PTC Therapeutics Reports Positive Phase II Huntington’s Disease Data, FDA Lifts Partial Clinical Hold

1. Positive Phase II Data: PTC Therapeutics announced positive interim results from the Phase 2 PIVOT-HD study of PTC518 in Huntington's disease patients, demonstrating dose-dependent reductions in mutant huntingtin protein levels and favorable clinical trends.
2. FDA Lifts Partial Clinical Hold: The FDA lifted the partial clinical hold on PTC518 after reviewing the PIVOT-HD data, allowing the program to continue.
3. Dose-Dependent Reductions: At the 12-month mark, PTC518 treatment led to dose-dependent reductions in mutant huntingtin protein levels in both blood and cerebrospinal fluid (CSF), with 22% and 43% reductions for 5mg and 10mg dose levels, respectively.
4. Clinical Trends: The study showed encouraging trends on several HD-relevant clinical assessments, including the Total Motor Score (TMS) and Composite Unified Huntington's Disease Rating Scale (cUHDRS).
5. Safety and Tolerability: PTC518 continued to be safe and well tolerated after 12 months of treatment.
6. Conference Call and Webcast: PTC Therapeutics hosted a conference call and webcast to discuss the findings and provide further details.