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1、Vertex Reports First Quarter 2025 Financial ResultsMay 5,2025 Total revenue of$2.77 billion,a 3%increase compared to Q1 2024;raised the low end of total revenue guidance by$100 million to a new range of$11.85 to$12 billion Strong progress with CASGEVY,ALYFTREK and JOURNAVX launches Povetacicept(pove

2、)IgAN Phase 3 interim analysis(IA)cohort fully enrolled and zimislecel Phase 3 program to complete dosing this quarter,settingup potential filings in 2026;inaxaplin Phase 3 IA cohort on track to complete enrollment in the second half of 2025 Four programs already in pivotal development,with pivotal

3、study of pove in primary membranous nephropathy(pMN)to start this year BOSTON-(BUSINESS WIRE)-May 5,2025-Vertex Pharmaceuticals Incorporated(Nasdaq:VRTX)today reported consolidated financial results forthe first quarter ended March 31,2025,and raised the low end of its total revenue guidance range b

4、y$100 million,from$11.75 billion to$12 billion to anew range of$11.85 billion to$12 billion.“Vertex delivered a strong start to 2025 with notable execution across the business as we grow and diversify the revenue base,progress multiplelaunches and advance the R&D pipeline.We continued to expand our

5、leadership in CF and build global momentum for CASGEVY,and we launchedJOURNAVX in moderate-to-severe acute pain,”said Reshma Kewalramani,M.D.,Chief Executive Officer and President of Vertex.“With multipleprograms in pivotal development including povetacicept,which continues to make rapid progress in

6、 achieving its potential as a pipeline-in-a-product,and additional programs in early and mid-stage development,Vertex is poised to continue to deliver value for years to come.”First Quarter 2025 ResultsTotal revenue increased 3%to$2.77 billion compared to the first quarter of 2024,primarily driven b

7、y the continued performance ofTRIKAFTA/KAFTRIO and an early contribution from the U.S.launch of ALYFTREK.In the U.S.,total revenue increased 9%to$1.66 billion due tocontinued strong patient demand and higher net realized pricing.Outside the U.S.,total revenue decreased 5%to$1.11 billion as strong pa

8、tientdemand in both established and newer markets was offset by the expected revenue decline in Russia,where Vertex is experiencing violation of itsintellectual property rights.Vertex has strong intellectual property protection for its medicines around the world and believes this violation in Russia

9、 is alimited and isolated matter.Combined GAAP and Non-GAAP R&D,Acquired IPR&D and SG&A expenses were$1.4 billion and$1.2 billion,respectively,compared to$1.2billion and$1.0 billion,respectively,in the first quarter of 2024.The increases were primarily due to continued R&D investment in support of m

10、ultiplemid-and late-stage clinical development programs and increased commercial investment to support the launch of JOURNAVX.Intangible asset impairment charge of$379.0 million associated with VX-264(the“cells plus device”program)in patients with type 1 diabetes,which will not be advancing further

11、in clinical development,was included in GAAP operating income in the first quarter of 2025.GAAP effective tax rate was 11.5%compared to 14.0%for the first quarter of 2024.Non-GAAP effective tax rate was 18.8%compared to 17.4%for the first quarter of 2024.The difference between the GAAP and non-GAAP

12、effectivetax rates was primarily due to tax benefits related to stock-based compensation.GAAP net income was$646 million compared to$1.1 billion for the first quarter of 2024,as a result of increased operating expenses and theintangible asset impairment charge.Non-GAAP net income was$1.1 billion com

13、pared to$1.2 billion in the first quarter of 2024,as a result of increased operating expenses.Cash,cash equivalents and total marketable securities as of March 31,2025,were$11.4 billion,compared to$11.2 billion as of December 31,2024.The increase was primarily due to cash flows from operating activi

14、ties,partially offset by repurchases of Vertexs common stock pursuant to itsshare repurchase program.Full Year 2025 Financial GuidanceVertex today raised the low end of its full-year 2025 revenue guidance range from$11.75 to$12 billion to$11.85 billion to$12 billion,which assumescontinued growth in

