Turner Syndrome

Launch of TransCon™ PTH underway with full commercial availability in Germany and Austria; U.S. PDUFA date of May 14, 2024 TransCon CNP pivotal ApproaCH Trial on track for topline results in Q4 2024 Total Q4 revenue of €138 million including SKYTROFA® revenue of €64 million; Q4 operating expenses of €155 million Conference call today at 4:30 pm ET COPENHAGEN, Denmark, Feb. 07, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma, Inc. (Nasdaq: ASND) today announced financial results for the fourth quarter and full year ended December 31, 2023 and provided a business update. “In 2023, we streamlined Ascendis, including our structure, processes, and operating expense allocation, and at the same time we believe we remain on track to achieve Vision 3x3,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “SKYTROFA is the leading growth hormone product in value in the U.S., the launch of YORVIPATH® in our Europe Direct and International Markets segments is underway beginning with Germany and Austria, and our clinical program for TransCon CNP is advancing. Ascendis is a leaner, more efficient organization in 2024, well-positioned to fulfill the strategic goals outlined in our Vision 2030, including achieving blockbuster status for each of our three independent Endocrinology Rare Disease products and expanding our engine for future innovation.” Select 2023 Highlights & Anticipated 2024 Milestones TransCon hGH: (lonapegsomatropin, approved as SKYTROFA in the U.S. and EU) Fourth quarter SKYTROFA revenue totaled €64 million, a 37% sequential increase. Full year 2023 SKYTROFA revenue totaled €179 million compared to €36 million the year prior. Q4-2022 Q1-2023 Q2-2023 Q3-2023 Q4-2023 SKYTROFA revenue (millions) €17 €32 €36 €47 €64 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 second 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) Launch of TransCon PTH underway with full commercial availability in Germany and Austria. 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. TransCon CNP: (navepegritide) First patient enrolled in Phase 2 reACHin Trial in infants with achondroplasia (age 0-2 years). Estimated total enrollment of 72 patients. 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. Topline data from Week 26 of the COACH Trial (TransCon hGH/TransCon CNP combination) expected in children with achondroplasia (age 2-11 years) in the fourth quarter of 2024. During the fourth quarter of 2024, plan to submit an Investigational New Drug application or similar in adults with achondroplasia. TransCon IL-2 β/γ: (onvapegleukin alfa) During the fourth quarter of 2024, plan to provide a clinical update from the Phase 2 portion of indication-specific, dose expansion cohorts in the IL Believe trial. TransCon TLR7/8 Agonist: During the fourth quarter of 2024, plan to provide a clinical update from the Phase 2 portion of indication-specific, dose expansion cohorts in the transcendIT-101 trial. Ophthalmology In January 2024, announced the formation and launch of Eyconis, Inc., a separate company created to develop, manufacture, and commercialize TransCon ophthalmology assets globally. Financial Update and Outlook Based on Current Plans Ended 2023 with cash, cash equivalents, and marketable securities totaling €399 million. 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. Fourth Quarter and Full Year 2023 Financial Results Total revenue for the fourth quarter of 2023 was €137.7 million compared to €22.9 million during the same period for 2022. The increase was primarily attributable to the $70.0 million upfront payment from Teijin, reported in license revenue and higher SKYTROFA revenue of €64.2 million compared to €17.1 million in the same period last year. Total revenue for 2023 was €266.7 million compared to €51.2 million in 2022. The increase was primarily attributable to higher SKYTROFA revenue of €178.7 million in 2023 compared to €35.7 million last year and the $70.0 million upfront payment from Teijin, reported in license revenue. Research and development (R&D) costs for the fourth quarter were €90.9 million compared to €108.6 million during the same period in 2022. The decrease was tied to lower external development costs for TransCon hGH and Oncology programs. R&D costs for 2023 were €413.5 million compared to €379.6 million in 2022. The higher R&D costs in 2023 reflect an increase in TransCon CNP, Oncology and Ophthalmology program development activities and an increase in employee related costs attributable to organizational growth, partially offset by lower TransCon hGH program development costs. Selling, general, and administrative (SG&A) expenses for the fourth quarter were €64.0 million compared to €56.6 million during the same period in 2022. This increase was primarily due to higher employee related expenses attributable to organizational growth. SG&A expenses for 2023 were €264.4 million compared to €221.2 million in 2022. Higher SG&A expenses were primarily due to organizational