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1、2025 Vertex Pharmaceuticals Incorporated 2025 Vertex Pharmaceuticals Incorporated FIRST QUARTER 2025 FINANCIAL RESULTS2025 Vertex Pharmaceuticals IncorporatedFirst Quarter 2025Financial ResultsMay 5,20252025 Vertex Pharmaceuticals Incorporated 2AgendaIntroduction Susie Lisa,CFA,Senior Vice President

2、,Investor RelationsCEO Perspective and Pipeline Update Reshma Kewalramani,M.D.,Chief Executive Officer and PresidentCommercial Update Stuart Arbuckle,Executive Vice President and Chief Operating Officer Duncan McKechnie,SVP,North America Commercial Operations Chief Commercial Officer(as of July 1,20

3、25)Financial Results Charlie Wagner,Executive Vice President and Chief Financial Officer2025 Vertex Pharmaceuticals Incorporated 3Safe harbor statement&non-GAAP financial measuresThis presentation contains forward-looking statements that are subject to risks,uncertainties and other factors.All state

4、ments other than statements of historical fact are statements that could be deemed forward-looking statements,including all statements regarding the intent,belief,or current expectation of Vertex and members of the Vertex senior management team.Forward-looking statements are not purely historical an

5、d may be accompanied by words such as“anticipates,”“may,”“forecasts,”“expects,”“intends,”“plans,”“potentially,”“believes,”“seeks,”“estimates,”and other words and terms of similar meaning.Such statements include,without limitation,the information provided regarding and expectations for future financi

6、al and operating performance,the section captioned“Updated full year 2025 financial guidance,”expectations for financial performance in Q1 2025,and statements regarding(i)expectations,development plans and timelines for Vertexs products and pipeline programs,including beliefs regarding the status of

7、 product launches,achievement of key enrollment milestones in 2025,advancement of multiple programs across multiple modalities,significantly expanding the number of patients Vertex serves,relevant estimated patient populations,expectations with respect rapid advancement in Vertexs clinical portfolio

8、 and for five launches over five years(by 2028),and expectations for increased revenue contributions from CASGEVY,ALYFTREK,and JOURNAVX in 2025,(ii)expectations regarding ALYFTREK,including those related to ALYFTREKs clinical benefits and potential to set a new standard of care in CF,for additional

9、potential approvals of ALYFTREK in 2025 and launching in the U.K.later this year,expectations regarding U.S.patients switching to ALYFTREK,and expectations with respect to a lower royalty burden,(iii)expectations for Vertexs CF programs,including those related to the potential benefits of VX-828 as

10、a next generation CFTR corrector and the initiation of the VX-828 study in CF patients in 2025,and expectations related to the VX-522 clinical trial,(iv)expectations for Vertexs T1D programs,including beliefs regarding a potentially curative treatment and treatable patient population,expectations to

11、 complete enrollment and dosing in the ongoing zimislecel pivotal trial in Q2 2025,and potential global regulatory submissions in the first half of 2026,(v)expectations regarding the therapeutic scope,potential benefits,and target patient population for pove,including its best-in-class and pipeline-

12、in-a-product potential,expectations for poves clinical progress,including with respect to an interim analysis in the Phase 3 RAINIER study and the potential to file for accelerated approval in the U.S.in the first half of 2026 if positive,plans to advance pove into pivotal development for pMN,and ex

13、pectations for the RUBY-3 and RUBY-4 Phase 2 basket studies,(vii)expectations for VX-407 in ADPKD,including regarding advancement to Phase 2 in the second half of 2025,(vii)expectations for CASGEVY,including building global momentum in 2025 and reaching more eligible patients across geographies with

14、 regulatory approval and access,and plans to expand manufacturing to support global demand,(viii)status and expectations for the U.S.JOURNAVX launch in acute pain,beliefs regarding the commercial potential of JOURNAVX,including expectations for sales volume and revenue,beliefs regarding momentum wit

