Tag Archive for: amyotrophic lateral sclerosis

At this week’s American Academy of Neurology annual meeting, Amylyx provided additional data from its Phase III amyotrophic lateral sclerosis study showing the full extent of Relyvrio’s failure.

Amylyx Pharmaceuticals on Thursday released interim results from its ongoing Phase II HELIOS trial, showing that its lead asset AMX0035 (sodium phenylbutyrate and taurursodiol) improved pancreatic function and glycemic control in adult patients with the rare, inherited neurodegenerative disease Wolfram syndrome.

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.

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.