Palopegteriparatide

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 2023U.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 2024Raising full year 2023 U.S. SKYTROFA®* revenue expectations to €165 - €170 millionConference 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. Q2-2022Q3-2022Q4-2022Q1-2023Q2-2023SKYTROFA 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 Ascendis Pharma will host a conference call and webcast today at 4:30 pm Eastern Time (ET) to discuss its second quarter 2023 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 our 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, the company uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Germany (Heidelberg, Berlin and Munich) and the United States (Palo Alto and Redwood City, California, and Princeton, New Jersey). Please visit ascendispharma.com to learn more. Forward-Looking StatementsThis 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) Ascendis’ plan to resubmit an NDA for TransCon PTH in October 2023; (ii) the timing and results of the EC decision on the TransCon PTH MAA; (iii) the timing of topline results of the ApproaCH trial; (iv) Ascendis’ expectations regarding 2023 SKYTROFA revenues in the United States; (v) Ascendis’ ability to achieve its Vision 3x3; (vi) Ascendis’ expectations regarding regulatory approval for three independent product candidates by 2025; (vii) Ascendis’ ability to capture the opportunities presented by its product candidates and realize long-term profitability and sustainability; (viii) Ascendis’ SKYTROFA revenue expectations; (ix) Ascendis’ expectations regarding the launch of SKYTROFA in Germany; (x) Ascendis’ expectations regarding EU approval at Lonza for a high-capacity drug substance manufacturing site; (xi) the timing of Topline results from Phase 3 foresiGHt trial and the potential for the first label expansion opportunity; (xii) Ascendis’ expectations regarding the launch of TransCon PTH in Germany; (xiii) the timing of topline results from the ApproaCH trial; (xiv) the timing of one-year follow-up data from the OLE portion of the Phase 2 ACcomplisH trials; (xv) the timing of topline/interim analysis from Phase 2 dose-expansion cohorts of transcendIT-101 and IL-Believe trials; (xvi) the ability of the new TransCon technology carrier platform to unlock large market opportunities where high volume and low-cost manufacturing is required; (xvii) the potential best-in-class weekly and monthly administration profiles of Ascendis’ new TransCon technology carrier platform; (xviii) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated global biopharma company; and (xix) 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, including inflation, the effects on its business from the worldwide COVID-19 pandemic and ongoing conflicts such as that in the region surrounding Ukraine and Russia. 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 16, 2023 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, 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 Revenue47,393 6,160 80,982 12,988 Cost of sales12,929 1,086 17,551 5,332 Gross profit34,464 5,074 63,431 7,656 Research and development costs105,021 90,383 211,134 173,576 Selling, general and administrative expenses70,281 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 income35,761 71,127 80,374 84,171 Finance expenses9,334 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 assets4,606 4,828 Property, plant and equipment125,362 129,095 Investment in associate14,111 22,932 Other receivables2,066 1,920 Marketable securities- 7,492 146,145 166,267 Current assets Inventories167,919 130,673 Trade receivables20,212 11,910 Income tax receivable1,360 883 Other receivables14,127 12,833 Prepayments42,958 31,717 Marketable securities36,880 290,688 Cash and cash equivalents394,222 444,767 677,678 923,471 Total assets823,823 1,089,738 Equity and liabilities Equity Share capital7,699 7,675 Distributable equity57,142 255,673 Total equity64,841 263,348 Non-current liabilities Borrowings479,374 482,956 Derivative liabilities86,385 157,950 Contract liabilities949 14,213 566,708 655,119 Current liabilities Borrowings26,564 25,421 Contract liabilities4,146 - Trade payables and accrued expenses122,120 101,032 Other liabilities22,860 31,989 Income tax payables5,773 5,490 Provisions10,811 7,339 192,274 171,271 Total liabilities758,982 826,390 Total equity and liabilities823,823 1,089,738 Investor Contacts:Media Contact:Tim LeeMelinda BakerAscendis PharmaAscendis Pharma+1 (650) 374-6343+1 (650) 709-8875tle@ascendispharma.com media@ascendispharma.com ir@ascendispharma.com Patti Bank ICR Westwicke +1 (415) 513-1284 patti.bank@westwicke.com * Registered in the U.S. as SKYTROFA® (longapegsomatropin-tcgd) and in the EU as SKYTROFA® (lonapegsomatropin). SKYTROFA® is not marketed in the EU.

