Amylyx Secures Funds for Phase II Trial of AMX0035 in ALS

Cambridge, MA-based startup company, Amylyx Pharmaceuticals has secured funding from The ALS Association and ALS Finding a Cure for a Phase II trial of the company’s candidate ALS therapy, called AMX0035. Amylyx’s oral therapeutic candidate is a combination of two drugs, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA), which have been shown to exert synergistic neuroprotective and anti-infammatory effects in preclinical models. Each individual compound has exhibited efficacy in pre-clinical models  (Ryu et al., 2005, Vaz et al., 2015) and demonstrated safety and tolerability in ALS clinical trials (Cudkowicz et al., 2009, Elia et al., 2016), as well as preliminary signs of efficacy. The clinical trial, which is expected to begin later this year at Massachusetts General Hospital (MGH) in Boston, MA, will test the safety and tolerability of AMX0035, as well as functional outcomes.

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