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 treatment 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2026-05-07

申办/合作机构

Ascendis Pharma Bone Diseases A/S

NCT07706764

/ Recruiting临床3期

A Phase 3, Multicenter, Open-Label Single-Arm Clinical Trial to Assess the Safety, Tolerability, Pharmacokinetics, and Efficacy of Palopegteriparatide Administered Subcutaneously Daily in the Adolescent Population (12 Years to Less Than 18 Years of Age) With Chronic Hypoparathyroidism

This trial will enroll adolescents between ages of ≥12 and <18 years with clinically diagnosed hypoparathyroidism . The purpose of the study is to see how well treatment with once-daily palopegteriparatide works and how safe it is. At least 12 participants will receive palopegteriparatide for 234 weeks. This trial will be conducted in Europe.

开始日期2026-04-22

申办/合作机构

Ascendis Pharma A/S

NCT07264634

/ RecruitingN/A

A Study to Assess the Amount of Palopegteriparatide in Breast Milk of Lactating Females Requiring YORVIPATH® (Palopegteriparatide)

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2026-03-19

申办/合作机构

Ascendis Pharma 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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6,156

项与帕罗培特立帕肽相关的文献（医药）

2026-12-01·BONE

The associations of obesity and other covariates with bone turnover markers in children and adolescents

Article

作者: Ward, Leanne M ; Thabane, Lehana ; Rauch, Frank ; Wong, Andy Kin On ; Kim, Hyunwoo ; Adachi, Jonathan Derrick ; Papaioannou, Alexandra ; Ma, Jinhui

BACKGROUND:

Procollagen type 1 N-terminal propeptide (P1NP) and C-terminal telopeptide of type 1 collagen (CTX) reflect skeletal metabolism during growth. We examined associations of obesity and other biological and behavioral factors with P1NP and CTX and assessed whether obesity-related associations varied by sex and across age groups approximating the pre-pubertal, pubertal, and post-pubertal periods.

METHODS:

Data were obtained from Canadian Health Measures Survey participants aged 6-20 years. Analyses included 3278 participants for P1NP (1658 males, 1620 females; 367 with obesity) and 1484 for CTX (762 males, 722 females; 162 with obesity). Survey-weighted multiple regression models were used to examine their associations with obesity status, physical activity, parathyroid hormone (PTH), serum 25-hydroxyvitamin D₃ [25(OH)D₃], and high-sensitivity C-reactive protein (hs-CRP), with all variables included simultaneously. Given the cross-sectional design, associations were interpreted as correlational.

RESULTS:

Overweight and obesity were associated with lower P1NP z-scores (β = -0.320 and - 0.378, both p < 0.001) and lower CTX z-scores (β = -0.378, p < 0.001; β = -0.278, p < 0.05). Obesity-status-by-age-group interactions were observed for P1NP and CTX (p < 0.001 and p = 0.002, respectively), whereas sex interactions were not significant. Obesity was not associated with lower marker levels in the age group approximating the pre-pubertal period but was associated with lower levels in age groups approximating the pubertal and post-pubertal periods. Cross-sectional age-related curves suggested that the highest estimated P1NP and CTX levels may occur at younger ages among participants with obesity, whereas marker levels were lower at older ages. High hs-CRP was associated with lower P1NP z-scores (β = -0.577, p < 0.001), while PTH was positively associated with P1NP and CTX z-scores (β = 0.117 and 0.103, both p < 0.01). Serum 25(OH)D₃ status and physical activity were not significantly associated with either marker.

CONCLUSIONS:

Obesity-related differences in bone turnover varied across age groups, and the observed pattern may be broadly consistent with previously reported obesity-related differences in height velocity.

2026-12-01·IBRO Neuroscience Reports

Causal effect of thyroid function on cortical brain structure: A two-sample Mendelian randomization study

Article

作者: Chen, Jianhuai ; Wang, Ruifeng ; Shi, Jianxin ; Zhao, Heng

Background:

The causal relationship between variations in thyroid dysfunction and brain structure remains elusive. We intended to probe into this causal connection using a Mendelian randomization (MR) approach.

Methods:

Aggregated statistics from the genome-wide Association Study (GWAS) on Autoimmune thyroid dysfunction were obtained from the FinnGen database, including autoimmune hyperthyroidism (N = 2,190) and autoimmune hypothyroidism (N = 49,055). Aggregate statistics on cortical area and thickness for GWAS were obtained from the Enhancing NeuroImaging Genetics through Meta-Analysis (ENIGMA) Consortium (N = 51,665). The main method employed is inverse variance weighting (IVW), supplemented by weighted median, Mr-Egger regression, MR-pleotropy residuals, and outliers. Some sensitivity analyses were conducted to determine heterogeneity and pleiotropy.

