Ascendis Pharma A/S Announces Preliminary Six-Month Data from Open-Label Extension of Phase 2 PaTH Forward Trial and Files IND Amendment to Initiate Phase 3 PaTHway Trial of TransCon PTH in Adult Hypoparathyroidism
- 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.