Pharmaceutical Industry News Roundup: Gene Therapy Sales Dip, New Obesity Research, and FDA Approvals

Sarepta's Elevidys Faces Challenges Amid Safety Concerns

Sarepta Therapeutics reported a significant decrease in sales for its Duchenne muscular dystrophy gene therapy, Elevidys, in the second quarter of 2025. The therapy generated $282 million, marking a 25% decline from the previous quarter. Despite this dip, the figures slightly exceeded consensus estimates.

Elevidys recently returned to the market for select Duchenne patients following a brief pause in shipments due to a standoff with the Food and Drug Administration. However, analysts remain cautious about its future prospects, citing safety concerns that may impact demand among patients and physicians.

Leerink Partners analyst Joseph Schwartz predicts further sales decline between the second and third quarters, emphasizing the uncertainty surrounding the therapy's long-term market performance.

Neumora Ventures into Obesity Research as Industry Focus Shifts

Neumora Therapeutics, a prominent neuroscience biotech company, has announced its entry into obesity research. The company plans to prioritize obesity as the lead indication for its preclinical compound, NMRA-215. With aims to advance the drug into clinical testing by early next year, Neumora is currently conducting experiments on mice.

CEO Paul Berns highlighted the growing evidence supporting centrally acting drugs for weight loss in obesity treatment. He expressed confidence that Neumora's expertise in developing highly brain-penetrant chemistry would support their advancement in this field.

This move comes as part of a broader industry trend, with increasing focus on obesity research and treatment options.

FDA Approvals and Clinical Advancements

The FDA has granted approval to Jazz Pharmaceuticals' drug Modeyso for a rare and aggressive type of brain cancer. The treatment is indicated for adults and children aged one year or older with diffuse midline glioma positive for a specific mutation and who have progressive disease following prior therapy.

Modeyso represents the first treatment to reach the market for this tumor type, which affects approximately 2,000 people in the U.S. annually. Jazz Pharmaceuticals acquired the drug through a $935 million deal to purchase Chimerix earlier this year.

In other clinical developments, Novo Nordisk announced plans to advance a Prothena drug for transthyretin amyloidosis cardiomyopathy into Phase 3 testing. The program for the drug, coramitug, is set to begin this year. Prothena has already earned $100 million through its deal with Novo Nordisk and could receive up to $1.2 billion overall.

AI and Investment in Drug Discovery

Chai Discovery, an AI-focused biotech startup, has secured a $70 million Series A funding round led by Menlo Ventures. The company utilizes computing tools to predict biochemical molecule interactions, aiming to accelerate drug development processes.

This latest funding follows a nearly $30 million seed round from investors including Thrive Capital, OpenAI, and Dimension last year. Former Pfizer Chief Scientific Officer Mikael Dolsten has joined Chai's board of directors alongside the recent funding, further strengthening the company's position in the AI-driven drug discovery landscape.