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BridgeBio(BBIO) - 2024 Q4 - Annual Results
BBIOBridgeBio(BBIO)2025-02-20 12:31

Market Size and Opportunity - The global ATTR market is estimated to be 15B−15B-20B, with significant growth driven by increased disease awareness and adoption of non-invasive diagnostic tools[17][18] - Infigratinib targets a 4B+marketopportunitywith55,000+peopleintheUS/EUwithachondroplasiaandhypochondroplasia[42]−BBP−418targetsa4B+ market opportunity with 55,000+ people in the US/EU with achondroplasia and hypochondroplasia[42] - BBP-418 targets a 1B+ market opportunity with 7,000+ patients in the US/EU with LGMD2I/R9[48] - Encaleret targets a 2B+marketopportunitywith25,000+patientsintheUS/EUwithADH1and200,000+patientswithPSH[52][53]RegulatoryApprovalsandDesignations−AttrubyreceivedregulatoryapprovalforthetreatmentofadultpatientswithATTR−CMtoreducecardiovasculardeathandhospitalization[8]−InfigratinibreceivedBreakthroughTherapyDesignation(BTD)forAchondroplasia,BBP−418receivedRarePediatricDiseaseDesignation(RPDD)forLimb−GirdleMuscularDystrophy,andBBP−812receivedRegenerativeMedicineAdvancedTherapyDesignation(RMAT)forCanavanDisease[6]ClinicalTrialsandEnrollment−ThreefullyenrolledPhase3trials:FORTIFY(112patients),CALIBRATE(70participants),andPROPEL3(114participants)evaluatingBBP−418,Encaleret,andInfigratinibrespectively[5]−Phase3study(PROPEL3)ofInfigratinibforachondroplasiaisfullyenrolledwithLPLVexpectedin2H2025[43]−Phase3study(FORTIFY)ofBBP−418inLGMD2I/R9isfullyenrolledwithtoplineinterimanalysisdataexpectedin2H2025[49]−Phase3study(CALIBRATE)ofEncaleretforADH1isfullyenrolledwithtoplineresultsexpectedin2H2025[55]MarketAccessandDistribution−Attruby′smarketaccessstrategyincludesa28−dayfreetrial,independentspecialtypharmacydistribution,anddedicatedpatientassistanceprograms[26]PipelineandPortfolio−Thecompany′spipelineincludesAttruby(500,000+patients,2B+ market opportunity with 25,000+ patients in the US/EU with ADH1 and 200,000+ patients with PSH[52][53] Regulatory Approvals and Designations - Attruby received regulatory approval for the treatment of adult patients with ATTR-CM to reduce cardiovascular death and hospitalization[8] - Infigratinib received Breakthrough Therapy Designation (BTD) for Achondroplasia, BBP-418 received Rare Pediatric Disease Designation (RPDD) for Limb-Girdle Muscular Dystrophy, and BBP-812 received Regenerative Medicine Advanced Therapy Designation (RMAT) for Canavan Disease[6] Clinical Trials and Enrollment - Three fully enrolled Phase 3 trials: FORTIFY (112 patients), CALIBRATE (70 participants), and PROPEL3 (114 participants) evaluating BBP-418, Encaleret, and Infigratinib respectively[5] - Phase 3 study (PROPEL 3) of Infigratinib for achondroplasia is fully enrolled with LPLV expected in 2H 2025[43] - Phase 3 study (FORTIFY) of BBP-418 in LGMD2I/R9 is fully enrolled with topline interim analysis data expected in 2H 2025[49] - Phase 3 study (CALIBRATE) of Encaleret for ADH1 is fully enrolled with topline results expected in 2H 2025[55] Market Access and Distribution - Attruby's market access strategy includes a 28-day free trial, independent specialty pharmacy distribution, and dedicated patient assistance programs[26] Pipeline and Portfolio - The company's pipeline includes Attruby (500,000+ patients, 2B+ market), Infigratinib (55,000+ patients), BBP-418 (7,000+ patients), Encaleret (25,000+ patients), and BBP-812 (1,000 patients)[15] - BridgeBio Oncology Therapeutics has progressed two potentially first-in-class molecules into the clinic with a third expected in 1H 2025[65] Clinical Data and Efficacy - Attruby demonstrated a 42% reduction in cardiovascular-related hospitalization events and a 50% reduction in all-cause mortality and recurrent cardiovascular hospitalization events at Month 30[21] - Attruby's clinical data showed near-complete stabilization of TTR, with acoramidis being 4 times more potent than tafamidis at a fixed plasma concentration[22] - V122I ATTRv-CM has a median survival of 40 months compared to 60 months for ATTRwt-CM[34] Company Achievements and Milestones - Attruby team has successfully led $5B+ portfolio with 12 FDA approvals spanning 6 disease states and 7 indications[31] - BridgeBio expects to hit numerous milestones in 1H and 2H 2025, including EU and Japan approvals for Acoramidis and LPLV for Infigratinib and BBP-418[68] Ecosystem and Innovation - The company's ecosystem leverages a diversified portfolio, operational nimbleness, and world-class decentralized R&D to drive innovation[13][14] Market Penetration and Prescription - Attruby achieved 430 scripts to date, with 77% of Medicare lives in formulary position and 248 unique prescribing HCPs[28]