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].

Summary of PTC Therapeutics Conference Call Company Overview - **Company**: PTC Therapeutics - **Sector**: Biotechnology - **Key Executives**: Matt Klein (CEO), Pierre Grave (CFO) Key Points Industry and Company Performance - PTC Therapeutics is experiencing a pivotal year with strong execution across all areas of the company, following a successful 2024 [2][3] - The company has submitted four FDA New Drug Applications (NDAs), with one approval received and three pending, including the Suppiance NDA expected to be approved within six weeks [3] Financial Position - As of Q1, PTC Therapeutics has over $2 billion in cash, providing a solid foundation for planned product launches and R&D advancements [3] Product Pipeline and Launches - Upcoming products include Suppiance for PKU and potential approvals for Friedreich ataxia and Translarna in the U.S. [3][4] - The company is preparing for the launch of Suppiance in Europe, with an early access program already initiated in Germany [23][24] Translarna in Europe - Despite the lack of marketing authorization renewal, over half of European countries wish to continue commercializing Translarna, allowing PTC to maintain 25-30% of European revenue, which is about 40% of overall Translarna revenue for 2024 [7][8][10] Market Opportunity for Suppiance - Suppiance is positioned as a highly differentiated therapy for PKU, addressing a significant unmet need as only 10-15% of the estimated 15,000-17,000 patients in the U.S. are currently on existing therapies [12][13] - The company plans to price Suppiance at a premium to existing therapies, with payer support anticipated [13][14] Commercial Strategy - PTC has a well-established commercial team experienced in rare disease therapies, which will facilitate the launch of Suppiance [14] - The company has identified 103 PKU expert centers in the U.S., many of which overlap with existing treatment centers, aiding in the transition to new therapies [17][18] Friedreich Ataxia Program - Vatiquinone is positioned as a therapy for both pediatric and adult patients, with a significant market opportunity due to its safety and tolerability profile [39][40] - The company expects to target pediatric patients first, followed by adults who may have had issues with existing therapies [41][42] Regulatory and Clinical Development - The PDUFA date for vatiquinone is August 19, with positive feedback from the FDA regarding the absence of an AdCom meeting [49] - PTC518 for Huntington's disease has shown promising Phase II results, with plans for accelerated approval discussions based on the data [51][55] Cash Management and Future Plans - PTC Therapeutics has a strong cash position, allowing for flexibility in internal development and potential business development activities [66][67] - The company aims to reach $2 billion in top-line revenue, with significant contributions expected from PKU and Friedreich ataxia products [67][68] Conclusion - PTC Therapeutics is well-positioned for growth with a robust pipeline, strong financials, and a strategic approach to market entry and commercialization across its product offerings. The company is focused on addressing unmet needs in rare diseases while maintaining a solid cash position to support its initiatives.

PTC Therapeutics June 2025 Matthew B. Klein, MD CEO Pam and Kelsey living with PKU 1 Corporate Presentation – June 2025 Forward Looking Statements This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this presentation, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC, including with respect to the ...

NEW YORK, May 09, 2025 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of PTC Therapeutics, Inc. (“PTC” or the “Company”) (NASDAQ: PTCT).   Such investors are advised to contact Danielle Peyton at newaction@pomlaw.com or 646-581-9980, ext. 7980.    The investigation concerns whether PTC and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices.  [Click here for information about joining the class action] On May 5, 2025 ...

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].

Financial Data and Key Metrics Changes - The company reported total product and royalty revenue of $190 million for Q1 2025, with a narrowed full-year revenue guidance of $650 million to $800 million [5][6][20] - The cash position at the end of Q1 was over $2 billion, providing resources for commercial and R&D efforts [5][10][20] Business Line Data and Key Metrics Changes - The DMD franchise generated $134 million in revenue, with Translarna contributing $86 million and Emflaza $48 million [12][20] - The company is preparing for the launch of SUFIANCE, with a positive CHMP opinion and expected broad label for PKU patients [6][16] Market Data and Key Metrics Changes - The company anticipates revenue from SUFIANCE in 2025, leveraging early access programs in Germany and other markets [6][37] - Approximately 58,000 addressable patients with PKU are expected to be targeted in the global launch [16] Company Strategy and Development Direction - The company aims to achieve cash flow breakeven through ramping up PKU commercial sales and pending FDA approvals [10][11] - Strategic focus on business development opportunities while maintaining a strong financial position [38] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing FDA review processes, indicating no expected delays [28][29] - The company is well-positioned to navigate global macro uncertainties, with minimal expected impact from tariffs and regulatory changes [97][99] Other Important Information - The company is actively engaging with healthcare providers and patients to prepare for the launch of SUFIANCE, emphasizing the importance of diet liberalization for PKU patients [17][31] - The FDA is conducting inspections for the Pitiquinone NDA, with no AdCom meeting planned, indicating a smooth review process [9][78] Q&A Session Summary Question: On SUFIANCE, what is the dynamic of patient inquiries? - Management noted that inquiries are coming from both therapy-naive patients and those who have tried other therapies, driven by social media engagement [24][25][29] Question: Expectations for SUFIANCE sales in Europe? - Management confirmed expectations for revenue in 2025 from early access programs in Germany and other countries [35][37] Question: Feedback from the nutritionist community regarding SUFIANCE? - Management highlighted the involvement of nutritionists in the launch plan, emphasizing their role in patient management [41][42] Question: Insights on Friedreich's ataxia and potential patient access? - Management indicated a significant unmet need for patients under 16 and expressed confidence in rapid uptake post-approval [80][82] Question: Impact of global macro factors on financial guidance? - Management stated minimal expected impact from tariffs and regulatory changes, maintaining confidence in their pricing strategy [97][99]

PTC Therapeutics (PTCT) Q1 2025 Earnings Call May 06, 2025 04:30 PM ET Speaker0 Ladies and gentlemen, thank you for standing by. Welcome to PTC Therapeutics First Quarter twenty twenty five Earnings Conference Call. All participants are in a listen only mode. After the presentation, there will be a question and answer session. Today's conference is being recorded. I would now like to turn the conference over to Ellen Cavallari, Head of Investor Relations. Please go ahead. Speaker1 Good afternoon and thank y ...

– Strong revenue performance of $190 million – – Positive CHMP opinion for Sephience™ (sepiapterin) received in April 2025, NDA review remains on track for July 29, 2025 PDUFA date – – Global Sephience launch activities progressing well – – Strong cash position of over $2.0 billion as of March 31, 2025 – WARREN, N.J., May 6, 2025 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ended March 31, 2025. "Following a year of out ...

PTC Therapeutics, Inc. PTCT on Monday announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington’s disease (HD) patients.Huntington’s disease is a progressive neurodegenerative disorder caused by mutation of the huntingtin (HTT) gene, leading to the breakdown of brain cells. It is characterized by uncontrolled movements (chorea), cognitive decline, and psychiatric disturbances.The study met its primary endpoint of reduction in blood Huntingtin (HTT) protein leve ...

This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this presentation, other than statements of historic fact, are forward-looking statements, including statements with respect to the future expectations, plans and prospects for PTC, PTC's strategy, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses and other matters, ...