On 28 April 2026, PTC Therapeutics hosted a webcast outlining results of the 24-month interim analysis of the PIVOT-LTE long-term extension study of votoplam (formerly PTC518). Following the 12-month randomised placebo-controlled PIVOT-HD trial, participants were eligible to continue to the long-term extension. Participants already receiving 5 mg or 10 mg of votoplam continued with the same dose, and those on placebo were randomised to either active dose. The data suggest dose-dependent slowing of disease progression, as measured by cUHDRS at 24 months in HD-ISS Stage 2 participants compared with a matched natural history cohort from Enroll-HD. No treatment-related spikes in NfL were observed at 24 months, and mean NfL levels in Stage 2 participants were reported to be lower than baseline levels.

For details of all ongoing and completed EHDN-endorsed studies, click here.