Amylyx Pharmaceuticals said that the last enrolled patient in the CENTAUR clinical trial, which is evaluating AMX0035 for amyotrophic lateral sclerosis (ALS), has finished the 24 weeks of study treatment.
The CENTAUR clinical study is a randomized, double-blind, placebo-controlled phase 2 clinical trial. The primary goals of the mid-stage clinical trial are to assess the safety and tolerability of AMX0035 and its impact on disease progression as gauged by the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) through the 24-week study duration.
The CENTAUR clinical trial will also assess AMX0035’s impact on isometric strength as measured by respiratory function, ATLIS, and exploratory biomarkers of neuronal death. According to Amylyx Pharmaceuticals, there will also be an open-label extension to the mid-stage clinical trial that will allow the enrolled patients to continue to get AMX0035.
CENTAUR clinical trial Principal Investigator Dr. Sabrina Paganoni said: “This trial has been an important partnership between industry and academia, and we look forward to analyzing the results gathered in this study.
“Thank you to the participants and site study teams who have made this work possible.”
AMX0035 combines two small molecules – sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). According to Amylyx Pharmaceuticals, both the compounds had shown strong efficacy in various cellular and animal models of ALS.
When tested individually across ALS clinical trials, PB and TUDCA have both demonstrated safety, tolerability, and preliminary signs of efficacy. In preclinical studies, Amylyx Pharmaceuticals showed a synergistic effect between the two compounds in the prevention of nerve cell death and degeneration.
Patrick Yeramian – chief medical officer of Amylyx Pharmaceuticals said: “We’re thankful to everyone involved, and we are working hard to bring topline results to the community as quickly as possible over the coming months.”
In 2017, AMX0035 secured orphan drug status from the US Food and Drug Administration (FDA) as a potential drug for ALS.