Neurodegenerative disease
Avidity has been given the go-ahead by the FDA to request accelerated approval of delpacibart braxlosiran—potentially the first disease-modifying treatment for facioscapulohumeral muscular dystrophy—with an application in the second half of 2026.
After a major shareholder pushed back, Keros is returning half of its capital to investors in a move that Guggenheim analysts called “a positive step forward.”
Analysts reacted positively to the news that uniQure is in alignment with the FDA on an accelerated approval pathway and on target for a Q1 2026 submission for its one-time gene therapy for Huntington’s disease—but patients have been here before.
Disappointing results for iluzanebart come shortly after Vigil Neuroscience struck a buy-out deal with Sanofi, but analysts say the outcome is unsurprising and shouldn’t affect the deal.
With $90 million to start, Syndeio has a lead asset in Phase II clinical trials for major depressive disorder, with plans to soon launch a biomarker trial in Alzheimer’s disease.
Analysts at BMO Capital Markets said in a weekend note that a non-invasive blood test could help boost uptake of Alzheimer’s disease therapies.
The FDA also changed its tune and is now planning to convene an advisory committee to discuss Biohaven’s application.
To more effectively treat neurodegenerative conditions, we first need diagnostic tools that lend a more complete picture of protein aggregates in the brain.
The Alchemab deal will further strengthen Lilly’s early-stage pipeline for amyotrophic lateral sclerosis, coming less than a year after the pharma licensed QurAlis’ antisense oligonucleotide to correct a specific protein alteration in ALS.
The biotech’s Huntingtin-targeting molecule lowered blood levels of the protein and elicited functional improvements in earlier-stage patients, but results were not as robust in other biomarkers or with patients at later stages of the disease.
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