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0Argenx 2023 7 17|S0120521080001 1 100 5%30 Efgartigimod 70 efgartigimod a pipeline in a drug PD-1 TNF-a efgar Keytruda 2023 efgar 4 12 4 PC-POTS Benchmark efgar SOC+IVIg+RTX off-label Argenx biotech Vertex/18.8 vs.25.3 Argenx 5.2 01 03.1 VYVGART(efgar)-gMG02 03.2 efgar 03 03.2.1 ITP04 03.2.2 CIDP05 03.2.3 PPG03.2.4 PC-POTS03.3 efgar 2024 03.3.1 IIM03.3.2 BP03.3.3 SS03.3.4 MN03.3.5 TED03.3.6 AAC03.3.7 LN03.4 03.5 ARGX-1173 014 Argenx 2008 2021 VYVGART(efgartigimod)2022 4 efgar 2014 FcRn FIC Biotech A pipeline in a drug efgar 13 PD-1 FIC efgar ARGX-117 ARGX-119 FIC a pipeline in a drug 2008 SIMPLE ANTIBODY 2010 IL-6 Gerilimzumab 2011 Eli Lilly ARGX-110 CD70 2012 Shire NHance ARGX-111 c-Met 2013 ARGX-110 1 ARGX-112 IL22R1 ARGX-111 1 2014 ARGX-113 efgar Bayer Euronext Brussels 4000 2015 ARGX-115 GARP efgar 1 2016 Abbvie ARGX-113 ARGX-110 1/2 2017 efgar 2 MGefgar 2 ITP Abbvie 1000 ARGX-110 2 R/R CTCL NASDAQ 1 efgar MGefgar 2 PV efgar SC 1 efgar MG 2 2.66 2018 ARGX-117 C2 efgar 2 PV efgar MG 3 efgar 2 ITP POC 3 ASH cusatuzumab 1/2 ORR 92%cusatuzumab Cilag(JNJ)3 13 2019 Halozyme Abbvie 3000 ARGX-115 ARGX-118 Galectin-10 Global offering 4.84 2020 efigartigimod 3 MG 7.5 9800 Bayer PRV 2021 efgar efgar CIDP 10 JNJ cusatuzumab VYVGART(efgar)2022 efgar SC 3 MG 7 efgar 3 ITP VYVGART(efgar)Bluebird Bio 1.02 PRV2023 FDA efgar PDUFA 2023/6/20 Genmab Genmab Argenx5 efgar 2 ASHcusatuzumab(ARGX-110)1/2ORR 92%efgar 3 MG ADAPT efgar 3 CIDP(efgar)VYVGART efgar SC PDUFA 6 1688%2017 Nasdaq IPO 1 efgar gMG 3 2020 efgar 3 3 gMG 2021 VYVGART(efgar)2023 3 2022()4 Nasdaq 2023/6/12 1688%2022 VYVGART 4 Argenx Argenx wind6 2022 VYVGART 4 2023Q1 2.18 26%4 CAGR 10%2020 efgar 3 1 PRV 81%2 PRV 2022 4 Argenx wind7 2023Q1 VYVGART 90%23%22.5(2022/8)2023Q1 1000 83%VYVGART 42/1/2 gMG 2.9 2023Q1 16%2023Q1 23%VYVGART gMG Argenx8/Argenx biotech Vertex/18.8 vs.25.3 Argenx Vertex Trikafta CF efgar efgar 3 Argenx Argenx(25%)(CRO 55%)(50%)(38%)Vertex 6/29 Vertex 18.8 903 4800Argenx 25.3 213 843 2022 Argenx Argenx Vertex Vertex(2022 89,121,273,263,193,102)Argenx SeekingAlpha(6/28)wind Argenx(2022 4.1,36.3,10.4,14.2,21,185 9 02 10 100 30 Off-label/IVIg RTX off-label CNI IVIg IVIg 100($)TNF-aHumira Adalimumab 212.37 2022 RA PA PP AS UC CD HSEnbrel etanercept 59.65 2016 RA PA PPRemicade infliximab 70 2016 RA PA PP AS UC CDSimponi golimumab 22.76 2021 RA PA AS UCIL-17Cosentyx secukinumab 47.88 2022 RA PA PP AS nr-axSpATaltz ixekizumab 24.82 