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

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 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 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

7,395

Literatures (Medical) associated with Palopegteriparatide

01 Jan 2026·JOURNAL OF ETHNOPHARMACOLOGY

Pien-Tze-Huang ameliorates autoimmune hepatitis by promoting hepatocyte autophagy via inhibiting miR-342-3p/mTOR signal

Article

Author: Liu, Miaohua ; Wang, Yang ; Xiong, Yi ; Zeng, Xin ; Zheng, Linxin ; Liu, Duanyong ; Li, Shanshan ; Zhou, Bugao

ETHNOPHARMACOLOGICAL RELEVANCE:

Pien Tze Huang (PTH), as a national protected species with a history of more than 450 years, is known for its efficacy in heat clearance, detoxification, blood cooling, blood stasis removal, and is commonly used in the treatment of liver-related diseases. However, it remains unclear whether PTH regulates autophagy by modulating miR-342-3p/mTOR signaling pathway for the treatment of autoimmune hepatitis (AIH).

AIM OF THE STUDY:

To explore the therapeutic effects and molecular mechanisms of PTH on AIH, focusing on the analysis of its regulatory effects on miR-342-3p/mTOR pathway and autophagy.

MATERIALS AND METHODS:

The chemical composition of PTH was analyzed by LC-MS/MS; AIH model was constructed in C57BL/6J mice to assess the effects of PTH on symptoms, pathological damage and cytokines; lysosomal changes were observed by transmission electron microscopy, and autophagy-related proteins (LC3B, Beclin-1) expression was detected; differential miRNAs and mRNAs were screened by whole-transcriptome sequencing and verified by RT-PCR for key miRNAs and mRNAs (miR-342-3p); induction of AML-12 hepatocyte inflammation model, combined with autophagy modeling experiments, lentivirus-mediated overexpression/knockdown of miR-342-3p and mTOR pathway interventions, to further clarify the molecular mechanism of PTH for AIH.

RESULTS:

PTH is mainly composed of amino acids and their derivatives, lipids and organic acids; PTH intervention significantly improved the symptoms of AIH mice, attenuated liver injury, regulated cytokines, and promoted lysosomal restitution and autophagy protein expression. Whole transcriptome sequencing and experiments confirmed that PTH downregulated miR-342-3p, inhibited mTOR signaling and enhanced autophagy. Cellular experiments further demonstrated that PTH activated autophagy and alleviated inflammation by directly regulating the miR-342-3p/mTOR axis.

CONCLUSION:

Pien-Tze-Huang ameliorates autoimmune hepatitis by promoting hepatocyte autophagy via inhibiting miR-342-3p/mTOR signal. This study elucidates a clear molecular mechanism for PTH's therapeutic action, highlighting its potential as a targeted treatment for AIH, and the miR-342-3p/mTOR axis serves as a viable target for future anti-AIH drug design.

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.

31 Dec 2025·ANNALS OF MEDICINE

Is it true that lower PTH control is associated with lower levels of inflammatory cytokines in patients with chronic kidney disease?

Article

Author: Fengmin, Shao ; Huixia, Cao ; Shasha, Wang ; Qin, Xu ; Yifeng, Hu ; Qiuyue, Xiong ; Ning, Wang ; Lei, Yan

BACKGROUND:

The body of patients with chronic kidney disease (CKD)is in a state of microinflammation, which further aggravates the progression of CKD and the occurrence and development of its complications. The inflammatory state of the body is significantly increased in patients with secondary hyperparathyroidism of CKD.

METHODS:

Patients diagnosed with chronic kidney disease in the department of Nephrology of Henan Provincial People's Hospital were selected as the experimental group, and 58 normal subjects were selected as the control group. Collecting patient's Clinical date, whole blood of the experimental group and control group was collected, and hs-CRP was detected by enzyme-linked immunosorbent assay. Compare the differences in clinical indicators and serum hs-CRP between the two groups. Taking PTH and hs-CRP as dependent variables, correlation analysis was conducted to clarify the relationship between PTH and hs-CRP, and further curve fitting was carried out.

