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BridgeBio Pharma Initiates CALIBRATE, A Pivotal Phase 3 Study of Encaleret in Autosomal Dominant Hypocalcemia Type 1
- The CALIBRATE Phase 3 study design incorporates feedback from global regulatory agencies and will compare the effects of encaleret to standard of care on
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"- The CALIBRATE Phase 3 study design incorporates feedback from global regulatory agencies and will compare the effects of encaleret to standard of care on blood and urinary calcium concentrations over a 24 week treatment period in patients with autosomal dominant hypocalcemia type 1 (ADH1) - The primary composite endpoint evaluates the proportion of participants with ADH1 achieving blood and urinary calcium within widely accepted normal ranges - In an ongoing single arm, open-label, Phase 2 safety and efficacy study of patients with ADH1, 69% of participating patients achieved concurrent values of both blood calcium and 24hr urinary calcium excretion in the reference range. None of these individuals had attained this dual therapeutic goal while on standard of care - If approved, encaleret would be the first therapy specifically indicated for ADH1 - Population genetics analyses estimate that there are approximately 25,000 carriers of gain-of-function variants of the calcium sensing receptor (CaSR), the underlying cause of ADH1, in the United States (US) and European Union (EU) - Current standard of care (oral supplements of calcium and/or vitamin D) inadequately addresses both the short- and long-term clinical manifestations related to hypocalcemia and hypercalciuria PALO ALTO, Calif., Dec. 22, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today 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 complications1. Secondary endpoints will evaluate other measures of mineral homeostasis, quality of life, and kidney function. “We are excited to have reached this important mile...