Core Viewpoint - The Schall Law Firm is investigating PTC Therapeutics, Inc. for potential violations of securities laws related to misleading statements and failure to disclose important information to investors [1][2]. Group 1: Investigation Details - The investigation centers on whether PTC issued false or misleading statements regarding the results of the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Huntington's disease patients [2]. - PTC announced that the study met its primary endpoint of reducing blood Huntingtin (HTT) protein levels with a significant p-value of less than 0.0001 at Week 12, along with favorable safety and tolerability [2]. - Analysts suggest that a Phase 3 study is necessary to confirm if the treatment effectively slows the progression of Huntington's disease, leading to a significant drop in PTC's share price by over 18.6% on the announcement day [2].

Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices involving PTC Therapeutics, Inc. and its officers or directors [1] Group 1: Company Developments - On May 5, 2025, PTC announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disease patients, which met its primary endpoint of reducing blood Huntingtin protein levels with a p-value of less than 0.0001 at Week 12 [3] - Analysts suggest that PTC may need to conduct a Phase 3 study to confirm whether the treatment effectively slows Huntington's disease [3] Group 2: Market Reaction - Following the announcement of the study results, PTC's stock price dropped by $9.30 per share, or 18.62%, closing at $40.65 per share on May 5, 2025 [4]

Core Viewpoint - PTC Therapeutics, Inc. is under investigation for potential securities fraud and unlawful business practices following the announcement of Phase 2 study results for its treatment of Huntington's disease, which led to a significant drop in stock price [1][2][3]. Group 1: Company Developments - PTC announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disease patients, achieving its primary endpoint of reducing blood Huntingtin protein levels with a p-value of less than 0.0001 at Week 12 [2]. - Despite meeting the primary endpoint, analysts suggest that a Phase 3 study may be necessary to confirm the treatment's efficacy in slowing the progression of Huntington's disease [2]. Group 2: Market Reaction - Following the announcement of the study results, PTC's stock price fell by $9.30 per share, representing an 18.62% decline, closing at $40.65 per share on May 5, 2025 [3].

Core Insights - PTC Therapeutics announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) for Huntington's disease, achieving its primary endpoint of reducing blood HTT protein levels with significant statistical significance [1][2] Study Results - The study demonstrated a reduction in blood HTT protein levels of 23% at the 5mg dose and 39% at the 10mg dose for Stage 2 patients, while Stage 3 patients showed a 36% reduction at the 10mg dose [4] - The treatment exhibited favorable safety and tolerability across all dose levels and disease stages, with no serious adverse events reported [5] Clinical Implications - There were observed dose-dependent trends in clinical scales for Stage 2 patients, including improvements in the Composite Unified Huntington's Disease Rating Scale (cUHDRS) and Total Motor Score (TMS) [4] - For Stage 3 patients, the 5mg dose showed favorable trends compared to placebo, indicating potential differences in treatment effects between stages [5] Long-term Data - 24-month treatment data indicated dose-dependent trends on cUHDRS, Total Function Capacity (TFC), and Symbol Digit Modalities Test (SDMT) subscales, with a notable lowering of plasma NfL levels at both 5mg and 10mg doses [6] Market Reaction - Following the announcement, PTC Therapeutics' stock fell by 18.60% to $40.65, reflecting investor concerns regarding the differing treatment effects observed in Stage 3 patients [5][7]