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PTC Therapeutics Reports Positive Topline Results from Month 24 Interim Analysis of PIVOT-HD Extension Study of Votoplam
PTC Therapeutics, Inc. (NASDAQ: PTCT) today reported positive topline results from the 24-month interim analysis of the PIVOT-HD long-term extension study, with favorable dose-dependent effects on disease progression for Stage 2 Huntington's disease (HD) patients following 24 months of votoplam treatment compared to an external natural history cohort.
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"- Dose-dependent benefit on cUHDRS in Stage 2 participants compared to matched natural history cohort, with 52% slowing at 10 mg dose -","length":135,"tagName":"p","attribs":{}},{"type":"text","content":"- Continued evidence of favorable safety profile - ","length":50,"tagName":"p","attribs":{}},{"type":"text","content":"- Novartis initiated global Phase 3 INVEST-HD study of votoplam -","length":65,"tagName":"p","attribs":{}},{"type":"text","content":"- PTC will host a conference call to discuss results today, April 28, at 4:30 pm ET - ","length":85,"tagName":"p","attribs":{}},{"type":"text","content":"WARREN, N.J., April 28, 2026 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today reported positive topline results from the 24-month interim analysis of the PIVOT-HD long-term extension study, with favorable dose-dependent effects on disease progression for Stage 2 Huntington's disease (HD) patients following 24 months of votoplam treatment compared to an external natural history cohort.","length":407,"tagName":"p"},{"type":"text","content":""These results give us confidence in the potential for votoplam to deliver long-term meaningful effect on slowing Huntington's disease progression," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. "In particular, the evidence of dose-dependent slowing of progression on the cUHDRS disease rating scale in the Stage 2 study participants supports the Novartis-initiated Phase 3 INVEST-HD study. We look forward to continuing to review the data and aligning on potential regulatory interactions based on the results with our partner Novartis."","length":588,"tagName":"p"},{"type":"text","content":"The PIVOT-HD study was a 12-month placebo-controlled study of two dose levels of votoplam in participants with Stage 2 and Stage 3 HD. The study met the primary endpoint of blood Huntingtin (HTT) protein lowering at 12 weeks, with persistent dose-dependent lowering at Month 12. PIVOT-HD participants then enrolled in the PIVOT-HD extension study in which those originally randomized to receive 5 mg or 10 mg of votoplam remained on those dose levels. Participants initially randomized to receive placebo were randomized to receive 5 mg or 10 mg. All participants and investigators remain blinded to initial PIVOT-HD treatment assignment. The ...