BridgeBio JPM Presentation_January 2025.pdf

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1、J.P.Morgan PresentationJanuary 13,20252Forward Looking Statements and DisclaimerThe presentation contains forward-looking statements.Statements made or presented may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securitie

2、s Act of 1933,as amended,and Section 21E of the Securities Exchange Act of 1934,as amended.Words such as“believe,”“anticipate,”“plan,”“expect,”“intend,”“will,”“may,”“goal,”“potential,”“should,”“could,”“aim,”“estimate,”“predict,”“continue”and similar expressions or the negative of these terms or othe

3、r comparable terminology are intended to identify forward-looking statements,though not all forward-looking statements necessarily contain these identifying words.We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Sectio

4、n 27A of the Securities Act and Section 21E of the Exchange Act.These forward-looking statements,including express and implied statements relating to the commercial success of Attruby,the timing of ongoing clinical trials,including BridgeBio Oncology Therapeutics and Gondola Bios clinical trials,the

5、 clinical,therapeutic and market potential of our clinical development programs and our pipeline,BridgeBio Oncology Therapeutics pipeline and Gondola Bios pipeline,our speed of creating new and meaningful drugs and related impact on patients,the efficiency of our engine to rapidly and efficiently de

6、liver medicines,our value creation potential for patients,the potential market sizes and opportunities,the safety,efficacy and mechanisms of our newly FDA-approved Attruby(acoramidis)and other later-stage products including infigratinib,BBP-418 and encaleret,the timing of approval of Attruby for ATT

7、R-CM in the European Union and Japan,our financial position,including our expectations regarding reaching regulatory milestones and the receipt of milestone payments,the potency and safety of our product candidates,the potential benefits of our product candidates,the potential for greater patient ac

8、cess to medications,the affordability and availability of insurance coverage of our medications,and the timing and expectations regarding results of our various clinical trials,reflect our current views about our plans,intentions,expectations and strategies,which are based on the information current

9、ly available to us and on assumptions we have made.Such statements reflect the current views of the Company with respect to future events and are subject to known and unknown risks,including business,regulatory,economic and competitive risks,uncertainties,contingencies and assumptions about the Comp

10、any,including,without limitation,risks inherent in developing therapeutic products,and those risks and uncertainties described under the heading“Risk Factors”in the Companys most recent Annual Report on Form 10-K filed with the U.S.Securities and Exchange Commission(“SEC”)and in subsequent filings m

11、ade by the Company with the SEC,which are available on the SECs website at www.sec.gov.In light of these risks and uncertainties,many of which are beyond the Companys control,the events or circumstances referred to in the forward-looking statements,express or implied,may not occur.The actual results

12、 may vary from the anticipated results and the variations may be material.You are cautioned not to place undue reliance on these forward-looking statements,which speak to the Companys current beliefs and expectations only as of the date of the presentation.Except as required by law,the Company discl

13、aims any intention or responsibility for updating or revising any forward-looking statements made or presented at the presentation in the event of new information,future developments or otherwise.No representation is made as to the safety or effectiveness of the product candidates for the therapeuti

14、c use for which such product candidates are being studied.Certain information communicated at the presentation may relate to or is based on studies,publications,surveys and other data obtained from third-party sources and the Companys own internal estimates and research.While the Company believes th

15、ese third-party sources to be reliable as of the date of the presentation,it has not independently verified,and makes no representation as to the adequacy,fairness,accuracy or completeness of,any information obtained from third-party sources.In addition,certain information to be communicated at the

16、presentation involves a number of assumptions and limitations,and there can be no guarantee as to the accuracy or reliability of such assumptions.Finally,such research has not been verified by any independent source.Such information is provided as of the date of the presentation and is subject to ch

17、ange without notice.The Company has not verified,and will not verify,any part of this presentation,and the Company makes no representation or warranty,express or implied,as to the accuracy or completeness of the information to be communicated at the presentation or as to the existence,substance or m

18、ateriality of any information omitted from the presentation at the presentation.The Company disclaims any and all liability for any loss or damage(whether foreseeable or not)suffered or incurred by any person or entity as a result of anything contained or omitted from this document or the related pr

19、esentation and such liability is expressly disclaimed.3Key recent achievements:clinical impact4Key recent achievements:3 fully enrolled Phase 3 trialsTrial enrolled 112 patients to evaluate BBP-418 in Limb-Girdle Muscular Dystrophy 2I/R9 Trial enrolled 70 participants to evaluate encaleret in Autoso

