Neurodegenerative disease
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.
Biohaven will use the money to bankroll commercial preparations for the spinocerebellar ataxia drug candidate troriluzole, which is currently under FDA review with a decision expected in the third quarter.
GSK is paying to access ABL Bio’s Grabody-B platform, which potentially enables therapies to cross the blood-brain barrier.
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.
Biopharma leaders react to the forced resignation of CBER Head Peter Marks as RFK Jr.’s promised job cuts begin at the FDA; Novo Nordisk presents mixed results from oral semaglutide in cardiovascular disease; the EU’s Committee for Medicinal Products for Human Use declines to recommend Eli Lilly’s Alzheimer’s drug; and pharma R&D returns grew in 2024.
The European Union’s CHMP said that the benefits of the drug, already approved in the U.S., do not outweigh the risk of potentially fatal brain swelling and bleeding.
WVE-N531, an oligonucleotide, elicited significant functional benefit and reversal of muscle damage in the Phase II FORWARD-53 trial. Wave plans to file for accelerated approval of the candidate in 2026.
Eisai’s new fiscal 2027 forecast for Leqembi is roughly 50% lower than its projections a year ago.
President Donald Trump continues to warn of tariffs on the pharmaceutical industry; Susan Monarez replaces Dave Weldon as CDC director nominee; Novo Nordisk joins the triple-G race; Alnylam wins approval for Amvuttra in ATTR-CM; and Cassava Sciences ends development of simufilam in Alzheimer’s.
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