- Results for all three TransCon hGH starting dose cohorts, in first clinical trial of an indication outside of growth hormone deficiency, showed a safety and tolerability profile comparable to daily somatropin - Annualized height velocity was similar at Week 26 in once-weekly TransCon hGH-treated and daily somatropin-treated children COPENHAGEN, Denmark, Dec. 16, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (NASDAQ:ASND) today announced positive Week 26 topline results from New InsiGHTS, its Phase 2 rando ...

Core Viewpoint - Ascendis Pharma A/S has received FDA acceptance for the review of its supplemental Biologics License Application for TransCon hGH in adult growth hormone deficiency, with a PDUFA goal date set for July 27, 2025 [1][2]. Company Overview - Ascendis Pharma is focused on developing innovative therapies using its TransCon technology platform, aiming to make a significant impact on patients' lives [4]. Product Development - The sBLA submission is based on the Phase 3 foresiGHt trial, which demonstrated the efficacy and safety of TransCon hGH compared to placebo and daily human growth hormone in adults with GHD [2]. - The trial involved 259 adults aged 23 to 80, showing that TransCon hGH led to a significant reduction in trunk fat and an increase in total body lean mass at Week 38 compared to placebo [2]. Market Need - Adult growth hormone deficiency is significantly undertreated, with only 5-10% of patients receiving treatment, indicating a high unmet medical need [2][3]. - Symptoms of adult GHD include central obesity, metabolic syndrome, decreased bone density, and psychological issues, highlighting the importance of effective treatment options [3].

Financial Data and Key Metrics Changes - Product revenues have significantly increased, with SKYTROFA volume rising over 60% year-over-year in Q3 2024, although reported revenue was EUR 47.2 million compared to EUR 47 million in Q3 2023 due to higher sales deductions [32][34] - Total revenue for Q3 was EUR 57.8 million, including EUR 2.1 million from services and license revenue [37] - Cash, cash equivalents, and marketable securities totaled EUR 626 million at the end of Q3 2024, up from EUR 399 million at the end of 2023 [41] Business Line Data and Key Metrics Changes - SKYTROFA's revenue for the first nine months of 2024 totaled EUR 138.5 million, a 21% increase year-over-year [34] - YORVIPATH revenue outside the US increased over 60% to EUR 8.5 million in Q3, driven by growing demand [36] - The company expects full-year 2024 SKYTROFA revenue to be between EUR 200 million and EUR 220 million, excluding prior year sales deductions [42] Market Data and Key Metrics Changes - SKYTROFA penetration in the overall treated growth hormone market was approximately 6% by the end of Q3 2024, with daily growth hormone products representing about 85% of total US prescriptions [35] - The market for YORVIPATH is estimated to have around 70,000 to 90,000 adults with hypoparathyroidism in the US [10] Company Strategy and Development Direction - The company aims to expand SKYTROFA's label to include adult growth hormone deficiency and to successfully launch YORVIPATH in the US and Europe [31] - A new partnership with Novo Nordisk is expected to enhance the TransCon technology platform, targeting obesity and type 2 diabetes [24][25] - The company is focused on achieving blockbuster status for its endocrinology programs and expanding its market share [28] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in maintaining patient retention for YORVIPATH due to its unique therapeutic profile and alignment with treatment guidelines [44][60] - The goal of achieving operating cash flow breakeven by the end of 2025 remains intact, supported by current cash reserves [48] - Management highlighted the strong position of SKYTROFA in a consolidating market, with expectations for continued growth [15][56] Other Important Information - The company has a robust supply chain for YORVIPATH, ensuring no patient will be unable to receive their prescription [111] - The company is preparing for the imminent launch of YORVIPATH in the US, with product availability expected in mid-January 2025 [16][42] Q&A Session Summary Question: Confidence in retaining patients for YORVIPATH amid competition - Management emphasized the unique replacement therapy characteristics of YORVIPATH, which are unmatched by competitors [44][45] Question: Expectation to reach operating cash flow breakeven by end of 2025 - Management confirmed that this remains the goal, supported by current cash and equivalents [48] Question: Update on CNP preparation for FDA meeting - Management stated that all necessary data for the submission is ready, with no missing information [51][52] Question: Progress with payers for SKYTROFA approval - Management indicated improvements in market access and a strong positioning strategy for SKYTROFA [55][56] Question: Timing for NATPARA inventory depletion affecting YORVIPATH - Management noted that the timing is outside their control but expects a transition to occur next year [65][66] Question: Uptake of PTH experienced patients onto YORVIPATH - Management believes that many patients will seek treatment quickly once YORVIPATH is available [71][72] Question: Revenue contribution from adult growth hormone market - Management highlighted the potential for significant growth in the adult market, which is currently underpenetrated [75][76] Question: Comparison of YORVIPATH launch in the US versus Europe - Management expects a faster uptake in the US due to a more unified market mindset [81][82] Question: Adjustments to 2024 revenue guidance for SKYTROFA - Management explained that lower volume and fewer selling days contributed to the adjustments [84] Question: Next milestones for oncology pipeline - Management expressed encouragement from recent results and plans to focus on creating value for patients [92]

