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

/ Not yet 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2026-01-01

申办/合作机构

Ascendis Pharma A/S

NCT07081997

/ Not yet recruiting临床3期

A Phase 3, Multicenter, Single Arm, 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2025-11-01

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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6,594

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.

230

项与帕罗培特立帕肽相关的新闻（医药）

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

2026-02-11

·肽研社

Tides | 药王易主，诺和诺德不得不All In Wegovy

温馨提示肽研社建读者交流群啦~ 行业交流、商务合作、报告咨询请添加小编微信，后续入群哦👇 研究进展 01 Ayrmid 2026-02-04爱尔兰，Ayrmid公布的真实世界研究数据显示，其长效 CXCR4 抑制肽药物 motixafortide 在镰状细胞病和 β-地中海贫血患者中展现出显著的造血干细胞（HSC）动员能力，为基因治疗的实施扫清了关键障碍。相关研究结果已在 2026 年 TANDEM（ASTCT® 与 CIBMTR® 联合年会）上发布。 Motixafortide 是一种结构优化的 CXCR4 拮抗肽分子，目前已获 FDA 批准，与 G-CSF（filgrastim）联用用于多发性骨髓瘤的干细胞动员。其独特的药代动力学特征和持久 CXCR4 阻断能力，使其在传统动员方案效果受限的遗传性血液病患者中受到关注，尤其是在镰状细胞病这一干细胞采集长期受限的治疗场景中。本次来自五个治疗中心的真实世界研究共纳入 15 名 14–50 岁患者。结果显示，73% 的患者（11/15）成功采集到足量 HSC 以进入基因治疗生产阶段，其中 5 名患者已完成基因治疗并实现成功植入，另有 5 名患者的治疗产品正在生产中。值得注意的是，这 11 名成功动员的患者此前均曾在 plerixafor 动员方案下采集失败，凸显了 motixafortide 作为 CXCR4 靶向肽类动员剂在困难人群中的临床价值。此外，还有 3 名患者将 motixafortide 作为一线动员方案，同样实现了有效采集。在已完成或正在进行的治疗中，患者接受了包括 Casgevy、Lyfgenia 和 Zynteglo 在内的多种基因治疗产品，显示由 motixafortide 动员获得的干细胞可适配不同的基因治疗制造平台，进一步验证了其临床通用性和技术兼容性。 02 Pharmazz 2026-02-04，Pharmazz在2026年国际卒中大会（ISC 2026）上公布其内皮素B（ET-B）受体激动剂 sovateltide（Tycamzzi®）治疗急性缺血性脑卒中的IV期临床研究中期分析结果。数据显示，该药物在功能恢复方面展现出显著疗效，并保持良好的安全性。该项IV期研究（NCT05955326）为一项在印度开展的多中心随机对照试验，旨在评估sovateltide在卒中发病24小时内给药的真实世界疗效与安全性。