Gonadal Dysgenesis

Ad hoc announcement pursuant to Art. 53 LR MILAN--(BUSINESS WIRE)-- Newron Pharmaceuticals S.p.A. (“Newron”) (SIX: NWRN, XETRA: NP5), a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and peripheral nervous system, today announced its financial results and operational highlights for the business year ended December 31, 2023, and provided an outlook for 2024. Highlights 2023: Evenamide (Schizophrenia) Compelling efficacy results across multiple investigation timepoints up to and including one-year from study 014/015 evaluating evenamide as an add-on therapy to a single antipsychotic in patients with treatment-resistant schizophrenia (TRS) Presentation of data to the medical community at several international scientific conferences, highlighting the clinically meaningful response to evenamide in TRS patients, across various timepoints Publication of data from study 014 and results from first 100 patients of study 015 in peer-reviewed journal, the International Journal of Neuropsychopharmacology, outlining the continued improvement in symptoms of psychosis in TRS patients treated with evenamide The findings from study 014/015 support the initiation of a potentially pivotal, Phase III, randomized, double-blind, placebo-controlled study with evenamide as an add-on treatment in patients with TRS Study 008A, a potentially pivotal study of evenamide (30 mg bid) in patients with chronic schizophrenia demonstrating inadequate benefit to their current second-generation antipsychotic, completed patient enrollment; top-line results are expected in April 2024 Xadago®/safinamide (Parkinson’s disease) Newron and its partners reached agreement with generic pharmaceutical manufacturers who submitted Paragraph IV Notice Letters regarding Abbreviated New Drug Applications in the US In Europe, Supplementary Protection Certificates (SPCs) have been approved in most territories of relevance Corporate Newron’s senior management team strengthened by two key promotions: Laura Faravelli, previously Director Business Development at Newron, was promoted to Vice President Business Development; Roberto Galli, Vice President Finance since 2012, was promoted to Chief Financial Officer Gillian Dines appointed as Non-Executive Director to the Board of Newron at the AGM 2023 Margarita Chavez appointed as advisor to the Board of Newron; she is nominated for election as Non-Executive Director to the Board at the next Ordinary AGM on April 17, 2024 Post-period, Newron entered into an agreement for the subscription of up to 2.05 million newly issued shares with an institutional investor focused on investing in high-growth firms across sectors including biotech and healthcare, to finance the Company’s operations beyond the current key inflection points in its pipeline Post-period, agreement with European Investment Bank (EIB) to extend the near-term tranche repayment dates of Newron’s 2018 financing agreement Stefan Weber, CEO of Newron, commented: “Against a backdrop of challenging global biotechnology market conditions, we have continued to make huge strides in advancing our evenamide development program for treatment-resistant schizophrenia (TRS) through the clinic. We have reported multiple data sets across three different study timepoints, each showing compelling evidence of the benefit of evenamide in schizophrenia patients no longer responding to their first- or second-generation antipsychotic treatments. Post-period, we also optimized our financing structure by agreeing with the EIB to extend the near-term tranche repayment dates of our financing agreement. And we welcomed an institutional investor that signed a subscription agreement for new shares to finance our operations beyond the current key inflection points in the pipeline. We thank our shareholders for their ongoing support and for sharing our vision to improve the lives of patients suffering from diseases of the CNS, by now for 25 years, since Newron was founded.” Evenamide Newron made substantial progress across its evenamide development program over the reporting year and into early 2024. The published compelling data is demonstrating the benefit of the Company’s new chemical entity for patients with TRS: In Q1 2023, Newron disclosed interim analyses of study 014, a Phase II, international, randomized, open label, rater-blinded trial evaluating evenamide (7.5, 15 or 30 mg bid) as an add-on to a single antipsychotic (excluding clozapine) in patients with moderate to severe TRS, not responding to their current antipsychotic medication, and study 015, its extension to one year of treatment. Results from the first 100 patients to complete six months (30 weeks) of treatment with evenamide were reported in January, followed by results in February 