This is an observational, opportunistic lactation study to be conducted in lactating female participants who are currently receiving therapeutic doses of YORVIPATH as part of their usual care and who have chosen to breastfeed their infant(s). The potential transfer of palopegteriparatide into breast milk will be assessed.

开始日期2026-01-01

申办/合作机构

Ascendis Pharma A/S

NCT07345494

/ RecruitingN/A

A Global Pregnancy Registry to Assess Maternal, Fetal, and Infant Outcomes Following Exposure to YORVIPATH® (Palopegteriparatide) During Pregnancy and Breastfeeding

The purpose of this registry study is to collect both prospective and retrospective data in women exposed to palopegteriparatide during pregnancy to assess risk of pregnancy and maternal complications, and adverse effects on the developing fetus, neonate, and infant and to assess infant outcomes through at least the first year of life.

开始日期2026-02-18

申办/合作机构

Ascendis Pharma A/S

NCT07081997

/ Not yet recruiting临床3期

A Phase 3, Multicenter, Randomized, Open-Label Trial Investigating the Safety, Tolerability and Efficacy of Palopegteriparatide Administered Subcutaneously Daily at Doses Greater Than 30 μg/Day in Adult Participants With Hypoparathyroidism

This trial has a duration of 78 weeks and will include adult participants already on treatment with palopegteriparatide at doses at or greater than 30 mcg/day. All participants will receive subcutaneous palopegteriparatide during the trial and will be individually and progressively titrated to an optimal dose at pre-specified dose levels. The primary purpose of the trial is to provide additional evidence of treatment effect and safety of palopegteriparatide at doses greater than 30 mcg/day in adults with hypoparathyroidism. The trial will be conducted in the US.

开始日期2026-04-01

申办/合作机构

Ascendis Pharma Bone Diseases A/S

100 项与帕罗培特立帕肽相关的临床结果

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100 项与帕罗培特立帕肽相关的转化医学

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100 项与帕罗培特立帕肽相关的专利（医药）

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6,616

项与帕罗培特立帕肽相关的文献（医药）

2026-05-01·Journal of Orthopaedics

Teriparatide enhances vertebral bone quality: Quantitative analysis using the VBQ score in lumbar fusion patients

Article

作者: Sako, Noriaki ; Miyazaki, Masashi ; Abe, Tetsutaro ; Kaku, Nobuhiro

Background:

Vertebral bone quality is a critical factor influencing spinal stability and surgical planning. The vertebral bone quality (VBQ) score, derived from routine MRI, has emerged as a noninvasive imaging biomarker of bone quality; however, its clinical implications in the perioperative spinal fusion setting remain incompletely understood.

Methods:

This retrospective study included patients who underwent single-level lumbar fusion at L4-5. VBQ was measured at T12-L2 to avoid instrumentation-related artifacts. Associations between VBQ and radiographic parameters were evaluated using correlation and multivariable regression analyses. Inter- and intra-rater reliability were assessed using kappa statistics.

Results:

VBQ scores significantly decreased in the PTH group (ΔVBQ -0.31 ± 0.56), indicating improved bone quality, whereas no improvement was observed in controls (ΔVBQ 0.08 ± 0.61; p = 0.03). HU changes did not differ between groups. ΔVBQ correlated with postoperative BMD (r = -0.28, p = 0.04) and PTH duration (r = -0.39, p = 0.01). Regression analysis identified PTH use (β = -0.39, 95%CI: -0.72 to -0.06, p = 0.02) and treatment duration (β = -0.27, 95%CI: -0.52 to -0.02, p = 0.03) as independent predictors of VBQ improvement with R² of 0.32.

Conclusion:

Perioperative teriparatide significantly enhances vertebral bone quality, demonstrated by reduced VBQ scores over 12 months. VBQ appears more sensitive than HU or BMD to anabolic therapy-induced qualitative changes and may serve as a practical imaging biomarker for optimizing perioperative bone management in osteoporotic spinal fusion patients.

2026-04-01·VETERINARY JOURNAL

Markers of bone turnover, parathyroid hormone, calcium, and magnesium concentrations in horses with acute colitis

Article

作者: Gomez, Diego E ; Toribio, Ramiro E ; Fortin-Trahan, Rosalie ; Arroyo, Luis G ; Hostnik, Laura D ; Kamr, Ahmed

