RegulationOrphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Japan), Orphan Drug (Australia), Priority Review (Australia), Orphan Drug (United Kingdom), Priority Review (United States)

Structure/Sequence

Sequence Code 4953

Source: *****

Clinical Trials associated with Palopegteriparatide

NCT07706764

/ RecruitingPhase 3

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.

Start Date22 Apr 2026

Sponsor / Collaborator

Ascendis Pharma A/S

NCT07081997

/ RecruitingPhase 3

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.

Start Date16 Apr 2026

Sponsor / Collaborator

Ascendis Pharma Bone Diseases A/S

NCT07264634

/ RecruitingNot Applicable

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.

Start Date19 Mar 2026

Sponsor / Collaborator

Ascendis Pharma A/S

100 Clinical Results associated with Palopegteriparatide

100 Translational Medicine associated with Palopegteriparatide

100 Patents (Medical) associated with Palopegteriparatide

6,504

Literatures (Medical) associated with Palopegteriparatide

01 Dec 2026·IBRO Neuroscience Reports

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

Article

Author: 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.

01 Oct 2026·BONE

PTH treatment is effective in the Aga2/+ mouse model of moderate to severe osteogenesis imperfecta

Article

Author: Zieba, Jennifer ; Kot, Alexander ; Bagheri, Roya ; Krakow, Deborah ; Wight, Caroline ; Wachtell, Davis

Osteogenesis imperfecta (OI) is a genetically heterogenous disorder characterized by brittle bone and recurrent fractures. The majority of OI is clinically categorized into mild, moderate, severe, and perinatal lethal forms. A clinical study on the use of intermittent parathyroid hormone (PTH) as an anabolic therapy for OI found PTH was effective in mild, but not in individuals with moderate/severe OI. However, this sub-group analysis was relatively small and considered preliminary by the authors. Since then, research on PTH therapy in OI has focused on mild OI and no studies of PTH use in moderate/severe OI patients or animal models have been reported. In this work, we used the Aga2/+ mouse, an established autosomal dominant model of moderate/severe OI, to investigate whether PTH treatment has an effect on bone. In vitro, Aga2/+ osteoblasts were sensitive to PTH and exhibited similar transcriptional changes compared to WT. In vivo, PTH treatment improved lumbar trabecular bone, increased cortical bone area fraction in the mid-femur, and, in females, led to improved biomechanical properties. These results demonstrate the Aga2/+ model responded to PTH and support further investigation into whether PTH is effective in adult individuals with moderate/severe OI who have limited treatment options.

01 Oct 2026·Journal of Stomatology Oral and Maxillofacial Surgery

Synergistic effects of intermittent parathyroid hormone (1–34) and masticatory force for alveolar bone remodeling in the maxilla of dogs

Article

Author: Kyoung, Do-Kyoung ; Kim, Min-Ji ; Kim, Jin-Woo ; Moon, Ho-Jin ; Kim, Ji-Yeon ; Kim, Se-Hyang

INTRODUCTION:

Parathyroid hormone (PTH) is a key regulator of calcium homeostasis and bone remodeling. While intermittent PTH (1-34) (teriparatide) is an established treatment for osteoporosis, its effects on alveolar bone regeneration remain unclear. This study evaluated intermittent PTH (1-34) impact on the microstructure and remodeling of canine maxillary alveolar bone under physiological masticatory force.

METHODS:

In this exploratory pilot study, three male mongrel dogs were each assigned to a single dose group (n = 1 per group) and injected weekly for 20 weeks with vehicle (CON), 1.6 μg/kg PTH (PTH1.6), or 3.2 μg/kg PTH (PTH3.2). Bone microarchitecture and remodeling were assessed using micro-computed tomography (micro-CT), fluorescence labeling, and hematoxylin and eosin (H&E) staining.

RESULTS:

Micro-CT revealed bone mass and density increased with higher occlusal force in all groups. Trabecular bone volume and mineral density were higher in PTH1.6 than in controls, whereas PTH3.2 values were lower than controls; given n = 1 per group, these differences are reported as individual-animal observations without statistical inference. Fluorescence labeling demonstrated the greatest new bone formation in PTH1.6. H&E staining showed no apparent differences in osteoclast or osteoblast numbers among those groups.