15、h payers and retailers,and expectations regarding JOURNAVXs addition to the list of medicines covered under the NOPAIN Act,(ix)expectations for the studies of the intravenous and oral formulations of VX-993 in acute pain and DPN,including expectations to complete the ongoing oral study of VX-993 in

16、acute pain this quarter and to share data in the second half of 2025,and expectations to progress additional Nav1.8 and NaV1.7 inhibitors,(x)expectations to complete enrollment in IA cohort of inaxaplin study in 2025 and the potential to file for U.S.accelerated approval,(xi)expectations for the Pha

17、se 3 trial of suzetrigine in DPN and plans to advance the pivotal program,and expectations to advance study of suzetrigine in LSR into Phase 3 pending regulatory discussions,and(xii)plans to advance the MAD portion of the DM1 study.While Vertex believes the forward-looking statements contained in th

18、is presentation are accurate,these forward-looking statements represent the companys beliefs as of the date of this presentation and there are risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements.Tho

19、se risks and uncertainties include,among other things,that data from clinical trials,especially if based on a limited number of patients,may not to be indicative of final results,the companys regulatory submissions may be delayed,actual patient populations eligible for our products may be smaller th

20、an anticipated,the company may not be able to commercialize its products successfully or in the manner anticipated,data from the companys development programs may not be available on expected timelines,or at all,support registration or further development of its potential medicines due to safety,eff

21、icacy or other reasons,and other risks listed under the heading“Risk Factors”in Vertexs annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the companys website at .You should not place any undue reliance on these stat

22、ements,or the data presented.Vertex disclaims any obligation to update the information contained in this presentation as new information becomes available.In this presentation,Vertexs financial results and financial guidance are provided in accordance with accounting principles generally accepted in

23、 the United States(GAAP)and using certain non-GAAP financial measures.In particular,non-GAAP financial results and guidance exclude from Vertexs pre-tax income(i)stock-based compensation expense,(ii)intangible asset amortization expense,(iii)gains or losses related to the fair value of the companys

24、strategic investments,(iv)increases or decreases in the fair value of contingent consideration,(v)acquisition-related costs,(vi)an intangible asset impairment charge,and(vii)other adjustments.The companys non-GAAP financial results also exclude from its provision for income taxes the estimated tax i

25、mpact related to its non-GAAP adjustments to pre-tax income described above and certain discrete items.For full-year 2024,the companys non-GAAP weighted-average common shares outstanding included the estimated effect of potentially dilutive securities that was not used in the calculation of GAAP dil

26、uted weighted-average common shares outstanding because the company incurred a GAAP net loss for the period.These results should not be viewed as a substitute for the companys GAAP results and are provided as a complement to results provided in accordance with GAAP.Management believes these non-GAAP

27、 financial measures help indicate underlying trends in the companys business,are important in comparing current results with prior period results and provide additional information regarding the companys financial position that the company believes is helpful to an understanding of its ongoing busin

28、ess.Management also uses these non-GAAP financial measures to establish budgets and operational goals that are communicated internally and externally,to manage the companys business and to evaluate its performance.The companys calculation of non-GAAP financial measures likely differs from the calcul

29、ations used by other companies.The company provides guidance regarding combined R&D,AIPR&D and SG&A expenses and effective tax rate on a non-GAAP basis.Unless,otherwise noted,the guidance regarding combined R&D,AIPR&D and SG&A expenses does not include estimates associated with any potential future

30、business development transactions,including collaborations,asset acquisitions and/or licensing of third-party intellectual property rights.The company does not provide guidance regarding its GAAP effective tax rate because it is unable to forecast with reasonable certainty the impact of excess tax b

31、enefits related to stock-based compensation and the possibility of certain discrete items,which could be material.Non-GAAP financial measures are presented compared to corresponding GAAP measures in the appendix hereto.A reconciliation of the GAAP financial results to non-GAAP financial results is i

32、ncluded in the companys Q1:25 and Q4:24 press releases dated May 5,2025 and February 10,2025.2025 Vertex Pharmaceuticals Incorporated 2025 Vertex Pharmaceuticals Incorporated 4Vertex delivered a strong start to 2025,as we extend leadership in CF,execute multiple launches and advance the pipelineCFTR

