Neurodegenerative disease

Misses from amyotrophic lateral sclerosis hopefuls Denali Therapeutics and partners AbbVie and Calico Life Sciences mark the latest setbacks for the controversial platform trial, the results from which have largely mirrored the dismal success rate in ALS overall.

Denali’s failure on Monday continues biopharma’s losing streak against amyotrophic lateral sclerosis. PTC Therapeutics and Amylyx have seen similarly disappointing results.

Among the FDA’s pending decisions for this quarter are Vertex’s non-opioid pain drug and Sanofi’s RNA interference therapy for hemophilia A and B.

Effectively treating and preventing this common form of dementia will require a cocktail of drugs and a combination of approaches, as well as a drive toward early detection.

With two earlier trials meeting their primary endpoints, Axsome claimed it has the data to support a filing for FDA approval in the second half of 2025.

The report comes just two days after Novartis announced its own Parkinson’s drug failure.

Even as Biogen and Eisai’s Leqembi and Eli Lilly’s Kisunla slowly roll out onto the market, experts question the efficacy of these anti-amyloid antibodies and the amyloid hypothesis overall.

GSK, Gilead and Arcellx, Vertex and more present new data at the American Society of Hematology annual meeting just as sickle cell therapies Casgevy and Lyfgenia have a new outcomes-based payment model; Eli Lilly and Novo Nordisk pump new funds into manufacturing; and AbbVie makes a Cerevel comeback while uniQure clears a path toward accelerated approval in Huntington’s disease.

In a Type B meeting, the FDA signified that it will allow uniQure to use a natural history control, the composite Unified Huntington’s Disease Rating Scale, and neurofilament light chain levels to support the accelerated approval of its gene therapy AMT-130.

Tavapadon improved motor and daily living complications at week 26. The news comes nearly one month to the day after AbbVie announced the Phase II failure of another key Cerevel asset, emraclidine.