BridgeBio(BBIO) - 2025 Q4 - Annual Report

Financial Performance - Attruby generated $362.4 million in U.S. net product revenues in 2025, reflecting increasing patient adoption and prescriber utilization [44]. - Beyonttra contributed $105 million in license and services revenue in 2025 following approvals in Europe and Japan [47]. - BridgeBio has obtained FDA approval for three products and has treated over 8,500 patients with its approved medicines [21]. - The company received gross cash proceeds of $500.0 million in December 2024 following FDA approval of Attruby on November 22, 2024 [184]. Market Potential - The total global addressable market for ATTR therapeutic interventions could exceed $20.0 billion, with diagnosed ATTR-CM patients in the U.S. growing from fewer than 5,000 in 2019 to more than 50,000 in 2025 [48]. - The estimated global prevalence of ATTRwt-CM and ATTRv-CM is over 400,000 and 40,000 patients, respectively, indicating significant underdiagnosis in the population [53]. - The market opportunity for achondroplasia and other FGFR-driven skeletal dysplasias is estimated to exceed $5.0 billion globally [82]. - The total global market opportunity for hypochondroplasia is expected to approach that of achondroplasia, driven by growing awareness and clinical trials [120]. Clinical Development - Positive Phase 3 results for infigratinib in achondroplasia, BBP-418 in limb-girdle muscular dystrophy type 2I/R9, and encaleret in autosomal dominant hypocalcemia type 1 are expected to lead to NDA submissions in 2026 [42]. - The Phase 3 PROPEL 3 study for low-dose infigratinib showed a statistically significant improvement in annualized height velocity of +2.10 cm/year compared to placebo [79]. - Enrollment in the Phase 2 portion of the ACCEL 2/3 trial for hypochondroplasia has been completed, with proof-of-concept results expected in the second half of 2026 [122]. - The company plans to initiate a registrational Phase 3 study for encaleret in pediatric ADH1 in Q1 2026 [99]. Regulatory and Compliance - The company is subject to extensive government regulations regarding the research, development, and marketing of drug products, which require significant time and financial resources [186]. - The FDA regulates drugs and biologics under the Federal Food, Drug, and Cosmetic Act, requiring substantial data to demonstrate quality, safety, and efficacy before marketing [188]. - The FDA targets ten months for the initial review of a new molecular entity NDA or original BLA, and six months for priority review applications [203]. - Manufacturers must comply with ongoing FDA regulations post-approval, including monitoring adverse experiences and adhering to advertising standards [219]. Competitive Landscape - Acoramidis (Attruby/Beyonttra) competes with Pfizer's Vyndaqel and Alnylam's vutrisiran for the treatment of ATTR-CM [137]. - The company faces competition from established pharmaceutical and biotechnology companies, which may have greater resources [135][136]. Partnerships and Agreements - Bayer License Agreement granted an exclusive license for acoramidis with an upfront payment of $135.0 million and potential milestone payments up to $600.0 million through 2026 [156]. - Eidos received an upfront payment of $25.0 million from Alexion for the license to commercialize Beyonttra in Japan, with a regulatory milestone payment of $30.0 million recognized in June 2025 [160]. - QED entered into a license agreement with Kyowa Kirin for infigratinib, receiving an upfront payment of $100.0 million and potential milestone payments up to $81.4 million [168]. Product Development and Innovation - The decentralized hub-and-spoke model allows multiple programs to advance concurrently, enhancing the probability of success over time [33]. - BBP-812 has the potential to become the first disease-modifying therapy for Canavan disease, having received multiple designations from the FDA and EMA [124]. - BridgeBio has initiated a next-generation depleter program for ATTR-CM, which could expand the addressable patient population by removing existing amyloid deposits [127].