Clinical TrialApr 28, 2026, 04:33 PM
PTCT Votoplam PIVOT-HD Study Shows 52% Slowing in HD Progression
AI Summary
PTC Therapeutics announced positive topline results from the 24-month interim analysis of the PIVOT-HD long-term extension study for votoplam in Huntington's disease. The study demonstrated a favorable dose-dependent slowing of disease progression in Stage 2 participants, with a 52% slowing at the 10 mg dose and 28% at the 5 mg dose compared to a natural history cohort. The safety profile remained favorable, and Novartis has initiated the global Phase 3 INVEST-HD study for votoplam, which will enroll approximately 770 early-stage HD patients.
Key Highlights
- PTCT reported positive topline results from 24-month interim analysis of PIVOT-HD extension study for votoplam.
- Votoplam showed dose-dependent benefit on cUHDRS in Stage 2 participants.
- 52% slowing of disease progression observed at 10 mg dose in Stage 2 participants.
- 28% slowing of disease progression observed at 5 mg dose in Stage 2 participants.
- Favorable safety profile for votoplam was maintained at 24 months.
- Novartis initiated global Phase 3 INVEST-HD study for votoplam.
- INVEST-HD study will enroll approximately 770 individuals with early-stage HD.
- Primary endpoint for INVEST-HD is change from baseline up to month 36 in cUHDRS.