PTC Therapeutics to Share 24-Month PIVOT-HD Extension Data Supporting Phase 3 INVEST-HD
PTC Therapeutics will share 24-month interim results from its PIVOT-HD long-term extension study of votoplam on April 28, reinforcing Novartis’s initiation of the INVEST-HD Phase 3 trial. The companies plan to discuss next development steps with the FDA based on these supportive data.
1. Webcast Conference Call for 24-Month Data
PTC Therapeutics will host a webcast on April 28 at 4:30 p.m. ET to present the 24-month interim analysis from the long-term extension of the PIVOT-HD study evaluating votoplam in Huntington’s disease patients. The live call will be available on the company’s Investors page, with a 30-day replay following the event.
2. Phase 3 INVEST-HD Initiation and FDA Strategy
Novartis has already initiated the INVEST-HD Phase 3 trial, citing supportive long-term extension data from PIVOT-HD. Novartis and PTC will assess optimal next steps with the FDA, potentially including regulatory filings or accelerated approval pathways based on these interim results.
3. PIVOT-HD Study Design and Extension
Originally a 12-month placebo-controlled trial of 5 mg and 10 mg votoplam in Stage 2 patients, PIVOT-HD added a Stage 3 cohort to refine patient selection. After 12 months, all participants entered a blinded extension receiving active drug at their initial dose or randomized if they began on placebo.
4. Votoplam Mechanism and Partnership
Votoplam is a small molecule splicing modifier designed to induce HTT mRNA degradation by promoting inclusion of a pseudoexon with a premature stop codon. Discovered by PTC and partnered with Novartis in December 2024, Novartis now leads development, manufacturing and potential commercialization.