PTC Therapeutics, Inc. (NASDAQ: PTCT) has announced interim results from the phase 2 pivot-hd study of PTC518 in Huntington’s disease (HD) patients. At month 12, PTC518 treatment resulted in notable changes in various metrics.
The interim results demonstrated a dose-dependent lowering of mutant huntingtin (mhtt) protein in the blood and cerebrospinal fluid (CSF) in the cohort of patients. The durability of dose-dependent mhtt lowering in the blood was shown with lowering of 22% and 43% for 5mg and 10mg dose levels, respectively. In the CSF, dose-dependent mhtt lowering was also demonstrated with lowering of 21% and 43% for 5mg and 10mg dose levels, respectively.
Furthermore, the treatment resulted in a notable slowing in the progression of motor symptoms as assessed by the total motor score (TMS), with 2.0 points worsening for the 5mg dose and 1.3 points worsening for the 10mg dose, compared to 4.9 points worsening for the placebo.
The company also announced that the FDA has lifted the partial clinical hold on the program based on a review of the pivot-hd data.
Huntington’s disease is a rare, hereditary, genetic disorder of the central nervous system caused by a defective gene that produces a toxic and abnormal huntingtin protein, leading to neuron damage and death.
PTC Therapeutics, Inc. is a global biopharmaceutical company focused on the discovery, development, and commercialization of clinically differentiated medicines for patients with rare disorders.
For further details, PTC will hold a conference call at 8:00 am EDT to discuss the news. Following these announcements, the company's shares moved -6.6%, and are now trading at a price of $33.27. For more information, read the company's full 8-K submission here.
