Ascendis Pharma A/S, a clinical-stage biopharmaceutical company, has recently announced favorable top-line results from a global phase 2 trial. The trial with the fixed dose of 4 weeks, blinded portion of PaTH Forward, evaluates the efficacy, safety and tolerability of TransCon™ PTH among adults with HP (hypoparathyroidism).
TransCon PTH is an investigational prodrug of PTH (parathyroid hormone) and a replacement therapy for the adult hypoparathyroidism. It has been designed to replace the parathyroid hormone at physiologic levels for 24 hours daily and address both long-term complications and short-term symptoms of HP.
A total number of 59 adult subjects had been randomized for TransCon PTH’s fixed doses at 15, 18 or 21 µg per day or placebo for 4 weeks by using a prefilled pen injector. All doses were well-tolerated without any sign of adverse impacts at any point. In addition, there were no dropouts during the fixed dose period of 4 weeks. No TEAEs (treatment-emergent adverse events) disrupted the trial process of the investigational drug. The overall TEAEs incidence was also comparable between placebo and TransCon PTH.
According to David B. Karpf, M.D., VP of Clinical Development at Ascendis Pharma, the investigational prodrug TransCon PTH has been adopted to treat hypoparathyroidism patients by developing a replacement therapy which restores PTH’s physiologic level 24 hours each day. The data highlights the potential of the drug to remove the standard of care while maintaining the urinary calcium and serum levels at normal.
The study results demonstrated that the investigational prodrug can increase the level of serum calcium, discontinue calcium reduction and active D over the 4-week period. Despite the increased levels of serum calcium, it leads to the reduction of urinary calcium excretion as well as reductions of calcium-phosphate and serum phosphate products. At the 4th week, in the per protocol analysis, the TransCon PTH dosage arms and 21 µg/day arm showed a significant response with p-value less than 0.05 in the primary composite endpoint as compared to the placebo.