1. Oct 19, 2020

Investigational treatment prolongs survival in ALS

The experimental treatment AMX003 prolonged survival in patients with amyotrophic lateral sclerosis (ALS), according to results from the CENTAUR trial.

In the trial, patients with ALS were randomized 2:1 to receive AMX003, which is combination of sodium phenylbutyrate and taurursodiol, or placebo. Patients who completed a 24-week randomized phase were eligible to receive AMX003 in the open‐label extension. At 35‐month maximum follow‐up post‐randomization, an all‐cause mortality analysis included all randomized patients.

Of the 135 of the 137 participants that vital status was obtained for, the median overall survival was 25 months and 18.5 months for patients randomized to receive AMX003 and placebo, respectively.

“These findings are an important step forward because, in this trial, early treatment with AMX0035 was associated with longer survival in people with ALS,” said the study's leader Sabrina Paganoni, MD, PhD, investigator at the Healey & AMG Center for ALS and assistant professor of Physical Medicine and Rehabilitation at Harvard Medical School and Spaulding Rehabilitation Hospital in a press release. “These results provide substantial evidence supporting the role of AMX0035 for the treatment of ALS. Next steps will depend on ongoing discussions with regulatory agencies.”

Paganoni S, Hendrix S, Dickson SP, et al. Long‐term survival of participants in the CENTAUR Trial of sodium phenylbutyrate‐taurursodiol in ALS. Muscle Nerve. 2020; DOI: 10.1002/mus.27091.