Developing disease-modifying treatments for amyotrophic lateral sclerosis (ALS), a rare and aggressive disease, has proven challenging. The high failure rate in ALS clinical trials is often attributed to the disease’s unclear etiology and complex pathophysiology. At the 10th Congress of the European Academy of Neurology (EAN) 2024, Prilenia Therapeutics presented promising results from the additional analyses of the Phase II Healey ALS Platform Trial evaluating pridopidine in ALS patients. The positive results will pave the way for the Phase III trial of pridopidine, says GlobalData.
Erela Dana, Director of Neurology at GlobalData, comments, “The Healey ALS Platform Trial aims to accelerate the path to approval for ALS drugs by testing drugs simultaneously and adoptively. Given the challenges in developing treatments for this disease, the platform trial is anticipated to find an effective therapy more quickly, with fewer total participants and fewer participants on placebo.”
GlobalData forecasts that the ALS market will reach sales of approximately $1.3 billion in the eight major markets (US, France, Germany, Italy, Spain, UK, Japan, and Canada) by 2029, with $375.3 million being contributed by 12 late-stage agents, including pridopidine. Should pridopidine demonstrate statistically significant efficacy results in pivotal Phase III trials following the positive results of the Healey additional analysis presented at EAN, it could account for approximately $11.1 million in sales in 2029.
Pridopidine is an oral sigma-1 receptor (S1R) activator currently in Phase III for the treatment of ALS. Key opinion leaders (KOLs) previously interviewed by GlobalData highlight that pridopidine is likely to be positioned as the potential add-on drug of choice for bulbar onset ALS patients due to the potential positive effects of the mechanism on speech and bulbar function.
Dana comments, “The results presented at EAN showed significant benefits in definite, probable, and early (<18mo) ALS patients at 24 weeks follow-up for ALSFRS-R Total, ALSFRS-R respiratory, bulbar, quality of life, and quantitative speech characteristics.”
Beyond the Healey trial, Prilenia also enrolled its first patient in the National Institutes of Health (NIH)-sponsored Accelerating Access to Critical Therapies for ALS Act (ACT for ALS) expanded access protocol (EAP2) at the end of March 2024. Prilenia is planning to use the pre-specified and additional analysis data from the Healey Platform Trial to inform its pivotal Phase III trial to further evaluate the efficacy and safety of pridopidine beyond the ongoing EAP.
Dana concludes, “If the Phase III outcomes follow the positive outcomes from the Healey study, they will pave the way for pridopidine to become a key part of the treatment landscape for ALS patients with the bulbar onset form of the disease.”

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