BioMarin JPM 2025.pdf

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1、BioMarin Pharmaceutical Inc.Alexander Hardy,President and Chief Executive OfficerJanuary 13,202543rd AnnualJ.P.Morgan Healthcare Conference2Forward Looking StatementsThis presentation and the associated conference call and webcast contain forward-looking statements about the business prospects of Bi

2、oMarin Pharmaceutical Inc.(BioMarin),including,without limitation,statements about:future financial performance,including the expectations of Total Revenues,Non-GAAP Operating Margin percentage,Operating Cash Flow and Revenue Compound Annual Growth Rate(CAGR)for future periods and the underlying ass

3、umptions;BioMarins plans and expectations regarding innovation and growth,including plans regarding business development and clinical-stage external innovation,as well as the anticipated benefits of such plans;ability of BioMarins approved products,including VOXZOGO and BioMarins enzyme therapies,to

4、 drive long-term revenue growth;the clinical development and commercialization of BioMarins product candidates and commercial products,including plans and expectations regarding(i)the ability to expand BioMarins leadership in achondroplasia with VOXZOGO and leverage VOXZOGO in other skeletal conditi

5、ons,including hypochondroplasia,idiopathic short stature,Noonan Syndrome,Turner Syndrome and SHOX deficiency;(ii)development of BMN 333 for the treatment of achondroplasia and hypochondroplasia,(iii)expansion of PALYNZIQ for the treatment of adolescents with phenylketonuria(PKU),(iv)development of B

6、MN 390 for the treatment of PKU,(v)development of BMN 351 for the treatment of Duchenne Muscular Dystrophy,(vi)development of BMN 349 for the treatment of alpha-1 antitrypsin deficiency,and(vii)development of BMN 370 for the treatment of von Willebrand disease;the expected benefits and availability

7、of BioMarins product candidates and commercial products;the timing of BioMarins clinical development and commercial prospects,including announcements of data from clinical studies and trials;potential growth opportunities and trends,including the assumptions and expectations regarding Total Addressa

8、ble Patient Population with respect to the conditions targeted by BioMarins product candidates and commercial products;and the legal action BioMarin initiated against Ascendis Pharma A/S at the Unified Patent Court for infringement of BioMarins patent rights,including BioMarins expectations regardin

9、g the timing and resolution of such legal action.These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements.These risks and uncertainties include,among others,those factors detailed in BioMarins filings wi

10、th the Securities and Exchange Commission,including,without limitation,the factors contained under the caption Risk Factors in BioMarins Quarterly Report on Form 10-Q for the quarter ended September 30,2024 as such factors may be updated by any subsequent reports.You should carefully consider that i

11、nformation before you make an investment decision.You should not place undue reliance on forward-looking statements,which speak only as of the date hereof.These forward-looking statements are based on the beliefs and assumptions of the Companys management based on information currently available to

12、management and should be considered in connection with any written or oral forward-looking statements that the Company may issue in the future as well as other cautionary statements the Company has made and may make.Except as required by law,BioMarin does not undertake any obligation to update or al

13、ter any forward-looking statement,whether as a result of new information,future events or otherwise.3Non-GAAP Financial MeasuresThis presentation includes both GAAP information and Non-GAAP information.Non-GAAP Operating Margin percentage is defined by the company as GAAP Income from Operations,excl

14、uding amortization of intangible assets,stock-based compensation expense,and,in certain periods,certain other specified items,divided by GAAP Total Revenues.Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP Weighted-Average Diluted Shares Outstanding.Non-GAAP Inco

15、me is defined by the company as GAAP Net Income excluding amortization of intangible assets,stock-based compensation expense and,in certain periods,certain other specified items,as detailed below when applicable.Non-GAAP Weighted-Average Diluted Shares Outstanding is defined by the company as GAAP W

16、eighted-Average Diluted Shares Outstanding,adjusted to include any common shares issuable under the companys equity plans and convertible debt in periods when they are dilutive under Non-GAAP.BioMarin regularly uses both GAAP and Non-GAAP results and expectations internally to assess its financial o

