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

NCT07081997

/ Not yet recruitingPhase 3

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 ?g/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 Date01 Nov 2025

Sponsor / Collaborator

Ascendis Pharma Bone Diseases A/S

JPRN-jRCT2051210058

/ Not yet recruitingPhase 3IIT

A Phase 3, Multicenter, Single Arm, Open-Label Trial, Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Subcutaneously Daily in Japanese Adult Subjects with Hypoparathyroidism - PaTHway Japan Trial

Start Date02 Nov 2021

Sponsor / Collaborator-

NCT05387070

/ RecruitingPhase 3

PaTHway CHINA TRIAL: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Trial, With an Open-Label Extension, Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Subcutaneously Daily in Adults With Hypoparathyroidism

This study is limited to conduct in China only. The primary objective is to assess the treatment effect of daily TransCon PTH on serum calcium (sCa) levels within the normal range and stopping from therapeutic doses of active vitamin D (calcitriol) or active vitamin D analogue (alfacalcidol) and calcium at 26 weeks of treatment. All subjects will start with 18 mcg of study drug and will be individually and progressively titrated to an optimal dose over a 26-week double blind period, followed by an open label extension period up to 156 weeks. TransCon PTH or placebo will be administered as a subcutaneous injection using a pre-filled injection pen. Neither trial participants nor their doctors will know who has been assigned to each group. After the 26 weeks, participants will continue in the trial as part of a long-term extension study. During the extension, all participants will receive TransCon PTH, with the dose adjusted to their individual needs.

Start Date28 Jul 2021

Sponsor / Collaborator

VISEN Pharmaceuticals (Shanghai) Co., Ltd.

100 Clinical Results associated with Palopegteriparatide

100 Translational Medicine associated with Palopegteriparatide

100 Patents (Medical) associated with Palopegteriparatide

7,597

Literatures (Medical) associated with Palopegteriparatide

31 Dec 2025·RENAL FAILURE

Persistent hyperparathyroidism after kidney transplantation in children

Article

Author: Lee, Hyun Kyung ; Ha, Jongwon ; Kang, Hee Gyung ; Han, Ahram ; Min, Sangil ; Ahn, Yo Han ; Park, Peong Gang

BACKGROUND:

Persistent hyperparathyroidism after kidney transplantation (KT) has been reported in up to 50% of adult recipients, but pediatric data remain limited. We evaluated the prevalence, skeletal manifestations, and risk factors for persistent hyperparathyroidism in children following KT.

METHODS:

In this retrospective cohort study, 107 pediatric KT recipients (58% male; median age 10.3 years) transplanted between 2004 and 2019 were analyzed. Persistent hyperparathyroidism was defined as a median parathyroid hormone (PTH) > 65 pg/mL between 3 and 12 months post-KT. Risk factors for persistent hyperparathyroidism, post KT clinical features, and treatment status were analyzed.

RESULTS:

Thirty-six patients (33.6%) had persistent hyperparathyroidism after KT. On univariable analysis, dialysis duration of 24 months or longer (p = 0.028) and pretransplant hyperphosphatemia (p = 0.026) were significantly associated with persistent hyperparathyroidism. The multivariable model identified pretransplant hyperphosphatemia as an independent predictor (OR 2.70, 95% CI 1.10-6.87; p = 0.030). There was no significant difference in height Z score change between patients with and without persistent hyperparathyroidism (p = 0.97). However, persistent hyperparathyroidism was associated with poorer graft survival (log-rank p = 0.049). Six patients received cinacalcet and one underwent subtotal parathyroidectomy for refractory hypercalcemia.

CONCLUSIONS:

Persistent hyperparathyroidism is relatively common in pediatric KT recipients, affecting one-third of patients by one-year post-transplant. Prolonged dialysis and pre-existing hyperphosphatemia before KT may be risk factors. These findings underscore the importance of optimizing chronic kidney disease-mineral bone disease management and routine PTH monitoring before and after transplant in children.

31 Dec 2025·CLINICAL AND EXPERIMENTAL HYPERTENSION

Parathyroid hormone and vitamin D modulate nocturnal blood pressure dipping: a retrospective cohort study in primary hypertension

Article

Author: Liang, Xiaohui ; Xu, Yixin ; Deng, Changjiang

BACKGROUND:

Non-dipper hypertension, defined by reduced nocturnal blood pressure (BP) decline, increases cardiovascular risk. Calcium-phosphate metabolism (parathyroid hormone [PTH], vitamin D [25(OH)D]) may influence circadian BP rhythms, but their independent roles remain unclear.

