Tag Archive for: ALS

The companies announced they will terminate the development of their experimental treatment for amyotrophic lateral sclerosis (ALS) after failing to show improvement in patients in an early-to-mid stage study.

After a years-long drought, progress in the amyotrophic lateral sclerosis (ALS) space began to accelerate with the 2022 approval of Amylyx’s Relyvrio and Biogen and Ionis’ Qalsody in 2023. But now, that progress appears to have stalled. Earlier this month, Amylyx announced it would pull Relyvrio from the market after a failed Phase III trial, and closely watched trials from Seelos Therapeutics and Sanofi and Denali Therapeutics recently missed their primary endpoints.

The company will withdraw its amyotrophic lateral sclerosis (ALS) drug – its only product in the market – from the U.S. and Canada after the treatment failed in a key late-stage trial.

Topline data from the Phase III PHOENIX trial of Relyvrio, which won approval in 2022, showed no significant difference on either the primary or secondary endpoints, according to Amylyx.

The past two years have seen a pair of new treatments approved for a particularly intractable neurodegenerative disease —amyotrophic lateral sclerosis. But while Amylyx’s Relyvrio and Biogen’s Qalsody have crossed the FDA finish line, others have stumbled in a regulatory space that experts say is still evolving.

The agency found data from the company’s mid-stage study for the treatment of a type of neurodegenerative disease to be insufficient to support accelerated approval.

The decision comes after Amylyx in June requested a formal re-examination of the initial negative opinion adopted by the Committee for Medicinal Products for Human Use of the European Medicines Agency.

The Japanese multinational pharma is pledging up to $580 million in a development and commercialization deal with AcuraStem for the latter’s PIKFYVE program for amyotrophic lateral sclerosis.

The study will use a robot to surgically place a brain-computer interface (BCI) implant in a region of the brain that controls the intention to move, Neuralink said, adding that its initial goal is to enable people to control a computer cursor or keyboard using their thoughts alone.

After its Biologics License Application was rejected twice by the FDA, BrainStorm’s Phase III data suggest its amyotrophic lateral sclerosis candidate significantly lowers neurofilament light chain levels.