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.

NCT04701203 / Active, not recruitingPhase 3

PaTHway 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

During the first 26 weeks of the trial, participants will be randomly assigned to one of two groups: one group will receive TransCon PTH and one group will receive placebo. All subjects will start with a fixed dose of study drug and will be individually and progressively titrated to an optimal dose over a 10 week period, followed by an individualized dosing period up to 16 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. This is a global trial that will be conducted in, but not limited to, the United States, Canada, Germany, and Denmark.

Start Date16 Feb 2021

Sponsor / Collaborator

Ascendis Pharma Bone Diseases A/S

100 Clinical Results associated with Palopegteriparatide

100 Translational Medicine associated with Palopegteriparatide

100 Patents (Medical) associated with Palopegteriparatide

Literatures (Medical) associated with Palopegteriparatide

01 Jun 2024·ADVANCES IN THERAPY

Palopegteriparatide Treatment Improves Renal Function in Adults with Chronic Hypoparathyroidism: 1-Year Results from the Phase 3 PaTHway Trial

Article

Author: Tsourdi, Elena ; Ominsky, Michael S ; Imanishi, Yasuo ; Sprague, Stuart ; Zhao, Carol ; Lai, Bryant ; Sibley, Christopher T ; Palermo, Andrea ; Makita, Noriko ; Khan, Aliya A ; Gosmanova, Elvira O ; Rejnmark, Lars ; Rubin, Mishaela R ; Takeuchi, Yasuhiro ; Makara, Michael A ; Kohlmeier, Lynn ; Ukena, Jenny ; Gagnon, Claudia ; Shoback, Dolores M ; Schwarz, Peter ; Shu, Aimee D

INTRODUCTION:

Individuals with chronic hypoparathyroidism managed with conventional therapy (active vitamin D and calcium) have an increased risk for renal dysfunction versus age- and sex-matched controls. Treatments that replace the physiologic effects of parathyroid hormone (PTH) while reducing the need for conventional therapy may help prevent a decline in renal function in this population. This post hoc analysis examined the impact of palopegteriparatide treatment on renal function in adults with chronic hypoparathyroidism.

METHODS:

PaTHway is a phase 3 trial of palopegteriparatide in adults with chronic hypoparathyroidism that included a randomized, double-blind, placebo-controlled 26-week period followed by an ongoing 156-week open-label extension (OLE) period. Changes in renal function over 52 weeks (26 weeks blinded + 26 weeks OLE) were assessed using estimated glomerular filtration rate (eGFR). A subgroup analysis was performed with participants stratified by baseline eGFR < 60 or ≥ 60 mL/min/1.73 m².

RESULTS:

At week 52, over 95% (78/82) of participants remained enrolled in the OLE and of those, 86% maintained normocalcemia and 95% achieved independence from conventional therapy (no active vitamin D and ≤ 600 mg/day of calcium), with none requiring active vitamin D. Treatment with palopegteriparatide over 52 weeks resulted in a mean (SD) increase in eGFR of 9.3 (11.7) mL/min/1.73 m² from baseline (P < 0.0001) and 43% of participants had an increase ≥ 10 mL/min/1.73 m². In participants with baseline eGFR < 60 mL/min/1.73 m², 52 weeks of treatment with palopegteriparatide resulted in a mean (SD) increase of 11.5 (11.3) mL/min/1.73 m² (P < 0.001). One case of nephrolithiasis was reported for a participant in the placebo group during blinded treatment; none were reported through week 52 with palopegteriparatide.

CONCLUSION:

In this post hoc analysis of the PaTHway trial, palopegteriparatide treatment was associated with significantly improved eGFR at week 52 in addition to previously reported maintenance and normalization of serum and urine biochemistries. Further investigation of palopegteriparatide for the preservation of renal function in hypoparathyroidism is warranted.

TRIAL REGISTRATION:

ClinicalTrials.gov NCT04701203.

30 Apr 2024·Endocrinology and metabolism (Seoul, Korea)

Treatment of Hypoparathyroidism by Re-Establishing the Effects of Parathyroid Hormone

Review

Author: Rejnmark, Lars

The conventional treatment of hypoparathyroidism (HypoPT) includes active vitamin D and calcium. Despite normalization of calcium levels, the conventional treatment is associated with fluctuations in calcium levels, hypercalciuria, renal impairment, and decreased quality of life (QoL). Replacement therapy with parathyroid hormone (PTH)(1-84) is an option in some countries. However, convincing beneficial effects have not been demonstrated, which may be due to the short duration of action of this treatment. Recently, palopegteriparatide (also known as TransCon PTH) has been marketed in Europe and is expected also to be approved in other countries. Palopegteriparatide is a prodrug with sustained release of PTH(1-34) designed to provide stable physiological PTH levels for 24 hours/day. A phase 3 study demonstrated maintenance of normocalcemia in patients with chronic HypoPT, with no need for conventional therapy. Furthermore, this treatment lowers urinary calcium and improves QoL. Another long-acting PTH analog with effects on the parathyroid hormone receptor (eneboparatide) is currently being tested in a phase 3 trial. Furthermore, the treatment of autosomal dominant hypocalcemia type 1 with a calcilytic (encaleret) is also being tested. All in all, improved treatment options are on the way that will likely take the treatment of HypoPT to the next level.

