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 - Seite 2

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.”