854A (Kindeva Drug Deliver)

— Rollout of YORVIPATH® initiated in Germany and Austria, with an estimated 55 doctors writing prescriptions and ~100 patients receiving commercial product as of March 31 — TransCon™ PTH (palopegteriparatide) PDUFA date of May 14, 2024, for adults with hypoparathyroidism. If approved, U.S. launch planned in Q3 — TransCon CNP (navepegritide) pivotal ApproaCH Trial on track for topline results in Q4 2024 — SKYTROFA® Q1 revenue more than doubled year-over-year to €65 million; Q1 operating expenses fell by 20% year-over-year to €137 million — Ascendis remains on track for achieving full-year 2024 SKYTROFA revenue of €320 to €340 million and full year 2024 operating expenses of €600 million — Conference call today at 4:30 pm ET COPENHAGEN, Denmark, May 02, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced financial results for the first quarter ended March 31, 2024, and provided a business update. “With SKYTROFA revenue more than doubling in the U.S. compared to the first quarter of 2023, and our successful initial launch of YORVIPATH in Germany and Austria, we believe Ascendis is on the path to achieving sustainable growth and operating cash flow breakeven on a quarterly basis by the end of 2024,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Our achievements this quarter give me further confidence that all elements are in place to deliver three independent Endocrinology Rare Disease blockbuster products and a strong pipeline in larger therapeutic areas such as Oncology, Ophthalmology, and Metabolic Diseases as outlined in Vision 2030.” Select Highlights & Anticipated 2024 Milestones TransCon hGH:(lonapegsomatropin, approved as SKYTROFA in the U.S., EU, European Economic Area (EEA) countries, and Great Britain) First quarter 2024 SKYTROFA revenue totaled €65 million, a 106% year over year increase with a steady quarter to quarter increase in treated patients.Full year 2024 SKYTROFA revenue expected to be €320 million to €340 million (based on average 2023 exchange rates).Plan to submit a supplemental Biologics License Application to FDA for adult growth hormone deficiency (GHD), in the third quarter of 2024.Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024. TransCon PTH:(palopegteriparatide, approved as YORVIPATH in the EU, EEA countries, and Great Britain) Commercial rollout of YORVIPATH continues in Germany and Austria with an estimated 55 doctors writing prescriptions and ~100 patients receiving commercial product as of March 31. First quarter YORVIPATH revenue totaled €1.5 million reflecting first two months of delivery to patients.In the U.S., Prescription Drug User Fee Act (PDUFA) date of May 14, 2024; if approved, U.S. commercial launch planned in the third quarter of 2024.Granted marketing authorization for the treatment of adults with chronic hypoparathyroidism and orphan drug status in Great Britain. TransCon CNP:(navepegritide) Topline data from pivotal ApproaCH Trial expected in the fourth quarter of 2024, and plan to submit a New Drug Application to FDA for children with achondroplasia (age 2-11 years) in the same quarter.Expect to initiate and complete enrollment in the combination TransCon hGH and TransCon CNP COACH trial of children with achondroplasia (ages 2-11) during the second quarter of 2024; topline Week 26 data expected in the fourth quarter of 2024. Oncology Programs New data from the ongoing Phase 1/2 IL-Believe Trial has been accepted for a poster presentation at the American Society for Clinical Oncology (ASCO) May 31-June 4. The presentation will provide initial data from the combination of TransCon IL-2 β/γ and TransCon TLR7/8 Agonists in patients with melanoma who have progressed on anti-PD1 therapy.During the fourth quarter of 2024, plan to provide a clinical update from the Phase 2 indication-specific, dose expansion cohorts from our TransCon IL-2 β/γ and TransCon TLR7/8 Agonist clinical trials. Financial Update and Outlook Based on Current Plans As of March 31, 2024, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €320 million compared to €399 million as of December 31, 2023.Full year 2024 SKYTROFA revenue expected to be €320 million to €340 million (based on average 2023 exchange rates).Expect total operating expenses (SG&A and R&D) of approximately €600 million for 2024.Expect to be operating cash flow breakeven on a quarterly basis by the end of 2024. First Quarter 2024 Financial ResultsTotal revenue for the first quarter of 2024 was €95.9 million compared to €33.6 million during the same period for 2023. The increase was primarily attributable to higher SKYTROFA revenue of €65.0 million compared to €31.6 million in the same period last year and non-cash license revenue of €24.8 million related to the license agreement with Eyconis in January 2024. Total Revenue (In EUR'000s) Three Months Ended March 31, 2024 2023 Revenue from external customers Commercial sale of products 66,499 31,551 Licenses 24,770 614 Other 4,625 1,424 Total revenue from external customers 95,894 33,589 Research and development (R&D) costs for the first quarter of 2024 were €70.7 million compared to €106.1 million