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PTC518 PIVOT-HD Study Achieves Primary Endpoint

- Study met primary endpoint with dose-dependent blood HTT protein lowering at Week 12 - - Favorable dose-dependent trends across clinical scales in Stage 2

articlePtc Therapeutics, Inc.May 5, 20253/company/ptc-therapeutics-inc/news/ptc518-pivot-hd-study-achieves-primary-endpoint-2025-05-05
PTC518 PIVOT-HD Study Achieves Primary Endpoint

About this update from Ptc Therapeutics, Inc.

[{"type":"text","content":"- Study met primary endpoint with dose-dependent blood HTT protein lowering at Week 12 -\n- Favorable dose-dependent trends across clinical scales in Stage 2 patients at Month 12 -\n- 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 -\n- Continued favorable safety and tolerability profile with no treatment-related NfL spikes -\n- PTC will host a conference call on May 5, 2025, at 8:00 am ET- \nWARREN, 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","length":1197,"tagName":"div"}]

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