01 Jan 2022·The Journal of clinical endocrinology and metabolismQ2 · MEDICINE

PaTH Forward: A Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial of TransCon PTH in Adult Hypoparathyroidism

Q2 · MEDICINE

Article

Author: Karpf, David B ; Pagotto, Uberto ; Rubin, Mishaela ; Vokes, Tamara ; Eriksen, Erik ; Palermo, Andrea ; Brod, Meryl ; Clarke, Bart ; Mourya, Sanchita ; Khan, Aliya A ; Smith, Alden ; Schwarz, Peter ; Shu, Aimee D ; Pihl, Susanne ; Rejnmark, Lars ; Marcocci, Claudio ; Markova, Denka ; Ahmed, Intekhab ; Hofbauer, Lorenz

Abstract:

Context:

Hypoparathyroidism is characterized by insufficient levels of parathyroid hormone (PTH). TransCon PTH is an investigational long-acting prodrug of PTH(1-34) for the treatment of hypoparathyroidism.

Objective:

This work aimed to investigate the safety, tolerability, and efficacy of daily TransCon PTH in adults with hypoparathyroidism.

Methods:

This phase 2, randomized, double-blind, placebo-controlled 4-week trial with open-label extension enrolled 59 individuals with hypoparathyroidism. Interventions included TransCon PTH 15, 18, or 21 µg PTH(1-34)/day or placebo for 4 weeks, followed by a 22-week extension during which TransCon PTH dose was titrated (6-60 µg PTH[1-34]/day).

Results:

By Week 26, 91% of participants treated with TransCon PTH achieved independence from standard of care (SoC, defined as active vitamin D = 0 μg/day and calcium [Ca] ≤ 500 mg/day). Mean 24-hour urine Ca (uCa) decreased from a baseline mean of 415 mg/24h to 178 mg/24h by Week 26 (n = 44) while normal serum Ca (sCa) was maintained and serum phosphate and serum calcium-phosphate product fell within the normal range. By Week 26, mean scores on the generic 36-Item Short Form Health Survey domains increased from below normal at baseline to within the normal range. The Hypoparathyroidism Patient Experience Scale symptom and impact scores improved through 26 weeks. TransCon PTH was well tolerated with no treatment-related serious or severe adverse events.

Conclusion:

TransCon PTH enabled independence from oral active vitamin D and reduced Ca supplements (≤ 500 mg/day) for most participants, achieving normal sCa, serum phosphate, uCa, serum calcium-phosphate product, and demonstrating improved health-related quality of life. These results support TransCon PTH as a potential hormone replacement therapy for adults with hypoparathyroidism.

103

News (Medical) associated with Palopegteriparatide

25 Dec 2024

·www.globenewswire.com

YORVIPATH® (Palopegteriparatide) Now Commercially Available in the United States for the Treatment of Hypoparathyroidism in Adults

Dec. 19, 2024 -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that YORVIPATH® (palopegteriparatide; developed as TransCon PTH) is now commercially available by prescription in the United States. YORVIPATH is a prodrug of parathyroid hormone (PTH [1-34]), administered once-daily, designed to provide continuous exposure to active PTH over the 24-hour dosing period. It is the first and only medicine approved by the U.S. Food & Drug Administration (FDA) for the treatment of hypoparathyroidism in adults. “Members of our community have shared so many stories of positive changes in their lives while participating in clinical trials of YORVIPATH,” said Patty Keating, Executive Director of the HypoPARAthyroidism Association. “We are thrilled that YORVIPATH is now available in the United States, giving patients and physicians here a new approach to treating the underlying cause of this often-debilitating hormone deficiency.” “We are pleased to make YORVIPATH commercially available in the United States, where it is the first and only FDA-approved treatment of hypoparathyroidism in adults,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “We recognize the urgent unmet medical needs of the hypoparathyroidism community and are committed to doing all we can to ensure patients have affordable and broad access to YORVIPATH.” Reflecting this commitment, Ascendis has established a dedicated YORVIPATH team within the U.S. Ascendis Signature Access Program® to support the patient treatment journey, which is staffed by specially trained nurses and offers a full suite of programs designed to help patients, caregivers, and physicians navigate each step of the treatment process. These include clinical education, assistance with prior authorization and appeals, training on proper injection procedures, and co-pay and other related assistance for eligible patients. 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, 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. Ascendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of Patients, Science, and Passion, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more. The content above comes from the network. if any infringement, please contact us to modify.

