The Sean M. Healey & AMG Center for ALS and Prilenia Therapeutics Announce First Participant Enrolled in NIH-Sponsored ACT for ALS Expanded Access Protocol for Pridopidine

in Portfolio News

BOSTON―The Healey & AMG Center for ALS at Massachusetts General Hospital in collaboration with Prilenia Therapeutics B.V is thrilled to announce the enrollment of the first participant in the National Institutes of Health (NIH) – National Institute of Neurological Disorders and Stroke (NINDS) sponsored Expanded Access to Pridopidine Protocol (EAP) in Amyotrophic Lateral Sclerosis (ALS), supported by the U.S. government’s Accelerating Access to Critical Therapies for ALS Act (ACT for ALS).

The EAP will include up to 200 ALS individuals treated with pridopidine for up to two years, at up to 45 sites across the U.S, and is open to those who are not eligible for other clinical trials. Pridopidine, an investigational product from Prilenia, showed encouraging results and favorable safety profile in its previous Phase 2 HEALEY ALS Platform Trial. Prilenia is planning a global phase 3 study starting in H2 2024.

EAPs, also referred to as Compassionate Use, are pathways for people with a serious and life-threatening disease to access investigational products that are part of clinical trials, but not yet approved by the FDA. The EAP program brings new options to be part of research to people by oordinating access to experimental drugs and enables the collection of safety and biomarker data in a population not studied in clinical trials. This data can help inform the next trial or help support market approval for a broader group of people with ALS than those typically included in clinical trials.

“There is an urgent need for more effective therapies to help people suffering from ALS,” said Walter Koroshetz, M.D., director of NINDS. “The data collected through the Expanded Access Protocol may help improve our understanding of the biological mechanisms underlying ALS, especially in individuals who are in later stages of the disease and are not eligible for other clinical trials.”

The study is led by principal investigators James Berry, MD, MPH, Suma Babu, MPH, MBBS, and Sabrina Paganoni MD, PhD, physician investigators at the Healey & AMG Center for ALS at Massachusetts General Hospital.

“NIH/NINDS-funded EAPs represent an important addition to ALS research,” said the investigators. “These studies pave the way for access to investigational drugs to people living with ALS who are not be eligible for clinical trials, thus advancing research and innovation in ALS.”

Pridopidine is a highly selective and potent sigma-1 receptor (S1R) agonist. The S1R is highly expressed in the brainstem and spinal cord, areas implicated in ALS and important for bulbar, speech, and limb function. The S1R positively regulates key cellular pathways that are commonly impaired in ALS and other neurodegenerative diseases. In ALS cell culture and mouse models, pridopidine has been shown to be neuroprotective, specifically mediated via the activation of S1R. Pridopidine is being developed by Prilenia, a clinical-stage biotech company focused on developing novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders.

This EAP is also supported by the Northeast ALS Consortium, an international, independent, non-profit group of 134 research sites around the world that collaboratively conduct clinical research in ALS and other motor neuron diseases. More information can be found on their website.

Watch this video for more information on the mechanism of action behind pridopidine.

This study is supported by NIH (1U01NS136021-01).

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 dozens of active 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.

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