CONCLUSION:

In this pilot large-animal model, moderate-dose intermittent PTH (1-34) administration was associated with greater alveolar anabolic response than higher-dose administration, and the magnitude of response appeared to increase with regional masticatory loading. These hypothesis-generating observations warrant confirmatory studies in appropriately powered cohorts. Optimizing PTH dosing and considering mechanical loading is critical for effective alveolar bone regeneration in the dental medicine scenarios.

144

News (Medical) associated with Palopegteriparatide

15 Jun 2026

·www.biospace.com

Phase 3 Data Show TransCon® PTH Replicated Systemic Actions of Endogenous PTH Through Week 182 in Adults with Hypoparathyroidism

Multi-organ system and quality-of-life benefits sustained through three and a half years 86% response rate for the multi-component endpoint 89% of patients completed the three-and-a-half-year trial COPENHAGEN, Denmark, June 13, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced Week 182 data from its completed Phase 3 PaTHway Trial showing that long-term treatment with TransCon PTH (palopegteriparatide) demonstrated sustained efficacy and safety in adults with hypoparathyroidism. Over the three-and-a-half-year duration of the trial, TransCon PTH replicated the systemic actions of endogenous PTH, with a balanced, beneficial impact on the main target organ systems – CNS, kidney, small intestine, and bone – as demonstrated by improved quality of life and normalized and stable urine calcium, serum calcium, serum phosphate, and bone mineral density. These benefits were sustained while enabling independence from conventional therapy with active vitamin D and calcium. “With its unique ability to replicate the systemic actions of endogenous parathyroid hormone, TransCon PTH has successfully addressed the physical and psychological burdens of hypoparathyroidism for the majority of treated patients,” said Aliya Khan, M.D., Clinical Professor of Medicine, Division of Endocrinology & Geriatrics, and Director of the Calcium Disorders Clinic at McMaster University in Canada. “These Phase 3 data reinforce the rapid and sustained benefits seen throughout clinical trials of TransCon PTH, including compelling improvements in quality of life reported by patients previously limited by the fatigue, cognitive challenges, and reduced physical functioning and well-being that are the hallmarks of this disease.” Highlights of Week 182 Results from the Phase 3 PaTHway Trial 86% of patients were responders for the multi-component endpoint of (1) serum calcium in the normal range, (2) taking no active vitamin D, and (3) taking ≤600 mg/day of calcium. 89% of patients had normal albumin-adjusted serum calcium levels and a mean value of 8.8 mg/dL. 100% of patients achieved independence from active vitamin D, defined as not taking calcitriol or alfacalcidol. 96% of patients achieved independence from therapeutic doses of calcium, defined as taking < 600 mg/day of calcium. Significant improvements in kidney function were maintained, with mean (SE) eGFR of 80.2 (1.8) mL/min/1.73 m 2 at Week 182, reflecting a mean (SE) increase of 11.0 (1.4) mL/min/1.73 m 2 from baseline. Among patients randomized to TransCon PTH, eGFR increased from baseline through Week 38 and stabilized thereafter. After initiation of open-label treatment at Week 26, patients who had been receiving placebo in the double-blind period experienced a similar increase in eGFR. Following these eGFR increases, mean eGFR values were maintained through Week 182, in contrast to the expected typical age-related decline in eGFR in adults. i Mean 24-hour urine calcium decreased substantially, normalized within 