For patients with hypoparathyroidism, TransCon PTH normalizes calcium
TransCon PTH normalized calcium levels in the blood and urine of adults with hypoparathyroidism and eliminated the need for vitamin D supplements and high doses of calcium, according to over a year and a half of data from trial.
These data – totaling 84 weeks – come from the global Phase 2 PaTH Forward trial, which involved 59 patients with hypoparathyroidism, and its open label extension.
“We believe that these data, combined with the fact that almost all of the patients continue in the open end of the trial, indicate 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, MD, chief medical officer at Ascendis Pharma, the developer of the experimental therapy, in a press release.
The company is also evaluating TransCon PTH in 76 adults with hypoparathyroidism in the global Phase 3 PaTHway trial (NCT04701203). Data from this trial is expected in early 2022.
If it is positive, Ascendis hopes to submit a new drug application or NDA to the United States Food and Drug Administration (FDA) later next year for regulatory approval for TransCon PTH.
“We look forward to sharing our key Phase 3 data in the first quarter of 2022 and, if positive, expect to complete an NDA submission to the FDA in mid-2022,” Pizzuti added.
TransCon PTH is a precursor to the parathyroid hormone, known as PTH, which occurs at low levels in people with hypoparathyroidism. Ascendis’ long-acting investigational therapy is designed to normalize calcium and phosphate levels.
Due to hypoparathyroidism, altered calcium and phosphate levels can lead to generalized weakness, muscle cramps, seizures, and abnormal tingling sensations in the lips, fingers and toes. It can also cause headaches, memory loss, and mood swings.
The four-week PaTH Forward Phase 2 trial (NCT04009291) evaluated the safety and efficacy of three daily doses of TransCon PTH – 15, 18, or 21 micrograms – given as an injection subcutaneously, compared to to a placebo.
TransCon PTH was found to be generally safe and well tolerated, and 82% of patients treated eliminated the need for conventional therapy – vitamin D supplements and up to 1000 mg of calcium per day.
In addition, 50% of those who received the treatment achieved all of the trial’s main goals: the elimination of conventional therapies as well as the normalization of calcium levels in the blood and urine.
Of the 59 participants in PaTH Forward, all but one, 58, participated in the open-label extension study, in which all patients received the treatment. After more than a year and a half of TransCon PTH treatment, the average calcium levels in the blood and urine have remained stable and within the normal range.
All of the study participants stopped taking vitamin D supplements within the first few weeks of the trial and no longer needed this conventional treatment. In addition, 93% of patients took 600 mg or less of calcium supplements.
At all doses, TransCon PTH was well tolerated with no serious or serious treatment-related events (side effects) reported. In addition, no participant discontinued the study due to treatment-related adverse events.
“The long-lasting and continuous improvements seen in this study over a year and a half of treatment with TransCon PTH underscore the importance of a sustained recovery of parathyroid hormone to normal physiological levels,” said Professor Peter Schwarz, MD, professor of endocrinology at Rigshospitalet and at the University of Copenhagen. , in Denmark.
“Parathyroid hormone plays an essential role in the regulation of serum and urinary calcium and phosphate, thus impacting the function of nerves and muscles, and I am delighted that we may soon be able to offer a new treatment paradigm for addressing the health issues and quality of life issues that these patients face every day, ”added Schwarz.