growth including commercial personnel to support existing SKYTROFA sales in the U.S. and Germany, and in preparation for future product launches. Total operating expenses for the fourth quarter were €154.9 million compared to €165.2 million during the same period in 2022. Total operating expenses for 2023 were €677.9 million compared to €600.9 million in 2022. Net finance expenses were €41.6 million in the fourth quarter compared to €46.7 million in the same period in 2022. Net finance expenses were €0.2 million in 2023 compared to a net finance income of €1.7 million in 2022. For the fourth quarter of 2023, Ascendis Pharma reported a net loss of €86.9 million, or €1.54 per share (basic and diluted) compared to a net loss of €207.4 million, or €3.71 per share (basic and diluted) for the same period in 2022. For the full year 2023, Ascendis Pharma reported a net loss of €481.4 million, or €8.55 per share (basic and diluted) compared to a net loss of €583.2 million, or €10.40 per share (basic and diluted) in 2022. As of December 31, 2023, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €399.4 million compared to €742.9 million as of December 31, 2022. As of December 31, 2023, Ascendis Pharma had 57,707,439 ordinary shares outstanding, including 1,093,054 ordinary shares represented by ADSs held by the company. Conference Call and Webcast Information Ascendis Pharma will also host a conference call and webcast today at 4:30 p.m. Eastern Time (ET) to discuss 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 . 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/S Ascendis 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 Germany 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 timing of topline results from the ApproaCH Trial, (iii) Ascendis’ ability to achieve Vision 3x3, (iv) Ascendis’ ability to fulfill its strategic goals outlined in Vision 2030, including achieving blockbuster status for each of its three independent Endocrinology Rare Disease products and expanding its engine for future innovation, (v) Ascendis’ expectations regarding full year 2024 revenue for SKYTROFA, (vi) Ascendis’ plan to submit a Biologics License Application for SKYTROFA for adult GHD in the second quarter of 2024, (vii) the timing of topline results from the Phase 2 trial in Turner syndrome, (viii) the launch of TransCon PTH in the U.S., if approved, (ix) the estimated total enrollment in the Phase 2 reACHin Trial, (x) the timing of topline data from the ApproaCH Trial, (xi) Ascendis’ plan to submit a New Drug Application for TransCon CNP for children with achondroplasia, (xii) the timing of topline data from Week 26 of the COACH Trial, (xiii) Ascendis’ plan to submit an Investigational New Drug application or similar for TransCon CNP in adults with achondroplasia, (xiv) Ascendis’ plan to provide a clinical update from the Phase 2 portion of indication-specific, dose expansion cohorts of the IL Believe trial, (xv) Ascendis’ plan to provide a clinical update from the Phase 2 portion of indication-specific, dose expansion cohorts in the transcendIT-101 trial during the fourth quarter of 2024, (xvi) Eyconis, Inc.’s ability to develop, manufacture, and commercialize TransCon ophthalmology assets globally, (xvii) Ascendis’ expectations regarding its total operating expenses for 2024, (xviii) Ascendis’ expectation that it will be operating cash flow breakeven on a quarterly basis by the end of 2024, (xix) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (xx) 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. © February 2024 Ascendis Pharma A/S. Investor Contacts: Tim Lee Ascendis Pharma +1 (650) 374-6343 tle@ascendispharma.com ir@ascendispharma.com Media Contact: Melinda Baker Ascendis Pharma +1 (650) 709-8875 media@ascendispharma.com Patti Bank ICR Westwicke +1 (415) 513-1284 patti.bank@westwicke.com 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 December 31, Twelve Months ended December 31, 2023 2022 2023 2022 Revenue 137,703 22,895 266,718 51,174 Cost of sales 19,457 5,111 44,395 12,137 Gross profit 118,246 17,784 222,323 39,037 Research and development costs 90,881 108,618 413,454 379,624 Selling, general and administrative expenses 63,975 56,553 264,410 221,227 Operating profit / (loss) (36,610 ) (147,387 ) (455,541 ) (561,814 ) Share of profit / (loss) of associate (2,924 ) (7,962 ) (18,395 ) (17,697 ) Finance income 22,727 3,305 43,857 52,181 Finance expenses 64,280 50,027 44,065 50,487 Profit / (loss) before tax (81,087 ) (202,071 ) (474,144 ) (577,817 ) Income taxes (expenses) (5,791 ) (5,348 ) (7,303 ) (5,377 ) Net profit / (loss) for the period (86,878 ) (207,419 ) (481,447 ) (583,194 ) Attributable to owners of the Company (86,878 ) (207,419 ) (481,447 ) (583,194 ) Basic and diluted earnings / (loss) per share € (1.54 ) € (3.71 ) € (8.55 ) € (10.40 ) Number of shares used for calculation (basic and diluted) 56,560,368 55,941,261 56,287,060 56,071,793 Net profit / (loss) for the period (86,878 ) (207,419 ) (481,447 ) (583,194 ) Other comprehensive income / (loss) Items that may be reclassified subsequently to profit or loss: Exchange differences on translating foreign operations (1,498 ) 2,211 (2,731 ) (327 ) Other comprehensive income / (loss) for the period, net of tax (1,498 ) 2,211 (2,731 ) (327 ) Total comprehensive income / (loss) for the period, net of tax (88,376 ) (205,208 ) (484,178 ) (583,521 ) Attributable to owners of the Company (88,376 ) (205,208 ) (484,178 ) (583,521 ) Ascendis Pharma A/S Consolidated Statements of Financial Position (In EUR'000s) December 31, December 31, 2023 2022 Assets Non-current assets Intangible assets 4,419 4,828 Property, plant and equipment 110,634 129,095 Investment in associate 5,686 22,932 Other receivables 2,127 1,920 Marketable securities - 7,492 122,866 166,267 Current assets Inventories 208,931 130,673 Trade receivables 35,874 11,910 Income tax receivables 802 883 Other receivables 19,097 12,833 Prepayments 38,578 31,717 Marketable securities 7,275 290,688 Cash and cash equivalents 392,164 444,767 702,721 923,471 Total assets 825,587 1,089,738 Equity and liabilities Equity Share capital 7,749 7,675 Distributable equity (153,446 ) 255,673 Total equity (145,697 ) 263,348 Non-current liabilities Borrowings 534,246 387,556 Lease liabilities 84,619 95,400 Derivative liabilities 143,296 157,950 Contract liabilities 5,949 14,213 Deferred tax liabilities 5,830 - 773,940 655,119 Current liabilities Borrowings 11,226 11,630 Lease liabilities 14,174 13,791 Contract liabilities 1,184 - Trade payables and accrued expenses 94,566 101,032 Other liabilities 41,176 31,989 Income tax payables 2,299 5,490 Provisions 32,719 7,339 197,344 171,271 Total liabilities 971,284 826,390 Total equity and liabilities 825,587 1,089,738

Researchers have developed a new technique to measure the number of chromosomes in ancient genomes more precisely, using it to identify the first prehistoric person with mosaic Turner syndrome (characterized by one X chromosome instead of two [XX]), who lived about 2500 years ago. Researchers at the Francis Crick Institute, working with University of Oxford, University of York and Oxford Archaeology, have developed a new technique to measure the number of chromosomes in ancient genomes more precisely, using it to identify the first prehistoric person with mosaic Turner syndrome (characterised by one X chromosome instead of two [XX]), who lived about 2500 years ago. As part of their research published today in Communications Biology, they also identified the earliest known person with Jacob's syndrome (characterised by an extra Y chromosome -- XYY) in the Early Medieval Period, three people with Klinefelter syndrome (characterised by an extra X chromosome -- XXY) across a range of time periods and an infant with Down Syndrome from the Iron Age. Most cells in the human body have 23 pairs of DNA molecules called chromosomes, and the sex chromosomes are typically XX (female) or XY (male), although there are differences in sexual development. 'Aneuploidy' occurs when a person's cells have an extra or missing chromosome. If this occurs in the sex chromosomes, a few differences like delayed development or changes in height can be seen around puberty. Ancient DNA samples can erode over time and can be contaminated by DNA from other ancient samples or from people handling them. This makes it difficult to accurately capture differences in the number of sex chromosomes. The team at the Crick developed a computational method which aims to pick up more variation in sex chromosomes. For the sex chromosomes, it involves counting the number of copies of X and Y chromosomes, and comparing the outcome to a predicted baseline (what you would expect to see). The team used the new method to analyse ancient DNA from a large dataset of individuals collected as part of their Thousand Ancient British Genomes project across British history, identifying six individuals with aneuploidies across five sites in Somerset, Yorkshire, Oxford and Lincoln2. The individuals lived across a range of time periods, from the Iron Age (2500 years ago) up to the Post-Medieval Period (about 250 years ago). They identified five people who had sex chromosomes which fell outside of the XX or XY categories. All were buried according to their society's customs although no possessions were found with them to shed more light on their lives. The three individuals with Klinefelter syndrome lived across very different time periods, but they shared some similarities -- all were slightly taller than average and showed signs of delayed development in puberty. By investigating details on the bones, the research team could see that it was unlikely that the individual with Turner syndrome had gone through puberty and started menstruation, despite their estimated age of 18-22. Their syndrome was shown to be mosaic -some cells had one copy of chromosome X and some had two. Kakia Anastasiadou, PhD student in the Ancient Genomics