COPENHAGEN, Denmark — Ascendis Pharma A/S (Nasdaq: ASND) announced updates on its Vision 3x3 strategic roadmap and key milestones for 2022 during the 40th Annual J.P. Morgan Healthcare Conference. CEO Jan Mikkelsen highlighted the company's achievements in 2021, including becoming a sustainable biopharmaceutical company and launching SKYTROFA®, the first U.S. FDA-approved once-weekly treatment for pediatric growth hormone deficiency.
SKYTROFA® Launch Success: Since its U.S. launch, SKYTROFA® has seen significant uptake, with 10% of target prescribers opting for it over daily growth hormone treatments within two months. The company also anticipates European Commission approval for TransCon hGH by the end of January 2022.
2022 Clinical and Regulatory Milestones: Ascendis plans to share multiple clinical data readouts across its endocrinology rare disease and oncology portfolios. Key highlights include:
- TransCon hGH: Plans to submit a protocol to the FDA for Turner Syndrome subjects in Q2 2022 and complete enrollment in the foresiGHt Trial for adult growth hormone deficiency.
- TransCon PTH: Expects top-line results from the Phase 3 PaTHway Trial in Q1 2022, with NDA and MAA submissions planned for Q3 and Q4, respectively.
- TransCon CNP: Awaits top-line data from the ACcomplisH Trial in Q4 2022 and plans to file an IND for the ACcomplisH Infants Trial in Q2 2022.
- TransCon TLR7/8 Agonist and TransCon IL-2 β/γ: Expects top-line data from monotherapy and combo-therapy dose escalation in Q3 and Q4 2022, respectively.
Future Directions: Ascendis aims to announce a third independent therapeutic area with its own diversified pipeline by Q4 2022, further solidifying its position as a leading global biopharmaceutical company.
Presentation Details: The live webcast of the J.P. Morgan presentation is available on the Ascendis Pharma website, with a replay accessible for 30 days post-event.
About Ascendis Pharma A/S: Ascendis Pharma is dedicated to creating innovative therapies using its TransCon technologies, aiming to make a significant impact on patients' lives. The company operates globally, with headquarters in Copenhagen, Denmark, and facilities in Germany and the United States.
Forward-Looking Statements: This update contains forward-looking statements subject to risks and uncertainties, including the timing of clinical trials, regulatory approvals, and market acceptance of new therapies. Ascendis Pharma does not assume any obligation to update these statements, except as required by law.