Drug Approval

11 Dec 2024

·www.pharmaceutical-technology.com

Er-Kim and Ascendis’ endocrine therapy agreement extended in Eurasia

TransCon CNP, one of the therapies in the agreement, is currently in clinical development for achondroplasia. Credit: Louis Reed on Unsplash. Er-Kim has extended its exclusive agreement with Ascendis Pharma , broadening the reach of three endocrinology treatments across multiple countries in Eurasia. This follows the initial exclusive distribution arrangement announced in January 2024 which covered Turkey along with Central and Eastern Europe. The extended agreement appoints Er-Kim as the exclusive distributor for Ascendis Pharma’s endocrinology rare disease portfolio in Azerbaijan, Armenia, Georgia, Kyrgyzstan, Kazakhstan, Turkmenistan, Tajikistan and Uzbekistan. The therapies included are once-weekly human growth hormone, SKYTROFA (lonapegsomatropin), along with TransCon CNP (navepegritide) and the first-in-class parathyroid hormone (PTH) replacement therapy, YORVIPATH (palopegteriparatide). SKYTROFA received approval in the European Union for growth hormone-deficient individuals aged three to 18. It is also approved in the US for children aged one year and above with growth failure due to inadequate secretion of growth hormone. TransCon CNP is currently in clinical development for achondroplasia, the widespread form of skeletal dysplasia causing disproportionate short stature. Ascendis Pharma is advancing this treatment following the completion of its pivotal ApproaCH trial. Ascendis Pharma Europe and international president and executive vice-president Camilla Harder Hartvig stated: “At Ascendis Pharma we are driven by our commitment to make a meaningful difference in the lives of patients. “We are therefore pleased to extend our existing partnership with Er-Kim to further broaden patient access to our innovative endocrinology rare disease treatment options across Eurasia.” YORVIPATH addresses chronic hypoparathyroidism, a condition characterised by low or absent levels of PTH. It received approval in the European Union in November 2023 for adults with this condition and in the US for the treatment of hypoparathyroidism in adults. Er-Kim CFO Mert Zorlular stated: “We are proud of what we’ve achieved together over the past year and look forward to our expanded collaboration with Ascendis Pharma in broadening the distribution of its rare endocrinology disease therapies to address the significant unmet need for access to treatments in these countries. “Our success in making life-saving treatments available to patients regardless of location, one country at a time, depends on our partnerships with innovative companies such as Ascendis Pharma.”

Drug ApprovalLicense out/in

09 Dec 2024

·www.prnewswire.com

Orsini chosen by Ascendis to distribute treatment for rare endocrine disorder in the U.S.

ELK GROVE VILLAGE, Ill., Dec. 9, 2024 /PRNewswire/ -- Orsini has been selected by Ascendis Pharma as a specialty pharmacy partner for YORVIPATH® (palopegteriparatide), an FDA-approved parathyroid hormone analog (PTH(1-34)) indicated for the treatment of hypoparathyroidism in adults. Read the US Full Prescribing Information here. Continue Reading Chronic hypoparathyroidism is a rare disorder that affects about 70,000 people in the U.S., caused when a patient's body produces abnormally low levels of parathyroid hormone. This leads to low calcium levels and high phosphorus levels in the blood, and those living with chronic hypoparathyroidism can experience a range of symptoms including chronic pain, muscle cramps, cataracts, bone damage and kidney stones. "We're thrilled to add YORVIPATH to our growing portfolio of endocrinology therapies." Post this YORVIPATH is designed to provide continuous exposure to released PTH over the 24-hour dosing period. It is currently approved in the European Union, U.K. and European Economic Area. "We're thrilled to add YORVIPATH to our growing portfolio of endocrinology therapies," Darin DeCarlo, Orsini's Chief Growth Officer, said. "Orsini is built to serve rare disease patients, and we're excited to partner with Ascendis to deliver this new treatment option to patients suffering from chronic hypoparathyroidism." About Orsini Providing compassionate care since 1987, Orsini is a leader in rare disease and gene therapy pharmacy solutions, built to simplify how patients connect to advanced medicines. Through our comprehensive commercialization solutions including a nationwide specialty pharmacy, patient services hub, home infusion and nursing network, and third-party logistics provider, we work with biopharma, providers, and payors to ensure No Patient is Left Behind™. Orsini holds accreditations with the Accreditation Commission for Health Care (ACHC), The Joint Commission, URAC and NABP. Orsini has earned URAC's Rare Disease Pharmacy Center of Excellence Designation and ACHC's Distinction in Rare Diseases and Orphan Drugs. For more information, contact Orsini at 847-734-7373 ext. 505, email us at [email protected], or visit . SOURCE Orsini WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug ApprovalGene Therapy