Results:

For autoimmune hypothyroidism (exposure), significant causal associations were observed with cortical thickness (outcome type) in the following regions: pars triangularis (β = -0.003, 95% CI: -0.005 to -0.001, PTH = 0.023; PTH: P-value for cortical thickness,), posterior cingulate (β = 0.003, 95% CI: 0.000-0.006, PTH = 0.034), and transverse temporal (β = 0.006, 95% CI: 0.002-0.010, PTH = 0.004; β = 0.006, 95% CI: 0.001-0.011, PTH = 0.010). For autoimmune hyperthyroidism (exposure), significant causal associations were observed with cortical surface area (outcome type) in the entorhinal (β = -2.576, 95% CI: -4.714 to -0.437, PSA = 0.018; PSA: P-value for cortical surface area), frontal pole (β = -1.195, 95% CI: -2.118 to -0.271, PSA = 0.011), lateral occipital (β = -15.787, 95% CI: -31.171 to -0.403, PSA = 0.044), lingual (β = -10.778, 95% CI: -20.888 to -0.668, PSA = 0.037), postcentral (β = -16.360, 95% CI: -28.410 to -4.309, PSA = 0.008), precentral (β = -16.089, 95% CI: -29.281 to -2.897, PSA = 0.017), and superior parietal (with global weighting; β = 11.360, 95% CI: 0.435-22.285, PSA = 0.042). Additionally, autoimmune hyperthyroidism was causally associated with cortical thickness (outcome type) in the entorhinal (β = 0.009, 95% CI: 0.000-0.018, PTH = 0.039) and posterior cingulate (β = 0.004, 95% CI: 0.001-0.008, PTH = 0.025). These findings remained reliable in the sensitivity analyses.

Conclusions:

These findings suggest that autoimmune hyperthyroidism and autoimmune hypothyroidism may have potential causal effects on the surface area and thickness of specific regions of the cerebral cortex, but further studies are needed to fully understand the nature of this relationship.

2026-12-01·Radiology case reports

Multimodality imaging localization of a giant functional parathyroid adenoma in a young fitness trainer with delayed recognition of hypercalcemia: A case report

Article

作者: Sarin, Deepak ; Sharma, Vivek ; Sharma, Naman ; Uppal, Geetanjali ; Banka, Devansh ; Gupta, Dipti

Giant parathyroid adenoma (GPA) is a rare cause of primary hyperparathyroidism (PHPT) whose symptoms overlap with normal training fatigue and dietary shifts that calcium testing gets delayed in active people. A 36-year-old male fitness trainer presented with 13 kg of unintentional weight loss over six months with weakness, and nocturia, all of which he and the physicians who first saw him attributed to his training regimen and protein supplementation. Serum calcium was 16.4 mg/dL and parathyroid hormone (PTH) 1127.4 pg/mL, with acute kidney injury and bilateral nephrolithiasis; protein electrophoresis and free light chains excluded a paraproteinemic cause. Ultrasonography, ¹⁸F-FDG PET-CT, and Technetium-99m sestamibi scintigraphy were concordant, identifying a right-sided parathyroid adenoma of approximately 2.3 × 2.3 × 5.9 cm extending into the upper mediastinum. Focused right parathyroidectomy was performed under intraoperative PTH monitoring, and PTH fell from 2233 pg/mL before excision to 172 pg/mL ten minutes after. Histopathology confirmed a benign adenoma weighing 18 g, the diagnosis resting on the absence of capsular and vascular invasion; the Ki-67 index of 3 to 4% was supportive rather than defining. Serum calcium normalized to 9.0 mg/dL by the fourth postoperative day without complications. Here the nonspecific symptoms were initially attributed to physical training and protein supplementation, and calcium was not measured until the disease was advanced. The frequency of this diagnostic pattern cannot be established from a single case. What the case does show is that concordant multimodality localization enabled focused surgery for an adenoma of unusual size and anatomical extent.

