Detailed narrative
Q1 FY26 Performance Highlights
Ascendis Pharma achieved significant milestones in Q1 FY26, including the FDA approval of YUVIWEL, marking their third TransCon product. The company reported rapid revenue growth, profitability, and a robust pipeline. Global YORVIPATH revenue reached EUR 197 million, while SKYTROFA contributed EUR 44 million, leading to total Q1 revenue of EUR 247 million. The company reported an operating profit of EUR 25 million and a non-IFRS operating profit of EUR 55 million, reflecting a 22% margin.
YORVIPATH Commercial Progress
YORVIPATH continued strong uptake in the U.S., with over 1,000 new patients prescribed in Q1 FY26 and a cumulative 6,300+ patients by March 2026, served by over 2,700 unique healthcare providers. Insurance approval rates and median time to approval are improving, with 80% upstream coverage and over half of patients approved within 8 weeks. The product is commercially available or in early access programs in 35 countries outside the U.S., with additional launches expected through 2026. The company is pursuing label expansion for doses up to 60 micrograms in the U.S. and global expansion to pediatric patients.
YUVIWEL Launch and Early Success
Following FDA approval, YUVIWEL became commercially available in early April and has already been prescribed for over 60 children by more than 35 unique healthcare providers in less than 4-5 weeks. This rapid uptake reflects the significant unmet medical need and compelling clinical data, including improvements in final dimensions, body proportionality, physical function, and health-related quality of life, without compromising safety. An EU regulatory decision on the Marketing Authorization Application for YUVIWEL is expected in Q4 FY26. Label expansion trials are ongoing for infants under 2 years of age and geographic expansion.
SKYTROFA and Growth Disorder Portfolio
SKYTROFA maintained consistent performance as a premium product, holding a 7% share of the overall growth hormone market. Expected label expansion could double the addressable patient population in the U.S., and geographic expansion outside the U.S. is also planned. The company is also developing a combination therapy of once-weekly TransCon CNP and TransCon Growth Hormone for hypochondroplasia. Phase II COACH trial 52-week data showed triple efficacy compared to TransCon CNP monotherapy, improved body proportionality, lower limb alignment, spinal canal dimensions, and arm strength, potentially eliminating the need for invasive surgeries.
Pipeline and Strategic Focus
Ascendis has more than 20 ongoing or planned clinical trials, including 4 new clinical entities in preclinical development. The company decided to discontinue internal oncology development of TransCon IL-2 beta gamma, as it does not align with its strategic focus, and will explore other ways to maximize the value of these assets. Partnerships with Novo Nordisk for once-monthly TransCon semaglutide and with an undisclosed partner for TransCon anti-VEGF are advancing towards the clinic, highlighting the broader potential of the TransCon technology platform.
Financial Performance and Capital Allocation
The company reported total operating expenses of EUR 204 million, net profit of EUR 629 million (including a EUR 679 million deferred tax asset recognition), and non-IFRS net profit of EUR 18 million (EUR 0.27 per share). Ascendis ended Q1 FY26 with EUR 573 million in cash and cash equivalents, after a EUR 60 million impact from share repurchases and RSU settlements. The company completed a direct listing on NASDAQ, fully redeemed outstanding convertible senior notes, and sold a Priority Review Voucher for USD 187.5 million, with a primary focus on R&D investment for long-term growth.