Ascendis Pharma Reports Second Quarter 2023 Financial Results
Ascendis Pharma announces Q2 2023 financial results and business updates
Positive
Raising full year 2023 U.S. SKYTROFA revenue expectations to €165 - €170 million
FDA approval of Lonza as a high-capacity drug substance manufacturing site to support worldwide demand of SKYTROFA
TransCon PTH NDA to be resubmitted to FDA in October 2023
EU approval for TransCon PTH expected in Q4 2023
TransCon PTH-treated patients showed substantial increases in eGFR, indicating improved kidney function
Negative
Negative adjustment to provision for estimated sales rebates related to SKYTROFA revenue from prior periods
Negative foreign currency impact due to weaker U.S. dollar
09/05/2023 - 04:03 PM
Expect to resubmit NDA to FDA for TransCon™ PTH in October 2023; EC decision on MAA for TransCon PTH expected during the fourth quarter of 2023 U.S. and EU regulatory authorities endorsed ApproaCH as a pivotal Phase 3 trial of TransCon CNP in children with achondroplasia; enrollment completed, and topline results expected in second half of 2024 Raising full year 2023 U.S. SKYTROFA ® * revenue expectations to €165 - €170 million Conference call today at 4:30 pm ET COPENHAGEN, Denmark, Sept. 05, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced financial results for the second quarter ended June 30, 2023 and provided business updates.
“We have built a fully integrated Endocrinology Rare Disease franchise with a growing commercial reach and are on our way to achieving our Vision 3x3, including regulatory approval for three differentiated product candidates by 2025,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “With a proven U.S. and expanding global commercial infrastructure positioned to capture the multi-billion-dollar opportunities that each represents, coupled with an R&D engine for label expansion and future innovation for new patient populations, Ascendis has many routes to realize long-term profitability and sustainability.”
Corporate Highlights
TransCon hGH (marketed in the U.S. as SKYTROFA): Second quarter 2023 SKYTROFA revenue totaled €36 million , and the Company is increasing full year 2023 U.S. SKYTROFA revenue expectations from €150 - €160 million to €165 - €170 million . Second quarter 2023 SKYTROFA revenue included a negative adjustment to provision for estimated sales rebates related to sales from prior periods of €2.1 million as a result of expanding payer coverage and a negative foreign currency impact of €0.6 million compared to the first quarter of 2023, due to a weaker U.S. dollar. Q2-2022 Q3-2022 Q4-2022 Q1-2023 Q2-2023 SKYTROFA revenue (millions) €4.4 €12.3 €17.1 €31.6 €35.9
Based on reported revenues, SKYTROFA was the leading U.S. growth hormone product in the second quarter, with a market share of less than 10% of treated U.S. pediatric GHD patients. First commercial launch of SKYTROFA in the EU expected in September in Germany. Received FDA approval of Lonza as a high-capacity drug substance manufacturing site to support worldwide demand of SKYTROFA. Expect EU approval in first half of 2024. Topline results from Phase 3 foresiGHt Trial in adult growth hormone deficiency investigating the metabolic impact of TransCon hGH expected in the fourth quarter of 2023, opening up the first label expansion opportunity. TransCon PTH: In June 2023, the Company requested a Type A meeting with FDA and submitted an updated control strategy. The Company’s Type A meeting was held with FDA in late August based on the Agency’s availability. Following a constructive Type A meeting, the Company submitted additional information to FDA supporting the updated control strategy. Ascendis believes the materials submitted to FDA combined with the Type A meeting discussions will position the Company to resubmit the NDA for TransCon PTH for adults with hypoparathyroidism in October 2023. Remain on track for European Commission decision on Marketing Authorisation Application (MAA) for TransCon PTH during the fourth quarter of 2023. If approved, first country launch planned in Germany in early 2024. New post hoc analysis showed TransCon PTH-treated patients demonstrated substantial increases in estimated glomerular filtration rate (eGFR), indicating improved kidney function. More information on this analysis can be found in a separate press release issued today and available here on the Investors & News section of the Ascendis Pharma website. 