在研究设计方面，入组对象为18–78岁、影像学确诊缺血性脑卒中且NIHSS评分≥6分的患者。受试者随机接受sovateltide（0.3 μg/kg）或生理盐水对照治疗。药物于第1、3、6天分别给药，每次给药包含3次静脉推注，间隔3±1小时。主要终点为第90天mRS功能评分。中期分析共纳入80例完成90天随访的患者，其中39例接受sovateltide治疗，41例为对照组。结果显示： - 92.3%的sovateltide治疗患者在第90天达到改良Rankin量表（mRS）0–2分，而对照组为58.5%（OR=8.50，p=0.0005） - 有利功能结局绝对提高33% - 84.6%的治疗组患者mRS评分改善≥2分，对照组为51.2%（p=0.0014） - 两组患者基线特征（年龄、NIHSS评分、治疗时间窗等）均具有可比性 - 未观察到药物相关不良事件，整体安全性与既往研究一致 Sovateltide是一种全球first-in-class高选择性ET-B受体激动剂，可通过神经保护、促血管生成以及促进神经再生发挥作用。该药物已于2023年在印度获批上市，迄今累计治疗患者超过10万人。目前，Pharmazz正在推进全球III期随机双盲安慰剂对照研究RESPECT-ETB（NCT05691244），该研究计划在美国、欧洲、英国及澳大利亚入组超过500例急性缺血性脑卒中患者。 03 先为达 2026-02-05，先为达的XW014片获得CDE临床默示许可，用于肥胖或超重合并至少一种体重相关合并症人群的体重管理。 04 甘李药业 2026-02-06，甘李药业的注射用GLR1059临床申请获得CDE受理（CXSL2600182）。 05 Novo Nordisk 2026-02-09，根据Clinicaltrials官网显示，Novo Nordisk注册了UBT251用于治疗肥胖的全球二期临床试验。该候选药物为联邦制药自主研发的GLP-1/GIP/GCG受体激动剂。根据2024年披露的一期临床数据，半衰期为136-170小时，支持每周一次给药，1.0-4.5mg剂量范围内，体重下降幅度为3.19-2.80kg，明显优于安慰剂对照组。2025年3月，联邦制药宣布将该分子的大中华区外全球权益授权给Novo Nordisk，后者支付2亿美元预付款、18亿美元里程碑金额，以及一定比例的销售分成。 06 先为达 2026-02-10，先为达的埃诺格鲁肽注射液临床申请获得CDE受理。 07 MannKind 2026-02-10美国，MannKind宣布其针对新诊断儿童及青少年 1 型糖尿病患者的临床研究 INHALE-1ST 已完成首例受试者入组。该研究旨在评估在确诊初期即启动 Afrezza® 吸入式人胰岛素治疗，在10至未满18岁青少年患者中的安全性、有效性及治疗体验。MannKind 是一家专注于心代谢及罕见肺部疾病的生物制药公司，此项研究由 Jaeb Center for Health Research 牵头开展。 INHALE-1ST 研究设计为单臂、多中心临床研究，评估 Afrezza 联合每日一次皮下注射基础胰岛素用于新诊断 1 型糖尿病青少年患者的治疗效果。研究不仅关注血糖控制等临床终点，还将系统评估患者及照护者在疾病早期使用吸入式胰岛素进行餐时血糖管理的满意度与体验。研究计划在美国约 10 家临床中心入组约 100 名患者，首例患者已在科罗拉多州奥罗拉的 Barbara Davis 糖尿病中心完成入组。该研究主阶段为期 13 周，随后设有可选延展阶段，允许持续使用 Afrezza 联合基础胰岛素治疗最长 26 周。主要终点为在第 13 周访视前 14 天内，佩戴连续血糖监测仪（CGM）的受试者中，血糖处于 70–180 mg/dL 目标范围（TIR）时间比例达到或超过 70% 的患者比例。