PTC Therapeutics released top-line results from the 24-month interim analysis of Pivot-HD, reporting dose-dependent “favorable” effects on disease progression for stage 2 Huntington’s disease patients treated with votoplam versus an external natural-history cohort. The data come from the long-term extension portion of the phase II program. Votoplam (PTC-518) is an oral pre-mRNA splicing modifier given daily, with partner Novartis running an ongoing phase III study. PTC said the outcomes were consistent with prior signals from the original Pivot-HD trial, which met its primary endpoint of blood huntingtin lowering at 12 weeks. Investors will watch how the interim extension dataset informs the design and readouts of the ongoing late-stage program, especially for sustained progression measures beyond initial biomarkers.