Combination Therapy Improved Amyotrophic Lateral Sclerosis Outcomes in Phase 2 Clinical Trial

Results of the CENTAUR clinical trial (NCT03127514) reveal that a combination therapy of phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA) (AMX0035; Amylyx Pharmaceuticals) lowered risk of death, permanent assisted ventilation (PAV), and first hospitalization in people with amyotrophic lateral sclerosis (ALS) (hazard ratio [HR], 0.66; P<.05). These data were presented at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference

In the CENTAUR trial, individuals age 18 to 80 who had onset of ALS within 18 months of enrollment were randomly assigned to have treatment with PB/TUDCA or placebo. Participants were able to continue taking edaravone or riluzole. Disease progression was measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) on which statistically significant slowing was seen for those who received PB/TUDCA (P<0.05).  

“We are strongly encouraged by the results from the CENTAUR trial,” said Sabrina Paganoni, MD, PhD, investigator at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and Assistant Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital. “These results show a consistent effect with AMX0035 across endpoints of survival, hospitalization, and permanent ventilation. I am enthusiastic about these results which continue to support AMX0035 as a potential therapeutic for ALS.”