2022 PA PP AS nr-axSpAIL-12/23Tremfya guselkumab 22.68 2022 PA PPSkyrizi risankizumab 51.65 2022 PA PP UC CDStelara ustekinumab 97.23 2022 PA PP UC CDIL-4/13 Dupixent dupilumab 82.93 2022 AD ASM NP EE PN COPDPDE4 Otezla Apremilast 22.88 2022 PA PP OUIL-6 Actemra tocilizumab 35.62 2021 RA GCA CRS IDL COVIDJAKRinvoq upadacitinib 25.22 2022 RA PA AS UC nr-axSpA CD ADOlumiant baricitinib 11.15 2021 RA COVID AAXeljanz tofacitinib 24.55 2021 RA PA AS UCCD20Ocrevus ocrelizumab 60.36 2022 MSKesimpta ofatumumab 10.92 2022 MS 882.67/RTX off-label IVIg Argenx FDA HealthAdvances11 0312 1 efgar gMG 12 2 FIC ARGX-117 ARGX-119 13 Argenx Argenx13 10 2023H2 10 1 efgar 2 ADDRESS ADVANCE 3 PC-POTS 4 ARGX-117 Argenx 2023 Argenx14 03.115 9 AAb 1)2)3)4)5)6)7)8)9)AAb JAK VYVGART AAb AAb 1.6 AAb 100 5%40%(2%)1.6 AAb neuropathyandhiv16 AAb AAb IgM IgG FcRn IgG IgG 4 FcRn IgG AAb 1 MG 2 Argenx VYVGART CNI RTX IVIg FcRn IgG AAb AAb IgG Pyzik,M.et al.The therapeutic age of the neonatal Fc receptor.Arthritisresearch17 efgar FcRn AAb PD-1 TNF-a 10 Argenx MG ITP Argenx 13 140 20 FcRn IgG efgar PD-1 IgG FcRn efgar IgG Argenx Pyzik,M.et al.The therapeutic age of the neonatal Fc receptor.18 MG 12/10 96 7 85%AAb AAb AchR VYVGART MG CNI C5 Soliris Ultomiris DMT MG Acetylcholinesterase AchR Mestinon(pyridostigmine)C5 AchR Soliris(eculizumab)Ultomiris(ravulizumab)autobody AchR CD20/CD19 B RTX(RTX)off-label CNI prednisone AAb IVIg-MG gMG gMG Myastheniagravisnews Jobilize19 3 ADAPT efgar C5 AchR+efgar(67.7%vs 29.7%p0.0001)C5 ravulizumab efgar MG-ADL 6 40%ravulizumab 23%efgar(AEs 77%vs 84%)MG 3 efgar 3 ADAPT efgar 3 ADAPT Ravulizumab 3(AD Tuan Vu,M.D.et al.Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis20 VYVYGART C5 FcRn VYVGART 23 Ultomiris 46 C5 Soliris 41 Ultomiris MG VYVGART 2023/6/20 VYVGART UCB UCB 2023 1 FDA rozanolixizumab gMG 6/27 RYSTIGGO nipocalimab 3 FcRn C5 MG CD20 CD19 CD38 IL-6 BAFF BTK 2022 GSK CSL BioMarin Curavac Regeneron Alnylam MG R-CAR-T Cartesian Therapeutics mRNA CAR-T 1/2 VYVGART Argenx efgar efgar ravulizumab zilucoplan rozanolixizumab nipocalimab batoclimab FcRn C5 C5 FcRn FcRn FcRn Argenx Astrazeneca UCB UCB Immunovant 3 3 3 3 Min2 MG-ADL 77.8%vs 48.3%63.9%vs 53%79%vs 67%-57%vs.33%30%11%12%-24%Min3 MG-ADL 73%vs 36.7%56.7%vs 34.1%78%vs 53%72%vs 31%52%vs 15%(2)-36%23%25%41%37%-A77%vs 84%91%vs 87%77%vs 70%8183%vs 67%SAE SAE MG(AD Tuan Vu,M.D.et al.Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis FDA 21 VYVGART 2022 VYVGART 2021 1 7500 1.75 VYVGART 15%25%(mid-teens to low-twenties)/6/30 2023 2021 VYVGART gMG 14 Medison 2022 Genpharm VYVGART Gulf 2022 4()94%2023/6/20 VYVGART Hytrulo Hytrulo HALOZYME HALO ENHANCE HALO/UCB 6/27 rozanolixizumab-noli)HALOZYME IP RYSTIGGO 6050/6 7.3 Hytrulo 15773/4 6.3 Hytrulo VYVGART 2023 VYVGART Argenx22 03.2efgar23 03.2.1ITP24 AAb ITP(210)/10 5%16 160 5.7 ITP AAb SOC CNI RTX ITP ITP 1 prednisone Ashwell-Morell 2 CD20 RTX off-label2 SYK fostamatinib 1843%2 rhTPO Eltrombopagavatrombopag 40%60%3 B T azathioprine 3060%3 FcRn AAb IVIg-ITP SOC ITP Newland,A.C.et al.Phase 2 study of efgartigimod,a novel FcRn antagonist,in adult patients with primary immune thrombocytopenia.Argenx Nichola Cooper,M.D.et al.Immune Thrombocytopenia25 CNI C1 B BLyS CD40L BTK FcRn ITP AAb 3 ITP 3 FcRn efgar rozanolixizumab BTK rilzabrutinib 3 2 53.8%54.5%40%()2 efgar ITP efgar 2 ITP Nichola Cooper,M.D.et al.Immune Thrombocytopenia26 2 50 109/L 46.2%25%33%50 25%efgar(5 mg/kg)46.2%64 7.7%Rozanolixizumab 2 20mg/kg 54.5%efgar Efgar(ADVANCE)Rozanolixizumab(myOpportunITy3)3 2022 2 12 ADVANCE 2023 2 7 BTKi Rilzabrutinib 2 40%BTK efgar ITP 2023 3 efgar 2 ITP Rozanlixizumab 2 ITP Rilzabrutinib 2 ITP Newland,A.C.et al.Phase 2 study of efgartigimod,a novel FcRn antagonist,in adult patients with primary immune thrombocytopenia.Argenx Nichola Cooper,M.D.et al.Immune Thrombocytopenia27 03.2.2CIDP28 CIDP AAb myelin sheath CIDP IgG4 AAb nodal paranodal 10%(118%)CIDP 0.6710.3 10 1.6 CIDP SOC IVIg 85%IVIg 30 CNI IgG4 IVIg RTX B CD52 alemtuzumab a4 integrin natalizumab CIDP AAb CIDP Carina Bunschoten,MD et al,Progress in diagnosis and treatment of chronic inflammatory demyelinating polyradiculoneuropathy Kieseier,B.C.,Mathey,E.K.,Sommer,C.et al.Immune-mediated neuropathies.29 IVIg CIDP Grand View Research 2023 IVIg 60 CAGR 68%CIDP 24%IVIg 10000 4.3 4.2 13 IVIg VYVGART 22.5 IVIg CIDP IVIg IVIg IVIg IVIg CIDP CIDP IVIg IVIg CIDP Grand View Research Pubmed Argenx IVIg30 2 ADHERE(NCT04281472)2023Q3 360 UCB 2020 rozanolixizumab CIDP CIDP AAb 30%nipocalimab 2/3 CIDP 2022 2026CIDP efgar Adhere Adhere Argenx Argenx 2021 Argenx 30 efgar CIDP POC 31 03.2.3PPG32 PPG desmoglein AAb 10%3 1)PV 2)PF 3)PP PP T B 5.2/10 1.9 SOC RTX CNI IVIg RTX RTX(2018)59100%CR 4081%B PPG PPG Kasperkiewicz,M.et al.Pemphigus.Nat Rev Dis Primers 3,17026(2017).33 efgar 2 efgar PPG(3 AE)31 28(90%)17 DC 64%92(13)CR RTX 60%CR 290 efgar AAb 50 RTX 180PPG efgar PPG efgar 2 Argenx BJD Pascal J et al First-line rituximab combined with