Ascendis Pharma(ASND) - 2025 Q2 - Quarterly Report

FORM 6-K Information Ascendis Pharma A/S filed Form 6-K for August 2025 as a foreign private issuer, with annual reports under Form 20-F Filing Information Ascendis Pharma A/S filed Form 6-K for August 2025 as a foreign private issuer, submitting annual reports under Form 20-F - Ascendis Pharma A/S filed Form 6-K for August 2025 as a foreign private issuer[2] - The company files annual reports under Form 20-F[2] Incorporation by Reference The Form 6-K report is incorporated by reference into Ascendis Pharma A/S's Form S-8 and Form F-3 registration statements - The Form 6-K is incorporated by reference into multiple Form S-8 and Form F-3 registration statements of Ascendis Pharma A/S[5] Signatures The report was signed by Michael Wolff Jensen, Executive Vice President, Chief Legal Officer, on August 7, 2025 - The report was signed by Michael Wolff Jensen, Executive Vice President, Chief Legal Officer, on August 7, 2025[10] Unaudited Condensed Consolidated Interim Financial Statements The unaudited condensed consolidated interim financial statements show financial performance, position, equity changes, and cash flows Statements of Profit or (Loss) and Other Comprehensive Income or (Loss) Ascendis Pharma A/S reported significant revenue and gross profit increases, with narrowed net losses for the three and six months ended June 30, 2025 Consolidated Statement of Profit or (Loss) (EUR'000) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | :--- | :--- | | Revenue | 158,045 | 35,998 | 258,998 | 131,892 | | Cost of sales | 31,447 | 11,465 | 48,963 | 19,034 | | Gross profit | 126,598 | 24,533 | 210,035 | 112,858 | | Operating profit/(loss) | (52,951) | (133,257) | (157,164) | (182,402) | | Net profit/(loss) for the period | (38,855) | (109,380) | (133,482) | (240,415) | | Basic earnings/(loss) per share | (0.64) | (1.91) | (2.22) | (4.21) | | Diluted earnings/(loss) per share | (0.82) | (2.21) | (2.22) | (4.21) | - Revenue for the three months ended June 30, 2025, increased by 339% YoY to €158,045 thousand, and for the six months, it increased by 96% YoY to €258,998 thousand[15] - Net loss for the three months ended June 30, 2025, improved by 64% YoY to (€38,855) thousand, and for the six months, it improved by 44% YoY to (€133,482) thousand[15] Statements of Financial Position As of June 30, 2025, total assets and cash decreased, while total liabilities increased, leading to a larger negative equity balance Consolidated Interim Statements of Financial Position (EUR'000) | Metric | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Total assets | 1,087,902 | 1,179,495 | | Total equity | (187,572) | (105,706) | | Total liabilities | 1,275,474 | 1,285,201 | | Cash and cash equivalents | 494,046 | 559,543 | | Trade receivables | 110,452 | 166,280 | | Inventories | 303,381 | 295,609 | | Borrowings (Non-current) | 330,186 | 365,080 | | Borrowings (Current) | 418,073 | 458,207 | | Derivative liabilities | 186,579 | 150,670 | - Total equity decreased from (€105,706) thousand as of December 31, 2024, to (€187,572) thousand as of June 30, 2025[16] - Cash and cash equivalents decreased by 11.89% from €559,543 thousand to €494,046 thousand[16] Statements of Changes in Equity Total equity decreased from January 1 to June 30, 2025, primarily due to net loss and treasury share acquisitions, partially offset by a capital increase Consolidated Interim Statements of Changes in Equity (EUR'000) | Metric | Equity as of January 1, 2025 | Equity as of June 30, 2025 | | :--- | :--- | :--- | | Total Equity | (105,706) | (187,572) | | Net profit/(loss) for the period | — | (133,482) | | Share-based payment | — | 55,580 | | Acquisition of treasury shares | — | (17,396) | | Capital increase | — | 26,302 | - Total equity decreased by €81,866 thousand from January 1, 2025, to June 30, 2025[17] - Net loss for the period was (€133,482) thousand, contributing to the decrease in equity[17] Cash Flow Statements Cash flows used in operating activities significantly improved, while investing and financing activities shifted to net cash usage, decreasing cash and cash equivalents Consolidated Interim Cash Flow Statements (EUR'000) | Metric | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | | Operating activities | (21,654) | (162,890) | | Investing activities | (5,041) | 7,763 | | Financing activities | (11,043) | 15,968 | | Increase/(decrease) in cash and cash equivalents | (37,738) | (139,159) | | Cash and cash equivalents at June 30 | 494,046 | 258,696 | - Cash flows used in operating activities improved by €141,236 thousand, from (€162,890) thousand in 2024 to (€21,654) thousand in 2025[18] - Cash flows from investing activities decreased by €12,804 thousand, shifting from a net inflow of €7,763 thousand in 2024 to a net outflow of €5,041 thousand in 2025[18] Notes to the Unaudited Condensed Consolidated Interim Financial Statements The notes provide detailed context for the interim financial statements, covering accounting policies, significant events, and financial instrument specifics General Information Ascendis Pharma A/S is a global biopharmaceutical company headquartered in Denmark, with ADSs listed on Nasdaq since 2015 - Ascendis Pharma A/S is a global biopharmaceutical company founded in 2006, headquartered in Hellerup, Denmark[20] - The company's ADSs are listed on the Nasdaq Global Select Market under 'ASND' since February 2015[21] Summary of