Steven Arnold, MD: Assessing AMX0035 Efficacy in Alzheimer Disease


The managing director of the Interdisciplinary Brain Center at Massachusetts General Hospital discussed the process of determining the best situations for use of AMX0035.

“The combination of biomarkers and clinical outcomes into a composite measure really is important and I think that [it]is an interesting and novel approach that can inform future trials.”

Amylyx Pharmaceuticals recently completed the final patient visit in its phase 2 PEGASUS trial (NCT03533257) of AMX0035. The trial aimed to evaluate the safety and biological activity of AMX0035 administration in people with Alzheimer's disease.1 AMX0035 has also been assessed in amyotrophic lateral sclerosis (ALS), with recent success shown in the phase 2/3 CENTAUR trial (NCT03127514).2

NeurologyLive spoke with study investigator Steven Arnold, MD, professor of neurology, Harvard Medical School, and managing director and translational neurology head, Interdisciplinary Brain Center, Massachusetts General Hospital, to learn about the potential of AMX0035 for the treatment of Alzheimer disease and other diseases, as well as to improve neuronal survival. 

Arnold offered insight into how the investigators started broad in terms of subject eligibility, and his hopes to identify situations in which AMX0035 is more or less effective so that future studies can be more specific in evaluating that efficacy. He also discussed the difficulties of assessing Alzheimer disease and cognition.

Topline results from PEGASUS are expected to be released sometime in the first half of 2021.

1. Amylyx Pharmaceuticals Announces Last Patient Completes Final Study Visit in PEGASUS Trial of AMX0035 in Alzheimer’s Disease. News release. Amylyx Pharmaceuticals. November 9, 2020. Accessed November 24, 2020.
2.Paganoni S, Hendrix S, Dickson SP, et al. Long‐Term Survival of Participants in the CENTAUR Trial of Sodium Phenylbutyrate‐Taurursodiol in ALS. Muscle Nerve. Published online October 16, 2020. doi: 10.1002/mus.27091.

Related Videos
Mony de Leon, EdD
Ray Dorsey, MD
Krystyn J. Van Vliet, PhD & Anna Jagielska, PhD, MSc
Hai Sun, MD, PhD
Svetlana Blitshteyn, MD, FAAN, director and founder of Dysautonomia Clinic
Shiv Saidha, MBBCh
Marjan Gharagozloo, PhD
© 2024 MJH Life Sciences

All rights reserved.