Benzinga | Mar 23, 2021 07:10AM EDT

Ascendis Pharma A/S Highlights Presentation Of 6-Month Open-Label Extension Data From Phase 2 PaTH Forward Trial Of TransCon PTH In Adult Hypoparathyroidism At ENDO 2021

Ascendis Pharma A/S (NASDAQ:ASND), a biopharmaceutical company that utilizes its innovative TransCon(tm) technologies to create product candidates that address unmet medical needs, today presented the safety and efficacy results of TransCon PTH after six-months of treatment in patients with hypoparathyroidism enrolled in the open-label extension period (OLE) of the phase 2 PaTH Forward Trial, at the Endocrine Society's annual meeting, taking place virtually from March 20-23, 2021.

"The study demonstrated that a majority of subjects were able to achieve independence from conventional therapeutic supplements, while maintaining serum calcium in the normal range and having clinically meaningful improvements in urine calcium levels, bone turnover markers and bone mineral density and quality of life," said Mishaela Rubin, M.D. M.S., Associate Professor of Medicine in the Metabolic Bone Disease Unit at the Vagelos College of Physicians & Surgeons at Columbia University Irving Medical Center in New York City. "Importantly, we are presenting the first data on the treatment effect of TransCon PTH on the Hypoparathyroidism Patient Experience Scale (HPES). In this study, HPES scores all decreased from baseline to Week 26, indicating fewer and less impact of disease-specific symptoms. These data are promising and TransCon PTH may offer a new potential treatment option for patients with hypoparathyroidism, many of whom have their daily lives disrupted by the disorder."

Presentation Details and Data Highlights1

Presentation title: TransCon PTH as a Hormone Replacement Therapy for Patients with Hypoparathyroidism: 6-Month Update from the PaTH Forward Open-Label Extension in 58 subjects.

Oral session: Novel Treatments for Metabolic Bone Diseases; March 23, 2021 at 2:15 to 2:25 p.m. EDT.

Presenter: Mishaela Rubin, M.D. M.S.

Summary of findings: Six-month results from the PaTH Forward OLE demonstrated:

* Over 90 percent of subjects demonstrated independence from standard-of-care (calcium ? 500 mg/day and active vitamin D = 0 ?g/day) and 76 percent of subjects eliminated all supplements while: Maintaining mean serum calcium in the normal range Reducing mean 24-hour urine calcium to within the normal range Achieving sustained reductions in mean serum phosphate and calcium x phosphate product Demonstrating enhanced quality of life on both SF-36 Functional Health Survey and HPES Trending towards normalization of skeletal remodeling.

* TransCon PTH continues to be well-tolerated: No adverse events of hypocalcemia or hypercalcemia requiring visit to hospital, emergency room or urgent care.

"All 58 subjects in the open-label extension have now completed 12 months of treatment on TransCon PTH without any additional dropouts, which is a reflection of patient comfort with this once-daily injection as a chronic therapy for hypoparathyroidism," said Mark Bach, M.D., Ph.D., Senior Vice President of Endocrine Medical Sciences at Ascendis Pharma. "TransCon PTH is a potential hormone replacement therapy that was designed to restore calcium metabolism and improve quality of life for people with hypoparathyroidism, and we plan to present the 12-month results at an upcoming scientific meeting."

In addition, to PaTH Forward, Ascendis Pharma is conducting the PaTHway Trial, a phase 3 clinical study evaluating the safety, tolerability and efficacy of TransCon PTH in adults with HP. Topline results are expected from PaTHway in the fourth quarter of 2021.

About the PaTH Forward Trial1

PaTH Forward is a global, phase 2, randomized, double-blind, placebo-controlled group trial evaluating the safety and efficacy of three fixed doses of TransCon PTH (15, 18 or 21 ?g/day or placebo). The trial enrolled 59 adult subjects with chronic HP who received standard of care or were previously treated with PTH therapies. The goal of PaTH Forward is to evaluate TransCon PTH control of serum and urinary calcium and identify a titration regimen for complete withdrawal of standard of care (i.e., active vitamin D and calcium supplements). PaTH Forward has introduced a ready-to-use pre-filled pen injector and assesses disease-specific patient-reported outcomes. After four weeks of fixed dosing, all subjects were eligible to enter an open-label extension period with the opportunity to receive a customized maintenance dose of TransCon PTH to evaluate long-term safety and efficacy.