Blueprint JPM 2025 BPMC slides vF.pdf

《Blueprint JPM 2025 BPMC slides vF.pdf》由會員分享，可在線閱讀，更多相關《Blueprint JPM 2025 BPMC slides vF.pdf（25頁珍藏版）》請在三個皮匠報告上搜索。

1、Blueprint MedicinesDriving growth and innovation with operational excellenceKate Haviland,Chief Executive OfficerJ.P.Morgan Healthcare ConferenceJanuary 13,2025Forward-Looking StatementsThis presentation contains forward-looking statements within the meaning of the Private Securities Litigation Refo

2、rm Act of 1995,as amended,including,without limitation,statements regarding plans,strategies,timelines and expectations for the companys operations,including its growth strategies,opportunities and expectations for 2025 and beyond;the companys expectations regarding its estimated peak revenue for AY

3、VAKIT and the systemic mastocytosis market;continued growth in the breadth and depth of prescribing for AYVAKIT;the companys development plans and expectations regarding BLU-808,including its potential treatment for mast cell disorders,as well as other potential related allergic-inflammatory indicat

4、ions;expectations related to the markets for the companys current or future approved drugs and drug candidates,including expectations regarding the size or scale of patient opportunities that its current or future approved drugs and drug candidates;statements regarding anticipated clinical milestone

5、s;the potential benefits of any of the companys current or future approved drugs or drug candidates in treating patients;statements related to the companys liquidity and capital position,product revenues,run-rate,financial performance,strategy,goals and anticipated milestones,business plans and focu

6、s,including expectations regarding its 2025 capital allocation strategy,its anticipated cash burn,and path to profitability.The words“aim,”“may,”“will,”“could,”“would,”“should,”“expect,”“plan,”“anticipate,”“intend,”“believe,”“estimate,”“predict,”“project,”“potential,”“opportunity,”“continue,”“target

7、”and similar expressions are intended to identify forward-looking statements,although not all forward-looking statements contain these identifying words.Any forward-looking statements in this presentation are based on managements current expectations and beliefs and are subject to a number of risks,

8、uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this presentation,including,without limitation:the risk that the marketing and sale of AYVAKIT/AYVAKYT or any future approve

9、d drugs may be unsuccessful or less successful than anticipated,or that AYVAKIT/AYVAKYT may not gain market acceptance by physicians,patients,third-party payors and others in the medical community;the risk that the market opportunities for AYVAKIT/AYVAKYT or the companys drug candidates are smaller

10、than it estimates or that any approval it obtains may be based on a narrower definition of the patient population that it anticipates;the risk of delay of any current or planned clinical trials or the development of the companys current or future drug candidates;risks related to the companys ability

11、 to successfully demonstrate the safety and efficacy of its drug candidates and gain approval of its drug candidates on a timely basis,if at all;the risk that preclinical and clinical results for the companys drug candidates may not support further development of such drug candidates either as monot

12、herapies or in combination with other agents or may impact the anticipated timing of data or regulatory submissions;the risk that the timing of the initiation of clinical trials and trial cohorts at clinical trial sites and patient enrollment rates may be delayed or slower than anticipated;the risk

13、that actions of regulatory agencies may affect the companys approved drugs or its current or future drug candidates,including affecting the initiation,timing and progress of clinical trials;risks related to the companys ability to obtain,maintain and enforce patent and other intellectual property pr

14、otection for its products and current or future drug candidates it is developing;risks related to the success of the companys current and future collaborations,financing arrangements,partnerships,licensing and other arrangements;risks related to the companys liquidity and financial position,includin

15、g the risk that it may be unable to generate sufficient future product revenues to maintain a self-sustainable financial profile and to achieve profitability;and risks related to the accuracy of the companys estimates of revenues,expenses and capital requirements.These and other risks and uncertaint

16、ies are described in greater detail in the section entitled“Risk Factors”in the companys filings with the Securities and Exchange Commission(SEC),including the companys most recent Annual Report on Form 10-K,as supplemented by its most recent Quarterly Report on Form 10-Q and any other filings that

17、the company has made or may make with the SEC in the future.The forward-looking statements in this presentation are made only as of the date hereof,and except as required by law,the company undertakes no obligation to update any forward-looking statements contained in this presentation as a result o

18、f new information,future events or otherwise.Accordingly,readers are cautioned not to place undue reliance on these forward-looking statements.This presentation also contains estimates,projections and other statistical data made by independent parties and by the company relating to market size and g

19、rowth and other data about the companys industry.These data involve a number of assumptions and limitations,and you are cautioned not to give undue weight to such estimates.In addition,projections,assumptions and estimates of the companys future performance and the future performance of the markets

20、in which the company operates are necessarily subject to a high degree of uncertainty and risk.Blueprint Medicines,AYVAKIT,AYVAKYT and associated logos are trademarks of Blueprint Medicines Corporation.2Targeting the mast cell to fundamentally change the treatment of allergic and inflammatory diseas

