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Interim PIVOT-HD Results Demonstrate Evidence of Favorable CNS Biomarker and Clinical Effects at Month 12 in Huntington's Disease Patients
- FDA lifts PTC518 partial clinical hold based on PIVOT-HD data - - Conference call and webcast to be held June 20th at 8:00 am EDT - WARREN, N.J., June 20,
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"- FDA lifts PTC518 partial clinical hold based on PIVOT-HD data -\n- Conference call and webcast to be held June 20th at 8:00 am EDT - \nWARREN, N.J., June 20, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared interim results from the Phase 2 PIVOT-HD study of PTC518 in Huntington's disease (HD) patients. 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. 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, following 12 months of treatment, PTC518 continues to be safe and well tolerated.\n\n\"The evidence of both CNS biomarker and early clinical effects at Month 12 along with the continued favorable tolerability profile supports the promise of PTC518 to address the need for an effective and safe disease-modifying therapy for patients living with Huntington's disease,\" said Dr. Matthew B. Klein, Chief Executive Officer, PTC Therapeutics, Inc. \"With these data in hand, we look forward to the next steps in the PTC518 development program.\"\nAt Month 12, durability of dose-dependent mHTT lowering in the blood was demonstrated 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. In addition, at Month 12, PTC518 treatment resulted in a notable slowing in 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).\nIn addition, PTC announced that the FDA has lifted the partial clinical hold on the program based on review of the PIVOT-HD data.\nToday's Conference Call and Webcast PTC will hold a conference call at 8:00 am EDT today to discuss this news. To access the call by phone, please click here to register and you will be provided with dial-in details. To avoid delays, we recommend participants dial in to the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investor section of the PTC website at https://ir.ptcbio.com/events-...