276

项与帕罗培特立帕肽相关的新闻（医药）

2026-10-26

·BioSpace

TransCon® PTH (Palopegteriparatide) Demonstrates Sustained, Clinically Meaningful Improvements in Kidney Function in Adults with Hypoparathyroidism Treated Up to 5 Years

COPENHAGEN, Denmark, Oct. 26, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced new data demonstrating early, sustained, and clinically meaningful improvements in kidney function in adults with hypoparathyroidism treated with TransCon ® PTH (palopegteriparatide) for up to 5 years in the Company’s Phase 2 PaTH Forward and Phase 3 PaTHway trials. Results from the pooled analysis were shared in a poster presented by nephrologist Elvira Gosmanova, M.D., Professor of Medicine at Albany Medical College, during Kidney Week 2026, the annual meeting of the American Society of Nephrology. “These groundbreaking improvements in renal function underscore the unique ability of TransCon PTH to replicate the systemic and balanced actions of endogenous PTH across multiple target organ systems,” said Aimee Shu, Executive Vice President and Chief Medical Officer at Ascendis Pharma. “We are pleased to showcase these benefits of TransCon PTH showing simultaneous improvements across key measures that include maintained normal serum calcium, decreased urine calcium, and sustained increases in eGFR, while enabling independence from conventional therapy.” Historically, management of hypoparathyroidism with conventional therapy consisting of active vitamin D and calcium with the aim of raising serum calcium to the lower half or just below the reference range to alleviate symptoms of hypocalcemia and avoid hypercalciuria, but did not address the missing parathyroid hormone (PTH). i Today, in contrast, it is possible to provide or restore the full range of effects of PTH that are missing in hypoparathyroidism with TransCon PTH, which provides active PTH within the physiological range for 24 hours/day. The active PTH released from TransCon PTH includes the amino acid sequence identical to the region of endogenous PTH required for receptor activation and enables simultaneous improvements across multiple key clinical and quality-of-life measures. ii , iii Data Highlights Increases in mean estimated glomerular filtration rate (eGFR) from baseline were statistically significant across patients from Week 26 of treatment through trial completion (p 10 mL/min /1.73 m 2 ; eGFR remained stable through the end of the trials. From Week 52 to Week 266, the mean eGFR slope was 0.28 mL/min/1.73 m 2 /year, indicating clinically meaningful early improvements in eGFR sustained through up to 5 years, in contrast to the expected typical age-related decline in eGFR in adults. vi Patients with baseline eGFR values < 60 mL/min/1.73 m 2 had a numerically greater increase in mean eGFR than those with a baseline eGFR ≥ 60 mL/min/1.73 m 2 . 24-hour urine calcium excretion normalized by Week 26 from mean 396 mg/day at baseline to mean 206 mg/day at Week 26 and further decreased from Week 26 to mean 170 mg/day at Week 52, with improvements sustained through the end of the trials. 98% of patients achieved independence from active vitamin D, defined as taking no calcitriol or alfacalcidol, at final trial visit. 95% of patients achieved independence from therapeutic doses of calcium, defined as taking < 600 mg/day of calcium, at final trial visit. Mean serum calcium increased within the normal range to 8.9 mg/dL, with 88% of patients having normal albumin-adjusted serum calcium levels at final trial visit. In both trials, TransCon PTH was generally well tolerated, with no new safety signals identified. Most treatment-emergent adverse events (TEAEs) were mild to moderate (Grade 1 or 2). The PaTH Forward and PaTHway trials included a combined total of 141 adults with hypoparathyroidism. The pooled kidney data analysis covers the 139 patients who received at least one dose of TransCon PTH. Of these, 129 completed the final trial visit. The PaTH Forward and PaTHway trials comprised randomized, double-blind, placebo-controlled periods through Weeks 4 and 26 and open-label extension periods through Weeks 266 and 182, respectively. An eGFR ≥30 mL/min/1.73 m 2 was required for trial eligibility. Mean (SD) baseline eGFR for the pooled analysis was 69 (17) mL/min/1.73 m 2 . The pooled data were analyzed post-hoc and included evaluation of the impact of TransCon PTH on renal function, as