Denali Therapeutics Inc.
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Biogen, Eli Lilly, Merck and Novartis spent more than $20 billion to absorb biotechs with promising or approved drugs; the rare disease space notched approvals for therapies from Denali Therapeutics, Rocket Pharmaceuticals and Biogen; and Wave’s stock lost half its value after its RNA-based obesity candidate failed to impress investors.
The FDA has some big verdicts lined up in the second quarter, including one for a closely watched obesity drug that many anticipate will further intensify competition in weight loss.
Avlayah is the first Hunter syndrome therapy approved to address the condition’s neurologic complications, according to Tracy Beth Høeg, acting director of the Center for Drug Evaluation and Research.
The Hunter syndrome space suffered a setback in February when the FDA turned down REGENXBIO’s investigational gene therapy, raising urgent questions about whether competitor Denali Therapeutics can clear the agency’s bar next month.
The discovery of a tumor in a patient who received REGENXBIO’s gene therapy for Hurler syndrome prompted the FDA to place a hold on that program along with the company’s Hunter syndrome program, which is awaiting an FDA decision on or before Feb. 8.
From opening new therapeutic mechanisms to repairing neuronal damage, investigational molecules from Ventyx Therapeutics, AC Immune, Gain Therapeutics and more could shape the future of Parkinson’s disease treatment.
Hypersensitivity reactions in a mouse model prompted the agency to suspend Denali’s planned Phase I development for DNL952 for Pompe disease.
After a season of regulatory upheaval, obesity and rare genetic diseases will likely remain major themes for biopharma in 2025, according to Jefferies.
After some high-profile crashes, the one-time biotech darling is inching toward success with its Hunter syndrome treatment, which today began a rolling BLA for accelerated approval.
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