OBJECTIVE:

To investigate associations between PTH, 25(OH)D, and nocturnal BP dipping in primary hypertension, addressing prior inconsistencies.

METHODS:

This retrospective cohort included 585 hypertensive adults stratified by nocturnal systolic BP dipping (dippers [≥10% decline, n = 250]; non-dippers [<10%, n = 335]) using 24-hour ambulatory monitoring. Serum PTH, 25(OH)D, renal/metabolic parameters, and vascular indices were analyzed. Multivariate linear regression assessed associations with dipping, adjusted for age, sex, renal function, and arterial stiffness.

RESULTS:

Non-dippers showed elevated PTH (median: 5.23 vs. 4.70 pmol/L, p = .011) and lower 25(OH)D (30.89 vs. 36.79 nmol/L, p < .001). Adjusted models confirmed PTH (β=-0.21, 95% CI:-0.34--0.08; p = .002) and 25(OH)D (β = 0.03, 95% CI:0.01-0.04; p < .001) as associated factors of dipping. Although statistically significant, these correlations were modest and suggested possible trends rather than strong causal relationships. Age/sex effects on BP patterns became nonsignificant after adjusting for calcium-phosphate markers.

CONCLUSION:

Calcium-phosphate dysregulation (elevated PTH, low vitamin D) independently contributes to non-dipping, suggesting biomarker-guided approaches for circadian BP control. Demographic disparities in dipping may reflect underlying mineral metabolism disturbances. Prospective trials should explore calcium-modulating therapies (e.g. vitamin D supplementation) in non-dipper hypertension.

01 Dec 2025·Current Pain and Headache Reports

Functional Magnetic Resonance Imaging of Post-Traumatic Headache: A Systematic Review

Review

Author: Christensen, Rune H ; Ashina, Håkan ; Szabo, Edina ; Al-Khazali, Haidar M ; Iljazi, Afrim

PURPOSE OF REVIEW:

To evaluate existing functional magnetic resonance imaging (fMRI) studies on post-traumatic headache (PTH) following traumatic brain injury (TBI).

RECENT FINDINGS:

We conducted a systematic search of PubMed and Embase databases from inception to February 1, 2024. Eligible fMRI studies were required to include adult participants diagnosed with acute or persistent PTH post-TBI in accordance with any edition of the International Classification of Headache Disorders. We identified five eligible fMRI studies: two on acute PTH and three on persistent PTH. These studies assessed resting-state functional connectivity involving comparisons with one or more of the following groups: people with migraine, those with mild TBI but no PTH, and healthy controls. In acute PTH, studies focused exclusively on functional connectivity between the periaqueductal gray or hypothalamus and other brain regions. In persistent PTH, evidence of altered functional connectivity was identified primarily within cingulate, sensorimotor, and visual regions, indicating a hypersensitivity to sensory stimuli in PTH. Despite these insights, the fMRI data remains sparse and is limited by inconsistent results and small samples. The paucity of fMRI studies on PTH limits our understanding of its neurobiological basis. The available evidence suggests that alterations in functional connectivity occur within brain areas involved in emotional and sensory discriminative aspects of pain processing. However, inconsistent results and small sample sizes underscore a critical need for larger, more rigorous fMRI studies. Future studies should also consider using task-based fMRI to investigate possible hypersensitivity to different sensory stimuli in PTH after TBI.

