AMX0035’s Safety as a Treatment for Patients with ALS: Lawrence Steinman, MD

WATCH TIME: 5 minutes

“The good news here is that it's a reasonably safe drug, the safety profile is pretty good. You're not exposing people to undue risk. I would have to say that if I were in the shoes of somebody with that disease, I'd want to take it.”

The FDA recently approved AMX0035 (Relyvrio; Amylyx Pharmaceuticals), a coformulation of sodium phenylbutyrate and taurursodiol, on September 29, 2022, as a treatment for patients with amyotrophic lateral sclerosis (ALS) to help slow the progression and mortality of the disease. It became the third approved treatment to show such ability, and was greenlit based on findings from the phase 2 CENTAUR trial (NCT03127514).

Published in the New England Journal of Medicine, CENTAUR was a randomized, double-blind trial that included 137 patients with ALS who were randomly assigned 2:1 to AMX0035 (3 g sodium phenylbutyrate and 1 g taurursodiol) or placebo. Patients were administered the treatment once per day for 3 weeks, and increased to double the dosage per day during the 24-week period.¹

In a recent conversation with NeurologyLive^®, Lawrence Steinman, MD, Zimmermann Professor of Neurology and Neurological Sciences and Pediatrics, Stanford University, discussed the satisfactory outcome of the AMX0035 approval, and the work done with the ALS patient community. He also spoke on future research regarding the dosage of the therapy, as well as a program for those who do not have insurance to access the drug.

REFERENCES
1. Paganoni S, Macklin EA, Hendrix S, et al. Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis. N Engl J Med. 2020;383:919-930. doi:10.1056/NEJMoa1916945