1、argenx The Next ChapterJ P M O R G A N H E A L T H C A R E C O N F E R E N C EJ A N U A R Y 8,2 0 2 41Forward Looking StatementsThis presentation has been prepared by argenx SE(“argenx”or the“company”)for informational purposes only and not for any other purpose.Nothing contained in this presentatio

2、n is,or should be construed as,a recommendation,promise or representation by the presenter or the company or any director,employee,agent,or adviser of the company.This presentation does not purport to be all-inclusive or to contain all of the information you may desire.Certain information contained

3、in this presentation relates to or is based on studies,publications,surveys and other data obtained from third-party sources and the companys own internal estimates and research.While argenx believes these third-party studies,publications,surveys and other data to be reliable as of the date of this

4、presentation,it has not independently verified,and makes no representation as to the adequacy,fairness,accuracy or completeness of,any information obtained from third-party sources.In addition,no independent source has evaluated the reasonableness or accuracy of argenxs internal estimates or researc

5、h and no reliance should be made on any information or statements made in this presentation relating to or based on such internal estimates and research.The financial results presented in this presentation are preliminary,estimated,and unaudited.They are subject to the completion and finalization of

6、 argenxs financial and accounting closing procedures.They reflect managements estimates based solely upon information available to management as of the date of this presentation.Further information learned during that completion and finalization may alter the final results.In addition,the preliminar

7、y estimates should not be viewed as a substitute for full quarterly and annual financial statements prepared in accordance with IFRS.There is a possibility that argenxs financial results for the quarter ended December 31,2023,and full year financial results for 2023 could vary materially from these

8、preliminary estimates.In addition to the completion of the financial closing procedures,factors that could cause actual results to differ from those described above are set forth below.Accordingly,you should not place undue reliance upon this preliminary information.Additional information regarding

9、the Companys fourth quarter 2023 financial results and full year financial results for 2023 will be available in the Companys annual report and Form 20-F,which will be filed with the Netherlands Authority for the Financial Markets and U.S.Securities and Exchange Commission(the“SEC”),respectively.The

10、se forward-looking statements can be identified by the use of forward-looking terminology,including the terms“plans,”“aims,”“believes,”“continues,”“hope,”“estimates,”“preliminary,”“anticipates,”“expects,”“intends,”“may,”“will,”“should,”or“commitment”and include statements argenx makes concerning its

11、 preliminary financial results for the full year 2023;its expansion efforts,including reaching more patients with VYVGART within the MG treatment paradigm,through geographic expansion and into new autoimmune indications,expanding into CIDP,and the anticipated development of empasiprubart and ARGX-11

12、9;the anticipated timing of its launch of SC efgartigimod for CIDP in the U.S.;the initiation,timing,progress and results of its anticipated clinical development,data readouts and regulatory milestones and plans;its strategic priorities,including the timing and outcome of regulatory filings and regu

13、latory approvals;its expectations of future profitability;the potential for innovation of its clinical programs;its pipeline;and the nomination of new development candidates.By their nature,forward-looking statements involve risks and uncertainties and readers are cautioned that any such forward-loo

14、king statements are not guarantees of future performance.argenxs actual results may differ materially from those predicted by the forward-looking statements as a result of various important factors,including but not limited to argenxs ability to successfully execute its business and growth strategie

15、s,the inherent uncertainties associated with development of novel drug therapies,preclinical and clinical trial and product development activities and regulatory approval requirements,the ability of our clinical trials to reach their endpoints,the ability to maintain,expand,and deliver on our pipeli

16、ne;the acceptance of our products and product candidates by our patients as safe,effective and cost-effective,volatile market conditions,and the impact of governmental laws and regulations on our business.A further list and description of these risks,uncertainties and other risks can be found in arg

17、enxs U.S.Securities and Exchange Commission(SEC)filings and reports,including in argenxs most recent annual report on Form 20-F filed with the SEC as well as subsequent filings and reports filed by argenx with the SEC.Given these uncertainties,the reader is advised not to place any undue reliance on

18、 such forward-looking statements.These forward-looking statements speak only as of the date of publication of this document.argenx undertakes no obligation to publicly update or revise the information in this presentation,including any forward-looking statements,except as may be required by law.This

19、 presentation contains trademarks,trade names and service marks of other companies,which are the property of their respective owners.2On a Journey to Transform AutoimmunityPioneering novel target biology Leading antibody engineering capabilitiesPipeline-in-a-product opportunitiesCreating optionality

20、 across and within molecules3We are Hearing Transformational Stories with VYVGART4Mike,VYVGART PatientPFS in development15 indications in development by 2025ITP MAA filedCIDP sBLA submitted$1.2B in gMG revenue in 2023ARGX-109(Anti-IL-6)ARGX-213(Anti-FcRn)ARGX-220ARGX-121EmpasiprubartPOC established

21、in MMNTrials in DGF and DMARGX-119Phase 1b/2a trials in CMS and ALSOur Innovation Horizons5PFS in development15 indications in development by 2025ITP MAA filedCIDP sBLA submitted$1.2B in gMG revenue in 2023ARGX-109(Anti-IL-6)ARGX-213(Anti-FcRn)ARGX-220ARGX-121EmpasiprubartPOC established in MMNTrial

