Phase 3 PHOENIX Study of AMX0035: Sabrina Paganoni, MD, PhD

“A feature of PHOENIX that I am particularly excited about is that we will leverage telemedicine to facilitate access and participation. We learned a lot about telemedicine during the pandemic, so I'm really delighted that we can apply these learnings to make the trial more accessible.”

The investigational agent AMX0035 will be further evaluated for the treatment of amyotrophic lateral sclerosis (ALS) in the upcoming phase 3 study A35-004 PHOENIX. Amylyx Pharmaceuticals, the agent’s developer, announced the trial design of the study evaluating its agent at the European Network to Cure ALS (ENCALS) Meeting 2021, May 12-14.

The trial will be a 48-week, randomized, placebo-controlled study that will assess ALS Functional Rating Scale–Revised (ALSFRS-R) over 48 weeks and survival as primary outcomes. Secondary efficacy outcomes will include change in slow vital capacity (SVC) 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.

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 NEALS, to learn more about the PHOENIX study. She discussed how the results from the CENTAUR study paved the way for the new study.

REFERENCE

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. 
https://www.businesswire.com/news/home/20210512005352/en/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