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1、J.P.MORGAN HEALTHCARE CONFERENCEJANUARY 2024RESHMA KEWALRAMANI,M.D.CEO AND PRESIDENT2024 Vertex Pharmaceuticals IncorporatedSAFE HARBOR STATEMENT&NON-GAAP FINANCIAL MEASURESThis presentation contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995,includ
2、ing,without limitation,the information provided regarding and expectations for future financial and operating performance andstatements regarding(i)expectations,development plans and timelines for the companys medicines and pipeline programs,including anticipated 10 disease areas in clinical develop
3、ment in 2024,expectations for five potential launches in five years,anticipated near-term commercial opportunities in TDT,CF and acute pain,anticipated benefits of new products and relevant patientpopulations,and plans to broaden and deepen R&D pipeline across modalities,(ii)plans to launch CF medic
4、ines in younger agegroups globally and to bring additional molecules to market to get CF patients to carrier levels of SwCl,(iii)plans to advance VX-522 toreach the 5,000 CF patients who cannot benefit from a CFTR modulator,(iv)expectations for CASGEVY,including the potentialbenefits for patients wi
5、th SCD and TDT,expectations for broad access in the US,expectations for ex-US access initially through EarlyAccess Programs and efforts for long-term reimbursement agreements,plans to obtain approvals in additional geographies,and plansto complete dosing in younger age groups,(v)expectations for our
6、 pain program,including plans for near-term launch andcommercial potential in acute pain,expectation for treatment of acute pain without side effects of addictive properties of opioids,plans to share data from the acute pain pivotal program and submit regulatory filings,plans to advance VX-993 into
7、Phase 2 for acutepain and to initiate Phase 1 for VX-993 intravenous formulation,plans to engage in meetings with regulators with goal of broad PNPlabel,advance VX-548 in DPN into pivotal development in 2024,and to enroll and dose VX-548 Phase 2 study in LSR,and plans toadvance NaV 1.7 and NaV1.8 in
8、hibitors,(vi)expectations for our T1D program,including plans for VX-880 and VX-264 studies,(vii)expectations for vanzacaftor triple combination therapy,including our plan share Phase 3 data in early 2024,(viii)expectations forinaxaplin,including dose selection and movement into Phase 3 in Q1 2024,e
9、xpectations for accelerated approval in US,and potentialcommercial opportunity,(ix)expectations for our ability to reinvest in our pipeline,(ix)expectations for our DM1 program,and(x)plans to file an IND and CTA for VX-407 and initiate first in human trial.While Vertex believes the forward-looking s
10、tatementscontained in this presentation are accurate,these forward-looking statements represent the companys beliefs as of the date of thispresentation and there are risks and uncertainties that could cause actual events or results to differ materially from those expressedor implied by such forward-
11、looking statements.Those risks and uncertainties include,among other things,that data from clinicaltrials,especially if based on a limited number of patients,may not to be indicative of final results,the company may not be able toscale up manufacturing of our product candidates,actual patient popula
12、tions eligible for our products may be smaller thananticipated,data from the companys development programs may not be available on expected timelines,or at all,supportregistration or further development of its potential medicines due to safety,efficacy or other reasons,and other risks listed under t