15、CF,including the launch of ALYFTREK;as well as continued uptake of CASGEVY in multiple regions;and early contributions fromthe launch of JOURNAVX.Vertex reiterated its guidance for both combined GAAP and Non-GAAP R&D,AIPR&D and SG&A expenses,whichincludes expectations for continued investment in mul

16、tiple mid-and late-stage clinical development programs and commercial capabilities,andAIPR&D expenses of approximately$100 million.This guidance also includes an immaterial cost impact from tariffs in 2025 based on currently knowntariff rates and regulations.Vertexs financial guidance is summarized

17、below:Current FY 2025 FY 2025 Total revenue$11.85 to$12.0 billion$11.75 to$12.0 billion Combined GAAP R&D,AIPR&D and SG&A expenses*Unchanged$5.55 to$5.7 billionCombined Non-GAAP R&D,AIPR&D and SG&A expenses*Unchanged$4.9 to$5.0 billionNon-GAAP effective tax rateUnchanged 20.5%to 21.5%*The difference

18、 between the combined GAAP R&D,AIPR&D and SG&A expenses and the combined non-GAAP R&D,AIPR&D and SG&A expensesguidance relates primarily to$650 million to$700 million of stock-based compensation expense.*Combined GAAP and Non-GAAP R&D,AIPR&D and SG&A expenses guidance includes approximately$100 mill

19、ion of AIPR&D expenses.Key Business HighlightsCystic Fibrosis(CF)PortfolioMarketed ProductsVertex has worked for more than 20 years to discover and develop medicines to treat the underlying cause of CF.Vertex CFTR modulators can treat95 percent of all people living with CF in markets with the highes

20、t prevalence and are approved for patients as young as one month old.ALYFTREK,the newest marketed CFTR modulator,is approved in the U.S.and the United Kingdom(U.K.)for the treatment of patients 6 years and older.Vertexanticipates the number of CF patients taking its medicines will continue to grow t

21、hrough new approvals and reimbursement,treatment of youngerpatients,increased survival,and expansion into additional geographies.Recent and anticipated progress includes:ALYFTREK,the once-daily,next-in-class combination CFTR modulator for the treatment of people with CF ages 6 yearsand older who hav

22、e at least one F508del mutation or another responsive mutation in the CFTR gene,is now approved inthe U.S.and the U.K.Vertex is working with the National Health Service(NHS)in the U.K.to secure coverage for eligiblepatients.In April,the European Medical Agencys(EMA s)Committee for Medicinal Products

23、 for Human Use(CHMP)adopted apositive opinion for ALYFTREK for the treatment of CF patients ages 6 years and older.European Commission approval isexpected in the second half of 2025.Regulatory reviews are underway for ALYFTREK in Canada,Switzerland,Australiaand New Zealand.Vertex secured European Co

24、mmission approval for the label expansion of KAFTRIO in combination with ivacaftor for CFpatients ages 2 years and older who have at least one non-class I mutation in the CFTR gene.With this approval,approximately 4,000 people in the European Union are newly eligible for a medicine that treats the u

25、nderlying cause oftheir disease.Work to ensure access for all eligible patients is underway.CASGEVY for the treatment of sickle cell disease(SCD)and transfusion-dependent beta thalassemia(TDT)CASGEVY is a non-viral,ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT th

26、at has been shown to reduce oreliminate vaso-occlusive crises(VOCs)for patients with SCD and transfusion requirements for patients with TDT.CASGEVY is approved in the U.S.,Great Britain,the EU,the Kingdom of Saudi Arabia(KSA),the Kingdom of Bahrain(Bahrain),Canada,Switzerland and the United Arab Emi

27、rates(UAE)for the treatment of both SCD and TDT.Recent highlights include:In Europe,Vertex secured a formal reimbursement agreement with NHS England for SCD patients to access CASGEVY,following the agreement reached in August 2024 for eligible TDT patients to access CASGEVY.Vertex recently enteredin

28、to a similar agreement in Wales for both eligible SCD and TDT patients.Following a positive assessment,Vertex alsofinalized national reimbursement in Austria.In the Middle East,following regulatory approval in the UAE,Vertex securedreimbursement in the majority of emirates.Since launch through May 1