Hypocalcemia is frequent in horses with colitis. Information on serum parathyroid hormone (PTH) concentrations and its association with bone turnover biomarkers in horses with colitis is lacking. We aimed to determine the association between serum bone resorption biomarkers (C-terminal telopeptide of type I collagen [CTX-I]) and bone formation (osteocalcin [OCN]) with blood PTH, total calcium (tCa), ionized calcium (iCa), phosphorus (Pi), and total magnesium (tMg) concentrations, and mortality in horses with acute colitis. A total of 163 horses were divided into colitis (n = 127) and healthy (n = 36) groups. Blood samples were collected from all horses. Non-parametric methods were used for data analysis. In horses with colitis, serum CTX-I and PTH concentrations were significantly higher, whereas OCN, tCa, iCa, Pi, and tMg concentrations were lower compared to healthy horses (P < 0.05). In colitis horses with ionized hypocalcemia, serum CTX-I concentrations were positively correlated with PTH concentrations (P < 0.05). Colitis horses with iCa concentrations < 1.4 mmol/L and PTH > 92.9 pg/mL were more likely to die ([OR = 6.1; 95 % CI = 1.2-24.5; P < 0.05]; [OR = 3.6; 95 % CI = 1.3-10.1; P < 0.05]), respectively. In colitis horses, elevated PTH and CTX-I concentrations, together with decreased OCN concentrations, suggests that bone turnover activity was increased to offset hypocalcemia. Elevated PTH and hypocalcemia were associated with non-survival, indicating that in non-surviving horses, bone resorption activity was not sufficient to restore normocalcemia. PTH resistance could have contributed to hypocalcemia in some horses with acute colitis.

2026-04-01·BONE

Improvements in bone quality by parathyroid hormone treatment are enhanced in the Nmp4 knockout mouse model

Article

作者: Bidwell, Joseph P ; Vashishth, Deepak ; Chen, Runkang ; Wang, Bowen ; Stephen, Samuel J

Osteoporosis is linked to increased bone fragility. Unlike anti-resorptive therapies, the analogue of parathyroid hormone, PTH 1-34, is an FDA-approved therapeutic for osteoporosis that enhances bone formation. However, as PTH treatment potency declines over time, it is necessary to investigate the mechanisms involved in this attenuation to reinforce its long-term efficacy. This need has led to investigations into the transcription factor nuclear matrix protein 4 (Nmp4), in which PTH treatment of mice globally lacking Nmp4 (Nmp4^-/-) enhanced bone formation. Yet, the changes in the compositional quality of PTH-stimulated bone in Nmp4^-/- mice are unknown, which in turn could impact the efficiency of this approach. To this end, we characterized cortical bone quality in Nmp4^-/- mice and wild-type littermates treated with PTH for 8 weeks, starting at 16 weeks of age, using micro-computed tomography, Raman spectroscopy, X-ray diffraction, biochemical assays, and biomechanical characterization (whole-bone strength, fracture toughness). PTH treatment and Nmp4 ablation increased tissue and marrow area and maximum moment of inertia. Femora from PTH-treated mice exhibited increased stiffness, maximum load, and fracture resistance. Bone in Nmp4^-/- mice with PTH treatment demonstrated lower mineral crystallinity, decreased mineral-to-matrix ratio, lattice spacing, altered levels of advanced glycation end-products, increased levels of osteocalcin, and increased matrix phosphorylation levels. These results suggest that ablation of Nmp4, in concert with PTH treatment, improved bone function by modulating bone structure and matrix composition. Our findings demonstrate the potential utility of targeting Nmp4 to improve PTH potency and bone quality.

