On January 10, 2022 Ascendis Pharma A/S (Nasdaq: ASND) reported that the Company will today provide an update on its Vision 3×3 and planned 2022 key milestones at the 40th Annual J.P. Morgan Healthcare Conference. Details of the update are outlined below, and CEO Jan Mikkelsen will give a virtual presentation to attendees from 3:00-3:40 p.m. Eastern Time (Press release, Ascendis Pharma, JAN 10, 2022, View Source;3-strategic-roadmap [SID1234598467]).
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"We took major steps in 2021 to becoming a viable, sustainable, and profitable biopharmaceutical company," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "A strong flow of clinical results and our first commercial TransCon product showcased how we are leveraging our technology platform to build a leading global biopharmaceutical company able to successfully meet the needs of patients with differentiated product candidates."
"This year, I am happy to report that our U.S. launch of SKYTROFA (lonapegsomatropin-tcgd), the first U.S. FDA-approved once-weekly treatment for pediatric growth hormone deficiency is off to a strong start," continued Mr. Mikkelsen. "In only two short months since launch, we have already seen 10% of our target prescribers’ writing prescriptions for SKYTROFA instead of daily growth hormone."
"During 2022, we look forward to sharing multiple clinical data readouts across our high-value endocrinology rare disease and oncology portfolios," continued Mr. Mikkelsen. "We believe the clinical readouts will demonstrate our ability to deliver therapies that make a meaningful impact on patients’ lives. In addition, we look forward to announcing a third independent therapeutic area with its own diversified pipeline in the fourth quarter of 2022."
Pipeline Updates
TransCon hGH: TransCon hGH is an investigational once-weekly prodrug designed to deliver somatropin over a one-week period. TransCon hGH is approved by the FDA in the U.S. under the brand name SKYTROFA (lonapegsomatropin-tcgd) for the treatment of pediatric patients one year and older who weigh at least 11.5 kg and have growth failure due to inadequate secretion of endogenous growth hormone.
In mid-October, the Company commercially launched in the U.S. TransCon hGH under the brand name SKYTROFA. Since launch, physician enthusiasm for SKYTROFA is reflected by an increase in prescriptions, submission of formulary exceptions, and repeat prescribers. During the fourth quarter, 369 SKYTROFA prescriptions were written by 139 targeted prescribers, which includes 42% repeat prescribers.1
In November 2021, the Company received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency for TransCon hGH for patients with pediatric growth hormone deficiency. The European Commission’s approval of the Marketing Authorisation Application (MAA) is expected by the end of January 2022.
During the second quarter of 2022, the Company plans to submit a protocol to the FDA for TransCon hGH in Turner Syndrome subjects.
Ascendis is targeting completion of enrollment in foresiGHt, a global Phase 3 trial evaluating the safety and efficacy of TransCon hGH in adult patients with growth hormone deficiency during the second quarter of 2022.
TransCon PTH: TransCon PTH is an investigational long-acting prodrug of parathyroid hormone (PTH) in development as a potential once-daily replacement therapy for adult hypoparathyroidism (HP):
During the first quarter of 2022, top-line results are expected from PaTHway, a Phase 3 randomized, double-blind, placebo-controlled clinical trial in North America and Europe, investigating the safety, tolerability, and efficacy of TransCon PTH in adults with HP.
If the Phase 3 PaTHway Trial results are positive, Ascendis plans to submit a New Drug Application (NDA) to the FDA in the third quarter of 2022 followed by a MAA submission to the EMA in the fourth quarter of 2022.
Top-line results from PaTHway Japan, a single-arm Phase 3 trial of TransCon PTH in a minimum of 12-Japanese subjects with HP are expected in the third quarter of 2022.
Initiation of a pediatric HP program is planned for the fourth quarter of 2022.
TransCon CNP: TransCon CNP, an investigational long-acting prodrug of C-type natriuretic peptide (CNP), as a potential therapeutic option for patients with achondroplasia (ACH):
Top-line data from the ACcomplisH Trial, a Phase 2 randomized, double-blind, placebo-controlled clinical trial in North America, Europe, and Oceania in subjects with achondroplasia (age 2–10) are expected in the fourth quarter of 2022.
During the second quarter of 2022, the Company plans to file an Investigational New Drug (IND) application or similar for the ACcomplisH Infants Trial in subjects with achondroplasia (age 0–2).
TransCon TLR7/8 Agonist: TransCon TLR7/8 Agonist is an investigational long-acting prodrug of resiquimod, a small molecule agonist of Toll-like receptors (TLR) 7 and 8 designed to provide sustained activation of intratumoral antigen-presenting cells driving tumor antigen presentation and induction of immune stimulatory cytokines for weeks or months with a single intratumoral injection:
Enrollment continues in transcendIT-101. Top-line data from monotherapy and combo-therapy dose escalation expected in the third quarter of 2022.
TransCon IL-2 β/γ: TransCon IL-2 β/γ is an investigational long-acting prodrug designed to improve cancer immunotherapy by sustained exposure to an IL-2 variant that selectively activates the IL-2Rβ/γ, with minimal binding to IL-2Rα:
Top-line monotherapy data from the IL-βelieγe Trial are expected in the fourth quarter of 2022.
The Company expects to dose the first patient in the combo-therapy and dose escalation arm of the IL-βelieγe Trial in the first quarter of 2022.
TransCon TLR7/8 Agonist and TransCon IL-2 β/γ Combinations:
During the fourth quarter of 2022, the Company plans to submit an IND or similar for Phase 2 cohort expansion for TransCon TLR7/8 Agonist and TransCon IL-2 β/γ.
Presentation at J.P. Morgan Healthcare Conference on Monday, January 10th
The live webcast of the J.P. Morgan presentation will be available on the Investors & News section of the Ascendis Pharma website at www.ascendispharma.com. A webcast replay will be available on this website shortly after conclusion of the event for 30 days. The Company’s slides from the J.P. Morgan presentation also will also be available on the Investor Relations website.