Sabrina Paganoni, MD, PhD: Takeaways From the CENTAUR Trial of AMX0035

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The investigator at the Healey & AMG Center for ALS spoke to the newly released findings of the phase 2/3 trial of the investigational combination ALS agent AMX0035.

“Patients tell us that their number one desire is to be able to maintain physical function for as long as possible. For example, they want to continue to be able to walk or to continue to be able to negotiate stairs. They want to be able to use their hands so they can cut their food, or type emails, or use the phone. That’s exactly what we measured in the trial.”

The full dataset from the phase 2/3 CENTAUR trial (NCT03127514) of the investigational combination therapy AMX0035 suggests that the sodium phenylbutyrate–taurursodiol agent slows functional decline in patients with amyotrophic lateral sclerosis (ALS) compared with placebo. Analysis of the change from baseline revealed a 2.92-point higher mean Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score for the AMX0035 group (P = .01).

Lead investigator Sabrina Paganoni, MD, PhD, investigator, Healey & AMG Center for ALS, Massachusetts General Hospital, told NeurologyLive in this interview that these findings for the Amylyx Pharmaceuticals agent mark a milestone moment for the ALS community. Paganoni offered insight into the findings of the trial, and what sensitivity analyses suggest about its possible role in the landscape of treatment.

She shared her perspective on the level of cooperation that ultimately resulted in the CENTAUR, noting that members of the ALS community came from all corners to fund and design the study. CENTAUR received the ALS ACT grant and was funded in part by the ALS Ice Bucket Challenge, in addition to support from a number of advocacy and clinician organizations.