On February 16, 2023 Ascendis Pharma A/S (Nasdaq: ASND) reported financial results for the full year ended December 31, 2022 and provided a business update (Press release, Ascendis Pharma, FEB 16, 2023, View Source [SID1234627326]).

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"Our unique TransCon technology platform and algorithm for product innovation enables us to address major unmet medical needs with a diverse, growing pipeline of highly differentiated product candidates," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "With our demonstrated ability to take a product from concept through approval and launch, we will continue to focus on building long term value for patients and other stakeholders, establishing Ascendis Pharma as a leading sustainable, profitable biopharma company."

Select Highlights & Anticipated 2023 Milestones

TransCon hGH:
First European SKYTROFA (lonapegsomatropin) commercial launch in Germany on track for the third quarter of 2023.
In the third quarter of 2023, we anticipate completing enrollment in New InsiGHTS, a four-armed Phase 2 trial designed to investigate the safety, tolerability, and efficacy of different dose levels of TransCon hGH in patients with Turner Syndrome.
We expect Phase 3 topline results from foresiGHt in adult growth hormone deficiency in the fourth quarter of 2023.
Fourth quarter 2022 SKYTROFA (lonapegsomatropin-tcgd) U.S. revenue grew to €17.1 million.
TransCon PTH:
U.S. FDA Priority Review continues for use in adult patients with hypoparathyroidism, with a PDUFA date of April 30, 2023. If approved, U.S. commercial launch expected by the end of the second quarter of 2023.
European Commission decision on MAA anticipated during the fourth quarter of 2023. If approved, first European country launch expected in early 2024.
In anticipation of U.S. and EU approvals, commercial, medical affairs, product supply and other teams continue launch readiness activities.
Phase 3 PaTHway Japan trial achieved its primary objectives; topline results consistent with North American and EU trials.
Enrollment opened in January 2023 for U.S. Expanded Access Program.
Q1-2022 Q2-2022 Q3-2022 Q4-2022 2022
SKYTROFA revenue (millions) € 1.9 € 4.4 € 12.3 € 17.1 € 35.7

TransCon CNP:
Announced positive topline data from the Phase 2 ACcomplisH Trial, with results in children with achondroplasia down to 2 years of age; as of February 14, 2023 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 plan to submit an IND or similar in children under the age of two years with achondroplasia.
TransCon TLR7/8 Agonist:
Reported topline data and recommended Phase 2 dose from the dose escalation portion of the Phase 1/2 transcendIT-101 Trial. Early signs of clinical activity were observed in patients receiving TransCon TLR7/8 Agonist as monotherapy or in combination with pembrolizumab.
Enrollment in the dose expansion phase of transcendIT-101 continues, with a focus on investigating TransCon TLR7/8 Agonist in combination with pembrolizumab in four different 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. Dose escalation combination therapy results expected during the third quarter of 2023.
Preparing to initiate βelieγe-IT-201, a randomized Phase 2 trial of TransCon IL-2 β/γ and TLR7/8 combination therapies, in the second quarter of 2023.
TransCon RBZ:
Ophthalmology selected as the third therapeutic area; TransCon RBZ (ranibizumab) selected as the first investigational pipeline candidate, designed for higher efficacy with 6-month dosing intervals.
Ended the fourth quarter of 2022 with cash, cash equivalents, and marketable securities totaling €742.9 million.
Full-Year 2022 Financial Results

Total revenue for 2022 was €51.2 million compared to €7.8 million in 2021. Revenue for 2022 include SKYTROFA U.S. revenue, and license, clinical supply and services provided to third parties, primarily VISEN Pharmaceuticals. Revenue in 2022 benefited from a full-year contribution of SKYTROFA U.S. revenue of €35.7 million compared to €0.9 million in 2021.

Research and development (R&D) costs for 2022 were €379.6 million compared to €295.9 million in 2021. The higher R&D costs in 2022 reflect a one-time reversal of pre-launch inventories in 2021, following the U.S. FDA approval of SKYTROFA in August 2021. In addition, higher R&D costs in 2022 reflect manufacturing of pre-launch inventories for TransCon PTH and an increase in employee and other costs attributable to organizational growth.

Selling, general, and administrative (SG&A) expenses for 2022 were €221.2 million compared to €160.2 million in 2021. Higher SG&A expenses were primarily due to an increase in commercial and administrative personnel following the launch of SKYTROFA in the U.S. and preparation for future product launches.

Our share of net loss of associate was €17.7 million in 2022, compared to a net gain of €12.0 million in 2021. For 2021, the net profit of associate included a non-cash gain of €42.3 million as a result of a financing round in VISEN.

Net finance income was €1.7 million in 2022 compared to a net finance income of €55.8 million in 2021.

For the full year 2022, Ascendis Pharma reported a net loss of €583.2 million, or €10.40 per share (basic and diluted) compared to a net loss of €383.6 million, or €7.00 per share (basic and diluted) for the same period in 2021.

As of December 31, 2022, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €742.9 million compared to €789.6 million as of December 31, 2021. As of December 31, 2022, Ascendis Pharma had 57,152,295 ordinary shares outstanding.

Conference Call and Webcast Information

Ascendis Pharma will host a conference call and webcast today at 4:30 pm Eastern Time (ET) to discuss its full year 2022 financial results.

Those who would like to participate may access the live webcast here, or register in advance for the teleconference here. The link to the live webcast will also be available on the Investors & News section of the Ascendis Pharma website at View Source A replay of the webcast will be available on this section of our website shortly after conclusion of the event for 30 days.