Posted October 23, 2020

(Distributed by Amylyx Pharmaceuticals on June 15, 2020)

Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, today announced it has completed enrollment in the Phase II PEGASUS trial assessing AMX0035 in individuals with Alzheimer’s disease. Amylyx completed enrollment, dosing 96 of the previously planned 100 participants.

The PEGASUS trial will provide data on the safety and tolerability profile of AMX0035 in people living with Alzheimer’s disease, biological activity on biomarkers related to disease processes and preliminary information on cognitive and functional effects of AMX0035. Trial collaborators include the Alzheimer’s Association, the Alzheimer’s Drug Discovery Foundation (ADDF) and Cure Alzheimer’s Fund.

In April 2019, Amylyx doubled the PEGASUS trial’s planned enrollment to approximately 100 participants with additional funding support from Cure’s Alzheimer’s Fund. The trial was first supported in 2017 through the Alzheimer’s Combination Therapy Opportunities (ACTO) program, a joint research funding initiative created by the Alzheimer’s Association and the ADDF.

“Alzheimer’s disease is a complex, progressive brain disorder with no disease-modifying treatments that impact the neurodegenerative process,” said Steven E. Arnold, M.D., Translational Neurology Head of the Interdisciplinary Brain Center at Massachusetts General Hospital and Harvard Medical School and the PEGASUS trial’s principal investigator. “Due to COVID-19, we must pay close attention to the safety of older adults whose associated health conditions and age may pose a risk. We are pressing ahead safely as we expect the trial will generate meaningful insights about the ability of AMX0035 to target mechanisms involved in the progression of Alzheimer’s.”

Patient safety is Amylyx’ priority. In an effort to mitigate possible delays due to the coronavirus pandemic, multiple steps have been taken in the PEGASUS trial to reduce patient exposure. Amylyx has worked with clinicians to switch to virtual visits and expand clinic visitation windows when possible. Amylyx also capped trial enrollment early at 96 to ensure patient safety and to responsibly respond to clinical trial site closures. Topline data remain targeted for early 2021.

“Alzheimer’s disease remains one of the most critically unmet needs in healthcare and we desperately need new treatments for patients,” said Rudolph Tanzi, Ph.D., Kennedy Professor of Neurology, Massachusetts General Hospital and Harvard Medical School, chair of the Amylyx Scientific Advisory Board and chair of the Cure Alzheimer’s Fund Research Leadership Group. “AMX0035 recently showed benefit in patients with ALS, and the same mechanism of neuroprotection may also be helpful in Alzheimer’s disease. We are very fortunate that the PEGASUS trial has continued, despite COVID-19, and eagerly await data in Q1 2021.”

Last year, Amylyx announced that AMX0035 met its clinical endpoint in the CENTAUR study in people with ALS. In the study, participants taking AMX0035 had a statistically significant slowing of ALS disease progression as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) compared to placebo (p<0.05), the primary efficacy outcome of the trial. Detailed results from this study will be published shortly.

“We deeply appreciate the participation from patients and their families and the support we have received, particularly as we address new challenges during this global crisis,” said Kent Leslie, Chief Scientific Officer at Amylyx. “We remain committed to those battling Alzheimer’s disease and to the research that will propel us to bring timely answers to the community later this year. Thank you to the patients, investigators, site teams, Alzheimer’s Association, Alzheimer’s Drug Discovery Foundation and Cure Alzheimer’s Fund whose rigor and dedication make this trial possible.”


PEGASUS (NCT03533257) is a 3:2 randomized, double-blind, multi-center, placebo-controlled study evaluating the safety, tolerability and activity of AMX0035 in patients with late mild cognitive impairment or early dementia due to Alzheimer’s disease over 24 weeks. The biomarker-focused trial design will assist in understanding the effects of AMX0035 and its potential in treating Alzheimer’s disease.

About AMX0035

AMX0035 is a first-in-class investigational therapy designed to reduce neuronal death and dysfunction. AMX0035 targets mitochondrial and endoplasmic reticulum-dependent neuronal degeneration pathways.

About Amylyx Pharmaceuticals

Amylyx Pharmaceuticals, Inc. is a pharmaceutical company working on developing a novel therapeutic for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases. For more information, visit and follow us on LinkedIn and Twitter.

Alzheimer’s Association

The Alzheimer’s Association is the leading voluntary health organization in Alzheimer’s care, support and research. It is the largest nonprofit funder of Alzheimer’s research. The Association’s mission is to eliminate Alzheimer’s disease through the advancement of research; to provide and enhance care and support for all affected; and to reduce the risk of dementia through the promotion of brain health. Its vision is a world without Alzheimer’s. Visit or call 800.272.3900.

Alzheimer’s Drug Discovery Foundation

Founded in 1998 by Leonard A. Lauder and Ronald S. Lauder, the Alzheimer’s Drug Discovery Foundation is the only philanthropy solely focused on accelerating the development of drugs to prevent and treat Alzheimer’s disease. Its venture philanthropy approach and scientific expertise allows the ADDF to support the most promising ideas around the world. And 100% of every donation funds drug research programs. To learn more, visit

Cure Alzheimer’s Fund

Cure Alzheimer’s Fund is a non-profit dedicated to funding the most promising research to prevent, slow or reverse Alzheimer’s disease. Since its founding in 2004, Cure Alzheimer’s Fund has contributed over $110 million to research, and its funded initiatives have been responsible for several key breakthroughs – including the groundbreaking Alzheimer’s in a Dish. Cure Alzheimer’s Fund has received a 4 star rating for 9 consecutive years regarding its overall financial health from Charity Navigator. With 100 percent of funds raised going directly to research, Cure Alzheimer’s Fund has been able to support some of the best scientific minds in the field of Alzheimer’s research. For more information, please visit