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BridgeBio Pharma Announces Positive Phase 1 Data and Phase 2/3 Trial Design for BBP-711, a Potentially Best-In-Class GO Inhibitor for Primary Hyperoxaluria Type 1 (PH1) and Recurrent Kidney Stone Formers
– BBP-711 led to near complete inhibition of glycolate oxidase throughout the dosing period and greater than 10-fold increases in plasma glycolate, suggesting
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"– BBP-711 led to near complete inhibition of glycolate oxidase throughout the dosing period and greater than 10-fold increases in plasma glycolate, suggesting it has the potential to be both a best-in-class therapy and the first oral therapy for PH1 and recurrent kidney stone formers – Based on the tolerability and potency of the oral therapy, BridgeBio has met with regulators and intends to initiate a Phase 2/3 pivotal study by the end of 2022 – At the end of 2022, BridgeBio also intends to launch a Phase 2 study of BBP-711 in adult recurrent kidney stone formers, which affects an estimated 1.5 million individuals in the United States and European Union PALO ALTO, Calif., June 27, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced positive Phase 1 data for BBP-711 in healthy volunteers, supporting the development of the investigational therapy for patients with primary hyperoxaluria type 1 (PH1) and recurrent kidney stone formers. The data were shared in a feature oral presentation at European Society for Pediatric Nephrology (ESPN) 2022, taking place in Ljubljana, Slovenia. BBP-711 is an orally-administered small molecule inhibitor of glycolate oxidase (GO) that is being developed to treat conditions of excess oxalate. Overproduction of oxalate in hyperoxaluria, including PH1 and recurrent kidney stone formers with elevated oxalate, can lead to kidney stone formation, nephrocalcinosis and renal impairment. PH1 affects an estimated 5,000 patients in the United States and European Union, while recurrent stone formers are much more common, affecting an estimated 1.5 million individuals in the United States and European Union. “PH1 is a severe genetic condition with a high degree of morbidity, including kidney stones, calcification in the kidneys and deposition of calcium oxalate in many vital organs. Despite major advances in the field of hyperoxaluria, PH1 remains a disease with high unmet medical need in many parts of the world,” said Scott Adler, M.D., chief medical officer and vice president of clinical development at Cantero Therapeutics, the BridgeBio affiliate developing BBP-711. “These data are encouraging for patients with PH1 as BBP-711 could become a novel oral therapy to prevent hyperoxaluria and its long...