COPENHAGEN, Denmark, July 06, 2021 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to create new product candidates that address unmet medical needs, today announced it has reached the target enrollment in the phase 3 PaTHway Trial, a trial evaluating the safety, tolerability, and efficacy of TransCon PTH (palopegteriparatide) in addition to providing a comprehensive global clinical program update.
“The durable benefit demonstrated in our phase 2 PaTH Forward Trial Week 58 data supports the use of TransCon PTH as a potential new treatment paradigm for adults with HP. We are committed to bringing TransCon PTH as quickly as possible to all patients with HP independent of etiology,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Given the strong demand and our commitment to patients and clinical investigators, although we stopped screening new subjects in early June, if any subject had started the screening process by June 7th, they will be enrolled in the phase 3 PaTHway Trial if all the criteria are met. We anticipate announcing top-line results from the PaTHway Trial in the first quarter of 2022, and if successful, followed by an expected NDA submission in mid-2022.”
“We are pleased to continue extending the global clinical reach for TransCon PTH for all HP patients as a key component of our Vision 3x3. With recent acceptances from regulatory authorities relating to the PaTHway Japan Trial and the PaTHway China Trial through VISEN Pharmaceuticals (VISEN), we believe we now have a defined pathway to obtain regulatory approval in countries representing over 400,000 patients,” continued Mr. Mikkelsen.
The PaTHway Trial is a phase 3, randomized, double-blind, placebo-controlled trial in North America and Europe evaluating the safety, tolerability, and efficacy of palopegteriparatide in adults with HP. The primary efficacy endpoint is the proportion of subjects with albumin-adjusted serum calcium within the normal range, and independent from active vitamin D and therapeutic doses of calcium (≤ 600 mg/day) at 26 weeks. If successful, Ascendis expects to submit an NDA to the U.S. Food and Drug Administration (FDA) in mid-2022 and subsequently submit a Marketing Authorisation Application to the European Medicines Agency.
Ascendis also announced the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) acceptance of the clinical trial notification for the PaTHway Japan Trial, a single-arm, phase 3 trial of palopegteriparatide in a minimum of 12 Japanese subjects with HP. Subjects will start with an 18 µg dose of palopegteriparatide and be followed over a 26-week period during which they will be titrated to an optimal dose. In addition, MHLW granted ODD to palopegteriparatide for the treatment of HP. In Japan, ODD is granted to therapies intended for use in less than 50,000 patients in Japan and for which significant unmet medical need exists. The designation is granted by the MHLW based on the opinion of the Pharmaceutical Affairs and Food Sanitation Council.
In Greater China, VISEN recently announced that they obtained the IND approval from the Center for Drug Evaluation of the National Medical Products Administration for the phase 3 China clinical trial (PaTHway China Trial) of TransCon PTH on June 1, 2021 and is soon expected to initiate the study of TransCon PTH in patients with HP in China. The design of the PaTHway China Trial mirrors the design of the PaTHway Trial.
All subjects from phase 3 PaTHway trials in North America, Europe, Japan and Greater China will be eligible to enter into open-label extensions to collect long-term follow-up data.
Palopegteriparatide was granted ODD from the FDA in June 2018 and from the European Commission in October 2020.