BridgeBio initiates Phase 3 trial CALIBRATE study of encaleret

BridgeBio Pharma announced the initiation of its multicenter, international, pivotal Phase 3 randomized trial of encaleret in patients with autosomal dominant hypocalcemia type 1, ADH1, CALIBRATE. The design of the CALIBRATE study incorporates feedback from global regulatory authorities and patients, with a primary composite endpoint of blood and urine calcium concentrations within normal ranges in participants treated with encaleret compared to standard of care, SoC. SoC for ADH1 consists of extra-dietary supplementation with calcium and/or active vitamin D analogs. While SoC may address acute symptoms related to hypocalcemia, it may exacerbate high urine calcium levels, which can cause renal complications. Secondary endpoints will evaluate other measures of mineral homeostasis, quality of life, and kidney function. "We are excited to have reached this important milestone in the development of encaleret," said Mary Scott Roberts, executive director of clinical development at BridgeBio and clinical lead of the encaleret program. "The pivotal CALIBRATE study will evaluate the potential for encaleret to provide a meaningful benefit to patients and families living with ADH1. If successful, this study could lead to an approval of encaleret for the treatment of ADH1, a disease with significant unmet medical need and no interventions specifically indicated for its treatment."