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 ...

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]