相关研究信息已在 ClinicalTrials.gov 登记（NCT07224321）。在监管进展方面，MannKind于2025年10月向 FDA 递交的 Afrezza 儿科适应症补充生物制品许可申请（sBLA）已获受理，PDUFA 目标审评日期为2026-05-29。若获批准，Afrezza有望成为 100 多年来首个适用于儿童和青少年患者的无针胰岛素治疗选择。注册获批 01 银诺医药 2026-02-04，银诺医药（02591.HK）宣布其核心产品依苏帕格鲁肽 α 用于治疗宠物糖尿病的新兽药临床试验申请已获农业部正式受理，预计将于 2026Q1开始 I 期临床试验。紧接着2月6日，公司宣布成立宠物事业部，将战略性布局宠物用创新药领域。依托其在代谢性疾病领域的核心技术积淀，充分复用在GLP-1领域的技术积累与产业化经验，针对性治疗宠物糖尿病、肥胖等宠物常见病症。 2025 年 1 月，依苏帕格鲁肽 α 在国内获批上市，用于成人 2 型糖尿病治疗，包括单药以及接受二甲双胍药物治疗血糖仍控制不佳的患者，每周皮下注射一次。同年，该产品还通过谈判顺利进入了医保目录，其减重适应症也正在申请中。 02 Insulet Corporation 2026-02-05，Insulet Corporation (NASDAQ: PODD) 宣布其Omnipod 5自动化胰岛素输注系统（AID）已在中东多个核心市场实现商业化落地，包括沙特阿拉伯、科威特、卡塔尔和阿联酋。该系统可与雅培FreeStyle Libre 2 Plus及Dexcom G7连续血糖监测（CGM）传感器兼容。相关进展于迪拜举行的第16届阿联酋糖尿病与内分泌大会（EDEC）期间正式发布。 Omnipod 5是一款无管路（tubeless）自动化胰岛素输注系统，通过每5分钟自动调节胰岛素给药，实现全天候动态控糖，减少多次每日注射（MDI）的负担。临床数据显示，该系统可显著改善血糖达标时间（Time in Range）并降低HbA1c水平。其防水、隐蔽、可穿戴的设计，使其成为目前首个可与CGM联动、主动纠正高血糖并预防低血糖的无管路AID系统。在适应症方面，Omnipod 5适用于2岁及以上1型糖尿病患者。在沙特、科威特和阿联酋，该系统可同时兼容雅培FreeStyle Libre 2 Plus和Dexcom G7传感器；在卡塔尔则兼容Dexcom G7传感器。与此同时，Insulet还在上述中东市场首次推出Omnipod Discover数据管理平台。该平台是一款基于网络的回顾性数据分析与报告工具，面向患者、照护者及医疗专业人员，旨在整合血糖与胰岛素输注数据，将复杂的糖尿病管理信息转化为清晰、可操作的趋势和洞察。Omnipod Discover可直观展示Omnipod 5算法如何自动调整胰岛素给药，帮助医生和患者更高效地理解数据、优化决策，从而降低临床管理和日常使用负担。 Insulet表示，Omnipod Discover将以中东市场作为首发区域，未来计划扩展至其他已上市Omnipod 5的国家和地区。随着此次中东市场的拓展，Omnipod 5目前已在全球19个国家实现商业化。公司预计将于2026年进入西班牙市场，并计划在2027年上半年陆续在希腊和克罗地亚上市。 03 南京海融制药 2026-02-06，南京海融制药的注射用醋酸西曲瑞克获得NMPA批准（国药准字H20263289）。 04 Ascendis 2026-02-06加拿大，Pendopharm（Pharmascience旗下部门）宣布，加拿大卫生部（Health Canada）已批准 Yorvipath®（palopegteriparatide 注射液）在加拿大上市，用于治疗成人慢性甲状旁腺功能减退症。