Amylyx ALS drug falls short in phase 3 trial

March 8, 2024

Amylyx Pharmaceuticals reported this week that a confirmatory study evaluating its approved amyotrophic lateral sclerosis (ALS) drug, Relyvrio, did not meet its primary or secondary objectives.

According to the drugmaker, the phase 3 PHOENIX trial, which enrolled 664 adults living with ALS, did not meet its primary endpoint of reaching statistical significance as measured by change from baseline in the revised ALS functional rating scale after 48 weeks.

Accordingly, Amylyx will pause its promotion of Relyvrio. The drugmaker is contemplating a potential market withdrawal, pending discussions with regulatory bodies and the ALS community within the next eight weeks.

Following a challenging journey, Relyvrio's 2022 approval came shortly after an FDA Advisory Committee, convening for the second time, changed its previous stance to endorse the drug.

Relyvrio, a pill that combines sodium phenylbutyrate and taurursodiol, aims to protect neurons by inhibiting pathways that lead to cell death within the mitochondria and endoplasmic reticulum. It has been authorized for use either as a standalone treatment or alongside other therapies. In previous clinical studies, Relyvrio demonstrated potential in decelerating the deterioration of physical abilities in individuals diagnosed with ALS, marking a significant advancement in the management of the neurodegenerative disease. 

Amylyx says it is still committed to investigating the drug's potential for treating other neurodegenerative diseases like Wolfram syndrome and progressive supranuclear palsy, with studies ongoing.