Ascendis Pharma(ASND) - 2025 Q1 - Quarterly Report

Financial Performance - Revenue for the three months ended March 31, 2025, was €100,954,000, an increase of 5.4% compared to €95,894,000 for the same period in 2024[15] - Gross profit decreased to €83,437,000 from €88,325,000, reflecting a decline of 5.1% year-over-year[15] - Research and development expenses rose significantly to €86,603,000, up 22.5% from €70,687,000 in the prior year[15] - Selling, general, and administrative expenses increased to €101,046,000, a 51.4% rise compared to €66,783,000 in the same quarter of 2024[15] - The net loss for the period was €94,626,000, compared to a net loss of €131,035,000 in the same period last year, representing a 27.8% improvement[15] - The company reported a basic and diluted loss per share of €(1.58) for the period, improving from €(2.30) in the previous year[15] - Revenue from commercial products reached €96.028 million, up 44.5% from €66.499 million in Q1 2024, driven by significant sales of YORVIPATH® and SKYTROFA®[45] - The net loss for the period was €94.626 million, an improvement from a net loss of €131.035 million in Q1 2024[19] - Cash and cash equivalents at March 31, 2025, totaled €517.923 million, compared to €320.239 million at the same date in 2024, reflecting an increase of 62%[19] Assets and Liabilities - Total assets decreased to €1,061,438,000 as of March 31, 2025, down from €1,179,495,000 at the end of 2024[16] - Total equity as of March 31, 2025, was €(189,807,000), compared to €(105,706,000) at the end of 2024, indicating a decline in equity[16] - Cash and cash equivalents decreased to €517,923,000 from €559,543,000, reflecting a reduction of 7.4%[16] - Total financial liabilities increased to €1,110.1 million as of March 31, 2025, compared to €1,104.0 million at the end of 2024, marking a rise of 0.1%[61] - Total financial liabilities as of March 31, 2025, amount to €935,512,000, with contractual cash flows totaling €1,227,447,000, indicating a significant portion of liabilities maturing within 1-5 years[78] Shareholder Information - The company had 60,018,550 shares used for the calculation of basic and diluted earnings per share, an increase from 56,883,257 shares in the prior year[15] - The company has authorized a share repurchase program of up to $18.25 million, which was fully executed by March 4, 2025[39] - The exercise price of outstanding warrants ranged from €11.98 to €145.50, with a total of 6,017,157 warrants outstanding as of March 31, 2025[58] Product Development and Pipeline - The company aims to achieve blockbuster status (>$1B) for TransCon PTH, TransCon hGH, and TransCon CNP through worldwide commercialization[95] - The current pipeline includes two marketed products and four product candidates in clinical development, focusing on Endocrinology Rare Disease and Oncology[94] - The company plans to expand its pipeline with Endocrinology Rare Disease blockbuster product opportunities, targeting unmet medical needs[95] - The oncology pipeline includes TransCon IL-2 b/g, currently in Phase 2 trials for various tumor types, aiming to enhance anti-tumor effects and immune activation[98] - The company is focused on applying its TransCon technology platform to develop differentiated product candidates, aiming to address unmet medical needs in large markets[90] Clinical Trials and Regulatory Approvals - TransCon PTH (palopegteriparatide) received FDA approval in August 2024 for the treatment of hypoparathyroidism in adults, with seven years of market exclusivity in the U.S.[147] - The Phase 3 PaTHway Trial demonstrated a mean increase in estimated glomerular filtration rate (eGFR) of 8.9 mL/min/1.73m² at Week 52, sustained at Week 104 with a mean change of 9.0 mL/min/1.73m²[154] - The company has submitted a New Drug Application (NDA) for TransCon CNP for achondroplasia in children aged 2-11, and a supplemental Biologics License Application (sBLA) for TransCon hGH for adult GHD[98] - The ongoing reACHin Trial is evaluating TransCon CNP in infants aged 0 to <2 years, with an IND amendment filed in Q3 2023[183] Market and Competitive Landscape - The global market for daily hGH products is primarily dominated by Novo Nordisk, Pfizer, Eli Lilly, Sandoz, and Merck KGaA, which together account for most of the market share[120] - Pediatric indications comprise up to 90% of the total hGH market, with approximately half attributed to pediatric GHD[121] - Approximately 68% of commercial patients and 63% of Medicaid patients with growth hormone deficiency (GHD) received daily growth hormone treatment, while 32% of commercial and 37% of Medicaid patients remained untreated[118] Technology and Innovation - TransCon technologies aim to provide sustained drug release, potentially extending dosing frequency from daily to six months or more, improving treatment safety and efficacy[107] - Early clinical studies indicate sustained activation of cytotoxic immune cells, leading to robust anti-tumor responses with infrequent administration of TransCon product candidates[199] - The company believes that TransCon technologies may enhance the efficacy of existing therapies without increasing toxicity, potentially allowing for new combination regimens[197]