Ascendis Pharma Provides Update on Vision 3x3 Strategic Roadmap at 41st Annual J.P. Morgan Healthcare Conference
– On track to fulfill all elements of Vision 3x3 to build a sustainable, profitable, leading biopharma company
“As we approach anticipated launch of our second endocrinology rare disease product, TransCon PTH for hypoparathyroidism, we remain on track to achieve Vision 3x3 to become a sustainable, profitable leading biopharma company,” said
- 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
Germanyfor 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, 2023PDUFA date; if approved, U.S.commercial launch planned in the second quarter of 2023.
- Enrollment opened in
January 2023for U.S.Expanded Access Program (EAP). European Commissiondecision 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 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.
A live webcast of the event will be available via the Investors & News section of the
The Company’s slides from the J.P. Morgan presentation will be available on the same Investor Relations website at https://investors.ascendispharma.com.
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) the expected regulatory approval and launch of TransCon PTH in 2023, (ii) the timing and announcement of top-line results from the foresiGHt Trial and the Phase 1/2 IL-βelieγe Trial, (iii) the timing of completion of patient enrollment in the New InsiGHTS Trial and the ApproaCH Trial, (iv) Ascendis’ PDUFA date of
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Source: Ascendis Pharma