Shares of Ascendis Pharma (ASND) lost more than 5% on Tuesday after management announced that the FDA extended the review period for its regulatory filing for a hormone replacement therapy by an additional three months. A final decision is now expected by Aug 14, 2024.The company’s filing seeks approval for TransCon PTH (palopegteriparatide) to treat adults with hypoparathyroidism, a rare condition in which the body produces abnormally low levels of parathyroid hormone (PTH). Low production of this hormone ...

- Prescription Drug User Fee Act (PDUFA) goal date extended by three months for further review of submission to August 14, 2024 COPENHAGEN, Denmark, May 14, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (NASDAQ:ASND) today announced that the U.S. Food and Drug Administration (FDA) notified the Company that information submitted in response to the FDA's ongoing review of the New Drug Application (NDA) for TransCon PTH (palopegteriparatide) for adults with hypoparathyroidism constituted a major amendment to th ...

COPENHAGEN, Denmark, May 13, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (NASDAQ:ASND) today announced 2-year results from a post-hoc analysis of the Company's Phase 3 PaTHway Trial demonstrating significant and sustained improvements in renal function in adults with chronic hypoparathyroidism treated with TransCon PTH (palopegteriparatide). The data were shared in an oral presentation on May 12 by Peter Schwarz, M.D., Professor of Clinical Medicine at the University of Copenhagen, during the European Cong ...

COPENHAGEN, Denmark, May 09, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (NASDAQ:ASND) today announced that new TransCon PTH (palopegteriparatide) data supporting clinical and quality-of-life benefits in adult patients with chronic hypoparathyroidism will be presented at the European Congress of Endocrinology being held May 11-14, 2024, in Stockholm. Topics include an oral presentation of new 2-year results from a post-hoc analysis of the Phase 3 PaTHway Trial demonstrating sustained improvements in kidney ...

Ascendis Pharma A/S (ASND) Q1 2024 Earnings Conference Call May 2, 2024 4:30 PM ET Company Participants Tim Lee - Senior Director, Investor Relations Jan Mikkelsen - President & Chief Executive Officer Scott Smith - Executive Vice President & Chief Financial Officer Stina Singel - Executive Vice President, Clinical Development, Oncology Joe Kelly - US General Manager Conference Call Participants Jessica Fye - JPMorgan David Lebowitz - Citi Li Watsek - Cantor Joseph Schwartz - Leerink Partners Gavin Clark-Ga ...

[FORM 6-K Filing Information](index=1&type=section&id=FORM%206-K%20Filing%20Information) [Incorporation by Reference](index=2&type=section&id=Incorporation%20by%20Reference) This Form 6-K report is incorporated by reference into Ascendis Pharma A/S's existing registration statements on Form S-8 and Form F-3 - The Form 6-K is incorporated by reference into multiple **Form S-8** and **Form F-3** registration statements of Ascendis Pharma A/S[2](index=2&type=chunk) [Information Contained in this Form 6-K Report](index=2&type=section&id=Information%20Contained%20in%20this%20Form%206-K%20Report) This report contains the Company's Unaudited Condensed Consolidated Interim Financial Statements and Management's Discussion and Analysis for the period ended March 31, 2024 - The report contains **Unaudited Condensed Consolidated Interim Financial Statements** and **Management's Discussion and Analysis** for the period ended March 31, 2024[3](index=3&type=chunk) [Signatures](index=3&type=section&id=SIGNATURES) The report was signed by Michael Wolff Jensen, Executive Vice President, Chief Legal Officer, on May 2, 2024 - The report was signed by **Michael Wolff Jensen**, Executive Vice President, Chief Legal Officer, on May 2, 2024[4](index=4&type=chunk) [Unaudited Condensed Consolidated Interim Financial Statements – March 31, 2024](index=5&type=section&id=Unaudited%20Condensed%20Consolidated%20Interim%20Financial%20Statements%20%E2%80%93%20March%2031%2C%202024) [Unaudited Condensed Consolidated Interim Statements of Profit or (Loss) and Other Comprehensive Income or (Loss)](index=6&type=section&id=Unaudited%20Condensed%20Consolidated%20Interim%20Statements%20of%20Profit%20or%20%28Loss%29%20and%20Other%20Comprehensive%20Income%20or%20%28Loss%29) Ascendis Pharma A/S reported a net loss of **€131.0 million** for Q1 2024, up from **€110.9 million** in Q1 2023, despite revenue increasing to **€95.9 million** Unaudited Condensed Consolidated Interim Statements of Profit or (Loss) and Other Comprehensive Income or (Loss) | Metric | Three Months Ended March 31, 2024 (EUR'000) | Three Months Ended March 31, 2023 (EUR'000) | | :-------------------------- | :------------------------------------------ | :------------------------------------------ | | Revenue | 95,894 | 33,589 | | Cost of sales | 7,569 | 4,621 | | Gross profit | 88,325 | 28,968 | | Research and development costs | 70,687 | 106,114 | | Selling, general and administrative expenses | 66,783 | 66,539 | | Operating profit/(loss) | (49,145) | (143,685) | | Finance income | 3,575 | 45,135 | | Finance expenses | 77,161 | 9,840 | | Net profit/(loss) for the period | (131,035) | (110,914) | | Basic and diluted earnings/(loss) per share | (€2.30) | (€1.98) | - Revenue increased significantly from **€33.6 million** in Q1 2023 to **€95.9 million** in Q1 2024, a **185.5% increase**[10](index=10&type=chunk) - Net loss widened from **€110.9 million** in Q1 2023 to **€131.0 million** in Q1 2024, primarily due to a substantial increase in finance expenses and a decrease in finance income[10](index=10&type=chunk) [Unaudited