Material Accounting Policies Interim financial statements adhere to IAS 34, maintaining consistency with prior year policies, with IFRS 18 changes not expected to materially impact operations - Interim financial statements are prepared under IAS 34, consistent with prior year's IFRS policies[23][24] - IFRS 18, effective January 1, 2027, will introduce new categories in the statement of profit or loss but is not expected to have a material impact on operations or financial statements[27][29][30] Significant Accounting Judgements and Estimates Accounting judgments and estimates are consistently applied, with no material impact from revisions or changes in significant uncertainties since December 31, 2024 - Accounting judgments and estimates are based on historical experience and relevant factors, with ongoing reviews[31][33] - No material impact from revisions to critical accounting estimates, and no changes in significant accounting judgments compared to December 31, 2024[34] Significant Events in the Reporting Period Key events include VISEN's IPO, a share repurchase program, an impairment charge, and a negative equity balance not requiring re-establishment - VISEN Pharmaceuticals completed its IPO on the Hong Kong Stock Exchange on March 21, 2025, resulting in a non-cash gain of €33.6 million for Ascendis Pharma[35][36] - Ascendis Pharma A/S owned 39.2% in VISEN post-IPO, with a market value of approximately €203 million as of June 30, 2025[36] - A $18.25 million share repurchase program was fully executed by March 4, 2025[38] - An impairment charge of €7.5 million was recognized for property, plant and equipment due to changes in activities at a U.S. site[40] - The company reported a negative equity balance of €187.6 million as of June 30, 2025, but is not subject to legal requirements to re-establish it due to the parent company's positive equity[41] Revenue Substantial revenue growth for the three and six months ended June 30, 2025, was driven by commercial product sales, especially YORVIPATH, with North America as the largest contributor Revenue by Category (EUR'000) | Category | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | :--- | :--- | | Commercial products | 153,663 | 31,389 | 249,690 | 97,888 | | Rendering of services and clinical supply | 3,570 | 3,740 | 7,094 | 8,365 | | Licenses | 812 | 869 | 2,214 | 25,639 | | Total revenue | 158,045 | 35,998 | 258,998 | 131,892 | Revenue from Commercial Products (EUR'000) | Product | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | :--- | :--- | | SKYTROFA® | 50,706 | 26,202 | 102,044 | 91,207 | | YORVIPATH® | 102,957 | 5,187 | 147,646 | 6,681 | | Total commercial products | 153,663 | 31,389 | 249,690 | 97,888 | - Total revenue increased by €122.0 million (339%) for the three months and €127.1 million (96%) for the six months ended June 30, 2025, primarily driven by YORVIPATH launch in the U.S[43][44] - North America contributed €182.1 million to revenue for the six months ended June 30, 2025, compared to €122.1 million in the prior year[43] Earnings Per Share Basic and diluted earnings per share improved due to reduced net loss, with Q2 2024 diluted EPS restated for convertible notes impact Earnings Per Share (EUR) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 (Restated) | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | :--- | :--- | | Basic earnings per share | (0.64) | (1.91) | (2.22) | (4.21) | | Diluted earnings per share | (0.82) | (2.21) | (2.22) | (4.21) | | Weighted average number of ordinary shares for basic EPS | 60,454,589 | 57,345,613 | 60,237,774 | 57,114,435 | | Weighted average number of ordinary shares for diluted EPS | 63,911,374 | 60,802,398 | 60,237,774 | 57,114,435 | - Diluted earnings per share for the three months ended June 30, 2024, was restated from (€1.91) to (€2.21) to reflect the dilutive impact from convertible notes[48] Segment Information The company operates as a single business unit, with geographical revenue breakdown provided in Note 5 - Ascendis Pharma A/S is managed and operated as one business unit[49] - Revenue is specified by geographical area based on customer location, as detailed in Note 5[49] Share-based Payment Share-based compensation costs for the six months ended June 30, 2025, were €55.6 million, with RSU/PSU increases and warrant decreases - Share-based compensation costs recognized for the six months ended June 30, 2025, were €55.6 million, up from €43.3 million in the prior year[51] RSU and PSU Activity (Number) | Metric | Restricted Stock Units | Performance Stock Units | Total | | :--- | :--- | :--- | :--- | | Outstanding January 1, 2025 | 993,807 | 156,667 | 1,150,474 | | Granted during the period | 634,589 | 73,583 | 708,172 | | Settled during the period | (60,056) | (15,716) | (75,772) | | Transferred during the period | (321,392) | (46,588) | (367,980) | | Forfeited during the period | (43,820) | (2,688) | (46,508) | | Outstanding June 30, 2025 | 1,203,128 | 165,258 | 1,368,386 | Warrant Activity (Number and EUR) | Metric | Warrants (Number) | Weighted Average Exercise Price (EUR) | | :--- | :--- | :--- | | Outstanding January 1, 2025 | 6,204,122 | 93.25 | | Granted during the period | 246,743 | 131.88 | | Exercised during the period | (461,976) | 55.66 | | Forfeited during the period | (75,422) | 100.21 | | Outstanding June 30, 2025 | 5,913,467 | 97.74 | Share Capital As of June 30, 2025, share capital comprised 61,151,463 DKK 1 nominal value