Ascendis Pharma A/S Announces Expansion of Global Clinical Reach for TransCon PTH with Filing of the Clinical Trial Notification for Phase 3 Clinical Trial in Adults with Hypoparathyroidism in Japan
– Marks next step to bring the potential first ever hormone replacement therapy for hypoparathyroidism to Japan –
COPENHAGEN, Denmark, May 12, 2021 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to create product candidates
that address unmet medical needs, today announced that it has filed a Clinical Trial Notification (CTN) with the Pharmaceuticals and Medical Devices Agency (PDMA) in Japan to initiate the company’s
phase 3 clinical trial of TransCon PTH in adult subjects with hypoparathyroidism (HP), the PaTHway Japan Trial.
“The CTN filing for TransCon PTH represents another milestone as we continue to lay the groundwork to make this important therapy available globally to address a significant unmet need for patients with hypoparathyroidism worldwide. The phase 3 PaTHway Japan Trial, which has been the subject of careful negotiations with the PDMA, will enroll a predetermined cohort of patients so that the combined global program will support submission of a marketing authorization application in Japan. This study, along with long-term data from our phase 2 PaTH Forward Trial and data from our phase 3 PaTHway trial, should potentially enable rapid access to an important hormone replacement therapy for patients living with HP in Japan,” said Jan Mikkelsen, President and CEO of Ascendis Pharma.
“An objective of our Vision 3x3 is to obtain global clinical reach for our endocrinology rare disease pipeline. Starting first with North America and Europe, then expanding to Greater China through our investment in VISEN Pharmaceuticals, our recent expansion of our clinical reach into Japan with TransCon hGH and TransCon PTH represents an important milestone in realizing this vision,” continued Mr. Mikkelsen.
The phase 3 PaTHway Japan Trial is designed to be a single arm study with the objective to evaluate the safety and efficacy of TransCon PTH in adults with hypoparathyroidism.
About TransCon PTH1
TransCon PTH is an investigational once-daily long-acting prodrug of parathyroid hormone (PTH[1-34]) in development as a treatment for adult hypoparathyroidism (HP) designed to restore PTH at physiologic levels for 24 hours each day to address both the short-term symptoms and long-term complications of the disease. TransCon PTH was granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) in June 2018, and in October 2020 was granted Orphan Designation by the European Commission for the treatment of hypoparathyroidism.