Reactions to FDA Advisory Panel Decision on AMX0035: Justin Klee; Josh Cohen, BSc

WATCH TIME: 3 minutes

"I thought the presentation did a great job of addressing those concerns, and also highlighting the strength of the data. The data, as Josh said, were first published in the New England Journal of Medicine, then in Nerve & Muscle, showing a benefit first on the ALS Functional Rating Scale—the gold standard in the field of ALS—and then on overall survival in muscle and nerve, which is critical in any disease, but particularly in a rapidly fatal disease like ALS."

In March 2022, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee (AdCom) voted that the current data on AMX0035 (Amylyx Pharmaceuticals), an investigational fixed-dose coformulation of sodium phenylbutyrate-taurursodiol for the treatment of amyotrophic lateral sclerosis (ALS), did not demonstrate sufficient enough efficacy, which the FDA would require for approval. Many in the community await the FDA’s final decision, expected to come at the end of June, as to whether AMX0035 will become just the third approved treatment for the fatal neuromuscular disease.¹

The committee voted 6-4 (6 no; 4 yes; 0 abstain) that the data from the phase 2/3 CENTAUR trial (NCT03127514)—the main study supporting the new drug application submission—and open-label extension, did not adequately establish the medication as an effective treatment for ALS. Several of the AdCom members who voted against the drug raised concerns over the trial’s conduct and robustness of the data, while others felt that more questions would be answered with the ongoing phase 3 PHOENIX study (NCT05021536).

At the 2022 American Academy of Neurology (AAN) Annual Meeting, April 2-7, in Seattle, Washington, Amylyx presented new safety data from CENTAUR, as well as from PEGASUS (NCT03533257), another trial evaluating the agent in patients with Alzheimer disease. To understand whether these concerns were of worry, NeurologyLive^® sat down with Justin Klee, BSc, and Josh Cohen, BSc, cofounders and co-CEOs of Amylyx. They shared their general reactions to the meeting, stressing that the evidence speaks for itself. Additionally, the duo discussed why the opinion of Dean Follman, PhD, one of the AdCom members who supported the decision, holds immense weight.

REFERENCES
1. FDA. Peripheral and Central Nervous System Drugs Advisory Committee Meeting. FDA. March 30, 2022. Accessed May 18, 2022.