Tag Archive for: amyotrophic lateral sclerosis

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.

In its briefing documents, the agency said it does not believe there is sufficient evidence to support NurOwn’s clinical benefit, adding there were large amounts of missing data.

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.

The Massachusetts-based biopharma became the second company this week to drop its amyotrophic lateral sclerosis hopeful after it failed to elicit significant improvement compared with placebo.

Biogen Inc. said on Tuesday it would pause or discontinue studies of some experimental drugs and therapies and focus on more promising treatments, in the latest attempt by the new chief to cut costs.