2023 from the same 100-person cohort at the one-year (52 weeks) timepoint. The results at the six-week, six-month and one-year timepoints demonstrated a clinically important and sustained improvement in symptoms, and importantly, a substantially greater benefit at one-year than at the six-week and six-month datapoints. Topline data from all 161 patients at the six-week primary endpoint were reported in March. Although the primary objective of the study was safety and tolerability of evenamide, efficacy over baseline was also assessed. The mean Positive and Negative Syndrome Scale (PANSS) total score, Clinical Global Impression of Severity (CGI-S) rating, and the Strauss-Carpenter Level of Functioning (LOF) total score significantly improved compared to baseline (p<0.001). The results from the complete study population of 161 patients were fully consistent with the findings from the first 100 patients at this timepoint. Notably, evenamide was safe and well-tolerated at all doses, with almost all (95%) patients completing six weeks of treatment. The incidence of treatment-emergent adverse events was very low, and more than 90% of the completers chose to continue with evenamide treatment into the long-term extension study (study 015). In the beginning of Q4, Newron provided data from the full study population in the extension study (study 015) at the 36th ECNP Congress in Barcelona, Spain, demonstrating the positive effect of evenamide at six months of treatment. Results showed that benefits continued to accrue over time, and many patients who did not respond early achieved clinically important benefits later on in the study. Remarkably, following treatment with evenamide, approximately 40% of patients at six months no longer met the protocol severity criteria used to diagnose TRS. These data were further strengthened, post-period in January 2024, with the final results from study 015 reviewing all study participants after one year of treatment. The data showed that treatment with evenamide was associated with sustained, clinically significant benefit that increased throughout the one-year course of treatment. Gradual and sustained improvement was demonstrated across all efficacy scales used. More than 70% of patients experienced clinically important reduction in disease severity. Review of the efficacy data indicated that treatment with evenamide resulted in approximately 50% of patients at one-year no longer meeting any of the protocol severity criteria used to diagnose treatment resistance. Significantly 25% of all patients achieved “remission” (no/minimal symptoms for at least six months), never described before in TRS patients. Moreover, in contrast to common clinical experience, no patient relapsed during the one-year treatment period. The durability and longevity of these clinical benefits is unprecedented in this indication and suggest that the glutamate modulating effect of evenamide could lead to a progressive and long-standing alteration in brain processes, synergizing with the effect of the antipsychotic to which the patient had become resistant. The next R&D activities will be: In their totality, the results from study 014/015 support the initiation of a potentially pivotal Phase III, randomized, double-blind, placebo-controlled study of evenamide as an add-on treatment in patients with TRS, which will hopefully confirm the benefit of evenamide seen so far. If approved, the compound would be the first add-on drug that improves the symptoms of TRS, offering a much-needed new therapeutic option for those who are not responding to existing antipsychotics. Newron is also investigating evenamide in a potentially pivotal study (study 008A) in a separate indication, in patients with chronic schizophrenia currently being treated with a second-generation antipsychotic, but who demonstrate an inadequate response to that treatment. Study 008A is a four-week, randomized, double-blind and placebo-controlled study assessing the efficacy, tolerability, and safety of evenamide (30 mg bid). Patient enrollment has completed and results from this study are expected in April 2024. Xadago®/safinamide In partnership with Zambon and Supernus, Newron continued to further develop and market its product, Xadago®/safinamide. In reference to the receipt of several Paragraph IV Notice Letters in May 2021 regarding the submission by some generic manufacturers of an Abbreviated New Drug Application ANDA to the US Food and Drug Administration (FDA), seeking approval to engage in the commercial manufacture, use or sale of safinamide mesylate drug product in the US before expiration of the three US patents listed in the FDA Orange Book for Xadago®, Newron and its partners Zambon and Supernus have reached settlement