17、perating performance and evaluate key business decisions related to its principal business activities:the discovery,development,manufacture,marketing and sale of innovative biologic therapies.Because Non-GAAP Income,Non-GAAP Operating Margin percentage,Non-GAAP Diluted EPS and Non-GAAP Weighted-Aver

18、age Diluted Shares Outstanding are important internal measurements for BioMarin,the company believes that providing this information in conjunction with BioMarins GAAP information enhances investors and analysts ability to meaningfully compare the companys results from period to period and to its fo

19、rward-looking guidance,and to identify operating trends in the companys principal business.BioMarin also uses Non-GAAP Income internally to understand,manage and evaluate its business and to make operating decisions,and compensation of executives is based in part on this measure.Non-GAAP measures ar

20、e not meant to be considered in isolation or as a substitute for,or superior to,comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP.Investors should note that the Non-GAAP information is not prepared under any compre

21、hensive set of accounting rules or principles and does not reflect all of the amounts associated with the companys results of operations as determined in accordance with GAAP.Investors should also note that these Non-GAAP financial measures have no standardized meaning prescribed by GAAP and,therefo

22、re,have limits in their usefulness to investors.In addition,from time to time in the future there may be other items that the company may exclude for purposes of its Non-GAAP financial measures;likewise,the company may in the future cease to exclude items that it has historically excluded for purpos

23、es of its Non-GAAP financial measures.Because of the non-standardized definitions,the Non-GAAP financial measure as used by BioMarin in this presentation may be calculated differently from,and therefore may not be directly comparable to,similarly titled measures used by other companies.BioMarin does

24、 not provide guidance for GAAP reported financial measures(other than revenue)or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the company is unable to predict with reasonable certainty the financial impact of

25、 changes resulting from its strategic portfolio and business operating model reviews;potential future asset impairments;gains and losses on investments;and other unusual gains and losses without unreasonable effort.These items are uncertain,depend on various factors,and could have a material impact

26、on GAAP reported results for the guidance period.As such,any reconciliations provided would imply a degree of precision that could be confusing or misleading to investors.With respect to historical Non-GAAP adjusted financial information,see the appendix beginning on slide 22 for the reconciliations

27、 to the comparable information reported under U.S.GAAP.4BioMarin is the global leader in treating genetically defined conditions.SCIENTIFICEXCELLENCEUncovering and solving for the root cause of conditionsACCELERATED INNOVATIONRapidly transforming insights into category-defining medicinesIN-HOUSE EXP

28、ERTISE Driving the development and manufacturing of complex medicinesGLOBAL DELIVERY Ensuring life-changing products reach patients counting on us mostWe pioneer new medicines through four connected strengths.5Building on Strong Execution in 20243Q24 Year-to-date FinancialsFinancial Outlook Provided

29、 through 2034$2.1B+19%TotalRevenues27.7%+7.6 pptsNon-GAAPOperatingMargin1$2.60+63%Non-GAAPDiluted Earnings Per Share11Refer to slide 3 for more detail on Non-GAAP financials;2Hypochondroplasia,Idiopathic Short Stature,Noonan Syndrome,Turner Syndrome,SHOX deficiency;3Duchenne Muscular Dystrophy,Alpha

30、-1 Antitrypsin Deficiency;4Revenue growth shown on a non-risk-adjusted basis.New Strategy to Drive Value CreationINNOVATIONGROWTHVALUE COMMITMENTMilestones Anticipated over Next 18 MonthsLow-to-Mid 40%s,starting with 40%in 2026Non-GAAP Operating Margin1$4B2027 Total RevenuesTargeting Mid-teenCAGR202

31、3 2034 Revenue Growth4$1.25B+2027 Operating Cash FlowSkeletal Conditions 5 indications2advancing in VOXZOGO and BMN 333 PK dataEnzyme Therapies Palynziq(12-17 yrs)Phase 3 Data and U.S.&EU sBLA Filings BMN 390(PKU with novel PEG)First-in-human study startPipeline BMN 351(DMD3)Clinical POC Data;BMN 34