21、es3Degranulatingmast cellSkinRespiratoryGastrointestinalSystemicKITPro-inflammatory mediatorselenestinibNearly 15 years ofscientific leadership inmast cell biology BLU-808,oral wtKIT inhibitor for mast cell diseasesClinical-stage therapywith multibillion-dollarpipeline-in-a-product potentialestimate

22、d peaksystemic mastocytosis franchise revenueestimated AYVAKIT revenue by 2030$4B$2BwtKIT,wild-type KIT.Looking back at a year of achievement in 2024$53$111$2042021202220232024$475-4801130%AYVAKITREVENUEGROWTH$,MILLIONS4Achieved inflection in AYVAKIT revenue toward multibillion-dollar peak potential

23、Expanded global reach with AYVAKYT reimbursed in 15 countries to dateInitiated registrational Phase 3 study of elenestinib in ISMSubmitted BLU-808 IND to FDA and completed healthy volunteer trialStrengthened financial profile,with 50%reduction in anticipated cash burn11 Based on 2024 revenue guidanc

24、e.Plan to report Q4 and full-year 2024 financial results in February 2025.FDA,U.S.Food and Drug Administration;IND,investigational new drug application;ISM,indolent systemic mastocytosis.Our core growth drivers in 20255DURABLEFINANCIALPROFILECONTINUOUSINNOVATIONDrive continued AYVAKIT revenue growth

25、Extend long-term SM franchise with elenestinib developmentAccelerate and expand development of BLU-808Advance R&D innovation to fuel long-term growthHIGHGROWTHSM,systemic mastocytosis.Our capital allocation strategy aligns with core growth drivers6DURABLEFINANCIALPROFILECONTINUOUSINNOVATIONDrive con

26、tinued AYVAKIT revenue growthExtend long-term SM franchise with elenestinib developmentAccelerate and expand development of BLU-808HIGHGROWTHAdvance R&D innovation to fuel long-term growthSM,systemic mastocytosis.“I think it was just the hope to be done with all this sort of cumbersome palliative ca

27、re.I just wanted to feel better.The goal for me was to start cutting out all that stuff from my life and get back to living.Sometimes I reflect now and think wow.I feel way better than I did before.AYVAKIT streamlines and simplifies my life and plans.”Andrew,AYVAKIT patientWere hearing life-changing

28、 stories with AYVAKIT7Systemic mastocytosis is a fast-growing rare disease market8LARGE,GROWING MARKET OPPORTUNITY1 Internal Blueprint Medicines analyses of U.S.claims data.2 Bergstrom et al;Acta Oncologica(2024).CAGR,compound annual growth rate.GLOBAL SM REVENUE POTENTIAL$4B estimated SM franchise

29、peak revenue$2Bestimated annual AYVAKIT revenue by 2030Increase in diagnosed SM patients 25K patients observable in U.S.claims data today1Widening lens on who is an AYVAKIT patient Supported by growing body of long-term safety&efficacy dataHigher estimated SM prevalence 2x prior estimate,based on re

30、cent large independent study220192020202120222023202425KOBSERVABLESM PATIENTSIN U.S.CLAIMS DATA1Building off AYVAKITs strong launch to drive growth to peak Strong and steady new patient starts Significant,growing number of patients on AYVAKIT Trend toward multi-year duration of therapy Growing bread

31、th and depth of prescribing in hem/onc and A/I specialties Robust payer coverage and fast time to fill Reach a wider group of providers and specialties,including derm and GI Optimized diagnosis and care championed by an empowered global SM community More patients activated to seek out AYVAKIT Diseas

32、e control redefined with long-term data on AYVAKIT and real-world SM burden Continued global geographic expansionPEAK POTENTIAL(FUTURE)STRONG LAUNCH(TODAY)Planned 2025investmentsIncrementalfield forceexpansionNew HCPand patientinitiativesAdditionaldatageneration9A/I,allergy/immunology;derm,dermatolo

33、gy;GI,gastroenterology;HCP,healthcare provider;hem/onc,hematology/oncology.Driving innovation with elenestinib to extend SM franchise lifecycle10Registrational Phase 3 HARBOR trial initiated in Q4 2024Mean change in ISM-SAF TSS from baseline,leveraging AYVAKIT precedentReduction in anaphylaxis frequ

34、ency,improvement in bone health,additional biomarkersTwo active arms,75&100 mg elenestinib selected based on Part 1 data,versus placeboPrimary endpointNovel endpointsMultiple dosesHARBOR Registrational Trialof elenestinib,a next-genKIT D816V inhibitor,in patients with ISMSymptom Improvement Reducing

35、 symptoms Impacting mast cell burden Improving quality of lifeCURRENTDisease Modification Improve bone health Reduce anaphylaxis Address chronic inflammation Minimize progressionFUTUREISM-SAF,indolent systemic mastocytosis symptom assessment form.TSS,total symptom score.Our core growth drivers in 20

