Healey & AMG Center for ALS Announces New Investigational Drug Monepantel from PharmAust Limited will begin design for inclusion in HEALEY ALS Platform Trial

BOSTON- The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) has contracted with PharmAust Limited to begin designing a regimen in the HEALEY ALS Platform Trial.

The HEALEY ALS Platform Trial is led by the Healey & AMG Center in collaboration with the Northeast ALS Consortium (NEALS). The trial is the first of its kind, allowing for multiple investigational drugs to be tested and evaluated simultaneously to accelerate the development of potential new ALS therapies. Drug candidates that enter the platform trial are chosen by a group of expert ALS scientists and members of the Healey & AMG Center Science Advisory Committee.

“We are thrilled to work with PharmAust leadership to design their regimen to investigate Monepantel for people with ALS” said Merit Cudkowicz, M.D., M.S.c., principal investigator and sponsor of the HEALEY ALS Platform Trial, director of the Sean M. Healey & AMG Center for ALS, chair of the Department of Neurology at MGH, and the Julieanne Dorn Professor of Neurology at Harvard Medical School. “We thank the Therapy Evaluation Committee for their review of potential therapies to add to the HEALEY ALS Platform Trial.” 

The HEALEY ALS Platform Trial is a multicenter, double-blind, placebo-controlled, perpetual, adaptive platform trial testing multiple investigational products in parallel. The seventh regimen completed enrollment in May 2024. 

“We are grateful for the continued support of our industry partners, research collaborators, clinical trial sites, and the contributions made by our patients and their families” said Sabrina Paganoni, MD, PhD, co-principal investigator of the trial, physician scientist at the Healey & AMG Center, Co-Director of the Neurological Clinical Research Institute (NCRI) at MGH and Associate Professor at Harvard Medical School. “We look forward to using the information we learn by studying Monepantel to better understand ALS and continue in the fight against the disease.” 

About the Sean M. Healey & AMG Center for ALS at Mass General

At the Sean M. Healey & AMG Center for ALS at Mass General, we are committed to bringing together a global network of scientists, physicians, nurses, foundations, federal agencies, and people living with ALS, their loved ones, and caregivers to accelerate the pace of ALS therapy discovery and development.

Launched in November 2018, the Healey & AMG Center, under the leadership of Merit Cudkowicz, MD and a Science Advisory Council of international experts, is reimagining how to develop and test the most promising therapies to treat the disease, identify cures and ultimately prevent it.

With many clinical trials and lab-based research studies in progress right now, we are ushering in a new phase of ALS treatment and care. Together, we will find the cures.