during the same period in 2023. The 33% decline was largely tied to lower external development costs for TransCon hGH, TransCon PTH (including a reversal of prior period write-downs of pre-launch inventories) and Oncology programs, partially offset by an increase in TransCon CNP costs. Selling, general, and administrative (SG&A) expenses for the first quarter of 2024 were €66.8 million compared to €66.5 million during the same period in 2023. Higher employee costs, including the impact from commercial expansion, partly offset by lower external pre-launch and administrative expenses. Total operating expenses for the first quarter of 2024 were €137.5 million compared to €172.7 million during the same period in 2023. Net finance expenses for the first quarter of 2024 were €73.6 million compared to a net finance income of €35.3 million during the same period in 2023. For the first quarter of 2024, Ascendis Pharma reported a net loss of €131.0 million, or €2.30 per share (basic and diluted) compared to a net loss of €110.9 million, or €1.98 per share (basic and diluted) for the same period in 2023. As of March 31, 2024, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €320.2 million compared to €399.4 million as of December 31, 2023. As of March 31, 2024, Ascendis Pharma had 58,224,419 ordinary shares outstanding, including 881,730 ordinary shares represented by ADSs held by the company. Conference Call and Webcast InformationAscendis Pharma will host a conference call and webcast today at 4:30 pm Eastern Time (ET) to discuss its first 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 https://investors.ascendispharma.com. 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. About Ascendis Pharma A/SAscendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of Patients, Science and Passion, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more. Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) TransCon PTH’s PDUFA date of May 14, 2024, (ii) the commercial launch of TransCon PTH in the U.S., if approved, (iii) the timing of topline results from the ApproaCH Trial, (iv) Ascendis’ expectations regarding full year 2024 revenue for SKYTROFA, (v) Ascendis’ ability to fulfill its strategic goals to deliver three independent Endocrinology Rare Disease blockbuster products and a strong pipeline in larger therapeutic areas such as Oncology, Ophthalmology, and Metabolic Diseases, (vi) Ascendis’ plan to submit a supplemental Biologics License Application for SKYTROFA for adult GHD in the third quarter of 2024, (vii) the timing of topline results from the Phase 2 trial in Turner syndrome, (viii) the timing of topline data from the ApproaCH Trial, (ix) Ascendis’ plan to submit a New Drug Application for TransCon CNP for children with achondroplasia, (x) Ascendis’ ability to initiate and complete enrollment in the COACH Trial, (xi) the timing of topline data from Week 26 of the COACH Trial, (xii) Ascendis’ plan to present initial data from the Phase 1/2 IL-Believe Trial, (xiii) Ascendis’ plan to provide a clinical update from the Phase 2 portion of indication-specific, dose expansion cohorts of the IL Believe trial, (xiv) Ascendis’ expectations regarding its total operating expenses for 2024, (xv) Ascendis’ expectation that it will be operating cash flow breakeven on a quarterly basis by the end of 2024, (xvi) Ascendis’ belief that it is on a path to achieving sustainable growth and that all elements are in place to deliver three independent Endocrinology Rare Disease blockbuster products, (xvii) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (xviii) Ascendis’ use of its TransCon technologies to create new and potentially best-in-class therapies. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 7, 2024 and Ascendis’ other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law. Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, TransCon, SKYTROFA®, and YORVIPATH® are trademarks owned by the Ascendis Pharma group. © May 2024 Ascendis Pharma A/S. Investor Contacts:Media Contact:Tim LeeMelinda BakerAscendis PharmaAscendis Pharma+1 (650) 374-6343+1 (650) 709-8875tle@ascendispharma.commedia@ascendispharma.comir@ascendispharma.com Patti Bank ICR Westwicke +1 (415) 513-1284 patti.bank@westwicke.com FINANCIAL TABLES FOLLOW Ascendis Pharma A/SConsolidated Statements of Profit or Loss and Comprehensive Income / (Loss)(In EUR'000s, except share and per share data)Three Months Ended March 31, 2024 2023 Consolidated Statement of Profit or Loss Revenue 95,894 33,589 Cost of sales 7,569 4,621 Gross profit 88,325 28,968 Research and development costs 70,687 106,114 Selling, general and administrative expenses 66,783 66,539 Operating profit/(loss) (49,145) (143,685)Share of profit/(loss) of associate (5,796) (1,227)Finance income 3,575 45,135 Finance expenses 77,161 9,840 Profit/(loss) before tax (128,527) (109,617)Income taxes/(expenses) (2,508) (1,297)Net profit/(loss) for the period (131,035) (110,914)Attributable to owners of the Company (131,035) (110,914)Basic and diluted earnings/(loss) per share€(2.30) €(1.98)Number of shares used for calculation (basic and diluted) 