36、2511DURABLEFINANCIALPROFILECONTINUOUSINNOVATIONDrive continued AYVAKIT revenue growthExtend long-term SM franchise with elenestinib developmentAccelerate and expand development of BLU-808Advance R&D innovation to fuel long-term growthHIGHGROWTHBLU-808 targets wild-type KIT(wtKIT),the master regulato

37、r of the mast cellKITDegranulating mast cellVALIDATED TARGET KIT is the master regulator of mast cell activity Activation triggers an inflammatory responseand broad symptomology Inhibition proven to have therapeutic effectsUNMET NEED Highly potent and selective oral wtKIT inhibitor Once-daily dosing

38、 Wide therapeutic index enabling tunable approach to optimize benefit-risk12BLU-808 designed to be a best-in-class oral,once-daily wtKIT inhibitor13BLU-808 OPTIMIZED PROFILE1POTENCYpKIT cellular IC50(nM)0.37WT KIT-dependent proliferation IC50(nM)1.3Inhibition of CD63 extracellular expression IC50(nM

39、)2.7Inhibition of histamine degranulation IC50(nM)8.6SELECTIVITYS(10)3 M0.042PDGFRA selectivity300 xPDGFRB selectivity400 xFLT3 selectivity9600 xCSF1R Kd selectivity800 xBrain penetrance(Kpu,u)0.0211 mg5 mg10 mg20 mg30 mg42 mg1 mg QD3 mg QD6 mg QD12 mg QDPART 1:SINGLEASCENDING DOSEN=56Daily(QD)dosin

40、g;14 days+follow-upPART 2:MULTIPLEASCENDING DOSEN=312PHASE 1 HEALTHY VOLUNTEER STUDYTo support tunable dosing potentialand meaningful therapeutic differentiation1 Grassian,A.et al.Presentation at AAAAI(2024).In single ascending dose(SAD)study,42 mg was selected to achieve 50%greater exposure over 30

41、 mg,based on preclinical data.2Two patients did not complete the protocol including one subject in the placebo cohort who was removed at Day 12 due to violation of study site policy and one subject in the 6 mg cohort who was found to be ineligible at Day 8 due to a medical history of benign ethnic n

42、eutropenia and was removed.All available data for both subjects are included.Assessments:safety,pharmacokinetics,&pharmacodynamicsSingle ascending dose(SAD)Dose-dependent PK and tryptase response with single BLU-808 dose14IC50,predicted 50%inhibitory concentration based on cellular proliferation ass

43、ays;IC90,predicted 90%inhibitory concentration based on cellular proliferation assays;AST,aspartate aminotransferase;ALT,alanine aminotransferase;PK,pharmacokinetics;T1/2,terminal half-life;%CV,coefficient of variation(percent);SD,standard deviation.Tryptase values below lower limit of quantificatio

44、n(LLOQ;1ng/mL)were imputed at 0 ng/mL.T1/2 40 hours supports once-daily dosing Low PK variability(%CV 30%)No food effect Dose-dependent serum tryptase decrease Single dose of BLU-808 reduced serum tryptase by more than 60%Single doses of BLU-808 were well-tolerated(1-42 mg),with no significant chang

45、es in labs,including AST/ALTSINGLE DOSE PHARMACOKINETICS(N=56)CHANGE IN SERUM TRYPTASE(N=56)HoursHoursMultiple ascending dose(MAD)Consistent,dose-dependent PK with multiple BLU-808 doses Sustained target coverage with once-daily dosing at all doses Doses 3mg reached concentrations above IC90 Low var

46、iability(50%reductionin anticipated cash burn in 2024over prior year1$882.4 millioncash and cash equivalents at end of Q3 2024anticipate$80M in proceeds from GSK acquisition of IDRx due to equity stake,upon closingAYVAKITREVENUEGROWTH$,MILLIONS202420231 Based on 2024 revenue guidance.2025 strategic

47、priorities to unlock the next stage of growth24STRATEGIC PRIORITYGOAL1H 20252H 2025Grow leadership insystemic mastocytosisDeliver continued strong and steady AYVAKIT revenue growthPresent additional long-term data from PIONEER trial of AYVAKITAchieve reimbursement of AYVAKYT in 20 countries overallA

48、ctivate sites and drive enrollment of HARBOR trial of elenestinibAchieve BLU-808clinical proof-of-conceptPresent Phase 1 healthy volunteer trial results Initiate POC trials in CSU,CindU,AR and ACInitiate POC trials in allergic asthma and MCASDrive research innovationNominate 2 development candidates,including first protein degraderAC,allergic conjunctivitis;AR,allergic rhinitis;CindU,chronic inducible urticaria;CSU,chronic spontaneous urticaria,MCAS,mast cell activation syndrome;POC,proof of conceptBlueprint MedicinesDriving growth and innovation with operational excellence