On january 8, 2023 Ascendis Pharma A/S (Nasdaq: ASND) reported an update on its Vision 3×3 strategic roadmap and planned 2023 key corporate milestones. Ascendis President and CEO Jan Mikkelsen will present the update tomorrow, January 9, at the 41st Annual J.P. Morgan Healthcare Conference (Press release, Ascendis Pharma, JAN 8, 2023, View Source;3-strategic-roadmap-0 [SID1234626004]).

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"As we approach anticipated launch of our second endocrinology rare disease product, TransCon PTH for hypoparathyroidism, we remain on track to achieve Vision 3×3 to become a sustainable, profitable leading biopharma company," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "We are deeply committed to advancing our pipeline of endocrinology rare disease, oncology, and now ophthalmology product candidates designed to make a meaningful difference in the lives of patients."

Key Updates

TransCon hGH:

During the fourth quarter, 2022, we completed recruitment into the Phase 3 foresiGHt Trial in adult growth hormone deficiency (GHD). Topline results from foresiGHt are expected in the fourth quarter of 2023.
During the third quarter of 2023, we anticipate completing enrollment in New InsiGHTS, a Phase 2 trial evaluating the safety and efficacy of TransCon hGH in patients with Turner Syndrome.
First European SKYTROFA (lonapegsomatropin) commercial launch planned in Germany for the third quarter of 2023.
SKYTROFA (lonapegsomatropin-tcgd) U.S. preliminary, unaudited fourth quarter 2022 revenue is expected to be approximately €17.1 million. This includes an estimated negative foreign currency translation impact of €0.4 million, compared to a benefit of €0.5 million in the third quarter of 2022.
TransCon PTH:

Phase 3 PaTHway Japan trial achieved its primary objectives; topline results consistent with North American and EU trials.
FDA Priority Review continues for use in adult patients with hypoparathyroidism, with an April 30, 2023 PDUFA date; if approved, U.S. commercial launch planned in the second quarter of 2023.
Enrollment opened in January 2023 for U.S. Expanded Access Program (EAP).
European Commission decision anticipated during the fourth quarter of 2023; if approved, EU commercial launch planned shortly thereafter.
Once-weekly TransCon PTH in preclinical development for patients on stable daily TransCon PTH dose.

TransCon CNP:

First-ever randomized, double-blind, placebo-controlled Phase 2 trial (ACcomplisH) suggests a potential for safety, efficacy, tolerability, and convenience in children with achondroplasia as young as two years of age; all 57 patients currently remain in the trial with treatment duration up to 3 years.
During the second quarter of 2023, we expect to complete enrollment in ApproaCH, a global randomized, double-blind, placebo-controlled Phase 2b trial in children ages 2–11 years with achondroplasia. The trial targets enrollment of ~80 patients.
During the third quarter of 2023, we expect to submit an IND or similar in children under the age of two years with achondroplasia.

TransCon TLR7/8 Agonist:

Reported topline data from the dose escalation portion of the Phase 1/2 transcendIT-101 Trial at SITC (Free SITC Whitepaper) 2022. Early signs of clinical activity were observed in patients receiving TransCon TLR7/8 Agonist as monotherapy or in combination with pembrolizumab.
Enrollment in transcendIT-101 continues with dose expansion focused on investigating TransCon TLR7/8 Agonist in combination with pembrolizumab in four cancer types.

TransCon IL-2 β/γ

The Phase 1/2 IL-βelieγe Trial evaluating TransCon IL-2 β/γ monotherapy in patients with locally advanced or metastatic solid tumors continues to enroll patients. Results from monotherapy dose escalation are expected during the first quarter of 2023.

Ophthalmology selected as the third therapeutic area:

In vivo data demonstrates, TransCon Hydrogel Platform supports continuous local drug release over at least 6 months supporting twice yearly administration.
TransCon RBZ (ranibizumab) selected as the first product candidate.

Presentation at J.P. Morgan Healthcare Conference on Monday, January 9th

A live webcast of the event will be available via the Investors & News section of the Ascendis Pharma website at View Source The presentation will begin at 12:00 p.m. Eastern Time / 9:00 am Pacific Time. A webcast replay will be available for 30 days.

The Company’s slides from the J.P. Morgan presentation will be available on the same Investor Relations website at View Source