BridgeBio Pharma, Inc. Announces Updated Phase 2B Data for Encaleret in Autosomal Dominant Type 1 Hypocalcemia Demonstrating Normalization of Blood and Urinary Calcium in Trial Participants



BridgeBio Pharma, Inc. announced updated results from its ongoing Phase 2b open-concept proof-of-concept study of encaleret for the treatment of autosomal dominant hypocalcemia type 1 (ADH1). Within five days of individual titration of the dose in 13 participants, the encaleret normalized the mean levels of calcium in the blood and the urinary excretion of calcium over 24 hours. The results are presented in an oral presentation entitled “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 2021 American Society for Bone and Mineral Research (ASBMR) Annual Meeting, to be held in San Diego, Calif. On October 1? 4, 2021. In this update of the ongoing Phase 2b open-label dosing study, 13 adults with ADH1 with nine distinct CASR variants received encaleret. Calcitriol (active vitamin D) and extra-dietary calcium supplementation above the recommended daily allowance (current standard of care) were discontinued during the study. During the inpatient observation periods of defined dose escalation and individualized dose titration, encaleret was well tolerated without any serious adverse events, severe adverse events or discontinuation of treatment due to ‘reported adverse events. In 13 trial participants, encaleret normalized mean blood calcium levels and 24-hour urinary calcium excretion during periods 1 and 2. Mineral responses after administration of encaleret demonstrate that encaleret may become an effective treatment option for ADH1 patients. At ASBMR 2021, BridgeBio will also present a retrospective systematic review of ADH1 literature and clinical study designs for its PROPEL and PROPEL2 studies of low-dose infigratinib in people with achondroplasia, which is the most common form of small genetic size with a prevalence of greater than 55,000 cases in the United States and the European Union. Low-dose infgratinib is the only known product candidate in clinical development for achondroplasia designed to target disease at its genetic source and the only orally administered product candidate in clinical development. BridgeBio’s experimental therapies for ADH1 and achondroplasia are two of the company’s 14 programs that are advanced in clinical or commercial settings for patients living with genetic diseases and cancers of genetic origin. BridgeBio’s first wave of programs are for drugs now approved for molybdenum cofactor deficiency (MoCD) type A and previously treated locally advanced or metastatic cholangiocarcinoma (CCA) harboring FGFR2 fusion or rearrangement. The second wave of programs includes the Company’s four main short-term catalysts for its product candidates for the treatment of ADH1 and achondroplasia, as well as transthyretin amyloidosis (TTR) (ATTR) and l congenital adrenal hyperplasia (CAH). The third wave under development of BridgeBio includes a variety of cancer and Mendelian space programs already in clinical practice.


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