80% of PKU centers of excellence in the U.S. had written prescriptions for one or more patientsSephience contributed $92 million of revenue in the fourth quarter and $111 million worldwide since launch in 2025. Pauwels said fourth-quarter Sephience revenue was approximately $92 million, including $81 million in the U.S. and $11 million ex-U.S.For the fourth quarter, PTC reported total net product and royalty revenue of $263 million . Full-year 2025 total net product and royalty revenue was $831 million , wh ...

PTC Therapeutics (NasdaqGS:PTCT) Q4 2025 Earnings call February 19, 2026 04:30 PM ET Company ParticipantsEllen Cavaleri - Head of Investor RelationsEric Pauwels - Chief Business OfficerJarwei Fang - VP of Large-Cap Biotechnology and Pharma Equity ResearchJoe Heidt - Equity Research AssociateMatthew Klein - CEOPierre Gravier - CFOTazeen Ahmad - Managing DirectorConference Call ParticipantsBen Burnett - Executive Director and Senior Equity AnalystBrian Cheng - Executive Director and Senior Biotech AnalystElli ...

Summary of PTC Therapeutics FY Conference Call Company Overview - **Company**: PTC Therapeutics (NasdaqGS:PTCT) - **Industry**: Biotechnology, focusing on rare diseases and innovative therapies Key Points and Arguments 2025 Performance Highlights - **Objectives Achieved**: Successfully gained approvals for Safiyance in multiple regions, including the US, Europe, and Japan, within six months [2][3] - **Revenue Performance**: Total revenue for 2025 was $823 million, surpassing guidance of $750 million-$800 million. Product revenue was $588 million, driven by Safiyance and mature products [4] - **Safiyance Launch**: Generated $112 million in total revenue since launch, with 946 patients on commercial therapy worldwide [4][5] Safiyance Product Insights - **Mechanism of Action**: Safiyance has a dual mechanism that benefits a wide range of PKU patients, including those with severe forms of the disease [7][8] - **Clinical Efficacy**: In a head-to-head study, Safiyance showed over 70% greater reduction in phenylalanine levels compared to BH4 [9] - **Dietary Impact**: 69% of participants in a long-term study were able to increase protein intake while maintaining control of phenylalanine levels [10] - **Market Potential**: The total addressable market for PKU patients in the US is approximately 17,000, with significant unmet needs despite existing therapies [11][12] 2026 Outlook - **Revenue Guidance**: Expected revenue for 2026 is $700-$800 million, primarily from Safiyance, reflecting a 19%-36% year-over-year growth [5][6] - **Operational Expenses**: Operating expenses are projected to be $680-$720 million, indicating a potential path to cash flow breakeven in 2026 [6] - **Focus Areas**: Continued emphasis on Safiyance's global launch, Vodaplam for Huntington's disease, and advancing early-stage R&D programs [6][14] Vodaplam and Other Programs - **Vodaplam Development**: Leading oral disease-modifying therapy for Huntington's disease, with positive results from the PIVOT-HD phase 2 study [14][15] - **FDA Engagement**: Alignment reached with FDA for phase 3 study, with potential for accelerated approval due to significant unmet needs [16][41] - **Vatiquinone for Friedreich's Ataxia**: Demonstrated significant effects on disease progression, with ongoing discussions with FDA for next steps [17][18] R&D and Future Innovations - **RNA Splicing Platform**: PTC's proprietary platform is expected to yield new therapies and strategic partnerships in various therapeutic areas [19][20] - **Upcoming Programs**: Plans to advance several innovative programs targeting diseases like sickle cell disease and neurodegenerative disorders [20] Market Strategy and Global Expansion - **Geographical Focus**: Expansion plans include launching Safiyance in Japan, Brazil, and other countries, leveraging early access programs [12][39] - **Payer Mix**: Current payer mix for Safiyance is approximately 70% commercial, with expectations to move towards a more balanced mix over time [34] Challenges and Considerations - **DMD Franchise Erosion**: Anticipated continued revenue decline in the DMD franchise due to increased generic competition [28][29] - **Market Dynamics**: Ongoing assessment of market conditions and revenue guidance adjustments based on performance and insights throughout the year [30][31] Additional Important Insights - **Patient Engagement**: High prescription refill rates and low discontinuation rates indicate strong patient adherence to Safiyance therapy [4][5] - **Social Impact**: Positive patient stories highlight the transformative potential of Safiyance in improving quality of life for individuals with PKU [10][11] This summary encapsulates the key points discussed during the PTC Therapeutics FY Conference Call, providing insights into the company's performance, product developments, and strategic outlook for the future.

