Approval of AMX0035 and the Future of Clinical Trials for ALS: Lawrence Steinman, MD

WATCH TIME: 4 minutes

“It's not a disease that comes on like a stroke. If you have sensitive devices, or some knowledge, you can begin to tell years or maybe decades before somebody becomes clinically diagnosed with ALS.”

CENTAUR (NCT03127514), which was a phase 2, randomized, double-blind trial, had data that supported AMX0035’s new drug application submitted in November 2021. Published in the New England Journal of Medicine, the data included 137 patients with amyotrophic lateral sclerosis (ALS) who were assigned randomly 2:1 to AMX0035 (3 g sodium phenylbutyrate and 1 g taurursodiol) or placebo. Each patient had treatment administered once per day over the course of 3 weeks, which was then increased to twice per day over a treatment period of 24-weeks.¹

On September 29, 2022, the FDA approved AMX0035 a coformulation of sodium phenylbutyrate and taurursodiol, as a treatment for patients with ALS based on findings of the CENTAUR trial. It became the third approved treatment to help slow the progression and mortality of the disease.

In a recent conversation with NeurologyLive^®, Lawrence Steinman, MD, Zimmermann Professor of Neurology and Neurological Sciences and Pediatrics, Stanford University, discussed the previous studies that have been done on ALS before the approval of the AMX0035. In addition, he mentioned that there might be a way to prolong life longer if there was research on how to intervene earlier before the diagnosis of ALS. He also mentioned that the only way to find promising data is by performing clinical trials which both the pharmaceutical industry and academia are interested in conducting.

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