Yorvipath® 是一种甲状旁腺激素（PTH）替代治疗方案，由 Ascendis开发。此次获批标志着该创新疗法正式进入加拿大市场，为长期缺乏新治疗选择的患者提供了新的疾病管理手段。此次监管批准基于 Pendopharm 与 Ascendis 于 2024 年 7 月签署的加拿大独家分销协议。根据该协议，Pendopharm 负责 Yorvipath® 在加拿大的注册、商业化及市场推广工作。该产品此前已在美国、欧盟、英国、澳大利亚、瑞士、以色列和日本获批，并正在其他全球市场推进注册或开发。Pendopharm 表示，目前正与多方关键合作伙伴紧密协作，推进产品顺利上市，并同步开展公共和私人医保体系的准入与报销谈判。 Yorvipath® 的获批进一步巩固了 Ascendis 基于 TransCon® 技术平台在内分泌罕见病领域的布局，也凸显了 Pendopharm 在加拿大引入创新专科药物、解决未满足医疗需求方面的能力与执行力。市场营销 01 Novo Nordisk 2026-02-08美国，Novo Nordisk在“超级碗”赛事期间，首次发布品牌重磅广告，正式向大众介绍Wegovy®（司美格鲁肽）口服片剂25mg。该产品为目前美国唯一获FDA批准、用于体重管理的口服GLP-1受体激动剂，适用于肥胖成人，或伴有体重相关合并症的超重成人，在控制热量摄入和增加运动的基础上，用于减重及维持体重。此次名为 “A New Way to Wegovy®” 的传播活动，通过轻松幽默的方式，尝试打破公众对减重药物“走捷径”的刻板印象，将讨论重点从羞耻感和犹豫，转向基于医学与科学证据的体重管理选择。在商业可及性方面，Wegovy®口服片剂已在美国7万多家药房广泛上市，包括CVS、Costco等连锁药房，并通过Ro、LifeMD、Weight Watchers等部分远程医疗平台，以及NovoCare® Pharmacy和GoodRx等渠道提供。对于自费患者，起始剂量（1.5 mg）可通过官方渠道以每月$149的价格获得；符合条件的商业保险患者，在优惠计划下最低可支付每月$25。目前，美国已有超过5500万人享有FDA批准的肥胖治疗药物保险覆盖。 02 Novo Nordisk 2026-02-09美国&丹麦，Novo Nordisk宣布已正式对远程医疗公司 Hims & Hers提起诉讼，指控其在美国市场销售的复方司美格鲁肽产品侵犯了Novo Nordisk的美国专利 US 8,129,343，并通过大规模营销未经FDA批准的产品误导患者和医疗专业人士，对公共健康构成严重风险。 Novo Nordisk表示，Hims & Hers长期开展针对复方司美格鲁肽产品的推广活动，夸大其临床获益和安全性，使消费者和医务人员误以为这些未经批准的产品与原研药具有同等质量与疗效。公司特别指出，Hims & Hers在Novo Nordisk推出全球首款、也是唯一获得FDA批准的GLP-1减重口服药 Wegovy® pill 后不久，曾高调推出所谓“复方GLP-1口服片”，但仅两天后即匆忙下架；与此同时，Hims & Hers仍在继续非法大规模复方配制并销售注射用司美格鲁肽产品，其所使用的API并非正品来源，存在严重安全隐患。诉讼中，Novo Nordisk请求法院永久禁止Hims & Hers销售侵犯其专利的未经批准复方药物，并追究其造成的经济损失。公司同时警告称，Hims & Hers与部分复方药房的大规模营销行为已导致市场充斥Wegovy®和Ozempic®的“仿制品”。这些复方司美格鲁肽产品可能含有危险杂质或错误剂量的活性成分，从而引发严重免疫反应、住院治疗、严重药物相互作用甚至过量用药等风险。根据Novo Nordisk的检测结果，由复方药房配制的注射用司美格鲁肽产品中，杂质含量最高可达86%，而口服复方司美格鲁肽产品中的杂质比例最高可达75%。