Strategic Roadmap to Achieve Blockbuster Status
Ascendis Pharma A/S (Nasdaq: ASND) has introduced Vision 2030, a strategic roadmap aimed at achieving blockbuster status for multiple products and expanding the company's engine for future innovation. This announcement was made by Ascendis President and CEO Jan Mikkelsen, who will present the update at the 42nd Annual J.P. Morgan Healthcare Conference.
Key Updates on Product Development
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TransCon™ hGH (lonapegsomatropin): Following positive Phase 3 foresiGHt Trial results in adult growth hormone deficiency (GHD), Ascendis plans to submit a supplemental Biologics License Application to the FDA in Q2 2024. Topline results from a Phase 2 trial in Turner syndrome are expected in Q4 2024. SKYTROFA, the brand name for TransCon hGH, has achieved U.S. market value leadership in 2023, with preliminary unaudited Q4 2023 revenue expected to be approximately €64 million.
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TransCon PTH (palopegteriparatide): The first launch is planned in Germany in January 2024, with FDA regulatory review ongoing for the treatment of adult patients with hypoparathyroidism. A Prescription Drug User Fee Act (PDUFA) date is set for May 14, 2024, with a U.S. commercial launch planned in Q3 2024 if approved.
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TransCon CNP (navepegritide): Treatment in children with achondroplasia has shown consistent growth and improvements in health-related quality of life. A Clinical Trial Application (CTA) for an infant trial was submitted in Q3 2023, with topline results from the pivotal ApproaCH Trial expected in Q4 2024. Plans include submitting a New Drug Application to the FDA for children with achondroplasia and an Investigational New Drug application for adults.
Global Expansion and New Therapeutic Areas
Ascendis is expanding its global reach through exclusive sales and distribution agreements and is exploring new therapeutic areas, including oncology and ophthalmology. The company expects to complete enrollment in a Phase 2 trial for advanced head and neck squamous cell carcinoma (HNSCC) and provide a clinical update from the Phase 2 portion of the IL-Believe Trial in Q4 2024. Additionally, the creation of Ophthalmology NewCo, financed by institutional investors, is expected in Q1 2024.
Financial Outlook
Preliminary unaudited cash, cash equivalents, and marketable securities as of December 31, 2023, are approximately €400 million. Full-year 2024 SKYTROFA revenue is expected to be between €320 million and €340 million, based on average 2023 exchange rates. Ascendis expects total operating expenses of approximately €600 million for 2024 and aims to be operating cash flow breakeven on a quarterly basis by the end of 2024.
Vision 2030: Strategic Roadmap
Vision 2030 outlines Ascendis Pharma's ambitions to achieve blockbuster status for multiple products, expand its pipeline with Endocrinology Rare Disease product opportunities, and create value in additional therapeutic areas through innovative business models. The company aims to differentiate itself with its product innovation algorithm and remain independent as a profitable biopharma.
Presentation at J.P. Morgan Healthcare Conference
A live webcast of the presentation will be available on the Ascendis Pharma website, with a replay accessible for 30 days. The company's slides from the presentation will also be available on the Investor Relations website.
