WALTHAM, Mass.– Prilenia Therapeutics B.V. and Ferrer announced the presentation of five posters at the 2025 Huntington Study Group (HSG) Huntington’s Disease (HD) Clinical Research Congress, held October 10–13, 2025, showcasing new analyses demonstrating pridopidine’s potential to slow disease progression and improve multiple clinical outcomes in HD patients.
The new data include two-year follow-up analyses of patients not taking antidopaminergic medicines (ADMs) across both the double-blind and open-label phases of the PROOF-HD study. Findings demonstrated significant and clinically meaningful slowing of progression, with markedly less decline in function, cognition, and motor performance among pridopidine-treated patients compared with both placebo and propensity-matched natural history cohorts from Track-HD and Enroll-HD.
“The data show pridopidine’s ability to deliver consistent and sustained slowing of disease progression and significantly less decline across multiple endpoints of function, cognition and motor features, measured by scales including cUHDRS, TFC, SWR and Q-Motor, and compared to both placebo and two natural history cohorts, in HD patients not taking antidopaminergic medicines (ADMs),” said Michal Geva, Head of Research at Prilenia. “This is further evidenced by the Phase 3 data published in Nature Medicine showing a significant reduction in decline from baseline of 0.41 cUHDRS points at week 52 for pridopidine-treated patients compared to placebo (p = 0.035), with clinically meaningful differences extending to week 65 (0.27) and beyond.”
“It is clear that the HD community needs access to new therapies that improve multiple independent clinical endpoints that matter most to people with HD and their families, including function, disease progression, cognition and motor function,” said Oscar Pérez, Chief Scientific Officer at Ferrer. “We have a carefully designed roadmap aimed at paving the way to availability of pridopidine as an oral and easy to administer disease-modifying therapy capable of significantly slowing down clinical progression of HD.”
Prilenia and Ferrer plan to initiate a global confirmatory clinical trial of pridopidine in early-stage HD patients not taking ADMs in the first half of 2026. The trial will also include a small proof-of-concept arm of patients taking low-dose ADMs as per label guidance.
Pridopidine is an investigational, orally administered small-molecule sigma-1 receptor (S1R) agonist that crosses the blood-brain barrier and binds with high affinity across the brain. It has shown a favorable safety and tolerability profile in placebo-controlled trials and clinical experience involving more than 1,600 participants with follow-up extending up to seven years.
