watch will be between Ionis and Arrowhead, both of whom presented positive data on their ApoC3-targeting drugs for diseases associated with elevated triglyceride levels.

Meanwhile, as the BIOSECURE Act sits before Congress, biopharma companies detail the potential fallout to the growing geopolitical tensions between the U.S. and China.

WuXi AppTec was once again in the news this week, with speculation that the China-based biotech allegedly handed a U.S. client’s intellectual property over to the Chinese government without consent.

Meanwhile, Lonza’s $1.2 billion buy of a Roche biologics plant in California—one of the world’s largest biologics manufacturing facilities by volume—bodes well for the CDMO market, and BMS pulls ahead of Amgen in the race to bring a fully approved KRAS inhibitor to market for patients with certain types of non-small cell lung cancer, acing a confirmatory Phase III trial for Krazati.

The pharmaceutical industry is facing critical attention, particularly around drug pricing and development costs. Drug development cost is about 10% of the total healthcare spend in the United State. Broader issues such as local monopolies, utilization, unit, and costs and local monopolies, politics and a fragmented payer system contribute to the increasingly high costs to patient.

Challenging as these issues are, they must be addressed to lower patient costs. In this episode, our guests discuss more efficient drug development practices but also some of the broader issues within the United States healthcare system and the IRA’s impacts on not only US patients but the world. 

Host 

⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace  

Guests

⁠⁠Ali Pashazadeh⁠⁠, CEO,Treehill Partners

⁠⁠Dan Smithey⁠⁠, President, CEO and Co-founder, Serán BioScience

⁠⁠Matthew Price⁠⁠, COO and Co-founder, Promontory Therapeutics

⁠⁠Peter Rubin⁠⁠, Executive Director, No Patient Left Behind

⁠⁠Rob Williamson⁠⁠, President and COO, Triumvira Immunologics