29、st,Vertex has activated more than 65 authorized treatment centers(ATCs)globally andapproximately 90 patients have had their first cell collection.Vertex recently filed a manufacturing license submission with the FDA and expects to begin manufacturing CASGEVY inPortsmouth,NH,in the second half of 202

30、5.This submission is part of the planned ramp of CASGEVY manufacturingcapacity as demand for the therapy increases.JOURNAVX(suzetrigine)for the treatment of moderate-to-severe acute painJOURNAVX is a first-in-class,oral,selective,non-opioid NaV1.8 pain signal inhibitor,approved in the U.S.for the tr

31、eatment of moderate-to-severeacute pain.Vertex continues to advance a portfolio of selective NaV1.8 and NaV1.7 pain signal inhibitors for stand-alone use or in combination,withpotential to provide effective pain relief without the limitations of opioids and other available medicines.On January 30,20

32、25,the FDA approved JOURNAVX for the treatment of adults with moderate-to-severe acute pain,and itis now available and stocked at pharmacies across the U.S.,including major national and regional retail pharmacy chains.Since JOURNAVX became available in early March through April 18th,more than 20,000

33、 prescriptions have been writtenand filled across the hospital and retail settings in different acute pain conditions,consistent with JOURNAVXs broad label.As of May 1st,across commercial and government payers,approximately 94 million lives already have covered access toJOURNAVX,and approximately 42

34、 million have unrestricted access(i.e.,without the need for prior authorization or stepedits).Vertex has reached a formal coverage agreement with a large national Pharmacy Benefit Manager(PBM)to makeJOURNAVX available to their customers,representing approximately 22 million commercial lives.A total

35、of ten stateMedicaid plans are also providing unrestricted access to JOURNAVX and 20 more are currently evaluating their policies.More than 50 large healthcare systems have taken steps to initiate pharmacy and therapeutics(P&T)committee reviews ofJOURNAVX,and some have already added it on formularie

36、s.Vertex recently initiated two Phase 4 studies in various moderate-to-severe acute pain conditions to provide additional dataon the effectiveness and safety of JOURNAVX as part of real-world clinical practice,in both inpatient and outpatientsettings.The Alternatives to Pain Act has been reintroduce

37、d in both the House and Senate of the new U.S.Congress,while nearly35 U.S.states to date have passed or introduced new legislation in support of non-opioid options.Vertex also expectsJOURNAVX to be added to the list of treatments eligible for an add-on payment under the NOPAIN Act,which becameeffect

38、ive on January 1,2025.Select Clinical-Stage R&D PipelineCystic FibrosisVertex is enrolling and dosing studies in younger age groups a Phase 3 study of TRIKAFTA/KAFTRIO in 1-to2-year-olds and a Phase 3 study of ALYFTREK in 2-to 5-year-olds to expand the labels and enable earlier treatment ofchildren

39、with CF.Consistent with its commitment to serial innovation and bringing as many patients as possible to normal levels of CFTRfunction,Vertex continues to advance new oral small molecule combination therapies through preclinical and clinicaldevelopment.Vertex expects to advance the once-daily,next-g

40、eneration 3.0 VX-828 combination into a clinical trial inpeople with CF this year.Vertex has implemented a temporary pause to the multiple ascending dose(MAD)portion of the Phase 1/2 study ofVX-522,a nebulized CFTR mRNA therapy,in order to assess a tolerability issue.Sickle Cell Disease and Transfus

41、ion-Dependent Beta ThalassemiaVertex has completed enrollment of children 5 to 11 years of age with SCD or TDT in two global Phase 3 studies ofCASGEVY.Vertex continues to advance preclinical assets for gentler conditioning for CASGEVY,which could broaden the eligiblepatient population.Acute PainVert

42、ex expects to complete the Phase 2 study for an oral formulation of VX-993 this quarter.VX-993 is a next-generationselective NaV1.8 pain signal inhibitor,for the treatment of moderate-to-severe acute pain following bunionectomy surgery.Vertex expects to report results from the Phase 2 study in the s

43、econd half of 2025.VX-993 has Fast Track designation formoderate-to-severe acute pain.Vertex has completed the Phase 1 study of an intravenous formulation of VX-993 in healthy volunteers.The FDA hasgranted the intravenous formulation of VX-993 Fast Track designation for moderate-to-severe acute pain