32、9(A1AT3)Study StartPrioritizing Business Development6Building on Strong Execution in 20243Q24 Year-to-date FinancialsFinancial Outlook Provided through 2034 Milestones Anticipated over Next 18 MonthsNew Strategy to Drive Value CreationINNOVATIONGROWTHVALUE COMMITMENTLow-to-Mid 40%s,starting with 40%

33、in 2026$4BTargeting Mid-teenCAGR2023 2034 Revenue Growth4$1.25B+2027 Operating Cash FlowNon-GAAP Operating Margin1$2.1B+19%TotalRevenues27.7%+7.6 pptsNon-GAAPOperatingMargin1$2.60+63%Non-GAAPDiluted Earnings Per Share12027 Total Revenues1Refer to slide 3 for more detail on Non-GAAP financials;2Hypoc

34、hondroplasia,Idiopathic Short Stature,Noonan Syndrome,Turner Syndrome,SHOX deficiency;3Duchenne Muscular Dystrophy,Alpha-1 Antitrypsin Deficiency;4Revenue growth shown on a non-risk-adjusted basis.Skeletal Conditions 5 indications2advancing in VOXZOGO and BMN 333 PK dataEnzyme Therapies Palynziq(12-

35、17 yrs)Phase 3 Data and U.S.&EU sBLA Filings BMN 390(PKU with novel PEG)First-in-human study startPipeline BMN 351(DMD3)Clinical POC Data;BMN 349(A1AT3)Study StartPrioritizing Business Development7Building on Strong Execution in 2024Financial Outlook Provided through 2034 New Strategy to Drive Value

36、 CreationINNOVATIONGROWTHVALUE COMMITMENTMilestones Anticipated over Next 18 MonthsLow-to-Mid 40%s,starting with 40%in 2026$4BTargeting Mid-teenCAGR2023 2034 Revenue Growth4$1.25B+2027 Operating Cash FlowNon-GAAP Operating Margin13Q24 Year-to-date Financials$2.1B+19%TotalRevenues27.7%+7.6 pptsNon-GA

37、APOperatingMargin1$2.60+63%Non-GAAPDiluted Earnings Per Share12027 Total Revenues1Refer to slide 3 for more detail on Non-GAAP financials;2Hypochondroplasia,Idiopathic Short Stature,Noonan Syndrome,Turner Syndrome,SHOX deficiency;3Duchenne Muscular Dystrophy,Alpha-1 Antitrypsin Deficiency;4Revenue g

38、rowth shown on a non-risk-adjusted basis.Skeletal Conditions 5 indications2advancing in VOXZOGO and BMN 333 PK dataEnzyme Therapies Palynziq(12-17 yrs)Phase 3 Data and U.S.&EU sBLA Filings BMN 390(PKU with novel PEG)First-in-human study startPipeline BMN 351(DMD3)Clinical POC Data;BMN 349(A1AT3)Stud

39、y StartPrioritizing Business Development8Building on Strong Execution in 2024Financial Outlook Provided through 2034 New Strategy to Drive Value CreationINNOVATIONGROWTHVALUE COMMITMENTMilestones Anticipated over Next 18 MonthsLow-to-Mid 40%s,starting with 40%in 2026$4BTargeting Mid-teenCAGR2023 203

40、4 Revenue Growth4$1.25B+2027 Operating Cash FlowNon-GAAP Operating Margin13Q24 Year-to-date Financials$2.1B+19%TotalRevenues27.7%+7.6 pptsNon-GAAPOperatingMargin1$2.60+63%Non-GAAPDiluted Earnings Per Share12027 Total Revenues1Refer to slide 3 for more detail on Non-GAAP financials;2Hypochondroplasia