shares, increasing by 461,976 shares - Share capital consists of 61,151,463 fully paid shares at DKK 1 nominal value each[59] - Share capital increased by 461,976 shares for the six months ended June 30, 2025[59] Treasury Shares Treasury share holdings decreased from 1.4% to 1.0% of outstanding shares, primarily due to stock incentive program transfers Treasury Shares Holding | Metric | January 1, 2025 | June 30, 2025 | | :--- | :--- | :--- | | Nominal values (EUR'000) | 113 | 80 | | Holding (Number) | 845,887 | 597,055 | | % of total outstanding shares | 1.4% | 1.0% | - Treasury shares decreased by 248,832 shares from January 1, 2025, to June 30, 2025[60] Financial Assets and Liabilities Financial assets at amortized cost decreased, while derivative liabilities and borrowings increased, with complex instruments measured using Level 3 fair value inputs Financial Assets and Liabilities (EUR'000) | Category | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Financial assets measured at amortized cost | 608,919 | 729,787 | | Total financial assets | 608,919 | 729,787 | | Financial liabilities measured at amortized cost | 889,823 | 953,321 | | Derivative liabilities | 186,579 | 150,670 | | Total financial liabilities | 1,076,402 | 1,103,991 | - Financial assets measured at amortized cost decreased by €120,868 thousand[61] - Derivative liabilities, measured at fair value through profit or loss, increased by €35,909 thousand[61][80] Borrowings Borrowings primarily consist of $575.0 million convertible senior notes and capped synthetic royalty funding agreements for YORVIPATH and SKYTROFA - Issued $575.0 million of 2.25% convertible notes in March 2022, maturing April 1, 2028, with a carrying amount of €418.1 million as of June 30, 2025[62][65] - Entered into a $150.0 million capped synthetic royalty funding agreement for YORVIPATH in September 2024, exchanging a 3% royalty on U.S. net revenue until 2.0x (or 1.65x by Dec 31, 2029) the purchase price is reached[68][69] - Entered into a $150.0 million capped synthetic royalty funding agreement for SKYTROFA in September 2023, exchanging a 9.15% royalty on U.S. net revenue (starting Jan 1, 2025) until 1.925x (or 1.65x by Dec 31, 2031) the purchase price is reached[71][72] Derivative Liabilities Derivative liabilities, from convertible notes' foreign currency conversion option, are fair valued using Black-Scholes, sensitive to volatility and share price changes - Derivative liabilities are tied to the foreign currency conversion option in convertible notes[74] - Measured at fair value using the Black-Scholes option pricing model, with key assumptions including conversion price, share price, maturity, risk-free rate, and expected volatility (49.5% as of June 30, 2025)[75] - A 10% relative increase in volatility would increase derivative liabilities by €14.0 million, and a 10% increase in share price would increase them by €37.6 million, both negatively impacting profit or loss and equity[76][77] Fair Value Measurement Convertible notes, royalty funding, and derivative liabilities are Level 3 fair value measurements, using unobservable inputs Fair Value Measurement (EUR'000) | Item | June 30, 2025 Carrying Amount | June 30, 2025 Fair Value | December 31, 2024 Carrying Amount | December 31, 2024 Fair Value | Fair value level | | :--- | :--- | :--- | :--- | :--- | :--- | | Convertible senior notes | 418,073 | 411,689 | 458,207 | 438,288 | 3 | | Royalty funding liabilities | 283,530 | 289,322 | 305,379 | 305,673 | 3 | | Derivative liabilities | 186,579 | 186,579 | 150,670 | 150,670 | 3 | - Fair values of convertible senior notes, royalty funding liabilities, and derivative liabilities are classified as Level 3, relying on unobservable inputs[78][79] Maturity Analysis Maturity analysis of non-derivative financial liabilities shows significant contractual cash flows beyond one year, mainly from borrowings and lease liabilities Maturity Analysis of Non-Derivative Financial Liabilities (EUR'000) | Category | < 1 year | 1-5 years | >5 years | Total contractual cash-flows | Carrying amount | | :--- | :--- | :--- | :--- | :--- | :--- | | Borrowings (excluding lease liabilities) | 41,635 | 899,938 | — | 941,573 | 701,603 | | Lease liabilities | 15,270 | 58,290 | 29,897 | 103,457 | 90,931 | | Trade payables, accrued expenses and other liabilities | 97,289 | — | — | 97,289 | 97,289 | | Total financial liabilities | 154,194 | 958,228 | 29,897 | 1,142,319 | 889,823 | - Total contractual cash flows for financial liabilities amount to €1,142,319 thousand, with the majority (€958,228 thousand) due within 1-5 years[81] Subsequent Events In July 2025, the company extended its headquarters lease, increasing lease liability by approximately €9.0 million - Headquarters lease extended in July 2025, increasing lease liability by approximately €9.0 million[82] Management's Discussion and Analysis of Financial Condition and Results of Operations This section discusses the company's financial condition, operational results, strategic vision, product pipeline, and market risks Introduction and Forward-Looking Statements This section provides an overview of financial condition and results, with a cautionary note on forward-looking statements and inherent risks - Discussion and analysis are based on unaudited condensed consolidated interim financial statements prepared under IAS 34[84] - Forward-looking statements are subject to known and unknown risks and uncertainties, which