22、s in DGF and DMARGX-119Phase 1b/2a trials in CMS and ALSVYVGART Opportunity Horizon6Leadership in FcRnPioneering FcRnUnique modulation of FcRn 15 indications by 2025*Fc fragment and proprietary ABDEG mutationsTransformational data in gMG and CIDPGenerating key learnings*Indications in development7Es

23、timated 4,000 patient years of safety follow-up between clinical trial and real-world experience45%MSE QoL comparable to healthy population*78%MG-ADL 4*Meaningful steroid tapering by at least 5mg/day within first 6 monthsSuperior cost/benefitover IVIg*Enables significantly faster access to treatment

24、VYVGART is Setting New Expectations in gMG*Real world evidence*Source:ADAPT and ADAPT+clinical trial data*Leading Health Technology Assessment agency825%YoY increase2,300*Prescribers in the USP R E S C R I B E R E X P A N S I O NStrong Commercial Execution21%2023 CAGR$1.2B*Global Product RevenueG R

25、O W T H2023 PerformanceFavorable payor policies90%Access VYVGART after 2 OralsB R O A D P A T I E N T A C C E S S55%patients from orals6,000*Global VYVGART PatientsE A R L I E R L I N E P A T I E N T S*Preliminary Financials.Unaudited and subject to change*As of Q3 2023 Financial Results9Innovation

26、Builds Autoimmune Market Opportunities1017,0006,000*Growing MG MarketgMG Patients on VYVGARTCurrent VYVGART TAMExpanding VYVGART TAMGrowing VYVGART ShareUSRoW argenx markets(exc.China)Global31,000Label-enabling trials in broader gMG populationsPhase 3b studies and externally sponsored researchGeogra

27、phic expansion,including South Korea and AustraliaGrowing VYVGART shareUS:VYVGART Hytrulo J-Code in effect;field force expansionPFS developmentAddition to China NRDLExpanding VYVGART TAMGrowing MG marketTargeted biologics are expanding gMG market by providing patients more treatment optionsBased on

28、argenx market research*As of Q3 2023 Financial ResultsVYVGART Has Potential to Transform CIDP 1167%Response rate demonstrates IgG autoantibodies play significant role in underlying CIDP biologyHR:0.39P=0.00003999%Rollover of eligible patients to open-label extensionFavorable safety and tolerability

29、profile consistent with previous clinical trialsStage BsBLA submitted with priority review voucherSET NEW STANDARD FOR HOW CIDP TRIALS ARE RUNSIGNIFICANT IMPACT ON CIDP PATIENTS99%Study ComplianceStage AESTABLISHED CIDP AS IgG MEDIATED61%reduced risk of relapse 11CONFIDENTIAL FOR INTERNAL USE ONLY.2

30、023 argenx.We Aim to Address the UnseenSuffering in CIDP20%of patients achieve remission on current SOC(CDAS=2)*50%of patients are dissatisfied with their symptom burden*42K treated CIDP patients in US&ROW argenx markets(ex-China)*Gorson KC,et al.2010*Mendoza M,et al.2023*argenx market research1212T

31、ransforming the Patient Treatment Experience13Pre-filled SyringeAutoinjectorSingle 30-90s injection HCP administeredIncreasingly convenient delivery Preparing for self-administrationApproved June 2023Ongoing in clinical trialsIndustrialization phaseExclusive FcRn license to ENHANZEHigh concentration

32、 formulation with low viscosity,no back pressureThis isJust the Beginning 14Generalized MyastheniaGravisChronic InflammatoryDemyelinating PolyneuropathyExpected 2024 PoC study readoutsITPLaunched2022Anticipatedapproval 2024Japan submissionin reviewTED100KIMNM6KSjogrens330KPC-POTS500KBeyond 2024 PoC

33、studiesBP52KLN40KMN80KAAV105KAMR8KPotential 2024 Phase 3 StartPhase 2 Proof of Concept65K24K17K2024 Phase 3 StartASyS11KDM70K#14#15*argenx market research;US prevalence numbers(except Japan ITP)Phase 2 Readouts Present Significant Commercial Opportunities15 Anti-Ro/Anti-La AutoAbs Passive transfer m

34、odel evidence IgG reduction associated with improvementRCT-Phase 2CRESS/ESSDAISteroids/NSISTsCholinergic agonists Artificial tears Anti-adrenergic receptor AutoAbs IVIG/PLEX effectiveRCT-Phase 2MaPS/COMPASSNo approved therapies Myositis AutoAbs Passive transfer model evidence(IMNM)AutoAb titer corre

35、lates with disease activityRCT-P2/P3TISBIOLOGICRATIONALESteroidsIVIg330K500K6K IMNM11K ASyS70K DMSjogrens SyndromePC-POTSMyositis(IMNM,ASyS,DM)CLINICALFEASIBILITYU.S.COMMERICALOPPORTUNITYPFS in development15 indications in development by 2025ITP MAA filedCIDP sBLA submitted$1.2B in gMG revenue in 20