Condensed Consolidated Interim Statements of Financial Position](index=7&type=section&id=Unaudited%20Condensed%20Consolidated%20Interim%20Statements%20of%20Financial%20Position) Total assets decreased to **€802.5 million** as of March 31, 2024, with total equity further declining to a negative **€238.2 million** Unaudited Condensed Consolidated Interim Statements of Financial Position | Metric | March 31, 2024 (EUR'000) | December 31, 2023 (EUR'000) | | :-------------------------- | :----------------------- | :-------------------------- | | Total assets | 802,504 | 825,587 | | Total equity | (238,179) | (145,697) | | Total liabilities | 1,040,683 | 971,284 | | Cash and cash equivalents | 320,239 | 392,164 | - Total assets decreased by **€23.1 million** from December 31, 2023, to March 31, 2024[13](index=13&type=chunk) - Total equity remained negative and further decreased by **€92.5 million**, from **(€145.7 million)** to **(€238.2 million)**[13](index=13&type=chunk) - Cash and cash equivalents decreased by **€71.9 million** from **€392.2 million** to **€320.2 million**[13](index=13&type=chunk) [Unaudited Condensed Consolidated Interim Statements of Changes in Equity](index=8&type=section&id=Unaudited%20Condensed%20Consolidated%20Interim%20Statements%20of%20Changes%20in%20Equity) Total equity decreased from **(€145.7 million)** at January 1, 2024, to **(€238.2 million)** at March 31, 2024, driven by a **€131.0 million** net loss Unaudited Condensed Consolidated Interim Statements of Changes in Equity | Metric | Three Months Ended March 31, 2024 (EUR'000) | | :------------------------------------ | :------------------------------------------ | | Equity at January 1, 2024 | (145,697) | | Net profit/(loss) for the period | (131,035) | | Share-based payment | 17,281 | | Capital increase | 21,209 | | Equity at March 31, 2024 | (238,179) | - Total comprehensive loss for the period was **€131.0 million**[16](index=16&type=chunk) - Share-based payment contributed **€17.3 million** to equity, and capital increase added **€21.2 million**[16](index=16&type=chunk) [Unaudited Condensed Consolidated Interim Cash Flow Statements](index=9&type=section&id=Unaudited%20Condensed%20Consolidated%20Interim%20Cash%20Flow%20Statements) Operating cash flow improved to **(€101.6 million)** in Q1 2024, while investing cash flow decreased significantly to **€7.2 million**, and financing cash flow turned positive at **€18.4 million** Unaudited Condensed Consolidated Interim Cash Flow Statements | Cash Flow Activity | Three Months Ended March 31, 2024 (EUR'000) | Three Months Ended March 31, 2023 (EUR'000) | | :---------------------------------- | :------------------------------------------ | :------------------------------------------ | | Operating activities | (101,582) | (147,044) | | Investing activities | 7,155 | 210,646 | | Financing activities | 18,423 | (702) | | Net increase/(decrease) in cash and cash equivalents | (76,004) | 62,900 | | Cash and cash equivalents at March 31 | 320,239 | 501,281 | - Cash used in operating activities decreased by **€45.5 million**, reflecting improved operational performance[20](index=20&type=chunk) - Cash from investing activities decreased by **€203.5 million**, mainly due to lower settlements of marketable securities[20](index=20&type=chunk) - Cash from financing activities increased by **€19.1 million**, driven by warrant exercises[20](index=20&type=chunk) [Notes to the Unaudited Condensed Consolidated Interim Financial Statements](index=10&type=section&id=Notes%20to%20the%20Unaudited%20Condensed%20Consolidated%20Interim%20Financial%20Statements) [Note 1—General Information](index=10&type=section&id=Note%201%E2%80%94General%20Information) Ascendis Pharma A/S, a global biopharma company headquartered in Hellerup, Denmark, has its ADSs listed on The Nasdaq Global Select Market under 'ASND' since February 2015 - Ascendis Pharma A/S was incorporated in 2006 and is headquartered in **Hellerup, Denmark**[21](index=21&type=chunk) - The company's ADSs have been listed on **The Nasdaq Global Select Market** under the symbol **'ASND'** since **February 2, 2015**[21](index=21&type=chunk) [Note 2—Summary of Material Accounting Policies](index=10&type=section&id=Note%202%E2%80%94Summary%20of%20Material%20Accounting%20Policies) Interim financial statements adhere to IAS 34, with IAS 1 amendments effective January 1, 2024, reclassifying convertible notes and derivative liabilities to current - Unaudited condensed consolidated interim financial statements are prepared in accordance with **International Accounting Standard 34, 'Interim Financial Reporting'**[23](index=23&type=chunk) - Amendments to **IAS 1**, effective **January 1, 2024**, required reclassification of **convertible notes (€407.1 million)** and **derivative liabilities (€143.3 million)** from non-current to current liabilities as of December 31, 2023, due to the embedded derivative not being an equity instrument[25](index=25&type=chunk)[27](index=27&type=chunk) - **Lease liabilities** were reclassified from a separate line item to be part of borrowings in the consolidated statements of financial position[28](index=28&type=chunk) [Note 3—Significant Accounting Judgements and Estimates](index=12&type=section&id=Note%203%E2%80%94Significant%20Accounting%20Judgements%20and%20Estimates) Management's accounting judgments and estimates, based on historical experience, show no material impact from revisions or changes compared to December 31, 2023 - Management's ongoing revisions of critical accounting estimates have not revealed any **material impact** in the periods presented[33](index=33&type=chunk) - **No changes** to the application of significant accounting judgments or estimation uncertainties compared to December 31, 2023[33](index=33&type=chunk) [Note 4—Significant Events in the Reporting Period](index=12&type=section&id=Note%204%E2%80%94Significant%20Events%20in%20the%20Reporting%20Period) Significant events include the formation of Eyconis, Inc. in January 2024 and a negative consolidated equity balance of **€238.2 million**, though capital resources are sufficient for twelve months - Ascendis Pharma A/S announced the formation and launch of **Eyconis, Inc.