PTC Therapeutics Says FDA Lifts Clinical Hold On Investigational Study In Patients With Rare Weak Nerve Cell Disorder

Thursday, PTC Therapeutics Inc PTCT shared interim results from the Phase 2 PIVOT-HD study of PTC518 in Huntington’s disease (HD) patients.

HD is an inherited condition in which nerve cells in the brain break down over time, resulting in progressive movement, thinking (cognitive), and psychiatric symptoms.

At Month 12, PTC518 treatment resulted in dose-dependent lowering of mutant huntingtin (mHTT) protein in the blood and cerebrospinal fluid (CSF) in the interim cohort of patients.

Related: PTC Therapeutics Says Interim Data For Huntington’s Disease Candidate Shows Promise In Mid-Stage Study.

In addition, favorable trends were demonstrated on several relevant HD clinical assessments, including Total Motor Score (TMS) and Composite Unified Huntington’s Disease Rating Scale (cUHDRS).

Furthermore, after 12 months of treatment, PTC518 remains safe and well tolerated.

“With these data in hand, we look forward to the next steps in the PTC518 development program,” said Dr. Matthew Klein, CEO of PTC Therapeutics

At Month 12, the durability of dose-dependent mHTT lowering in the blood was demonstrated with a lowering of 22% and 43% for 5mg and 10mg doses, respectively.

In the CSF, dose-dependent mHTT lowering was also demonstrated, with lowering of 21% and 43% for 5mg and 10mg dose levels, respectively.

In addition, at Month 12, PTC518 treatment resulted in a notable slowing in the progression of motor symptoms as assessed by the TMS (2.0 points worsening for 5mg and 1.3 points worsening for 10mg vs. 4.9 points worsening for placebo).

Also, PTC announced that the FDA lifted the partial clinical hold on the program based on a review of the PIVOT-HD data.

Related: PTC Therapeutics Pauses Enrollment In Huntington’s Candidate Trial In US.

Price Action: PTCT shares are down 4.10% at $34.16 at last check Thursday.

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