may cause actual results to differ materially from current expectations[85][86] - The company assumes no obligation to update or revise forward-looking statements, and readers are cautioned not to rely on them as predictions of future events[86] Company Overview and Vision Ascendis Pharma A/S leverages its TransCon platform for rare diseases and oncology, with Vision 2030 targeting blockbuster products and pipeline expansion - Ascendis Pharma is a global biopharmaceutical company focused on its TransCon technology platform to develop best-in-class therapies[90] - Vision 2030 aims to achieve blockbuster status (>$1B) for TransCon PTH, TransCon hGH, and TransCon CNP, become a leader in growth disorders and hypoparathyroidism, and expand into other therapeutic areas through collaborations[91][93] - The company's algorithm for product innovation focuses on clinically validated parent drugs/pathways to optimize safety, efficacy, tolerability, and convenience, aiming to reduce traditional drug development risks[92][95] Company Overview Ascendis Pharma A/S is a global biopharmaceutical company using its TransCon platform for Endocrinology Rare Disease, Oncology, and partnered therapeutic areas - Ascendis Pharma applies its TransCon technology platform to develop therapies for patients, with a portfolio in Endocrinology Rare Disease and Oncology[90] - The company has established partnerships for TransCon-based products in Metabolic and Cardiovascular diseases and Ophthalmology[90] Our Vision Vision 2030 targets blockbuster status for key TransCon products, leadership in specific rare diseases, and pipeline expansion through collaborations - Vision 2030 aims for blockbuster status (>$1B) for TransCon PTH, TransCon hGH, and TransCon CNP through worldwide commercialization[93] - The company plans to expand its pipeline with Endocrinology Rare Disease blockbuster product opportunities and pursue TransCon product opportunities in >$5B indications in other therapeutic areas[92][93] - A core part of the vision is to maximize value creation through collaboration with therapeutic area market leaders and outperform industry drug development benchmarks[93] Ascendis Algorithm for Product Innovation The innovation algorithm applies TransCon technologies to validated parent drugs, optimizing safety and efficacy to reduce development risks for unmet medical needs - The algorithm focuses on applying TransCon technologies to clinically validated parent drugs/pathways to optimize safety, efficacy, tolerability, and convenience[95] - This approach aims to identify indications with unmet medical needs, potential for clearly differentiated products, and established development pathways[95] - Leveraging established clinical safety and efficacy data is believed to increase the probability of success compared to traditional drug development[95] Program Summaries and Pipeline Ascendis Pharma has two marketed products and a pipeline of clinical-stage candidates, including TransCon CNP under FDA review, leveraging its TransCon technologies globally - YORVIPATH® (TransCon PTH) is approved in the U.S. (since August 2024) and EU (since November 2023) for adult hypoparathyroidism, with ~3,100 unique patients prescribed in the U.S. through June 30, 2025[96][125] - SKYTROFA® (TransCon hGH) is approved in the U.S. (since October 2021) and EU (since September 2023) for pediatric GHD, and recently approved by FDA in July 2025 for adult GHD[96][150][151] - TransCon CNP (navepegritide) is under FDA priority review for achondroplasia in children, with a PDUFA goal date of November 30, 2025[96][165] TransCon Product Candidates Pipeline | Therapeutic Area | Product Candidate | Indication | Status | Region | | :--- | :--- | :--- | :--- | :--- | | Endocrinology Rare Diseases | TransCon CNP | Achondroplasia (children aged 2-11) | NDA Accepted | Multinational | | | TransCon CNP | Achondroplasia (children) | Long-Term Extension Trial | Multinational | | | TransCon hGH | Turner Syndrome (children aged 1-10) | Phase 2 | U.S. | | | TransCon PTH | Hypoparathyroidism in adults | Phase 3 | Multinational | | | TransCon CNP | Achondroplasia (infants) | Phase 2 | Multinational | | | TransCon CNP | Achondroplasia (adolescents) | Phase 2 | Multinational | | | TransCon CNP + TransCon hGH | Achondroplasia (children aged 2-11) | Phase 2 | Multinational | | Partner Programs | TransCon hGH | Pediatric GHD | BLA submitted | China | | | TransCon hGH | Pediatric GHD | Phase 3 | Japan | | | TransCon PTH | Hypoparathyroidism in adults | Completed Phase 3 | China | | | TransCon PTH | Hypoparathyroidism in adults | J-NDA submitted | Japan | | | TransCon CNP | Achondroplasia | Completed Phase 2 | China | | Oncology | TransCon IL-2 B/y | Various tumor types | Phase 2 | Multinational | Global Commercialization Strategy The company is building a global commercial presence through integrated U.S. operations, direct European expansion, international distribution, and strategic partnerships - Established an integrated organization in the U.S. for commercializing approved Endocrinology Rare Disease products (SKYTROFA and YORVIPATH)[102] - Expanding direct commercial presence in select European countries ('Europe Direct'), starting with Germany, and building infrastructure in other clusters[103] - Utilizes exclusive sales and distribution agreements for 'International Markets' (over 75 countries) and strategic collaborations with partners like VISEN (Greater China) and Teijin (Japan)[104][105] - The company believes the impact of 'Reciprocal Tariffs' on pharmaceuticals will be immaterial, but continues to monitor[106] TransCon Technologies TransCon technologies extend drug action duration via predictable, sustained release of unmodified parent drugs, optimizing safety and efficacy across therapeutic classes - TransCon technologies combine a parent drug, an inert carrier, and a linker to extend drug action duration, aiming for optimized safety, efficacy, tolerability, and convenience[107][108] - The technology enables predictable, sustained release of unmodified active parent drugs, potentially improving injection site tolerability and reducing systemic adverse effects[108][113] - By leveraging clinically validated parent drugs or pathways, the approach aims for a higher development and regulatory success rate[114][115] Overview TransCon technologies provide predictable, sustained delivery of active, unmodified parent drugs, improving safety, efficacy, and convenience with flexible dosing frequencies - TransCon technologies combine a parent drug, an inert carrier, and a linker to extend drug action duration[108] - The goal is to optimize safety, efficacy, tolerability, and convenience, with predictable release over time[107] - This approach can potentially support dosing frequencies from daily up to six months or more and offers intellectual property benefits[107] TransCon Technology Components TransCon technologies use carrier platforms and reversible linkers for predictable, sustained release of unmodified parent drugs, leveraging validated targets to reduce risk - TransCon carriers (systemic like mPEG, and localized hydrogels) inactivate and protect the drug, minimizing excretion and degradation, and improving tolerability[111][112] - Reversible TransCon linkers enable transient conjugation and predictable, self-cleaving release of unmodified parent drugs, tailored for various dosing frequencies and pharmacokinetic profiles[113] - The strategy focuses on clinically validated parent drugs to leverage existing knowledge, design optimal drug levels, and reduce development risk[114][115] TransCon Products and Product Candidates - Endocrinology Rare Disease Ascendis Pharma develops and commercializes TransCon products for Endocrinology Rare Diseases, including YORVIPATH, SKYTROFA, and TransCon CNP for achondroplasia - YORVIPATH (TransCon PTH) is approved in the U.S. and EU for adult hypoparathyroidism, demonstrating durable response, improved kidney function, and quality of life in long-term clinical trials[125][128][129] - SKYTROFA (TransCon hGH) is approved for pediatric GHD in the U.S. and EU, and recently for adult GHD in the U.S., with an auto-injector designed for patient convenience[150][151][153] - TransCon CNP is under FDA priority review for achondroplasia, with pivotal ApproaCH trial data showing superiority over placebo in annualized growth velocity and improvements in bone morphometry[165][168][171] - Combination therapy of TransCon CNP and TransCon hGH in achondroplasia showed accelerated improvements in growth and proportionality, exceeding the 97th percentile of average-stature children[190][191][193] Hypoparathyroidism YORVIPATH (TransCon PTH) is approved in the U.S. and EU for hypoparathyroidism, demonstrating durable response, improved kidney function, and quality of life - Hypoparathyroidism affects over 250,000 patients in the U.S. and Europe, leading to severe short-term and long-term complications[116][117][123] - TransCon PTH (YORVIPATH) is approved in the U.S. (August 2024) and EU (November 2023) for adult hypoparathyroidism, providing once-daily PTH (1-34) replacement[124][125] - Long-term data from PaTHway and PaTH Forward trials show sustained normalization of serum calcium, independence from conventional therapy, improved eGFR, and enhanced quality of life, with no new safety signals[128][129][131] Growth Disorders SKYTROFA (TransCon hGH) is a once-weekly prodrug approved for pediatric and adult GHD in the U.S. and EU, aiming to improve adherence and outcomes - GHD affects children and adults, with low adherence rates (approx. 50-60%) to daily hGH therapy, impacting treatment outcomes[139][141][142] - SKYTROFA (TransCon hGH) is a once-weekly prodrug of unmodified somatropin, approved for pediatric GHD in the U.S. (Oct 2021) and EU (Sep 2023), and for adult GHD in the U.S. (July 2025)[149][150][151] - The Phase 2 New InsiGHTS Trial in children with Turner syndrome showed TransCon hGH improved growth similar to daily somatropin, with comparable safety and tolerability[154] - SKYTROFA includes a proprietary auto-injector designed for room temperature storage, empty-all design, low-volume injection, and potential Bluetooth connectivity for adherence tracking[153] Achondroplasia TransCon CNP for achondroplasia is under FDA priority review, with pivotal trial data showing superior growth velocity and improved bone morphometry - Achondroplasia is a rare genetic condition affecting over 250,000 worldwide, causing skeletal dysplasia and severe complications, with significant unmet medical needs[156] - TransCon CNP (navepegritide) is an investigational once-weekly prodrug designed for sustained CNP release, aiming to avoid hypotension and effectively penetrate growth plates[163] - NDA for TransCon CNP for children with achondroplasia accepted for FDA priority review, with a PDUFA goal date of November 30, 