44、.Peripheral Neuropathic Pain(PNP)Vertex continues to enroll and dose patients with diabetic peripheral neuropathy(DPN)in a Phase 3 pivotal trial ofsuzetrigine.The FDA has granted suzetrigine Fast Track designation in PNP and Breakthrough Therapy designation inDPN.Vertex continues to enroll and dose

45、patients in a Phase 2 study for the oral formulation of VX-993 for the treatment ofDPN.Type 1 Diabetes(T1D)Vertex is evaluating stem cell-derived,fully differentiated islet cell therapies for patients suffering from T1D,with the goal of developing a potentialone-time functional cure for this disease

46、.Zimislecel(VX-880),fully differentiated islet cells with standard immunosuppression:Vertex continues to enroll and dose patients in the Phase 3 portion of the Phase 1/2/3 study of zimislecel inpatients with T1D with severe hypoglycemic events and impaired awareness of hypoglycemia in the U.S.,Canad

47、a,U.K.,and EU.Vertex expects to complete enrollment and dosing of the pivotal study in the second quarter of 2025and submit marketing applications to global regulators in 2026.Zimislecel has been granted Regenerative Medicine Advanced Therapy(RMAT)and Fast Track designations fromthe U.S.Food and Dru

48、g Administration,Priority Medicines(PRIME)designation from the EMA,and has secured anInnovation Passport under the Innovative Licensing and Access Pathway(ILAP)from the UK Medicines andHealthcare products Regulatory Agency(MHRA).Vertex will present updated clinical data from the 12 participants who

49、received the full dose of zimislecel as a singleinfusion with over one year of follow-up in the Phase 1/2 portion of the study in an oral presentation at theAmerican Diabetes Association 85th Scientific Sessions.Vertex will host an in-person investor event at the ADAconference on Friday,June 20,2025

50、 at 7:15 p.m.CT/8:15 p.m.ET.The investor event will also be webcast live,and a link to the webcast can be accessed through Vertexs website at in the“Investors”section.Additional approaches:Vertex previously announced that it has discontinued development of the VX-264(“cells plus device”program),asth

51、e Phase 1/2 study did not meet its efficacy endpoint.Vertex is pursuing research-stage programs to evaluate additional approaches that could provide transformativebenefit to people with T1D and reduce or eliminate the need for standard immunosuppressive regimens.Theseapproaches include alternative i

52、mmunosuppressives,gene editing,and novel immunoprotection to encapsulate theislet cells.IgA Nephropathy(IgAN),Primary Membranous Nephropathy(pMN)and Other B Cell-Mediated DiseasesVertex is developing povetacicept,a dual antagonist of the BAFF and APRIL cytokines,which play key roles in the pathogene

53、sis of multiple Bcell-mediated diseases.Pove represents a potentially best-in-class approach to treat IgAN and pMN and has pipeline-in-a-product potential.The global Phase 3 RAINIER trial of povetacicept in patients with IgAN has completed enrollment of the interim analysiscohort.The interim analysi

54、s will be conducted once this cohort reaches 36 weeks of treatment,with the potential to file forAccelerated Approval in the U.S.in the first half of 2026,if results are supportive.Studies to support the launch ofpovetacicept for home administration are underway.Based on positive results of povetaci

55、cept in primary membranous nephropathy(pMN)in the RUBY-3 study,Vertex hasreached agreement with the FDA to advance povetacicept into pivotal development for this disease.Vertex will initiate asingle,adaptive Phase 2/3 trial of povetacicept vs.standard-of-care this year.In the Phase 2 portion,both 80

56、 mg and 240mg of povetacicept will be assessed and the selected dose will advance to Phase 3,where the primary endpoint ofcomplete clinical remission at 72 weeks of treatment will be evaluated.Vertex plans to present updated data from the IgAN and pMN cohorts of the RUBY-3 study at upcoming medicalc

57、ongresses.Vertex expects to share data and next steps from other RUBY-3 renal diseases and the hematologic conditions in theRUBY-4 study later this year.APOL1-Mediated Kidney Disease(AMKD)Vertex has discovered and advanced multiple oral,small molecule inhibitors of APOL1 function,pioneering a new cl

58、ass of medicines that targets theunderlying genetic driver of this kidney disease.Vertex expects to complete enrollment in the interim analysis cohort of the Phase 3 portion of the AMPLITUDE trial ofinaxaplin in the second half of 2025.Vertex will conduct the pre-planned interim analysis once this c

59、ohort has been treatedfor 48 weeks,with potential to file for accelerated approval in the U.S.if the results are supportive.Vertex continues to enroll and dose patients in the AMPLIFIED Phase 2 study of inaxaplin in people with AMKD anddiabetes or other co-morbidities.Myotonic Dystrophy Type 1(DM1)V

60、ertex is evaluating multiple approaches that target the underlying cause of DM1.Vertexs lead approach,VX-670,is an oligonucleotide linked to acyclic peptide,which holds the potential to address the underlying cause of DM1.Vertex continues to enroll and dose the MAD portion of the global Phase 1/2 cl

61、inical trial of VX-670 in people with DM1,which will assess both safety and efficacy.Autosomal Dominant Polycystic Kidney Disease(ADPKD)Vertex is developing small molecule correctors that restore function to the variant polycystin 1(PC1)protein,with the goal of addressing the underlyingcause of ADPK

62、D.Vertex has completed the Phase 1 study of VX-407 in healthy volunteers.Vertex expects to advance VX-407 into a Phase 2 proof-of-concept study this year in patients with a subset of variants inthe PKD1 gene,which encodes the PC1 protein,estimated to be up to 30,000(or 10%)of the overall patient pop

63、ulation.Non-GAAP Financial MeasuresIn this press release,Vertexs financial results and financial guidance are provided in accordance with accounting principles generally accepted in theUnited States(GAAP)and using certain non-GAAP financial measures.In particular,non-GAAP financial results and guida

64、nce exclude from Vertexspre-tax income(i)stock-based compensation expense,(ii)intangible asset amortization expense,(iii)gains or losses related to the fair value of thecompanys strategic investments,(iv)increases or decreases in the fair value of contingent consideration,(v)acquisition-related cost

65、s,(vi)anintangible asset impairment charge,and(vii)other adjustments.The companys non-GAAP financial results also exclude from its provision for incometaxes the estimated tax impact related to its non-GAAP adjustments to pre-tax income described above and certain discrete items.These resultsshould n

66、ot 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 financial measures help indicate underlying trends in the companys business,are important in comparingcurrent results with prior

67、period results and provide additional information regarding the companys financial position that the company believes ishelpful to an understanding of its ongoing business.Management also uses these non-GAAP financial measures to establish budgets and operationalgoals that are communicated internall

68、y and externally,to manage the companys business and to evaluate its performance.The companys calculationof non-GAAP financial measures likely differs from the calculations used by other companies.A reconciliation of the GAAP financial results tonon-GAAP financial results is included in the attached

69、 financial information.The company provides guidance regarding combined R&D,AIPR&D and SG&A expenses and effective tax rate on a non-GAAP basis.Unlessotherwise noted,the guidance regarding combined R&D,AIPR&D and SG&A expenses does not include estimates associated with any potentialfuture business d

70、evelopment transactions,including collaborations,asset acquisitions and/or licensing of third-party intellectual property rights.Thecompany does not provide guidance regarding its GAAP effective tax rate because it is unable to forecast with reasonable certainty the impact ofexcess tax benefits rela

71、ted to stock-based compensation and the possibility of certain discrete items,which could be material.Vertex Pharmaceuticals IncorporatedConsolidated Statements of Income(unaudited,in millions,except per share amounts)Three Months Ended March 31,2025 2024 Revenues:Product revenues,net$2,760.2$2,690.

