Project ALS and Amylyx Enter Collaboration to Test AMX0035

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.

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