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BridgeBio Pharma Announces Updated Phase 2B Data for Encaleret in Autosomal Dominant Hypocalcemia Type 1 (ADH1) Demonstrating Blood and Urine Calcium Normalization in Trial Participants
- Data presented at American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting - Achieving simultaneous blood and urine calcium normalization
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"- Data presented at American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting\n - Achieving simultaneous blood and urine calcium normalization is a challenge for patients with ADH1 due to the limitations of current standard-of-care\n - Encaleret could be the first approved therapy indicated specifically for the treatment of ADH1 if the development program is successful\n\n\nPALO ALTO, Calif., Oct. 1, 2021 /PRNewswire/ -- BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, today announced updated results from its ongoing Phase 2b proof-of-concept, open-label study of encaleret for the treatment of Autosomal Dominant Hypocalcemia Type 1 (ADH1). Within five days of individualized dose titration in 13 participants, encaleret normalized mean blood calcium levels and 24 hour urine calcium excretion. The results are featured in an oral presentation titled 'The Effects of Encaleret (CLTX-305) on Mineral Physiology in Autosomal Dominant Hypocalcemia Type 1 (ADH1) Demonstrate Proof-of-Concept: Early Results from an Ongoing Phase 2B, Open-Label, Dose-Ranging Study' at the American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting, taking place in San Diego, California on October 1 – 4, 2021.\n\n \n \n \n \n \n \n\n \n\"Autosomal dominant hypocalcemia type 1 is a rare genetic form of hypoparathyroidism caused by gain-of-function variants of the calcium-sensing receptor (CASR) gene. The current standard of care consists of calcium and active vitamin D supplements, which do not address the root cause of ADH1,\" said Rachel Gafni, M.D., Senior Research Physician and Head, Mineral Homeostasis Studies Group of the National Institute of Dental and Craniofacial Research, National Institutes of Health (NIH). \"These updated results from the ongoing Phase 2b study of encaleret demonstrate consistent improvements in mineral homeostasis and support further study.\"\nIn this update from the ongoing Phase 2b open-label, dose-ranging study, 13 adults with ADH1 with nine distinct CASR variants were administered encaleret. Calcitriol (active Vitamin D) and extra-dietary calcium supplementation beyond the recommended daily intake (current standard of car...