** on **January 29, 2024**, granting exclusive rights to TransCon ophthalmology assets globally in exchange for approximately **42% ownership** of Eyconis[34](index=34&type=chunk) - The unaudited condensed consolidated interim statements of financial position presented a **negative balance of equity of €238.2 million** as of March 31, 2024[35](index=35&type=chunk) - Despite negative consolidated equity, the parent company (Ascendis Pharma A/S) holds a **positive equity balance**, and existing capital resources are believed to be **sufficient for at least twelve months**[35](index=35&type=chunk)[36](index=36&type=chunk) [Note 5—Revenue](index=13&type=section&id=Note%205%E2%80%94Revenue) Total revenue for Q1 2024 significantly increased to **€95.9 million** from **€33.6 million** in Q1 2023, driven by commercial product sales and **€24.8 million** in Eyconis license revenue Revenue | Revenue Category | Three Months Ended March 31, 2024 (EUR'000) | Three Months Ended March 31, 2023 (EUR'000) | | :------------------------------------ | :------------------------------------------ | :------------------------------------------ | | Sale of commercial products | 66,499 | 31,551 | | Rendering of services | 4,624 | 1,170 | | Sale of clinical supply | 1 | 254 | | Licenses | 24,770 | 614 | | **Total revenue** | **95,894** | **33,589** | | Attributable to Commercial customers | 66,499 | 31,551 | | Attributable to Collaboration partners and license agreements | 29,395 | 2,038 | | Recognized over time | 4,624 | 1,170 | | Recognized at a point in time | 91,270 | 32,419 | | Europe | 1,567 | — | | North America | 92,681 | 33,070 | | Asia | 1,646 | 519 | - Revenue from commercial products (SKYTROFA and YORVIPATH) increased by **110.8%** to **€66.5 million** in Q1 2024[38](index=38&type=chunk)[39](index=39&type=chunk) - License revenue significantly increased to **€24.8 million** in Q1 2024, primarily from a non-cash upfront payment related to the Eyconis equity position[38](index=38&type=chunk)[44](index=44&type=chunk) - **North America** accounted for the vast majority of revenue (**€92.7 million**) in Q1 2024[38](index=38&type=chunk) [Note 6—Segment Information](index=15&type=section&id=Note%206%E2%80%94Segment%20Information) The Company operates as a single business unit, thus no additional segment or geographical information beyond revenue is disclosed - The Company is managed and operated as **one business unit**[47](index=47&type=chunk) [Note 7—Share-based Payment](index=15&type=section&id=Note%207%E2%80%94Share-based%20Payment) Share-based compensation costs increased to **€17.3 million** in Q1 2024, with **1,226,958** total RSU and PSU units outstanding as of March 31, 2024 - Share-based compensation costs recognized in Q1 2024 were **€17.3 million**, up from **€13.7 million** in Q1 2023[49](index=49&type=chunk) Restricted Stock Units (RSU) and Performance Stock Units (PSU) Activity | Metric | Restricted Stock Units (Number) | Performance Stock Units (Number) | Total (Number) | | :-------------------------- | :------------------------------ | :------------------------------- | :------------- | | Outstanding January 1, 2024 | 576,625 | 105,023 | 681,648 | | Granted during the period | 694,908 | 92,655 | 787,563 | | Transferred during the period | (176,317) | (35,007) | (211,324) | | Forfeited during the period | (30,929) | — | (30,929) | | Outstanding March 31, 2024 | 1,064,287 | 162,671 | 1,226,958 | Warrant Activity | Warrant Activity | Weighted Average Exercise Price (EUR) | | :-------------------------- | :------------------------------------ | | Outstanding January 1, 2024 | 86.38 | | Granted during the period | 133.09 | | Exercised during the period | 39.72 | | Forfeited during the period | 107.73 | | Outstanding March 31, 2024 | 90.52 | [Note 8—Share Capital](index=18&type=section&id=Note%208%E2%80%94Share%20Capital) As of March 31, 2024, share capital comprises **58,224,419** fully paid shares, each with a nominal value of **DKK 1** - Share capital consists of **58,224,419** fully paid shares with a nominal value of **DKK 1** each[60](index=60&type=chunk) [Note 9—Treasury Shares](index=18&type=section&id=Note%209%E2%80%94Treasury%20Shares) Treasury shares decreased from **1,093,054** (1.9%) at January 1, 2024, to **881,730** (1.5%) at March 31, 2024, due to stock incentive transfers Treasury Shares | Treasury shares | Nominal values (EUR'000) | Holding (Number) | Holding in % of total outstanding shares | | :-------------------------------- | :----------------------- | :--------------- | :--------------------------------------- | | January 1, 2024 | 146 | 1,093,054 | 1.9% | | Transferred under stock incentive programs | (28) | (211,324) | — | | March 31, 2024 | 118 | 881,730 | 1.5% | - Treasury shares decreased by **211,324 units**, representing a reduction from **1.9%** to **1.5%** of total outstanding shares[62](index=62&type=chunk) [Note 10—Financial Assets and Liabilities](index=19&type=section&id=Note%2010%E2%80%94Financial%20Assets%20and%20Liabilities) Total financial assets decreased to **€366.2 million**, while total financial