An experimental drug has successfully slowed the progress of amyotrophic lateral sclerosis in a phase 2 study, its developer announced on Tuesday.
Participants taking AMX0035, designed to reduce neuronal death and dysfunction, had slower ALS disease progression than those given a placebo, according to Amylyx Pharmaceuticals Inc.
The CENTAUR trial assessed the drug’s safety and tolerability along with its effect on muscle strength, lung vital capacity, and biomarkers of neuronal degeneration, reported principal investigator Sabrina Paganoni, M.D., Ph.D., and colleagues.
Nearly 90% of participants who completed the trial elected to enroll in an extension study. Interim data from that investigation will be presented in 2020, the company added.
“With these results, Amylyx now has a responsibility to move ahead as efficiently as possible, as people living with ALS don’t have time to wait,” said Justin Klee, president and co-founder of Amylyx.
Detailed results from the trial will be submitted for publication and presented at a future medical congress, the company reported. Amylyx will work with the U.S. Food and Drug Administration and the ALS community to decide next steps, Klee concluded.