assessed by, change from baseline in eGFR and proportion of patients with an increase in eGFR ≥5 mL/min/1.73 m 2 , as well as 24-hour urinary calcium. About TransCon PTH TransCon PTH is a prodrug of PTH (1-34), administered once daily, designed to provide active PTH within the physiological range for 24 hours/day, approved as YORVIPATH ® in the United States, European Union, European Economic Area, and other jurisdictions as a treatment for adults with hypoparathyroidism. About Hypoparathyroidism Hypoparathyroidism is an endocrine disease caused by insufficient levels of parathyroid hormone (PTH), the primary regulator of calcium and phosphate balance in the body, acting directly on bone and kidney and indirectly on the intestine. Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment. Post-surgical hypoparathyroidism accounts for the majority of cases (70-80%), while other etiologies include autoimmune, idiopathic, and genetic causes, including ADH1. About Ascendis Pharma A/S Ascendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. 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 within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Examples of such statements include, but are not limited to, statements relating to (i) TransCon PTH (palopegteriparatide) data from the Phase 2 PaTH Forward and Phase 3 PaTHway trials, (ii) TransCon PTH’s ability to replicate the systemic and balanced actions of endogenous PTH across multiple target organ systems and to provide or restore the full range of effects of PTH that are missing in hypoparathyroidism, (iii) TransCon PTH’s differentiated profile, including its potential to show simultaneous improvements across key measures while enabling independence from conventional therapy, (iv) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients, and (v) Ascendis’ use of TransCon to develop new and potentially best-in-class therapies to address unmet medical needs. 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, without limitation: dependence on third-party manufacturers, distributors, and service providers for Ascendis’ products and product candidates; risks related to regulatory review and approval, including the possibility of delays, requests for additional data or analyses, restrictions or limitations on use, approval with labeling that is more limited than expected, or failure to obtain approval in the United States, European Union, or other jurisdictions; clinical development risks, including that results from ongoing or future trials may not confirm earlier data; unforeseen safety or efficacy findings in development programs or on-market products; manufacturing, supply chain, quality, or logistics issues that could delay development or commercialization; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen research and development or selling, general and administrative expenses and other costs impacting Ascendis’ business generally; market acceptance, pricing, and reimbursement challenges, including payer coverage decisions and health technology assessments; competitive developments, including new or improved therapies; intellectual property protection, freedom-to-operate, and litigation risks; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; cybersecurity, data privacy, and information technology disruptions; and the impact of international economic, political, legal, compliance, public health, and business factors, including tariffs, trade policies, currency fluctuations, and geopolitical events. 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 11, 2026, 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, and YORVIPATH ® are trademarks owned by the Ascendis Pharma group. © October 2026 Ascendis Pharma A/S. Investor Contact: Media Contact: Chad Fugere Melinda Baker Ascendis Pharma Ascendis Pharma +1 (650) 519-7494 +1 (650) 709-8875 __________________________________ i Khan AA, Bilezikian JP, Brandi ML, et al. J Bone Miner Res . 2022; 37(12):2568-2585. ii Holten-Andersen L, Pihl S, Rasmussen CE, et al. J Bone Miner Res . Nov 2019;34(11):2075-2086. doi:10.1002/jbmr.3824. iii Karpf DB, Pihl S, Mourya S, et al. J Bone Miner Res . Aug 2020;35(8):1430-1440. doi:10.1002/jbmr.4016. iv Mayne TJ, et al. Clin Transplant . 2021;35(7):e14326. v Ku E, et al. J Am Soc Nephrol . 2016;27(7):2196-204. vi Guppy M et al. BMJ Open. 2024;14(11):e089783. doi:10.1136/bmjopen-2024-089783.