liabilities increased to **€961.1 million**, with convertible notes and derivative liabilities reclassified as current Financial Assets and Liabilities | Financial Assets by Category | March 31, 2024 (EUR'000) | December 31, 2023 (EUR'000) | | :---------------------------------- | :----------------------- | :-------------------------- | | Trade receivables | 41,092 | 35,874 | | Other receivables | 4,845 | 3,909 | | Marketable securities | — | 7,275 | | Cash and cash equivalents | 320,239 | 392,164 | | **Total financial assets** | **366,176** | **439,222** | | | | | | Financial Liabilities by Category | March 31, 2024 (EUR'000) | December 31, 2023 (EUR'000) | | :---------------------------------- | :----------------------- | :-------------------------- | | Convertible senior notes | 424,984 | 407,095 | | Royalty funding liabilities | 146,233 | 138,377 | | Lease liabilities | 97,797 | 98,793 | | Trade payables and accrued expenses | 94,526 | 94,566 | | Other liabilities | 299 | — | | Derivative liabilities | 197,291 | 143,296 | | **Total financial liabilities** | **961,130** | **882,127** | - Convertible senior notes increased to **€425.0 million** (fair value **€387.3 million**) at March 31, 2024, from **€407.1 million** (fair value **€385.4 million**) at December 31, 2023[65](index=65&type=chunk)[68](index=68&type=chunk)[76](index=76&type=chunk) - Derivative liabilities, related to the foreign currency conversion option in convertible notes, increased to **€197.3 million** at March 31, 2024, from **€143.3 million** at December 31, 2023, measured using the Black-Scholes option pricing model[65](index=65&type=chunk)[72](index=72&type=chunk)[76](index=76&type=chunk) - A **10% relative increase in volatility** would increase derivative liabilities by approximately **€16.2 million**, and a **10% increase in share price** would increase them by approximately **€35.5 million**[74](index=74&type=chunk) [Note 11—Subsequent Events](index=24&type=section&id=Note%2011%E2%80%94Subsequent%20Events) No subsequent events have occurred after the balance sheet date that would influence these interim financial statements - **No subsequent events** occurred after the balance sheet date that would influence the financial statements[81](index=81&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=25&type=section&id=Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) [Special Note Regarding Forward-Looking Statements](index=25&type=section&id=Special%20Note%20Regarding%20Forward-Looking%20Statements) This section cautions that forward-looking statements involve risks and uncertainties, and actual results may differ materially, advising readers to consult the annual report's risk factors - The report contains **forward-looking statements** about business, operations, and financial performance, identifiable by terms like 'aim,' 'anticipate,' 'believe,' 'expect,' 'plan,' 'will,' etc[83](index=83&type=chunk) - These statements are based on current expectations and involve **known and unknown risks, uncertainties**, and other factors that may cause actual results to differ materially[85](index=85&type=chunk) - Readers are cautioned **not to rely on forward-looking statements** as predictions of future events and are advised to review risk factors from the **Annual Report on Form 20-F**[85](index=85&type=chunk)[86](index=86&type=chunk) [Overview](index=26&type=section&id=Overview) Ascendis Pharma A/S utilizes its TransCon platform to develop best-in-class therapies in Endocrinology Rare Disease and Oncology, aiming for blockbuster status for multiple products by 2030 - Ascendis Pharma A/S uses its **TransCon technology platform** to build a leading, fully integrated, global biopharma company[87](index=87&type=chunk) - The company's **Vision 2030** aims to achieve **blockbuster status (>$1 billion)** for **TransCon PTH, TransCon hGH, and TransCon CNP**, and expand into other therapeutic areas like oncology and GLP-1[88](index=88&type=chunk)[91](index=91&type=chunk) [Our Vision](index=26&type=section&id=Our%20Vision) Vision 2030 targets **blockbuster status** for TransCon PTH, TransCon hGH, and TransCon CNP, aiming for leadership in endocrinology rare diseases and expansion into oncology - **Vision 2030** aims for **blockbuster status (>$1 billion)** for **TransCon PTH, TransCon hGH, and TransCon CNP** through worldwide commercialization[88](index=88&type=chunk) - The company plans to expand its pipeline with **Endocrinology Rare Disease** **blockbuster product opportunities** and pursue TransCon product opportunities in **>$5 billion indications** in other therapeutic areas[88](index=88&type=chunk)[89](index=89&type=chunk) - A key part of the vision is to **remain independent** as a **profitable biopharma company**, driven by values of Patients, Science, and Passion[89](index=89&type=chunk) [Ascendis Algorithm for Product Innovation](index=27&type=section&id=Ascendis%20Algorithm%20for%20Product%20Innovation) The Ascendis Algorithm applies TransCon technologies to clinically validated parent drugs, optimizing safety and efficacy while reducing development risks for unmet medical needs in large markets - The algorithm focuses on identifying indications with **unmet medical needs**, **clinically validated parent drugs/pathways**, suitability for **TransCon technologies**, and potential for **differentiated products** in **large markets**[90](index=90&type=chunk) - This approach leverages **established clinical safety and efficacy data**, which is believed to **increase the probability of success** and **reduce risks** compared to traditional drug development[90](index=90&type=chunk) [TransCon Product Candidates Pipeline](index=27&type=section&id=TransCon%20Product%20Candidates%20Pipeline) Ascendis Pharma markets **SKYTROFA** and **YORVIPATH**, with a pipeline of five clinical-stage candidates in Endocrinology Rare Disease and Oncology, and has formed Eyconis for ophthalmology assets - **SKYTROFA (TransCon hGH)** is marketed in the **U.S.