即使是少量杂质，也可能对药物的安全性和有效性产生重大不利影响，包括诱发过敏性休克等严重免疫反应。 Hims & Hers于上周四正式推出其所谓的“司美格鲁肽口服片”，初期促销价为每月49美元，随后上调至99美元；相比之下，Novo Nordisk的原研产品 Wegovy® pill 起始价为149美元，常规月费为199美元。然而，在面临司法部潜在调查风险及Novo Nordisk明确的法律行动威胁后，Hims & Hers在产品上市仅几天后便宣布停止提供该治疗方案。Hims & Hers于2月8日在X平台发布简短声明称，公司已与行业相关方进行了“建设性沟通”，并据此决定下架该产品。该消息发布后，Hims & Hers股价在盘后交易中一度下跌近16%。就在上周，美国FDA也再次明确指出，包括Hims & Hers在内的复方药房所大规模销售的GLP-1类产品属于“FDA无法核实其质量、安全性或有效性的药物”。美国医学会（AMA）、美国糖尿病协会（ADA）及内分泌学会等权威医学组织也多次对GLP-1仿制产品表达担忧。ADA明确建议不要使用此类产品，原因在于其成分、安全性、质量一致性和疗效均存在高度不确定性。此次诉讼是Novo Nordisk多年持续行动的一部分，旨在保护患者免受不安全复方产品的侵害。此前，公司已发起包括“Check Before You Inject”和“Choose The Real Thing”在内的多项公众教育活动，提醒患者警惕使用来源不明、含有境外非正品API的未经批准药物。企业发展 01 Lilly 2026-02-04美国，Lilly公布了2025年全年财务业绩——在GLP-1产品持续放量的推动下实现强劲增长。Mounjaro（替尔泊肽降糖适应症）全年销售额$22965Mn（增长99%），Zepbound（替尔泊肽减重适应症）全年销售额$13542Mn（增长175%），替尔泊肽累计2025年销售额达到$36507Mn。 02 OPKO Health & Entera Bio 2026-02-04美国，OPKO Health（NASDAQ: OPK）通过其全资子公司 OPKO Biologics，与口服肽与蛋白替代疗法领域的领先企业 Entera Bio（NASDAQ: ENTX）宣布，双方将扩展于2025年签署的合作与许可协议，推进全球首个口服长效甲状旁腺激素（LA-PTH）肽类似物，用于甲状旁腺功能减退症患者的一日一次口服给药治疗。该新增项目将 OPKO 专有的长效PTH肽变体与 Entera 的 N-Tab® 口服肽递送平台相结合。这是双方第三个成功整合口服肽技术与先进蛋白/肽工程能力的研发项目。基于2025年12月公布的积极PK/PD数据，双方决定加速该项目开发，预计将于2026H2向美国FDA提交IND申请。根据扩展后的合作协议，OPKO 与 Entera 将以 50:50 的比例共同持有 LA-PTH 项目的权益，并各自承担一半的研发费用；而在另一条口服胃泌酸调节素（OXM）肽项目中，双方仍维持此前公布的 OPKO 60%、Entera 40% 的权益与成本分担结构。在管线进展方面，双方正同步推进可注射与口服两种剂型的OXM项目。OXM 是一种天然存在的GLP-1 / 胰高血糖素双重激动肽，在体重管理、代谢性疾病及抗纤维化领域具有潜在优势。注射型OXM的一期临床初步数据预计将于2026年末公布，随后口服OXM肽制剂将进入临床阶段。目前，全球尚无获批的GLP-1/胰高血糖素双重激动剂。 03 云顶新耀 & 麦科奥特 2026-02-05，云顶新耀（HKEX 1952.HK），一家专注于创新药研发、临床开发、制造及商业化的生物制药公司宣布与麦科奥特签署独家商业化许可协议，获得MT1013在中国及亚太区（日本除外）的独家商业化授权。