Ascendis Pharma said that its prodrug candidate TransCon PTH has met key objectives in the phase 2 PaTH Forward trial in adult subjects with hypoparathyroidism.
The Danish pharma company said that the four-week fixed dose, blinded portion of PaTH Forward, which is assessing the safety, tolerability and efficacy of TransCon PTH in the subjects has yielded positive top-line results.
According to Ascendis Pharma, TransCon PTH is a long-acting prodrug candidate of parathyroid hormone (PTH). The investigational prodrug is being developed as a once-daily replacement therapy for hypoparathyroidism in adults.
It has been designed by the Danish pharma company to replace PTH at physiologic levels for 24 hours each day and for addressing the short-term symptoms and also long-term complications of hypoparathyroidism.
Ascendis Pharma randomly grouped 59 patients in a blinded manner in the PaTH Forward trial, who were given fixed doses of TransCon PTH at 15, 18 or 21 µg/day or placebo for four weeks with a ready-to-use prefilled pen injector.
As per Ascendis Pharma, TransCon PTH eliminated standard of care in all the subjects in the highest dose arm and in 82% of subjects across all the dosage arms.
The Danish pharma company said that all doses of the prodrug candidate were well-tolerated, and no serious or severe adverse incidents were observed at any point.
Ascendis Pharma clinical development vice president David Karpf said: “With TransCon PTH, our goal has always been to improve the lives of patients with hypoparathyroidism, by designing a replacement therapy that restores physiologic levels of PTH 24 hours a day and that sets a new standard of care.
“These PaTH Forward data show the potential to completely remove standard of care while maintaining normal serum and urinary calcium levels, which could represent a major advance for patients with this complex, debilitating disease.”
Ascendis Pharma revealed plans to engage with regulatory authorities across the world on further steps for the development of TransCon PTH apart from submitting regulatory filings to begin a global phase 3 trial across North America, Europe, and Asia in the fourth quarter of this year.
Ascendis Pharma president and CEO Jan Mikkelsen said: “Hypoparathyroidism has a significant negative impact on over 200,000 patients worldwide and is one of the last hormonal insufficiency disorders without an effective replacement therapy. With TransCon PTH, we have a unique opportunity to make a meaningful difference for patients on a global basis.”
