CAMBRIDGE, Mass. - Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) today disclosed that its Phase 3 clinical trial for AMX0035, aimed at treating amyotrophic lateral sclerosis (ALS), failed to meet the primary efficacy endpoint. The study, known as PHOENIX, did not show statistical significance in changing the ALS Functional Rating Scale-Revised (ALSFRS-R) total score at 48 weeks.
Despite the setback, the trial reinforced AMX0035's safety and tolerability profile. The company plans to review the data with regulatory authorities and the ALS community within the next eight weeks to determine the future of RELYVRIO/ALBRIOZA in the ALS market, which may include voluntary withdrawal.
AMX0035, which is sold as RELYVRIO in the U.S. and ALBRIOZA in Canada, will remain available for ALS patients during this period. However, Amylyx has paused its promotion. Patient support services related to the medication will continue to be provided.
The PHOENIX study enrolled 664 adults with ALS, with participants randomized to receive either AMX0035 or a placebo. No significant differences in ALSFRS-R total score change from baseline at Week 48 were observed between the treatment group and the placebo group.
The company's co-CEOs, Justin Klee and Joshua Cohen, expressed their disappointment but emphasized their commitment to the ALS community, stating that the trial's findings will contribute to future ALS research. Amylyx remains dedicated to exploring AMX0035's potential in other neurodegenerative diseases such as Wolfram syndrome and progressive supranuclear palsy.
The ongoing Phase 3 ORION study of AMX0035 in progressive supranuclear palsy and the Phase 2 HELIOS study in Wolfram syndrome will continue as planned, with preliminary data from the HELIOS study expected in the second quarter of 2024.
Amylyx hosted an investor conference call today to discuss the PHOENIX trial results. The company has stated that the information provided is based on a press release statement.
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