– 58 subjects continue in the open-label extension study for TransCon PTH –
COPENHAGEN, Denmark, May 10, 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 preliminary 58-week results from the continuing open-label extension (OLE) portion of the PaTH Forward Trial, a global phase 2 trial evaluating the safety, tolerability, and efficacy of its investigational product candidate TransCon PTH in adult subjects with hypoparathyroidism (HP).
"Today we announced results which demonstrated, after one year of treatment, that TransCon PTH was well-tolerated at all doses and provided durable benefit in adults with HP. Data from these subjects provided evidence suggesting they are establishing physiologic calcium metabolism based upon normalization of 24-hour urine calcium excretion and a downward trend toward mid-normal levels in bone markers without the use of therapeutic calcium and vitamin D supplementation. In addition, patient data also showed continued normalized quality of life as measured by the SF-36 questionnaire. Taken together, we believe these results further support the use of TransCon PTH as a potential new treatment paradigm for adults living with HP," said Jan Mikkelsen, President and CEO of Ascendis Pharma.
"What is particularly rewarding to me is that all 58 patients continue to participate in the open label extension. I believe this is a reflection of the positive impact TransCon PTH is having on these individuals' lives," continued Mr. Mikkelsen.
Key Findings of the Preliminary OLE Results of PaTH Forward Trial at 58 weeks
- 58 subjects continue in the open-label extension beyond 58 weeks as of May 7, 2021
- Continued treatment with TransCon PTH demonstrated that:
- 91% of subjects were off standard of care therapy defined as no active vitamin D and ≤600 mg/day of calcium supplements
- Urinary calcium maintained in the normal range
- Bone markers trended towards the mid-normal levels
- Quality of life benefits measured by SF-36 continued within 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
- No change to the safety profile in the OLE portion of the study
A slide presentation with these data is available at the Investors & News section of the company's website here:https://investors.ascendispharma.com/
TransCon PTH Program Update
In addition to PaTH Forward, Ascendis Pharma is conducting the PaTHway Trial, a North American and European phase 3 clinical study evaluating the safety, tolerability and efficacy of TransCon PTH in adults with HP. Topline results are expected from PaTHway in the fourth quarter of 2021.
Ascendis plans to submit a Clinical Trial Notification to the Pharmaceuticals and Medical Devices Agency in Japan during the second quarter of 2021 to initiate a phase 3 trial of TransCon PTH in adults with hypoparathyroidism.