Pharmaceutical Industry Update: Gene Therapy Sales Dip, New Approvals, and AI-Driven Drug Discovery

Sarepta's Elevidys Faces Challenges Amid Safety Concerns

Sarepta Therapeutics reported a 25% quarter-on-quarter decline in sales for its Duchenne muscular dystrophy gene therapy, Elevidys. Despite the dip, second-quarter sales of $282 million slightly exceeded consensus estimates. The therapy, which recently returned to the market following a brief FDA-mandated pause, faces an uncertain future due to safety concerns that may impact demand. Leerink Partners analyst Joseph Schwartz anticipates further sales decline in the third quarter, with long-term prospects remaining unclear.

FDA Approves Jazz Pharmaceuticals' Brain Cancer Drug

In a significant development for patients with rare and aggressive brain cancer, the FDA has approved Jazz Pharmaceuticals' Modeyso. The drug is indicated for adults and children aged one year or older with diffuse midline glioma positive for a specific mutation and showing progressive disease after prior therapy. Modeyso, acquired through Jazz's $935 million acquisition of Chimerix, is the first treatment to reach the market for this type of tumor, which affects approximately 2,000 people annually in the U.S.

Emerging Players in Neuroscience and AI-Driven Drug Discovery

Neumora Therapeutics, a neuroscience-focused biotech that has raised over $800 million, is expanding into obesity research. The company plans to prioritize obesity as the lead indication for its preclinical compound NMRA-215, with clinical testing expected to begin early next year. CEO Paul Berns emphasized the importance of centrally acting drugs in driving weight loss and the company's expertise in developing brain-penetrant chemistry.

Meanwhile, AI-focused biotech startup Chai Discovery has secured a $70 million Series A funding round led by Menlo Ventures. The company, which utilizes computing tools to predict biochemical molecule interactions, previously raised a $30 million seed round from investors including Thrive Capital, OpenAI, and Dimension. Former Pfizer Chief Scientific Officer Mikael Dolsten has joined Chai's board of directors following this latest funding round.

Advancements in Cardiac and Obesity Treatments

Novo Nordisk announced plans to advance Prothena's drug for transthyretin amyloidosis cardiomyopathy into Phase 3 testing. The program for coramitug, acquired by Novo in 2021, is set to begin this year. Prothena has already earned $100 million through the deal and could receive up to $1.2 billion overall, with additional milestone payments tied to enrollment criteria for the Phase 3 trial.