** for pediatric GHD since **October 2021** and in **Germany** since **September 2023**[92](index=92&type=chunk) - **YORVIPATH (TransCon PTH)** is marketed in the **EU, EEA, and Great Britain** for chronic hypoparathyroidism, commercially available in **Germany and Austria** since **January 2024**[93](index=93&type=chunk) - The pipeline includes **TransCon hGH** for adult GHD and Turner syndrome; **TransCon PTH** for U.S. adult hypoparathyroidism; **TransCon CNP** for achondroplasia; and oncology candidates **TransCon TLR7/8 Agonist** and **TransCon IL-2 b/g**[93](index=93&type=chunk) - **Eyconis, Inc.** was formed in **January 2024** to develop and commercialize TransCon ophthalmology assets globally[93](index=93&type=chunk) [Global Commercialization Strategy](index=30&type=section&id=Global%20Commercialization%20Strategy) Ascendis Pharma is building a global commercial presence through U.S. operations, expanding 'Europe Direct' in key countries, and leveraging distribution and license agreements in International Markets - The company has established a multi-faceted organization in the **U.S.** for **SKYTROFA commercialization**, serving as a foundation for future Endocrinology Rare Disease product launches[96](index=96&type=chunk) - Expanding **'Europe Direct'** presence in select countries, starting with **Germany** for **SKYTROFA and YORVIPATH**, and establishing organizations for country clusters like DACH, France & BeNeLux, Iberia, Nordics, and UK & Ireland[96](index=96&type=chunk) - Global reach is extended through exclusive distribution agreements in **'International Markets'** (e.g., Asia, Central & Eastern Europe, Middle East, Israel) and exclusive license agreements with partners like **VISEN (Greater China)** and **Teijin Limited (Japan)**[96](index=96&type=chunk) [TransCon Technologies](index=30&type=section&id=TransCon%20Technologies) TransCon technologies optimize drug safety and efficacy through predictable, sustained release of unmodified parent drugs via a three-component system, reducing development risk - TransCon technologies combine conventional prodrug and sustained release benefits to extend drug action, aiming for **optimized safety, efficacy, tolerability, and convenience**[97](index=97&type=chunk) - TransCon molecules consist of a **parent drug, an inert carrier, and a linker** that temporarily binds them, enabling **predictable release** of the active, unmodified parent drug[98](index=98&type=chunk) - Carriers include **systemic mPEG** for systemic exposure (used in TransCon hGH, PTH, CNP) and **localized hydrogels** for targeted delivery, with a **novel prolongation technology** developed in 2023[100](index=100&type=chunk)[101](index=101&type=chunk) - **Self-cleaving TransCon linkers** enable transient conjugation and **predictable release rates**, avoiding metabolic variability of conventional prodrugs[102](index=102&type=chunk) [TransCon Products – Endocrinology Rare Disease](index=33&type=section&id=TransCon%20Products%20%E2%80%93%20Endocrinology%20Rare%20Disease) Ascendis Pharma advances its Endocrinology Rare Disease portfolio with marketed **SKYTROFA** and **YORVIPATH**, and **TransCon CNP** in pivotal trials for achondroplasia, demonstrating efficacy across multiple indications - **SKYTROFA (TransCon hGH)** is approved in the **U.S. and EU** for **pediatric GHD** and is being evaluated for adult GHD and Turner syndrome[92](index=92&type=chunk)[115](index=115&type=chunk)[121](index=121&type=chunk) - **YORVIPATH (TransCon PTH)** received regulatory approval in the **EU, EEA, and Great Britain** for **chronic hypoparathyroidism** and is commercially available in Germany and Austria[93](index=93&type=chunk)[131](index=131&type=chunk) - **TransCon CNP** is in **pivotal trials** for **achondroplasia** (ApproaCH, ACcomplisH, reACHin, COACH) and plans to initiate a trial for adults with achondroplasia in Q4 2024[93](index=93&type=chunk)[150](index=150&type=chunk) [TransCon Growth Hormone (hGH)](index=33&type=section&id=TransCon%20Growth%20Hormone%20%28hGH%29) SKYTROFA (TransCon hGH), approved in the U.S. and EU for pediatric GHD, demonstrated superiority in adult GHD, with a BLA resubmission planned for Q3 2024 - **SKYTROFA (TransCon hGH)** is approved in the **U.S.** for **pediatric GHD** (since Oct 2021) and in the **EU** for children and adolescents (since Sep 2023 in Germany)[92](index=92&type=chunk)[115](index=115&type=chunk) - The **foresiGHt Trial** demonstrated TransCon hGH's **superiority in adult GHD**, with a mean difference of **-2.04% in trunk percent fat** compared to placebo (**p < 0.0001**)[122](index=122&type=chunk)[123](index=123&type=chunk) - A **supplemental BLA** for **adult GHD** is planned for submission to the **FDA** in **Q3 2024**[122](index=122&type=chunk) - The **enliGHten Trial** showed **59%** of **pediatric GHD** treatment completers met or exceeded their average parental height SDS after a mean of 3.2 years of TransCon hGH treatment[116](index=116&type=chunk) [TransCon PTH](index=37&type=section&id=TransCon%20PTH) YORVIPATH (TransCon PTH) is approved in the EU/EEA/GB for chronic hypoparathyroidism, with U.S. NDA resubmission accepted and clinical trials showing improved kidney function - **TransCon PTH** received regulatory approval in the **EU, EEA, and Great Britain**, marketed as **YORVIPATH**, and