MT1013为全球首创的双靶点受体激动剂多肽，可同时靶向钙敏感受体(CaSR)及成骨生长肽(OGP)受体，开发主要用于治疗继发性甲状旁腺功能亢进症(SHPT)。此次战略合作将与云顶新耀现有肾科管线形成协同效应，强化产品布局，巩固公司在亚洲肾脏及自身免疫疾病领域的领导地位，并将产品覆盖从IgA肾病拓展至更广泛的慢性肾脏病领域。根据协议，云顶新耀将向麦科奥特支付人民币2亿元首付款，以及最高不超过人民币10.40亿元的潜在监管及商业里程碑付款。中国III期临床研究正在进行中，相关临床开发费用将由麦科奥特承担。 MT1013是由麦科奥特自主研发的全球首创双靶点多肽新药，2025年获美国肾脏病学会（ASN）年会“Late-Breaking”收录。其开创性地融合了“钙敏感受体（CaSR）+成骨生长肽（OGP）模拟”双重作用机制，解决了PTH、钙、磷代谢平衡的问题，这一全新设计使其在治疗继发性甲状旁腺功能亢进（SHPT）及相关骨代谢疾病时，首次创造性的从源头上控制病情，且能通过直接激活成骨通路，主动促进骨形成与修复，实现从“间接抑制骨吸收”到“主动促进骨生成”的治疗创新升级。临床研究证实，MT1013在慢性肾病接受维持性血液透析伴继发性甲状旁腺功能亢进患者中，起效迅速、效力强且持久，安全性良好，在综合达标率（iPTH，血钙，血磷均达标）、血钙钙化指标的控制及心血管获益前景上均展示出超越现有疗法的潜力。目前，MT1013正在中国开展针对该患者群体的III期临床研究，已入组超50%。 MT1013已完成关键II期临床研究，数据显示，MT1013在维持性血液透析伴SHPT患者中，展现出强效且持久的iPTH抑制能力，并在与依特卡肽头对头比较中实现了综合管理的优势——其iPTH、血钙、血磷三项同时达标率更高，降磷及降低心血管风险标志物FGF-23的优势更强，加之MT1013在长达52周的治疗中也为患者人群带来了显著的骨密度提升及骨代谢标志物改善，逐步验证了其独特的双重机制在SHPT患者中综合管理及改善骨骼健康方面的潜在临床获益。目前MT1013已进入以西那卡塞为阳性对照的确证性III期临床试验。该III期试验已在全国范围内启动超过100家研究中心，计划招募约424名患者，主要针对慢性肾脏病维持性血液透析伴SHPT的患者群体。 2026-02-05，云顶新耀（HKEX 1952.HK），一家专注于创新药研发、临床开发、制造及商业化的生物制药公司宣布与麦科奥特签署独家商业化许可协议，获得MT1013在中国及亚太区（日本除外）的独家商业化授权。MT1013为全球首创的双靶点受体激动剂多肽，可同时靶向钙敏感受体(CaSR)及成骨生长肽(OGP)受体，开发主要用于治疗继发性甲状旁腺功能亢进症(SHPT)。此次战略合作将与云顶新耀现有肾科管线形成协同效应，强化产品布局，巩固公司在亚洲肾脏及自身免疫疾病领域的领导地位，并将产品覆盖从IgA肾病拓展至更广泛的慢性肾脏病领域。根据协议，云顶新耀将向麦科奥特支付人民币2亿元首付款，以及最高不超过人民币10.40亿元的潜在监管及商业里程碑付款。中国III期临床研究正在进行中，相关临床开发费用将由麦科奥特承担。 MT1013是由麦科奥特自主研发的全球首创双靶点多肽新药，2025年获美国肾脏病学会（ASN）年会“Late-Breaking”收录。其开创性地融合了“钙敏感受体（CaSR）+成骨生长肽（OGP）模拟”双重作用机制，解决了PTH、钙、磷代谢平衡的问题，这一全新设计使其在治疗继发性甲状旁腺功能亢进（SHPT）及相关骨代谢疾病时，首次创造性的从源头上控制病情，且能通过直接激活成骨通路，主动促进骨形成与修复，实现从“间接抑制骨吸收”到“主动促进骨生成”的治疗创新升级。