Investigational drug ABBV-CLS-7262 initiates design phase for entry into the HEALEY ALS Platform Trial

BOSTON – The HEALEY ALS Platform Trial led by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) today announced topline results from the regimen evaluating verdiperstat, an oral myeloperoxidase inhibitor that is thought to reduce neuro-inflammation, developed by Biohaven Pharmaceutical Holding Company Ltd., in adults with amyotrophic lateral sclerosis (ALS).

The HEALEY ALS Platform Trial is designed to accelerate the development of breakthrough treatments for persons with ALS by testing multiple drugs at the same time. Verdiperstat is among the initial investigational treatment regimens tested using this innovative paradigm.

The primary endpoint of the verdiperstat regimen was not met; there was no clinically meaningful or statistically significant slowing of disease progression as measured by ALS Functional Rating Scale-Revised (ALSFRS-R, a 12-item functional activity scale) and survival through 24 weeks among participants randomized to active treatment with verdiperstat compared to placebo. No statistically significant benefits were seen on key secondary measures of disease progression including muscle strength, respiratory function, and survival. Full study results, including exploratory outcomes, biomarker, and subgroup analyses, are expected in the coming months and will be presented at an upcoming scientific meeting.

“We are thankful to the many people who participated in this study. We will learn from these results and continue to use this data to inform future advances in ALS trial design," says Merit Cudkowicz, MD, MSc, principal investigator and sponsor of the HEALEY ALS Platform Trial, director of the Sean M. Healey & AMG Center for ALS, chief of the Department of Neurology at MGH, and the Julieanne Dorn Professor of Neurology at Harvard Medical School. “Though we did not achieve positive results with this regimen, we were able to get a decisive answer with a more efficient approach than traditional stand-alone trials."

“This regimen demonstrated the efficiency of the innovative platform design in evaluating drugs for ALS,” said Jinsy Andrews, MD, MSc, verdiperstat regimen lead, assistant professor of neurology, and Director of Neuromuscular Clinical Trials at Columbia University Irving Medical Center, NY.

“We are at an exciting time for therapeutic development, and we continue to strive for future successes for the ALS community,” said Suma Babu, MD, MPH, verdiperstat regimen co-lead and assistant professor of neurology at MGH/Harvard Medical School. 

“We would like to extend our sincere gratitude to participants in the verdiperstat regimen who committed their invaluable time and hope in the trial as well as the NEALS investigators and study staff who dedicated their effort and expertise," says Sabrina Paganoni, MD, PhD, co-principal investigator of the HEALEY ALS Platform Trial and co-Director of the Neurological Clinical Research Institute at MGH. “Even though the trial results were negative, participants' involvement in the trial contributed greatly to ALS research by providing substantial clinical and biomarker data that will help us better understand ALS disease mechanisms.”

This important milestone was made possible by funding from The AMG Charitable Foundation, Tackle ALS, The ALS Association, ALS Finding A Cure^®, ALS One, The Muscular Dystrophy Association, Arthur M. Blank Family Foundation, I AM ALS, Tambourine ALS Collaborative and countless individuals who support the HEALEY ALS Platform Trial.

Background on ALS
Amyotrophic lateral sclerosis, ALS, is the most prevalent adult-onset progressive motor neuron disease, affecting approximately 30,000 people in the U.S. and an estimated 500,000 people worldwide. ALS causes the progressive degeneration of motor neurons, resulting in progressive muscle weakness and atrophy. There are currently few FDA therapies approved for treating ALS—riluzole, edaravone (IV and oral formulation), and Nuedexta.

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 on a quest to discover life-saving therapies for all individuals affected by ALS. Launched in November 2018, the Healey Center leverages a global network of scientists, physicians, nurses, caregivers, people with ALS and families working together to accelerate the pace of ALS therapy discovery and development.

Under the leadership of Merit Cudkowicz, MD and a Science Advisory Council of international experts, we are reimagining how to develop and test the most effective therapies to treat the disease, identify cures and, ultimately, prevent it.

The key to our success is our tightly integrated research and clinical efforts, encouraging opportunities to bring the challenges our patients face every day into our laboratories, focusing investigations on finding solutions that will make a meaningful difference to our patients without delay. Our collaborative efforts are designing more efficient and effective clinical trials while broadening access to these trials for people with ALS.

About the Massachusetts General Hospital
Massachusetts General Hospital, founded in 1811, is the original and largest teaching hospital of Harvard Medical School. The Mass General Research Institute conducts the largest hospital-based research program in the nation, with annual research operations of more than $1 billion and comprises more than 9,500 researchers working across more than 30 institutes, centers and departments. In July 2022, Mass General was named #8 in the U.S. News & World Report list of "America’s Best Hospitals." MGH is a founding member of the Mass General Brigham healthcare system.