33、m=cystic fibrosis transmembrane conductance regulator modulators;MAD:Multiple Ascending Dose;T1D:type 1 diabetes;IgAN:IgA nephropathy;AMKD:APOL-1 mediated kidney disease;pMN:primary membranous nephropathy.Expand CF leadership ALYFTREK:Approved in the U.S.and U.K.for patients with CF ages 6+;strong U

34、.S.launchVX-828 combo(next gen 3.0 CFTRm regimen):on track to initiate clinical trial in CF patients by EoYVX-522(mRNA):Implemented pause to MAD portion of Ph 1/2 study to assess a tolerability issueDrive commercial diversificationCASGEVY:Launch gathering momentum across all regionsJOURNAVX:Approved

35、 in U.S.for moderate-to-severe acute pain;strong early uptake across the hospital and retail settingsPreparing for additional launches:4 programs in pivotal development+5th to start in 2025(pMN)Advance broad and deep clinical-stage pipeline Povetacicept(IgAN)Phase 3 interim analysis(IA)cohort fully

36、enrolled,potential U.S.filing H1:26 Zimislecel(T1D)Phase 3 dosing to complete in Q2:25,potential global filings in 2026Inaxaplin(AMKD)Phase 3 IA cohort on track to complete enrollment by end of yearRapid progress with earlier stage programs:VX-993(oral)acute pain study to complete this quarter and d

37、ata expected in H2:25VX-407 to advance to Phase 2 POC for ADPKD in H2:25Deliver strong financial performanceQ1:25 revenue$2.77B;raised low end of 2025 total revenue guidance to$11.85-$12.0BDrive revenue growth:CF as foundation with increasing contributions from CASGEVY,ALYFTREK and JOURNAVX through

38、2025Deliver attractive operating margin while continuing to invest in pipeline;disciplined spend;commitment to specialty model2025 Vertex Pharmaceuticals Incorporated 5Expanding CF leadership:ALYFTREK approved in U.S.&U.K.and Positive CHMP Opinion in EUVertex CFTR modulators have the potential to tr

39、ansform the lives of nearly 95%of patients with CFNG:next generation;CFTR:cystic fibrosis transmembrane conductance regulator;HBE:human bronchial epithelial cells;MAD:multiple ascending dose.VX-522 mRNA approach for 5,000 patients who cannot benefit from CFTRm Temporary pause implemented to MAD port

40、ion of Phase 1/2 study to assess a tolerability issueSerial innovation:fifth CF launch since 2012Potential to set new standard of CF careLabel includes additional 31 rare mutations beyond TRIKAFTAPotential approvals in 2025:EU,Australia,New Zealand,Canada,Switzerland Next-generation CFTRm VX-828 com

41、bination therapy:Most efficacious CFTR corrector Vertex has ever studied in vitro in HBE assaysExpect to initiate study in CF patients in 2025Patients 1 month and olderPatients 1 year and olderPatients 6 years and olderN.G 1.0 regimen,ages 2+N.G 2.0 regimen,ages 6+N.G 3.0 regimen2025 Vertex Pharmace

42、uticals Incorporated 6Vertex is committed to long-term leadership in pain with a broad and deep pipeline JOURNAVX NaV1.8 inhibitor-AcuteOralSuzetrigine NaV1.8 inhibitor-DPNOralSuzetrigine NaV1.8 inhibitor-LSROralVX-993 NaV1.8 inhibitor-AcuteOralVX-993 NaV1.8 inhibitor-DPNOralVX-993 NaV1.8 inhibitor

43、IVVX-973 NaV1.8 inhibitorOralAdditional NaV1.8 inhibitors Oral and IVNaV1.7 inhibitorsOralFORMULATIONRESEARCHPHASE 1PHASE 2PHASE 3APPROVEDDPN:diabetic peripheral neuropathy;LSR:lumbosacral radiculopathy;IV:intravenous.Acute PainPeripheral Neuropathic Pain(PNP)2025 Vertex Pharmaceuticals Incorporated