56,883,257 56,091,927 Consolidated Statement of Comprehensive Income or (Loss) Net profit/(loss) for the period (131,035) (110,914)Items that may be reclassified subsequently to profit or loss: Exchange differences on translating foreign operations 63 (787)Other comprehensive income/(loss) for the period, net of tax 63 (787)Total comprehensive income/(loss) for the period, net of tax (130,972) (111,701)Attributable to owners of the Company (130,972) (111,701) Ascendis Pharma A/SConsolidated Statements of Financial Position(In EUR'000s) March 31, 2024 December 31, 2023Assets Non-current assets Intangible assets 4,301 4,419 Property, plant and equipment 107,164 110,634 Investment in associates 24,797 5,686 Other receivables 2,129 2,127 138,391 122,866 Current assets Inventories 232,681 208,931 Trade receivables 41,092 35,874 Income tax receivables 742 802 Other receivables 26,857 19,097 Prepayments 42,502 38,578 Marketable securities — 7,275 Cash and cash equivalents 320,239 392,164 664,113 702,721 Total assets 802,504 825,587 Equity and liabilities Equity Share capital 7,818 7,749 Distributable equity (245,997) (153,446)Total equity (238,179) (145,697) Non-current liabilities Borrowings 229,627 222,996 Contract liabilities 5,000 5,949 Deferred tax liabilities 7,085 5,830 241,712 234,775 Current liabilities Convertible notes, matures in April 2028 Borrowings 424,984 407,095 Derivative liabilities 197,291 143,296 622,275 550,391 Other current liabilities Borrowings 14,403 14,174 Contract liabilities 1,183 1,184 Trade payables and accrued expenses 94,526 94,566 Other liabilities 22,698 41,176 Income tax payables 3,336 2,299 Provisions 40,550 32,719 176,696 186,118 798,971 736,509 Total liabilities 1,040,683 971,284 Total equity and liabilities 802,504 825,587

WASHINGTON, DC / ACCESSWIRE / April 3, 2024 / In a significant stride towards active immune therapy of cancer, a recently featured article in Experimental Biology and Medicine (Volume 249, Issue 1) describes a Phase 1 clinical trial, led by James Spicer and colleagues at King's College London. Entitled "A phase 1 trial of human telomerase reverse transcriptase (hTERT) vaccination combined with therapeutic strategies to control immune-suppressor mechanisms", this study showcases promising developments in the fight against solid tumours. The primary objective of the trial was to assess the safety and tolerability of the vaccine and adjuvants, with exploratory endpoints, including immune response and the detection of antigen-specific T cells, analysed by Shahram Kordasti's immunopathology team. The results demonstrate not only the safety and feasibility of this vaccination-mediated immune therapy for solid tumours but also the induction of immunological responses. Remarkably, the study demonstrated the clonal expansion of hTERT reactive T cells, as well as disease stabilization for at least six months in a quarter of therapy-resistant patients. Professor Spicer spearheaded the trial with a focus on addressing key challenges in cancer treatment; specifically, the difficulty in generating activated effector T cells and the suppression of inhibitory immune cells. The investigation involved patients with diverse multiply pre-treated solid tumours. The primary target was hTERT, known to be overexpressed in more than 90% of malignancies. The study employed a novel vaccination strategy. An hTERT-derived 7-peptide library, manufactured in-house at King's by Farzin Farzaneh's group, ensured presentation by both HLA class-I and class-II in 90% of a European population. Montanide adjuvant and a topical TLR-7 agonist were chosen to optimize antigen presentation. The combination of oral low-dose cyclophosphamide to modulate regulatory T cells, and celecoxib to counter cyclooxygenase-2-mediated immunosuppression, added a further potential boost to the therapeutic approach. Professor Spicer expressed his excitement, stating, "Our findings represent a significant advance in cancer immunotherapy. The safety and efficacy demonstrated in this Phase 1 trial underscore the potential of hTERT vaccination and immune-suppressor control strategies in reshaping cancer treatment." Dr. Steven R. Goodman, Editor-in-Chief of Experimental Biology and Medicine, commended the research, stating, "This Phase 1 trial unveils a promising frontier in cancer treatment. The outcomes suggest a paradigm shift in addressing immune-suppressive mechanisms, offering hope for improved therapeutic strategies for cancer patients." This study was funded by the generous support received from the Candles Charity. Experimental Biology and Medicine is a global journal dedicated to the publication of multidisciplinary and interdisciplinary research in the biomedical sciences. The journal was first established in 1903. Experimental Biology and Medicine is the journal of the Society of Experimental Biology and Medicine. To learn about the benefits of society membership, visit If you are interested in publishing in the journal, please visit Contact Information: Benjamin Zimmer bzimmer@sebm.org SOURCE: Experimental Biology and Medicine View the original press release on newswire.com.