agreement with said generic manufacturers, thus resolving the legal action. All three patents remain valid and in force. Under the agreement, the generic manufacturers will be allowed to enter the US market with a safinamide mesylate drug product no earlier than December 1, 2027. In the EU, Supplementary Protection Certificates (SPCs) have already been approved in most territories of relevance; Newron and Zambon are confident that upon completion of the still ongoing procedures and targeted activities, the SPCs will be granted in all key territories. ESG reporting The Annual Report 2023 provides further transparency on Newron’s corporate ESG commitments. The relevant section covers information on the following topics: employee aspects, environmental aspects including climate, social aspects, human rights, and anti-corruption as well as cyber security – given their relevance to Newron and its stakeholders and given the Company’s ability as a company to have a positive impact on the listed areas. Newron’s 2023 ESG Report follows the most recent edition of the Directive on Information relating to Corporate Governance (Annex 7) of SIX Exchange Regulation (SER). Financial key takeaways 2023: In 2023, Newron reported a net loss of EUR 16.2 million, slightly lower compared to EUR 17.5 million in 2022 Cash used in operating activities has decreased to EUR 10.1 million from EUR 11.1 million in 2022 Xadago® revenues from Zambon increased from EUR 6.0 million in 2022 to EUR 9.0 million in the reporting year Newron’s R&D expenses have risen to EUR 13.2 million from EUR 12.0 million in 2022 G&A expenses slightly increased from EUR 7.4 million in 2022 to EUR 7.5 million Cash and Other current financial assets as of December 31, 2023 were at EUR 12.6 million, compared to EUR 22.8 million at the beginning of the year Post-period, in March 2024, Newron announced it has entered into an agreement for the subscription of up to 2.05 million shares with an institutional investor focused on investing in high-growth firms across sectors including biotech and healthcare, raising gross proceeds of up to EUR 15.0 million (at current exchange rate CHF-EUR). Under the agreement, the fund subscribes to an initial 750,000 newly issued shares at a subscription price of EUR 7.33 per share, which corresponds to gross proceeds of approximately EUR 5.5 million. In addition, the fund has a right to subscribe to an additional up to 1,300,000 newly issued shares until no later than January 31, 2025, at a subscription price to be calculated pursuant to an agreed formula. The share subscriptions are governed by the capital increase authorised by Newron’s shareholders in 2018 and approved and empowered by the Company’s Board of Directors in 2023. Newron intends to use the net proceeds of the fundraise for general corporate purposes, including the financing of its operations and research programs and for the development of current and future pipeline products. Newron also continues to seek opportunistic additions to its pipeline portfolio. Post-period, and also in March 2024, Newron announced that the Company has agreed with the European Investment Bank (EIB) to extend the near-term tranche repayment dates of its loan agreement. Under the amendment to certain terms of the financing agreement with the EIB, the repayment of tranches one to three (out of a total of five) has been shifted substantially, with tranche one now scheduled for repayment by 25 November 2025, tranche two by April 2026 and tranche three by June 2026. The EIB qualifies now for certain performance-based remuneration. Newron appreciates the support of the EIB in helping the Company to align its contractual obligations with the potential timing of certain upcoming inflection points from its pipeline. Financial Summary (IFRS) 2023 and 2022: In thousand EUR (except per share information) 2023 2022 Licence income from contracts with customers 58 14 Royalties from contracts with customers 6,735 5,936 Other income from contracts with customers 2,264 144 Revenue 9,057 6,094 Research and development expenses, net (13,152) (12,005) Operating Result (11,629) (13,302) Financial result, net (4,571) (4,170) Net loss (16,224) (17,493) Loss per share (0.91) (0.98) Cash used in operating activities (10,140) (11,092) Cash, cash equivalents and Other current financial assets 12,599 22,774 Total assets 25,866 37,195 Newron’s Annual Report 2023 is available for download on the Company’s website at: Outlook 2024: Following the remarkable evenamide data to date, Newron plans to initiate a potentially pivotal trial evaluating evenamide in TRS, once preparations are completed and a partner has been identified. If this trial replicates the data published in study 014/015, the Company