PTC Therapeutics announced on May 5, 2025, 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 dose-dependent reduction in blood Huntingtin (HTT) protein levels (p<0.0001) at Week 12, with durable lowering observed at Month 12.
For Stage 2 patients, the study demonstrated favorable dose-dependent trends across clinical scales, including the Composite Unified Huntington's Disease Rating Scale (cUHDRS) and Total Motor Score (TMS) subscale. However, for Stage 3 patients, trends favored the 5mg dose group relative to placebo but not the 10mg dose group, suggesting potential differences in treatment effect based on disease stage.
PTC518 maintained a favorable safety and tolerability profile across all dose levels and disease stages, with no treatment-related serious adverse events or neurofilament light chain protein (NfL) spikes. The company plans to engage with the FDA to discuss next development and regulatory steps, including potential accelerated approval pathways.
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