26 weeks, and remained normal through Week 182. As measured by Hypoparathyroidism Patient Experience Scales (HPES), patients reported improvements in symptoms and health-related quality of life across all domains. Hypoparathyroidism-related physical and cognitive symptoms and impacts on physical functioning and daily life improved rapidly with TransCon PTH treatment and were maintained through Week 182. As measured by SF-36, all subscale scores and component summary scores demonstrated rapid and clinically meaningful improvements with TransCon PTH treatment which were sustained through Week 182. Mean BMD Z-scores (matched for age and sex) corrected from high baseline levels through Week 26 and remained above 0 through Week 182. In the trial, TransCon PTH treatment was generally well-tolerated, with no new safety signals identified. Treatment-emergent adverse events (AEs) were mostly mild or moderate, and no discontinuations were related to study drug. Over three and a half years of treatment, no patients developed anti-PTH antibodies. “Regardless of disease origin, TransCon PTH has normalized key biochemistries and skeletal health while significantly improving kidney function and quality of life beginning at the earliest timepoints and continuing through multiple years of treatment,” said Aimee Shu, M.D., Executive Vice President, Chief Medical Officer at Ascendis Pharma. “We remain committed to continuing our work to advance treatment options for patients around the world living with this often-debilitating chronic disease.” The PaTHway Trial of 82 adults with hypoparathyroidism (85% post-surgical, 15% non-surgical) included a 26-week randomized, double-blind, placebo-controlled period followed by a 156-week open-label extension (OLE) period, and measured a wide array of clinical, biochemical, and quality of life endpoints, consistent with the breadth of negative long-term impacts experienced by patients with hypoparathyroidism. Seventy-three of the original 82 patients enrolled (89%) completed the three-and-a-half-year trial. Endpoints included independence from conventional therapy (defined as < 600 mg/day of calcium and no active vitamin D) and maintenance of normocalcemia (8.3 to 10.6 mg/dL). Renal function was assessed by estimated glomerular filtration rate (eGFR). Bone mineral density (BMD) measured by DXA scan was assessed at baseline and regular intervals through Week 182. Hypoparathyroidism-related symptoms and functional impacts were measured using the HPES. Health-related quality of life was measured using the 36-Item Short Form Survey (SF-36 version 2). Safety assessments included treatment-emergent AEs and 24-hour urine calcium excretion. TransCon PTH 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, approved as YORVIPATH ® in the United States, European Union, European Economic Area, and certain other jurisdictions as a treatment for adults with hypoparathyroidism. A slide presentation with these data will be made available on the Investor Relations & News section of the Ascendis Pharma website: . 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’s ability to replicate endogenous parathyroid hormone and address the physical and psychological burdens of hypoparathyroidism, (ii) Ascendis’ commitment to continuing its work to advance treatment options for patients around the world with hypoparathyroidism, (iii) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients and (iv) 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, TransCon, and YORVIPATH ® are trademarks owned by the Ascendis Pharma group. © June 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 Guppy M et al. BMJ Open . 2024;14(11):e089783. doi:10.1136/bmjopen-2024-089783