13、heheading“Risk Factors”in Vertexs annual report and subsequent quarterly reports filed with the Securities and Exchange Commissionat www.sec.gov and available through the companys website at .You should not place any undue reliance on thesestatements,or the data presented.Vertex disclaims any obliga
14、tion to update the information contained in this presentation as newinformation becomes available.In this presentation,Vertex references financial guidance and results that have been provided in accordance with US GAAP andcertain non-GAAP financial measures.Management believes these non-GAAP financi
15、al measures help indicate underlying trends inthe companys business,are important in comparing current results with prior period results and provide additional informationregarding the companys financial position.A reconciliation of the GAAP financial results to non-GAAP financial results is include
16、d inthe appendix hereto.2024 Vertex Pharmaceuticals Incorporated2DIFFERENTIATED VERTEX BUSINESS MODEL AND R&D STRATEGY2024 Vertex Pharmaceuticals IncorporatedDIFFERENTIATED BUSINESS MODELInvestment of majority of OpEx in R&D and BD/external innovation Creation of high-value transformative medicines
17、for specialty marketsLimited SG&A expenses and infrastructureRevenue growth:high operating margins and significant cash flowWE FOCUS ON Diseases where causal human biology is knownValidated targets Biomarkers that translate from bench to bedside Best modality(i.e.,modality agnostic)Efficient develop
18、ment®ulatory pathwaysIN ORDER TO DELIVER Transformative medicines for patientsGreater likelihood of clinical success Sustained innovationShareholder value3Approved medicinesacross CF,sickle cell disease and beta thalassemiaMultiple near-term commercial opportunities*CASGEVY(TDT U.S.;SCD and TDT E
19、U,KSA)Vanzacaftor triple(CF)VX-548(acute pain)Broad,diversified pipeline in clinical development Inaxaplin(AMKD)VX-548(DPN)VX-548(LSR)VX-880(T1D)VX-264(T1D)VX-522(CF-mRNA)VX-670(DM1)4TDT:transfusion-dependent beta thalassemia;AMKD:APOL1-mediated kidney disease;T1D:type 1 diabetes;DPN:diabetic periph
20、eral neuropathy;LSR:lumbosacral radiculopathy;DM1:myotonic-dystrophy type 1*Subject to regulatory approval 2024 Vertex Pharmaceuticals IncorporatedEXPANDING LEADERSHIP IN CF AND ENTERING A NEW ERA OF DIVERSIFICATION IN MULTIPLE SERIOUS DISEASES2024 Vertex Pharmaceuticals Incorporated5IN 2024,WE ANTI
21、CIPATE 10 DISEASE AREAS IN CLINICAL DEVELOPMENTSTRONG PROGRESS TOWARDS OUR GOAL OF FIVE LAUNCHES OVER FIVE YEARS(2028)Type 1 DiabetesAcute Pain Neuropathic PainAPOL1-Mediated Kidney DiseaseSickle Cell Disease Beta ThalassemiaAlpha-1 Antitrypsin Deficiency Cystic Fibrosis92,000+additional countries10
22、0,000100,0003.8M90M(U.S.)Images not to scale;Illustrative purposes.Patient populations include U.S.,Europe,and select geographies.*Over 5,000 people with CF cannot benefit from CFTR modulators and thus may potentially benefit from VX-522,our mRNA program.#Select PKD1 mutations.5,000*35,000 severe+KS
23、A&BahrainMyotonic Dystrophy Type 1110,000260,000Autosomal Dominant Polycystic Kidney Disease25,000#125,000 DURABLE GROWTH DRIVERSTreating younger patientsPatients living longerSerial CFTRm innovation mRNA for last 5,000 patients6Vanzacaftor tripleVX-522mRNABest-in-classmedicinesCystic Fibrosis Appro
