PTC Therapeutics Says Interim Data For Huntington's Disease Candidate Shows Promise In Mid-Stage Study

PTC Therapeutics Inc PTCT shared interim data from the 12-week portion of the PIVOT-HD Phase 2 study of PTC518 in Huntington's disease (HD) patients. 

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.

PTC518 treatment was also well tolerated, with no treatment-related serious adverse events and 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.

PIVOT-HD is a global, placebo-controlled study and consists of two parts - an initial 12-week placebo-controlled phase focused on PTC518 pharmacology and pharmacodynamic effect, followed by a 9-month placebo-controlled portion. 

The study will initially include two dose levels, 5 milligrams and 10 milligrams, with the ability to include a third dose level of up to 20 milligrams.

Price Action: PTCT shares are up 4.05% at $43.63 on the last check Wednesday.