Clinical ResultPhase 3

12 Jun 2026

·www.biospace.com

5-Year Phase 2 Data Show TransCon® PTH Replicated Systemic Actions of Endogenous PTH in Adults with Hypoparathyroidism

– Multi-organ system and quality-of-life benefits sustained through five years of treatment – 82% response rate for the multi-component endpoint – 95% of patients completed five years of treatment COPENHAGEN, Denmark, June 11, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced 5-year (Week 266) data from its Phase 2 PaTH Forward Trial showing that long-term treatment with TransCon PTH (palopegteriparatide) demonstrated sustained efficacy and safety in adults with hypoparathyroidism. Over the five-year duration of the trial, TransCon PTH replicated the systemic actions of endogenous PTH, with a balanced, beneficial impact on the main target organ systems – kidney, small intestine, CNS, and bone – as demonstrated by normalized and stable urine calcium, serum calcium, quality of life, and bone mineral density. These benefits were sustained while enabling independence from conventional therapy with active vitamin D and calcium. “Moving from symptom management to addressing the underlying hormone deficiency requires normalization of PTH biology to mitigate the multi-organ impacts of hypoparathyroidism. TransCon PTH has achieved this, meeting the high bar for the treatment of chronic hypoparathyroidism,” said Andrea Palermo, M.D., Ph.D., an endocrinologist at Campus Bio-Medico University (Rome), who presented the five-year data during the European Congress of Endocrinology (ECE) 2026. “These data demonstrate the consistent, long-term benefits of this therapy and reinforce its potential as the emerging standard of care for the treatment of hypoparathyroidism.” Highlights of Week 266 Results from the Phase 2 PaTH Forward Trial 82% of patients were responders for the multi-component endpoint of (1) serum calcium in the normal range, (2) taking no active vitamin D, and (3) taking < 600 mg/day of calcium. 88% of patients had normal albumin-adjusted serum calcium levels, with a mean value of 9.0 mg/dL. 96% of patients achieved independence from active vitamin D, defined as not taking calcitriol or alfacalcidol. 95% of patients achieved independence from therapeutic doses of calcium, defined as taking < 600 mg/day of calcium. Significant improvements in kidney function were maintained, with mean (SE) eGFR of 78.0 (3.0) mL/min/1.73 m 2 at Week 266, reflecting a mean (SE) increase of 9.4 (1.9) mL/min/1.73 m 2 from baseline. Improvements were evident as early as Week 4, increased through Week 58, and were sustained over five years of treatment, in contrast to the expected normal age-related decline in eGFR in adults. i Mean 24-hour urine calcium decreased substantially, normalized within 26 weeks, and remained normal through Week 266. As measured by Hypoparathyroidism Patient Experience Scales (HPES), patients reported improvements in symptoms and health-related quality of life across all domains. Hypoparathyroidism-related physical and cognitive symptoms and impacts on physical functioning and daily life improved rapidly with TransCon PTH treatment and were maintained through Week 266. As measured by SF-36, all mean health-related quality of life subscale and component summary scores rapidly normalized with TransCon PTH treatment and remained in the normative range through Week 266. Mean BMD Z-scores (matched for age and sex) corrected from high baseline levels through Week 26 and remained above 0 through Week 266. In the trial, TransCon PTH treatment was generally well-tolerated, with no new safety signals identified. Treatment-emergent adverse events (AEs) were mostly mild or moderate, and no discontinuations were related to study drug. One patient developed transient, low-titer and non-neutralizing anti-PTH antibodies, with no impact on safety or efficacy. Over five years of treatment, no other patients developed anti-PTH antibodies. “Across clinical trials and etiologies, TransCon PTH has shown a unique ability to replicate the actions of endogenous PTH, normalizing biochemistries and skeletal health and significantly improving kidney function and quality of life,” said Aimee Shu, M.D., Executive Vice President, Chief Medical Officer at Ascendis Pharma. “We are pleased to see TransCon PTH working as designed to transform treatment for patients living with this often-debilitating chronic disease.” The PaTH Forward Trial of 59 adults with hypoparathyroidism (80% post-surgical, 20% non-surgical) included a 4-week randomized, double-blind, placebo-controlled period followed by a 262-week open-label extension (OLE) period, and measured a wide array of clinical, biochemical, and quality of life endpoints, consistent with the breadth of negative long-term impacts experienced by patients with hypoparathyroidism. Fifty-six of the original 59 patients enrolled (95%) completed the five-year trial. Endpoints included independence from conventional therapy (defined as < 600 mg/day of calcium and no active vitamin D) and maintenance of normocalcemia (8.3 to 10.6 mg/dL). Renal function was assessed by estimated glomerular filtration rate (eGFR). Bone mineral density (BMD) measured by DXA scan was assessed at baseline and regular intervals through Week 266. Hypoparathyroidism-related symptoms and functional impacts were measured using the HPES. Health-related quality of life was measured using the 36-Item Short Form Survey (SF-36 version 2). Safety assessments included treatment-emergent AEs and 24-hour urine calcium excretion. TransCon PTH 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, approved as YORVIPATH ® in the United States, European Union, European Economic Area, and certain other jurisdictions as a treatment for adults with hypoparathyroidism. A slide presentation with these data will be made available on the Investor Relations & News section of the Ascendis Pharma website: . 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) the consistent, long-term benefits of TransCon PTH and its potential as the emerging standard of care for the treatment of hypoparathyroidism, (ii) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients and (iii) 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, TransCon, and YORVIPATH ® are trademarks owned by the Ascendis Pharma group. © June 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 Guppy M et al. BMJ Open . 2024;14(11):e089783. doi:10.1136/bmjopen-2024-089783

09 Jun 2026

·www.biospace.com

Ascendis to Showcase Advances in Treatment of Rare Endocrine Diseases at ENDO 2026