20、mal Dominant Hypocalcemia Type 1Trial enrolled 114 participants to evaluate infigratinib in AchondroplasiaNEW INFORMATION5Key recent achievements:regulatory advancement for late-stage programsReceives Breakthrough Designation(BTD)for AchondroplasiaReceives Rare Pediatric Disease Designation(RPDD)for

21、 Limb-Girdle Muscular Dystrophy 2I/R9Receives Regenerative Medicine Advanced Therapy Designation(RMAT)for Canavan DiseaseInfigratinib BBP-418BBP-8126Key recent achievement:Attruby regulatory approvalIndicated for the treatment of adult patients with ATTR-CM to reduce cardiovascular death and cardiov

22、ascular-related hospitalization7Remarkable commercial momentum430Attruby ScriptsNEW INFORMATION8BridgeBios growth is underpinned by an innovative corporate model,as highlighted in a recent publicationApplications of Portfolio Theory to Accelerating Biomedical InnovationNeil Kumar,Andrew W.Lo,Chinmay

23、 Shukla,and Brian StephensonOperational nimblenessCentralized infrastructure for functions with economies of scale Hyper-focused and agile decision makingCreative financing toolkitToolkit of strategic options(asset&portfolio levels)Costs are variablized and flexible across central functions and affi

24、liatesWorld class,decentralized R&DLeverage hyper-experienced,science-focused R&D practitionersAsset-level incentives preserve biology-level focusDiversified portfolioUncorrelated assets enable multiple“shots on goal”Modality and therapeutic area agnostic selection of programs9The BridgeBio ecosyste

25、m has a dynamic pipeline of productsProgramIndicationPre-clinicalPhase 1Phase 2Phase 3 ApprovedPatients(US+EU)Market OpptyEstimated US LOE*Attruby(acoramidis)Transthyretin Amyloidosis(ATTR-CM)500,000+$20B+2039InfigratinibAchondroplasia(ACH)Fully Enrolled55,000+$2B+2041Hypochondroplasia(HCH)55,000+$2

26、B+2041BBP-418Limb-Girdle Muscular Dystrophy Type 2I/R9(LGMD2I/R9)Fully Enrolled7,000+$1B+2041EncaleretAutosomal Dominant Hypocalcemia Type 1(ADH1)Fully Enrolled25,000+$1B+2041Post-Surgical Hypoparathyroidism(PSH)200,000+$1B+2044BBP-812Canavan Disease1,000TBD2039BridgeBio Oncology TherapeuticsOncolog

27、y,variousVariousVariousVariousGondolaBioRare disease,variousVariousVariousVariousPhase 1/2 Pivotal45%ownership*38%ownership*BridgeBio Oncology Therapeutics and GondolaBio are separate,independent companies from BridgeBio.BridgeBios initial interest in GondolaBio is subject to reduction as additional

28、 tranches of capital contributions are funded.*Approximate estimated US LOE dates considering various factors including issued patents,pending patent applications,potential patent term extensions,and regulatory exclusivities.11Market growth drivers include:With more sponsors,there is expanding disea

29、se awareness Increased global adoption of non-invasive diagnostic toolsGlobal annual ATTR market sales1$B$0.7$1.7$2.6$3.2$4.3201920202021202220232024Peak Year$6.4$15-$202+57%1ATTR market includes all approved drugs for ATTR-PN and ATTR-CM.2024 sales annualized as of Q3 2024.2Consensus estimates of$1

30、5B-$20B ATTR market.ATTR is a multi-billion-dollar market primed for continued expansion12+Compelling Clinical Data1Market Access:Make it Easy2The way we win13The most rapid benefit seen in any Phase 3 study of ATTR-CM to dateIn composite of all-cause mortality and recurrent cardiovascular-related h

31、ospitalization events at Month 30In the cumulative frequency of cardiovascular-related hospitalization events at Month 303 Months42%Reduction50%ReductionKey Data114Data:Attruby achieved near-complete stabilization of TTR1Maurer,M.et al.,Eur.Heart Jour,2024;45(Suppl 1).2Nelson,L.et al.,Amyloid,2021;2

32、8(1):24-29.3Penchala,S.et al.,PNAS,2013;110(24):9992-97.Preclinical signals of superior potency&selectivity“We carried out the subunit exchange in human plasma to address the relative selectivity of AG10 acoramidis vs.tafamidisit is obvious that tafamidis is inferior to AG10 acoramidis,but nothing l

33、ike the degree you claim it is.”Prof.Jeffery Kelly(inventor of tafamidis)in email correspondence with Dr.Isabella Graef,February 27,2013.1Differential TTR selectivity“It is clear that AG10 acoramidis binds more selectively to TTR in serum than tafamidis.”3Evidence of potency on TTR stabilization“AG1

