Steven Arnold, MD: Assessing AMX0035 in Alzheimer Disease


The managing director of the Interdisciplinary Brain Center at Massachusetts General Hospital discussed the potential of AMX0035 for the treatment of Alzheimer disease and other neurological diseases.

“It's shown efficacy for things related to Parkinson's, Huntington's, ALS, and of course, Alzheimer disease...The other interesting thing about it is that while each of the components has shown some benefit in these models or in clinical populations, the preclinical model suggested that the combination works synergistically to enhance neuronal survival and neuronal functioning.”

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 and to improve neuronal survival. He offered insight into the outcome measures of the trial and his hopes for the ongoing data analysis.

Arnold also discussed the effect the pandemic has had on the study, which resulted in a number of site closures, though the majority of these delays were mitigated. The trial assessed 95 patients, almost double the original enrollment goal of 50. The topline results 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.

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