COPENHAGEN, Denmark, June 08, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the latest data from its Endocrinology Rare Disease programs in hypoparathyroidism, achondroplasia, and pediatric growth hormone deficiency will be shared in two oral presentations and three posters at ENDO 2026, the annual meeting of the Endocrine Society being held in Chicago from June 13-16, 2026. “As a leader in rare endocrine diseases, we are committed to sharing comprehensive clinical trial results in peer-reviewed forums that reinforce the transformative potential of our innovative therapies,” said Aimee Shu, M.D., Executive Vice President, Chief Medical Officer at Ascendis Pharma. “This year at ENDO, we look forward to showcasing the long-term safety and efficacy of TransCon PTH, demonstrated through multiple clinical trials, as well as data demonstrating the long-term benefits of TransCon CNP alone and in combination with TransCon hGH in children with achondroplasia.” Ascendis presentations at ENDO 2026 include: ORAL PRESENTATIONS Saturday June 13 2:45-3:00pm CT W187 Hypoparathyroidism Abstract 2312489 Session: Bone & Mineral Metabolism: Parathyroid Disease ORF16 Palopegteriparatide Treatment in Adults with Hypoparathyroidism: Final Results of the Phase 3 PaTHway Trial Presented by Aliya Khan, M.D. Monday June 15 2:30–2:45pm CT W187 Achondroplasia Abstract 2317908 Session: Growth & Puberty ORF47 Navepegritide Combined with Lonapegsomatropin for the Treatment of Children with Achondroplasia: 52-Week Results from the Phase 2 COACH Trial Presented by Ciara McDonnell, M.D. POSTERS Sunday June 14 9am-4pm CT Poster Hall Hypoparathyroidism Poster SUN-770 (Abstract 2311239) Palopegteriparatide Enhances Work Functioning and Productivity in Employed Adults with Chronic Hypoparathyroidism: 156-Week Results from the PaTHway Trial Authors: Wahid Noori et al Sunday June 14 9am-4pm CT Poster Hall Hypoparathyroidism Poster SUN-769 (Abstract 2312938) Global Prevalence Estimates for Post-Surgical and Non-Surgical Chronic Hypoparathyroidism (HypoPT): A Systematic Literature Review and Meta-Analysis Authors: Subhara Raveendran et al Sunday June 14 9am-4pm CT Poster Hall Pediatric Growth Hormone Deficiency Poster SUN-201 (Abstract 2328928) Safety and Effectiveness of Lonapegsomatropin in the SkybriGHt Registry: Analysis of the First 100 Patients Enrolled With 2 Years of Follow-Up Authors: Aristides Maniatis, M.D. et al 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 Achondroplasia Achondroplasia 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, and is associated with an increased risk of muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia. Medical complications of achondroplasia can vary from individual to individual and across different stages of life. Throughout infancy and childhood, observed complications include spinal abnormalities, enlarged brain ventricles, impaired muscle strength and reduced stamina, hearing deficits and chronic ear infections, upper airway obstructions, sleep-disordered breathing, hip problems, leg bowing, and chronic pain; some of which 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 Pediatric Growth Hormone Deficiency Growth hormone plays an essential role in the health of children and adults, promoting growth in children and maintenance of healthy body composition, cardiovascular function, and metabolism at all ages. Inadequate secretion of growth hormone from the anterior pituitary gland leads to impaired growth and function in many organs and tissues of the body where growth hormone is active, including liver, muscle, fat, and bone. Pediatric growth hormone deficiency (pediatric GHD), may be present at birth (congenital GHD) or may develop at any time during infancy, childhood, or adolescence, with the most visible sign being a noticeable slowing of growth; it may be isolated or occur with multiple pituitary hormone deficiencies. 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) Ascendis’ planned presentations and posters at ENDO 2026, (ii) Ascendis’ commitment to sharing comprehensive clinical trial results in peer-reviewed forums that reinforce the transformative potential of its innovative therapies, (iii) the long-term safety and efficacy of TransCon PTH and the long-term benefits of TransCon CNP alone and in combination with TransCon hGH in children with achondroplasia, (iv) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients, and (v) Ascendis’ ability to apply TransCon to develop new therapies that demonstrate best-in-class potential 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, and TransCon are trademarks owned by the Ascendis Pharma group. © June 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

Phase 2Phase 3

100 Deals associated with Palopegteriparatide

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R&D Status

Approved

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Indication	Country/Location	Organization	Date
Hypoparathyroidism	European Union	Ascendis Pharma Bone Diseases A/S	17 Nov 2023
Hypoparathyroidism	Iceland	Ascendis Pharma Bone Diseases A/S	17 Nov 2023
Hypoparathyroidism	Liechtenstein	Ascendis Pharma Bone Diseases A/S	17 Nov 2023
Hypoparathyroidism	Norway	Ascendis Pharma Bone Diseases A/S	17 Nov 2023

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Achondroplasia	NDA/BLA	United States	Ascendis Pharma A/S	-
Achondroplasia	NDA/BLA	European Union	Ascendis Pharma A/S	-

