8 April 2019 - - US-based clinical-stage pharmaceutical company Amylyx Pharmaceuticals, Inc., in collaboration with the Alzheimer's Association, the Alzheimer's Drug Discovery Foundation and the Cure Alzheimer's Fund, has dosed its first patients in a recently expanded Phase 2 clinical trial (PEGASUS) to assess AMX0035 in individuals with Alzheimer's disease, the company said.

AMX0035 is Amylyx's proprietary two-drug combination therapy in development to prevent nerve cell death and degeneration.

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.

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 program, a joint research funding initiative created by the Alzheimer's Association and the ADDF to support clinical trials combining multiple treatment approaches.

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.

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.

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.

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 USD 88m 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 % 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.

Amylyx Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company developing a novel therapeutic for Amyotrophic Lateral Sclerosis, Alzheimer's disease and other neurodegenerative diseases.

The company's therapeutic, AMX0035, targets the neuroinflammation and nerve cell death that characterise these diseases.

AMX0035 is a proprietary combination of existing compounds that act synergistically to prevent cell death and neurotoxic inflammation in multiple preclinical models.