Ascendis Pharma(ASND)2025-02-13 04:03Financial Data and Key Metrics Changes - In 2024, SKYTROFA revenue reached approximately €200 million, with a 6.5% market share of the total growth hormone market in the U.S. and around 45% of the total U.S. long-acting growth hormone market [10][11] - Total revenue for the fourth quarter was €173.9 million, including a $100 million upfront fee from Novo Nordisk, while total revenue for the full year 2024 was €363.6 million [35][36] - For the fourth quarter, SKYTROFA revenue was €58.5 million, down from €64.2 million in the same quarter of 2023, but benefited from a favorable sales adjustment of €4.6 million [32][34] Business Line Data and Key Metrics Changes - SKYTROFA volume increased 84% in 2024, with a premium net pricing of 3x compared to daily growth hormone [18][10] - YORVIPATH revenue for the fourth quarter increased to €13.6 million, bringing total 2024 revenue to €28.7 million [33][34] - TransCon CNP demonstrated significant improvements in linear growth and body proportionality compared to placebo in clinical trials [25][26] Market Data and Key Metrics Changes - The U.S. market for chronic hypoparathyroidism is estimated to have about 70,000 to 90,000 patients, with a significant portion currently using conventional therapy [19][20] - Initial demand for YORVIPATH is strong, with 908 patients prescribed as of February 7, 2025, including 539 unique prescribers across 44 states [21][22] Company Strategy and Development Direction - The company aims to expand SKYTROFA's addressable market and plans to submit an NDA for adult growth hormone deficiency this year [17][31] - YORVIPATH is expected to become the standard-of-care for hypoparathyroidism, with plans for further commercial launches in multiple international markets [34][22] - The company is expanding its TransCon technology platform beyond endocrine rare diseases into metabolic diseases and ophthalmology [14][29] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong launch of YORVIPATH and its potential to become a multi-billion-dollar product [11][34] - The company is optimistic about the growth potential of SKYTROFA and YORVIPATH, with expectations for sustained revenue growth [10][31] - Management highlighted the importance of ongoing discussions with payers and the evolving reimbursement landscape for YORVIPATH [61][62] Other Important Information - The company ended 2024 with cash, cash equivalents, and marketable securities totaling €560 million, compared to €399 million at the end of 2023 [38] - The company plans to submit an IND application for a combination treatment of TransCon CNP and TransCon Growth Hormone for achondroplasia [27][26] Q&A Session Summary Question: Can you confirm the 908 figure is unique patients and not cumulative scripts? - Yes, the 908 figure represents unique patient enrollments, with about 80% being new to YORVIPATH and 20% from EAP or clinical trials [41][44] Question: How many of the 908 scripts have converted to actual therapy? - The company is still gathering data on this, but many of the patients are expected to be from the uncontrolled group [50][54] Question: What type of doctors are prescribing YORVIPATH? - Prescribers include a broad range of physicians, with over 50% reach among priority physicians, and many are new prescribers not previously associated with NATPARA [82][87] Question: What are the expectations for prior authorizations for YORVIPATH? - The company anticipates some prior authorization requirements, but discussions with payers have been productive [61][96] Question: What is the market opportunity for adult growth hormone deficiency? - The penetration rate is currently low, presenting a significant growth opportunity, especially with the upcoming PDUFA date [126][127]