41、,Idiopathic Short Stature,Noonan Syndrome,Turner Syndrome,SHOX deficiency;3Duchenne Muscular Dystrophy,Alpha-1 Antitrypsin Deficiency;4Revenue growth shown on a non-risk-adjusted basis.Skeletal Conditions 5 indications2advancing in VOXZOGO and BMN 333 PK dataEnzyme Therapies Palynziq(12-17 yrs)Phase

42、 3 Data and U.S.&EU sBLA Filings BMN 390(PKU with novel PEG)First-in-human study startPipeline BMN 351(DMD3)Clinical POC Data;BMN 349(A1AT3)Study StartPrioritizing Business Development9Percentage of Infants and Children with Achondroplasia Eligible for VOXZOGO Treatment;(24K Global TAPP)VOXZOGO Pres

43、ents Attractive Global Expansion Opportunities with 90%of Total Addressable Patient Population(TAPP)Outside the U.S.Rest of World:68%of VOXZOGO TAPP Leveraging 20-year global capabilities in patient finding,diagnosis,access,and support for first-in-therapy treatments VOXZOGO available in majority of

44、 ROW countries with plans to add remaining by 2027 Applying learnings from successful ROW launchesTAPP defined as the diagnosed,clinically eligible patients for a given product or program in a defined footprint;footprint defined as all global markets included in BioMarins internal projections for VO

45、XZOGO revenues.Rest of World:Primarily Latin America,Middle East,and Asia.Europe,Japan,Australia,Canada:22%of VOXZOGO TAPPUnited States:10%of VOXZOGO TAPPHighly Penetrated,Large Strategic Markets GermanyItalyFranceSpainJapanAUS10Plans for Strong Growth in the U.S.with VOXZOGO in AchondroplasiaU.S.Ac

46、hondroplasia TAPP:2,400 Expected to be the only approved product for achondroplasia,from birth,through 2027 Majority of new patients are in 0-5 age group since label expansion in 2023 Continued expansion of U.S.prescriber base,especially pediatric endocrinologists Strong progress raising awareness t

47、o drive referrals from pediatricians New treatment guidelines for VOXZOGO and growing body of clinical benefit data beyond height2024E2027EVOXZOGO U.S.StrategyU.S.Market Treated ACH PatientsU.S.ACH TAPPPotential competitor(s)Assumes multiple products:2022A11New Publications Highlight Importance of E

48、arly VOXZOGO TreatmentVOXZOGO Treated Children Demonstrated Statistically Significantly Improvements in Proportionality vs.Untreated Control ArmNew International Treatment Guidelines Recommend Early Diagnosis and Treatment with VOXZOGOVOXZOGO phase 3 treatment in achondroplasia(n=48)Early screening&

49、detection of achondroplasia(prenatally,where possible)Education&consent for VOXZOGO treatmentBegin VOXZOGO treatment as early as possibleNew International Treatment Guidelines:Published in Med,December 20241Published in Nature Reviews Endocrinology,January 202521Savarirayan et al.,Sustained growth-p

50、romoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study,Med(2024),https:/doi.org/10.1016/j.medj.2024.11.019 2Savarirayan,R.,Hoover-Fong,J.,Ozono,K.et al.International consensus guidelines on the implementation and monitoring of vosoritide therapy in ind

51、ividuals with achondroplasia.Nat Rev Endocrinol(2025).https:/doi.org/10.1038/s41574-024-01074-9123Monitor treatment outcome and response after VOXZOGO initiation4Ongoing monitoring until growth plate closure512VOXZOGO for Hypochondroplasia(HCH)Demonstrated a 1.81cm Increase in Annualized Growth Velo

52、city in Phase 2 Rapid recruitment of pivotal study,expected to be fully enrolled in 1Q25 Direct-to-registrational opportunity enabled with evidence fromachondroplasia and early proof-of-concept in HCH Targeting 14,000 most severely impacted HCH patients(those with height Z-score -2.0 SDs)Leveraging

53、5-year global achondroplasia leadership Focus on increasing diagnosis and establishing treatment paradigmClearly Defined Go-to-market StrategyPotential First-in-class Approved Treatment Option;Tracking to Expected 2027 LaunchAnnualized Growth VelocityPhase 2 data1:N=24,1.81 cm/year increase in mean