RESULTS:

The levels of hemoglobin, total protein and albumin in CKD group were significantly lower than those in control group (p < 0.001). Alkaline phosphatase, creatinine, uric acid, Cyc and blood phosphorus in CKD group were higher than those in control group, and the P values were 0.016, <0.001, <0.001, <0.001, <0.001, respectively. Blood calcium was lower than control group, p < 0.001. The levels of hs-CRP in CKD group were significantly higher than those in control group, p < 0.001. Serum PTH was positively correlated with serum hs-CRP (r = 0.299, p = 0.002) in a normalized holistic analysis of CKD patients. In order to further clarify the correlation between PTH and hs-CRP, quadratic method was used to conduct curve fitting on PTH and hs-CRP and found that R = 0.361, F = 7.795, p < 0.001. Quadratic method was used to quadratic curve fitting at PTH levels <600 pg/mL, <500 pg/mL, <400 pg/mL, <300 pg/mL and <200 pg/mL, respectively, and it was found that the corresponding R = 0.281, F = 4.158, p < 0.001. R = 0.292, F = 4.397, p < 0.001; R = 0.546, F = 18.456, p < 0.001; R = 0.415, F = 7.471, p < 0.001; R = 0.403, F = 5.220, p < 0.001. It was found that there was no correlation between PTH and hs-CRP in hemodialysis patients and peritoneal dialysis patients. In order to exclude the influence of drug use on the statistical results, after excluding the patients who took over phosphorus binder and calcium binder, curve fitting method was used to find that PTH and CRP were correlated, in peritoneal dialysis group and hemodialysis group, R = 0.953, F = 39.913, p < 0.001; R = 0.448, F = 3.391, p = 0.010 respectively.

CONCLUSION:

CKD patients have the lowest levels of inflammatory factors within a certain range of PTH.

128

News (Medical) associated with Palopegteriparatide

22 Sep 2025

·endpoints.news

MBX Biosciences shares Phase 2 hypoparathyroidism data as it seeks to compete with Ascendis, AstraZeneca

MBX Biosciences said Monday that its experimental drug canvuparatide cleared the bar in a Phase 2 trial in a rare endocrine disorder called hypoparathyroidism. The once-weekly drug helped 63% of patients (30 out of 48 participants) maintain a normal range of albumin-adjusted serum calcium levels while also staying off conventional therapy of active vitamin D and daily calcium supplements at week 12, the Indiana-based drug developer said. At the same time point, 31% of patients on placebo (5 out of 16 people) achieved the composite primary endpoint. The study results were statistically significant at a p-value of 0.042. Investors were anticipating strong results from MBX’s Phase 2 study, codenamed Avail. The company’s share price $MBX rose 36% in post-market trading on Friday night after MBX said it would present the topline data before Monday’s opening bell. If Monday’s data are replicated in a larger Phase 3 trial, anticipated to begin next year, the biotech’s contender could potentially offer an alternative to an approved daily medicine marketed as Yorvipath by Ascendis Pharma. Other biopharmas also aim to treat the rare endocrine disorder, like Septerna and AstraZeneca, whose Amolyt-developed drug succeeded in a Phase 3 in March. Hypoparathyroidism impacts about 250,000 people in the US and Europe and can sometimes lead to chronic kidney disease. Takeda previously marketed a treatment for the rare endocrine condition, which is marked by reduced calcium levels and impairments to daily quality of life, but the Japanese pharma took it off the market due to manufacturing hurdles. “Hypoparathyroidism poses a substantial burden to patients, who often face complex treatment regimens and unpredictable swings in calcium levels that can lead to serious complications,” Mishaela Rubin, study investigator and Columbia University professor, said in a press release. “The 12-week and 6-month data provide promising early evidence that this investigational therapy may offer a potential option for long-term management, pending further study.” The study’s placebo-adjusted response rate of 32% came in below management expectations. In a note to clients last month, Mizuho analyst Uy Ear said MBX management anticipated a placebo-adjusted rate of 48%. While the response rate ended up being higher in the treatment arm, at 63% versus a projection of 55%, the placebo performed much better than expected. MBX had anticipated a 7% placebo response rate. The end result was 31%. In an interview with Endpoints News , CEO Kent Hawryluk said the placebo group was a “small sample size.” “While the placebo response was higher than originally planned, it’s not unusual. If we looked at the once-daily injectable,” Hawryluk said, referring to Ascendis’ Yorvipath, “in their Phase 2, they had a 27% placebo response for their primary endpoint. Importantly, that reduced in their longer Phase 3 study. “There’s a logical reason for that,” the MBX CEO continued. Hypoparathyroidism patients “can’t stay in the normal range of serum calcium for long without their conventional therapy,” he said. MBX said canvuparatide, previously known as MBX 2109, was “generally well-tolerated.” No treatment-related discontinuations were reported in the Phase 2 trial. The biotech also said 94% of patients elected to enroll in the open-label extension, with a 79% responder rate at six months. “We hope [to] provide a best-in-class treatment option for patients, which gives them freedom from having to take a daily injection, which is a daily reminder of their disease,” Hawryluk said. “We believe that we’ve shown with these results that we offer continuous infusion-like PTH exposure, which brings back their natural physiology, which they’re lacking with their disease.” Three doses were tested in the Phase 2. Chief medical officer Sam Azoulay told Endpoints that the biotech is still sifting through the full data package to determine the dose for the upcoming Phase 3. The topline data marked the second clinical readout for MBX since the biotech went public . The only other key clinical results since last year’s $163 million IPO were Phase 1 data for a different drug in treating post-bariatric hypoglycemia. That investigational medicine, called MBX 1416 or imapextide, is now in Phase 2 . Another drug, nicknamed MBX 4291, entered Phase 1 for obesity earlier this month. The biotech hopes MBX 4291 could compete in the obesity drug market as a once-monthly GLP-1 and GIP co-agonist. All of the drug candidates derive from the same platform at MBX, which aims to extend the half-life of peptide therapies. Hawryluk said Monday’s results could have “readthrough” to those other pipeline drug candidates. With canvuparatide, he noted, MBX was able to take a hormone peptide with a natural half-life of about one hour and turn it into a once-weekly treatment candidate.