Summary of PTC Therapeutics Conference Call Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on rare disease therapies with a robust commercial portfolio of six marketed products [3][4] - The company has launched Safiance, an oral therapy for Phenylketonuria (PKU), which is expected to be a multi-billion dollar opportunity due to significant unmet needs in the PKU patient population [3][4][5] Core Points and Arguments Unmet Need in PKU - Approximately 17,000 individuals in the U.S. and 58,000 worldwide suffer from PKU, with a significant unmet need despite two existing therapies [4][5] - Current therapies do not sufficiently lower phenylalanine levels or allow dietary liberalization, leading to a burdensome diet for patients [5][6] - Safiance offers a differentiated solution with significant efficacy, allowing 97% of patients to liberalize their diet [7][8] Launch and Market Reception - Safiance was approved in July 2025, generating $19.6 million in revenue within the first six weeks of launch, with broad penetration among prescribers [10][27] - Physicians express strong interest in prescribing Safiance to all PKU patients, regardless of their current treatment status [11][18] - The launch strategy involved building relationships with Centers of Excellence and integrating with patient communities [10][12] Market Access and Payer Dynamics - Positive feedback from payers with minimal restrictions on Safiance prescriptions, primarily requiring prior authorization [19][22] - The company has engaged with payers to demonstrate the value of Safiance through clinical data, which has helped avoid step edits in access [21][22] International Expansion - The European launch began in Germany, maintaining a pricing corridor consistent with the U.S. [24][25] - Plans for launches in Japan and Brazil are underway, with expectations of favorable pricing dynamics [25][26] Financial Outlook - Revenue guidance for the year is set at $750 million to $800 million, with expectations that Safiance will drive the company towards profitability [29] - The company anticipates a decrease in operating expenses as expensive trials conclude [29] Additional Important Points Pipeline and Future Developments - PTC is focused on maintaining revenue from its Duchenne Muscular Dystrophy (DMD) franchise despite generic competition, achieving over 70% of peak revenue post-exclusivity [30][31] - Upcoming FDA meetings for Vatiquinone (Friedreich's ataxia) and PTC518 (Huntington's disease) are aimed at discussing potential paths for resubmission and trial designs [34][36] R&D Focus - The company plans to showcase progress on its scientific platforms at an upcoming R&D day, highlighting advancements in its splicing platform and other therapeutic areas [37] This summary encapsulates the key insights from the conference call, focusing on PTC Therapeutics' strategic initiatives, market dynamics, and future outlook.

Financial Performance and Goals - PTC aims for a \$2 billion topline revenue through commercial launches and innovative R&D platforms[9],[48] - The company is focused on reaching cash flow breakeven without additional capital[11],[48] Sephience (Sepiapterin) for PKU - Sephience global launch has generated \$19.6 million in revenue, including \$14.4 million in the US and \$5.2 million ex-US[16] - 341 patients are on commercial Sephience therapy worldwide[16] - Sephience treatment resulted in a 63% mean blood Phe reduction in the overall primary analysis population compared to a 1% reduction with placebo (p<0.0001)[18] - In classical PKU patients, Sephience treatment resulted in a 69% mean blood Phe reduction compared to a 3% reduction with placebo (p<0.001)[18] - Sephience showed a 70.3% greater reduction in blood Phe concentration compared to sapropterin (BH4) (p < 0.0001)[24] Votoplam for Huntington's Disease - Novartis will provide a \$1 billion upfront payment for the votoplam collaboration[35] - PTC could receive up to \$1.9 billion in development, regulatory, and sales milestones from Novartis[35] - PTC will receive a 40% U S profit share and double-digit tiered royalties on ex-U S sales from Novartis[35] Vatiquinone for Friedreich's Ataxia - Vatiquinone demonstrated a 42% slowing (p=0.021) of disease progression in the Upright Stability score over 72 weeks compared to placebo[39] - Long-term extension studies showed a 50% slowing (p<0.0001) of disease progression over 3 years[40] - A 4.8-point benefit (p<0.0001) on mFARS over 2 years in ambulatory and non-ambulatory adults was observed[40]

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.