231

项与帕罗培特立帕肽相关的新闻（医药）

2026-03-02

·FiercePharma

Ascendis gains more altitude with FDA approval for dwarfism drug Yuviwel

Ascendis Pharma of Denmark has gained its third FDA new drug approval in the last six years as the U.S. regulator has signed off on Yuviwel as a treatment for achondroplasia. Ascendis Pharma is spiraling upward by way of its TransCon platform. For the third time in six years, the Copenhagen-based biopharma has scored an FDA approval for a rare disease medicine that was developed with its “transient conjugation” drug delivery technology. The U.S. regulator has signed off on Yuviwel, a once-weekly injection to treat children with achondroplasia, a rare genetic disorder that causes dwarfism. The accelerated approval covers children age 2 and older who have open growth plates, which are areas where cartilage develops at the end of bones, allowing them to lengthen.By providing continuous exposure to C-type natriuretic peptide (CNP) receptors, Yuviwel can counter the effects of the fibroblast growth factor receptor 3 (FGFR3) gene, which causes the protein to become overactive and restrict growth. This continuous systemic exposure to CNP over the weekly dosing interval sets Yuviwel apart in the achondroplasia treatment landscape, according to Ascendis.“The approval of once-weekly Yuviwel is a major step forward in the treatment of children with achondroplasia, giving physicians for the first time the option of prescribing a once-weekly medicine backed by compelling efficacy and excellent tolerability data from three randomized, double-blind, placebo-controlled clinical trials,” Carlos Bacino, M.D., of Baylor University and Texas Children’s Hospital, said in a release.The three trials and up to three years of open-label extension data demonstrated the ability of Yuviwel to provide improvements in annualized growth velocity versus placebo.Achondroplasia is the most common form of short-limbed dwarfism, affecting between 1 in 10,000 and 1 in 30,000 live births and preventing adults from growing taller than four feet,10 inches. The condition can increase the likelihood of spinal cord compression and respiratory blockages early in life along with other muscular and neurological problems.The company said it expects to make Yuviwel available early in the second quarter of this year. In a conference call Monday morning, Ascendis said it will reveal the price in the coming days.The nod comes with a valuable pediatric disease priority review voucher, which can be used to speed the review of a future drug application."Most of the products we're developing are getting priority review, so I'm not sure that we actually need to keep it. I think we'll sell it," Scott Smith, Ascendis' chief financial officer, said in a lighter moment during the conference call.Yuviwel’s approval comes after the FDA delayed its decision on the medicine by three months back in November. Ascendis will compete with BioMarin in the indication. The California company gained approval for its CNP treatment Voxzogo in 2021. The daily injected medicine generated sales of $927 million last year, which were up 26% and accounted for 29% of the company’s revenue in 2025.BioMarin is working on a longer-acting version of Voxzogo, BMN 333, which has shown promise in a phase 1 trial and could be launched by 2030, the company has said. BMN 333 has shown area-under-the-curve (AUC) levels greater than three times those observed in other long-acting CNP studies, BioMarin said in August of last year, referring to Ascendis’ trials for Yuviwel. AUC is a pharmacokinetic measure indicating the total exposure to a drug over time.Ascendis’ current advantage with Yuviwel is its consistent, sustained release over its weekly dosing as opposed to Voxzogo’s daily administration.Last month, BridgeBio revealed the success of a phase 3 trial of its FGFR1-3 selective tyrosine kinase inhibitor infigratinib in children with achondroplasia. BridgeBio and its partner Kyowa Kirin plan to apply for approval of infigratinib in the second half of this year.Ascendis used its TransCon technology to develop Skytrofa, a long-acting growth hormone for children which was approved in the U.S. in 2021, and Yorvipath, a hormone replacement therapy for hypoparathyroidism, which scored an FDA nod in 2024. Sales for Skytrofa reached 206 million euros ($242 million) last year, which were up 5% year over year. Yorvipath generated sales of 477 million euros ($561 million) last year, with 187 million euros ($220 million) coming in the fourth quarter."We expects Yuviwel revenue to be light this year but don't think that reflects on long-term value," analysts from ISI Evercore wrote in a note to investors. "We continue to believe our $1 billion CNP franchise peak is quite conservative."Evercore sees more sales potential for Yuviwel in Europe than in the U.S., citing sales results for Voxzogo. There are also other potential indications for the treatment. The company also is exploring the potential of Yuviwel in combination with other treatments.

上市批准临床3期临床结果临床1期优先审批

2026-02-12

·BioPharmaDive

Earnings roundup: Neurocrine slumps, Alnylam battles skeptics and Ascendis eyes a competitor

