Ascendis Pharma Provides Business and Strategic Roadmap Update at 43rd Annual J.P. Morgan Healthcare Conference

Business and Strategic Roadmap - Ascendis Pharma is positioned for rapid revenue growth driven by the launch of its first two Endocrinology Rare Disease medicines, SKYTROFA and YORVIPATH, and planned filings for its third product, TransCon CNP for achondroplasia [2] - The company's expanding TransCon technology platform and partnerships demonstrate a business model focused on fast, successful drug development, aiming to create substantial stakeholder value [2] Key Updates and Milestones TransCon hGH (SKYTROFA) - SKYTROFA full-year 2024 revenue expected to be ~€202 million, excluding sales deductions related to prior years [3][7] - U.S. SKYTROFA volume (mg) increased 84% year-over-year in 2024, capturing an estimated 6.5% market share of the total U.S. growth hormone market [3] - PDUFA goal date of July 27, 2025, for FDA review of supplemental BLA for adult growth hormone deficiency treatment, with a planned U.S. commercial launch in Q4 2025 [3] - Plan to submit an IND application or similar for a basket trial evaluating TransCon hGH in additional indications during Q3 2025 [3] TransCon PTH (YORVIPATH) - YORVIPATH full-year 2024 unaudited preliminary revenue estimate of ~€29 million [3][7] - Commercially available in Germany and Austria since January 2024, with ~700 patients on treatment in Europe Direct and International Markets by the end of 2024 [3] - Available for prescription in the U.S. since late December 2024, with 324 patients enrolled in the Ascendis Signature Access Program or direct with specialty pharmacy as of January 9, 2025 [3] - Expect commercial launch in at least five additional Europe Direct countries in 2025 [3] TransCon CNP (navepegritide) - Plan to submit NDA for the treatment of children with achondroplasia in Q1 2025 and Marketing Authorisation Application to the European Medicines Agency in Q3 2025 [3] - New data from the ApproaCH Trial showed significant improvements in leg bowing with TransCon CNP compared to placebo [3] - Topline Week 26 results from Phase 2 COACH Trial expected in Q2 2025 [3] - Plan to submit an IND or similar for the treatment of hypochondroplasia in Q4 2025 [3] Expanding the TransCon Platform & Pipeline - Development of a new TransCon protein degrader platform designed to enable efficient clearance of hormones, cytokines, and other targets [3] - First planned TransCon protein degrader product candidate aims to normalize excess FGF-23 hormone levels for patients with X-linked hypophosphatemia [3] Financial Update - Unaudited preliminary estimate of total full-year 2024 product revenue of ~€226 million [3] - Total full-year 2024 revenue, including a $100 million Novo Nordisk milestone payment, estimated at ~€364 million [7] - Pro forma cash balance as of December 31, 2024, estimated at ~€655 million, including the expected Novo Nordisk payment [7] Presentation at J.P. Morgan Healthcare Conference - Ascendis Pharma's President and CEO Jan Mikkelsen will present the business and strategic roadmap update at the 43rd Annual J.P. Morgan Healthcare Conference on January 13, 2025 [1][4] - A live webcast of the event will be available on the company's Investor Relations website, with a replay accessible for 30 days [4]