The ALS Association and ALS Finding a Cure Announce Joint Funding Support for Amylyx Clinical Trial of AMX0035 for the Treatment of ALS

WASHINGTON and CAMBRIDGE, Mass.July 20, 2016 /PRNewswire-USNewswire/ — The ALS Association and Amylyx Pharmaceuticals based in Cambridge, Mass. are pleased to announce that The ALS Association and ALS Finding a Cure® are jointly providing funding to support a Phase II trial of a potential new therapeutic approach being developed by Amylyx for the treatment of ALS. The $2.96 million grant is being awarded by a collaboration of The Association with ALS Finding a Cure as part of the ALS Accelerated Therapeutics Initiative (ALS ACT), undertaken by the two organizations in 2014. Funding support from The Association is dedicated to Massachusetts General Hospital (MGH) to run this clinical trial.

“To accelerate developing new treatments for ALS, the two foundations issued a global, competitive call for grant proposals for therapies that had a strong scientific rationale and where a biomarker of drug effect could be measured,” said Merit Cudkowicz, MD, Chief Medical Officer for ALS Finding A Cure and Chairman of Neurology at Massachusetts General Hospital. “AMX0035 was chosen as one of the top new therapeutic candidates to bring forward for ALS.”

“The ALS Association is pleased to support this important clinical trial designed to incorporate biomarkers to track changes in the disease process. Results from this trial will significantly contribute to biomarker development and inform us about the value of using this combined therapeutic approach,” said Lucie Bruijn, Ph.D., M.B.A., Chief Scientist for The ALS Association.

Amylyx’s oral therapeutic candidate, AMX0035, is a combination of two drugs, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). Each compound has exhibited strong efficacy in several cellular and animal models of ALS. Furthermore, PB and TUDCA have been individually tested in clinical trials of ALS and both showed safety and tolerability and preliminary signs of efficacy. In preclinical trials, Amylyx has shown a synergistic effect between the two compounds, suggesting that the combination may be more effective in people with ALS than either compound alone.

The trial, which is expected to begin later this year, will test the safety and tolerability of AMX0035, as well as functional outcomes. Analysis of biomarkers of cell function, neuronal damage, and inflammation will be included as a major part of the trial, along with a new measure of muscle strength that has been shown to be tightly correlated with disease progression.

ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which leads to total paralysis and death, usually within two to five years of diagnosis. For unknown reasons, veterans are twice as likely to develop ALS as the general population. There is no cure, and only one drug approved by the U.S. Food and Drug Administration (FDA) modestly extends survival.

“Amylyx is grateful for this support and acknowledgement from The ALS Association and ALS Finding a Cure Foundation,” said Justin Klee, Co-Founder and President of Amylyx. Josh Cohen, Co-Founder and CEO added, “The grant, which includes clinical trial support from the Massachusetts General Hospital Neurological Clinical Trial Institute and the Northeast ALS Consortium will accelerate the launch and execution of the first clinical study of AMX0035.”

“I am thrilled to evaluate AMX0035 in this upcoming study,” said Sabrina Paganoni, M.D., Ph.D., the principal investigator for the study and Assistant Professor at Harvard Medical School. “We also expect the biomarker and muscle strength analyses to advance our understanding of ALS and improve our ability to measure and target this disease.”

About The ALS Association
The ALS Association is the largest national non-profit organization fighting Lou Gehrig’s Disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure.  For more information about The ALS Association, visit our website at

About ALS Finding a Cure®
ALS Finding A Cure® is a new research organization dedicated to being a game changer in discovering a cure for this fatal disease. Founded in 2014 by noted philanthropist and Conair CEO, Leandro (Lee) Rizzuto, as a tribute to Christie Rizzuto, Lee’s daughter-in-law who was diagnosed with ALS in 2009 at the age of 41, and under the joint leadership of Lee Rizzuto and Peter N. Foss, ALS Finding A Cure is focused on identifying the gaps in the scientific understanding of ALS that are preventing the development of a cure. The organization, a program of The Leandro P. Rizzuto Foundation, collaborates with a wide range of companies, ALS organizations, other disease non-profits, and ALS patients and families to ensure research efforts are non-duplicative, synergistic and focused on the ultimate goal: getting to a cure. For more information about ALS Finding a Cure, visit

About Amylyx Pharmaceuticals
Amylyx is developing a novel therapeutic for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases that targets the neuroinflammation and nerve cell death that characterize these disorders. The company’s therapeutic, AMX0035, is a proprietary, fixed-dose combination therapy that blocks nerve cell death and neurotoxic inflammation. Learn more

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SOURCE The ALS Association

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