AMX0035 in the ALS Treatment Paradigm: Sabrina Paganoni, MD, PhD


The investigator at the Healey & AMG Center for ALS at Massachusetts General Hospital spoke to the data backing the investigational AMX0035 in the treatment of ALS. [WATCH TIME: 3 minutes]

WATCH TIME: 3 minutes

“In the [CENTAUR] clinical trial, we learned that AMX0035 is associated with longer retention of physical function and longer survival, and we also learned that the drug is overall safe and well-tolerated.”

Data from the phase 2/3 CENTAUR trial (NCT03127514)—which included a study population of 135 patients with amyotrophic lateral sclerosis (ALS)—suggest that AMX0035, an investigational therapy from Amylyx Pharmaceuticals, could offer an effective option for patients. The data published, in late 2020, showed that those treated with AMX0035 (n = 87) reported an average Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised score of 2.32 points higher than the placebo group (n = 48; = .03) in the primary prespecified analysis, after 24 weeks.1

Now, Amylyx Pharmaceuticals has announced that it is moving forward with a submission to the FDA of the investigational combination agent. This decision was made based on a recent discussion between the agency and company, with Joshua Cohen, co-CEO, chairman, and cofounder, Amylyx, saying in a statement that they are “thrilled to move toward the US submission” and that they “look forward to continuing to work with the FDA.”2

Sabrina Paganoni, MD, PhD, investigator, Healey & AMG Center for ALS, Massachusetts General, and member, Executive Committee of Northeast ALS Consortium, was an investigator in the CENTAUR trial and has been involved in its development. NeurologyLive inquired with Paganoni to find out more about AMX0035, what the clinical community should know ahead of its submission to the FDA, and what the data show to this point.

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
2. Amylyx Pharmaceuticals Announces Plan to Submit New Drug Application (NDA) for AMX0035 for the Treatment of ALS in the Coming Months. News release. Amylyx Pharmaceuticals. September 15, 2021. Accessed September 28, 2021.
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