126

News (Medical) associated with Palopegteriparatide

29 May 2025

·www.prnewswire.com

NEW THERAPY FOR CHILDREN WITH GROWTH HORMONE DEFICIENCY APPROVED IN AUSTRALIA

SKYTROFA® (lonapegsomatropin) is now registered by the Therapeutic Goods Administration (TGA) for the treatment of paediatric growth hormone deficiency.[1] SINGAPORE, May 29, 2025 /PRNewswire/ -- Independent biopharmaceutical company Specialised Therapeutics (ST) is pleased to announce the Australian registration of SKYTROFA® (lonapegsomatropin) as a new once-weekly injectable therapy for paediatric growth hormone deficiency (GHD).[2] The Therapeutic Goods Administration (TGA) has approved SKYTROFA as a treatment for "growth failure in children and adolescents aged from 3 years up to 18 years due to insufficient endogenous growth hormone secretion".[1,2] GHD is a rare disease affecting around 2-3/10,000 Australians, with approximately 2,000 children thought to be living with the condition.[3] It occurs when the pituitary gland, located at the base of the brain, does not produce an adequate level of growth hormone, which is essential for promoting healthy growth in children.[3] In children, GHD is typically characterised by slow height growth, resulting in short stature, and may also include delayed puberty, impaired hair growth and headaches.[3] The goal of treatment for GHD is to restore normal levels of growth hormone in the body, reduce physical symptoms, enhance metabolic health and improve quality of life.[3] ST Chief Executive Officer, Mr Carlo Montagner, said the TGA approval of SKYTROFA demonstrated the company's ongoing mission to support patients with rare diseases in Australia and the Asia-Pacific region. "We are delighted to have secured TGA registration of SKYTROFA for eligible Australian children with growth hormone deficiency. Beyond short stature, children and adolescents with the condition may experience considerable physical and psychosocial impacts on their daily life, such as poor concentration, decreased strength or muscle development, fatigue, and reduced quality of life.[4] "This announcement also represents a significant milestone for Specialised Therapeutics, marking the second endocrinology therapy we have successfully registered in Australia, following the approval of YORVIPATH® (palopegteriparatide) earlier this year," said Mr Montagner. SKYTROFA is being registered in Australia by ST, under an exclusive distribution agreement with biopharmaceutical company Ascendis Pharma A/S that covers Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand, and Vietnam. The Australian registration of SKYTROFA follows approvals issued to Ascendis Pharma by the United States Food and Drug Administration (US FDA)[5] in August 2021 and the European Medicines Agency (EMA)[6] in January 2022. Developed using Ascendis Pharma's proprietary TransConTM platform, SKYTROFA is delivered as a subcutaneous injection, available in various strengths.[2] The starting dose depends on the patient's body weight and is then adjusted individually by the treating endocrinologist based on the patient's response to treatment.[2] Ascendis Pharma's innovative TransConTM technology platform is designed to create new therapies with the potential to optimise therapeutic effect, including improving treatment efficacy, safety and dosing frequency.[7] TransConTM molecules have three components: an unmodified parent drug, an inert TransCon™ carrier that protects it, and a TransConTM linker that temporarily binds the two.[7] SKYTROFA is a prodrug of somatropin administered once weekly, designed to provide sustained release of active, unmodified somatropin.[6] This allows the medicine to slowly release unmodified growth hormone into the body over the course of one week, removing the need for daily injections. SKYTROFA consists of three components: the parent drug somatropin, an inert methyloxypolyethlene glycol carrier (mPEG), and a proprietary TransConTM linker that transiently binds the other two elements.[8,9] While bound, the carrier inactivates the somatropin and shields it from renal excretion and receptor-mediated clearance.[10] Following injection, autocleavage of the linker occurs under physiologic conditions, and SKYTROFA releases fully active, unmodified somatropin in a predictable manner.[10] The TGA registration of SKYTROFA was based on the results of Ascendis Pharma's three pivotal Phase 3 clinical trials, heiGHt, fliGHt and enliGHten, which collectively treated more than 300 paediatric patients diagnosed with GHD, including from Australia.