34、0 acoramidis is 4 times more potent than tafamidis at a fixed plasma concentration(e.g.,10 M).”2Increase in serum TTR levels reflect in vivo stabilization“Change from baseline in serum TTR levels were higher in participants receiving acoramidis only than those receiving placebo+tafamidis at Month 30

35、.”115Make it easy:simplified,differentiated,generous access programs28-day supply for patients new to Attruby(newly diagnosed or switching from another therapy)Independent and rare/orphan diseasefocused Specialty Pharmacies(SPs)where Institutional SPs can keep prescriptionDedicated Attruby Field Rei

36、mbursement Managers and Patient Access Liaisons Easily accessible Copay and Patient Assistance ProgramFree TrialDistribution NetworkField Access TeamsPatient Assistance ProgramsCommitment to clinical trial patientsUS patients who participated in the acoramidis clinical trials may receive Attruby at

37、no cost for the duration of their medically indicated treatment216Performance to date indicates strong commercial momentum430 Attruby scripts written to date248 Unique prescribing HCPs 77%Medicare lives in equal formulary position to tafamidis17You priced responsibly and have a good label;we will ha

38、ve you at parity with tafamidis.Also,I have never seen a company offer free drug to their trial patients.Shows commitment.”-Payer“You did the right thing by being less than tafamidis both your label and price will play a role here.”-Payer“I want to reemphasize our gratitude for LDN inclusion.We obje

39、ctively see key metrics in the patient journey improve as a result of dispensing accessEager to leverage our resources to impact patient care in a positive way.”Manager of Health System SP“You are a smart company to set up your distribution this way.I wish all companies would do this!”Director of He

40、alth System SP“BridgeBios thoughtful resources and the structure of the networkaligns with the health systems mission of putting patients first.Pharmacist Holy Smokes!?I wish all manufacturers would provide this information.Do you have any idea how much time this is going to save our team?Pharmacist

41、“Given the combination of strong efficacy and safety data,alongside BridgeBios very health system friendly approach towards access for this product(in comparison to the many challenges we have had with Pfizer),we plan to begin preferring Attruby.AD Specialty Clinical Program Development“It takes les

42、s than 5 minutes-the easiest enrollment process of any drug we use.RN Heart Failure Coordinator“Pricing:Limited Distribution Network:ForgingBridges:“Positive feedback all around on Market Access:18Scott CollinsSVP,Market AccessAna MerzVP,SalesLaunched IMBRUVICA($5B+,12 FDA approvals,6 disease states

43、,7 indications in 10 years)and EPKINLY(3L+DLBCL)Liz ArnoldHead of Commercial StrategyMultiple global launches,expertise in strategy,consulting,and marketing across rare disease,hematology,and OTC Sean DohertySVP,MarketingBroad global sales and marketing launch experience including in rare,infectious

44、,and auto-immune diseasesHudson BoyerVP,Commercial Analytics&OpsLaunches in rare disease,hematology,and immunology;strategy consulting and equity analyst backgroundExtensive market access experience with consistent coverage acrossrare disease and oncology,leading large field-based access teamsJulie

45、EverettChief Business OfficerSuccessfully led cross-functional teams through multiple rare disease launches,including VOXZOGO and PALYNZIQ(BioMarin)Led commercial strategy/execution across$1B portfolio Decade of strategy consulting leadership focused on launch excellence and lifecycle maximization(T

46、rinity)John WhangChief Medical Affairs OfficerOrchestrated multiple successful launches with pioneering therapies in competitive segments STELARA(Janssen),REPATHA(Amgen),and CAMZYOS(BMS)8+launches as strategy consultant(McKinsey)Demonstrated strategic innovation(Heartline Study J&J/Apple collab)and

47、consistently built outstanding organizationsMatt OuttenChief Commercial OfficerBroad commercial leadership expertise with success across multiple competitive marketsLed$5B+portfolio,12 FDA approvals spanning 6 disease states and 7 indications(IMBRUVICA,Pharmacyclics)Commercial lead on$21B pharma M&A

48、 dealOur BridgeBio team is committed to providing industry-leading access and white glove service for all parties looking to bring Attruby to patients with ATTR-CM.Our Attruby team has experienced industry leaders who have built and launched blockbuster drugs19Looking Ahead:Attruby Delivers Outstand

