PTC Therapeutics shares positive 24-month data on votoplam for Huntington's disease, supporting Novartis Phase 3 trial.

PTC Therapeutics announced results from the 24-month interim analysis of the PIVOT-HD long-term extension study of votoplam, a drug targeting Huntington's disease. The data supports the ongoing Novartis Phase 3 INVEST-HD study, with both companies planning next steps including potential FDA actions. Votoplam works by reducing toxic Huntingtin protein levels linked to the disease, which currently has no cure. This progress is significant as it may lead to new treatment options for patients with this fatal genetic disorder.