Ascendis Pharma A/S Introduces Vision 2030
During the fourth quarter of 2024, plan to submit a New Drug Application to FDA for children with achondroplasia (age 2-11 years). Week 26 topline data from the COACH Trial (TransCon hGH/TransCon CNP combination) expected in the fourth quarter of 2024. During the fourth quarter of 2024, plan to submit an Investigational New Drug application or similar in adults with achondroplasia. Global commercial presence in Endocrinology Rare Disease During the fourth quarter of 2023, entered into an exclusive license agreement with Teijin Limited for TransCon hGH, TransCon PTH, and TransCon CNP in Japan. Phase 3 PaTHway Japan Trial of TransCon PTH completed, and Phase 3 riGHt Trial of TransCon hGH fully enrolled. VISEN?s TransCon hGH Phase 3 and TransCon CNP Phase 2 trials in China completed. Expanding global reach through exclusive sales & distribution agreements with geographic market leaders in International Markets, with three regional agreements signed to date: Specialised Therapeutics Asia Pte Ltd, Er-Kim, and Vector Pharma FZCO. Oncology: During the fourth quarter of 2024, expect to complete enrollment in BelieveIT-201, a Phase 2 trial in advanced head and neck squamous cell carcinoma (HNSCC). During the fourth quarter of 2024, plan to provide a clinical update from the Phase 2 portion of indication-specific, dose expansion cohorts in the IL-Believe Trial. Ophthalmology: Creation of Ophthalmology NewCo, financed by institutional investors, expected during the first quarter of 2024. Financial Update and Outlook Based on Current Plans: Preliminary unaudited December 31, 2023 cash, cash equivalents, and marketable securities of ~?400 million. Full year 2024 SKYTROFA revenue expected to be ?320 million to ?340 million (based on average 2023 exchange rates). Expect to provide YORVIPATH revenue update during 2024. Expect total operating expenses (SG&A and R&D) of approximately ?600 million for 2024. Expect to be operating cash flow breakeven on a quarterly basis by the end of 2024. Vision 2030: Ascendis Pharma?s 2025?2030 Strategic Roadmap Achieve blockbuster status for multiple products and expand our engine for future innovation. Be the Leading Endocrinology Rare Disease Company: Achieve blockbuster status (>$1B) for TransCon PTH, TransCon hGH, and TransCon CNP through worldwide commercialization, Be the leader in growth disorders and hypoparathyroidism, pursuing clinical conditions, innovative life cycle management, and complementary patient offerings, Expand pipeline with Endocrinology Rare Disease blockbuster product opportunities, Create Value in Additional Therapeutic Areas through Innovative Business Models, Obtain accelerated approval in oncology with registrational trials ongoing, Pursue TransCon product opportunities in >$5B indications, Maximize value creation of these product opportunities through collaboration with therapeutic area market leaders, Differentiate with Ascendis Fundamentals: Outperform industry drug development benchmarks with Ascendis? product innovation algorithm
Remain independent as a profitable biopharma through lean and flexible ways of working.