Amylyx Pharmaceuticals has reported that its investigational drug, AMX0035, slowed disease progression in the Phase II CENTAUR study for amyotrophic lateral sclerosis (ALS) treatment.
ALS, also called Lou Gehrig’s disease, is a neurodegenerative disease impacting nerve cells in the brain and the spinal cord. Over time, the condition results in death and loss of the brain’s control of muscle movement.
AMX0035 acts on mitochondrial and endoplasmic reticulum dependent neuronal degeneration pathways to decrease neuronal death and dysfunction.
The CENTAUR study investigated the safety and tolerability of the drug candidate for 24 weeks in patients aged 18-80.
The trial specifically compared AMX0035’s effect on disease progression, determined using the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), to placebo.
Other measures included muscle strength, lung vital capacity and neuronal degeneration biomarkers. The trial was conducted in alliance with Mass General’s Sean M Healey & AMG Center for ALS.
Data showed a statistically significant slowing of disease progression on ALSFRS-R with the Amylyx therapy compared to placebo.
Amylyx SAB chair Dr Rudolph Tanzi said that the mechanism of AMX0035 may provide a new treatment option for ALS, as well as Alzheimer’s disease patients.
Amylyx Pharmaceuticals president and co-founder Justin Klee said: “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.
“We will work closely with the FDA and the ALS community, including the wonderful Northeast ALS Consortium leadership and member sites that conducted the CENTAUR trial, to decide next steps and will continue to keep everyone informed.”
The company will announce detailed results from the CENTAUR trial in the future.
Patients enrolled in the Phase II trial were given an option to participate in a subsequent open-label extension study of AMX0035. Interim results from the ongoing extension study are expected next year.