On September 3, 2024 Ascendis Pharma A/S (Nasdaq: ASND) reported financial results for the second quarter ended June 30, 2024, and provided a business update (Press release, Ascendis Pharma, SEP 3, 2024, View Source [SID1234646284]).
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"The recent FDA approval of YORVIPATH demonstrates why our unrelenting focus on helping patients suffering from hypoparathyroidism and other serious diseases with considerable unmet need is so important for Ascendis," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "Already, two out of our three Endocrine Rare Disease TransCon product candidates have been approved by the FDA and European Commission. Our first, SKYTROFA, has achieved U.S. market value leadership, and, we believe, now with broader market access, remains well positioned to reach blockbuster status in the U.S. alone. Looking forward, we are preparing for our second U.S. launch with YORVIPATH and are on track to report pivotal data in the coming weeks for our third TransCon product candidate, TransCon CNP."
Select Highlights & Anticipated 2024 Milestones
TransCon hGH:
(lonapegsomatropin, marketed as SKYTROFA)
SKYTROFA revenue for the second quarter of 2024 totaled €26.2 million, a 27% year-over-year decrease compared to €35.9 million during the same period in 2023. 134% year-over-year volume growth was offset by the cost associated with broader market access for SKYTROFA which also resulted in a negative adjustment to prior period sales deductions of €27.1 million, where €19.5 million and €7.6 million were attributable to the three months ended March 31, 2024, and periods prior to January 1, 2024, respectively.
SKYTROFA revenue for the first half of 2024 totaled €91.2 million, a 35% year-over-year increase compared to €67.4 million during the same period of 2023. 159% year-over-year volume growth was offset by the cost associated with broader market access for SKYTROFA which also resulted in a negative adjustment to prior period sales deductions of €7.6 million, which were attributable to periods prior to January 1, 2024.
On track to submit a supplemental Biologics License Application to the FDA for adult growth hormone deficiency in the third quarter of 2024.
Topline results from Phase 2 New InsiGHTS Trial in Turner syndrome expected in the fourth quarter of 2024.
TransCon PTH:
(palopegteriparatide, marketed as YORVIPATH)
Received U.S. FDA approval for TransCon PTH, under the brand name YORVIPATH, for the treatment of hypoparathyroidism in adults.
Completing manufacturing of commercial product for the U.S. market and anticipate initial supply will be available in the first quarter of 2025. The Company is in dialogue with the FDA about commercialization of existing manufactured product, which if agreed, could be introduced in the U.S. in the fourth quarter of 2024.
Second quarter YORVIPATH revenue totaled €5.2 million, reflecting the first full quarter of commercial launch in Germany and Austria as well initial revenue in International Markets. Initial revenue in France expected starting in the fourth quarter of 2024.
TransCon CNP
(navepegritide)
Topline data from pivotal ApproaCH Trial expected in the coming weeks, and, if successful, plan to submit a New Drug Application to FDA for children with achondroplasia (age 2-11 years) in the first quarter of 2025.
Plan to complete enrollment in the combination TransCon hGH and TransCon CNP COACH trial of children with achondroplasia (ages 2-11 years) during the third quarter of 2024; topline Week 26 data expected in the second quarter of 2025.
Expect to initiate teACH, a Phase 2 trial in adolescents with achondroplasia, in the fourth quarter of 2024.
Oncology Programs
Presented new and updated results from the ongoing Phase 1/2 IL-Believe Trial of TransCon IL-2 β⁄γ in a poster presentation at ASCO (Free ASCO Whitepaper) 2024. As of the April 16, 2024, data cutoff, 40% of efficacy-evaluable patients (2 out of 5) in the initial cohort of patients with anti-PD-1 refractory melanoma treated with TransCon IL-2 β⁄γ in combination with TransCon TLR7/8 Agonist exhibited confirmed clinical responses with no new safety signals.
Initial results from the Phase 2 dose expansion cohort of the IL-Believe Trial of TransCon IL-2 β⁄γ in combination with chemotherapy in platinum-resistant ovarian cancer (PROC) will be presented later this month at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2024 Congress in Barcelona, Spain.
Financial Update and Outlook Based on Current Plans
As of June 30, 2024, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €259 million, compared to €399 million as of December 31, 2023.
Full year 2024 SKYTROFA revenue expected to be €220 million to €240 million.
Expect total operating expenses (SG&A and R&D) to be approximately €600 million for 2024.
Pending launch timing of YORVIPATH in the U.S., expect to be operating cash flow breakeven on a quarterly basis in 2024 or 2025
Subsequent to the quarter end, entered into a $150 million capped synthetic royalty funding agreement with Royalty Pharma relating to net sales of YORVIPATH in the United States. More information on this funding can be found in a separate press release issued today and available here on the Investors & News section of the Ascendis Pharma website.
Second Quarter 2024 Financial Results
Total revenue for the second quarter of 2024 was €36.0 million, compared to €47.4 million during the same period for 2023. Results in the quarter were primarily impacted by a negative adjustment to prior periods’ estimates and assumptions for sales deductions of €27.1 million, where €19.5 million and €7.6 million were attributable to the three months ended March 31, 2024, and periods prior to January 1, 2024, respectively. This was partially offset by increased demand for SKYTROFA in the U.S. and revenue contribution from YORVIPATH. In addition, non-product revenue was €4.6 million in the second quarter of 2024, compared to €11.5 million during the same period for 2023.
Total Revenue
(In EUR’000s) Three Months Ended
June 30, Six Months Ended
June 30,
2024 2023 2024 2023
Revenue from external customers
Commercial sale of products 31,389 35,895 97,888 67,446
Licenses 869 589 25,639 1,203
Other 3,740 10,909 8,365 12,333
Total revenue from external customers 35,998 47,393 131,892 80,982
Research and development (R&D) costs for the second quarter of 2024 were €83.5 million, compared to €105.0 million during the same period in 2023. The decline was largely tied to lower external development costs for TransCon TLR 7/8 Agonist and lower costs for TransCon PTH, as well as lower employee costs as a result of the Eyconis spin-off.
Selling, general, and administrative (SG&A) expenses for the second quarter of 2024 were €74.3 million, compared to €70.3 million during the same period in 2023. The increase was primarily due to higher employee costs, including the impact from commercial expansion.
Total operating expenses for the second quarter of 2024 were €157.8 million compared to €175.3 million during the same period in 2023.
Net finance income for the second quarter of 2024 was €29.4 million compared to a net finance income of €26.4 million during the same period in 2023.
For the second quarter of 2024, Ascendis Pharma reported a net loss of €109.4 million, or €1.91 per share (basic and diluted) compared to a net loss of €121.4 million, or €2.16 per share (basic and diluted) for the same period in 2023.
As of June 30, 2024, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €258.7 million compared to €399.4 million as of December 31, 2023. As of June 30, 2024, Ascendis Pharma had 58,231,484 ordinary shares outstanding, including 881,730 ordinary shares represented by ADSs held by the company.
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 second quarter 2024 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 the Ascendis Pharma website shortly after conclusion of the event for 30 days.