Sabrina Paganoni, MD, PhD: A Combination Approach to ALS


The investigator at the Healey & AMG Center for ALS detailed the combination of AMX0035 and other agents and how ALSFRS-R scores translate to daily life.

“We know that there are many different primary insults that cause ALS, but we also know that many of them converge on common mechanisms, including the activation of mitochondrial pathways and endoplasmic reticulum-dependent pathways.”

CENTAUR trial lead investigator Sabrina Paganoni, MD, PhD, told NeurologyLive in a recent interview that the results of the phase 2/3 assessment of AMX0035 were a milestone moment in the treatment of patients with amyotrophic lateral sclerosis (ALS). The agent, developed by Amylyx Pharmaceuticals, is a combination of sodium phenylbutyrate–taurursodiol.

Notably, 77% of patients assessed in the trial were already on an approved ALS therapy, either riluzole (n = 96), edaravone (Radicava, MT Pharma; n = 46), or both (n = 38), during and/or before trial entry. When Paganoni and colleagues conducted sensitivity analyses to correct for this, their findings suggested that AMX0035 had a clinical benefit independent of these other treatments with a well-tolerated safety profile. This, she explained, implies that the agent can be successfully combined with these agents to offer patients additional benefit.

Paganoni detailed the combination that comprises AMX0035 and the pathways being targeted, as well as the changes observed in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) scores and how those changes translate to improvements in patients’ daily lives.

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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