With quarterly earnings underway, BioPharma Dive is providing a snapshot of some companies results and how theyre being received by investors. Today, were offering insight into the latest numbers from Neurocrine Biosciences, Alnylam Pharmaceuticals and Ascendis Pharma. Neurocrine again disappoints some investorsShares of Neurocrine Biosciences fell by over 10% on Thursday, wiping more than $1 billion in value from the brain-focused biotechnology company.The drop followed a Wednesday afternoon earnings report in which Neurocrine said net product sales in the fourth quarter and 2025 overall totaled $798 million and $2.83 billion. Those figures are respectively 29% and 22% higher year-over-year. Ingrezza, a drug for movement issues associated with Huntington's disease, accounted for more than 80% of overall sales. The remainder mostly came from Crenessity, a treatment approved in 2024 for a rare, genetic condition that can cause life-threatening shortages of crucial hormones.Brian Abrahams, an analyst at RBC Capital Markets, suspects a combination of factors may be concerning some investors. One is Neurocrine's guidance. The company expects that, over the course of this year, net sales from Ingrezza will be in the range of $2.7 billion to $2.8 billion. That might not be as high as shareholders had hoped. Neurocrine executives also recently disclosed plans to spend another $150 million building out the sales teams for those two leading products a decision that could be seen as bad for near-term margins.Additionally, Abrahams noted how some are worried about Crenessitys growth prospects, specifically with regard to the number of new patients starting on the drug. Neurocrine says there are roughly 20,000 people in the U.S. with that rare condition, congenital adrenal hyperplasia, and, across last year, about 2,050 began taking Crenessity. Sales figures indicate the final three months of 2025 averaged fewer starts, at 431, than previous quarters.Even so, Abrahams and his team see the fears as overblown. Though we acknowledge investor consternation around margins and Crenessity launch dynamics could persist ... we believe todays downside reaction will prove to be a compelling buying opportunity, he wrote in a note to clients.Ingrezza looks poised for a major beat, he added, and Crenessity should be fine [as] we have always anticipated some continued slowdown in new patient starts.Overall, we see much growth left in both key franchises, wrote TD Cowen analyst Phil Nadeau in his own client note.Alnylam bitten by its successA similar situation played out with Alnylam Pharmaceuticals. The company, which specializes in so-called RNA interference drugs, gave a preliminary look at fourth-quarter and full-year earnings last month, then released its official report Thursday. It recorded nearly $3 billion across all of 2025 and almost $1 billion over those final three months, reflecting increases of 81% and 121% compared to the same periods the prior year.Despite that performance, Alnylam shares had slipped around 4% by the time trading closed Thursday afternoon.Though the company sells half a dozen drugs, nearly 80% of its product revenue comes from Amvuttra, a treatment for the nerve damage and heart failure associated with a rare disorder known as ATTR. Alnylam has said this disorder, which has a couple subtypes, affects north of a few hundred thousand people worldwide. For 2026, the company expects net product revenue to reach $4.9 billion to $5.3 billion, with Amvuttra and another ATTR medicine, Onpattro, responsible for up to $4.7 billion of that sum.To Paul Matteis, an analyst at Stifel, the main debate among investors is whether Amvuttra can meet or beat this guidance, and whether Alnylam can keep pace finding new patients to get on the drug.Alnylam is somewhat a victim of its own success after two very strong quarters for Amvuttra, Matteis wrote. Yet, while some investors may have wanted even more from the latest earnings, the underlying fundamentals here still seem strong.To that end, Alnylam said Amvuttras approval last spring for those cardiovascular problems tied to ATTR was a primary reason why patient demand for the drug grew roughly 250% in the fourth quarter. The company estimates that, globally, around 80% of patients with this condition, which stiffens the heart and impairs its function, remain undiagnosed.The number of identified patients has, on average, grown 40% annually since 2019, according to Alnylam.There are simply many tailwinds for the mid-to-long term, even if short-term expectations had gotten ahead of themselves, Matteis wrote.Ascendis numbers overshadowed by competitorDenmark-based Ascendis Pharma also provided an early glance at earnings in January. Its full report, out Wednesday, detailed fourth-quarter product revenue of 240 million euros, or about $279 million. Full-year product revenue hit 684 million euros, with 70% coming from Yorvipath, a drug for a rare illness where the body doesnt produce enough of a hormone that regulates vital minerals.The results were in-line with analyst forecasts. Joseph Schwartz, of Leerink Partners, expects Yorvipaths momentum to continue throughout this year, and described Ascendis goal of the drug surpassing $1 billion in revenue as achievable.The companys share price, however, slumped 5% at markets open Thursday. Though the stock would rebound over the following hours, Ascendis earnings appeared to take a back seat to data from a rival.Early that morning, BridgeBio Pharma disclosed positive findings from a late-stage study evaluating one of its most advanced drugs an oral enzyme-blocker named infigratinib as a growth-inducing treatment for achondroplasia, the most common type of dwarfism. Ascendis has been testing a once-weekly injectable therapy in this setting, and could receive approval from the Food and Drug Administration before the end of February.With few surprises in Ascendis results, the update from BridgeBio served as bigger news and reinforced that this oral option creates competition, wrote Li Watsek of Cantor Fitzgerald.Yaron Werber, a TD Cowen analyst who covers Ascendis, acknowledged that BridgeBios drug appears solid, with clean safety. And, if approved, it will likely dominate the achondroplasia market as both an initial and second-line treatment.In Werbers view, the key to Ascendis being able to compete is securing strong data from a Phase 3 study of its TransCon CNP therapy combined with its already approved product for growth hormone deficiency. In earlier testing, Ascendis said this pairing, when used in certain people with achondroplasia, tripled the effectiveness seen when TransCon CNP was used alone.The combination should eventually become the standard-of-care in achondroplasia, Werber wrote. '