[2,8,11,12] SKYTROFA was generally well-tolerated across all three clinical trials. The most commonly reported adverse events include viral infections, fever, cough, nausea and vomiting, haemorrhage, diarrhoea, abdominal pain, arthralgia, arthritis, and increased blood phosphate levels. PBS Information: SKYTROFA is not listed on the Pharmaceutical Benefits Scheme (PBS). About Specialised Therapeutics Founded in 2007, Specialised Therapeutics is the region's largest independent specialty pharmaceutical company, providing new therapies and technologies to patients in Australia, New Zealand and across Southeast Asia. Headquartered in Singapore, ST partners with global pharmaceutical, biotech and diagnostic companies to bring novel healthcare opportunities to patients who are impacted by a range of diseases. ST has built a strong track record of success, navigating complex regulatory, reimbursement and commercialisation environments in its diverse regions. The ST mission is to provide specialty therapies where there is an unmet need. The company's broad therapeutic portfolio currently includes novel agents in oncology, haematology, CNS, neurology, endocrinology, ophthalmology and supportive care, although it is not confined to these areas. ST is a member of the World Orphan Drug Alliance (WODA). Additional information can be found at About Ascendis Pharma Ascendis Pharma is a global biopharmaceutical company focused on applying its innovative TransConTM technology platform to make a meaningful difference for patients. Guided by core values of Patients, Science, and Passion, and following its algorithm for product innovation, Ascendis Pharma applies TransConTM 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. About Paediatric Growth Hormone Deficiency Paediatric growth hormone deficiency (GHD) is a serious orphan disease caused when the pituitary gland does not produce enough growth hormone. Physiological levels of growth hormone are required for overall endocrine health and development of healthy bone, muscle, and adipose tissue. Children with GHD are characterised by short stature and may also experience metabolic abnormalities, psychosocial challenges, and an overall poor quality of life. For decades, the standard of care for GHD has been a daily subcutaneous injection of human growth hormone (hGH) to improve growth and overall endocrine health. About SKYTROFA (lonapegsomatropin) Once-weekly SKYTROFA is a prodrug of somatropin, designed to provide sustained release of unmodified somatropin. The unmodified, unbound somatropin released from lonapegsomatropin has the same 191 amino acid sequence and size as endogenous growth hormone.[8] SKYTROFA single-use, prefilled cartridges are manufactured in nine dosage strengths, allowing for convenient dosing flexibility. They are designed for use only with the SKYTROFA Auto-Injector and may be stored at room temperature for up to six months. The recommended dose of SKYTROFA for treatment-naïve children with growth hormone deficiency and those switching from daily somatropin is 0.24 mg/kg body weight, administered once weekly.[2] The dose may be adjusted based on the child's weight and insulin-like growth factor-1 standard deviation score (IGF-1 SDS).[2] SKYTROFA was studied in over 300 children with GHD across the Phase 3 program, which consisted of the HeiGHt Trial[8] (for treatment-naïve patients), the FliGHt Trial[11] (for treatment-experienced patients), and the EnliGHten Trial[12] (a long-term extension trial). Patients who completed the HeiGHt or FliGHt Trials were able to continue in EnliGHten, with some on lonapegsomatropin treatment for over four years. Ascendis Pharma is also conducting the ongoing open-label portion of the global Phase 3 ForesiGHt Trial of SKYTROFA in adults with GHD. Ascendis®, TransConTM and SKYTROFA® are trademarks owned by the Ascendis Pharma group (NASDAQ: ASND). About TransConTM Technologies TransCon refers to "transient conjugation". Ascendis Pharma's innovative technology platform to create new therapies designed to potentially optimise therapeutic effect, including improving efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert TransCon carrier that protects it, and a TransCon linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (e.g. pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action is expected to be maintained. TransCon technology can be applied to a broad variety of therapeutics, such as antibodies, antibody fragments, proteins, peptides, or small molecules, and can be designed for systemic or local administration. References: SOURCE Specialised Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Drug ApprovalOrphan DrugClinical Result

