– Six-month data from PaTH Forward open-label extension support potential use of TransCon PTH as a hormone replacement therapy for adult hypoparathyroidism –– Conference call Tuesday, September 29 at 8:00 a.m. Eastern Time to review data –
COPENHAGEN, Denmark, Sept. 29, 2020 (GLOBE NEWSWIRE) — Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical needs, today announced preliminary six-month results from the open-label extension (OLE) portion of PaTH Forward, a global phase 2 trial evaluating the safety, tolerability and efficacy of TransCon PTH in adult subjects with hypoparathyroidism (HP).
“Today we announced preliminary clinical results which demonstrate that TransCon PTH can potentially provide a new treatment paradigm for over 150,000 HP patients in North America and Europe. These results showed that subjects in the trial continued on once-daily TransCon PTH independent of pill burden and consistently improved their quality of life, while at the same time demonstrating improvement in 24-hour urine calcium excretion and serum phosphate which may be associated with long-term complications1,2,” said Jan Mikkelsen, President and CEO at Ascendis Pharma. “We have now filed an IND amendment to initiate the U.S. sites of the phase 3 PaTHway Trial evaluating TransCon PTH in adult patients with hypoparathyroidism, which will enable us to eventually provide this therapy to patients as soon as possible.”TransCon PTH is an investigational long-acting prodrug of parathyroid hormone (PTH) in development as a once-daily hormone replacement therapy for adult hypoparathyroidism designed to replace PTH at physiologic levels for 24 hours each day and addresses both short-term symptoms and long-term complications of HP. Fifty-nine subjects participated in the phase 2 PaTH Forward Trial and continued in the OLE, where they all received a customized maintenance dose of TransCon PTH (6 to 30 µg) with a ready-to-use, room temperature, prefilled pen injector. One subject randomized to placebo withdrew after completing the four-week double-blinded fixed-dose period for reasons unrelated to safety or efficacy of the study drug. All of the other 58 subjects remained on TransCon PTH at the time of the six month data cutoff.IND Amendment Filed for Phase 3 PaTHway Trial
The company submitted an amendment to its investigational new drug application (IND) with the U.S. Food and Drug Administration (FDA) to initiate the U.S. sites of the PaTHway phase 3 clinical trial evaluating the safety, tolerability and efficacy of TransCon PTH in adults with HP following discussions with FDA and European regulatory authorities. The company expects to file the clinical trial application for the European arm later this year. The double-blind, placebo-controlled trial is expected to enroll approximately 76 subjects at sites in North America and Europe in order to obtain 68 evaluable subjects.The primary composite endpoint of the PaTHway Trial at 26 weeks is the proportion of subjects with (1) serum calcium in the normal range, (2) independence from active vitamin D, and (3) taking ≤600 mg/day of calcium supplements.Preliminary OLE Results of PaTH Forward Trial at 6 Months
Preliminary six-month results from the PaTH Forward OLE demonstrated:91 percent of all subjects eliminated standard of care (defined as (1) off active vitamin D and (2) ≤500 mg per day of calcium supplements), including 76 percent who eliminated all supplements.86 percent of all subjects normalized or reduced by 50 percent 24-hour urine calcium.71 percent of all subjects achieved a response on the composite endpoint of (1) serum calcium in the normal range, (2) independence from active vitamin D, (3) taking ≤500 mg/day of calcium supplements, and (4) 24-hour urine calcium in the normal range or 50 percent reduction from baseline, including 74 percent of subjects who were randomized to TransCon PTH.All mean summary and subdomain SF-36® Health Survey scores normalized despite all mean scores starting below norms at baseline including subjects randomized to placebo who switched to TransCon PTH group at week 4. Importantly, subjects randomized to TransCon PTH demonstrated continued improvements from week 4 to month 6.All doses of TransCon PTH were well-tolerated, and no treatment-related serious or severe adverse events were observed at any point. No subjects had PTH treatment-emergent adverse events related to hyper- or hypocalcemia leading to emergency visit, urgent care visit, or hospitalization.Adherence to daily injections of TransCon PTH was 99.7 percent.“For people living with hypoparathyroidism, this debilitating endocrine disease frequently disrupts daily activities, diminishes quality of life and often leads to long-term complications,” Aimee Shu, M.D., Senior Medical Director, Clinical Development at Ascendis Pharma. “We are very encouraged by the results of TransCon PTH in the PaTH Forward Trial, which may eventually represent a new treatment paradigm for patients with this disorder.” A slide presentation with these data is available at the Investors & News section of the company’s website here: https://investors.ascendispharma.com/Conference Call DetailsA live webcast of the conference call will be available on the Investors and News section of the Ascendis Pharma website at www.ascendispharma.com. A webcast replay will be available on this website shortly after conclusion of the event for 30 days.
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