44、 2025 Vertex Pharmaceuticals Incorporated 7Zimislecel:Pivotal trial will complete enrollment and dosing in Q2:25,with global regulatory submissions planned for 2026Total Daily InsulinEASD 2024Data presented on 12 patients who completed Day 180 visit:were no longer using exogenous insulin*911had elim

45、ination or reduction of exogenous insulin use*Although prohibited by protocol,two patients had steroid use in the peri-infusion period.One participant had one dose of steroids on the day of zimislecel infusion and did reduce(43%),but not eliminate,exogenous insulin.One participant received four dose

46、s of steroids in the peri-infusion period and saw a 12%increase in their use of exogenous insulin.HbA1c12All 12 patients achieved a reduction in HbA1c to 750K China)Povetacicept pMN150KPovetacicept LN225KPovetacicept AAV225KAPOL1 mediated kidney disease(AMKD)Inaxaplin Primary AMKD150KInaxaplin AMKD

47、with comorbidities3100KAdditional APOL1 inhibitors4250K(150K+100K)Autosomal dominant polycystic kidney diseaseVX-4075Up to 30KAdditional ADPKD serial innovation300K(incl.30K)Kidney:Broad pipeline of potentially transformative medicines in multiple serious renal diseases1.Estimated patient population

48、 in the U.S.and Europe,unless otherwise noted.2.IgAN patients continue to be studied in RUBY-3 3.AMPLIFIED Phase 2 trial began January 2025.4.Multiple programs in various phases.5.Targets a patient population with a subset of variants in the PKD1 gene.IgAN:IgA nephropathy;pMN:primary membranous neph

49、ropathy;LN:lupus nephritis;AAV:Antineutrophil cytoplasmic antibody(ANCA)-associated vasculitides.PATIENTS1RESEARCHPHASE 1PHASE 2PHASE 3APPROVEDEnrollment complete in IA cohortMoving to pivotalDevelopment H2:252025 Vertex Pharmaceuticals Incorporated 9Povetacicept:IgAN-completed enrollment of interim

50、 analysis cohort in global Phase 3 study;pMN-advancing to pivotal development Reached agreement with FDA to advance into pivotal development for pMN Single Phase 2/3 adaptive study vs.standard of care Primary endpoint:complete clinical remission at 72 weeks of treatmentStrong preclinical profile:Dua

51、l antagonist of BAFF/APRIL cytokines with high affinity and potencyCompelling RUBY-3 data(ASN 2024):Reduced UPCR mean 66%at 48 weeks;stable renal function(eGFR)63%achieved clinical remission*Convenient dosing:Once every four weeks,at home Subcutaneous Small volumeBest-in-class potential Pove 80mg vs

52、 placebo on top of standard of care(n=480)Completed enrollment of the interim analysis(IA)cohort:If IA is positive,potential to file for accelerated approval in the U.S.in H1:26RAINIER Phase 3 trial in IgAN*Defined as UPCR(urine protein creatinine ratio)65 ATCs activated and 90 patients have had fir

53、st cell collections*8 CASGEVY infusions in Q1 2025Expanding manufacturing to support global demand*From launch through May 1,2025.*Majority of Emirates.ATC:authorized treatment center;CMMI:Center for Medicare and Medicaid Innovation.OUS Reimbursed access in multiple countries:England,Wales,Denmark,A

54、ustria,Luxembourg,KSA,Bahrain,UAE*Payer Highlights2025 Vertex Pharmaceuticals Incorporated 2025 Vertex Pharmaceuticals Incorporated 14 Health economics research presented at AAPM:Replacing 25%acute pain opioid Rxs with non-opioids could deliver$4.5B annual savings Federal legislation:NOPAIN Act effe

55、ctive on Jan.1;expect JOURNAVX to be added near-term to list of medicines covered 35 states have enacted or proposed legislation to support use of non-opioids Retail pharmacy stocking:JOURNAVX was broadly available at 33,000 retail locations by mid-March 20,000 prescriptions filled through April 18t

