Eneboparatide met primary endpoint of normalising serum calcium in adults with hypoparathyroidism at 24 weeks in CALYPSO Phase III trial
Trial continues as planned to 52 weeks to further characterise the risk-benefit profile
High-level results from the CALYPSO Phase III trial showed that eneboparatide (AZP-3601), an investigational parathyroid hormone (PTH) receptor 1 agonist, met its primary endpoint with statistical significance in adults with chronic hypoparathyroidism (HypoPT) at 24 weeks, compared to placebo. The primary endpoint is a composite of normalisation of albumin-adjusted serum calcium levels and independence from active vitamin D and oral calcium therapy.
HypoPT is a rare endocrine disease caused by a deficiency of PTH and characterised by impaired regulation of calcium and phosphate levels in the blood. This dysregulation of the physiological action of PTH can lead to clinical manifestations, including negative impact on the kidney and bone.1 HypoPT is one of the largest known rare diseases, affecting over 200,000 people in the United States and the European Union, approximately 80% of whom are women.2-4
Marc Dunoyer, Chief Executive Officer, Alexion, AstraZeneca Rare Disease, said: “People living with HypoPT, a rare endocrine disease, are often at increased risk of hypercalciuria, osteopenia and osteoporosis, and these results from the CALYPSO trial underscore eneboparatide’s potential to be another option for these patients. We look forward to reviewing clinical results at 52 weeks to fully characterise the risk-benefit profile.”
Eneboparatide was well tolerated. After the 24-week randomised treatment period, all patients receive eneboparatide in the ongoing long-term extension period until 52 weeks. Full efficacy and safety data will be analysed at 52 weeks. Alexion plans to share these data with global health authorities and present them at forthcoming medical meetings.
References
1. Bilezikian JP. Hypoparathyroidism. J Clin Endocrinol Metab. 2020;105(6):1722-1736. doi:10.1210/clinem/dgaa113.
2. Vadiveloo T, et al. A population-based study of the epidemiology of chronic hypoparathyroidism. J Bone Miner Res. 2018;33(3):478-485.
3. Villarroya-Marquina I, et al. Influence of gender and women’s age on the prevalence of parathyroid failure after total thyroidectomy for multinodular goiter. Gland Surg. 2020;9(2):245-251.
4. Deering KL, et al. Economic burden of patients with post-surgical chronic and transient hypoparathyroidism in the United States examined using insurance claims data. Orphanet J Rare Dis. 2024;19(1):164.