PTC Therapeutics Reports 52% Progression Slowing at 10 mg in 24-Month PIVOT-HD Study
PTC Therapeutics’ 24-month PIVOT-HD extension analysis showed Stage 2 participants had 52% and 28% slowing of disease progression at 10 mg and 5 mg doses versus natural history controls. Safety remained favorable with no treatment-related NfL increases, supporting Novartis’s launch of the global Phase 3 INVEST-HD study.
1. Interim Analysis Shows Dose-Dependent Slowing
PTC Therapeutics reported that Stage 2 participants in the PIVOT-HD extension study experienced a 52% reduction in disease progression at the 10 mg dose and a 28% reduction at the 5 mg dose on the Composite Unified Huntington’s Disease Rating Scale compared to a propensity-weighted natural history cohort.
2. Favorable Safety and Biomarker Findings
Safety data at 24 months showed no treatment-related increases in neurofilament light chain protein, with mean NfL levels remaining below baseline for both dose cohorts, reinforcing the favorable safety profile observed in earlier phases.
3. Global Phase 3 INVEST-HD Study Launch
Novartis has initiated a global Phase 3 INVEST-HD study enrolling approximately 770 early-stage Huntington’s disease patients randomized 3:2 to receive 10 mg of votoplam or placebo, with the primary endpoint of change in cUHDRS score through month 36.
4. Conference Call and Regulatory Outlook
PTC will host a conference call on April 28 at 4:30 pm ET to discuss the interim results and plans for regulatory interactions with Novartis, with a replay available on the company’s website for 30 days.