Another Phase 3 Trial of PB-TURSO for Amyotrophic Lateral Sclerosis Being Initiated at FDA Request

A phase 3 trial of sodium phenylbutyrate-taurursodiol (PB-TURSO) (AMX0035; Amylyx Pharmaceuticals, Cambridge, MA) for amyotrophic lateral sclerosis (ALS) is expected to start in the 3rd quarter of 2021. In a nearly 3-year overall survival  analysis, those treated with PB-TURSO had a 44% decrease in likelihood of death, extending survival for approximately 6 months. PB-TURSO also provided improvements in functional status.
 
The Food and Drug Administration (FDA) showed interest in data from an additional placebo-controlled clinical trial prior to receiving a New Drug Application (NDA). The FDA also expressed that it would continue to discuss how regulatory requirements may be met in the most expeditious way possible.   
 
“People living with ALS need clinical answers and solutions quickly. We have been in close contact with physicians and global health authorities to rapidly evaluate AMX0035 for the treatment of ALS,” said Joshua Cohen, cochief executive officer, chairman and cofounder of Amylyx. “We are thrilled to plan our submissions in Europe and Canada and will continue working closely with regulators and the ALS community worldwide to determine the most expeditious and responsible pathways to advance AMX0035 through the clinical development process. We appreciate all of the advice and guidance from the regulators worldwide and will continue to act with haste and to keep the community updated.” 
 
“With the results from CENTAUR, we showed that AMX0035 may provide people living with ALS hope and the chance to function better and live longer lives,” said Sabrina Paganoni, MD, PhD, principal investigator of the CENTAUR trial, investigator at the Healey & AMG Center for ALS at Massachusetts General Hospital and Assistant Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital. “We are very excited to see AMX0035 advancing on multiple regulatory fronts and remain optimistic that it can potentially help people living with ALS around the world.”