100 Deals associated with Palopegteriparatide

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Indication	Country/Location	Organization	Date
Hypoparathyroidism	EU	Ascendis Pharma Bone Diseases A/S	17 Nov 2023
Hypoparathyroidism	IS	Ascendis Pharma Bone Diseases A/S	17 Nov 2023
Hypoparathyroidism	LI	Ascendis Pharma Bone Diseases A/S	17 Nov 2023
Hypoparathyroidism	NO	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


NCT05387070 (PaTHway, PRNewswire) Manual	Phase 3	Hypoparathyroidism	-	Palopegteriparatide	bcwsnrloua(odjyrmjndb) = qpswtobxtj lmccivywpq (vjjgmdxfcq ) View more	Positive	12 Aug 2024
				Placebo	bcwsnrloua(odjyrmjndb) = ewzkowvtsg lmccivywpq (vjjgmdxfcq )
NCT04701203 (FDA_CDER) Manual	Phase 3	Hypoparathyroidism	82	YORVIPATH	ejtpguwnlc(ixwnkqqnik) = hlrbynyktz jqpuqxenop (zwwmaahqgp ) View more	Positive	09 Aug 2024
				Placebo	ejtpguwnlc(ixwnkqqnik) = mfqidwlwui jqpuqxenop (zwwmaahqgp ) View more
NCT04701203 (PaTHway, GlobeNewswire) Manual	Phase 3	Hypoparathyroidism	82	TransCon PTH (palopegteriparatide)	lvwkijadwt(smysawitjh) = fgwqhqrujx jzxeoggorf (kcfoaobiko ) View more	Positive	13 May 2024
				TransCon PTH (palopegteriparatide) (eGFR < 60 mL/min/1.73m2)	lvwkijadwt(smysawitjh) = evjebuwvje jzxeoggorf (kcfoaobiko ) View more
PaTHway (ENDO2023)	Phase 3	Hypoparathyroidism	82	TransCon PTH	ekecbnchor(hhcywngcyw) = trended toward norms from baseline to Weeks 26 and 52 yoyeblodbs (jptfdyojxe ) View more	Positive	05 Oct 2023
NCT04009291 (PaTHway \| PaTH Forward, CTgov)	Phase 2	Hypoparathyroidism	59	TransCon PTH (TransCon PTH 15 mcg)	epitszzrqc(ktzmggvltb) = thytwmaute mtybeunkuv (artnlbaism, mvqvsbuxfl - nxeiuhigfi) View more	-	01 Sep 2023
				TransCon PTH (TransCon PTH 18 mcg)	epitszzrqc(ktzmggvltb) = nvokrhurlk mtybeunkuv (artnlbaism, njrcxkfuzt - qyowqkbhbu) View more
PaTH Forward Trial (ENDO2022)	Phase 2	Hypoparathyroidism	59	TransCon PTH 15 mcg/d	zbyvrongyp(ylxcdosarn) = mercxywaki dewluqsdwb (jrmmfpigio ) View more	Positive	01 Nov 2022
				TransCon PTH 18 mcg/d	zbyvrongyp(ylxcdosarn) = dqgishrbvn dewluqsdwb (jrmmfpigio ) View more
PaTHway (ENDO2022)	Phase 3	-	82	TransCon PTH 18 mcg/d	acvpkcviag(rilpoirids) = The most commonly reported study drug-related AEs were headaches jixhrwiviz (ozqvejtqks ) View more	Positive	01 Nov 2022
				Placebo
NCT04009291 (PaTH Forward, Corporate Publications) Manual	Phase 2	-	57	TransCon™ PTH	lnwduoofhy(gijducrhcs) = ubrgnxhyuz wwnsrybwho (hqvhewthet ) View more	Positive	12 Sep 2022
WCO_IOF_ESCEO2022	Not Applicable	Acromegaly PTH \| serum vitamin D metabolites	-	PTH (Acromegaly group)	smfxuhdoit(qhslaoksrj) = lower baseline levels yklmulpkbb (nlynxvzgcf ) View more	-	24 Mar 2022
				PTH (Control group)
NCT04701203 (Corporate Publications) Manual	Phase 3	Hypoparathyroidism	82	Palopegteriparatide	tztshemxzn(naqrmpfhcw) = dgnfffuwol cqaypdonse (pcpugansup, 66.3 - 88.1) View more	Positive	13 Mar 2022
				Placebo	tztshemxzn(naqrmpfhcw) = dbzxbncvza cqaypdonse (pcpugansup, 0.1 - 23.8) View more

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