临床结果临床3期财报上市批准

2026-02-12

·BioSpace

Ascendis Pharma Reports Fourth Quarter and Full-Year 2025 Financial Results

Q4 2025 product revenue of €240 million and FY 2025 product revenue of €684 million Q4 2025 operating profit of €10 million and cash flow from operating activities of €73 million TransCon ® CNP under FDA Priority Review, PDUFA action goal date of February 28, 2026 Conference call today at 4:30 pm ET COPENHAGEN, Denmark, Feb. 11, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced financial results for the fourth quarter and full year ended December 31, 2025, and provided a business update. “With a continued focus on making a meaningful difference for patients, we believe Ascendis is entering a steep growth phase as we transform into a leading global biopharma company,” said Jan Mikkelsen, President and CEO of Ascendis Pharma. “With strong execution and the power of our TransCon platform, we are positioned to generate approximately €500 million in operating cash flow in 2026 while aspiring to deliver at least €5 billion in global annual product revenue by 2030. At the same time, we are advancing a growing pipeline of highly differentiated programs to deliver durable, long-term growth.” Select 2025 Highlights & Anticipated 2026 Milestones TransCon PTH (palopegteriparatide, marketed as YORVIPATH) YORVIPATH revenue for the fourth quarter of 2025 totaled €187 million and €477 million for the full year 2025, as previously announced. More than 5,300 unique U.S. patient enrollments, with nearly 2,400 unique prescribing healthcare providers at year end. Outside the U.S., now available commercially or through named patient programs in more than 30 countries, with full commercial launches anticipated in 10 additional countries by year end 2026. Presented pooled analysis of 3-year data from PaTHway and PaTH Forward trials at the American Society of Nephrology (ASN) Kidney Week 2025, reinforcing that treatment with TransCon PTH led to rapid and sustained improvements in kidney function in adults with hypoparathyroidism. Ongoing label expansion trials through PaTHway60 (adults) and PaTHway Adolescent. Confirmed product pro once-weekly TransCon PTH, targeting patients who have been transitioned from conventional therapy to YORVIPATH and have achieved a stable daily dose. TransCon CNP (navepegritide, FDA NDA and EMA MAA filed) In the U.S., PDUFA action goal date of February 28, 2026 for pediatric achondroplasia. Submitted marketing authorization application (MAA) to the European Medicines Agency (EMA) in October 2025, with a regulatory decision on potential use in pediatric achondroplasia anticipated in the fourth quarter of 2026. Label expansion trial in infants with achondroplasia, reACHin, remains ongoing with enrollment completion anticipated later this year. TransCon CNP + TransCon hGH Combination Therapy ( navepegritide plus lonapegsomatropin ) Announced Week 52 topline results from Phase 2 COACH Trial, which demonstrated improvements in annualized growth velocity across both TransCon CNP treatment-naïve and TransCon CNP-treated children that exceeded the 97th percentile of average stature children, along with improvements in body proportionality and in arm span, and a safety pro with those observed for monotherapies of TransCon CNP and TransCon hGH on January 8, 2026. Held a successful end of Phase 2 meeting with the U.S. Food & Drug Administration (FDA) and Scientific Advice meeting in the EU regarding a Phase 3 trial of TransCon CNP and TransCon hGH in pediatric achondroplasia in the fourth quarter of 2025. Week 78 COACH data update anticipated in second quarter of 2026. Planned new trials to support TransCon CNP + TransCon hGH treatment in additional indications. TransCon hGH (lonapegsomatropin, marketed as SKYTROFA) SKYTROFA revenue for the fourth quarter of 2025 totaled €53 million and €206 million for the full year 2025, as previously announced. Received first label expansion in July 2025 with FDA approval for adult growth hormone deficiency (GHD). Initiated Phase 3 basket trial for additional indications: idiopathic short stature (ISS), SHOX deficiency, Turner syndrome, and small for gestational age (SGA). Oncology Program (onvapegleukin alfa) Expect to report median overall survival (OS) data for a cohort of 70 patients with late-line platinum-resistant ovarian cancer (PROC) from the IL-Believe Trial of TransCon IL-2 β/γ + weekly paclitaxel in the second quarter of this year. Strategic Collaborations & Investments Ongoing multi-product collaboration with Novo Nordisk for TransCon technology-based therapies in obesity and metabolic diseases, with the lead program, TransCon Semaglutide, on track to enter the clinic as anticipated. Eyconis lead program, TransCon aVEGF (EYC-0305), in development for wet AMD and other retinal diseases anticipated to enter the clinic in 2026. On January 26, 2026, VISEN Pharmaceuticals announced that China’s National