36、23ARGX-109(Anti-IL-6)ARGX-213(Anti-FcRn)ARGX-220ARGX-121EmpasiprubartPOC established in MMNTrials in DGF and DMARGX-119Phase 1b/2a trials in CMS and ALSPipeline Horizon16Rewriting Immunology Textbook with EmpasiprubartPioneering complement factor C2Unique sweeping antibodyOngoing development in 3 in

37、dications80-day half-life supports favorable dosingPOC established in MMNDefining MMN as auto-IgM mediated disease17Empasiprubart has Potential to Transform MMN18HR(95%CI)=0.09(0.02;0.44)94%of treated patients rated their condition improved since starting therapy,including 55%who were much/very much

38、 improved 8/9 placebo patients had no change or worsened(Patient Global Impression of Change scale)Empasiprubart demonstrated improvement compared to baseline on 6/6 efficacy measurementsSafety profile consistent with Phase 1 data91%reduction in need for IVIg rescue with empasiprubartCohort 2 is ong

39、oing;results to inform dose for Phase 3 study initiation18MMN Patients are WaitingClear opportunity for empasiprubartIm not asking to be able to run and jump like I used to.I just want to be able to stand like I used to.“”IVIg only treatment optionPatient journey characterized by deep frustration an

40、d anxietyto transform MMN outcomesA D D R E S S A B L E M A R K E T10k patientsUS+argenx ROW markets(ex China)*argenx market research;Arnold et al 2013;Park et al 2022 19ARGX-119:Enhancing Neuromuscular Junction Pioneering MuSK biology at NMJInitial development in CMS and ALSAgonistic SIMPLE Antibod

41、ySafety and tolerability data from extensive Phase 1 study support advancement into PoC studies20CMS and ALS Trials to Start in 202421ARGX-119 rescues early neonatal lethality and disease relapse in adult DOK7 CMS miceNature,Oury et al.2021ARGX-119 preserves NMJ numbers and restores muscle contracti

42、on in ALS patient derived NMJs on-a-chipFidelity(%)Biomaterials,Badu-Mensah et al.2022;Advanced Therapeutics,Guo et al.2020501000Frequency(Hz)ALSALS+ARGX-119Healthyargenx internal datamAbmAbPFS in development15 indications in development by 2025ITP MAA filedCIDP sBLA submitted$1.2B in gMG revenue in

43、 2023ARGX-109(Anti-IL-6)ARGX-213(Anti-FcRn)ARGX-220ARGX-121EmpasiprubartPOC established in MMNTrials in DGF and DMARGX-119Phase 1b/2a trials in CMS and ALSImmunology Innovation Horizon22ARGX-109(Anti-IL-6)Pipeline Growth Driven By Immunology Innovation Program23ARGX-213ARGX-220DISEASEINSIGHTANTIBODY

44、ENGINEERINGCLINICAL DEVELOPMENTContinued Leadership in FcRnBroad Immune System TargetsARGX-121First in class,novel targets4 I N D F I L I N G S B Y E N D O F 2 0 2 5Strong Track Record with Repeatable Innovation PlaybookLEADING TRANSLATIONAL BIOLOGY LABSTECHNOLOGYKNOW-HOWVYVGARTgMGDecision on approv

45、al:Switzerland,Australia,Saudi Arabia,South KoreaBy Year EndSeronegative trial initiationBy Year EndITPJapan decision on approval1Q 2024VYVGART SCgMGJapan decision on approvalBy 1Q 2024China decision on approval(Zai Lab)By Year EndCIDPU.S.launch,if approvedMid-2024Regulatory submissions Japan,Europe

46、,China,CanadaBy Year EndMG,CIDPUpdate on PFS development1H 2024EfgartigimodPrimary Sjogrens syndromeProof of concept data1H 2024PC-POTSProof of concept data1H 2024MyositisProof of concept data2H 2024EmpasiprubartMMNFull Phase 2 data2024ARGX-119CMS,ALSPhase 1b/2a study initiations2024IIPNot Disclosed

47、4 INDs filedBy End of 202524IndicationMilestoneTimingStrong Cadence of Milestones in 2024Q4 2023 Product Net Sales of$374 million282693293742022Q12022Q22022Q32022Q42023Q12023Q22023Q32023Q4*($B)2023Product Net Sales(1)1.2Cash,cash equivalents and current financial assets(1)3.22024 Financia

48、l Guidance(1)-Preliminary Financials.Unaudited and subject to change.($B)2024Cash burn(2)0.5Combined R&D and SG&A expenses 2.0*Preliminary Financials.Unaudited and subject to changePreliminary 2023 Financial Results(2)-Cash burn is equal to the decrease in our cash,cash equivalents and current financial assetsOn Track To Be Sustainable2023 CAGR:21%256Leading to multiple2024 Strategic PrioritiesCommitted to Driving Continued Growth26Broaden leadership in MG marketLaunch CIDPAdvance PFSPhase 2 data readoutsPhase 3 initiationsINDs by 20254THANK YOU27