54、AGV1Vosoritide treatment for children with hypochondroplasia:a phase 2 trial.Dauber,Andrew et al.eClinicalMedicine,Volume 71,10259113Building Leadership in Skeletal Conditions202520262027Continued Global ExpansionCommercialVOXZOGO for ACHVOXZOGO expected to be available in 60 countriesNew guidelines

55、,emerging datasets,and broad label support early treatment with VOXZOGOBMN 333 PKdataAnticipated Clinical MilestonesVOXZOGO&BMN 333ISS,Noonan,Turner,SHOX to enter phase 3Initiated an action against ASND at the EU Unified Patent Court(UPC)to protect EU patent rightsCurrent IP EnforcementActionsHCH la

56、unchHCH pivotal dataDecision from UPC expected;binding in 18 EU countriesBMN 333 initiated first-in-human study14Underlying,Established Strengths of the Enzyme Therapies BusinessHigh Patient Reach and Treatment ContinuityFirst-in-category MedicinesChallenging Path for Biosimilars Built and created g

57、lobal leadership in each therapeutic area 20-year track record building Enzyme Therapies infrastructure foundational to sustained growth Profile of these transformational medicines support high compliance and adherence rates$1.4B Enzyme Therapies Revenues 3Q24 Year-to-date15Strategy to Drive Long-Te

58、rm Growth across Enzyme Therapies Business$1.7B$1.3BTargeting High Single-digit CAGR(2023-2034)8%CAGR202020232034Enzyme Therapies RevenueFind&Start New PatientsGrow PKU OpportunityNote:Revenue figures include revenues for Vimizim,Naglazyme,Brineura,Palynziq,and Aldurazyme.2034 revenues include expec

59、ted revenues for BMN 390 for phenylketonuria.Expand access to diagnostic tools,including gene panels Implement initiatives to increase diagnosis,including cascade screening Leverage predictive analytics to pinpoint hot spots Expand label to include 12-17 y/o Broaden geographic reach Develop BMN 390:

60、Next-gen Palynziq with potential for lower immunogenicity16External Innovation Strategy to Complement Revenue Growth Leadership in Development&Commercialization of Medicines in Genetically Defined ConditionsRich Environment for Business DevelopmentScientific momentum allows clearly defined drug deve

61、lopmentRich biotech arena with many small companies at R&D stageBiotech remains capital constrainedBioMarin as the Partner of ChoiceProven world class research,clinical,and regulatory capabilitiesBest-in-class manufacturingStrong global commercial infrastructureFocus on Disciplined Deal Execution 10

62、%dystrophin levels at steady stateNear Full-Length Dystrophin produced,naturally-occurring versions of which preserve ambulation into adulthoodPhosphorothioate chemistry(non-morpholino)leverages in-house expertiseBMN 351 for Duchenne Muscular Dystrophy;Targeting 2025 POC1.Muscle cell penetrationWMS

63、20202.Binding at novel splice site with skip-inductionFUTURES 2022In vitro TIDES EU 20233.Functional dystrophin expressionWMS 2020TIDES USA 20204.Functional benefit WMS 2023SfN 2023Phase 1/22024Target POC2025Target Ph3Start:2026Target Launch20281.Muscle cell penetrationInterim 13-wk data2.Binding at

64、 novel splice site with skip-induction3.Functional dystrophin expression Anticipate release of 26-week data in 2H25WMS=World Muscle Society Annual Congress;FUTURES=CureDuchenne National Conference;TIDES=Oligonucleotide and Peptide Therapeutics Conference;SfN=Society for Neuroscience Annual Meeting C

65、linical fraction remaining ambulatoryAge(years)BMN-351-length dystrophinDMD102030405060708019CandidateCondition18-Month Look-AheadVOXZOGOHypochondroplasia 1H25:Pivotal trial fully enrolledVOXZOGOIdiopathic Short Stature 1H25:Phase 2 study enrollingVOXZOGONoonan/Turner/SHOX Syndrome1H25:Phase 2 study