Medical Products Administration approved its biologics license application for lonapegsomatropin (TransCon hGH) in China for the treatment of pediatric growth hormone deficiency (PGHD). In November 2025, Teijin Limited announced that YORVIPATH is commercially available for prescription in Japan. Financial Update For the fourth quarter of 2025, operating profit was €10 million and cash flow from operating activities was €73 million, primarily driven by continued growth of YORVIPATH revenue globally. Initiating previously announced $120 million share repurchase program in 2026. As of December 31, 2025, our cash balance was €616 million, an increase of €77 million compared to €539 million as of September 30, 2025. Fourth Quarter and Full-Year 2025 Financial Results Total revenue for the fourth quarter of 2025 was €248 million, compared to €174 million during the same period for 2024. The increase was primarily attributable to the continued growth of YORVIPATH global product revenue, partially offset by the recognition of a $100 million upfront payment in 2024 that did not recur in 2025. Total revenue for 2025 was €720 million compared to €364 million in 2024. The increase was primarily attributable to the continued growth of YORVIPATH partially offset by recognition of a $100 million upfront payment in 2024 that did not recur in 2025. Non-product revenue was €37 million in 2025, compared to €138 million in 2024. Total Revenue (In EUR'000s) Three Months Ended December 31, Twelve Months Ended December 31, 2025 2024 2025 2024 Revenue Commercial products 240,092 72,130 683,572 225,728 Services and clinical supply 4,780 5,933 18,008 15,570 Licenses 2,628 95,853 5,630 122,343 Milestones — — 12,922 — Total revenue 247,500 173,916 720,132 363,641 Commercial Products Revenue (In EUR'000s) Three Months Ended December 31, Twelve Months Ended December 31, 2025 2024 2025 2024 Revenue from commercial products YORVIPATH® 186,677 13,584 477,412 28,727 SKYTROFA® 53,415 58,546 206,160 197,001 Total revenue from commercial products 240,092 72,130 683,572 225,728 Research and development (R&D) expenses for the fourth quarter of 2025 were €78 million, compared to €79 million during the same period in 2024. R&D expenses for 2025 were €304 million compared to €307 million in 2024. The lower R&D expenses were driven by the completion of clinical trials and development activities within our Endocrinology Rare Disease pipeline partially offset by reversal (income) of prior period write-downs related to YORVIPATH pre-launch inventory. Selling, general, and administrative (SG&A) expenses for the fourth quarter of 2025 were €136 million, compared to €80 million during the same period in 2024. SG&A expenses for 2025 were €458 million compared to €291 million in 2024. Higher SG&A expenses were primarily due to the continued impact from global commercial expansion, including global launch activities for YORVIPATH. Total operating expenses for the fourth quarter of 2025 were €214 million compared to €160 million during the same period in 2024. Total operating expenses for 2025 were €761 million compared to €598 million in 2024. Net finance expenses were €38 million in the fourth quarter compared to €33 million in the same period in 2024. Net finance expenses for 2025 were €93 million compared to €74 million in 2024. The full year net finance expense increase was driven primarily by non-cash items. For the fourth quarter of 2025, Ascendis Pharma reported a net loss of €34 million, or €0.55 per share (basic and diluted) compared to a net loss of €38 million, or €0.64 per share (basic and diluted) for the same period in 2024. For the full year 2025, Ascendis Pharma reported a net loss of €228 million, or €3.76 per share (basic and diluted) compared to a net loss of €378 million, or €6.53 per share (basic and diluted) in 2024. As of December 31, 2025, Ascendis Pharma had cash and cash equivalents totaling €616 million compared to €560 million as of December 31, 2024. As of December 31, 2025, Ascendis Pharma had 61,977,408 ordinary shares outstanding, including 597,096 ordinary shares represented by ADSs held by the company. Conference Call and Webcast Information Ascendis Pharma will host a conference call and webcast today at 4:30 pm Eastern Time (ET) to discuss its fourth quarter and full year 2025 financial results. Those who would like to participate may access the live webcast here , or register in advance for the teleconference here . The link to the live webcast will also be available on the Investors & News section of the Ascendis Pharma website at . A replay of the webcast will be available on this section of the Ascendis Pharma website shortly after conclusion of the event for 30 days. About Ascendis Pharma A/S Ascendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of Patients, Science, and Passion, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more. Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) Ascendis’ expectation for durable, long-term growth, including its expectation to generate approximately €500 million in operating cash flow in 2026 and to deliver at least €5 billion in global annual product revenue by 2030; (ii) Ascendis’ expectations with respect to the commercial launches of TransCon PTH in additional countries; (iii) the PDUFA goal date for FDA review of TransCon CNP; (iv) the timing of EMA’s decision on potential use of TransCon CNP in pediatric achondroplasia; (v) the timing of enrollment completion of the label expansion trial in infants with achondroplasia, reACHin; (vi) the timing of Week 78 COACH data update; (vii) the planned new trials to support TransCon CNP + TransCon hGH treatment in additional indications; (viii) the timing of median OS data for the oncology program; (ix) the clinical entry of TransCon semaglutide; (x) the clinical entry of TransCon aVEGF program in 2026; (xi) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company; and (xii) Ascendis’ use of its TransCon technologies to create new and potentially best-in-class therapies. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; and the impact of international economic, political, legal, compliance, social and business factors. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 12, 2025, and Ascendis’ other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law. Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, TransCon, SKYTROFA ® , and YORVIPATH ® are trademarks owned by the Ascendis Pharma group. © February 2026 Ascendis Pharma A/S. Investor Contacts: Chad Fugere Ascendis Pharma ir@ascendispharma.com Media Contact: Melinda Baker Ascendis Pharma media@ascendispharma.com Patti Bank ICR Healthcare patti.bank@icrhealthcare.com FINANCIAL TABLES FOLLOW Ascendis Pharma A/S Consolidated Statements of Profit or (Loss) and Other Comprehensive Income or (Loss) (In EUR'000s, except share and per share data) Three Months Ended December 31, Twelve Months Ended December 31, 2025 2024 2025 2024 Consolidated Statement of Profit or (Loss) Revenue 247,500 173,916 720,132 363,641 Cost of sales (23,598 ) (14,023 ) (94,915 ) (44,258 ) Gross profit 223,902 159,893 625,217 319,383 Research and development expenses (78,151 ) (79,294 ) (303,621 ) (307,004 ) Selling, general, and administrative expenses (135,855 ) (80,216 ) (457,867 ) (291,142 ) Operating profit/(loss) 9,896 383 (136,271 ) (278,763 ) Share of profit/(loss) of associates 1,328 (4,575 ) 16,308 (20,060 ) Finance income 24,061 26,233 113,999 25,609 Finance expenses (61,990 ) (59,425 ) (206,687 ) (100,027 ) Profit/(loss) before tax (26,705 ) (37,384 ) (212,651 ) (373,241 ) Income taxes (expenses) (6,857 ) (1,085 ) (15,383 ) (4,843 ) Net profit/(loss) for the year (33,562 ) (38,469 ) (228,034 ) (378,084 ) Attributable to owners of the Company (33,562 ) (38,469 ) (228,034 ) (378,084 ) Basic earnings/(loss) per share € (0.55 ) € (0.64 ) € (3.76 ) € (6.53 ) Diluted earnings/(loss) per share € (0.55 ) € (0.64 ) € (3.76 ) € (6.53 ) Consolidated Statement of Comprehensive Income or (Loss) Net profit/(loss) for the year (33,562 ) (38,469 ) (228,034 ) (378,084 ) Other comprehensive income/(loss) Items that may be reclassified subsequently to profit or (loss): Exchange differences on translating foreign operations (490 ) 830 (3,538 ) 1,062 Other comprehensive income/(loss) for the year, net of tax (490 ) 830 (3,538 ) 1,062 Total comprehensive income/(loss) for the year, net of tax (34,052 ) (37,639 ) (231,572 ) (377,022 ) Attributable to owners of the Company (34,052 ) (37,639 ) (231,572 ) (377,022 ) Ascendis Pharma A/S Consolidated Statements of Financial Position (In EUR'000s) December 31, 2025 December 31, 2024 Assets Non-current assets Intangible assets 3,710 4,028 Property, plant and equipment 146,479 98,714 Investments in associates 32,526 13,575 Other receivables 10,870 2,317 193,585 118,634 Current assets Inventories 301,533 295,609 Trade receivables 141,333 166,280 Income tax receivables 1,781 1,775 Other receivables 14,582 9,385 Prepayments 33,715 28,269 Cash and cash equivalents 616,041 559,543 1,108,985 1,060,861 Total assets 1,302,570 1,179,495 Equity and liabilities Equity Share capital 8,322 8,149 Distributable equity (171,143 ) (113,855 ) Total equity (162,821 ) (105,706 ) Non-current liabilities Borrowings 385,254 365,080 Contract liabilities 1,123 5,000 Deferred tax liabilities 9,623 7,258 396,000 377,338 Current liabilities Convertible notes, matures in April 2028 Borrowings 429,391 458,207 Derivative liabilities 256,231 150,670 685,622 608,877 Other current liabilities Borrowings 57,141 33,329 Contract liabilities 4,944 936 Trade payables and accrued expenses 90,657 96,394 Other liabilities 58,204 67,956 Income tax payables 6,427 1,222 Provisions 166,396 99,149 383,769 298,986 1,069,391 907,863 Total liabilities 1,465,391 1,285,201 Total equity and liabilities 1,302,570 1,179,495