56、hWide availability,strong early uptake 94 million covered lives with access to JOURNAVX(as of May 1)Coverage across commercial and government plans 42 million with unrestricted access:no prior authorizations/step editsRapid progress with payersCompelling data support policy tailwindsJOURNAVX:FDA app

57、roved Jan 30,2025Strong early launch signalsAAPM:American Academy of Pain Medicine.2025 Vertex Pharmaceuticals Incorporated 15Q1 2025 financial highlights($in millions except where noted or per share data and percentages)Q1:24FY:24Q1:25Total revenue$2.69B$11.02B$2.77BTRIKAFTA/KAFTRIO2.48B10.24B2.54B

58、ALYFTREK-54Other product revenue*207782171Combined non-GAAP,Acquired IPR&D and SG&A expenses 1.02B8.82B1.23BNon-GAAP operating income 1.34B6961.18BNon-GAAP operating margin%50%6%43%Non-GAAP net income 1.24B1111.05BNon-GAAP net income per share diluted$4.76$0.42$4.06Cash,cash equivalents&total market

59、able securities(period-end)$14.6B$11.2B$11.4BNotes:An explanation of non-GAAP financial measures and reconciliation of combined non-GAAP R&D,Acquired IPR&D and SG&A expenses,non-GAAP operating income,non-GAAP net income and non-GAAP net income per share diluted to corresponding GAAP measures are inc

60、luded in the companys Q1:25 and Q4:24 press releases dated May 5,2025 and February 10,2025.Non-GAAP financial measures are presented compared to corresponding GAAP measures in the appendix of this presentation.Totals above may not add due to rounding.*Q1:25 includes$14 million CASGEVY revenues and a

61、n insignificant amount from JOURNAVX.FY:24 includes$10M CASGEVY revenues.2025 Vertex Pharmaceuticals Incorporated 16Updated full year 2025 financial guidanceCurrentFY 2025 GuidancePreviousFY 2025 GuidanceCommentaryTotal Revenue$11.85-$12.0B$11.75-$12.0BIncludes expectations for continued growth in C

62、F,including the launch of ALYFTREK;continued uptake of CASGEVY;and a contribution from the launch of JOURNAVX,primarily in the second half of 2025Combined GAAP R&D,Acquired IPR&D and SG&A ExpensesUnchanged$5.55-$5.7BReflects investment in our multiple mid-and late-stage clinical development programs

63、 and ramp of commercial capabilities;ranges include approximately$100 million in currently anticipated IPR&D expensesCombined Non-GAAP R&D,Acquired IPR&D and SG&A ExpensesUnchanged$4.9-$5.0BNon-GAAP Effective Tax RateUnchanged20.5%-21.5%*Note that GAAP 2025 operating income,net income,and EPS will r

64、eflect an intangible asset impairment charge of$379M recorded in Q1:25 associated with VX-264(the cells plus device program)in patients with type 1 diabetes(T1D),which will not be advancing further in clinical development.2025 Vertex Pharmaceuticals Incorporated 17Multiple catalysts throughout 2025A

65、NTICIPATED KEY MILESTONESALYFTREK(CF)Drive U.S.launch,secure reimbursement in U.K.and secure regulatory approvals in EU and other geographiesVX-522(CF)Temporary pause implemented to assess a tolerability issueNext-generation 3.0(CF)VX-828(next-generation CFTR corrector)combo on track to initiate CF

66、patient trial this yearCASGEVY(SCD/TDT)Reach more eligible 12+year-old patients across geographies Work to expand label to younger age groupsSuzetrigine(pain)Acute:JOURNAVX approved 1/30/25 for moderate to severe acute pain;U.S.launch underway PNP-DPN:Enroll and dose ongoing Phase 3 pivotal trial;Ad

67、vance LSR to Phase 3,pending regulatory discussionsVX-993(pain)Acute:Complete Phase 2 study(BUN;oral)in Q2:2025,share results in H2:2025 PNP-DPN:Continue to progress Phase 2 study(DPN;oral)Zimislecel/VX-880(T1D)Complete enrollment and dosing in pivotal trial Q2:25;potential for global regulatory sub

