Prilenia Announces Plans to Initiate Global Phase III Study of Novel ALS Treatment

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Prilenia Therapeutics B.V. has announced it will present clinical data supporting a future Phase III study of the investigational therapy pridopidine for amyotrophic lateral sclerosis (ALS). Prilenia, a clinical stage biotechnology company that develops treatments for neurodegenerative diseases and neurodevelopmental disorders, plans to present the data during the 14th Annual California ALS Research Summit in Los Angeles.

The future Phase III study will examine the effectiveness of pridopidine for ALS and is slated to start in the second half of 2024. According to a company press release from Prilenia, “Pridopidine is an investigational oral, small molecule, highly selective and potent Sigma-1 Receptor (S1R) agonist being studied as a potential treatment for ALS and Huntington’s disease (HD). The S1R is highly expressed in the brainstem and spinal cord, areas implicated in ALS and important for bulbar function and speech.”¹

According to an estimate from the Centers for Disease Control and Prevention, there are over 31,000 patients living with ALS in the United States as of 2017. On average 5,000 new patients are diagnosed with ALS every year.²

“Based on the clinical data generated to date, pridopidine has the potential to become a meaningful treatment for ALS, slowing key measures of disease progression, including function, respiration, quality of life and impact on speech, which is very meaningful for patients and caregivers, as well as prolonging overall survival,” said Michael R. Hayden, PhD, CEO and founder of Prilenia, in a press release. “We believe it is important for Prilenia to advance our clinical program in ALS, and we are actively planning a single, global, pivotal Phase 3 clinical trial. Our sincere appreciation to the many ALS patients, caregivers and healthcare providers who have contributed so much to our clinical studies as well as to those who will participate in future trials. We also are grateful to Dr. Merit Cudkowicz and the entire team at the Sean M. Healey & AMG Center for ALS for their innovative and passionate approach to evaluating pridopidine as we continue our close working relationship.”

According to the press release, pridopidine did not meet the primary outcome measure in an earlier Phase II study, the HEALEY ALS Platform Trial. However, there were data from the pre-specified subgroup that showed promising results with slower disease progression among participants with definite ALS who were also early in the course of the disease (less than 18 months from symptom onset).

“In the Phase II clinical trial, pridopidine showed encouraging results for the potential treatment of ALS across multiple secondary and exploratory measures with an excellent safety profile, and we are pleased to work closely with Prilenia on the advancement of this program into Phase III,” said Merit Cudkowicz, MD, MSc, principal investigator, and sponsor of the HEALEY ALS Platform Trial, Director of the Sean M. Healey & AMG Center for ALS, Chair of the Department of Neurology at Massachusetts General Hospital, and the Julieanne Dorn Professor of Neurology at Harvard Medical School said in a press release.

References

1. Prilenia Announces Clinical Data in Support of its Plans to Initiate Global Phase 3 Study in ALS. Prilenia Therapeutics B.V. News release. January 19, 2024. Accessed January 23, 2024. https://news.prilenia.com/press-releases/press-release-details/2024/Prilenia-Announces-Clinical-Data-in-Support-of-its-Plans-to-Initiate-Global-Phase-3-Study-in-ALS/default.aspx
2. Amyotrophic lateral sclerosis. Centers for Disease Control and Prevention, U.S. Department of Health & Human Services. Accessed January 23, 2024. https://www.cdc.gov/als/WhatisALS.html