72、6 Other revenues 10.0 Total revenues 2,770.2 2,690.6 Costs and expenses:Cost of sales 363.0 342.6 Research and development expenses 979.7 789.1 Acquired in-process research and development expenses 19.8 76.8 Selling,general and administrative expenses 396.4 342.7 Intangible asset impairment charge 3

73、79.0 Change in fair value of contingent consideration 2.2 (0.1)Total costs and expenses 2,140.1 1,551.1 Income from operations 630.1 1,139.5 Interest income 120.9 181.2 Interest expense(3.0)(10.4)Other expense,net(17.6)(31.2)Income before provision for income taxes 730.4 1,279.1 Provision for income

74、 taxes 84.1 179.5 Net income$646.3$1,099.6 Net income per common share:Basic$2.52$4.26 Diluted$2.49$4.21 Shares used in per share calculations:Basic 256.9 258.2 Diluted 259.5 261.1 Vertex Pharmaceuticals IncorporatedTotal Revenues(unaudited,in millions)Three Months Ended March 31,2025 2024TRIKAFTA/K

75、AFTRIO$2,535.5$2,483.6ALYFTREK 53.9 Other product revenues(1)170.8 207.0Product revenues,net 2,760.2 2,690.6Other revenues 10.0 Total revenues$2,770.2$2,690.6 1:In the first quarter of 2025,Other product revenues included$14.2 million from CASGEVY,and an insignificant amount from JOURNAVX.In thefirs

76、t quarter of 2024,there were no revenues for these products.The remaining“Other product revenues”are related to KALYDECO,ORKAMBI,andSYMDEKO/SYMKEVI,our other CF products.Vertex Pharmaceuticals IncorporatedReconciliation of GAAP to Non-GAAP Financial Information(unaudited,in millions,except percentag

77、es)Three Months Ended March 31,2025 2024 GAAP cost of sales$363.0$342.6 Stock-based compensation expense(2.6)(1.8)Intangible asset amortization expense(5.0)(5.0)Non-GAAP cost of sales$355.4$335.8 GAAP research and development expenses$979.7$789.1 Stock-based compensation expense(100.1)(119.4)Intangi

78、ble asset amortization expense(0.6)Non-GAAP research and development expenses$879.0$669.7 Acquired in-process research and development expenses$19.8$76.8 GAAP selling,general and administrative expenses$396.4$342.7 Stock-based compensation expense(63.4)(70.7)Non-GAAP selling,general and administrati

79、ve expenses$333.0$272.0 Combined non-GAAP R&D,AIPR&D and SG&A expenses$1,231.8$1,018.5 GAAP other expense,net$(17.6)$(31.2)Decrease in fair value of strategic investments 15.0 27.0 Non-GAAP other expense,net$(2.6)$(4.2)GAAP provision for income taxes$84.1$179.5 Tax adjustments(2)160.1 81.6 Non-GAAP

80、provision for income taxes$244.2$261.1 GAAP effective tax rate11.5%14.0%Non-GAAP effective tax rate18.8%17.4%Vertex Pharmaceuticals IncorporatedReconciliation of GAAP to Non-GAAP Financial Information(continued)(unaudited,in millions,except per share amounts)Three Months Ended March 31,2025 2024 GAA

81、P operating income$630.1$1,139.5 Stock-based compensation expense 166.1 191.9 Intangible asset impairment charge 379.0 Intangible asset amortization expense 5.6 5.0 Increase(decrease)in fair value of contingent consideration 2.2 (0.1)Non-GAAP operating income$1,183.0$1,336.3 GAAP net income$646.3$1,

82、099.6 Stock-based compensation expense 166.1 191.9 Intangible asset impairment charge 379.0 Intangible asset amortization expense 5.6 5.0 Decrease in fair value of strategic investments 15.0 27.0 Increase(decrease)in fair value of contingent consideration 2.2 (0.1)Total non-GAAP adjustments to pre-t

83、ax income 567.9 223.8 Tax adjustments(2)(160.1)(81.6)Non-GAAP net income$1,054.1$1,241.8 Net income per diluted common share:GAAP$2.49$4.21 Non-GAAP$4.06$4.76 Shares used in diluted per share calculations:GAAP and Non-GAAP 259.5 261.1 2:In the first quarter of 2025 and 2024,“Tax adjustments”included

84、 the estimated income taxes related to non-GAAP adjustments to the companyspre-tax income and excess tax benefits related to stock-based compensation.Vertex Pharmaceuticals IncorporatedCondensed Consolidated Balance Sheets(unaudited,in millions)March 31,2025 December 31,2024Assets Cash,cash equivale