05 May 2025

·www.globenewswire.com

Ascendis to Share Its Latest Endocrinology Rare Disease Data at ESPE & ESE 2025

COPENHAGEN, Denmark, May 05, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it will share the latest data from its hypoparathyroidism, achondroplasia, and growth hormone deficiency (GHD) programs during ESPE & ESE 2025, the joint congress of the European Society for Paediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE) being held May 10-13, 2025, in Copenhagen. Four oral presentations will feature Ascendis clinical trial results, including 4-year efficacy and safety data from the Phase 2 PaTH Forward Trial of TransCon PTH (palopegteriparatide) in adults with chronic hypoparathyroidism; Week 52 growth and bone morphometry data from the pivotal ApproaCH Trial of TransCon CNP (navepegritide) in children with achondroplasia; and efficacy and safety data from the Phase 3 foresiGHt Trial of TransCon hGH (lonapegsomatropin) in adults with growth hormone deficiency. “As new treatments and clinical practices transform the outlook for rare endocrine diseases, we are pleased to partner with trial investigators and leading experts to share new data highlighting clinical benefits of our novel therapies with attendees of ESPE & ESE 2025,” said Aimee Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Science and Chief Medical Officer at Ascendis Pharma. The full range of Ascendis Pharma events and updates at ESPE & ESE 2025 include the following: Hypoparathyroidism Sunday, May 1109:55 - 10:25 CEST Congress TheaterFoyer F5Product TheaterRestoring Physiological Levels of PTH in Chronic HypoparathyroidismClick here to see the full session outline.Sunday, May 1116:55-17-55 CEST& Monday, May 1217:15-17:45 CESTPoster HallPoster P238Estimating the Risk of Chronic Kidney Disease Progression in Chronic Hypoparathyroidism: A Restrospective Matched Cohort Study, Using Real-World Data from England Monday, May 1213:00 - 14:30 CESTRoom D5SymposiumBeyond Conventional Care: Redefining Treatment Success in Chronic HypoparathyroidismClick here to see the full session outline.Monday, May 1215:55-16:05 CESTRoom C2Oral PresentationLong-Term Efficacy & Safety of Palopegteriparatide Treatment in Adults with Chronic Hypoparathyroidism: 4-Year Results from the Phase 2 PaTH Forward TrialPresented by Dr. Andrea PalermoAchondroplasiaSunday, May 1115:55-17:25 CESTRoom C2SymposiumAssessing HRQoL in Achondroplasia Across the Life CourseClick here to see the full session outline.Monday, May 1215:15-15:25 CESTRoom C2Oral PresentationEffects of Navepegritide on Bone Morphometry in Children with Achondroplasia: 52-Week Results from the ApproaCH Clinical TrialPresented by Dr. Leanne WardTuesday, May 1315:05-15:15 CESTRoom D2Oral PresentationEffects of Navepegritide on Growth in Children with Achondroplasia: 52-Week Results from the ApproaCH Clinical TrialPresented by Dr. Hanne HoveGrowth Hormone DeficiencyTuesday, May 1314:45-14:55 CESTRoom D2Oral PresentationResults of the foresiGH Trial Support the Efficacy and Safety of Once-Weekly Lonapegsomatropin in Adults with Growth Hormone Deficiency (GHD)Presented by Dr. Aleksandra Gilis-Januszewska More information about the events and presentations listed here is available to registered attendees of the Joint ESPE & ESE 2025 congress. 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 and idiopathic causes. 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 deformities, 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 have detrimental effects on quality of life, physical functioning, and psychosocial function. Individuals with achondroplasia often require multiple surgeries and procedures to alleviate the condition’s many complications. About Adult Growth Hormone DeficiencyGrowth hormone plays an essential role in the health of children and adults, promoting normal growth in children and maintenance of normal body composition and cardiometabolic health throughout adulthood. In adults, growth hormone boosts protein production, promotes fat utilization, enhances muscle mass, and helps regulate blood sugar levels. Adult GHD is a condition in which an individual’s body does not produce enough growth hormone. Symptoms and morbidity can include central obesity, metabolic syndrome, decreased bone density, alterations in lipid profile and markers of cardiovascular risk, fatigue, general weakness, lack of muscle tone, and psychological symptoms such as cognitive impairment, social isolation, lack of motivation, and depression. 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. Examples of such statements include, but are not limited to, statements relating to (i) the clinical benefits of Ascendis’ novel therapies, (ii) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients, and (iii) Ascendis’ application of its TransCon technologies 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 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; the impact of international economic, political, legal, compliance, social and business factors, including tariffs and trade policies. 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, and TransCon are trademarks owned by the Ascendis Pharma group. © May 2025 Ascendis Pharma A/S. Investor Contacts: Scott Smith Ascendis Pharma ir@ascendispharma.com Media Contact:Melinda BakerAscendis Pharmamedia@ascendispharma.com Patti BankICR Healthcare+1 (415) 513-1284patti.bank@icrhealthcare.com

Phase 2Clinical Result

21 Mar 2025

·www.fiercepharma.com

Biotech designed to commercialize Ascendis' endocrinology drugs in China files $86M IPO

Visen Pharmaceuticals is designed to develop and commercialize Ascendis’ endocrinology therapies in greater China. Endocrine-disease-focused Visen Pharmaceuticals is making its public market debut in Hong Kong, filing papers for an $86.5 million IPO.The China-based biotech was formed in 2018 by Ascendis Pharma and an investor syndicate led by Vivo Capital. The biotech is designed to develop and commercialize Ascendis’ endocrinology therapies in greater China.Now, Visen is going public, with expected gross proceeds of $100 million, according to March 20 regulatory filings (PDF). The company has previously raised about $190 million, closing a $150 million series B financing in early 2021. That fundraise included OrbiMed, Logos Capital, Ascendis Pharma, Vivo Capital and Sofinnova Investments, among others.The company hosts three programs in its pipeline: Yorvipath (palopegteriparatide) for adults with chronic hypoparathyroidism; Skytrofa (lonapegsomatropin) for pediatric growth hormone deficiency; and navepegritide, an investigational prodrug of C-type natriuretic peptide for achondroplasia.

