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One-Year Data from Phase 3 Trial of TransCon™ PTH in Adults with Hypoparathyroidism Presented at ENDO 2023
Clinical ResultPhase 3
- Oral presentation of results showed sustained improvements through Week 52, as well as safety and tolerability similar to that previously reported for the initial 26-week blinded portion of the Phase 3 PaTHway Trial
- At Week 52, 95% of patients treated with TransCon PTH achieved independence from conventional therapy
- TransCon PTH treatment improved mean patient-reported disease symptom and health-related quality of life scores, starting at the first scheduled follow up after randomization or after switching from placebo and sustained through Week 52
Ascendis Pharma A/S (Nasdaq: ASND) today reported one-year (Week 52) data from its ongoing Phase 3 PaTHway Trial of TransCon PTH in adults with hypoparathyroidism. The data showed that treatment with TransCon PTH resulted in sustained improvements through Week 52, as well as safety and tolerability similar to that reported for the initial 26-week blinded portion of the trial. The data were presented by Bart Clarke, M.D., endocrinologist and Professor of Medicine at the Mayo Clinic (Rochester, MN), during ENDO 2023, the annual meeting of the Endocrine Society being held in Chicago.
“We are very pleased to see sustained improvements in clinical outcomes in this trial, including symptom and health-related quality of life measures, consistent with those reported earlier for the initial 26-week blinded portion,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “These clinical and patient-reported data, and the retention of 145 patients in our ongoing clinical trials, reinforces our confidence that TransCon PTH has the potential, if approved, to benefit adults with hypoparathyroidism regardless of disease etiology.”
Methods
PaTHway is a Phase 3 trial of TransCon PTH with a placebo (PBO)-controlled 26-week blinded portion and a 156-week open-label extension (OLE) portion, designed to evaluate the long-term efficacy and safety of TransCon PTH as a potential hormone therapy for those diagnosed with hypoparathyroidism. Results through Week 52 (26 weeks blinded + 26 weeks OLE) were reported at ENDO 2023. Of the 82 study participants dosed, 79 completed blinded treatment and entered the OLE, and 78 (59 TransCon PTH/TransCon PTH, 19 PBO/TransCon PTH) completed Week 52.
Week 52 Highlights
95% of patients in the OLE (74 out of 78) achieved independence from conventional therapy (defined as no active vitamin D and calcium supplements of 600mg/day), and none required active vitamin D.
At Week 52, 81% of participants treated with TransCon PTH achieved both normal serum calcium and independence from conventional therapy.
With TransCon PTH treatment, mean albumin-adjusted serum calcium levels were maintained within the normal range (8.3–10.6 mg/dL) through Week 52 of the OLE (8.9 mg/dL at Week 52).
Patient-reported scores on the Hypoparathyroidism Patient Experience Scale (HPES) and SF-36 Health Survey showed sustained improvements in disease-related physical and cognitive symptoms, as well as physical functioning and daily life, starting at the first scheduled follow up after randomization or switching from placebo and sustained through Week 52.
Bone mineral density (BMD) Z-scores continued to trend toward age- and sex-matched norms with 52 weeks of TransCon PTH treatment.
TransCon PTH normalized 24-hour urine calcium through Week 52, regardless of initial randomization (placebo or TransCon PTH).
TransCon PTH continued to be well-tolerated in the Phase 3 open-label extension, with no new safety signal identified. Most TEAEs were mild or moderate (Grades 1-2) and none reported during the open-label extension through Week 52 led to discontinuation of the study drug or trial.
Slides showing the data presented for Week 52 of the Phase 3 PaTHway trial of TransCon PTH in adults with hypoparathyroidism can be viewed on the Ascendis Pharma Investors & News website at https://investors.ascendispharma.com.
About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated, global biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Germany (Heidelberg, Berlin and Munich) and the United States (Palo Alto and Redwood City, California, and Princeton, New Jersey). Visit ascendispharma.com to learn more.
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