“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
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 forU.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 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
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.
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