PTC Therapeutics, Inc. PTCT on Monday announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington’s disease (HD) patients.Huntington’s disease is a progressive neurodegenerative disorder caused by mutation of the huntingtin (HTT) gene, leading to the breakdown of brain cells. It is characterized by uncontrolled movements (chorea), cognitive decline, and psychiatric disturbances.The study met its primary endpoint of reduction in blood Huntingtin (HTT) protein leve ...

Overall, 159 subjects entered the study. As you can see, there are notable differences between Stage two and Stage three patients. Stage three patients on average are several years older and had more severe disease. Now the results. Pivot HD met its primary endpoint of blood total Huntington protein lowering at 12. PTC Therapeutics (PTCT) Update / Briefing May 05, 2025 08:00 AM ET Speaker0 Good morning and thank you for standing by. Welcome to the Pivot HD Top Line Results Conference Call. At this time, all ...