Prilenia Therapeutics B.V. and Ferrertoday announced the publication, in the journal Nature Medicine, of a manuscript entitled “Pridopidine in Early-Stage Manifest Huntington Disease: A Phase 3 Trial”ii. The publication describes data showing that treatment with pridopidine slowed clinical progression in Huntington’s disease (HD) patients not taking antidopaminergic medicines (ADMs).
The published data, from pre-defined analyses of a subgroup of patients not taking ADMs from pridopidine’s Phase 3 PROOF-HD trial, show that treatment with pridopidine achieved a clinically meaningful improvement from baseline for one year and slowing of decline thereafter, as measured by cUHDRS, with change vs placebo of 0.46, 0.45, 0.41 and 0.27 at 26, 39, 52 and 65 weeks (the end of the double-blind trial). Annual reductions in cUHDRS of 0.1–0.3 points have been associated with a clinically meaningful benefit in HDiii.
Importantly, the benefits in cUHDRS in this subgroup of subjects with HD were seen across domains of function, cognition, and motor performance. Pridopidine also achieved similar maintenance of cognition with no deterioration, as measured by Stroop Word Reading Test (SWR), and motor performance, as measured by Quantitative Motor (Q-Motor).
Ralf Reilmann, MD, FAAN, Founding Director, George Huntington Institute and publication lead author, 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.”