66、 enrollingBMN 333ACH&HCH2H25:PK data;1H26:PK presentation PALYNZIQPhenylketonuria(12-17)1H25:Phase 3;2H25:sBLA filingBMN 390Phenylketonuria(Novel PEG)1H26:FIH study startBMN 351Duchenne Muscular Dystrophy2H25:Clinical POC presentation BMN 349Alpha-1 Antitrypsin Deficiency1H25:Patient study startBMN

67、370von Willebrand Disease2026:FIH study startAnticipated Near-Term R&D MilestonesSkeletal ConditionsEnzyme TherapiesPipeline20PURPOSE-DRIVEN.PATIENT-FOCUSED.FUTURE-READY.Q&AThank you.AppendixReconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information23Reconciliation of GAAP Reported to

68、 Selected Non-GAAP Adjusted Information(1)(1)Certain amounts may not sum or recalculate due to rounding.(2)Represents a payment triggered by a third partys attainment of a regulatory approval milestone related to previously sold intangible assets.(3)These amounts represent severance and restructurin

69、g costs related to the companys 2024 portfolio strategy review and the associated organizational redesign efforts announced in the second and third quarters of 2024.These amounts also include impairments of certain right-of-use and fixed assets.(4)Represents a downward adjustment to non-marketable e

70、quity securities recorded in Other income(expense),net.24Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information(1)(1)Certain amounts may not sum or recalculate due to rounding.(2)Represents a payment triggered by a third partys attainment of a regulatory approval milestone related

71、 to previously sold intangible assets.(3)These amounts represent severance and restructuring costs related to the companys 2024 portfolio strategy review and the associated organizational redesign efforts announced in the second and third quarters of 2024.These amounts also include impairments of ce

72、rtain right-of-use and fixed assets.GAAP Income from Operations Adjustments Stock-based compensation expense Amortization of intangible assets Gain on sale of nonfinancial assets(2)Severance and restructuring costs(3)Non-GAAP Income from Operations Nine Months Ended September 30,2024 Percent of GAAP

73、 Total Revenue 2023 Percent of GAAP Total Revenue$323 15.3%$158 8.9%150 7.2 152 8.6 34 1.6 47 2.7 (10)(0.5)87 4.1 (1)(0.1)$583 27.7%$357 20.1%25Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information(1)(1)Certain amounts may not sum or recalculate due to rounding.(2)Represents a pa

74、yment triggered by a third partys attainment of a regulatory approval milestone related to previously sold intangible assets.(3)These amounts represent severance and restructuring costs related to the companys 2024 portfolio strategy review and the associated organizational redesign efforts announce

75、d in the second and third quarters of 2024.These amounts also include impairments of certain right-of-use and fixed assets.(4)Represents a downward adjustment to non-marketable equity securities recorded in Other income(expense),net.GAAP Diluted EPS Adjustments Stock-based compensation expense Amort

76、ization of intangible assets Gain on sale of nonfinancial assets(2)Severance and restructuring costs(3)Loss on investments(4)Income tax effect of adjustments Non-GAAP Diluted EPS Nine Months Ended September 30,2024 2023$1.56$0.77 0.76 0.76 0.17 0.24 (0.05)0.44 0.03 0.06 (0.31)(0.23)$2.60$1.60 26Reco

77、nciliation of GAAP Reported to Selected Non-GAAP Adjusted Information(1)(1)Common stock issuable under the companys convertible debt was excluded from the computation of GAAP Weighted-Average Diluted Shares Outstanding when they were anti-dilutive.If converted,for the prior year comparative period,t

78、he company would have issued approximately 4.4 million shares under the convertible notes due in 2027.GAAP Weighted-Average Diluted Shares Outstanding Adjustments Common stock issuable under the companys convertible debt(1)Non-GAAP Weighted-Average Diluted Shares Outstanding Nine Months Ended September 30,2024 2023 196.7 195.0 4.4 196.7 199.4