BridgeBio(US:BBIO)2021-05-27 19:04Pipeline Highlights - BridgeBio aims to have 4+ NDAs on file, targeting blockbuster markets[6] - The company is targeting $1B+ opportunities in its pipeline, including Acoramidis for ATTR CM and PN, and low-dose infigratinib for achondroplasia[12] - BridgeBio has a deep pipeline of 30+ R&D programs[6] - The company anticipates positive pivotal data in a multi-billion market and positive POC data in multiple blockbuster indications[30] Clinical Programs & Data - Encaleret Phase 2 data showed normalization of blood calcium, PTH, and phosphate levels, and reduction of urinary calcium excretion in ADH1 patients[63] - Acoramidis Phase 2 data demonstrated near-complete TTR stabilization[72] - ATTRibute-CM Phase 3 study enrolled 632 participants with topline data expected in late 2021 or early 2022[91] - Low-dose infigratinib improved key drivers of clinical symptomology in a validated achondroplasia mouse model, showing a 17% increase in FM area, 12% increase in L4-L6 length, and 21% increase in femur length[103] - BBP-631: AAV5 gene therapy for CAH is targeting a market of 75,000 (US+EU) patients[110] Oncology Research - BridgeBio's SHP2 inhibitor (BBP-398) has a human half-life of ~10-15 hours and is being developed for multiple tumors, with a potential market of >500K patients[131] - The company's pan-mutant KRAS inhibitor (BBP-454) is also targeting multiple tumors, with a potential market of >500K patients[23]