PTC Therapeutics lifted by promising Huntington's trial

22 June 2023

Promising clinical results from the 12-week portion of the PIVOT-HD Phase II study of PTC518 in Huntington's disease (HD) lifted US pharma group PTC Therapeutics (Nasdaq: PTCT). The stock closed up 2.7% at $43.06 yesterday.

The study demonstrated dose-dependent lowering of Huntingtin (HTT) protein levels in peripheral blood cells, reaching a mean 30% reduction in mutant HTT levels at the 10mg dose level. In addition, PTC518 exposure in the cerebrospinal fluid (CSF) was consistent with or higher than plasma unbound drug levels.

The New Jersey, USA-based rare disease drug developer said PTC518 treatment was well tolerated, with no treatment-related serious adverse events, no reports of peripheral neuropathy or dose-limiting toxicities. Furthermore, there were no CSF neurofilament light chain protein (NfL) treatment-related spikes, with an overall trend towards lowering of CSF NfL levels following 12 weeks of PTC518 treatment.

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