Sean M. Healey & AMG Center for ALS Announces First Participant Enrolled in Regimen I of the HEALEY ALS Platform Trial Evaluating NUZ-001 by Neurizon Therapeutics

The Sean M. Healey & AMG Center for ALS at Mass General Brigham is pleased to announce that the first participant received study medication in Regimen I of the HEALEY ALS Platform Trial evaluating NUZ-001 by Neurizon Therapeutics.

The HEALEY ALS Platform Trial is a multicenter, double-blind, placebo-controlled, adaptive trial for amyotrophic lateral sclerosis (ALS) created in partnership with the Network of Excellence for ALS (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.

“We gained valuable experience and learned so much from data collected in the past seven regimens in the Platform Trial,” says Merit Cudkowicz, MD, MSc, Director of the Sean M. Healey & AMG Center and Executive Director of the Mass General Brigham Neuroscience Institute. “We look forward to implementing our updated master protocol in Regimen I with Neurizon and continuing to deepen our understanding of ALS.”

Regimen I is led by James Berry, MD, MPH, Chief of the Division of ALS and Motor Neuron Diseases at Mass General Brigham and Michael Weiss, MD, Director of the Neuromuscular Diseases Division at the University of Washington.

The trial will evaluate NUZ-001, an oral therapy that targets key pathological mechanisms in ALS, including TDP-43 protein aggregation and impaired autophagy, which are central features across multiple neurodegenerative diseases. Initial Phase 1 studies support favorable safety and pharmacokinetics of the drug. NUZ-001 is developed by Neurizon Therapeutics Limited, a biotechnology company based in Melbourne, Australia, and is not yet approved for use in any country.

“The Platform Trial is designed to collect both clinical data and biofluids to allow us to fully evaluate the effect of a study drug, when possible,” says Dr. Berry. “Regimen I has incorporated numerous novel biomarkers that will shed light on the biological engagement of NUZ-001.”

“This clinical trial will provide us with more data to understand NUZ-001’s effects in people living with ALS, ultimately bringing us closer to finding effective treatments,” says Dr. Weiss. “The initiation of participant enrollment marks an important milestone for this regimen.”

“This is an exciting step in the Platform Trial,” 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 Brigham. “This work is critical to accelerating research in ALS, and it would not be possible without the participants and their families, industry partners, research collaborators and clinical trial sites. Thank you for your contributions to this trial.”

On March 19th, there will be a Healey & AMG Center community webinar with Neurizon on Regimen I.

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 Brigham

At the Sean M. Healey & AMG Center for ALS at Mass General Brigham, 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 Brigham 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.