CAMBRIDGE, MA (BUSINESS WIRE) — Amylyx Pharmaceuticals, Inc., in collaboration with the Alzheimer’s Association, the Alzheimer’s Drug Discovery Foundation (ADDF) and the Cure Alzheimer’s Fund, today announced the dosing of its first patients in a recently expanded Phase 2 clinical trial (PEGASUS) to assess AMX0035 in individuals with Alzheimer’s disease. AMX0035 is Amylyx’s proprietary two-drug combination therapy in development to prevent nerve cell death and degeneration.

“The urgent need for a new approach to Alzheimer’s is clearer now than ever. As this is one of the first major combination therapy clinical trials for Alzheimer’s Disease, we’re optimistic about AMX0035’s potential to slow disease progression in individuals through its novel mechanisms of action,” said Steven E. Arnold, M.D., Translational Neurology Head of the Interdisciplinary Brain Center at Massachusetts General Hospital and Harvard Medical School and the study’s principal investigator.

Alzheimer’s is a complex disease that likely has multiple, interrelated causes. A growing number of experts believe combination therapy—with multiple therapeutics—will be required to effectively treat it.

“Although targeting the classic pathologies of Alzheimer’s disease, beta-amyloid and tau, has proven challenging in clinical trials, this research has awarded us with a wealth of knowledge in our understanding of the disease,” said Rudolph Tanzi, Ph.D., of Massachusetts General Hospital, chair of the Cure Alzheimer’s Fund Research Leadership Group and chair of the Amylyx SAB. “Cure Alzheimer’s Fund is proud to take initiative and provide additional funding to expand Amylyx’s trial to support the development of novel approaches that patients need,” Dr. Tanzi added.

With additional funding from the Cure’s Alzheimer’s Fund, the PEGASUS trial has doubled its planned enrollment to 100 participants, with 20 patients enrolled and dosed to date. 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 to support clinical trials combining multiple treatment approaches.

“There have been trials targeting amyloid, and there have been trials targeting tau, but this is the first trial to target two novel mechanisms in tandem,” said Howard Fillit, M.D., the ADDF’s founding executive director and chief science officer. “The ADDF was one of the earliest supporters of innovative targets for Alzheimer’s, and we believe combination therapies are a critical next step in finding effective treatments for the disease.”

“The rationale for combination therapies has already been demonstrated in diseases like HIV/AIDS, cancer and heart disease. We believe this type of approach will also work for Alzheimer’s and other dementias and are encouraged by the launch of this combination therapy trial,” said Maria C. Carrillo, Ph.D., Alzheimer’s Association’s chief science officer. “The Alzheimer’s Association is honored to be at the forefront of the mission to keep expanding the research pipeline for therapies that attack this complex disease from a variety of angles.”

PEGASUS 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 demonstrating the effects of AMX0035 and its potential in treating Alzheimer’s disease. Study results are expected in 2020.

“We believe AMX0035 has potential across multiple neurodegenerative diseases. We’re moving quickly to determine the synergistic efficacy of these two therapies and deeply value the support from the Alzheimer’s Drug Discovery Foundation (ADDF), the Alzheimer’s Association and the Cure Alzheimer’s Fund to expand this unique trial,” said Kent Leslie, Amylyx’s chief scientific officer.

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’s 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 $88 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 7 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

About Amylyx Pharmaceuticals

Amylyx Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company developing a novel therapeutic for Amyotrophic Lateral Sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases. The company’s therapeutic, AMX0035, targets the neuroinflammation and nerve cell death that characterize these diseases. AMX0035 is a proprietary combination of existing compounds that act synergistically to prevent cell death and neurotoxic inflammation in multiple preclinical models. This press release and further information about Amylyx can be found at:


Merissa Muller
Account Executive
Finn Partners
(212) 715-1699