145 out of 154 participants continue in the open-label extension (OLE) portions of the Phase 2 PaTH Forward, Phase 3 PaTHway, and PaTHway Japan trials. The U.S. Expanded Access Program and German Compassionate Use Program continue to be open for enrollment of eligible patients. TransCon CNP: U.S. and EU regulatory agencies have endorsed ApproaCH, a global randomized, double-blind, placebo-controlled trial in children ages 2–11 years with achondroplasia, as a pivotal Phase 3 trial. Enrollment is complete and topline results are expected in the second half of 2024. All 57 patients remain in the OLE portion of the Phase 2 ACcomplisH Trial, with treatment duration up to 3 years. One-year follow-up data from OLE expected in the fourth quarter of 2023. TransCon TLR7/8 Agonist: Enrollment continues in the Phase 2 portion of transcendIT-101 at the recommended Phase 2 dose (RP2D) in four indication-specific cohorts. Topline/interim analysis from Phase 2 dose-expansion cohorts expected in 2024. TransCon IL-2 β/γ Completed Phase 1 dose escalation in combination with pembrolizumab in the Phase 1/2 IL-Believe Trial with a total of 21 patients enrolled. RP2D was determined at 120 µg/kg IV every three weeks. No dose-limiting toxicity, vascular leak syndrome, or grade 3 or 4 cytokine release syndrome was observed at any dose level evaluated. Enrollment initiated in the Phase 2 portion in indication-specific cohorts, with expected topline/interim analysis from Phase 2 dose-expansion cohorts expected in 2024. Expanding leadership in long-acting prodrug technologies with new TransCon technology carrier platform to unlock large market opportunities where high volume and low-cost manufacturing is required. Proof-of-principle demonstrated for GLP-1 analogs (semaglutide), with data supporting potential best-in-class weekly and monthly administration profiles. More information on the pre-clinical data can be found in a presentation here on the Investors & News section of the Ascendis Pharma website. Ended the second quarter of 2023 with cash, cash equivalents, and marketable securities totaling €431.1 million . Subsequent to the quarter end, entered into a $150 million capped synthetic royalty funding agreement with Royalty Pharma. 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 2023 Financial Results
Total revenue for the second quarter of 2023 was €47.4 million compared to €6.2 million during the same period in 2022. The increase was primarily attributable to higher SKYTROFA revenue of €35.9 million compared to €4.4 million in the same period last year.
Research and development (R&D) costs for the second quarter were €105.0 million compared to €90.4 million during the same period in 2022. This increase was primarily due to higher development costs for Ascendis Pharma Oncology programs (TransCon IL-2 β/γ and TransCon TLR7/8 Agonist), increasing clinical trial activities for TransCon CNP, and higher employee-related costs, and was partly offset by lower development costs for TransCon hGH.
Selling, general, and administrative (SG&A) expenses for the second quarter were €70.3 million compared to €56.6 million during the same period in 2022. This increase was primarily due to higher external commercial expenses related to SKYTROFA, pre-launch activities for SKYTROFA outside the U.S., global pre-launch activities for TransCon PTH, higher employee related expenses, and an increase in other general and administrative expenses attributable to organizational growth.
Net finance income was €26.4 million in the second quarter compared to €61.7 million in the same period in 2022.
For the second quarter of 2023, Ascendis Pharma reported a net loss of €121.4 million , or €2.16 per share (basic and diluted) compared to a net loss of €81.3 million , or €1.46 per share (basic and diluted) for the same period in 2022.
As of June 30, 2023, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €431.1 million compared to €742.9 million as of December 31, 2022. As of June 30, 2023, Ascendis Pharma had 57,335,496 ordinary shares outstanding.
Conference Call and Webcast Information
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 https://investors.ascendispharma.com . A replay of the webcast will be available on this section of our website shortly after conclusion of the event for 30 days.
