BOSTON – The HEALEY ALS Platform Trial led by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital in collaboration with the Northeast ALS Consortium (NEALS) has successfully enrolled its first participants for ABBV-CLS-7262, an investigational product being developed by Calico Life Sciences LLC in collaboration with AbbVie.

The HEALEY ALS Platform Trial is designed to evaluate multiple investigational products simultaneously, thus accelerating the development of effective and breakthrough treatments for people living with ALS. The Sean M. Healey & AMG Center for ALS works closely with NEALS and industry partners to tailor the platform trial regimen to their investigational product. ABBV-CLS-7262 becomes the sixth regimen for this innovative design paradigm.

Investigational product candidates that enter the platform trial are selected by a group of expert ALS scientists and members of the Platform Trial Therapy Evaluation Committee. “We are excited to officially begin testing ABBV-CLS-7262 in the HEALEY ALS Platform Trial,” 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. “This is a new target and approach in ALS therapy development. We are grateful for the partnership and participation of people with ALS that allow trials like this to be possible.”

ABBV-CLS-7262 activates eIF2B, a key regulator of the integrated stress response (ISR), a pathway activated in people with ALS. In neurons exposed to cellular stressors, inhibition of the ISR by ABBV-CLS-7262 restores protein synthesis and dissolves pre-formed TDP-43 containing stress granules. This effect of ABBV-CLS-7262 is of clinical interest because TDP-43 containing stress granules are thought to lead to TDP-43 inclusions, a hallmark of ALS pathology.

“Launching enrollment for this novel investigational product, the sixth regimen in the Platform Trial, provides further hope towards discovering treatments for people living with ALS,” says Senda Ajroud-Driss, MD, Associate Professor of Neurology and Director of the Lois Insolia ALS Clinic at Northwestern University Feinberg School of Medicine. “We are grateful for the support from Tackle ALS, The ALS Association, ALS Finding A Cure®, ALS One, The Muscular Dystrophy Association, I AM ALS, and Tambourine ALS Collaborative.”

For updates on the trial, please join our weekly Healey ALS Platform webinars

Watch this video for more information on the mechanism of action behind ABBV-CLS-7262.

 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), Nuedexta, and Relyvrio.

 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.