临床结果临床2期上市批准

2026-10-06

·GlobeNewswire-Health Care

Ascendis to Share Latest Advances in Hypoparathyroidism and Achondroplasia Programs at ASBMR 2026

COPENHAGEN, Denmark, Oct. 06, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the latest advances in its hypoparathyroidism and achondroplasia programs will be showcased during ASBMR 2026, the annual meeting of American Society for Bone & Mineral Research being held in Boston from October 9-12, 2026. Updates include an oral presentation by Aliya Khan, M.D., of combined skeletal data from the Phase 2 PaTH Forward and Phase 3 PaTHway trials showing normalization of bone mass and microarchitecture in clinical trial patients treated with TransCon® PTH (palopegteriparatide) over a 5-year period. “Our growing body of clinical trial data continues to illustrate the unique potential for our differentiated TransCon-based therapies to improve patient health and quality of life,” said Aimee Shu, M.D., Executive Vice President, Chief Medical Officer at Ascendis Pharma. “Compelling long-term data such as these help inform treatment paradigms focused on outcomes that matter most to patients, and we look forward to sharing additional results as our programs advance.” Ascendis presentations at ASBMR 2026: ORAL PRESENTATIONHypoparathyroidismSunday, Oct. 11Session: 11:30a.m.-12:30p.m.Presentation: 12:18 PM – 12:30 Calcium Homeostasis DisordersBallroom A&BID #6080: Long-Term Effects of Palopegteriparatide on BMD and TBS in Adults With Chronic Hypoparathyroidism: Pooled Results from the PaTH Forward and PaTHway TrialsPresented by Aliya Khan, M.D.POSTERS HypoparathyroidismThursday, Oct. 85:30-6:30p.m.ASBMR/RBDA Pre-Meeting Symposium---Sunday, Oct. 112:00-3:00p.m.Poster Session IIID #6680: Hypoparathyroidism: Understanding the Impact of Palopegteriparatide on Patients’ Health Related Quality of Life, Functioning, and Well-Being Presented by Meryl Brod, Ph.D.AchondroplasiaFriday, Oct. 95:30-7:00p.m.Plenary Poster Session---Saturday, Oct. 102:00-3:30pm Poster Session 1ID 6329: Improved Growth and Physical Functioning in Children with Achondroplasia Treated with Navepegritide in the ApproaCH Trial Open-Label ExtensionPresented by Ciara McDonnell, M.D.Thursday, Oct. 85:30-6:30p.m.ASBMR/RBDA Pre-Meeting Symposium ---Friday, Oct. 95:30-7:00p.m.Plenary Poster Session---Saturday, Oct. 102:00-3:30pm Poster Session IID 6320: Continued Improvements in Lower Extremity Alignment in Navepegritide-Treated Children With Achondroplasia: Week 104 Results from the ApproaCH TrialPresented by Leanne Ward, M.D.Sunday, Oct. 112:00-3:00p.m.Late BreakingPoster Session IILate Breaker ID 8736: Navepegritide Combined with Lonapegsomatropin for the Treatment of Children with Achondroplasia: 78-Week Results from the Phase 2 COACH Trial Presented by Ciara McDonnell, M.D. About TransCon PTH TransCon PTH (palopegteriparatide) is a prodrug of PTH (1-34), administered once daily, designed to provide stable levels of active PTH within the physiological range for 24 hours/day. TransCon CNP is approved as YORVIPATH® in the United States (U.S.), European Union (EU), European Economic Area (EEA), and certain other jurisdictions as a treatment for adults with hypoparathyroidism. About TransCon CNPTransCon CNP (navepegritide) is a prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed to provide continuous exposure of active CNP to receptors on tissues throughout the body to counteract the overactive FGFR3 signaling in achondroplasia. TransCon CNP is approved as YUVIWEL® in the U.S. as a treatment for children with achondroplasia. Ascendis Pharma’s Marketing Authorisation Application for YUVIWEL is under review by the European Medicines Agency, with a decision anticipated in the fourth quarter of 2026. About TransCon hGHTransCon hGH (lonapegsomatropin) is a prodrug of somatropin administered once weekly, providing sustained release of active, unmodified somatropin. TransCon hGH is investigational in achondroplasia and is approved as SKYTROFA® in the U.S., EU, EEA, and elsewhere for the treatment of growth failure in children due to growth hormone deficiency and in the U.S. for the replacement of endogenous growth hormone in adults with growth hormone deficiency. About HypoparathyroidismHypoparathyroidism is an endocrine disease caused by insufficient levels of parathyroid hormone (PTH), the primary regulator of calcium and phosphate balance in the body, acting directly on bone and kidney and indirectly on the intestine. Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment. Post-surgical hypoparathyroidism accounts for the majority of cases (70-80%), while other etiologies include autoimmune, idiopathic, and genetic causes, including ADH1. About AchondroplasiaAchondroplasia is a rare genetic condition arising from a systemic fibroblast growth factor receptor 3 (FGFR3) variant that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways, estimated to affect more than 250,000 people worldwide. While historically considered a bone growth disorder, the FGFR3 variant seen in achondroplasia is expressed in tissues throughout the body, causing serious muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia. Medical complications of achondroplasia vary across different stages of life. Throughout infancy and childhood, observed complications include spinal abnormalities, enlarged brain ventricles, impaired muscle strength and stamina, hearing deficits and chronic ear infections, upper airway obstructions, sleep-disordered breathing, hip problems, leg bowing, and chronic pain; many of these persist or worsen in adulthood. These medical complications can affect physical well-being and quality of life, and may be impacted by a range of individual, clinical, and social factors. Some individuals with achondroplasia require multiple procedures and surgeries to address specific functional or anatomical concerns. About Ascendis Pharma A/SAscendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. 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 within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Examples of such statements include, but are not limited to, statements relating to (i) Ascendis’ planned oral presentation and posters at ASBMR 2026, (ii) the combined skeletal data from the Phase 2 PaTH Forward and Phase 3 PaTHway trials, including normalization of bone mass and microarchitecture in clinical trial patients treated with TransCon PTH (palopegteriparatide) over a 5-year period, (iii) the unique potential for Ascendis’ highly differentiated TransCon-based therapies to improve patient health and quality of life, (iv) the potential for long-term data to help inform treatment paradigms focused on outcomes that matter most to patients, (v) Ascendis’ plan to share additional results as its programs advance, (vi) Ascendis’ clinical development activities, including the ApproaCH Trial open-label extension and the Phase 2 COACH Trial of navepegritide combined with lonapegsomatropin, (vii) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients and (viii) Ascendis’ use of TransCon to create new and potentially best-in-class therapies to address unmet medical needs. 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, without limitation: dependence on third‑party manufacturers, distributors, and service providers for Ascendis’ products and product candidates; risks related to regulatory review and approval, including the possibility of delays, requests for additional data or analyses, restrictions or limitations on use, approval with labeling that is more limited than expected, or failure to obtain approval in the United States, European Union, or other jurisdictions; clinical development risks, including that results from ongoing or future trials may not confirm earlier data; unforeseen safety or efficacy findings in development programs or on‑market products; manufacturing, supply chain, quality, or logistics issues that could delay development or commercialization; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen research and development or selling, general and administrative expenses and other costs impacting Ascendis’ business generally; market acceptance, pricing, and reimbursement challenges, including payer coverage decisions and health technology assessments; competitive developments, including new or improved therapies; intellectual property protection, freedom‑to‑operate, and litigation risks; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; cybersecurity, data privacy, and information technology disruptions; and the impact of international economic, political, legal, compliance, public health, and business factors, including tariffs, trade policies, currency fluctuations, and geopolitical events. 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 11, 2026, 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®, YORVIPATH®, and YUVIWEL® are trademarks owned by the Ascendis Pharma group. © October 2026 Ascendis Pharma A/S. Investor Contact:Media Contact:Chad FugereMelinda BakerAscendis PharmaAscendis Pharma+1 (650) 519-7494+1 (650) 709-8875