2025[165] - Pivotal ApproaCH trial showed TransCon CNP superiority over placebo in annualized growth velocity (LS mean difference of 1.49 cm/year, p<0.0001) and improvements in bone morphometry, with a safety profile comparable to placebo[168][171][172] - The company is engaged in patent litigation with BioMarin regarding CNP therapies for achondroplasia[160] Combination Therapy (TransCon CNP + TransCon hGH) Combination therapy of TransCon CNP and TransCon hGH for achondroplasia shows significant improvements in growth and proportionality, with a consistent safety profile - Combination therapy of TransCon CNP and TransCon hGH is being evaluated for achondroplasia, aiming to improve health outcomes beyond monotherapy by combining two independent mechanisms of action[187] - Week 26 interim analysis from the COACH Trial showed TransCon hGH boosted TransCon CNP treatment benefits, resulting in significant growth and proportionality improvements[190] - The mean Annualized Growth Velocity (AGV) with combination treatment exceeded the 97th percentile of average-stature children, with a safety profile consistent with monotherapies[191][193] TransCon Product Candidates—Oncology Ascendis Pharma leverages TransCon technologies in oncology, with TransCon IL-2 b/g showing clinical activity in solid tumors with a well-tolerated safety profile - The company is leveraging TransCon technologies in oncology to enhance anti-tumor effects and provide sustained modulation of tumor microenvironments, aiming to improve efficacy and limit toxicities[198][199] - TransCon IL-2 b/g (onvapegleukin alfa) is an investigational long-acting prodrug designed for selective IL-2 activation, currently in Phase 1/2 clinical trials for various solid tumors[202] - Initial data from the IL-Believe Trial show clinical activity in heavily pre-treated patients with platinum-resistant ovarian cancer (29% response rate) and melanoma (40% response rate in combination with TransCon TLR7/8 Agonist), with a generally well-tolerated safety profile[203][204][205] Strategic Collaborations and Investments Strategic collaborations expand TransCon technologies globally and monetize product candidates through partnerships with Novo Nordisk, Teijin, VISEN, and Eyconis - Strategic collaborations aim to leverage TransCon technologies in new geographies and therapeutic areas, and monetize internal product candidates[209] - Partnerships include Novo Nordisk (metabolic/cardiovascular diseases), Teijin (Japan endocrinology rare diseases), VISEN Pharmaceuticals (Greater China endocrinology rare diseases), and Eyconis (global ophthalmology)[210][220][223][234] Novo Nordisk A/S Ascendis Pharma granted Novo Nordisk an exclusive worldwide license for TransCon technology in metabolic and cardiovascular diseases, including a $100 million upfront payment - Exclusive worldwide license granted to Novo Nordisk for TransCon technology in metabolic (including obesity and type 2 diabetes) and cardiovascular diseases[210][214] - Potential payments of up to $285 million for the lead program (TransCon Semaglutide), including a $100 million upfront fee received in January 2025[212] - Ascendis Pharma is eligible for additional development, regulatory, and sales-based milestone payments, plus tiered royalties on global net sales for licensed products[212] Teijin Limited Ascendis Pharma granted Teijin an exclusive license for TransCon hGH, PTH, and CNP in Japan, including a $70 million upfront payment and potential milestones - Exclusive license granted to Teijin for TransCon hGH, TransCon PTH, and TransCon CNP in Japan for endocrinology rare diseases[220] - Received $70 million upfront payment, with potential for up to $175 million in additional development and regulatory milestones[220] - Eligible to receive royalties on net sales in Japan, up to a mid-20s percentage, varying by product[220] - Teijin submitted an application for manufacturing and marketing approval of palopegteriparatide (TransCon PTH) for hypoparathyroidism in Japan in December 2024[221] VISEN Pharmaceuticals VISEN Pharmaceuticals, formed by Ascendis Pharma for Greater China, completed its IPO, with Ascendis retaining 39.2% ownership and positive clinical data reported - VISEN Pharmaceuticals was formed in November 2018 to develop and commercialize TransCon hGH, PTH, and CNP in Greater China[223] - VISEN completed its IPO on the Hong Kong Stock Exchange in March 2025, with Ascendis Pharma holding 39.2% ownership (market value ~€203 million as of June 30, 2025)[224] - VISEN reported positive Phase 3 data for TransCon PTH in adult hypoparathyroidism and Phase 2 data for TransCon CNP in achondroplasia in China[225][226] - BLA for lonapegsomatropin (TransCon hGH) was accepted by the China National Medical Products Administration in March 2024[226] Eyconis, Inc. Ascendis Pharma partnered with Frazier Life Sciences to form Eyconis for global TransCon ophthalmology assets, retaining a 40.8% equity position - Eyconis was formed in January 2024 to develop and commercialize TransCon ophthalmology assets globally[234] - Ascendis Pharma holds a 40.8% ownership in Eyconis as of June 30, 2025[235] - Eligible to receive development, regulatory, and sales milestone payments, plus single-digit royalties on global net sales[235] Results of Operations Significant revenue growth and narrowed net loss were reported, driven by YORVIPATH launch, with increased operating expenses and improved net finance income - Revenue increased by €122.0 million (339%) for Q2 2025 and €127.1 million (96%) for H1 2025, primarily due to YORVIPATH launch in the U.S[237][243] - Net loss improved by €70.5 million for Q2 2025 and €106.9 million for H1 2025, driven by higher revenue and a decline in net financial expenses[239][236] - Operating expenses increased by €21.8 million for Q2 2025 and €71.9 million for H1 2025, mainly due to commercial expansion and global launch activities for YORVIPATH[236][250] - Net finance income improved by €50.3 million for H1 2025, primarily due to net currency gains and reduced financial expenses[239][251] Financial Highlights Substantial revenue growth and reduced operating and net losses were reported, driven by YORVIPATH launch, with improved operating cash flows Financial Highlights (EUR'000) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Change | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change | | :--- | :--- | :--- | :--- | :--- | :--- | :--- | | Revenue | 158,045 | 35,998 | 122,047 | 258,998 | 131,892 | 127,106 | | Gross profit | 126,598 | 24,533 | 102,065 | 210,035 | 112,858 | 97,177 | | Operating profit/(loss) | (52,951) | (133,257) | 80,306 | (157,164) | (182,402) | 25,238 | | Net profit/(loss) for the period | (38,855) | (109,380) | 70,525 | (133,482) | (240,415) | 106,933 | | Cash flows from/(used in) operating activities | (7,342) | (61,307) | 53,965 | (21,654) | (162,890) | 141,236 | - Revenue increased by €122.0 million for the three months and €127.1 million for the six months ended June 30, 2025, primarily due to the launch of YORVIPATH in the U.S[237] - Operating loss improved by €80.3 million for the three months and €25.2 million for the six months, driven by higher revenue[238] Revenue Commercial product revenue significantly increased due to YORVIPATH launch and SKYTROFA demand, while license revenue decreased due to a prior-year non-cash agreement Revenue by Category (EUR'000) | Category | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Change | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change | | :--- | :--- | :--- | :--- | :--- | :--- | :--- | | Commercial products | 153,663 | 31,389 | 122,274 | 249,690 | 97,888 | 151,802 | | Rendering of services and clinical supply | 3,570 | 3,740 | (170) | 7,094 | 8,365 | (1,271) | | Licenses | 812 | 869 | (57) | 2,214 | 25,639 | (23,425) | | Total revenue | 158,045 | 35,998 | 122,047 | 258,998 | 131,892 | 127,106 | Revenue from Commercial Products (EUR'000) | Product | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Change | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change | | :--- | :--- | :--- | :--- | :--- | :--- | :--- | | SKYTROFA® | 50,706 | 26,202 | 24,504 | 102,044 | 91,207 | 10,837 | | YORVIPATH® | 102,957 | 5,187 | 97,770 | 147,646 | 6,681 | 140,965 | | Total commercial products | 153,663 | 31,389 | 122,274 | 249,690 | 97,888 | 151,802 | - Revenue from commercial products increased by €122.3 million for the three months and €151.8 million for the six months, primarily due to the launch of YORVIPATH in the U.S[243] - License revenue for the six months ended June 30, 2025, was lower by €23.4 million due to non-cash license revenue related to the Eyconis agreement in January 2024[244] Cost of Sales Cost of sales increased due to higher commercial sales volume and collaboration agreement costs - Cost of sales increased by €20.0 million for the three months and €29.9 million for the six months ended June 30, 2025[245] - The increase was primarily attributable to higher volume of commercial sales and costs related to collaboration agreements[245] Research and Development Expenses R&D expenses decreased for three months but slightly increased for six months, influenced by pipeline maturity, employee costs, and an impairment charge Research and Development Expenses (EUR'000) | Category | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Change | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change | | :--- | :--- | :--- | :--- | :--- | :--- | :--- | | Total external project costs | 30,639 | 44,881 | (14,242) | 67,492 | 77,220 | (9,728) | | Employee costs | 36,776 | 33,513 | 3,263 | 74,719 | 64,779 | 9,940 | | Impairment | — | — | — | 4,054 | — | 4,054 | | Total research and development expenses | 71,988 | 83,478 | (11,490) | 158,591 | 154,165 | 4,426 | - R&D expenses decreased by €11.5 million for the three months but increased by €4.4 million for the six months ended June 30, 2025[247] - The changes were primarily due to the maturity of the endocrinology rare disease pipeline, higher employee costs, and a €4.1 million impairment charge on property, plant and equipment for the six-month period[249] Selling, General and Administrative Expenses SG&A expenses significantly increased due to commercial expansion, global YORVIPATH launch activities, and an impairment charge Selling, General and Administrative Expenses (EUR'000) | Category | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Change | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change | | :--- | :--- | :--- | :--- | :--- | :--- | :--- | | Employee costs | 50,897 | 35,409 | 15,488 | 101,217 | 68,951 | 32,266 | | Other costs | 54,813 | 36,886 | 17,927 | 100,142 | 68,682 | 31,460 | | Impairment | — | — | — | 3,454 | — | 3,454 | | Total SG&A expenses | 107,561 | 74,312 | 33,249 | 208,608 | 141,095 | 67,513 | - SG&A expenses increased by €33.2 million for the three months and €67.5 million