Clinical ResultPhase 2Phase 1

22 Sep 2025

·firstwordpharma.com

MBX's weekly hypoparathyroidism drug hits key goals in Phase II

MBX Biosciences posted positive Phase II results for its experimental hypoparathyroidism treatment — prompting its share price to double on Monday — as the company looks to challenge Ascendis Pharma's Yorvipath (palopegteriparatide), the first US-approved treatment for the rare parathyroid hormone (PTH)-deficiency disorder.Canvuparatide is a long-acting PTH prodrug designed for once-weekly subcutaneous injection. Leveraging MBX's Precision Endocrine Peptide platform, the therapy is meant to help reduce daily swings in calcium levels that can lead to serious complications."We believe the totality of the data support a once-weekly product profile with continuous infusion-like PTH exposure," said CEO Kent Hawryluk. He added that preparations are underway to initiate a Phase III trial of canvuparatide in 2026. The Avail trial enrolled 64 adults with chronic hypoparathyroidism, a rare endocrine disease marked by low blood calcium and associated symptoms such as muscle cramping or spasm, and neurological symptoms such as depression, confusion and cognitive impairment. 63% responder rate In the 12-week randomised phase, 63% of patients on canvuparatide (30/48) achieved the primary composite endpoint — defined as normal serum calcium without dependence on active vitamin D and high-dose calcium supplements — compared with 31% for placebo (5/16). In the open-label extension portion, 79% of patients who received canvuparatide (44/56 evaluable) maintained responder status at six months, including those initially randomised to placebo.MBX said secondary endpoints showed positive results for bone turnover and formation markers, including BSAP, CTx, and P1NP, consistent with enhanced bone remodeling. Kidney activity also improved; in patients with elevated baseline urine calcium, mean levels fell 48% on canvuparatide at 12 weeks, versus a 33% reduction in placebo. MBX said there were no treatment-related serious adverse events or discontinuations reported. Mild injection site reactions occurred in 19% of treated patients in the pooled treatment groups, versus 13% on placebo.Another contender in the field is AstraZeneca, which is advancing eneboparatide following its acquisition of Amolyt Pharma for about $1.1 billion in 2024. The PTH receptor 1 agonist succeeded at normalising serum calcium in adults in the 202-patient CALYPSO study compared to placebo, according to topline data unveiled earlier this year.

