Hailshadow/iStock via Getty Images PTC Therapeutics ( NASDAQ: PTCT ) announced Thursday that the U.S. FDA lifted the partial clinical hold on its Phase 2 PIVOT-HD study for its experimental oral therapy PTC518 against the movement disorder Huntington's disease.
The decision followed encouraging interim data from the global study, the advancement of which had been paused in the U.S. pending additional data.
Citing 12-week results from PIVOT-HD, the New Jersey-headquartered biotech said PTC518 at 5 mg and 10 mg doses led to a 22% and 43% decline in mutant huntingtin (mHTT) protein in the blood and brain-related cerebrospinal fluid, respectively. According to PTC ( PTCT ), the patients also experienced positive clinical effects based on metrics such as Total Motor Score (TMS) and Composite Unified Huntington's Disease Rating Scale (cUHDRS). After 12 months of therapy, they continued to tolerate PTC518 with no safety concerns, the company added.
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