for the six months ended June 30, 2025[250] - The increase was primarily due to commercial expansion, global launch activities for YORVIPATH, and a €3.5 million impairment charge on property, plant and equipment[250] Finance Income and Finance Expenses Net finance income significantly improved for the six months ended June 30, 2025, driven by currency gains and reduced finance expenses Net Finance Income/(Expenses) (EUR'000) | Category | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Change | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change | | :--- | :--- | :--- | :--- | :--- | :--- | :--- | | Finance income | 55,059 | 49,052 | 6,007 | 83,912 | 14,395 | 69,517 | | Finance expenses | (33,018) | (19,624) | (13,394) | (77,803) | (58,553) | (19,250) | | Total net finance income/(expenses) | 22,041 | 29,428 | (7,387) | 6,109 | (44,158) | 50,267 | | Net currency gain/(loss) | 48,759 | (4,791) | 53,550 | 73,404 | (13,411) | 86,815 | - Total net finance income/(expenses) improved by €50.3 million for the six months ended June 30, 2025, shifting from a net expense to a net income[251] - This improvement was primarily driven by €86.8 million in net currency gains, mainly from U.S. dollar denominated positions, partly offset by higher remeasurement loss of financial liabilities and increased amortization charges[251] Liquidity and Capital Resources Liquidity is primarily from cash and cash equivalents (€494.0 million), deemed sufficient for twelve months, funded by equity, debt, and royalty agreements - Cash and cash equivalents amounted to €494.0 million as of June 30, 2025[253] - Existing capital resources are believed to be sufficient to meet projected cash requirements for at least twelve months from the report date[256] - Operations are funded through preference/ordinary shares, convertible debt, and royalty funding agreements, with total net proceeds of $2,580.2 million from public offerings since February 2015[257] Cash Flows Summary (EUR'000) | Category | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change | | :--- | :--- | :--- | :--- | | Operating activities | (21,654) | (162,890) | 141,236 | | Investing activities | (5,041) | 7,763 | (12,804) | | Financing activities | (11,043) | 15,968 | (27,011) | | Net increase/(decrease) in cash and cash equivalents | (37,738) | (139,159) | 101,421 | - Cash flows used in operating activities improved by €141.2 million, driven by commercial revenue growth and working capital improvements, including a $100 million upfront payment from Novo Nordisk[265] Off-Balance Sheet Arrangements The company has not entered into any off-balance sheet arrangements or holdings in variable interest entities - The company has no off-balance sheet arrangements or holdings in variable interest entities[268] Qualitative Disclosures about Market Risk The company is exposed to foreign currency, interest rate, credit, and equity risks, managed through cash holdings, investment policies, and fair value measurements - The company is exposed to foreign currency exchange rates (primarily USD), interest rates, credit risk, and equity risk[269][270] - Foreign currency risk is minimized by maintaining cash positions in currencies of expected future expenses[270] - Credit risk on bank deposits is limited by holding deposits with high credit-rated banks and an investment policy for marketable securities[273][275] - Derivative liabilities are measured at fair value through profit or loss, exposing the company to volatility from interest rate and share price changes[272][277][278] Foreign Currency Risk Foreign exchange risk, primarily from USD-denominated items, is managed by maintaining cash positions in relevant currencies - Exposed to foreign exchange risk, primarily with the U.S. Dollar, affecting revenue, operating expenses, convertible notes, and royalty funding liabilities[270] - Risk is minimized by maintaining cash positions in currencies where the majority of future expenses are expected[270] Interest Rate Risk Fixed-rate convertible notes and lease liabilities exist, but future indebtedness, interest income, and derivative liabilities are exposed to interest rate volatility - Convertible notes have a fixed 2.25% coupon rate, and lease liabilities have fixed interest rates[271] - Future indebtedness and interest income from bank deposits are exposed to changes in interest rates[271] - Derivative liabilities, measured at fair value through profit or loss, are exposed to volatility from interest rate development[272] Credit Risk Credit risk, primarily on cash and equivalents, is managed through an investment policy with minimum ratings for financial institutions - Credit risk primarily relates to cash and cash equivalents, managed by an investment policy with minimum ratings for financial institutions[273] - Deposits with major financial institutions may exceed insured limits, but counterparties are considered creditworthy[274] - No material impairment loss from expected credit loss has been recognized[276] Equity Risk Equity risk arises from share price changes impacting the fair value remeasurement of derivative liabilities, valued using the Black-Scholes model - Exposed to equity risk from changes in its share price, affecting the remeasurement of derivative liabilities at fair value[277][278] - Derivative liabilities are valued using the Black-Scholes option pricing model, which incorporates the company's share price as a key assumption[278]