24、valsEXPANDING LEADERSHIP IN CF AND RAISING THE BAR WITH SERIAL INNOVATION Next-in-class CFTR modulator with potential for improved benefit Convenient,once-daily dosing Meaningfully lower royalty burden Phase 3 studies completed;results in early 2024 For CF patients who cannot benefit from CFTR modul
25、ators Completed dosing SAD and initiated MAD portion of study in people with CFGoal:carrier levels of CFTR function2024 Vertex Pharmaceuticals Incorporated*Patient populations include North America,Europe,and Australia.92,000 patients with CF*patients with CF*20,000 eligible patients not eligible pa
26、tients not on CFTR on CFTR modulatorsmodulators2024 Vertex Pharmaceuticals IncorporatedCASGEVY(EXA-CEL):NEW ERA OF DIVERSIFICATION WITH LAUNCHES IN TWO DISEASES OUTSIDE CFMHRA:Medicines and Healthcare Products Regulatory Agency(Great Britain);BFDA:Bahrain FDACRISPR/Cas9 precisely targets the erythro
27、id-specific enhancer region of the BC11A gene.Rapid pace of global approvals:By the FDA for eligible patients ages 12+with sickle cell disease By the MHRA and BFDA for eligible patients ages 12+with sickle cell disease or transfusion-dependent beta thalassemiaThe first precise,durable,CRISPR/Cas9 ge
28、ne-edited therapy,delivering a potential one-time functional cure for patients with SCD and TDT.Payers81Source:Vertex market research,April-June 2022;patient research is U.S./EuropeATC:Authorized Treatment CenterCASGEVY REPRESENTS A POTENTIAL MULTI-$B OPPORTUNITY FOR VERTEXFOUNDATIONAL YEAR IN 2024
29、ProvidersPatients2024 Vertex Pharmaceuticals IncorporatedVictoria Gray,Sickle Cell Warrior First SCD patient dosed35,000Severe patients in U.S.,Europe+additional in KSA,Bahrain25%of surveyed SCD and TDT patients strongly believe genetic therapy is right choice for them175ATCs targeted in U.S.and Eur
30、ope;9 U.S.and 3 Europe ATCs activated75%of surveyed U.S.providers prefer a gene-edited approach over other gene therapy mechanisms1U.S.Anticipate broad access with government and commercial payersOUSAnticipate Early Access Programs initially;pursuing long-term reimbursement agreementsSERIAL INNOVATI
31、ON,BROAD/DEEP PIPELINE FOR LEADERSHIP IN MULTIPLE PAIN STATES GIVEN HIGH UNMET NEEDVERTEX IS COMMITTED TO TRANSFORMING THE TREATMENT OF PAIN2024 Vertex Pharmaceuticals Incorporated9VX-548 NaV1.8 inhibitor*-Acute painOralVX-548 NaV1.8 inhibitor-DPNOralVX-548 NaV1.8 inhibitor-LSROralVX-993 NaV1.8 inhi
32、bitorOralVX-973 NaV1.8 inhibitorOralVX-993 NaV1.8 inhibitor IVAdditional NaV1.8 inhibitors Oral and IVNaV1.7 inhibitorsOralFORMULATIONRESEARCHPHASE 1PHASE 2PHASE 3APPROVEDDPN:diabetic peripheral neuropathy;LSR:lumbosacral radiculopathy;IV:intravenous*Phase 3 program in acute pain has completed;data
33、expected in early 2024.Acute PainPeripheral Neuropathic Pain(PNP)VX-548 FOR ACUTE PAIN:POTENTIAL FOR EFFECTIVE PAIN RELIEF WITHOUT THE SIDE EFFECTS OR ADDICTIVE PROPERTIES OF OPIOIDS2024 Vertex Pharmaceuticals Incorporated10PIVOTAL PROGRAM COMPLETE;RESULTS EXPECTED EARLY 2024xSelectively inhibits Na
34、V1.8 channel in peripheral nervesInhibition of NaV1.8 reduces action potential signaling,preventing pain transmissionOrigin of pain NaV1.8 and 1.7 are validated targets for pain Phase 3 trials of VX-548 in abdominoplasty,bunionectomy and a single arm safety and effectiveness trial all complete;resul
35、ts early 2024 Program targets a broad label in moderate to severe acute pain Acute pain is a multi-billion-dollar market today with significant unmet need;80M patients annually U.S.Existing treatment options have limitations around efficacy,side effects and addiction potential We aim to transform th
