On May 11, 2022 Ascendis Pharma A/S (Nasdaq: ASND) reported financial results for the first quarter ended March 31, 2022 and provided a business update (Press release, Ascendis Pharma, MAY 11, 2022, View Source [SID1234614212]).
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"This continues to be a transformative time for Ascendis as we build on positive Phase 3 data for our first two endocrinology rare disease programs – TransCon hGH and TransCon PTH – and our first product approval and launch," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "2022 marks a key transition point for Ascendis. We have all the elements of success in place – a proven technology and algorithm for product innovation, the right capabilities, and a strong balance sheet that allows us to deliver on all components of our Vision 3×3 as we work to achieve sustainable growth through multiple approaches and long-term profitability."
Company Highlights & Progress
TransCon hGH:
TransCon hGH is commercially available in the U.S. under the brand name SKYTROFA (lonapegsomatropin-tcgd) as the only FDA-approved once-weekly treatment for 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. As of April 29, 2022, 1,231 unique patient prescriptions for SKYTROFA have been written by 404 prescribers and processed through Ascendis’ patient hub called the Ascendis Signature Access Program. Nearly 50% of the prescribers have written prescriptions for more than one patient.
Coverage for SKYTROFA continues to increase, with approximately 45% of U.S. lives covered at the end of April per MMIT, compared to approximately 36% at the end of February.
In January 2022, the Company received marketing authorization for Lonapegsomatropin Ascendis Pharma (developed under the brand name of TransCon hGH) in the European Union as a once-weekly subcutaneous injection for the treatment of children and adolescents ages 3 to 18 years with growth failure due to insufficient secretion of endogenous growth hormone.
On track for FDA protocol submission during the second quarter of 2022 to evaluate TransCon hGH for Turner Syndrome.
The ongoing conflict in the region surrounding Ukraine, Russia, and Belarus has impacted our ability to conduct clinical trials or to treat and evaluate patients there. As a result, we have shifted focus for our foresiGHt Trial in adult growth hormone deficiency to other countries and are now targeting completion of enrollment during the fourth quarter of 2022.
TransCon PTH:
On March 13, 2022, we announced top-line data from a randomized, double-blind, placebo-controlled Phase 3 Trial (PaTHway) of TransCon PTH in adults with hypoparathyroidism (HP), demonstrating statistically significant improvement with TransCon PTH compared to control for the primary composite endpoint and all key secondary endpoints. As of May 1, 2022, all 79 patients continued in the open-label extension portion of the PaTHway Trial.
On track for planned U.S. NDA submission during the third quarter of 2022 and expected EU MAA submission during the fourth quarter of 2022.
After more than two years of treatment in the open-label extension portion of the PaTH Forward Trial, as of May 1, 2022, 57 out of 59 original subjects continue in the trial.
In April 2022, enrollment completed in the Phase 3 PaTHway Japan Trial. Top-line results expected later this year.
TransCon CNP:
Top-line data from the ACcomplisH Trial, a Phase 2 randomized, double-blind, placebo-controlled clinical trial in North America, Europe, and Oceania in children ages 2-10 years with achondroplasia are expected in the fourth quarter of 2022.
TransCon TLR7/8 Agonist:
Enrollment continues in transcendIT-101, a Phase 1/2 study of TransCon TLR7/8 Agonist with or without pembrolizumab in patients with advanced or metastatic solid tumors. We expect top-line monotherapy and combo-therapy dose escalation data during the third quarter of 2022.
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. Top-line data are expected in the fourth quarter of 2022.
On track to dose the first patient in checkpoint combination dose-escalation arm of the IL-βelieγe Trial in the second quarter of 2022.
TransCon TLR7/8 Agonist and TransCon IL-2 β/γ Combination:
We plan to submit an IND or similar for Phase 2 cohort expansion for TransCon TLR7/8 Agonist and TransCon IL-2 β/γ during the fourth quarter of 2022.
Ended the first quarter of 2022 with cash, cash equivalents, and marketable securities totaling €1,065 million.
First Quarter 2022 Financial Results
Total revenue for the first quarter was €6.8 million, an increase of €6.1 million compared to €0.7 million in the same quarter of 2021. Revenues for the first quarter include U.S. commercial SKYTROFA sales, sales of clinical supplies, rendering of services, and recognition of internal profit from the license agreements with VISEN. The increase in revenue was primarily attributable to the €1.9 million commercial SKYTROFA sales, and €3.9 million higher sales of clinical supply to VISEN compared to the same period last year.
Research and development (R&D) costs for the first quarter were €83.2 million compared to €88.1 million during the same period in 2021. Following FDA approval of SKYTROFA (lonapegsomatropin-tcgd) in August 2021, commercial manufacturing is recognized as inventory while such costs were recognized as R&D costs prior to the FDA approval.
Selling, general, and administrative (SG&A) expenses for the first quarter were €47.4 million compared to €37.2 million during the same period in 2021. Higher SG&A expenses were primarily due to an increase in commercial and administrative personnel costs as well as an increase in commercial costs.
Net loss of associate was €4.9 million in the first quarter, compared to a net profit of €28.1 million during the same period in 2021.
Net finance income was €7.6 million in the first quarter compared to €33.6 million in the same period in 2021.
For the first quarter of 2022, Ascendis Pharma reported a net loss of €125.5 million, or €2.21 per share (basic and diluted) compared to a net loss of €62.8 million, or €1.17 per share (basic and diluted) for the same period in 2021.
As of March 31, 2022, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €1,065 million compared to €790 million as of December 31, 2021. As of March 31, 2022, Ascendis Pharma had 56,958,391 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 first quarter 2022 financial results. Details include:
A live webcast of the conference call will be accessible from the Investors and News section of the Ascendis Pharma website at www.ascendispharma.com. A replay of the webcast will be available on this website shortly after conclusion of the event for 30 days.