临床研究证实，MT1013在慢性肾病接受维持性血液透析伴继发性甲状旁腺功能亢进患者中，起效迅速、效力强且持久，安全性良好，在综合达标率（iPTH，血钙，血磷均达标）、血钙钙化指标的控制及心血管获益前景上均展示出超越现有疗法的潜力。目前，MT1013正在中国开展针对该患者群体的III期临床研究，已入组超50%。 MT1013已完成关键II期临床研究，数据显示，MT1013在维持性血液透析伴SHPT患者中，展现出强效且持久的iPTH抑制能力，并在与依特卡肽头对头比较中实现了综合管理的优势——其iPTH、血钙、血磷三项同时达标率更高，降磷及降低心血管风险标志物FGF-23的优势更强，加之MT1013在长达52周的治疗中也为患者人群带来了显著的骨密度提升及骨代谢标志物改善，逐步验证了其独特的双重机制在SHPT患者中综合管理及改善骨骼健康方面的潜在临床获益。目前MT1013已进入以西那卡塞为阳性对照的确证性III期临床试验。该III期试验已在全国范围内启动超过100家研究中心，计划招募约424名患者，主要针对慢性肾脏病维持性血液透析伴SHPT的患者群体。 04 LIR 2026-02-05加拿大，LIR Life Sciences宣布已根据现有服务协议，正式与 Neuland Laboratories启动 Project Phase 2肽设计工作，进一步推进其用于经皮递送平台的新一代细胞穿透肽（CPP）研发。本次二期项目在一期肽合成工作成功完成的基础上展开，核心聚焦于构效（SAR）分析，旨在设计并优化第三代肽候选物，以在保持强经皮转运能力的同时，提升可合成性、工艺效率及制剂灵活性，为后续规模化生产奠定基础。 Project Phase 2 将系统性探索一系列源自 LIR 铅型鱼精蛋白（protamine-based）序列的合成变体，通过理性设计筛选在经皮递送性能与制造可行性之间实现更优平衡的候选肽分子。LIR 与 Neuland 将协同推进代表性肽结构的优化，并确定后续优先合成与评估的目标序列。该阶段产生的结果将为未来的肽合成、制剂开发及生物学验证提供关键决策依据。 LIR Life Sciences 专注于通过创新递送方式开发可规模化、可负担的肥胖治疗方案，目前正推进基于 GLP-1 作用机制的经皮贴剂及其他非注射递送系统，目标是在全球范围内提升治疗可及性、依从性与成本效率。 05 Kashiv BioSciences 2026-02-10美国，Kashiv BioSciences宣布已与印度领先的跨国制药公司 Intas Pharmaceuticals 达成一项独家许可及供货协议，双方将就一款复杂肽类产品展开合作。根据协议安排，该产品将在印度由 Intas 负责商业化，在欧洲及英国市场则由其全资子公司 Accord Healthcare 进行推广和销售，合作范围覆盖泛欧洲、英国及印度市场。按照协议条款，Kashiv 将负责该产品的注册申报、生产制造及全球供应，而 Accord 将主导欧洲及英国地区的商业化和分销工作。Kashiv 表示，此次合作将借助 Intas 及 Accord 在欧洲市场成熟而广泛的商业化网络，进一步拓展公司在欧洲、英国及印度等关键市场的布局，并持续强化其区域产品组合。 Kashiv BioSciences 是一家总部位于美国的垂直整合型生物制药公司，拥有多项已商业化及处于临床后期阶段的产品管线，并跻身少数同时具备生物类似药生产能力并获得多项上市许可的美国企业之列。公司通过其在美国及印度的业务布局，形成了覆盖研发、临床、生产、注册及知识产权的全球化能力体系，持续推动重要药物的可及性与患者获益。部分图文来源于网络，如有侵权，请联系删除旗舰报告专栏推荐肽研社生物医药 · 美妆个护 · 营养健康动物健康 · 绿色农业 · 生物材料专业专注 | 成就客户 | 共生成长