36、e treatment of acute pain with effective and well-tolerated non-opioid optionsSelective NaV inhibitors,starting with VX-548Future selective NaV inhibitorsOTC onlyAnti-Inflammatory/AnalgesicsTODAYFUTUREOpioidsVX-548 FOR PERIPHERAL NEUROPATHIC PAIN:LARGE MARKET WITH HIGH UNMET NEED;10M PATIENTS ANNUAL
37、LY IN U.S.POSITIVE PHASE 2 RESULTS IN DPN ARE FIRST DEMONSTRATION OF SAFETY&EFFICACY IN THE CHRONIC SETTING2024 Vertex Pharmaceuticals Incorporated11LS Mean=least square mean,SE=standard error;Low Dose=23 mg qd,Mid Dose=46 mg qd,High Dose=69 mg qd;qd=once a day,TID=three times a day.The pregabalin a
38、rm serves as a reference.Study was not designed or powered for comparisons between the VX-548 arms or between VX-548 and pregabalin.Change from baseline in weekly average NPRS at 12 weeksLS Mean(SE)Pregabalin100mg TIDN=47-2.09(0.29)VX-548 Low DoseN=24VX-548 Mid DoseN=48VX-548 High DoseN=48-2.18(0.39
39、)-2.11(0.28)-2.26(0.28)P value$2.7B$0.5B2023EDIFFERENTIATED BUSINESS MODEL DELIVERS PROFITABLE REVENUE GROWTH,FUELING CONTINUED INVESTMENT IN INNOVATIONNote:2019 CF revenues are non-GAAP;See appendix for reconciliations of GAAP to non-GAAP 2019 CF revenues and GAAP to non-GAAP 2019-2023E research an
40、d development(“R&D”)expenses;2023E CF revenue reflects the product revenue guidance provided on 11/6/23;2023E non-GAAP R&D and Acquired IPR&D expenses are based on combined non-GAAP R&D,Acquired IPR&D and SG&A expenses guidance provided on 11/6/23;this slide is intended to be illustrative and is not
41、 intended to be a reiteration of guidance.$4.0B$6.2B$7.6B$8.9B200222023E$9.85BAcquired IPR&DCF RevenueNon-GAAP R&DBD Activity2024 Vertex Pharmaceuticals Incorporated.enables significant investment ininternal and external innovationStrong,sustained CF revenue growth.2024 Vertex Pharmaceuti
42、cals IncorporatedOUR GOAL IS TO CONSISTENTLY DELIVER TRANSFORMATIVE THERAPIES AND SHAREHOLDER VALUEExpand our leadership and raise the bar in CF qLaunch in younger age groups globallyqBring additional molecules to market to get CF patients to carrier levels of CFTR functionqAdvance VX-522 to reach t
43、he 5,000 patients who cannot benefit from a CFTR modulatorDrive era of diversification with multiple commercial launch opportunitiesqLaunch CASGEVY;obtain approvals in additional geographiesqPrepare to file and launch VX-548 in moderate to severe acute painqComplete late-stage clinical development p
44、rograms in support of five launches in five years(2028)goalBroaden and deepen R&D pipeline across modalitiesqAdvance multiple programs across multiple modalities into pivotal developmentqAchieve proof-of-concept for additional sandbox disease areasqProgress next wave of innovation into the clinic,st
45、arting with DM1 and ADPKDDeliver financial performanceqContinue CF product revenue growth;incremental sales from launches in new disease areasqSustain strong operating margins and continue to invest in pipeline,with focus on specialty model17APPENDIXMULTIPLE CATALYSTS THROUGHOUT 2024 AND BEYOND2024
46、Vertex Pharmaceuticals Incorporated19RECENT HIGHLIGHTSANTICIPATED KEY MILESTONES Received approvals for TRIKAFTA in EU,U.K.and Canada in patients with CF ages 2 to 5 Completed vanzacaftor/tezacaftor/deutivacaftor Phase 3 studies(ages 6-11 and 12+)VX-522 CFTR mRNA study:completed dosing SAD portion,i
