BridgeBio Reports Positive Phase 3 Topline Results for Encaleret in Patients with Autosomal Dominant Hypocalcemia Type 1

Core Insights - The CALIBRATE study of encaleret for patients with ADH1 met all pre-specified primary and key secondary efficacy endpoints [1] - Encaleret demonstrated significant efficacy in achieving target serum and urine calcium levels compared to conventional therapy [2] - The drug was well-tolerated with no discontinuations related to the study drug [1] Efficacy Results - 76% of participants treated with encaleret achieved both serum and urine calcium within target ranges at Week 24, compared to 4% on conventional therapy at Week 4 (p<0.0001) [1][2] - In a key secondary analysis, 91% of encaleret-treated participants had intact PTH above the lower limit of the reference range at Week 24, compared to 7% on conventional therapy at Week 4 (p<0.0001) [1] - By Day 3 after randomization, 71% of encaleret participants had an albumin-corrected serum calcium within the reference range [4] Safety and Tolerability - Encaleret was well-tolerated, with safety findings consistent with known ADH1 biology [12] - 65 of 67 randomized participants (97%) chose to continue in the long-term extension of the study [5] Future Plans - The company plans to submit a New Drug Application (NDA) to the FDA in the first half of 2026 [1][6] - Registrational studies of encaleret in chronic hypoparathyroidism and pediatric ADH1 are planned for 2026 [1][6] Study Design - The CALIBRATE study enrolled 70 adults with ADH1, randomizing 67 participants in a 2:1 ratio (encaleret: standard of care) [4] - The primary endpoint was defined as the proportion of participants achieving target serum and urine calcium levels [2]