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PTC Therapeutics Reports Favorable Interim Results in Huntington's Disease Study; US FDA Lifts Clinical Hold
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PTC Therapeutics Reports Favorable Interim Results in Huntington's Disease Study; US FDA Lifts Clinical Hold
Jun 20, 2024 6:21 AM

08:53 AM EDT, 06/20/2024 (MT Newswires) -- PTC Therapeutics ( PTCT ) said Thursday that the US Food and Drug Administration has lifted the partial clinical hold on its PTC518 program following favorable interim results from the phase 2 PIVOT-HD study in Huntington's disease patients.

The company said that after 12 months, PTC518 showed dose-dependent reductions in mutant huntingtin protein in blood and cerebrospinal fluid, alongside positive trends in clinical assessments like Total Motor Score and Composite Unified Huntington's Disease Rating Scale. The drug candidate also maintained a good safety profile, according to PTC.

At the 12-month mark, the 5mg and 10mg doses of PTC518 reduced mutant huntingtin protein in blood by 22% and 43%, and in cerebrospinal flued by 21% and 43%, respectively. The drug candidate also slowed the progression of motor symptoms, with lesser worsening scores compared with placebo, the company said.

Shares of the company were up over 3% in recent Thursday premarket activity.

Price: 36.80, Change: +1.18, Percent Change: +3.31

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