AMX0035 lowers risk for adverse events, death in patients with ALS

AMX0035, an investigational neuroprotective therapy, significantly lowered the long-term risk for death or need for permanent assisted ventilation, as well as risk for first hospitalization, according to a press release.

Sabrina Paganoni

“These results show a consistent effect with AMX0035 across endpoints of survival, hospitalization and permanent ventilation. I am enthusiastic about these results [that] continue to support AMX0035 as a potential therapeutic for ALS,” Sabrina Paganoni, MD, PhD, principal investigator of the CENTAUR study, investigator at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and assistant professor at Harvard Medical School and Spaulding Rehabilitation Hospital, said in the release.

CENTAUR, the study investigating the neuroprotective therapy, involved 137 patients with ALS over a 24-week placebo-controlled period. Researchers evaluated the safety and efficacy of AMX0035 using the ALS Functional Rating Scale-Revised.

The treatment demonstrated a statistically significant (P < .05) effect on ALSFRS-R. The researchers observed a 44% decreased risk for death during follow-up in those on the therapy compared to the placebo group, per the results of an overall survival analysis. Moreover, they observed similar rates of adverse events in all participants and found that the therapy succeeded in slowing disease progression.

“The results from CENTAUR, which showed a significantly lower risk of death or permanent assisted ventilation and first hospitalization, are an important step forward in a therapeutic area with a significant unmet need,” Justin Klee, co-CEO and co-founder of AMX0035 developer Amylyx Pharmaceuticals, said in the release.

The researchers presented the findings from CENTAUR at the 2021 Muscular Dystrophy Association Virtual Clinical & Scientific Conference, according to the release, with plans to publish the results at a later date. Additionally, Amylyx plans to introduce a new drug submission for the treatment of ALS to Health Canada in the first half of 2021.

Reference:

Amylyx. Amylyx Pharmaceuticals to present new long-term data from the pivotal CENTAUR trial in ALS at the Muscular Dystrophy Association (MDA) virtual clinical & scientific conference. Available at: https://www.amylyx.com/2021/03/18/amylyx-pharmaceuticals-to-present-new-long-term-data-from-the-pivotal-centaur-trial-in-als-at-the-muscular-dystrophy-association-mda-virtual-clinical-scientific-conference/. Accessed March 22, 2021.