85、nts and marketable securities$6,201.2$6,115.9Accounts receivable,net 1,805.1 1,609.4Inventories 1,359.7 1,205.4Prepaid expenses and other current assets 642.8 665.7Total current assets 10,008.8 9,596.4Property and equipment,net 1,295.9 1,227.8Goodwill and other intangible assets,net 1,529.2 1,913.9D

86、eferred tax assets 2,544.3 2,331.1Operating lease assets 1,338.5 1,356.8Long-term marketable securities 5,156.5 5,107.9Other long-term assets 1,007.3 999.3Total assets$22,880.5$22,533.2 Liabilities and Shareholders Equity Accounts payable and accrued expenses$3,396.8$3,201.6Other current liabilities

87、 386.4 363.0Total current liabilities 3,783.2 3,564.6Long-term operating lease liabilities 1,537.7 1,544.4Long-term finance lease liabilities 111.4 112.8Other long-term liabilities 951.9 901.8Shareholders equity 16,496.3 16,409.6Total liabilities and shareholders equity$22,880.5$22,533.2 Common shar

88、es outstanding 257.0 256.9About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.Thecompany has approved medicines that treat the underlying causes of multiple chronic,life-shortening genetic dise

89、ases cystic fibrosis,sickle celldisease and transfusion-dependent beta thalassemia and continues to advance clinical and research programs in these diseases.Vertex also has arobust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep

90、 insight into causal humanbiology,including acute and neuropathic pain,type 1 diabetes,APOL1-mediated kidney disease,IgA nephropathy,primary membranous nephropathy,autosomal dominant polycystic kidney disease,and myotonic dystrophy type 1.Vertex was founded in 1989 and has its global headquarters in

91、 Boston,with international headquarters in London.Additionally,the company hasresearch and development sites and commercial offices in North America,Europe,Australia,Latin America and the Middle East.Vertex is consistentlyrecognized as one of the industrys top places to work,including 14 consecutive

92、 years on Science magazines Top Employers list and one of Fortunes100 Best Companies to Work For.For company updates and to learn more about Vertexs history of innovation,visit or follow us onLinkedIn,Facebook,Instagram,YouTube and Twitter/X.Special Note Regarding Forward-Looking StatementsThis pres

93、s release contains forward-looking statements that are subject to risks,uncertainties and other factors.All statements other than statementsof historical fact are statements that could be deemed forward-looking statements,including all statements regarding the intent,belief,or currentexpectation of

94、Vertex and members of the Vertex senior management team.Forward-looking statements are not purely historical and may beaccompanied by words such as“anticipates,”“may,”“forecasts,”“expects,”“intends,”“plans,”“potentially,”“believes,”“seeks,”“estimates,”and otherwords and terms of similar meaning.Such

95、 statements include,without limitation,Dr.Kewalramanis statements in this press release,the informationprovided regarding future financial performance and operations,the section captioned“Full Year 2025 Financial Guidance”and statements regarding(i)expectations for Vertexs continued growth in CF,inc

96、luding by increasing the number of CF patients taking its medicines through new approvals andreimbursements,treatment of younger patients,increased survival,and expansion into additional geographies,(ii)expectations regarding securingcoverage for ALYFTREK in the U.K.,(iii)expectations regarding appr

97、oval of ALYFTREK in the E.U.in the second half of 2025,(iv)beliefs regardingthe anticipated benefits and commercial launch progress of CASGEVY,and with respect to increasing and initiating manufacturing activities forCASGEVY in the second half of 2025,(v)expectations regarding the potential benefits

98、 of JOURNAVX for the treatment of moderate-to-severe acutepain,including regarding the efficacy and safety of JOURNAVX,beliefs that JOURNAVX has potential to provide effective pain relief without thelimitations of opioids and other available medicines,expectations with respect to the commercial laun

99、ch progress and that commercial coverage ofJOURNAVX will continue to expand throughout 2025,and expectations that JOURNAVX will be included on the list of treatments that quality foradd-on payment under the NOPAIN Act,(vi)expectations to expand the labels for TRIKAFTA/KAFTRIO and ALYFTREK and enable

100、 earlier treatmentof children with CF,(vii)expectations to advance the VX-828 combination into people with CF this year,(viii)expectations for the VX-522 clinical trial,(ix)expectations regarding Vertexs SCD and TDT program,including with respect to gentler conditioning for CASGEVY broadening the el