About Ascendis Pharma A/S ascendispharma.com to learn more.
Forward-Looking Statements
Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, TransCon, and SKYTROFA ® are trademarks owned by the Ascendis Pharma group. © September 2023 Ascendis Pharma A/S.
FINANCIAL TABLES FOLLOW
Ascendis Pharma A/S Consolidated Statements of Profit or Loss and Comprehensive Income / (Loss) (In EUR’000s, except share and per share data) Three Months ended June 30, Six Months ended June 30, 2023 2022 2023 2022 Revenue 47,393 6,160 80,982 12,988 Cost of sales 12,929 1,086 17,551 5,332 Gross profit 34,464 5,074 63,431 7,656 Research and development costs 105,021 90,383 211,134 173,576 Selling, general and administrative expenses 70,281 56,584 136,820 104,002 Operating profit / (loss) (140,838 ) (141,893 ) (284,523 ) (269,922 ) Share of profit / (loss) of associate (7,451 ) (1,166 ) (8,677 ) (6,039 ) Finance income 35,761 71,127 80,374 84,171 Finance expenses 9,334 9,434 18,652 14,833 Profit / (loss) before tax (121,862 ) (81,366 ) (231,478 ) (206,623 ) Income taxes (expenses) 429 47 (868 ) (195 ) Net profit / (loss) for the period (121,433 ) (81,319 ) (232,346 ) (206,818 ) Attributable to owners of the Company (121,433 ) (81,319 ) (232,346 ) (206,818 ) Basic and diluted earnings / (loss) per share € (2.16 ) € (1.46 ) € (4.14 ) € (3.68 ) Number of shares used for calculation (basic and diluted) 56,218,257 55,805,486 56,155,441 56,260,248 Net profit / (loss) for the period (121,433 ) (81,319 ) (232,346 ) (206,818 ) Other comprehensive income / (loss) Items that may be reclassified subsequently to profit or loss: Exchange differences on translating foreign operations (1,016 ) (757 ) (1,803 ) (332 ) Other comprehensive income / (loss) for the period, net of tax (1,016 ) (757 ) (1,803 ) (332 ) Total comprehensive income / (loss) for the period, net of tax (122,449 ) (82,076 ) (234,149 ) (207,150 ) Attributable to owners of the Company (122,449 ) (82,076 ) (234,149 ) (207,150 )
Ascendis Pharma A/S Consolidated Statements of Financial Position (In EUR’000s) June 30, December 31, 2023 2022 Assets Non-current assets Intangible assets 4,606 4,828 Property, plant and equipment 125,362 129,095 Investment in associate 14,111 22,932 Other receivables 2,066 1,920 Marketable securities - 7,492 146,145 166,267 Current assets Inventories 167,919 130,673 Trade receivables 20,212 11,910 Income tax receivable 1,360 883 Other receivables 14,127 12,833 Prepayments 42,958 31,717 Marketable securities 36,880 290,688 Cash and cash equivalents 394,222 444,767 677,678 923,471 Total assets 823,823 1,089,738 Equity and liabilities Equity Share capital 7,699 7,675 Distributable equity 57,142 255,673 Total equity 64,841 263,348 Non-current liabilities Borrowings 479,374 482,956 Derivative liabilities 86,385 157,950 Contract liabilities 949 14,213 566,708 655,119 Current liabilities Borrowings 26,564 25,421 Contract liabilities 4,146 - Trade payables and accrued expenses 122,120 101,032 Other liabilities 22,860 31,989 Income tax payables 5,773 5,490 Provisions 10,811 7,339 192,274 171,271 Total liabilities 758,982 826,390 Total equity and liabilities 823,823 1,089,738
* Registered in the U.S. as SKYTROFA ® (longapegsomatropin-tcgd) and in the EU as SKYTROFA ® (lonapegsomatropin). SKYTROFA ® is not marketed in the EU.