49、ing Results in Patients with Poor Prognosis0102030405060Median Survival(months)ATTRwt-CMATTRv-CM(V122I)V122I ATTRv-CM has an aggressive phenotype and poor prognosis1N=413Statistically significant benefit on composite ACM or first CVH in ATTRv-CM participants vs.placebo2N(%)Hazard Ratio(95%CI)p value

50、Overall Population611(100%)0.65(0.50-0.83)0.0008ATTRv-CM59(9.7%)0.41(0.21-0.81)0.0109Natural HistoryATTRibute-CM mITT PopulationN=4131Razvi,Y.et al.,Eur Jour Heart Fail.,2024;26(2):383-393.2BridgeBio Data on File.Overall population and ATTRv-CM data vs.placebo.201Hornstrup,L.et al.Arterioscler Throm

51、b Vasc Biol,2013;33:1441-47.2Figure adapted from Christoffersen,M.et al.,JAMA Cardiology,2024.Hazard ratio(95%CI)TTR distributionAdditional data:Elevated TTR levels are associated with improved survivalHigher TTR concentration associated with greater life expectancy(N102K individuals)2Genetic stabil

52、ization of transthyretin associated with improved health outcomes(N69K individuals)1-WT-T119M CarriersInfigratinib22Design Criteria55,000+PeopleIn the US/EU with achondroplasia(ACH),the most common form of disproportionate short stature.55,000+PeopleIn the US/EU with hypochondroplasia(HCH),another f

53、orm of disproportionate short stature.Impact OpportunityPhase 2 Data$4B+Market OpportunityInfigratinib is an oral best-in-class FGFR3 inhibitor that targets achondroplasia and hypochondroplasia at their sourceAnnualized height velocity(To the sponsors knowledge,largest effect reported to date)Target

54、 FGFR3-driven skeletal dysplasias at their sourceProportionality(To the sponsors knowledge,largest effect reported to date)Demonstrate safety with low dosingWell-toleratedConvenient oral ROA to reduce patient burden23Key Secondary Endpoints:Change from baseline in height z-score Change from baseline

55、 in upper to lower body segment ratioPrimary Endpoint:Change from baseline in annualized height velocity at Wk 52Screening and Observational PeriodChildren are followed for a minimum of 6 monthsRandomization 2:1(N=114)Open Label ExtensionChildren are followed until adult height is reached0.25 mg/kg/

56、day Infigratinib(N 76)6 monthsPlacebo(N 38)12 monthsPhase 3 Trial Fully Enrolled;LPLV Expected 2H 2025We have fully enrolled a Phase 3 study(PROPEL 3)of Infigratinib in Achondroplasia with LPLV in 2H 2025BBP-41825Design Criteria7,000+PatientsIn the US/EU with LGMD2I/R9,a progressive neuromuscular di

57、sorder that leads to loss of ambulation,cardiomyopathy,and respiratory dysfunction.Impact OpportunityPhase 2 DataBBP-418 is a first-in-class,disease-modifying therapy positioned to be the first approval in any form of limb-girdle muscular dystrophyGlycosylated DG(Expected surrogate endpoint)Serum cr

58、eatine kinaseStabilization of clinical measuresWell-toleratedTargets the LMGD2I/R9 at its source by adding substrate to restore glycosylation of DGAvoid safety concerns by using a synthesized version of an endogenous compoundConvenient oral ROA to reduce patient burden$1B+Market Opportunity+addition

59、al opportunity with other forms of LGMD 26Screening Genetically confirmed,symptomatic LGMD2I/R9 12-60 years of ageRandomization 2:1(N 112)Open Label ExtensionBBP-418(N 75)BBP-418(N 75)Placebo(N 37)Placebo(N 37)12 months24 monthsFinal Analysis Endpoints:NSAD(primary)Ambulatory measures 10MWT 100MTT P

60、ulmonary function:FVC Upper limb function:PUL 2.0 QoL measuresInterim Endpoints:Glycosylated DG(primary)Serum creatine kinase(CK)Trends in clinical measuresInterim AnalysisTopline Data 2H 2025We have fully enrolled a Phase 3 study(FORTIFY)of BBP-418 in LGMD2I/R9 and expect topline interim analysis d

61、ata readout in 2H 2025Phase 3 Trial Fully Enrolled;LPLV&Topline Results Expected 2H 2025Encaleret28Design Criteria25,000+PatientsIn the US/EU with ADH1,a genetic disease resulting in the disruption of Ca homeostasis.200,000+PatientsIn the US/EU with Post-Surgical Hypoparathyroidism(PSH),a dysregulat

