PTC Therapeutics(PTCT)2023-02-22 03:35Financial Data and Key Metrics Changes - The company achieved total revenue of $699 million in 2022, representing a 30% growth over 2021, despite significant foreign exchange headwinds [169] - For Q4 2022, total revenue was $167 million, consisting of $128 million from net product revenue and $40 million from Evrysdi royalty revenue [14][169] - Non-GAAP R&D expenses for Q4 2022 were $175 million, up from $136 million in Q4 2021, reflecting increased investment in research programs [41] Business Line Data and Key Metrics Changes - The DMD franchise generated $507 million in revenue for 2022, a 20% year-over-year growth, with Q4 revenue of $114 million [162] - Translarna achieved $56 million in revenue for Q4 2022, totaling $289 million for the year, marking a 22% growth [12][178] - Emflaza reported Q4 revenue of $58 million, bringing total annual revenue to $218 million, a 17% increase over 2021 [207] Market Data and Key Metrics Changes - The company is expanding its geographic presence in Latin America, the Middle East, and Asia Pacific, with ongoing efforts to launch Upstaza and grow the DMD franchise [12][38] - The company anticipates significant growth in 2023, with revenue guidance set between $940 million to $1 billion, reflecting over 30% year-over-year growth [155] Company Strategy and Development Direction - The company aims to deliver one new therapy every two to three years, focusing on rare diseases by modulating gene and protein expression [168] - The company is preparing for the launch of Upstaza in the US, with a dedicated team focusing on disease awareness and key publications [125] - The company plans to request a Type C meeting with the FDA to discuss the potential path for NDA resubmission for Translarna [160] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong performance and growth trajectory for 2023, highlighting the excitement around upcoming clinical trial readouts [4][118] - The company noted the importance of addressing the unmet medical needs of patients, particularly in the PKU and mitochondrial disease markets [6][50] Other Important Information - The company was awarded the 2023 EURORDIS Black Pearl Company Award for Innovation, recognizing its contributions to rare disease research [154] - The company has initiated additional dosing cohorts in the PTC518 Huntington's disease program, with results expected in the second quarter of 2023 [36] Q&A Session Summary Question: Can you break down revenue components in your full year 2023 revenue guidance? - The company expects revenue guidance of $940 million to $1 billion, reflecting growth in the DMD franchise, Upstaza, and Evrysdi, including a $100 million milestone for Evrysdi [43] Question: Can you clarify the nature of your discussions with the agency on Translarna? - The company had an informal meeting with the FDA and plans to request another Type C meeting to review the totality of data collected [55][160] Question: What is the expected reduction in seizure frequency considered clinically meaningful for the MIT-E study? - The study is powered for a 40% differential reduction between treatment and placebo groups, with a hypothesized reduction of 50% in the active population [65] Question: Can you provide an update on the APHENITY study? - The company reported that 156 subjects passed screening, with 102 achieving greater than 30% reduction in phenylalanine levels, indicating a high responder rate [203] Question: What is the rationale for dose escalation in the Part 2 study? - The company plans to utilize a two-week dose escalation approach to assess the safety and efficacy of higher doses in the study [134]