became commercially available in **Germany and Austria** in **January 2024**[131](index=131&type=chunk) - The **FDA accepted the NDA resubmission** for **TransCon PTH** for **adult hypoparathyroidism**, with a **PDUFA goal date of May 14, 2024**, addressing prior manufacturing control strategy concerns[133](index=133&type=chunk) - Post hoc analysis of the **Phase 3 PaTHway Trial** showed TransCon PTH treatment resulted in a **mean increase in eGFR of 7.9 mL/min/1.73m2** at Week 26 (**p<0.0001**) and **8.9 mL/min/1.73m2** at Week 52 (**p<0.0001**)[134](index=134&type=chunk)[135](index=135&type=chunk) - In the **PaTHway Trial**, **52%** of patients with baseline eGFR < 60 mL/min/1.73m2 improved to eGFR ≥ 60 mL/min/1.73m2 by Week 26, increasing to **57%** by Week 52[136](index=136&type=chunk)[137](index=137&type=chunk) [TransCon CNP](index=42&type=section&id=TransCon%20CNP) TransCon CNP, a once-weekly prodrug for achondroplasia, is in pivotal trials, with ACcomplisH showing improved growth velocity and quality of life; IND for adults and ApproaCH topline results are expected in Q4 2024 - **TransCon CNP** is designed to provide **sustained release of active CNP** for **achondroplasia**, minimizing binding to NPR-C and NPR-B receptors to avoid hypotension[149](index=149&type=chunk) - The **ACcomplisH trial** demonstrated TransCon CNP at **100 µg/kg/week** was **superior to placebo** on **annualized growth velocity (AGV)** at 52 weeks (**p=0.0218**)[158](index=158&type=chunk) - ACcomplisH also showed **significant improvements in health-related quality of life** (**SF-10 Physical Summary, p=0.002**) and disease impacts (ACEM Daily Living Function p=0.047, ACEM Emotional Well-being p=0.045)[151](index=151&type=chunk)[152](index=152&type=chunk) - Enrollment in the pivotal **Phase 3 ApproaCH Trial** is complete, with **topline results expected in Q4 2024**, and an **IND for adults with achondroplasia** is planned for **Q4 2024**[150](index=150&type=chunk)[155](index=155&type=chunk) [TransCon Product Candidates—Oncology](index=45&type=section&id=TransCon%20Product%20Candidates%E2%80%94Oncology) Ascendis Pharma is developing two clinical-stage oncology candidates, TransCon TLR7/8 Agonist and TransCon IL-2 b/g, to enhance anti-tumor effects and limit toxicities, with initial data expected by end of 2024 - The company is leveraging **TransCon technologies** to **enhance anti-tumor effects** of clinically-validated parent drugs and pathways, aiming to **improve efficacy** while **limiting adverse events**[163](index=163&type=chunk) - **TransCon TLR7/8 Agonist** is an investigational long-acting prodrug for **sustained intratumoral release of resiquimod**, with dose escalation completed and enrollment ongoing in four indication-specific cohorts of the **transcendIT-101 Trial**[166](index=166&type=chunk)[167](index=167&type=chunk) - **TransCon IL-2 b/g (onvapegleukin alfa)** is an investigational long-acting prodrug for **sustained systemic release of an IL-2 variant**, with dose escalation completed and enrollment ongoing in multiple indication-specific dose expansion cohorts of the **IL-Believe Trial**[168](index=168&type=chunk)[170](index=170&type=chunk)[171](index=171&type=chunk) - **Initial data** from dose expansion cohorts for both oncology candidates are expected by the **end of 2024**, including evaluation as a potential combination therapy[166](index=166&type=chunk)[168](index=168&type=chunk)[169](index=169&type=chunk) [Strategic Collaborations](index=48&type=section&id=Strategic%20Collaborations) Ascendis Pharma uses strategic collaborations to expand TransCon technologies and product candidates globally, notably with Teijin Limited for TransCon hGH, PTH, and CNP in Japan - **Strategic collaborations** aim to leverage **TransCon technologies** in certain geographies and therapeutic areas where partners have expertise, capability, and capital[173](index=173&type=chunk) - In **November 2023**, an exclusive license agreement was signed with **Teijin Limited** for **TransCon hGH, TransCon PTH, and TransCon CNP in Japan**, including a **$70 million upfront payment**, up to **$175 million in development/regulatory milestones**, and royalties[174](index=174&type=chunk) [Strategic Investments](index=48&type=section&id=Strategic%20Investments) Strategic investments include VISEN Pharmaceuticals for Greater China and Eyconis, Inc. for ophthalmology assets, expanding global presence and product development - Ascendis Pharma formed **VISEN Pharmaceuticals** in **November 2018** to develop and commercialize its endocrinology rare disease therapies in **Greater China**, retaining **43.93% ownership** after Series B financing[175](index=175&type=chunk) - VISEN announced **BLA acceptance** for **lonapegsomatropin (TransCon hGH)** by the **China National Medical Products Administration** in **March 2024**[175](index=175&type=chunk) - In **January 2024**, Ascendis Pharma announced the formation of **Eyconis, Inc.