IPO

100 Deals associated with Palopegteriparatide

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R&D Status

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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

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04701203 (PaTHway, Company_Website) Manual	Phase 3	Hypoparathyroidism	73	Palopegteriparatide	hzwdxgwntz(ubbjnrumwd) = wzudwszadz bpeybumegq (eyppatseku ) View more	Positive	14 Jul 2025
NCT04009291 (PaTH Forward, NEWS \| Corporate Publications) Manual	Phase 2	Hypoparathyroidism	56	Palopegteriparatide	frmmuxfchf(lnwtnfmkav) = ldlltmwvpf vkdqiibvql (uqsvkuayiq ) View more	Positive	18 May 2025
NCT04701203 (PaTHway, CTgov)	Phase 3	Hypoparathyroidism	84	TransCon PTH (TransCon PTH)	ilsoejtoiq = qkyxnwzpsr andajrpvov (nutklrpehj, lmpqwbmwhw - tssuoiquyz) View more	-	28 Feb 2025
				Placebo (Placebo)	ilsoejtoiq = ykhipbhvfk andajrpvov (nutklrpehj, yqxrenrnla - orqasiivae) View more
NCT05387070 (PaTHway, PRNewswire) Manual	Phase 3	Hypoparathyroidism	-	Palopegteriparatide	drhgcokpfm(avliiqkbmy) = bjdkcvciae emexmibokv (oroxencxbz ) View more	Positive	12 Aug 2024
				Placebo	drhgcokpfm(avliiqkbmy) = wynnxjjlnc emexmibokv (oroxencxbz )
NCT04701203 (FDA_CDER) Manual	Phase 3	Hypoparathyroidism	82	YORVIPATH	lbpxwkqwru(jiyrpagwbm) = pttxlijasd lpnidpoaiv (kmilrulibk ) View more	Positive	09 Aug 2024
				Placebo	lbpxwkqwru(jiyrpagwbm) = keggxvpnya lpnidpoaiv (kmilrulibk ) View more
NCT04701203 (PaTHway, GlobeNewswire) Manual	Phase 3	Hypoparathyroidism	82	TransCon PTH (palopegteriparatide)	ieezpznxav(tbzpvhzoan) = jyrlvueexw uklpsqvurx (kqotofhwdk ) View more	Positive	13 May 2024
				TransCon PTH (palopegteriparatide) (eGFR < 60 mL/min/1.73m2)	ieezpznxav(tbzpvhzoan) = nryfqeefnt uklpsqvurx (kqotofhwdk ) View more
NCT04009291 \| NCT04701203 \| NCT05387070 (PaTHway, ENDO2023)	Phase 3	Hypoparathyroidism	82	TransCon PTH	owyxvrzhjh(muzufuoqbm) = trended toward norms from baseline to Weeks 26 and 52 bnmqgbqipf (ajkhgumifo ) View more	Positive	05 Oct 2023
NCT04009291 \| NCT04701203 \| NCT05387070 (PaTHway, ENDO2022)	Phase 3	Hypoparathyroidism	82	TransCon PTH 18 mcg/d	dhistjeost(erkpvybojt) = The most commonly reported study drug-related AEs were headaches fxhjmkvnpz (yhljnyhtlo ) View more	Positive	01 Nov 2022
				Placebo
PaTH Forward Trial (ENDO2022)	Phase 2	Hypoparathyroidism	59	TransCon PTH 15 mcg/d	dpdnmkammv(gomunkxkxi) = gsvsstprmp uxcduzygsu (eixraxkfip ) View more	Positive	01 Nov 2022
				TransCon PTH 18 mcg/d	dpdnmkammv(gomunkxkxi) = sznjphbcpq uxcduzygsu (eixraxkfip ) View more
NCT04009291 (PaTH Forward, Corporate Publications) Manual	Phase 2	-	57	TransCon™ PTH	mwfkmslnrd(dzwmkpbpia) = xzvrsxddrb cubipxifsi (msfcgqyikw ) View more	Positive	12 Sep 2022

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