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	繭觸廠構廠繭網觸鬱顧(觸醖鏇構觸窪艱蓋積憲) = 襯構積廠壓鹹選繭膚鏇艱鏇積餘簾網廠願餘衊 (廠簾淵簾製遞積願製蓋 ) 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	壓壓鹽鹽鬱鑰顧範鬱襯(選選淵築構鑰廠鹽鹽獵) = 齋艱鑰願鹹廠鏇製製廠顧夢蓋獵觸蓋製遞醖廠 (廠蓋糧製鑰鑰簾壓積鬱 ) 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)	淵廠糧築襯鏇鬱齋鹽憲 = 淵醖顧襯製範遞願積憲鹹膚積齋衊窪獵鑰簾鹹 (憲齋蓋觸壓鹹襯構觸鏇, 壓鬱憲積襯製衊齋繭願 ~ 壓網夢衊構製壓鑰鬱獵) 更多	-	2025-02-28
				Placebo (Placebo)	淵廠糧築襯鏇鬱齋鹽憲 = 醖餘艱蓋網遞夢遞糧繭鹹膚積齋衊窪獵鑰簾鹹 (憲齋蓋觸壓鹹襯構觸鏇, 簾簾鏇蓋觸願積鑰觸衊 ~ 襯淵鬱廠窪範艱鬱廠鹽) 更多
NCT05387070 (PaTHway, PRNewswire) 人工标引	临床3期	甲状旁腺功能减退症	-	Palopegteriparatide	壓顧蓋顧願憲顧膚獵鹹(築糧鏇廠衊獵糧鏇衊積) = 艱夢淵觸壓壓憲齋簾艱襯鏇築繭選築壓願繭網 (簾簾憲鑰鹹製餘鬱積淵 ) 更多	积极	2024-08-12
				Placebo	壓顧蓋顧願憲顧膚獵鹹(築糧鏇廠衊獵糧鏇衊積) = 窪襯糧範鑰獵餘壓顧選襯鏇築繭選築壓願繭網 (簾簾憲鑰鹹製餘鬱積淵 )
NCT04701203 (FDA_CDER) 人工标引	临床3期	甲状旁腺功能减退症	82	YORVIPATH	鬱範範簾繭廠淵積醖築(鏇夢願網鏇窪艱製獵獵) = 糧鏇壓網夢鹹窪蓋窪蓋鏇積艱窪衊積醖襯鏇鑰 (鑰蓋範製膚膚餘積積顧 ) 更多	积极	2024-08-09
				Placebo	鬱範範簾繭廠淵積醖築(鏇夢願網鏇窪艱製獵獵) = 願選鑰鏇膚積憲範範鏇鏇積艱窪衊積醖襯鏇鑰 (鑰蓋範製膚膚餘積積顧 ) 更多
NCT04701203 (PaTHway, Pubmed \| Adv Ther)	临床3期	甲状旁腺功能减退症	82	Palopegteriparatide	蓋鏇膚積憲獵構願製遞(齋遞觸醖艱願衊顧鹹積) = One case of nephrolithiasis was reported for a participant in the placebo group during blinded treatment; none were reported through week 52 with palopegteriparatide 製鹽獵獵願夢廠餘築鑰 (夢醖壓範窪簾選簾襯糧 )	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)	鏇蓋範窪鹽繭蓋衊襯遞(願窪夢襯選艱齋齋獵網) = 網齋憲衊壓選選願淵範鏇簾鏇齋鏇選選鹹鹽淵 (積窪遞遞蓋鹹鹽網獵構 ) 更多	积极	2024-05-13
				TransCon PTH (palopegteriparatide) (eGFR < 60 mL/min/1.73m2)	鏇蓋範窪鹽繭蓋衊襯遞(願窪夢襯選艱齋齋獵網) = 選夢壓顧窪網襯鏇餘壓鏇簾鏇齋鏇選選鹹鹽淵 (積窪遞遞蓋鹹鹽網獵構 ) 更多
NCT04009291 \| NCT04701203 \| NCT05387070 (PaTHway, ENDO2023)	临床3期	甲状旁腺功能减退症	82	TransCon PTH	鑰製製繭窪淵顧簾齋範(齋獵蓋鹽獵觸鑰艱襯遞) = trended toward norms from baseline to Weeks 26 and 52 窪鑰餘窪襯鑰膚鏇襯網 (製網鹹壓蓋鏇淵積蓋構 ) 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)	選範製夢願憲餘顧鹽觸 = 淵鹹餘餘觸網夢艱範壓繭網壓淵築壓糧範鬱壓 (鑰夢窪築獵獵簾簾醖窪, 壓網鹹範鏇衊窪膚廠願 ~ 簾顧鹹鹽製淵鏇衊築艱) 更多	-	2023-09-01
				TransCon PTH (TransCon PTH 18 mcg)	選範製夢願憲餘顧鹽觸 = 築鏇廠網鹽齋鹽選蓋築繭網壓淵築壓糧範鬱壓 (鑰夢窪築獵獵簾簾醖窪, 糧積糧壓鏇鏇襯顧鏇蓋 ~ 範範顧艱鏇獵範窪夢積) 更多
PaTH Forward Trial (ENDO2022)	临床2期	甲状旁腺功能减退症	59	TransCon PTH 15 mcg/d	遞夢艱網選繭壓餘窪廠(鑰醖積製積壓鏇淵夢鬱) = 願鑰窪構憲繭蓋廠艱艱構餘鑰齋願糧衊構襯廠 (糧遞繭鏇壓鹽製襯範積 ) 更多	积极	2022-11-01
				TransCon PTH 18 mcg/d	遞夢艱網選繭壓餘窪廠(鑰醖積製積壓鏇淵夢鬱) = 構衊醖鹽餘簾觸獵積獵構餘鑰齋願糧衊構襯廠 (糧遞繭鏇壓鹽製襯範積 ) 更多