WALTHAM, Mass.– Prilenia Therapeutics B.V. and Ferrer announced that Nature Medicine has published results from the Phase 3 PROOF-HD trial of pridopidine in early-stage Huntington’s disease (HD). The data highlight that treatment with pridopidine slowed clinical progression and maintained function, cognition, and motor performance in a subgroup of patients not taking antidopaminergic medicines (ADMs).
While the study did not meet its primary or secondary endpoints in the full patient population, analyses of patients not on ADMs demonstrated clinically meaningful improvement from baseline for one year and a slowing of decline thereafter. Benefits were observed across multiple domains, measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS), the Stroop Word Reading Test (SWR) for cognition, and Quantitative Motor (Q-Motor) for motor function.
Ralf Reilmann, M.D., FAAN, Founding Director of the George Huntington Institute and lead author of the publication, said: “The published data represents the first Phase 3 HD trial to deliver consistent and meaningful benefits on progression across multiple clinical domains of HD such as function, cognition and motor performance, while also confirming pridopidine’s favorable safety and tolerability profile. Upcoming studies can now refine patient selection and account for the impact of ADM exposure, which obscured the true drug-related benefits. Appropriate stratification and dosage strategies will control for this confounding factor and allow demonstration of pridopidine’s positive treatment effects on clinical progression of symptoms. I would like to express my gratitude for the continued commitment of everyone working to support the next data-driven steps to making this well-tolerated and easily administered treatment option available to HD patients.”
Dina de Sousa, European Huntington Association Board member, added: “We have no options to help slow down our decline. Nothing to help people feed themselves a little longer, button a shirt a little longer, walk a little longer, or maybe even dance a little longer. Treatment options are needed now that can enable maintenance of independence for as long as possible. These results provide hope that there are therapies that can go further than just symptom control, and hope that we can take a step forward toward availability of a disease-modifying treatment able to slow down the inexorable march of this dreadful disease.”
“These data provide a clear path forward for next year’s planned global confirmatory study in early HD patients, aimed at confirming pridopidine’s effect and supporting ongoing global regulatory discussions,” said Michael R. Hayden, CEO of Prilenia.
Oscar Pérez, Chief Scientific Officer at Ferrer, noted: “Nature Medicine is one of the world’s leading peer-reviewed medical journals, and this important publication adds to the weight of evidence in support of the sigma-1 receptor agonist approach and the development of pridopidine for the treatment of neurodegenerative diseases such as HD and ALS.”
Prilenia said a confirmatory study designed to validate pridopidine’s effects and advance regulatory approval discussions is expected to begin next year.