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04701203 (PaTHway, Company_Website) Manual	Phase 3	Hypoparathyroidism	73	Palopegteriparatide	wifpqttqyv(jjigdndonn) = oqknhtqnay bsbzsmibiw (hgoqtwffxg ) View more	Positive	14 Jul 2025
NCT04009291 (PaTH Forward, NEWS \| Corporate Publications) Manual	Phase 2	Hypoparathyroidism	56	Palopegteriparatide	luqjmmqkee(stxnsogfrg) = kyxxtzzrcz nuhfuzkxxt (lvkikhofgi ) View more	Positive	18 May 2025
NCT04701203 (PaTHway, CTgov)	Phase 3	Hypoparathyroidism	84	TransCon PTH (TransCon PTH)	cdmeibfxfu = huaksvesqp lazggpmdpk (owdkpkgikb, awpkhfclyd - mdebmddxry) View more	-	28 Feb 2025
				Placebo (Placebo)	cdmeibfxfu = yhptdziprq lazggpmdpk (owdkpkgikb, wwjbfilecy - yduegmuuwy) View more
NCT05387070 (PaTHway, PRNewswire) Manual	Phase 3	Hypoparathyroidism	-	Palopegteriparatide	azttjlqyrj(ltxqrgopdp) = okxsjsuyri zgzspehann (muwcbxyvfx ) View more	Positive	12 Aug 2024
				Placebo	azttjlqyrj(ltxqrgopdp) = zrndgvynrz zgzspehann (muwcbxyvfx )
NCT04701203 (FDA_CDER) Manual	Phase 3	Hypoparathyroidism	82	YORVIPATH	mdiyqwbiro(sklurolgiu) = eirokpltxy dsocizfjra (tesyhpavgq ) View more	Positive	09 Aug 2024
				Placebo	mdiyqwbiro(sklurolgiu) = kfppfmyfoy dsocizfjra (tesyhpavgq ) View more
NCT04701203 (PaTHway, Pubmed \| Adv Ther)	Phase 3	Hypoparathyroidism	82	Palopegteriparatide	gzyihkatsx(nxnfagjguk) = One case of nephrolithiasis was reported for a participant in the placebo group during blinded treatment; none were reported through week 52 with palopegteriparatide hpwdvrtlnb (mjcovkjfrp )	Positive	01 Jun 2024
NCT04701203 (PaTHway, GlobeNewswire) Manual	Phase 3	Hypoparathyroidism	82	TransCon PTH (palopegteriparatide)	qaiqtcllyn(syjdrckiuz) = bezzwqgijw xryeiesrcl (dympdnczxs ) View more	Positive	13 May 2024
				TransCon PTH (palopegteriparatide) (eGFR < 60 mL/min/1.73m2)	qaiqtcllyn(syjdrckiuz) = utyjnouxrm xryeiesrcl (dympdnczxs ) View more
NCT04009291 \| NCT04701203 \| NCT05387070 (PaTHway, ENDO2023)	Phase 3	Hypoparathyroidism	82	TransCon PTH	foqvnvqesk(wjkvsyoyix) = trended toward norms from baseline to Weeks 26 and 52 kdctocsthp (wkndxcgvvg ) View more	Positive	05 Oct 2023
NCT04009291 (PaTHway \| PaTH Forward, CTgov)	Phase 2	Hypoparathyroidism	59	Placebo for TransCon PTH (Placebo)	raefvrtmrj = ytmkimnapv ngxhfuljon (bfbiqmkzcm, njuliuiujb - czbvlwubdj) View more	-	01 Sep 2023
				TransCon PTH (Double Blind: TransCon PTH 15 mcg)	hkjwfcooef(petjxahkky) = fdarlirsqa pmukpmeqgi (fduflpyhui, sfzfseflmq - hzxhlstuee) View more
PaTH Forward Trial (ENDO2022)	Phase 2	Hypoparathyroidism	59	TransCon PTH 15 mcg/d	mpdwueqlhr(bqaogkcxvc) = hzaqpwrzfu vzyzbslfwj (eouyskcere ) View more	Positive	01 Nov 2022
				TransCon PTH 18 mcg/d	mpdwueqlhr(bqaogkcxvc) = mivmpfkbet vzyzbslfwj (eouyskcere ) View more

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