Friday, Amylyx Pharmaceuticals Inc ( AMLX ) announced topline results from the PHOENIX Phase 3 trial of AMX0035, also known as Relyvrio in the U.S. and Albrioza in Canada for amyotrophic lateral sclerosis (ALS). 

PHOENIX did not meet its primary endpoint of reaching statistical significance (p=0.667) as measured by change from baseline in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) total score at Week 48, nor was there statistical significance seen in secondary endpoints. 

ALSFRS-R is a disease-specific severity score that reflects motor impairment and functional deterioration in ALS patients.

Amylyx will continue to engage with regulatory authorities to discuss the results from PHOENIX within the next eight weeks and make informed decisions. 

The company intends to share plans for Relyvrio/Albrioza in ALS, which may include voluntarily withdrawing the drug from the market.

Amylyx has voluntarily decided to pause the medication promotion at this time.

No significant difference was observed in the subset of participants who met the CENTAUR trial criteria. 

No significant differences were observed across secondary endpoints.

AMX0035 was well-tolerated in the 664-subject PHOENIX study. There were no new safety signals.

European participants who completed the 48-week randomized phase had the option to enroll in an open-label extension of the trial of up to two years, which remains ongoing.

The Phase 3 ORION study of AMX0035 in Progressive Supranuclear Palsy (PSP) remains ongoing. 

PSP is a rare neurological condition that can cause problems with balance, movement, vision, speech, and swallowing.

The first participant was dosed in December 2023, and the company is planning for an interim analysis. Topline results continue to be anticipated in 2025 or 2026.

Data from the ongoing 12-participant Phase 2 HELIOS clinical study demonstrate evidence of clinical activity of AMX0035 in Wolfram syndrome, a rare neurodegenerative disorder characterized by juvenile-onset diabetes mellitus.

The study is fully recruited, and the company plans to present preliminary data in the second quarter of 2024.

Price Action: AMLX shares are down 83.30% at $3.17 on the last check Friday.

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