Detailed narrative
TransCon Technology Platform & Vision 2030
Ascendis Pharma is transforming into a leading global biopharma company, driven by its TransCon technology platform and strong development/commercialization capabilities. The company aims to achieve EUR 5 billion in revenues by 2030 and over EUR 10 billion in the next decades, supported by its existing portfolio and continuous innovation. The platform enables filing at least one IND for a new chemical entity (NCE) annually, laying a foundation for sustained growth and expansion into new therapeutic areas.
YORVIPATH Performance & Expansion
YORVIPATH, the first and only approved treatment for adult hypoparathyroidism, continues to see robust uptake in the U.S. and global expansion, now available in over 35 countries. Long-term Phase II/III data show sustained response rates of 82-86% for the multi-covenant endpoint and 95% patient retention after 5 years, highlighting its potential as a standard of care. The company is pursuing label expansion for 12-18 year olds, higher doses in the U.S., and developing a once-weekly product for patients on stable doses.
YUVIWEL Launch & Market Expansion
YUVIWEL's U.S. commercial launch has seen rapid uptake, generating EUR 8 million in its first quarter. Over 220 unique patients were enrolled and more than 65% approved for reimbursement by the end of July. Management believes YUVIWEL is expanding the achondroplasia market by attracting new patients and those returning to therapy, rather than just switches, with nearly 80% of centers of excellence already prescribing it. An EU regulatory decision is expected in Q4 2026.
Combination Therapy for Achondroplasia
Ascendis is developing a unique combination therapy of TransCon CNP and TransCon Growth Hormone for achondroplasia. Recent week 78 COACH trial data demonstrated sustained efficacy, good safety, and tolerability, showing unprecedented🌐 improvements in arm span (+9.4 cm for naive, 7.9 cm for treated cohorts). This combination aims to establish a new treatment standard, with Phase III trials beginning later this year, and is expected to transform treatment for achondroplasia and other indications.
SKYTROFA Leadership & Pipeline
SKYTROFA remains the #1 long-acting growth hormone in the U.S., with over 20,000 unique enrollments, demonstrating the strength of Ascendis's commercial infrastructure. The company is pursuing label and geographic expansions, including Phase III basket trials for TransCon Growth Hormone in ISS, SDA, and Turner syndrome. The broader pipeline includes a once-monthly TransCon semiglutide program with Novo Nordisk and an anti-VEGF treatment with Iconis in ophthalmology, which recently initiated a first-in-human clinical trial.
ITC Legal Battle Update
The company is engaged in an ITC legal battle with BioMarin regarding a YUVIWEL patent. The patent was invalidated in Europe. In the U.S., a first opinion from a single judge is expected in August, followed by a full ITC decision in December, and a potential presidential order 2-3 months later. Management emphasizes the public interest in patient access for YUVIWEL and believes the U.S. case will not materially impact Ascendis's long-term pathway or its Vision 2030 objectives.