62、ion of Ca homeostasis caused by impaired parathyroid function.Impact OpportunityPhase 2 DataEncaleret is a first-in-class,disease-modifying investigational therapy with potential to be the first approved intervention for ADH1Parathyroid hormone to normal rangeSerum calcium to normal rangeWell-tolera

63、tedUrine calcium to normal rangeTarget ADH1 at its source by desensitizing over-activeCa sensing receptorsNormalize PTH,serum Ca,and urine Ca levelsConvenient oral ROA to reduce patient burden$2B+Market Opportunity29Select Secondary Endpoints:Blood iPTH,1,25-(OH)2 Vitamin D,magnesium,and phosphate U

64、rine magnesium and phosphate Bone turnover markers Renal ultrasound and renal function ER/urgent care visits and/or hospitalizations Quality of life(SF-36)Primary Endpoint:Proportion of participants achieving:o Blood Ca within the target range AND o 24-hour urine Ca within the reference rangeScreeni

65、ng and SoC Optimization Period Age 16 years of age Documented pathogenic variant,or variant of uncertain significance,of the CASR gene associated with biochemical findings related to hypoparathyroidismSoC MaintenancePeriod(N 70)Randomization 2:12-14 daysLong-Term ExtensionEncaleretEncaleret Titratio

66、n Period(N 47)EncaleretMaintenance Period4 weeksSoC Titration Period(N 23)SoC Maintenance Period20 weeks4 weeksSoC=standard of care;a combination of oral activated Vitamin D and/or calcium supplements.CASR=calcium-sensing receptor gene.Phase 3 Trial Fully Enrolled;LPLV&Topline Results Expected 2H 20

67、25We have fully enrolled the Phase 3 study(CALIBRATE)of encaleret in ADH1 and expect topline results in 2H 2025Up to 4 yearsBBP-812Canavan Disease31BBP-812 has the potential to change the disease trajectory for children with Canavan,a severe,ultra-rare,autosomal recessive leukodystrophy“One patient

68、in particular is sitting independently and taking steps and walking,and that is certainly something Ive never seen with Canavan disease.”Dr.Florian Eichler,Mass General Hospital for Children“The participant was like a new child,reactive,holding hands,and clapping.”Dr Annette Bley,University Medical

69、Center Hamburg“Lots of improvement at 3-month visit,emotional presence,visual tracking,reaching for things”Dr.Alexander Fay,UCSF Benioff Childrens HospitalSafety data consistent with other AAV9 GTxsUrine NAA levelsPreliminary DataCNS NAA levelsGross Motor SkillsDevelopmental SkillsBridgeBioEcosystem

70、 Highlights33BBOT has progressed two potentially first-in-class molecules into the clinic with a third expected in 1H 2025ProgramMechanism of ActionStatusBBO-8520KRASG12C ON/OFFFirst direct inhibitor of KRASG12C ONInhibits both KRASG12C GTP(active)and GDP(inactive)statesDifferentiates from KRASG12C

71、GDP(inactive)-only inhibitorsEnrollingBBO-10203RAS:PI3K BreakerBlocks specific interaction between RAS and PI3KaRAS driver agnostic(KRAS,HRAS and NRAS)Selectively blocks PI3K/AKT effector signaling in the tumorNo hyperglycemia/hyperinsulinemiaEnrollingBBO-11818PanKRAS ON/OFFDirect inhibitor of KRASG

72、12X ONPotent panKRAS inhibitorDirectly binds mutant KRASIND exp.Q1 202534IndicationDiscoveryDiscoveryLead Lead OptimizationOptimizationIND IND EnablingEnablingPhase 1Phase 1Est.Patient Pop.(US+EU)Erythropoietic Protoporphyria(EPP)20kGalactosemia10kAlpha-1 Antitrypsin Deficiency(AATD)200kNeurofibroma

73、tosis Type 1(NF1)200kHereditary Pancreatitis30kFibrous Dysplasia50kCongenital Glycosylation Disorder Type Ia(PMM2-CDG)5kAutosomal Dominant Polycystic Kidney Disease(ADPKD)300kTuberous Sclerosis Complex 1/2(TSC)65kGenetic Epilepsy Driven by SynGAP1 Mutations10kThe GondolaBio pipeline features a diver

74、se set of programs across TAs35We are well-financed and expect to hit numerous milestones in 2025vision for 2030:$8B De-risked PYS100k lives Lives impacted Acoramidis:EU approval Acoramidis:Japan approval Q1 Earnings Call Q2 and Q3 Earnings Calls Encaleret:LPLV,Topline BBP-418:LPLV,Topline Infigratinib:LPLV+1H 20252H 2025