– YORVIPATH launching in U.S. with product availability expected mid-January 2025; ex-U.S. YORVIPATH revenue of €8.5 million in Q3 – TransCon CNP NDA submission for achondroplasia planned for Q1 2025 followed by MAA submission planned for Q3 2025 – SKYTROFA Q3 revenue of €47.2 million – 60%+ year-over-year volume growth offset by current and prior period sales deductions – Full year 2024 SKYTROFA revenue excluding sales deductions related to prior years expected to be €200 - €220 million, and full year 2024 ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 6-K REPORT OF FOREIGN PRIVATE ISSUER PURSUANT TO SECTION 13a-16 OR 15d-16 UNDER THE SECURITIES EXCHANGE ACT OF 1934 For the month of September, 2024 Commission File Number: 001-36815 Ascendis Pharma A/S (Exact Name of Registrant as Specified in Its Charter) Tuborg Boulevard 12 DK-2900 Hellerup Denmark (Address of principal executive offices) Indicate by check mark whether the registrant files or will file annual reports under cover ...

COPENHAGEN, Denmark, Sept. 30, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it has submitted a supplemental Biologics License Application (sBLA) to the U.S. Food & Drug Administration (FDA) for TransCon hGH (marketed as SKYTROFA® (lonapegsomatropin-tcgd) in the U.S. for pediatric growth hormone deficiency) for the treatment of adults with growth hormone deficiency (GHD). “The sBLA submission for TransCon hGH for adult GHD supports our strategy to develop SKYTROFA into a blockb ...

COPENHAGEN, Denmark, Sept. 20, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced the pricing of its underwritten public offering of 2,000,000 American Depositary Shares (“ADSs”), each of which represents one ordinary share of Ascendis, at a price to the public of $150.00 per ADS. All of the ADSs are being offered by Ascendis. The offering is expected to close on or about September 23, 2024, subject to the satisfaction of customary closing conditions. In addition, Ascendis has grant ...

COPENHAGEN, Denmark, Sept. 18, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that it has commenced an underwritten public offering of $300,000,000 of American Depositary Shares (“ADSs”), each of which represents one ordinary share of Ascendis. All of the ADSs are being offered by Ascendis. In addition, Ascendis expects to grant the underwriters a 30-day option to purchase up to an additional $45,000,000 of ADSs at the public offering price, less the underwriting commissions. Th ...

Shares of Ascendis Pharma (ASND) rose 17% on Monday after it reported top-line results from the pivotal ApproaCH study that evaluated investigational once-weekly administered TransCon CNP in children aged two to 11 years with achondroplasia, the most common form of dwarfism.ASND Drug Shows AGV Benefit in Achondroplasia PatientsThe ApproaCH study met its primary endpoint of annualized growth velocity (AGV) at 52 weeks — children treated with TransCon CNP achieved a mean AGV of 5.89 cm/year compared with 4.41 ...

Ascendis Pharma A/S (ASND) shares ended the last trading session 17.1% higher at $139.57. The jump came on an impressive volume with a higher-than-average number of shares changing hands in the session. This compares to the stock's 14.1% loss over the past four weeks.This surge in share price came after ASND reported topline results from the pivotal ApproaCH study which evaluated its investigational once-weekly administered TransCon CNP in children aged two to 11 years with achondroplasia, the most common f ...