BridgeBio Pharma, Inc. products

Encaleret is an investigational small molecule antagonist of the calcium sensing receptor (CaSR) being studied in disorders of calcium homeostasis, including autosomal dominant hypocalcemia type 1 (ADH1). Individuals with ADH1 have gain-of-function mutations in the CaSR, causing low serum calcium and a range of debilitating symptoms. ADH1 may also lead to relatively high levels of calcium in urine, a condition called hypercalciuria, which can result in impaired kidney function and can cause kidney stone formation. Encaleret has been administered to approximately 1,300 healthy volunteers and osteoporosis patients, demonstrating tolerability and showing clear modification of ADH1 disease drivers, encouraging our investigation of the compound in ADH1 patients. Encaleret is a potential first-in-class CaSR antagonist for ADH1 and initiation of Phase 3 is planned in 2022.

Acoramidis (AG10) is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin (TTR). Acoramidis?was designed to mimic a?naturally-occurring?variant of the TTR gene (T119M) that is considered a “rescue mutation” because it has been shown to prevent or minimize ATTR in individuals carrying pathogenic, or disease-causing, mutations in the TTR gene. Results from the ongoing Phase 3 study investigating acoramidis for symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM), which includes mortality and cardiovascular-related hospitalizations, are expected in 2023.

BBP-711 is an orally-administered small molecule inhibitor of glycolate oxidase (GO) that is being developed to treat conditions of excess oxalate, including primary hyperoxaluria type 1 (PH1) and frequent kidney stone formation. In PH1, loss of function mutation in the AGXT gene results in accumulation of glyoxylate, which is converted into oxalate and leads to kidney stones and organ damage. Targeting GO is a clinically validated approach to reduce urinary oxalate by lowering the concentration of glyoxylate. The first-in-human Phase 1 trial of BBP-711 for hyperoxaluria was initiated in May 2021.