临床2期上市批准临床结果临床3期

2026-10-06

·astrazeneca.com

Alexion to showcase Phase III data in hypophosphatasia and hypoparathyroidism at ASBMR 2026 Annual Meeting

Alexion, AstraZeneca Rare Disease, will deliver 16 presentations, including three oral presentations, across hypophosphatasia (HPP), chronic hypoparathyroidism (HypoPT) and early research at the American Society for Bone and Mineral Research (ASBMR) Annual Meeting in Boston, Massachusetts, 9 to 12 October 2026. Highlights include: HICKORY: results from Phase III trial of efzimfotase alfa (ALXN1850) in treatment-naïve adolescents and adults with HPP CALYPSO: data on skeletal parameters from Phase III trialevaluating eneboparatide, an investigational parathyroid hormone (PTH) 1 receptor agonist, in adults with HypoPT Gianluca Pirozzi, Senior Vice President, Head of Development, Regulatory and Safety, Alexion, said: “At this year’s ASBMR Annual Meeting, findings across 16 presentations will notably demonstrate the breadth of our research in HPP and HypoPT, two rare, multisystemic diseases that can have far-reaching impacts on patients’ daily lives. In HPP, results from HICKORY, the first Phase III clinical trial to include patients with adult-onset disease, will further reinforce the potential of efzimfotase alfa to reduce treatment burden and redefine care expectations, while real-world evidence will provide important insights into the impact of HPP. Together with new bone data from the CALYPSO Phase III trial of eneboparatide in HypoPT, our presence reflects our commitment to developing meaningful innovations that address the needs of patients and their families.” Alexion presentations during the 2026 ASBMR Annual Meeting Lead Author Abstract Title Presentation Details Lead Author HPP Lead Author Peroutka, C. Abstract Title Genetic Characterization and Clinical Manifestations in Adults with Hypophosphatasia in the United States Presentation Details Welcome Reception and Plenary Poster Session Abstract #FRI-465 9 October 2026 05:30 – 07:15 PM ET Poster Session I Abstract #SAT-465 10 October 2026 02:00 – 03:30 PM ET Lead Author Moss, K. Abstract Title Understanding Disease Burden and Characterizing Diagnosis, Assessment, and Management of Adults with Hypophosphatasia in the UK: A Delphi Study Presentation Details Late Breaking Poster Session I Abstract #SAT-551 10 October 2026 02:00 – 03:30 PM ET Lead Author Dahir, K. Abstract Title Assessing the compliance, usability, health insights, and perceived value from an at-home digital assessment toolkit for adults with hypophosphatasia: a multicenter observational study Presentation Details Poster Session I Abstract #SAT-489 10 October 2026 02:00 – 03:30 PM ET Lead Author Lynch, L. Abstract Title Treatment Experience in Adult Patients with Hypophosphatasia (HPP): Results of a US Survey Presentation Details Poster Session I Abstract #SAT-482 10 October 2026 02:00 – 03:30 PM ET Lead Author Ohata, Y. Abstract Title Clinical Course and Candidate Biomarkers in Perinatal Hypophosphatasia Presentation Details Poster Session I Abstract #SAT-483 10 October 2026 02:00 – 03:30 PM ET Lead Author Padidela, R. Abstract Title Diagnostic Delay and Clinical Burden in Hypophosphatasia: Results from a Cross-Sectional, Multinational, Mixed-Methods Study Presentation Details Poster Session I Abstract #SAT-481 10 October 2026 02:00 – 03:30 PM ET Lead Author Dhaliwal, R. Abstract Title A Real-World US Survey Study Characterizing the Holistic Disease Burden in Adults with Hypophosphatasia Presentation Details Poster Session II Abstract #SUN-480 11 October 2026 02:00 – 03:30 PM ET Lead Author Tanfous, M. Abstract Title Cascade Diagnosis of the Rare Metabolic Disease Hypophosphatasia (HPP) Within Families: A Case Series Presentation Details Poster Session II Abstract #SUN-492 11 October 2026 02:00 – 03:30 PM ET Lead Author Dahir, K. Abstract Title Efficacy and Safety of Alkaline Phosphatase (ALP) Enzyme Replacement Therapy (ERT) Efzimfotase Alfa in Adolescents and Adults with Hypophosphatasia (HPP): Results of HICKORY as Part of a Three-Trial Phase 3 Clinical Program Presentation Details Oral Presentation Abstract #1116 11 October 2026 05:00 – 05:12 PM ET Lead Author HypoPT Lead Author Vizcaya, D. Abstract Title Chronic Hypoparathyroidism in the US: Prevalence and Incidence From 2018 to 2024, With Baseline Clinical Characteristics Presentation Details Welcome Reception and Plenary Poster Session Abstract #FRI-005 9 October 2026 05:30 – 07:15 PM ET Poster Session I Abstract #SAT-005 10 October 2026 02:00 – 03:30 PM ET Lead Author Siggelkow, H. Abstract Title Leveraging Structured and Unstructured EHR Data to Improve Treatment and Clinical Context Capture in Chronic Hypoparathyroidism Presentation Details Poster Session I Abstract #SAT-015 10 October 2026 02:00 – 03:30 PM ET Lead Author Siggelkow, H. Abstract Title Bone Manifestations in Chronic Hypoparathyroidism: A Retrospective Cohort Study Presentation Details Poster Session I Abstract #SAT-019 10 October 2026 02:00 – 03:30 PM ET Lead Author Khan, A. Abstract Title Balanced Effects of Eneboparatide on Skeletal Parameters: Results From a Phase 3, Randomized, Placebo-Controlled Study Evaluating the Efficacy and Safety of Eneboparatide in Adults With Chronic Hypoparathyroidism (CALYPSO) Presentation Details Oral Presentation Abstract #1089 11 October 2026 12:06 – 12:18 PM ET Lead Author Erdmann, M. Abstract Title Methodological Differences Limit the Feasibility of an Indirect Treatment Comparison Between the Phase III CALYPSO (Eneboparatide) and PaTHway (Palopegteriparatide) Trials in Chronic Hypoparathyroidism Presentation Details Late Breaking Poster Session II Abstract #SUN-519 11 October 2026 02:00 – 03:30 PM ET Lead Author Vizcaya, D. Abstract Title Epidemiology of Low Bone Density and Osteoporosis in Chronic Hypoparathyroidism: Baseline Prevalence and Medication Usage from National US Claims Presentation Details Poster Session II Abstract #SUN-014 11 October 2026 02:00 – 03:30 PM ET Lead Author Early Research Lead Author Celen, I. Abstract Title Plasma Proteomics Distinguishes Clinical Expression in ADO and Highlights Differential Inflammatory and Matrix Remodeling Pathways Presentation Details Oral Presentation Abstract #1143 12 October 2026 12:30 – 12:42 PM ET Notes Alexion Alexion, AstraZeneca Rare Disease, is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more rare disease patients around the world. It is headquartered in Boston, US. AstraZeneca AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Disease, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca’s innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on social media @AstraZeneca. Contacts For details on how to contact the Investor Relations Team, please click here. For Media contacts, click here. Corporate and financial

临床3期

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
D12395	-	H05AA H05AA05	-

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	-
遗传性胰腺炎	临床3期	日本	Ascendis Pharma Bone Diseases A/S	2021-06-10
低钙血症	临床3期	日本	Ascendis Pharma Bone Diseases A/S	2021-06-10
甲状腺癌	临床3期	日本	Ascendis Pharma Bone Diseases A/S	2021-06-10

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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


