Benzinga | Nov 18, 2021 04:46PM EST

Ascendis Pharma A/S Announces Top-Line Results From Week 84 Of Phase 2 PaTH Forward Trial Demonstrating That TransCon PTH Provided Durable Benefit And Was Well-Tolerated In Adults with Hypoparathyroidism

Ascendis Pharma A/S (NASDAQ:ASND), today announced top-line results from Week 84 of the Company's Phase 2 PaTH Forward Trial, a global trial evaluating the safety, tolerability, and efficacy of its investigational TransCon PTH product candidate in adult patients with hypoparathyroidism (HP). TransCon PTH is an investigational once-daily prodrug of parathyroid hormone (PTH) designed to restore physiologic levels of PTH 24 hours a day. The week 84 data showed that subjects treated with TransCon PTH had both mean serum calcium levels and urinary calcium excretion that remained stable and in the normal range and that most subjects (93%) continued to be free from taking active vitamin D and were taking < 600 mg/day of calcium supplements.

"The continued durable improvements observed in this study over a year and a half of treatment with TransCon PTH highlight the importance of sustained restoration of parathyroid hormone to normal physiological levels," said Professor Peter Schwarz, M.D., DMSci, Professor of Endocrinology at Rigshospitalet and Copenhagen University. "Parathyroid hormone plays an essential role in the regulation of serum and urine calcium and phosphate, thus impacting the function of nerves and muscles, and I am excited that we may potentially soon be able to offer a new treatment paradigm to address the serious health and quality of life issues these patients face each day."

After an initial screening and baseline assessment period, patients in the Phase 2 PaTH Forward Trial were randomized to blinded treatment with TransCon PTH at 15, 18, or 21 ?g/day or placebo for 4 weeks, followed by a switch to open label treatment, during which physicians could optimize dosing of TransCon PTH to meet individual treatment objectives.

Key Findings at Week 84 of the Phase 2 PaTH Forward Trial:

* 58 out of the 59 original trial participants continued open-label treatment with TransCon PTH.

* Mean serum calcium levels remained stable and in the normal range.

* All study subjects discontinued active vitamin D supplements in the earliest weeks of the trial and have remained off it since then. In addition, 93% of study subjects were taking calcium supplements <600 mg/day.

* Mean urinary calcium excretion remained stable and in the normal range.

* TransCon PTH was well-tolerated at all doses administered. No treatment-related serious or severe adverse events occurred, and no treatment-emergent adverse events (TEAEs) led to discontinuation of study drug.

"We believe that these data, combined with nearly all patients continuing in the open label portion of the trial, indicates the potential for TransCon PTH to become the first hormone therapy to replace active vitamin D and therapeutic doses of calcium for adults living with hypoparathyroidism," said Dana Pizzuti, M.D., Ascendis Pharma's Chief Medical Officer and Senior Vice President of Development Operations. "We look forward to sharing our top-line Phase 3 data in Q1 2022 and, if positive, plan to complete an NDA submission to the FDA in mid-2022."

The Investor presentation on the Investor & Media section of the Ascendis Pharma website at https://ascendispharma.com has been updated to include the TransCon PTH Path Forward Trial Week 84 Week data.