Neurodegenerative disease
The majority of the large pharmas have now reported third quarter earnings—spoiler alert: Eli Lilly made more money than anyone expected. Plus, the FDA dropped another initiative aimed at accelerating rare disease treatments; HHS greenlights the compounding of six unapproved peptides; and uniQure’s embattled gene therapy for Huntington’s disease has finally been accepted for review.
Novartis is discontinuing a neurodegeneration alliance with BioArctic in which the companies aimed to develop a drug candidate that could cross the blood-brain barrier.
Acumen will implement cash-saving strategies and assess its options after sabirnetug failed to beat placebo, leaving the biotech without a clinical program.
RNAi specialist Alnylam Pharmaceuticals will advance the Huntington’s disease drug brezosiran into a placebo-controlled study in 2027 once regulatory alignment is achieved.
Troriluzole, in development for spinocerebellar ataxia, was rejected nearly a year ago when the FDA took issue with the design of Biohaven’s registrational study. The agency has now agreed to review a new application and render a decision in the first quarter of 2027.
Arrowhead Pharmaceuticals’ siRNA candidate achieved “compelling” tau reductions in an early study of healthy volunteers, with a magnitude of effect that seems to outperformother blood-brain barrier platforms, according to Stifel.
The Takeda spinout showed that a high dose of solengepras can shorten the periods during which difficult Parkinson’s disease symptoms like involuntary movements and sleepiness.
After a series of clinical-stage failures, Alector is resetting around its preclinical blood-brain barrier delivery technology, securing a big pharma partnership along the way.
In the Phase 3 TEMPO program, AbbVie’s Juvmo elicited significant improvements in motor symptoms and prolonged periods with good symptom control.
A growing number of biotechs are entering the market with newly launched medicines, a pattern indicative of “improving capital markets,” according to Jefferies’ Andrew Tsai.
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