PaTHway (ENDO2026)	临床3期	甲状旁腺功能减退症	82	Palopegteriparatide	醖簾願鹹築憲選淵構鹹(襯鹽範製鏇衊衊繭齋選) = 壓蓋構襯選鬱齋壓遞壓積衊鏇鹹餘築積餘遞獵 (觸膚鑰遞獵齋積廠鹽願 ) 更多	积极	2026-06-13
ENDO2026	N/A	甲状旁腺功能减退症	10	Palopegteriparatide	積範艱鹹廠簾窪願憲窪(構醖範鏇襯鏇願繭齋膚): MD = 9.0 (95.0% CI, 6.69 ~ 11.31) 更多	积极	2026-06-13
ENDO2026	N/A	甲状旁腺功能减退症	10	Placebo		积极	2026-06-13
WCO_IOF_ESCEO2026	N/A	甲状旁腺功能减退症	183	PTH (1-34)	襯構顧鹽鏇襯簾壓衊遞(窪觸鹹鏇簾糧鬱鏇製範) = 襯膚膚膚廠網夢鹹廠憲鹽獵積廠鏇夢繭糧壓繭 (窪鏇構觸齋鹽餘製膚淵 ) 更多	积极	2026-04-16
NCT04701203 (PaTHway, Company_Website) 人工标引	临床3期	甲状旁腺功能减退症	73	Palopegteriparatide	鹹夢餘築範範淵繭衊壓(範襯願夢蓋襯淵壓鬱鑰) = 壓獵遞憲膚壓遞鹹齋窪窪構範廠夢壓夢蓋網鏇 (觸糧鑰窪願醖觸衊淵簾 ) 更多	积极	2025-07-14
PaTHway (ENDO2025)	临床3期	甲状旁腺功能减退症	82	Palopegteriparatide	膚鑰鑰選選醖顧範構餘(膚糧艱簾夢餘鏇齋壓齋) = 範簾糧顧製獵醖遞淵積鬱觸憲選壓製網製築窪 (構鹹餘構壓獵窪醖壓鏇 ) 更多	积极	2025-07-12
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	積網簾齋鬱顧獵襯壓衊(淵鹹構淵觸艱築鹽獵齋) = 鏇艱蓋蓋選壓蓋鏇獵窪製醖觸願夢鹹簾窪選艱 (鬱鹹窪顧製壓觸醖積壓 ) 更多	积极	2025-05-18
path forward trial (ESPE2025)	临床2期	甲状旁腺功能减退症	59	Palopegteriparatide	範遞鬱壓餘憲顧積願範(蓋簾製鑰鹹鏇鹽襯觸襯) = 齋簾範顧艱糧淵築蓋繭艱築憲廠築齋鏇遞膚艱 (衊鏇鏇衊鏇積齋鑰醖糧 ) 更多	积极	2025-05-10
NCT04009291 (PaTH Forward, ESPE2025)	临床2期	甲状旁腺功能减退症	59	Palopegteriparatide	憲顧鏇觸選醖夢淵夢齋(獵觸醖鬱壓餘選築構艱) = 齋簾觸夢齋糧襯遞鹹夢積齋齋鏇齋繭範窪淵獵 (憲廠襯餘窪艱積憲構衊 ) 更多	积极	2025-05-10
ESPE2025	N/A	甲状旁腺功能减退症	40	TransCon PTH	壓鹹艱簾蓋鹽築鹹願窪(餘繭窪蓋壓鑰獵糧壓網) = From 34 patients documented, 38% (n = 13) reported headache, 38% (n = 13) muscular spasms or arthralgia, 24% sleep disturbance or fatigue, 21% (n = 7) nausea, 21% palpitations, 15% injections site reactions, and 15% other gastrointestinal symptoms (n = 5). Ten patients reported no negative symptoms. 艱獵鹽築淵餘構醖醖鹹 (壓醖膚艱夢鹹築艱鬱製 )	积极	2025-05-10
NCT04701203 (PaTHway, CTgov)	临床3期	甲状旁腺功能减退症	84	TransCon PTH (TransCon PTH)	餘襯築範顧獵簾製簾憲 = 繭齋廠網糧艱窪繭願鏇憲艱鬱蓋醖繭顧齋淵鏇 (憲衊網憲鹽範糧糧餘壓, 簾鹹襯顧壓鹹淵選構艱 ~ 艱壓夢衊積廠糧鹹醖餘) 更多	-	2025-02-28
NCT04701203 (PaTHway, CTgov)	临床3期	甲状旁腺功能减退症	84	Placebo (Placebo)	餘襯築範顧獵簾製簾憲 = 網選壓齋衊鬱艱艱鬱夢憲艱鬱蓋醖繭顧齋淵鏇 (憲衊網憲鹽範糧糧餘壓, 鬱衊襯襯壓壓憲製簾廠 ~ 顧鬱壓夢衊網鏇壓艱鹹) 更多	-	2025-02-28