临床结果临床3期财报申请上市

100 项与帕罗培特立帕肽相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	H05AA	-

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
甲状旁腺功能减退症	欧盟	Ascendis Pharma Bone Diseases A/S	2023-11-17
甲状旁腺功能减退症	冰岛	Ascendis Pharma Bone Diseases A/S	2023-11-17
甲状旁腺功能减退症	列支敦士登	Ascendis Pharma Bone Diseases A/S	2023-11-17
甲状旁腺功能减退症	挪威	Ascendis Pharma Bone Diseases A/S	2023-11-17

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
软骨发育不全	申请上市	美国	Ascendis Pharma A/S	-
软骨发育不全	申请上市	欧盟	Ascendis Pharma A/S	-

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04701203 (PaTHway, Company_Website) 人工标引	临床3期	甲状旁腺功能减退症	73	Palopegteriparatide	衊蓋衊鹹衊願夢憲蓋醖(鬱顧齋積獵遞糧夢簾範) = 築餘願壓選鬱觸醖選鹹糧膚獵鹽觸鹹廠鹹製窪 (夢廠淵鑰壓淵鬱遞觸獵 ) 更多	积极	2025-07-14
NCT04009291 (PaTH Forward, NEWS \| Corporate Publications) 人工标引	临床2期	甲状旁腺功能减退症	56	Palopegteriparatide	憲鹹鹹齋糧鹹遞構鏇觸(膚鹽膚壓鏇獵蓋衊構齋) = 鑰淵簾鏇構餘選網餘積窪網獵襯蓋膚廠網鹹構 (艱襯繭選襯鏇鏇衊餘鏇 ) 更多	积极	2025-05-18
NCT04701203 (PaTHway, CTgov)	临床3期	甲状旁腺功能减退症	84	TransCon PTH (TransCon PTH)	齋鑰鹹獵積窪鑰構窪鬱 = 鹹膚鹽壓壓糧糧膚構製鬱齋憲鹽齋遞鹹範鹽鑰 (簾築築膚遞製衊鏇積顧, 蓋衊齋網衊網繭憲憲鹽 ~ 獵夢壓壓壓範築餘築艱) 更多	-	2025-02-28
				Placebo (Placebo)	齋鑰鹹獵積窪鑰構窪鬱 = 鹹衊艱衊艱鹹鹹糧淵鬱鬱齋憲鹽齋遞鹹範鹽鑰 (簾築築膚遞製衊鏇積顧, 鏇艱餘淵窪積網艱襯網 ~ 醖齋範築範淵願遞鹹顧) 更多
NCT05387070 (PaTHway, PRNewswire) 人工标引	临床3期	甲状旁腺功能减退症	-	Palopegteriparatide	餘選遞觸壓築鏇蓋憲衊(憲繭範窪鏇衊願積鏇襯) = 窪醖蓋積範廠築遞糧艱鬱艱衊壓廠廠築簾鏇鏇 (繭範鏇鬱淵鑰鏇願齋窪 ) 更多	积极	2024-08-12
				Placebo	餘選遞觸壓築鏇蓋憲衊(憲繭範窪鏇衊願積鏇襯) = 觸蓋觸鹽餘鹹構窪夢憲鬱艱衊壓廠廠築簾鏇鏇 (繭範鏇鬱淵鑰鏇願齋窪 )
NCT04701203 (FDA_CDER) 人工标引	临床3期	甲状旁腺功能减退症	82	YORVIPATH	顧窪淵鹹糧遞憲鏇網餘(衊廠糧醖襯觸壓窪糧鏇) = 壓鏇積鹽範襯選齋襯觸觸餘夢醖壓獵衊襯獵繭 (窪壓願構遞餘範觸齋夢 ) 更多	积极	2024-08-09
				Placebo	顧窪淵鹹糧遞憲鏇網餘(衊廠糧醖襯觸壓窪糧鏇) = 衊壓襯窪壓顧窪齋鬱鬱觸餘夢醖壓獵衊襯獵繭 (窪壓願構遞餘範觸齋夢 ) 更多
NCT04701203 (PaTHway, Pubmed \| Adv Ther)	临床3期	甲状旁腺功能减退症	82	Palopegteriparatide	憲繭襯淵淵鬱糧鹹壓餘(網鹹餘窪鑰襯繭蓋選醖) = One case of nephrolithiasis was reported for a participant in the placebo group during blinded treatment; none were reported through week 52 with palopegteriparatide 構艱齋廠簾糧顧鬱餘構 (艱網膚簾淵選積餘顧醖 )	积极	2024-06-01
NCT04701203 (PaTHway, GlobeNewswire) 人工标引	临床3期	甲状旁腺功能减退症	82	TransCon PTH (palopegteriparatide)	膚糧選夢窪願積蓋夢齋(網窪積廠範繭繭壓積艱) = 獵積構願鬱網窪衊範廠壓憲積簾鹹繭遞繭鹽選 (廠顧鹽遞範選範憲選夢 ) 更多	积极	2024-05-13
				TransCon PTH (palopegteriparatide) (eGFR < 60 mL/min/1.73m2)	膚糧選夢窪願積蓋夢齋(網窪積廠範繭繭壓積艱) = 窪積製憲憲範餘觸淵糧壓憲積簾鹹繭遞繭鹽選 (廠顧鹽遞範選範憲選夢 ) 更多
NCT04009291 \| NCT04701203 \| NCT05387070 (PaTHway, ENDO2023)	临床3期	甲状旁腺功能减退症	82	TransCon PTH	網蓋窪艱鬱範鹹製範壓(鏇築襯網淵製顧憲淵襯) = trended toward norms from baseline to Weeks 26 and 52 鹽選衊遞觸遞餘糧壓壓 (範鏇繭壓餘憲遞範獵築 ) 更多	积极	2023-10-05
NCT04009291 (PaTHway \| PaTH Forward, CTgov)	临床2期	甲状旁腺功能减退症	59	TransCon PTH (TransCon PTH 15 mcg)	醖積顧遞齋鏇醖範繭憲 = 憲窪願蓋鹹夢選鬱鬱範鬱齋艱鏇遞積願製糧窪 (製遞顧顧淵衊襯簾範蓋, 淵窪憲鏇窪衊膚積鏇製 ~ 淵構醖艱襯觸齋獵憲夢) 更多	-	2023-09-01
				TransCon PTH (TransCon PTH 18 mcg)	醖積顧遞齋鏇醖範繭憲 = 鏇網衊鹹蓋觸獵齋夢積鬱齋艱鏇遞積願製糧窪 (製遞顧顧淵衊襯簾範蓋, 獵鏇廠壓蓋淵夢齋衊衊 ~ 鏇膚醖淵衊繭網網淵窪) 更多
NCT04009291 \| NCT04701203 \| NCT05387070 (PaTHway, ENDO2022)	临床3期	甲状旁腺功能减退症	82	TransCon PTH 18 mcg/d	蓋鬱獵網蓋醖蓋艱蓋醖(艱繭糧顧製襯糧鏇膚蓋) = The most commonly reported study drug-related AEs were headaches 鬱窪鬱顧鏇鏇觸壓餘鏇 (選顧廠鏇製鏇鬱選構積 ) 更多	积极	2022-11-01
				Placebo