believes there will be substantial improvements in the treatment paradigm and outlook for TRS patients. Newron is also primed to report data from study 008A, a potentially pivotal study assessing evenamide in schizophrenia patients not deemed to be treatment resistant. If positive, this would be the first well-controlled study to demonstrate the clinical utility of evenamide in schizophrenia patients who show an inadequate response to treatment with atypical antipsychotics. Newron is continuing to have a productive dialogue with industry players around potential future collaboration opportunities for evenamide. The Company also continues to review the CNS landscape for opportunities to expand its drug development pipeline. Newron’s total available cash resources together with the initial proceeds deriving from the subscription agreement signed post-period, will fund the Company’s planned development programs and operations well into 2025 and well beyond the current key value inflection points in our pipeline. Media/analyst/investor Conference Call today at 3 pm CET Newron’s management team will present today the 2023 full-year results and provide an update and guidance for 2024. Please dial in five to ten minutes prior to the beginning of the call using one of the following telephone numbers: Switzerland/Europe: +41 (0)58 310 50 00 United Kingdom: +44 (0)207 107 0613 United States: +1 (1)631 570 5613 The presentation is available at 2024 Shareholders’ Meeting Agenda: Newron’s Board of Directors has approved the below agenda for the April 17, 2024, Shareholders’ Ordinary and Extraordinary meeting, which will take place at the Company’s registered office (Via Antonio Meucci 3) in Bresso (Mi), Italy, starting at 10 am CET. The formal invitation to shareholders will be issued and disclosed in the statutory papers on or around March 26, 2023. The full invitation and supporting material will be made available on the Company’s website ( ) on the same day. The agenda is as follows: Ordinary part Approval of the financial statements as at 31 December 2023. Update – as per art. 2447 Civil Code - regarding the freezing of 2022 Newron’ standalone losses. Connected and consequent resolutions; Appointment of a new members of the Board of Directors, for the financial years 2024 and 2025 and, therefore, until the approval of the financial statements as of December 31st, 2025, as follows: determination of the relevant number, proposal to appoint Margarita Chavez*, in quality of new non-executive director Determination of the remuneration Connected and consequent resolutions. Extraordinary part Proposal to amend Art 14 of the by-laws. Related and consequential resolutions; Proposal of attribution to the Board of Directors of powers, pursuant to art. 2443 of the Civil Code, exercisable during the next 5 years, to increase the share capital, in one or more times, for a maximum amount of Euro 357.636,00, in addition to any premium, with or without option rights, pursuant to art. 2441, paragraphs 1 and/or 4, first and second part, and/or 5, 6 of the Civil Code. Related and consequential resolutions; Proposal of attribution to the Board of Directors of powers, pursuant to art. 2443 of the Civil Code, exercisable during the next 5 years, to increase the share capital, in one or more times, for a maximum amount of Euro 107.291,00, in addition to any premium, with exclusion of the option rights, pursuant to art. 2441, paragraphs 5, 6 and/or 8 of the Civil Code, for one or more stock option plans. Related and consequential resolutions; * Margarita Chavez brings to Newron over 20 years of dealmaking expertise and leadership in the pharmaceutical industry. Most recently, she was Managing Director of AbbVie Ventures, where she led investments and built biotech companies across the US and Europe. Ms. Chavez also served as a board member for several biotech companies across the US and Europe. As a Director in Abbott’s Global Pharmaceutical Licensing & Acquisitions Division, she was involved in the successful in-licensing of Elagolix and the acquisitions of Solvay, ImmuVen and the Lupron franchise. Before joining Abbott, Ms. Chavez practiced as a corporate and securities lawyer in the Silicon Valley, advising tech and biotech companies on strategic transactions including IPOs and mergers and acquisitions. She received her bachelor’s degree from Santa Clara University and her juris doctor from Santa Clara University School of Law (both California). Ms. Chavez is currently a Venture Partner at Wellington Partners and has been acting as advisor to Newron’s board since October 1, 2023. Financial calendar AGM and EGM 2024: April 17, 2024 Half-year report 2024: September 19, 2024 About Newron Pharmaceuticals Newron (SIX: NWRN, XETRA: NP5) is a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and peripheral nervous system. The Company is headquartered in Bresso near Milan, Italy. Xadago®/safinamide has received marketing authorization for the treatment of Parkinson’s disease in the European Union, Switzerland, the UK, the USA, Australia, Canada, Latin America, Israel, the United Arab Emirates, Japan and South Korea, and is commercialized by Newron’s Partner Zambon. Supernus Pharmaceuticals holds the commercialization rights in the USA. Meiji Seika has the rights to develop and commercialize the compound in Japan and other key Asian territories. Newron is also developing evenamide as the potential first add-on therapy for the treatment of patients with symptoms of schizophrenia. For more information, please visit: Important Notices This document contains forward-looking statements, including (without limitation) about (1) Newron’s ability to develop and expand its business, successfully complete development of its current product candidates, the timing of commencement of various clinical trials and receipt of data and current and future collaborations for the development and commercialization of its product candidates, (2) the market for drugs to treat CNS diseases and pain conditions, (3) Newron’s financial resources, and (4) assumptions underlying any such statements. In some cases, these statements and assumptions can be identified by the fact that they use words such as “will”, “anticipate”, “estimate”, “expect”, “project”, “intend”, “plan”, “believe”, “target”, and other words and terms of similar meaning. All statements, other than historical facts, contained herein regarding Newron's strategy, goals, plans, future financial position, projected revenues and costs and prospects are forward-looking statements. By their very nature, such statements and assumptions involve inherent risks and uncertainties, both general and specific, and risks exist that predictions, forecasts, projections and other outcomes described, assumed or implied therein will not be achieved. Future events and actual results could differ materially from those set out in, contemplated by or underlying the forward-looking statements due to a number of important factors. These factors include (without limitation) (1) uncertainties in the discovery, development or marketing of products, including without limitation difficulties in enrolling clinical trials, negative results of clinical trials or research projects or unexpected side effects, (2) delay or inability in obtaining regulatory approvals or bringing products to market, (3) future market acceptance of products, (4) loss of or inability to obtain adequate protection for intellectual property rights, (5) inability to raise additional funds, (6) success of existing and entry into future collaborations and licensing agreements, (7) litigation, (8) loss of key executive or other employees, (9) adverse publicity and news coverage, and (10) competition, regulatory, legislative and judicial developments or changes in market and/or overall economic conditions. Newron may not actually achieve the plans, intentions or expectations disclosed in forward-looking statements and assumptions underlying any such statements may prove wrong. Investors should therefore not place undue reliance on them. There can be no assurance that actual results of Newron's research programs, development activities, commercialization plans, collaborations and operations will not differ materially from the expectations set out in such forward-looking statements or underlying assumptions. Newron does not undertake any obligation to publicly update or revise forward-looking statements except as may be required by applicable regulations of the SIX Swiss Exchange or the Dusseldorf Stock Exchange where the shares of Newron are listed. This document does not contain or constitute an offer or invitation to purchase or subscribe for any securities of Newron and no part of it shall form the basis of or be relied upon in connection with any contract or commitment whatsoever. View source version on businesswire.com: Contacts For more information: Newron Stefan Weber – CEO, +39 02 6103 46 26, pr@newron.com UK/Europe Simon Conway / Ciara Martin / Natalie Garland-Collins, FTI Consulting, +44 20 3727 1000, SCnewron@fticonsulting.com Switzerland Valentin Handschin, IRF , +41 43 244 81 54, handschin@irf-reputation.ch Germany/Europe Anne Hennecke / Caroline Bergmann, MC Services, +49 211 52925222, newron@mc-services.eu USA Paul Sagan, LaVoieHealthScience, +1 617 374 8800, Ext. 112, psagan@lavoiehealthscience.com Source: Newron Pharmaceuticals S.p.A. View this news release online at:

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."