Financial Performance & Outlook - PTC achieved total revenue of $807 million in 2024[14] - PTC has a strong cash position of over $2 billion, enabling future revenue growth and R&D innovation[15] - PTC aims to reach cash flow break-even without additional capital[16] - PTC's vision includes a path to $2 billion in topline revenue[53] Key Product Programs & Milestones - Four U S regulatory approval applications were submitted in 2024[11] - AADC Gene Therapy BLA was approved[12] - Sephience (sepiapterin) in PKU has a potential revenue opportunity greater than $1 billion[28] - Vatiquinone for Friedreich's Ataxia (FA) has an NDA filing accepted with Priority Review, with an approval decision expected August 19, 2025[32] - PTC518 for Huntington's Disease (HD) has a development and commercialization collaboration with Novartis, including $1 billion upfront payment and up to $19 billion in milestones[45] Research & Development - PTC has innovative research platforms, including a validated splicing platform[48] - PTC has inflammation & ferroptosis programs targeting CNS and non-CNS disorders[50]

Core Insights - PTC Therapeutics reported strong revenue performance of $190 million for the first quarter of 2025, with a significant cash position exceeding $2 billion as of March 31, 2025, supporting ongoing commercial and R&D activities [1][8]. Financial Performance - Total net product and royalty revenue for Q1 2025 was $190 million, a decrease from $208.8 million in Q1 2024 [5]. - Revenue from the DMD franchise was $134 million, with Translarna™ contributing $86 million and Emflaza® contributing $48 million [5]. - Collaboration and license revenue included $986.2 million from the PTC518 agreement with Novartis, which closed in January 2025 [5][7]. - Net income for Q1 2025 was $866.6 million, compared to a net loss of $91.6 million in Q1 2024 [8]. Regulatory and Clinical Updates - Positive CHMP opinion for Sephience™ (sepiapterin) received on April 25, 2025, with an expected European Commission adoption in approximately two months [5]. - NDA for Sephience is under review by the FDA, with a target action date of July 29, 2025 [5]. - NDA for vatiquinone accepted and granted Priority Review by the FDA, with a target action date of August 19, 2025 [5]. - Phase 2 PIVOT-HD study for PTC518 met primary endpoint of dose-dependent blood HTT lowering at Week 12, with favorable safety and tolerability [5]. Guidance and Future Outlook - PTC anticipates full-year 2025 revenue between $650 million and $800 million, including in-line products and potential new launches [8]. - Full-year 2025 GAAP R&D and SG&A expenses are projected to be between $805 million and $835 million [8]. - Non-GAAP R&D and SG&A expenses are expected to be between $730 million and $760 million, excluding estimated non-cash, stock-based compensation of $75 million [8].

Financial Data and Key Metrics Changes - Fourth quarter revenue totaled $213 million, and full year 2024 revenue was $807 million, exceeding guidance [8][31] - Cash, cash equivalents, and marketable securities totaled approximately $1.1 billion as of December 31, 2024, compared to $877 million as of December 31, 2023 [35] Business Line Data and Key Metrics Changes - DMD franchise revenue for the full year 2024 was $547 million, with Translarna net product revenue of $94 million and Emflaza net product revenue of $50 million in the fourth quarter [32][33] - The company achieved all clinical and regulatory milestones on schedule, including four FDA approval applications submitted [12][11] Market Data and Key Metrics Changes - The US represents the largest opportunity for Sepiapterin, with approximately 17,000 PKU patients, most of whom are not on medical treatments [23] - The estimated prevalence of Friedreich's ataxia in the US is about 6,000 patients, with one-third being pediatric [27] Company Strategy and Development Direction - The company plans to support 2025 commercial launches and continue investing in R&D platforms while exploring business development opportunities [10][11] - Anticipated milestones for 2025 include the global launch of Sepiapterin and potential launch of Vatiquinone in the US [15][16] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's strong financial position and ability to reach cash flow breakeven without raising additional capital [10][11] - The management highlighted the excitement surrounding Sepiapterin due to its potential to address significant unmet needs in the PKU patient population [15] Other Important Information - The company received a $1 billion upfront payment from Novartis as part of the PTC518 collaboration, with potential for up to $1.9 billion in development and sales milestones [13][35] - The company is preparing for multiple new product launches throughout the year, leveraging its experienced global commercial infrastructure [29] Q&A Session Summary Question: What is the expected impact of diet liberalization for PKU patients? - Management highlighted that over 97% of patients in the feed protocol can liberalize their diet, with many achieving or exceeding the recommended daily allowance of protein [40][41] Question: What is the market opportunity for Friedreich's ataxia? - Management expressed excitement about the opportunity to provide therapy for all FA patients, including pediatric patients, and emphasized the strong safety profile of the treatment [45][46] Question: What is the visibility on the Translarna review process? - Management indicated that the review is ongoing, and while they do not expect a formal PDUFA date, they anticipate more information in the first half of the year [57] Question: How should investors frame expectations for the PIVOT-HD update? - Management stated that the update will include both stage 2 and stage 3 patients, with a focus on biomarker data and clinical outcome scales [68][69] Question: How is the company preparing for the Sepiapterin launch? - Management noted that they are actively engaging with key stakeholders and preparing for a strategic launch sequence targeting access for PKU patients [22][24]