AMX0035’s Potential and Mechanism in ALS: Sabrina Paganoni, MD, PhD


With AMX0035 expected to be submitted to the FDA for review in the coming months, the investigator at the Healey & AMG Center for ALS at Massachusetts General Hospital chimed in to share her thoughts on its potential. [WATCH TIME: 3 minutes]

WATCH TIME: 2 minutes, 52 seconds

“If approved, I really think that AMX0035 will become widely used for people with ALS. We know that the drug is effective, we know that it can be combined with standard of care medications, and so it can add value on top of the other drugs that are currently in use for ALS.”

In September 2021, Amylyx Pharmaceuticals announced that its investigational combination agent for the treatment of amyotrophic lateral sclerosis (ALS), AMX0035, would be submitted to the FDA for review in the coming months. This decision was made based on a recent discussion between the FDA and Amylyx, according to the company. Joshua Cohen, co-CEO, chairman, and cofounder, Amylyx, said in a statement that the company was “thrilled to move toward the US submission” and that they “look forward to continuing to work with the FDA.”1

The submission will likely be supported by data from the phase 2/3 CENTAUR trial (NCT03127514) that included a study population of 135 patients with ALS. Participants were randomly assigned in a 2:1 fashion to active treatment or placebo, and data published in late 2020 suggested 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.2

Sabrina Paganoni, MD, PhD, investigator, Healey & AMG Center for ALS, Massachusetts General Hospital, and member, Executive Committee of Northeast ALS Consortium, was an investigator in the CENTAUR trial and has gained important experience with AMX0035 through its clinical development. As such, NeurologyLive inquired with her to find out more about the agent’s potential in ALS pending the FDA’s decision. She spoke to the gaps it would help fill in clinical care and discussed the details that neuromuscular specialists should know about the sodium phenylbutyrate and taurursodiol combination.

1. 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 15, 2021.
2. 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
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