Amylyx announces end of enrollment for phase 3 trial of ALS therapeutic
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Amylyx Pharmaceuticals Inc. announced completion of enrollment for a global, phase 3 clinical trial of AMX0035, an oral, fixed-dose medication for ALS.
According to a company release, the PHOENIX study is a 48-week, randomized, placebo-controlled trial conducted at more than 65 sites throughout Europe and the United States and will include 664 participants with ALS.
The primary efficacy outcome of the study, Amylyx said in the release, will be joint assessment of ALS Functional Rating Scale-Revised total score progression over 48 weeks, adjusted for mortality. Participants have been randomized on a 3:2 basis to receive either AMX0035 (a combination of sodium phenylbutyrate and taurursodiol) or placebo during the study. Researchers will also assess safety and tolerability.
Participants in Europe who finish the 48-week trial may be enrolled in an open-label extension phase, during which all participants will receive AMX0035, which will be further evaluated for safety and efficacy, per the release.
“We are grateful for the people living with ALS and their families who are participating in PHOENIX and the dedication of the study investigators,” Joshua Cohen and Justin Klee, co-CEOs at Amylyx, stated in the release. “We remain committed to continuing research and exploring the full potential of AMX0035 as part of our mission to one day end the suffering caused by ALS and other neurodegenerative diseases.”
The PHOENIX trial design was informed by results of the phase 2 CENTAUR study, in which treatment with AMX0035 demonstrated a statistically significant benefit in function and an observed benefit on survival in post-hoc analysis, the release stated.
“We are pleased to share this milestone from the PHOENIX trial, a collaborative effort to advance our scientific understanding of ALS and build upon the positive data from our phase 2 CENTAUR trial,” Sabrina Paganoni, MD, PhD, principal investigator of the CENTAUR study and member of the executive committee of the Northeast ALS Consortium, said in the release.
Amylyx expects to report topline results from PHOENIX in 2024.