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 (PTCT) could be a solid choice for investors given its recent upgrade to a Zacks Rank #2 (Buy). An upward trend in earnings estimates -- one of the most powerful forces impacting stock prices -- has triggered this rating change.The Zacks rating relies solely on a company's changing earnings picture. It tracks EPS estimates for the current and following years from the sell-side analysts covering the stock through a consensus measure -- the Zacks Consensus Estimate.The power of a changing ear ...

PTC Therapeutics (PTCT) FY Conference June 03, 2025 10:20 AM ET Speaker0 Started, I'm obligated to direct everyone to WilliamBlair.com for a full list of disclosures. I'm very pleased to introduce doctor Matt Klein, the CEO of PTC Therapeutics, who's going to provide an overview of the company. Thanks, Matt. Speaker1 Thank you, Sammy. It's great to be here and be able to provide this update on PTC Therapeutics. Before I get started, I refer you to our forward looking statements, including those listed in ou ...

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 ...

Summary of Conference Call Company and Industry - **Company**: PTC Therapeutics - **Industry**: Biotechnology, specifically focusing on treatments for neurodegenerative diseases and metabolic disorders such as Huntington's disease and Phenylketonuria (PKU) Key Points and Arguments Huntington's Disease Program 1. **Phase II Study Results**: The Phase II study for Huntington's disease met primary endpoints, demonstrating target engagement and safety, which supports moving to Phase III trials [3][4][11] 2. **Biomarker Data**: Data showed dose-dependent lowering of neurofilament light chain (NfL), indicating neuroprotection, and early signs of clinical effect were observed at 24 months [4][11] 3. **Regulatory Discussions**: Ongoing discussions with the FDA regarding the potential for accelerated approval based on Huntington protein lowering as a surrogate marker [10][15] 4. **KOL Feedback**: Key opinion leaders (KOLs) have responded positively, indicating strong support for the drug's safety and efficacy in lowering Huntington's protein [5][6] 5. **Future Steps**: Plans to gather additional data from ongoing studies to support an accelerated approval application while preparing for an efficacy trial [12][16] PKU Market and Product Launch 1. **Regulatory Progress**: Product discussions with the FDA have been productive, with expectations for timely approval ahead of the PDUFA date [19][20] 2. **Commercial Infrastructure**: The company has redeployed its existing sales force and added case managers to support patient and physician engagement [22][23] 3. **Market Strategy**: Targeting specialty centers and understanding KOL prescribing habits to maximize market penetration [25][30] 4. **Patient Engagement**: High enthusiasm from the patient community for the new treatment, emphasizing diet liberalization benefits [28][37] 5. **Launch Dynamics**: Anticipated rapid uptake post-approval, although throughput at treatment centers may limit initial patient access [34][36] Vutiquinone Regulatory Process 1. **Mid-Cycle Meeting**: The FDA does not expect an advisory committee meeting, and the review process is progressing well [38][39] Capital Allocation and Business Development 1. **Strong Financial Position**: The company has a balance sheet of approximately $2 billion, allowing for strategic investments without immediate pressure [40][41] 2. **Business Development Opportunities**: Actively looking for late-stage commercial and development-stage assets to complement internal pipeline [42][45] 3. **European Market Strategy**: Plans to maintain a narrow price corridor and leverage early access programs in Germany to facilitate market entry [47][48] Other Important Content - **Regulatory Environment**: The current administration is perceived to be open to accelerated approval pathways for neurodegenerative diseases, which may benefit PTC's programs [14][15] - **Market Segmentation**: The company is aware of different patient segments and is prepared to address the needs of classical PKU patients who have not previously received treatment [31][32] - **Long-term Market Potential**: The European market is expected to represent about 50% of the overall opportunity, with strategic pricing and market entry plans in place [47][48]

