The AMG Center for ALS at Mass General recently announced, along with Amylyx Pharmaceuticals, Inc., that AMX0035 demonstrated a significant treatment benefit for people with ALS in the CENTAUR study. The team found that AMX0035 had a statistically significant slowing of ALS disease progression as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) compared to placebo (p<0.05), which was the primary outcome of the study.
Both the commercial biotech (Amylyx) and the research center (Mass General) will submit detailed results from the CENTAUR study to a peer-reviewed publication and presentation at a future medical congress.
The Drug: AMX0035
AMX0035 is Amylyx’ first-in-class investigational therapy designed to reduce neuronal death and dysfunction. It targets mitochondrial and endoplasmic reticulum-dependent neuronal degeneration pathways. In clinical trials, AMX0035 has overall been shown to be safe and effective. A preclinical study revealed that AMX0035 limited cell death and neurotoxic inflammation in differing ALS models. As reported, CENTAUR had 132 ALS participants to assess the safety and effectiveness of AMX0035. During the study, patients received either placebo or the experimental drug. As reported the study results reveal great promise.
Principal Investigator Comments
Sabrina Paganoni, MD, PhD, principal investigator of the CENTAUR study and investigator at the Healey Center for ALS at Mass General and Assistant Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital, reported, “Today marks a significant step forward in the fight to develop new treatments for ALS.” She continued, “The study results highlight AMX0035 as a potentially beneficial new treatment for people with ALS, and the design and execution of the CENTAUR trial are a testament to true collaboration across the many stakeholders in this fight.”
Participants in CENTAUR were given the option after the trial to enroll in an open-label extension study to receive treatment with AMX0035. Nearly 90 percent of participants who completed CENTAUR elected to enroll in the extension study. Interim data from the ongoing extension study will be presented in 2020. In addition, the company will provide an update on regulatory plans and further details on expanded access plans in early 2020. The CENTAUR study was conducted by investigators at 25 top ALS medical centers through the Northeast ALS (NEALS) consortium. NEALS is an international, independent, non-profit group of researchers who collaboratively conduct clinical research in Amyotrophic Lateral Sclerosis (ALS) and other motor neuron diseases. NEALS researchers are dedicated to finding treatments for people with ALS and motor neuron disease as rapidly as possible.
Other Organizations Comment
Dr. Merit Cudkowicz, Chief Medical Officer from ALS Finding a Cure® Director of the Healey Center of ALS, Chief of Neurology at Mass General, and the Julieanne Dorn Professor Neurology at Harvard Medical School, noted, “ALS Finding a Cure is product to have catalyzed and supported the CENTAUR study, and I am encouraged by what the results mean for people living with people with ALS. Our team at the Mass General Neurological Clinical Research Institute is product of this collaboration with Amylyx and our colleagues in the Northeast ALS Consortium on this important study.”
About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is a pharmaceutical company developing a novel therapeutic for Amyotrophic Lateral Sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases. For more information, visit their website.
About the Sean M. Healey & AMG Center for ALS at Mass General
The Sean M. Healey & AMG Center for ALS at Mass General includes a diverse team of researchers, clinicians, project managers, and information technologists who are determined to accelerate the pace of ALS therapy development. Under the leadership of Merit Cudkowicz, MD, and a Science Advisory Council of international experts, Healey faculty partners with scientists, clinicians, and people with ALS from all over the world in a shared mission to develop treatments to cure ALS. For more information, visit here.
Sabrina Paganoni, MD, Mass General