Amylyx Nixes AMX0035 Development After Failed Study

Amylyx Pharmaceuticals says it will discontinue development of its investigational therapy AMX0035 (sodium phenylbutyrate and taurursodiol) in progressive supranuclear palsy (PSP) after the drug failed to show benefit in a mid-stage clinical trial. The Phase 2b ORION study found no differences between AMX0035 and placebo on primary or secondary outcomes at 24 weeks, prompting the company to terminate both the trial and its open-label extension, the company says, adding that a planned Phase 3 study will also not move forward. Despite the lack of efficacy, the treatment continued to show a safety profile consistent with earlier studies and was generally well tolerated, the company says.

PSP is a rare and fatal neurodegenerative disorder that impairs movement, balance, speech, and vision, typically progressing within six to eight years after diagnosis, according to the company. There are no disease-modifying treatments currently approved, it adds.