Steven Arnold, MD: Phase 2 PEGASUS Trial in Alzheimer Disease


The managing director of the Interdisciplinary Brain Center at Massachusetts General Hospital discussed the trial design and outcomes of the PEGASUS trial of AMX0035.

“Some of the interesting things about the [PEGASUS] trial are [that] it does have a broad eligibility for people and we use a novel approach for a composite global outcome.”

In recent weeks, the final patient visit was completed for the phase 2 PEGASUS trial (NCT03533257), which is evaluating the safety and biological activity of AMX0035 administration in people with Alzheimer's disease.1 The investigational agent, a product of Amylyx Pharmaceuticals, has also been assessed in amyotrophic lateral sclerosis (ALS), with recent success shown in the phase 2/3 CENTAUR trial (NCT03127514).2

This assessment in Alzheimer had been marred slightly due to the ongoing pandemic, which resulted in a number of site closures, but the majority of these delays were mitigated, and the trial enrolled 96 patients, 46 more than originally planned but 4 shy of the adjusted enrollment goal. The topline results are expected to be released sometime in the first half of 2021.

To find out more about the trial design, what outcomes were assessed, and how AMX0035 might perform in Alzheimer, 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.

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.
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