101、igible patientpopulation,(x)plans with respect to the studies of the intravenous and oral formulation of VX-993 for the treatment of acute pain,includingexpectations to complete the Phase 2 study of the oral formulation of VX-993 in acute pain and expectations to report results from that study in th

102、esecond half of 2025,(xi)expectations regarding the pivotal study evaluating zimislecel in T1D,including the expectations to complete enrollment anddosing during the fiscal quarter and submit marketing applications to global regulators in 2026,expectations to present updated data in upcomingmedical

103、conferences,and expectations and plans to pursue additional approaches to standard immunosuppression regimens,(xii)expectations withrespect to povetacicept,including beliefs about its potential benefits and therapeutic scope,study designs,expectations regarding the Phase 3RAINIER study,including pla

104、ns to file for potential accelerated approval in the U.S.in the first half of 2026,beliefs with respect to the RUBY-3 basketstudy and plans to present updated data from this study at upcoming medical congresses,expectations and plans with respect to advancingpovetacicept into pivotal development in

105、pMN,beliefs with respect to the RUBY-4 basket study and expectations to share data and next steps later thisyear,(xiii)expectations regarding the AMPLITUDE trial in AMKD,including expectations to complete enrollment in the interim analysis cohort in thesecond half of 2025 and,assuming a positive int

106、erim analysis,apply for potential accelerated approval in the U.S.,(xiv)expectations for the potentialbenefits and clinical status of VX-670 for the treatment in people with DM1,and(xv)expectations regarding the ADPKD program,including thepotential benefits of VX-407 and expectations to advance VX-4

107、07 into a Phase 2 proof-of-concept study in 2025.While Vertex believes the forward-looking statements contained in this press release are accurate,these forward-looking statements represent the companys beliefs only as of the dateof this press release and there are a number of risks and uncertaintie

108、s that could cause actual events or results to differ materially from thoseexpressed or implied by such forward-looking statements.Those risks and uncertainties include,among other things,that the companys expectationsregarding its 2025 full year revenues,expenses and effective tax rates may be inco

109、rrect(including because one or more of the companysassumptions underlying its expectations may not be realized),that we may be unable to successfully commercialize ALYFTREK as a treatment for CFor JOURNAVX as a treatment for acute pain,that external factors may have different or more significant imp

110、acts on the companys business oroperations than the company currently expects,that data from preclinical testing or clinical trials,especially if based on a limited number of patients,may not be indicative of final results or available on anticipated timelines,that patient enrollment in the companys

111、 trials may be delayed,that thecompany may not realize the anticipated benefits from collaborations with third parties,that data from the companys development programs may notsupport registration or further development of its potential medicines in a timely manner,or at all,due to safety,efficacy or

112、 other reasons,and thatanticipated commercial launches may be delayed,if they occur at all.Forward-looking statements in this press release should be evaluated togetherwith the many uncertainties that affect Vertexs business,particularly those risks listed under the heading“Risk Factors”and the othe

113、r cautionaryfactors discussed in Vertexs periodic reports filed with the SEC,including Vertexs annual report on Form 10-K and its quarterly reports on Form 10-Qand current reports on Form 8-K,all of which are filed with the Securities and Exchange Commission(SEC)and available through the companysweb

114、site at and on the SECs website at www.sec.gov.You should not place undue reliance on these statements,or the scientific datapresented.Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.Conference Call and WebcastThe company

115、 will host a conference call and webcast at 4:30 p.m.ET.To access the call,please dial(833)630-2124(U.S.)or+1(412)317-0651(International)and reference the“Vertex Pharmaceuticals First Quarter 2025 Earnings Call.”The conference call will be webcast live and a link to the webcast can be accessed throu

116、gh Vertexs website at in the Investorssection.To ensure a timely connection,it is recommended that participants register at least 15 minutes prior to the scheduled webcast.An archivedwebcast will be available on the companys website.(VRTX-E)View source version on :https:/ Relations:Susie Lisa,CFA,617-341-6108Manisha Pai,617-961-1899Miroslava Minkova,617-341-6135Media:617-341-Source:Vertex Pharmaceuticals Incorporated