- Oral presentation of results showed sustained improvements through Week 52, as well as safety and tolerability similar to that previously reported for the initial 26-week blinded portion of the Phase 3 PaTHway Trial - At Week 52, 95% of patients treated with TransCon PTH achieved independence from conventional therapy - TransCon PTH treatment improved mean patient-reported disease symptom and health-related quality of life scores, starting at the first scheduled follow up after randomization or after switching from placebo and sustained through Week 52 Ascendis Pharma A/S (Nasdaq: ASND) today reported one-year (Week 52) data from its ongoing Phase 3 PaTHway Trial of TransCon PTH in adults with hypoparathyroidism. The data showed that treatment with TransCon PTH resulted in sustained improvements through Week 52, as well as safety and tolerability similar to that reported for the initial 26-week blinded portion of the trial. The data were presented by Bart Clarke, M.D., endocrinologist and Professor of Medicine at the Mayo Clinic (Rochester, MN), during ENDO 2023, the annual meeting of the Endocrine Society being held in Chicago. “We are very pleased to see sustained improvements in clinical outcomes in this trial, including symptom and health-related quality of life measures, consistent with those reported earlier for the initial 26-week blinded portion,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “These clinical and patient-reported data, and the retention of 145 patients in our ongoing clinical trials, reinforces our confidence that TransCon PTH has the potential, if approved, to benefit adults with hypoparathyroidism regardless of disease etiology.” Methods PaTHway is a Phase 3 trial of TransCon PTH with a placebo (PBO)-controlled 26-week blinded portion and a 156-week open-label extension (OLE) portion, designed to evaluate the long-term efficacy and safety of TransCon PTH as a potential hormone therapy for those diagnosed with hypoparathyroidism. Results through Week 52 (26 weeks blinded + 26 weeks OLE) were reported at ENDO 2023. Of the 82 study participants dosed, 79 completed blinded treatment and entered the OLE, and 78 (59 TransCon PTH/TransCon PTH, 19 PBO/TransCon PTH) completed Week 52. Week 52 Highlights 95% of patients in the OLE (74 out of 78) achieved independence from conventional therapy (defined as no active vitamin D and calcium supplements of 600mg/day), and none required active vitamin D. At Week 52, 81% of participants treated with TransCon PTH achieved both normal serum calcium and independence from conventional therapy. With TransCon PTH treatment, mean albumin-adjusted serum calcium levels were maintained within the normal range (8.3–10.6 mg/dL) through Week 52 of the OLE (8.9 mg/dL at Week 52). Patient-reported scores on the Hypoparathyroidism Patient Experience Scale (HPES) and SF-36 Health Survey showed sustained improvements in disease-related physical and cognitive symptoms, as well as physical functioning and daily life, starting at the first scheduled follow up after randomization or switching from placebo and sustained through Week 52. Bone mineral density (BMD) Z-scores continued to trend toward age- and sex-matched norms with 52 weeks of TransCon PTH treatment. TransCon PTH normalized 24-hour urine calcium through Week 52, regardless of initial randomization (placebo or TransCon PTH). TransCon PTH continued to be well-tolerated in the Phase 3 open-label extension, with no new safety signal identified. Most TEAEs were mild or moderate (Grades 1-2) and none reported during the open-label extension through Week 52 led to discontinuation of the study drug or trial. Slides showing the data presented for Week 52 of the Phase 3 PaTHway trial of TransCon PTH in adults with hypoparathyroidism can be viewed on the Ascendis Pharma Investors & News website at https://investors.ascendispharma.com. About Ascendis Pharma A/S Ascendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated, global biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Germany (Heidelberg, Berlin and Munich) and the United States (Palo Alto and Redwood City, California, and Princeton, New Jersey). Visit ascendispharma.com to learn more. The content above comes from the network. if any infringement, please contact us to modify.