PTC Therapeutics, Inc. (PTCT) said it will present new long-term results from its PIVOT-HD study, offering an updated look at how its experimental Huntington's disease treatment Votoplam. The company will discuss the 24-month interim results during a webcast later today.Company to Review 24-Month Findings
PTC will host a call at 4:30 p.m. ET to outline how patients have responded to Votoplam after two years in the long-term extension study. Its partner Novartis recently said the data support the ongoing Phase 3 INVEST-HD trial and will help guide next steps, including potential FDA discussions.
About the PIVOT-HD Study
PIVOT-HD began as a 12-month, placebo- controlled trial evaluating two doses of Votoplam - 5mg and 10 mg, in Stage 2 and later Stage 3 Huntington's disease patients. The study measured reductions in the Huntingtin (HTT) protein, safety outcomes, and changes in disease-related biomarkers. After the first year, all participants could join the long-term extension, where everyone received Votoplam while remaining blinded to their original treatment group.
About Votoplam
Votoplam (formerly PTC518) is a small-molecule therapy designed to lower production of the mutant Huntingtin protein by modifying RNA splicing. The drug comes from PTC's splicing platform, the same technology behind Evrysdi for spinal muscular atrophy. Novartis partnered on the program in 2024 and now leads development and commercialization.
PTCT has traded between $35.95 and $87.50 over the past year. The stock is currently trading at $67.63, down 2.30%.
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