Pre-clinical studies at Columbia University’s Motor Neuron Center Complement
Amylyx Phase 2 Clinical Studies of Lead Therapeutic Compound

New York, NY and Cambridge, MA (September 7, 2017) – Project ALS and Amylyx Pharmaceuticals today announced a collaboration to undertake pre-clinical studies with Amylyx’s oral compound AMX0035 to advance the understanding of the compound’s neurobiological effects. The studies to be conducted at the Project ALS Pre-Clinical Core at Columbia University’s Motor Neuron Center will complement the company’s recently initiated Phase 2 clinical program of AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS).

The Project ALS Pre-Clinical Core at the Columbia University’s Motor Neuron Center has established a unique integrated and standardized platform for the testing and validation of new therapeutic strategies in recognized experimental models of ALS and for biomarker discovery. The Core, developed in collaboration with Project ALS, will accelerate the translation of new promising therapies to patients by facilitating speedy testing of new therapeutic leads discovered by laboratories studying motor neuron biology, genetics and genomics.

“The collaboration will bring together a promising therapeutic candidate for a devastating disease with leading edge, fundamental neuroscientific research at Columbia,” said Valerie Estess, director of research for Project ALS. “The studies in this collaboration will provide greater insight into the neurobiological effects of AMX0035, and hopefully optimize its beneficial effects.”

The collaboration is an outgrowth of previous studies by The Project ALS Pre-Clinical Core at Columbia University of tauroursodeoxycholic acid (TUDCA), one of the components of AMX0035. “We look forward to evaluating AMX0035 in models of ALS. It is exciting that TUDCA has independently shown promise in both our labs and in studies conducted by the company,” said Drs. Hynek Wichterle and Serge Przedborski, co-directors of The Project ALS Pre-Clinical Core and tenure faculty in the Departments of Pathology and Neurology at Columbia University.

AMX0035 is a combination of two drugs, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). Previous studies of PB and TUDCA as individual agents demonstrated efficacy in cellular and animal models of ALS.  AMX0035 has been shown to synergistically prevent nerve cell death and neurotoxic inflammation, hallmarks of ALS, in preclinical models. In addition, PB and TUDCA have been individually tested in ALS clinical trials and each demonstrated safety, tolerability, and preliminary signs of efficacy.  AMX0035 recently entered Phase 2 clinical development to evaluate its safety and efficacy in ALS. The trial, called CENTAUR, is a 24-week, randomized, double-blind, placebo-controlled study in 132 participants with ALS. More information on the CENTAUR trial can be found at, NCT03127514 and at

“Amylyx is very excited to partner with Project ALS and the researchers at Columbia’s Motor Neuron Center to advance these experiments. We hope these studies will provide valuable insights into both AMX0035 and ALS biology that will ultimately improve the lives of patients with ALS,” said Kent Leslie, Chief Scientific Officer of Amylyx.

About ALS
ALS is a brain disease that is closely related to Parkinson’s, Alzheimer’s, and Huntington’s. Also known as Lou Gehrig’s disease, ALS targets brain cells called motor neurons. Motor neurons send messages from the brain to muscles throughout the body. In ALS, as motor neurons weaken and die, a person progressively loses the ability to walk, speak, swallow, and breathe. ALS affects adults of all ages, from teens to seniors.

About Project ALS
Project ALS is a non-profit 501(c)3 that identifies and funds the most promising scientific research that will lead to the first effective treatments and a cure for ALS. The Project ALS Pre-Clinical Core at Columbia University is supported by generous donations from the Kleiner Family Research Initiative, Stu and Leslie Hendel, Team Chris Combs, and other donors.

About Motor Neuron Disease Center
The Center for Motor Neuron Biology and Disease at Columbia University, in short the Motor Neuron Center, offers an unparalleled pre-clinical academic initiative integrated with the ALS Clinic Center, the Columbia Precision Medicine Initiative, the Institute of Genomic Medicine, and the Zuckerman Mind, Brain, and Behavior Institute.

For the past decade, the Motor Neuron Center faculty members have utilized existing and novel models of ALS as well as patient derived iPS cell motor neurons to test a variety of therapeutic strategies ranging from pharmacological approaches, using small molecules, to gene therapies, using viral vectors or CRISPR based genome engineering. MNC scientists work with internationally renowned clinicians experienced in clinical studies including therapeutic trials for ALS and other paralytic disorders, eager to provide advice about the feasibility of therapeutic approaches and to test promising leads in clinical trials.

About Amylyx
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.


For press and research information, please contact: Laura Nicholas at or 212-420-7382 or Peter Steinerman (for Amylyx) at or 516-641-8959.