Amylyx Discontinues Rare Disease Program After Relyvrio Fails to Show Benefit Over Placebo

Amylyx has discontinued its rare disease program involving Relyvrio (also called AMX0035) after the drug failed to demonstrate benefit over placebo in a phase 2/3b trial for progressive supranuclear palsy (PSP)1.

The trial found no difference in disease progression or severity between patients receiving AMX0035 and those receiving placebo at 24 weeks1.

Based on these results, the company stopped both the phase 2b trial and a related open-label extension study, and decided not to proceed with the phase 3 portion1.

Relyvrio was previously pulled from the ALS market after failing a confirmatory phase 3 trial, where it also did not show significant benefit over placebo2 3 4.

Amylyx stated the drug was generally well-tolerated, with safety data consistent with earlier trials, but the lack of efficacy led to the discontinuation decision1.

Relyvrio/AMX0035 had initially been approved for ALS after earlier, smaller-scale efficacy signals, but could not replicate those results in larger studies3 4 5.