** with institutional investors, granting **exclusive rights to develop and commercialize TransCon ophthalmology assets globally** in exchange for an equity position and eligibility for future milestone payments and single-digit royalties[184](index=184&type=chunk) [Results of Operations](index=52&type=section&id=Results%20of%20Operations) Revenue surged to **€95.9 million** in Q1 2024, but net loss widened to **€131.0 million** due to increased finance expenses, despite decreased R&D costs and stable SG&A Results of Operations | Metric | Three Months Ended March 31, 2024 (EUR'000) | Three Months Ended March 31, 2023 (EUR'000) | | :-------------------------- | :------------------------------------------ | :------------------------------------------ | | Revenue | 95,894 | 33,589 | | Cost of sales | 7,569 | 4,621 | | Gross profit | 88,325 | 28,968 | | Research and development costs | 70,687 | 106,114 | | Selling, general and administrative expenses | 66,783 | 66,539 | | Operating profit/(loss) | (49,145) | (143,685) | | Finance income | 3,575 | 45,135 | | Finance expenses | 77,161 | 9,840 | | Net profit/(loss) for the period | (131,035) | (110,914) | - Revenue increased by **€62.3 million (185.5%)** to **€95.9 million**, driven by SKYTROFA sales, **€24.8 million** non-cash license revenue from Eyconis, and YORVIPATH launch[187](index=187&type=chunk) - Net loss for the period increased by **€20.1 million** to **€131.0 million**, primarily due to a **€67.3 million increase** in finance expenses and a **€41.6 million decrease** in finance income[186](index=186&type=chunk)[195](index=195&type=chunk) - Research and development costs decreased by **€35.4 million** to **€70.7 million**, reflecting pipeline maturity and a **€10.6 million reversal** of prior period write-downs for TransCon PTH pre-launch inventories[191](index=191&type=chunk) [Revenue](index=52&type=section&id=Revenue) Q1 2024 revenue surged to **€95.9 million**, a **€62.3 million** increase, driven by SKYTROFA sales, **€24.8 million** Eyconis license revenue, and YORVIPATH launch - Revenue for Q1 2024 was **€95.9 million**, an increase of **€62.3 million** compared to Q1 2023[187](index=187&type=chunk) - Increase primarily attributable to sale of **SKYTROFA**, **€24.8 million non-cash license revenue** from **Eyconis**, and the launch of **YORVIPATH**[187](index=187&type=chunk) Sale of Commercial Products | Sale of commercial products | March 31, 2023 (EUR'000) | June 30, 2023 (EUR'000) | September 30, 2023 (EUR'000) | December 31, 2023 (EUR'000) | March 31, 2024 (EUR'000) | | :-------------------------- | :----------------------- | :---------------------- | :--------------------------- | :-------------------------- | :----------------------- | | Sale of commercial products | 31,551 | 35,895 | 46,968 | 64,249 | 66,499 | [Cost of Sales](index=52&type=section&id=Cost%20of%20Sales) Cost of sales for Q1 2024 increased by **€2.9 million** to **€7.6 million**, correlating with higher commercial revenue - Cost of sales for Q1 2024 was **€7.6 million**, an increase of **€2.9 million** compared to Q1 2023[189](index=189&type=chunk) - The increase was primarily attributable to **higher commercial revenue**[189](index=189&type=chunk) [Research and Development Costs](index=53&type=section&id=Research%20and%20Development%20Costs) R&D costs decreased by **€35.4 million** to **€70.7 million** in Q1 2024, driven by pipeline maturity and a **€10.6 million** reversal of TransCon PTH inventory write-downs - R&D costs for Q1 2024 were **€70.7 million**, a decrease of **€35.4 million** compared to Q1 2023[191](index=191&type=chunk) - This decrease was primarily due to **lower external project costs**, reflecting the **maturity of the endocrinology rare disease pipeline**[191](index=191&type=chunk) - Q1 2024 R&D costs include a **€10.6 million reversal (income)** of prior period write-downs of pre-launch inventories for **TransCon PTH** due to the launch of **YORVIPATH** in the EU[191](index=191&type=chunk) External Project Costs | External Project Costs | March 31, 2023 (EUR'000) | June 30, 2023 (EUR'000) | September 30, 2023 (EUR'000) | December 31, 2023 (EUR'000) | March 31, 2024 (EUR'000) | | :--------------------- | :----------------------- | :---------------------- | :--------------------------- | :-------------------------- | :----------------------- | | TransCon hGH | 18,010 | 12,882 | 20,892 | 12,197 | 11,816 | | TransCon PTH | 12,514 | 10,100 | 10,600 | 7,366 | (6,319) | | TransCon CNP | 10,945 | 13,473 | 18,627 | 15,570 | 15,744 | | TransCon IL-2 b/g | 11,195 | 8,532 | 10,279 | 1,987 | 6,964 | | TransCon TLR7/8 Agonist | 8,492 | 12,081 | 7,843 | 5,066 | 2,910 | | Ophthalmology | 1,721 | 3,038 | 5,543 | 6,419 | — | | Other project costs | 507 | 36 | 129 | 353 | 1,224 | | **Total external project costs** | **63,384** | **60,142** | **73,913** | **48,958** | **32,339** | [Selling, General and Administrative Expenses](index=53&type=section&id=Selling%2C%20General%20and%20Administrative%20Expenses) SG&A expenses slightly increased to **€66.8 million** in Q1 2024, driven by higher employee costs from commercial expansion, partially offset by lower external expenses - SG&A expenses for Q1 2024 were **€66.8 million**, a slight increase of **€0.2 million** compared to Q1 2023[193](index=193&type=chunk) - The increase was primarily due to **higher employee costs**, including the impact from **commercial expansion**, partly offset by lower external pre-launch and administrative expenses[193](index=193&type=chunk) Selling, General and Administrative Expenses | Selling, general and administrative expenses | March 31, 2023 (EUR'000) | June 30, 2023 (EUR'000) | September 30, 2023 (EUR'000) | December 31, 2023 (EUR'000) | March 31, 2024 (EUR'000) | | :------------------------------------------- | :----------------------- | :---------------------- | :--------------------------- | :-------------------------- | :----------------------- | | Employee costs | 27,473 | 30,225 | 28,381 | 29,627 | 33,543 | | Other costs | 37,573 | 38,876 | 34,133 | 32,016 | 31,795 | | Depreciation | 1,493 | 1,180 | 1,101 | 2,332 | 1,445 | | **Total selling, general and administrative expenses** | **66,539** | **70,281** | **63,615** | **63,975** | **66,783** | [Finance Income and Finance Expenses](index=54&type=section&id=Finance%20Income%20and%20Finance%20Expenses) Finance income