Laboratory at the Crick, and first author of the study, said: "Through precisely measuring sex chromosomes, we were able to show the first prehistoric evidence of Turner syndrome 2500 years ago, and the earliest known incidence of Jacob's syndrome around 1200 years ago. It's hard to see a full picture of how these individuals lived and interacted with their society, as they weren't found with possessions or in unusual graves, but it can allow some insight into how perceptions of gender identity have evolved over time." Pontus Skoglund, Group Leader of the Ancient Genomics Laboratory at the Crick, said: "Our method is also able to classify DNA contamination in many cases, and can help to analyse incomplete ancient DNA, so it could be applied to archaeological remains which have been difficult to analyse. "Combining this data with burial context and possessions can allow for a historical perspective of how sex, gender and diversity were perceived in past societies. I hope this type of approach will be applied as the common resource of ancient DNA data continues to grow." The team worked with archaeologists from the University of Oxford, the Wells and Mendip Museum, University of York, University of Bradford, Oxford Archaeology, York Osteoarchaeology and Network Archaeology, acknowledging support from Lincolnshire County Council, Magdalen College and Balfour Beatty for National Highways. Rick Schulting, Professor of Scientific and Prehistoric Archaeology at the University of Oxford, said: "The results of this study open up exciting new possibilities for the study of sex in the past, moving beyond binary categories in a way that would be impossible without the advances being made in ancient DNA analysis."

Strategic roadmap to achieve blockbuster status for multiple products and expand the Company’s engine for future innovation COPENHAGEN, Denmark, Jan. 07, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today introduced selected 2024 corporate milestones and Vision 2030, its strategic roadmap through 2030. Ascendis President and CEO Jan Mikkelsen will present the update tomorrow, January 8, at the 42nd Annual J.P. Morgan Healthcare Conference. “With unwavering focus on our values of Patients, Science, and Passion, we remain on track to fulfill Vision 3x3, with the approval already of two Endocrinology Rare Disease products and value leadership in the U.S. growth hormone market for SKYTROFA,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Today we are introducing Vision 2030, our strategic roadmap to attain blockbuster status for multiple products and expand our engine for future innovation.” Selected Key Updates TransCon™ hGH (lonapegsomatropin, approved as SKYTROFA® in the U.S. and EU) Following positive topline results from Phase 3 foresiGHt Trial in adult growth hormone deficiency (GHD), plan to submit a supplemental Biologics License Application to FDA in the second quarter of 2024.Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024.Attained U.S. market value leadership in 2023.SKYTROFA preliminary unaudited fourth quarter 2023 revenue is expected to be approximately €64 million. Full year 2024 SKYTROFA revenue expected to be €320 million to €340 million (based on average 2023 exchange rates). TransCon PTH (palopegteriparatide, approved as YORVIPATH® in the EU) First launch planned in Germany in January 2024, leveraging the Company’s existing commercial infrastructure.FDA regulatory review for the treatment of adult patients with hypoparathyroidism continues, with a Prescription Drug User Fee Act (PDUFA) date of May 14, 2024; if approved, U.S. commercial launch planned in the third quarter of 2024. TransCon CNP (navepegritide) Treatment with TransCon CNP at 100 mg/kg/week for one year in all 57 children with achondroplasia (age 2 to 10 years) in ACcomplisH, demonstrated growth consistent with results from the blinded period and achieved improvements in health-related quality of life and disease impacts assessed with SF-10 and Achondroplasia Child Experience Measure.During the third quarter of 2023, submitted Clinical Trial Application (CTA) for infant trial (age 0-2 years).Topline results from the pivotal ApproaCH Trial expected in the fourth quarter of 2024.During the fourth quarter of 2024, plan to submit a New Drug Application to FDA for children with achondroplasia (age 2-11 years).Week 26 topline data from the COACH Trial (TransCon hGH/TransCon CNP combination) expected in the fourth quarter of 2024.During the fourth quarter of 2024, plan to submit an Investigational New Drug application or similar in adults with achondroplasia. Global commercial presence in Endocrinology Rare Disease During the fourth quarter of 2023, entered into an exclusive license agreement with Teijin Limited for TransCon hGH, TransCon PTH, and TransCon CNP in Japan. Phase 3 PaTHway Japan Trial of TransCon PTH completed, and Phase 3 riGHt Trial of TransCon hGH fully enrolled.VISEN’s TransCon hGH Phase 3 and TransCon CNP