68、missions in 2026,after patients achieve insulin independence and reach 1 year of insulin-free follow-upInaxaplin(AMKD)Complete enrollment in IA cohort in 2025;following 48 weeks of treatment;potential to file for U.S.accelerated approvalPovetacicept(IgAN,etc.)IgAN:Completed enrollment in IA cohort;f

69、ollowing 36 weeks of treatment,potential to file for U.S.accelerated approval in H1:2026 pMN:Initiate Phase 2/3 pivotal trial in H2:2025 Other autoimmune renal/cytopenia indications:results from additional cohorts in RUBY-3/RUBY-4 Phase 2 basket studies in 2025VX-407(ADPKD)Begin Phase 2 proof-of-con

70、cept study in ADPKD patients in H2:2025VX-670(DM1)Advance MAD portion of Phase 1/2 study in DM1 patients,which will evaluate both safety and efficacy 2025 Vertex Pharmaceuticals Incorporated 2025 Vertex Pharmaceuticals Incorporated FIRST QUARTER 2025 FINANCIAL RESULTS2025 Vertex Pharmaceuticals Inco

71、rporatedFirst Quarter 2025Financial ResultsMay 5,20252025 Vertex Pharmaceuticals Incorporated 19Appendix AGAAP to non-GAAP Financial Information($in millions except as noted,per share data and percentages)Q1:24FY:24Q1:25Combined R&D,Acquired IPR&D and SG&AGAAP1.21B9.72B1.40BNon-GAAP1.02B8.82B1.23BOp

72、erating incomeGAAP1.14B(233)630Non-GAAP 1.34B6961.18BOperating Margin%:GAAP42%(2)%23%Non-GAAP 50%6%43%Net incomeGAAP1.10B(536)646Non-GAAP 1.24B1111.05BNet income per share dilutedGAAP$4.21$(2.08)$2.49Non-GAAP$4.76$0.42$4.06Shares used in diluted per share calculationsGAAP261.1257.9259.5Non-GAAP 261.

73、1260.9259.5Note:An explanation of non-GAAP financial measures and reconciliation of combined non-GAAP R&D,Acquired IPR&D and SG&A expenses,non-GAAP operating income,non-GAAP net income and non-GAAP net income per share diluted to corresponding GAAP measures are included in the companys Q1:25 and Q4:

74、24 press releases dated May 5,2025 and February 10,2025.2025 Vertex Pharmaceuticals Incorporated 20Vertex targeted disease area epidemiology estimates DISEASE STATEASSETAPPROACH/MODALITYPATIENT OPPORTUNITYCOMMERCIALIZEDCystic fibrosis5 approved,incl.ALYFTREKSmall molecules109,000 Sickle cell disease

75、+TDTCASGEVYCell and gene therapy60,000 severeAcute PainJOURNAVXSmall molecule NaV1.8 inhibitor80MIN PIVOTAL STUDIES(in progress or near-term)Peripheral neuropathic painSuzetrigineSmall molecule NaV1.8 inhibitor10MAMKDInaxaplinSmall molecule inhibitor250,000T1DZimislecelOther approachesCell and gene

76、therapy 60,000 w/initial filing*3.8MIgA nephropathyPovetaciceptFusion protein300K U.S./Europe750K ChinapMNPovetaciceptFusion protein150,000PIPELINEDM1VX-670Oligonucleotide with cyclic peptide110,000 CFVX-522mRNA5,000*ADPKDVX-407Small molecule corrector300,000*Zimislecel initial program seeks first a

77、pproval for 60,000 patients;Vertex will seek to serve the full 125,000 patient population with severe T1D over time.*VX-522 targets a patient population that does not make any CFTR protein and is a subset of the 109,000 overall CF patient population.*VX-407 targets a patient population with a subset of variants in the PKD1 gene,estimated at up to 30,000(or 10%)of the overall patient population.