47、nitiated MAD portion in CF patients Launch TRIKAFTA/KAFTRIO OUS in ages 2-5 years Share vanza triple Phase 3 data early 2024 Complete MAD portion of the VX-522 CFTR mRNA study Received approval for CASGEVY in U.S.for SCD;in U.K.and Bahrain for TDT and SCD Received CHMP positive recommendation for ex
48、a-cel in SCD and TDT in EU Regulatory reviews ongoing in U.S.for TDT;in KSA and Switzerland for SCD and TDT Enrollment completed in global Phase 3 studies in patients with SCD or TDT ages 5 to 11 Launch CASGEVY in U.S.,U.K.,Bahrain Secure additional global regulatory approvals:U.S.(TDT);EU,KSA,Switz
49、erland(SCD and TDT)Complete dosing in younger age group Completed VX-548 Phase 3 trials in acute pain Reported VX-548 positive Phase 2 PNP results in diabetic peripheral neuropathy(DPN)Initiated VX-548 Phase 2 PNP study in lumbosacral radiculopathy(LSR)Completed VX-993 Phase 1 study(oral);completed
50、IND-enabling studies(IV)VX-548:share acute pain data early 2024;prepare for filing,launch VX-548:DPN End-of-Phase 2 meeting Q1:24;initiate pivotal trials VX-548:LSR trial continue enrollment and dosing VX-993:initiate acute pain studies Phase 2 oral and Phase 1 IV VX-993:initiate neuropathic pain st
51、udies Phase 2 oral Completed enrollment in Phase 2B portion of Phase 2/3 pivotal trial of inaxaplin in AMKD Select dose and advance to Phase 3 portion of study in Q1:24 VX-880:Phase 1/2 trial fully enrolled(Parts A,B,C)for T1D VX-264(the“cells+device”program):enrolling and dosing in Phase 1/2 trial
52、VX-880:Review Phase 1/2 data,resume trial when appropriate VX-264:Complete Part A of Phase 1/2 trial,proceed to Part B CTAs cleared in Canada and U.K.for VX-670 in DM1;study initiated in Canada Enroll and dose VX-670 study in DM1 patients in Canada,start U.K.Work with FDA to address comments,lift ho
53、ld,initiate study in the U.S.Completed IND-enabling studies for VX-407 in ADPKD File IND and CTA;initiate first-in-human trialRECONCILIATION OF GAAP TO NON-GAAP FINANCIAL INFORMATION20022Q323YTDGAAP Research and Development(“R&D”)Expenses$1.44B$1.64B$1.94B$2.54B$2.34BStock-based compensat
54、ionexpense(225)(263)(268)(298)(232)Other adjustments(41)(10)(11)(38)(9)Non-GAAP R&D Expenses1.17B1.37B1.66B2.20B2.10B2019GAAP total revenues$4.16BORKAMBI adjustment (156)Non-GAAP total revenues4.01BNote:“ORKAMBI adjustment”represents a 2019 adjustment to reflect the conclusion of Vertexs early acces
55、s program for ORKAMBI in France.Prior to 2019,Vertex had only recognized a portion of net product revenues related to ORKAMBI distributed through the early access program in France.As a result,Vertex recognized an adjustment to increase net product revenues,which related to prior period shipments of
56、 ORKAMBI distributed through the early access program in France.Vertex removed this amount from its 2019 non-GAAP product revenues.All numbers in the reconciliation tables below are in millions except where noted.Note:Beginning in 2022,Vertex no longer excludes research and development charges resul
57、ting from upfront or contingent milestone payments in connection with collaborations,asset acquisitions and/or licensing of third-party intellectual property rights from its Non-GAAP financial measures.These charges are included as Acquired in-process research and development expenses,and were previously included in Research and development expenses,in Vertexs consolidated statements of operations.The non-GAAP R&D expenses for 2019-2021 above have been recast to reflect this change.Non-GAAP R&D expenses for 2023 are based on the nine-months ended September 30,2023.