Phase 2 trials in China completed.Expanding global reach through exclusive sales & distribution agreements with geographic market leaders in International Markets, with three regional agreements signed to date: Specialised Therapeutics Asia Pte Ltd, Er-Kim, and Vector Pharma FZCO. Oncology During the fourth quarter of 2024, expect to complete enrollment in BelieveIT-201, a Phase 2 trial in advanced head and neck squamous cell carcinoma (HNSCC).During the fourth quarter of 2024, plan to provide a clinical update from the Phase 2 portion of indication-specific, dose expansion cohorts in the IL-Believe Trial. Ophthalmology Creation of Ophthalmology NewCo, financed by institutional investors, expected during the first quarter of 2024. Financial Update and Outlook Based on Current Plans Preliminary unaudited December 31, 2023 cash, cash equivalents, and marketable securities of ~€400 million.Full year 2024 SKYTROFA revenue expected to be €320 million to €340 million (based on average 2023 exchange rates).Expect to provide YORVIPATH revenue update during 2024.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. Vision 2030: Ascendis Pharma’s 2025–2030 Strategic Roadmap Achieve blockbuster status for multiple products and expand our engine for future innovation. Be the Leading Endocrinology Rare Disease Company Achieve blockbuster status (>$1B) for TransCon PTH, TransCon hGH, and TransCon CNP through worldwide commercializationBe the leader in growth disorders and hypoparathyroidism, pursuing clinical conditions, innovative life cycle management, and complementary patient offeringsExpand pipeline with Endocrinology Rare Disease blockbuster product opportunities Create Value in Additional Therapeutic Areas through Innovative Business Models Obtain accelerated approval in oncology with registrational trials ongoingPursue TransCon product opportunities in >$5B indicationsMaximize value creation of these product opportunities through collaboration with therapeutic area market leaders Differentiate with Ascendis Fundamentals Outperform industry drug development benchmarks with Ascendis’ product innovation algorithmRemain independent as a profitable biopharma through lean and flexible ways of workingLet our values Patients, Science, Passion drive our decisions to success Presentation at J.P. Morgan Healthcare Conference on Monday, January 8th A live webcast of the event will be available via the Investors & News section of the Ascendis Pharma website at https://investors.ascendispharma.com. The presentation will begin at 12:00 p.m. Eastern Time / 9:00 am Pacific Time. A webcast replay will be available for 30 days. The Company’s slides from the J.P. Morgan presentation will be available on the same Investor Relations 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 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 Germany 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) Ascendis’ ability to achieve blockbuster status for multiple products and expand its engine for future innovation, (ii) Ascendis’ ability to achieve its 2024 corporate milestones and Vision 2030, (iii) Ascendis’ ability to fulfill Vision 3x3, (iv) Ascendis’ plan to submit a supplemental Biologics License Application for TransCon hGH in the second quarter of 2024, (v) the timing of topline results from the Phase 2 trial in Turner syndrome, (vi) Ascendis’ expectations with respect to SKYTROFA revenue for 2023 and 2024, (vii) Ascendis’ plan to launch TransCon PTH in Germany in January 2024, (viii) TransCon PTH’s PDUFA date of May 14, 2024, (ix) Ascendis’ plans to launch TransCon PTH in the U.S. in the third quarter of 2024, if approved, (x) the timing of topline results from the ApproaCH Trial, (xi) Ascendis’ plan to submit a New Drug Application for TransCon CNP for children with achondroplasia, (xii) Ascendis’ plan to submit an Investigational New Drug application or similar for TransCon CNP in adults with achondroplasia; (xiii) the timing of topline annualized height velocity data from the COACH Trial, (xiv) Ascendis’ ability to expand its global reach through exclusive sales & distribution agreements with geographic market leaders, (xv) Ascendis’ expectations with regard to the completion of enrollment in BelieveIT-201, (xvi) Ascendis’ plan to provide a clinical update from the Phase 2 portion of indication-specific, dose expansion cohorts in the IL-Believe Trial, (xvii) Ascendis’ expectations regarding its financial results and performance in 2023 and 2024, (xviii) Ascendis’ Vision 2030, (xix) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (xx) 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, SKYTROFA®, and YORVIPATH® are trademarks owned by the Ascendis Pharma group. © January 2024 Ascendis Pharma A/S. Investor Contacts: Media Contact:Tim Lee Melinda BakerAscendis Pharma Ascendis Pharma+1 (650) 374-6343 +1 (650) 709-8875ir@ascendispharma.com media@ascendispharma.com Patti Bank ICR Westwicke +1 (415) 513-1284 patti.bank@westwicke.com