PTC Therapeutics shares fall after Huntington’s study update
USA-based rare disease drug developer PTC Therapeutics (Nasdaq: PTCT) on Monday announced results from the Phase II PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disease (HD) patients.
The study met its primary endpoint of reduction in blood Huntingtin (HTT) protein levels (p<0.0001) at Week 12 and favorable safety and tolerability In addition, the 12-month data from the Stage 2 patients are consistent with the previously reported dose-dependent lowering of HTT protein and dose-dependent trends across clinical scales.
Despite meeting its primary endpoint and exhibiting a favorable safety profile, the market response was negative, reflecting skepticism about the drug’s potential for accelerated approval. PTC’s shares closed down 18.6% lower at $40.65 yesterday.
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