- Study met primary endpoint with dose-dependent blood HTT protein lowering at Week 12 -
- Favorable dose-dependent trends across clinical scales in Stage 2 patients at Month 12 -
- Signals of dose-dependent clinical benefit relative to matched natural history cohort as well as dose-dependent lowering of NfL in Stage 2 patients at Month 24 -
- Continued favorable safety and tolerability profile with no treatment-related NfL spikes -
- PTC will host a conference call on May 5, 2025, at 8:00 am ET-
WARREN, N.J., May 5, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disease (HD) patients. The study met its primary endpoint of reduction in blood Huntingtin (HTT) protein levels (p