PTC Therapeutics(PTCT) - 2024 Q2 - Earnings Call Transcript

Financial Data and Key Metrics - Q2 2024 total revenue was $187 million, with DMD franchise revenue contributing$118 million [4] - Translarna net product revenue for Q2 was $70 million, while EMFLAZA net product revenue was$47 million [17] - Royalty revenue from Evrysdi was $53 million, driven by Roche's global revenue of$535 million [17] - Non-GAAP R&D expense was $123 million, down from$170 million in Q2 2023, reflecting strategic portfolio prioritization [17] - Non-GAAP SG&A expense was $60 million, compared to$75 million in Q2 2023 [17] - Cash, cash equivalents, and marketable securities totaled $1.09 billion as of June 30, 2024, up from$877 million at the end of 2023 [18] Business Line Performance - DMD franchise delivered $118 million in Q2, with Translarna commercialization ongoing in Europe and international markets [11] - EMFLAZA net revenue was$47 million in Q2, supported by patient programs and strong payer relationships [12] - Sepiapterin NDA submitted to the FDA for PKU treatment, with plans for global launch in 2025 [6] - Translarna NDA resubmitted to the FDA, with positive results from Study 41 supporting the submission [7] - PTC518 for Huntington's disease showed promising 12-month interim data, with dose-dependent lowering of mutant Huntington protein and favorable clinical effects [7][8] Market Performance - Translarna commercialization continues in Europe and international markets, with new group purchase orders in Brazil [11] - Sepiapterin launch preparations are underway, with submissions planned for Japan, Brazil, and other countries in 2024 [6][12] - Upstaza received FDA priority review with a target action date of November 13, 2024, and global access discussions are progressing [15] - TEGSEDI and WAYLIVRA showed growth in Latin America, with recent approval in Mexico and new group purchase orders in Brazil [16] Strategic Direction and Industry Competition - Focus on execution and achieving regulatory milestones, including NDA submissions for sepiapterin, Translarna, and Vatiquinone [4][9] - Sepiapterin is positioned as a potential new standard of care for PKU, with data supporting differentiation from current therapies [13] - PTC518 is seen as a promising therapy for Huntington's disease, with plans to pursue accelerated approval based on biomarker and clinical data [8][29] - Utreloxastat for ALS is expected to address significant unmet needs, with top-line results from the CARDINALS trial anticipated in Q4 2024 [9][40] Management Commentary on Operating Environment and Future Outlook - Management highlighted strong execution across all areas, with solid revenue performance and effective expense management [4] - The company updated 2024 revenue guidance to $700 million to$750 million, driven by sustained Translarna revenue in Europe [16] - Management expressed confidence in the potential of sepiapterin, PTC518, and Vatiquinone to address unmet medical needs and drive future growth [6][8][9] Other Important Information - The company completed the sale of its gene therapy manufacturing business in Hopewell, New Jersey, receiving $27.5 million upfront and reducing operational expenses [5] - PTC amended its agreement with Royalty Pharma, exercising a put option for$250 million in cash [18] Q&A Session Summary Question: Huntington's Disease Biomarkers and Safety - PTC518 has shown no evidence of NFL spikes, indicating a favorable safety profile, with dose-dependent lowering of mutant Huntington protein and early clinical benefits [20][21][22] Question: PKU Launch Expectations - Sepiapterin launch is expected to have a fast uptake, particularly in the US, due to well-defined patient identification through newborn screening and strong preparation [23][24] Question: Revenue Guidance and Regulatory Path for PTC518 - Revenue guidance was updated due to sustained Translarna revenue in Europe, with the majority of revenue coming from PTC-marketed products [31] - PTC is preparing for FDA discussions on accelerated approval for PTC518, with plans to submit a Phase III efficacy trial design [26][29] Question: Translarna Regulatory Status in Europe and US - Translarna reexamination in Europe is ongoing, with a focus on addressing concerns around the STRIDE analysis [33] - Translarna NDA resubmission in the US is under review, with expectations of regulatory action within six months [34] Question: ALS Trial Expectations - The CARDINALS trial for utreloxastat in ALS is designed to support NDA submission, with success defined as statistically significant primary endpoint results [39][40] Question: Huntington's Disease Biomarker Correlation - Huntington protein lowering is the most reliable biomarker for clinical benefit, with evidence supporting its use as a surrogate endpoint for accelerated approval [43][44] Question: ALS Trial Design and FDA Expectations - The CARDINALS trial design aligns with FDA expectations, incorporating ALSFRS scale changes and time to death as key endpoints [47] Question: Translarna US Market Opportunity - Translarna is expected to have a rapid uptake in the US, driven by high unmet medical need and strong physician and patient demand [52][53][54] Question: SG&A Expenses and Vatiquinone Data - No significant increase in SG&A expenses is expected for 2025, as the company has built infrastructure to support multiple launches [58][59] - Vatiquinone data from the MOVE FA study will be shared once the long-term analysis is completed [60] Question: Huntington's Disease Surrogate Biomarkers - PTC believes Huntington protein lowering fits FDA guidance for surrogate endpoints, with strong scientific evidence supporting its use [64][65]