decreased to **€3.6 million** in Q1 2024, while finance expenses surged to **€77.2 million**, both significantly impacted by a **€54 million** fair value loss on derivative liabilities - Finance income decreased by **€41.6 million** to **€3.6 million** in Q1 2024, primarily due to a **€54 million fair value loss** on derivative liabilities, compared to a **€41.2 million gain** in Q1 2023[195](index=195&type=chunk) - Finance expenses increased by **€67.3 million** to **€77.2 million** in Q1 2024, driven by the **€54 million fair value loss** on derivative liabilities, **€8.1 million higher exchange rate losses**, and **€4.8 million in interest charges** on royalty funding liabilities[195](index=195&type=chunk) [Liquidity and Capital Resources](index=55&type=section&id=Liquidity%20and%20Capital%20Resources) Cash and cash equivalents were **€320.2 million** as of March 31, 2024; despite negative consolidated equity, capital resources are deemed sufficient for twelve months, funded by equity, convertible debt, and collaborations - Cash and cash equivalents amounted to **€320.2 million** as of March 31, 2024[197](index=197&type=chunk) - The company reported a **negative consolidated equity balance of €238.2 million**, but the parent company holds a **positive equity balance**, mitigating legal or regulatory requirements[198](index=198&type=chunk) - Existing capital resources are believed to be **sufficient to meet projected cash requirements for at least twelve months** from the report date[198](index=198&type=chunk) - Funding sources include **$575.0 million** in 2.25% **convertible notes** issued in March 2022 and a **$150.0 million capped synthetic royalty funding agreement** with Royalty Pharma in September 2023[200](index=200&type=chunk) Cash Flow Activity | Cash Flow Activity | 2024 (EUR'000) | 2023 (EUR'000) | Change (EUR'000) | | :---------------------------------- | :------------- | :------------- | :--------------- | | Operating activities | (101,582) | (147,044) | 45,462 | | Investing activities | 7,155 | 210,646 | (203,491) | | Financing activities | 18,423 | (702) | 19,125 | | Net increase/(decrease) in cash and cash equivalents | (76,004) | 62,900 | (138,904) | [Off-balance Sheet Arrangements](index=58&type=section&id=Off-balance%20Sheet%20Arrangements) Ascendis Pharma A/S has not entered into any off-balance sheet arrangements or holdings in variable interest entities - The company has **not entered into any off-balance sheet arrangements or holdings in variable interest entities**[209](index=209&type=chunk) [Qualitative Disclosures about Market Risk](index=58&type=section&id=Qualitative%20Disclosures%20about%20Market%20Risk) Ascendis Pharma faces financial risks from foreign currency, interest rates, and credit, managing liquidity through cash reserves without using derivative hedging instruments - The company is exposed to financial risks from **foreign currency exchange rates (primarily U.S. Dollar), interest rates, and credit risk**[210](index=210&type=chunk)[211](index=211&type=chunk) - Foreign currency risk is **minimized by maintaining cash positions** in currencies of expected future expenses[211](index=211&type=chunk) - **Derivative liabilities** are measured at **fair value through profit or loss**, exposing profit or loss to **volatility from interest rate and share price changes**[212](index=212&type=chunk)[217](index=217&type=chunk) - **Credit risk on bank deposits is considered low** due to high credit-rated counterparties, and **no material impairment loss** was recognized for expected credit loss[214](index=214&type=chunk)[216](index=216&type=chunk)

Core Viewpoint - Ascendis Pharma A/S is set to report its first quarter 2024 financial results and provide a business update on May 2, 2024, after U.S. market close, followed by a conference call and live webcast to discuss the results [1] Group 1 - Ascendis Pharma utilizes its innovative TransCon technology platform to develop new therapies aimed at improving patient outcomes [2] - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [2] - Ascendis Pharma emphasizes its core values of Patients, Science, and Passion in its operations [2]

COPENHAGEN, Denmark, April 25, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it will report first quarter 2024 financial results and provide a business update on Thursday, May 2, 2024, after the close of the U.S. financial markets. Ascendis Pharma will also host a conference call and live webcast on May 2, 2024, at 4:30 p.m. Eastern Time (ET) to discuss its first quarter 2024 financial results. Those who would like to participate may access the live webcast here, or register in ...

COPENHAGEN, Denmark, April 24, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (NASDAQ:ASND) today announced that the United Kingdom's Medicines & Healthcare products Regulatory Agency (MHRA) has granted marketing authorization for YORVIPATH® (palopegteriparatide; developed as TransCon™ PTH) in Great Britain as a parathyroid hormone (PTH) replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism, and has also granted YORVIPATH orphan drug status. YORVIPATH is a prodrug of parath ...

Things are looking better for the biotech industry in 2024 after a decent performance in 2023. The world is cautiously wading through an uncertain macroeconomic environment. This year is likely to bring more cheer for the industry as the Fed has given clear indication of multiple interest rate cuts.The majority of the biotech players do not generate enough revenues to fund their operations and are dependent on external funds. A company starts generating revenues following a successful FDA approval and the l ...