Summary of PTC Therapeutics Conference Call Company Overview - **Company**: PTC Therapeutics (PTCT) - **Industry**: Biotechnology, focusing on rare diseases and gene therapies Key Points and Arguments Executive Order and Market Position - The recent executive order has unclear ramifications for companies, but PTC Therapeutics expects minimal impact due to its revenue being more international than domestic [4][5] - The company is preparing for the launch of its PKU product, emphasizing a global pricing corridor to maintain product value [5][6] FDA Interactions - PTC has three applications pending with the FDA, with no significant impact from recent agency changes noted [10][12] - The PKU New Drug Application (NDA) is progressing well, with confidence in approval by the PDUFA date [11][12] Translarna Sales in Europe - Despite a negative opinion from the CHMP, PTC is pursuing a country-by-country strategy to commercialize Translarna, with about 50% of countries showing interest [16][17] - The company anticipates achieving 25-30% of European revenue for the year from Translarna [18][20] PKU Launch Strategy - PTC is ready for a global launch of its PKU product, with an early access program initiated in Germany [33][34] - The company has built a robust infrastructure to support the launch, including a patient services team to assist with prior authorizations and patient engagement [38][39] Competitive Landscape for PKU - There are approximately 17,000 individuals with PKU in the US, with a significant unmet need as 85-90% are not on current therapies [44][45] - PTC's PKU therapy is positioned as a better option compared to existing treatments, with a focus on diet liberalization and improved tolerability [47][50] Pricing Strategy - PTC plans to price its PKU therapy at a premium to Palynziq, justified by strong efficacy data and a favorable safety profile [54][55] - The company believes it could achieve a billion-dollar opportunity in the US alone with low double-digit market penetration [57][58] PTC 518 and Huntington's Disease - PTC 518 is positioned as a potential first-ever disease-modifying therapy for Huntington's disease, with promising early data [63][64] - The company is focused on understanding the optimal patient population for this therapy, emphasizing the importance of early-stage patients [66][68] Competitive Landscape for Huntington's Disease - PTC believes that its oral therapy will be more attractive than gene therapies due to its safety, tolerability, and ability to be administered early in the disease process [78][79] - The partnership with Novartis is expected to enhance commercialization efforts for PTC 518 [76][79] Additional Important Content - The company is optimistic about the global opportunity for its therapies, with plans for launches in Japan and other markets [59][61] - PTC is committed to addressing unmet needs in rare diseases, which is a key focus for its product development strategy [32][33]

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]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☑ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-35969 PTC Therapeutics, Inc. (Exact name of registrant as specified in its charter) Delaware 04-3416587 (Stat ...

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 ...

Revenue and Financial Performance - Total net product and royalty revenue for Q1 2025 was $190 million, compared to $208.8 million in Q1 2024[8]. - Revenue from the DMD franchise in Q1 2025 was $134 million, with Translarna™ generating $86 million and Emflaza® generating $48 million[4]. - Net income for Q1 2025 was $866.6 million, compared to a net loss of $91.6 million in Q1 2024[8]. - PTC anticipates full-year 2025 revenue to be between $650 million and $800 million, including in-line products and potential new product launches[8]. - Cash, cash equivalents, and marketable securities totaled $2.03 billion as of March 31, 2025, up from $1.14 billion at the end of 2024[8]. - Full-year 2025 GAAP R&D and SG&A expenses are projected to be between $805 million and $835 million[8]. Research and Development - GAAP R&D expenses for Q1 2025 were $109 million, down from $116 million in Q1 2024[8]. - PTC518 Phase 2 study met primary endpoint of dose-dependent blood HTT lowering at Week 12, with favorable safety and tolerability[4]. - The NDA for pediatric and adult patients with Friedreich's ataxia has been accepted and granted Priority Review by the FDA, with a target action date of August 19, 2025[4]. Regulatory and Market Approvals - Positive CHMP opinion for Sephience™ received on April 25, 2025, with an expected EC adoption in approximately two months[4]. - PTC's marketing authorization for Translarna in Brazil, Russia, and other regions is under scrutiny due to potential risks in pricing and reimbursement negotiations[21]. - The company is relying on results from Study 041, an 18-month clinical trial, to support marketing approval for Translarna in the United States[21]. Collaborations and Commercialization - PTC has a collaboration agreement with Novartis, which includes development, regulatory, and sales milestone payments, as well as profit sharing and royalties[21]. - Expectations for Upstaza/Kebilidi include commercialization and achieving sales milestones, with potential contingent payments[21]. - The commercialization of Evrysdi under PTC's SMA collaboration is anticipated, along with Tegsedi and Waylivra[21]. Risks and Strategic Considerations - PTC faces significant risks in the development and regulatory approval of its products, including Translarna, Emflaza, and vatiquinone[23]. - The company emphasizes the importance of considering various risk factors that could impact its business and financial performance[21]. - PTC's ability to secure adequate financing for operating expenses and capital expenditures is critical for its future operations[21]. - The eligible patient base and commercial potential of PTC's products are key considerations for the company's growth strategy[21]. - PTC does not guarantee that any of its products will achieve commercial success or maintain regulatory approval[23].