Clinical ResultPhase 2AcquisitionDrug ApprovalPhase 1

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

100 Deals associated with Palopegteriparatide

External Link

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R&D Status

10 top approved records.

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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

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04701203 (PaTHway, Company_Website) Manual	Phase 3	Hypoparathyroidism	73	Palopegteriparatide	zegfhcossh(lsufuuqhwz) = iqzapjnayo gmelyppqnc (wwglmlfuxo ) View more	Positive	14 Jul 2025
NCT04009291 (PaTH Forward, NEWS \| Corporate Publications) Manual	Phase 2	Hypoparathyroidism	56	Palopegteriparatide	zfmryqgyxs(fxfglyojcj) = uhvjnjilnk xhcxuxsuif (rwvisyxhdk ) View more	Positive	18 May 2025
NCT04701203 (PaTHway, CTgov)	Phase 3	Hypoparathyroidism	84	TransCon PTH (TransCon PTH)	cqfcjwopvh = jgcdsyoaco jgoxqwturo (dtqotcecpi, kszwtxrxir - sfbaaxjixx) View more	-	28 Feb 2025
				Placebo (Placebo)	cqfcjwopvh = xakqgsfdld jgoxqwturo (dtqotcecpi, fugjqsirkd - wgiwwsguan) View more
NCT05387070 (PaTHway, PRNewswire) Manual	Phase 3	Hypoparathyroidism	-	Palopegteriparatide	iityepilqd(shdevorooo) = litwgynrdq incwwxaual (dpbpjebivv ) View more	Positive	12 Aug 2024
				Placebo	iityepilqd(shdevorooo) = ldsreyxlnf incwwxaual (dpbpjebivv )
NCT04701203 (FDA_CDER) Manual	Phase 3	Hypoparathyroidism	82	YORVIPATH	yycccxztyg(sswsevddfo) = xrxlbajdje qpjnubbcxf (wdhliurkwy ) View more	Positive	09 Aug 2024
				Placebo	yycccxztyg(sswsevddfo) = jtgiiiflxw qpjnubbcxf (wdhliurkwy ) View more
NCT04701203 (PaTHway, GlobeNewswire) Manual	Phase 3	Hypoparathyroidism	82	TransCon PTH (palopegteriparatide)	gygsjyjric(pazgzznzly) = rcgwmhtkbl dzbvlmdyhy (lgkvijaxoz ) View more	Positive	13 May 2024
				TransCon PTH (palopegteriparatide) (eGFR < 60 mL/min/1.73m2)	gygsjyjric(pazgzznzly) = gbfvwjlhph dzbvlmdyhy (lgkvijaxoz ) View more
NCT04009291 \| NCT04701203 \| NCT05387070 (PaTHway, ENDO2023)	Phase 3	Hypoparathyroidism	82	TransCon PTH	avslsutbcj(dffuhtoxbw) = trended toward norms from baseline to Weeks 26 and 52 rsqlfpxrtp (cwyhidhtrk ) View more	Positive	05 Oct 2023
NCT04009291 \| NCT04701203 \| NCT05387070 (PaTHway, ENDO2022)	Phase 3	Hypoparathyroidism	82	TransCon PTH 18 mcg/d	recicgqnkd(fgqwbvyocb) = The most commonly reported study drug-related AEs were headaches jyigngugse (wwwlocazfe ) View more	Positive	01 Nov 2022
				Placebo
PaTH Forward Trial (ENDO2022)	Phase 2	Hypoparathyroidism	59	TransCon PTH 15 mcg/d	xipdoevkea(slvllnhovd) = rttqdscraf lvjnpfqyxj (trljvspksp ) View more	Positive	01 Nov 2022
				TransCon PTH 18 mcg/d	xipdoevkea(slvllnhovd) = qxgkbwaokb lvjnpfqyxj (trljvspksp ) View more
NCT04009291 (PaTH Forward, Corporate Publications) Manual	Phase 2	-	57	TransCon™ PTH	ztzlxapsjd(dhzsnsvkfs) = diflzzqvdx burvizorph (zhrrrvhdne ) View more	Positive	12 Sep 2022

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Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis