Combination Therapy in ALS: Sabrina Paganoni, MD, PhD


The investigator at the Healey & AMG Center for ALS at Massachusetts General Hospital discussed how adding AMX0035 to a therapeutic regimen might improve treatment of ALS.

“AMX0035 is thought to act on ALS by using different mechanisms compared to riluzole and edaravone. So, each of them has different profiles in terms of mechanism of action. Again, this tells us that we might need to attack ALS from different angles to really be able to make an impact.”

Amylyx Pharmaceuticals has announced the trial design for the upcoming phase 3 study A35-004 PHOENIX evaluating the investigational agent AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS) at the European Network to Cure ALS (ENCALS) Meeting 2021, May 12-14.

The 48-week, randomized, placebo-controlled trial will assess ALS Functional Rating Scale–Revised over 48 weeks and survival as primary outcomes. It will also assess change in slow vital capacity measured both at home with a self-administered spirometer to support virtual data collection and at clinic sites, serial assessments of patient-reported outcomes, ventilation-free survival rates, and others as secondary efficacy outcomes.

NeurologyLive spoke with principal investigator of the previous study, CENTAUR (NCT03127514), and investigator of the PHOENIX study, Sabrina Paganoni, MD, PhD, investigator, Healey & AMG Center for ALS, Mass General and member, Executive Committee of Northeast ALS Consortium, to learn more about the potential of AMX0035 to improve patient care in ALS. She stressed that combination therapy using different mechanisms to target ALS pathologies is key to successful treatment.

1. Amylyx Pharmaceuticals presents trial design of global phase 3 PHOENIX study of AMX0035 in ALS at the European Network to Cure ALS (ENCALS) meeting 2021. News release. Amylyx Pharmaceuticals. Published online May 12, 2021.
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