Amylyx Pharmaceuticals Announces First Patients Enroll in Open Label Extension of CENTAUR Phase 2 Clinical Trial of AMX0035 for Treatment of Amyotrophic Lateral Sclerosis

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Amylyx Pharmaceuticals, Inc., in collaboration with The ALS Association, ALS Finding a Cure®, the Northeast ALS Consortium, and the Massachusetts General Hospital Neurological Clinical Research Institute, today announced that the first patients to complete the CENTAUR phase II clinical trial have elected to continue treatment in an open label extension (OLE) study of AMX0035. All patients who complete the main phase of the study will be eligible for this extension and will receive AMX0035 with no placebo comparator.
“The CENTAUR-OLE study provides patients with the option of continuing treatment once they have completed the 24-week study period,” said Sabrina Paganoni, M.D., Ph.D., Principal Investigator for the study and physician at the Massachusetts General Hospital and Spaulding Rehabilitation Hospital. “The study will provide data on AMX0035’s long-term safety and tolerability as well as additional insights into its efficacy. There is a significant and urgent need for therapies for ALS, as there are very limited treatment options.”
The CENTAUR (“Combination of Phenylbutyrate and Tauroursodeoxycholic Acid”) trial, which began dosing patients in July 2017, is a randomized, double-blind, placebo-controlled Phase II clinical trial enrolling 132 ALS patients nationwide. The trial’s primary objectives are to evaluate the safety and tolerability of AMX0035 and to assess the drug’s impact on disease progression as measured by the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) over the 24-week study period. The CENTAUR trial is also the first drug trial to measure patient muscle strength using an innovative device called ATLIS (“Accurate Test of Limb Isometric Strength”). Additionally, the trial will include assessments of promising blood and imaging based biological markers of ALS to confirm target engagement.
Amylyx CEO and co-founder, Joshua Cohen commented, “We are excited to offer this extension to the participants in this study and are so grateful for their commitment.” Justin Klee, President and co-founder added, “Thank you also to The ALS Association, ALS Finding a Cure, the Northeast ALS Consortium, the Neurological Clinical Research Institute at MGH, and the ALS community for their outstanding support in helping us offer this open label extension.”
AMX0035 is an oral combination of two small molecules, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). Each compound has shown efficacy in several cellular and animal models of ALS. When individually tested in ALS clinical trials, PB and TUDCA have both met endpoints of safety, tolerability, and demonstrated preliminary signs of efficacy. In preclinical trials, Amylyx has demonstrated a synergistic effect between the two compounds, suggesting that the combination may have improved efficacy compared to the individual agents.
More information on the CENTAUR trial can be found at or, NCT03127514. Please contact (617-724-7210), or email Emily Engel at, if you are interested in participating. Contact information for trial sites is also available on
Background on ALS
Amyotrophic lateral sclerosis, ALS, is the most prevalent adult-onset progressive motor neuron disease, affecting more than 20,000 people in the U.S. and an estimated 450,000 people worldwide. ALS causes the progressive degeneration of motor neurons, resulting in rapidly progressing muscle weakness and atrophy that eventually leads to partial or total paralysis; on average, the disease is fatal within just three years from onset. There are currently just three therapies approved specifically for treating ALS—riluzole and the recently-approved edaravone.

About MGH Neurological Clinical Research Institute (NCRI)
Established in 1994 at the heart of Massachusetts General Hospital (MGH), the mission of the NCRI ( is to accelerate translational research in neurological disorders through initiating and testing novel therapies. The NCRI is a premier academic Contract Research Organization (CRO) that leverages the vast disease knowledge of extraordinary physician-investigators to provide high quality clinical trials design and conduct of multicenter neurological trials. The NCRI serves as both the Coordinating Center and a clinical research site for the Northeast ALS Consortium (NEALS) ( a network of ALS specialists at more than 100 locations worldwide.

About The ALS Association
The ALS Association is the only 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 dedicated to being a game changer in discovering a cure for this fatal disease. Founded in 2014 by noted philanthropist and Conair Founder and Chairman, 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 leadership of Peter N. Foss and a team of renowned ALS researchers, 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 research organizations, and ALS patients and families to ensure research efforts are non-duplicative, synergistic, and focused on the ultimate goal: finding a cure. For more information about ALS Finding a Cure®, visit

About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company currently developing a novel therapeutic for Amyotrophic Lateral Sclerosis (ALS) 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 have worked synergistically to prevent cell death and neurotoxic inflammation in multiple preclinical models. AMX0035 entered a Phase 2 clinical trial (CENTAUR) in ALS patients in June 2017. Learn more at:



Amylyx Pharmaceuticals Inc.
Josh Cohen, 617-682-0917
Steinerman Biomedical Communications
Peter Steinerman, 516-641-8959



Source: Amylyx Pharmaceuticals, Inc.

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