Sean M. Healey & AMG Center for ALS Announces New Regimen for Inclusion in the HEALEY ALS Platform Trial with Eli Lilly and Company

The Sean M. Healey & AMG Center for ALS is pleased to announce the inclusion of a new regimen, LY3873862, in the HEALEY ALS Platform Trial with Eli Lilly and Company (Lilly).

The HEALEY ALS Platform Trial is a multicenter, double-blind, placebo-controlled, adaptive trial for ALS created in partnership with the Northeast ALS Consortium (NEALS). The goal of the HEALEY ALS Platform trial is to accelerate the development of potential new ALS therapies. The trial tests and evaluates multiple investigational drugs simultaneously, shares infrastructure across trial sites, and improves start up and enrollment efficiencies, allowing for fast results.

The Therapy Evaluation Committee selected Lilly to work with the Healey & AMG Center on the design of a new regimen to include in the HEALEY ALS Platform Trial. Lilly received FDA May proceed of their investigational new drug (IND) and has begun operational start-up of LY3873862. Lilly is pleased to announce that further information regarding its LY3873862 regimen will be shared in a poster at the 2025 Annual NEALS Conference in October.

LY3873862 is a potent and selective oral small molecule that inhibits SARM1, a key regulatory protein activated when neurons are stressed or injured. Activation of SARM1 causes axonal degeneration (nerve fiber breakdown), a major driver of disease progression in ALS. Pre-clinical studies conducted at Lilly have demonstrated that blocking SARM1 function with LY3873862 strongly protects axons in laboratory studies and whole animal models. Additionally, human genetic studies have identified a connection between the SARM1 gene and sporadic ALS, revealing that certain genetic variants that make SARM1 more active are found more frequently in ALS patients. Therefore, blocking SARM1 function with LY3873862 represents a promising new therapeutic approach for the treatment of ALS.

“Together with Lilly, we designed a regimen that meets key scientific objectives within the flexible master protocol framework,” says Sabrina Paganoni, MD, PhD, co-principal investigator of the HEALEY ALS Platform Trial and co-director of the Neurological Clinical Research Institute (NCRI) at Mass General. “This is a testament to both the collaborative design process and the adaptability of the master protocol to accelerate the development of new investigational drugs.”

The Lilly regimen will be led by Dr. Paganoni and Terry Heiman-Patterson, MD, director of the Temple MDA/ALS Center of Hope and professor of the Department of Neurology at the Lewis Katz School of Medicine at Temple University.

“We are excited to begin this new collaboration with Lilly,” states Dr. Heiman-Patterson. “This furthers our mission to increase our knowledge of ALS and find meaningful therapies for people affected by ALS.”

“We remain hopeful in discovering effective treatments for ALS and appreciate the work of the Therapy Evaluation Committee in selecting LY3873862,” 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, and executive director of the Neuroscience Institute at Mass General Brigham. “We would like to extend a sincere thank you to industry partners, research collaborators, clinical trial sites and people living with ALS and their families for their continued support in this trial.”

For further information about the HEALEY ALS Platform Trial, please visit: HEALEY ALS Platform Trial

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.

About the Neurological Clinical Research Institute

The Neurological Clinical Research Institute (NCRI) at Mass General is an academic research organization composed of innovative researchers experienced and passionate about designing, developing, facilitating, and conducting multicenter clinical trials in neurological diseases. Our mission is to accelerate translational research in neurological disorders by initiating clinical development of novel therapies and leading trials of these compounds. We strive to be at the cutting edge of innovative trial design to accelerate therapy development by creating new trial methodology, discovering novel biomarkers and refining outcome measures.