short-term prednisone versus prednisone alone for the treatment of pemphigus(Ritux 3)RTX PPG 3 RTX efgar 90%prednisone 6000mg prednisone E-cadherin desmoglein prednisone 2023H2 ADDRESS34 NME B BTK CD20(RTX)CD20 FcRn IgG FcRn UCB(rozanolixizumab)(nipocalimab)Alexion 2500 Affibody AB FcRn ABY-039 PPG FcRn PPG PPG NCBI35 03.2.4PC-POTS36 12 10%6500 5070%1030%1012%PC()()()ME/CFS 200/1 2 3 4 5 6 PC-POTS POTS Davis,H.E.et al.Long COVID:major findings,mechanisms and recommendations.Fatema-Zahra El-Rhermoul et al.Autoimmunity in Long Covid and POTS 37 PC POTS PC 67%10 30 120bpm POTS SOC beta Cholinergic AAb:POTS PC AAb GPCR muscarinic acetylcholine-adrenoceptor adrenergic antinuclear Sage Journals POTS Nature AAb AAb PC-POTS AAb POTS POTS PC-POTS drvikram:How To Manage POTS Naturally?Mallick,Deobrat et al.COVID-19 Induced POTS:A Review.Davis,H.E.et al.Long COVID:major findings,mechanisms and recommendations.38 POTS AAb efgar Berlin Cures BC007 anti-GPCR AAb efgar 2 POC 42 2022 2023 11 Berlin Cures 2014 BC007 2023Q2 2 2024Q1 FcRn FcRn AAb POTS AAb GPCR FcRn IVIg POTS PC-POTS 50 POC efgar Argenx price in POTS POC PC-POTS IVIg PC-POTS BurlinCures Frontiers BurlinCures 39 03.3efgar202440 03.3.1IIM41 2.934/10 6.3 60%IIM MSA MSA 2021 Nature 20 2030%IIM AAb SOC CNI IVIg RTX(off-label)JAK IL-6 IVIg Rheumatology(Oxford)2020 IVIg IIM IVIg IIM 23L 1L IIM IVIg efgar IIM IIM AAb IIM Lundberg,I.E.et al.Idiopathic inflammatory myopathies.Lim,Johan et al.Intravenous immunoglobulins as first-line treatment in idiopathic inflammatory myopathies:a pilot study.creakyjoints IIM 10 20%90%42 IIM RTX TNF-a etanercept infliximab BMS Orencia(abatacept CD80/86)Corbus lenabasum(CB2)FcRn NME FcRn nipocalimab efgatigimod efgar efgar 2/3 ALKIVIA(NCT05523167)240 2026 2024 Asys IMNM DM 3 MSA MSA+3.3 Priovant Brepocitinib TYK2 DM 3 NCT05437263 2024/12Alexion Ravulizumab C5 DM 2/3 NCT04999020 2023/5Argenx efgar FcRn IIM 2/3 NCT05523167 2026/12Galapagos GLPG3667 TYK2 DM 2 NCT05695950 2025/3Kezar ZetomipzomibImmuno-proteasomeDM PM 2 NCT04628936 2023/3Janssen Nipocalimab FcRn IIM 2 NCT05379634 2026/3Horizon Daxdilimab ILT7 DM ASIM 2 NCT05669014 2026/10IIM IIM IIM AAb Argenx clinicaltrials.gov IVIg IIM43 03.3.2BP44 2.4%BP 2.423/80 190312/5.2 PD-(L)1 BP hemidesmosome BPAG(BP180,BP230)SOC CNI IVIg/CR 60100%off-lable RTX RTX 6070%RTX+IVIg 6 FcRn IVIg+CD20 FcRn IVIg CD20 IVIg+CD20 efgar BP PPG BP 45 efgar 2 POC 2022 BP 2/3 BALLAD(NCT05267600)160 2024H1 efgar FcRn BP 1 sutimlimab 2/dupilumab ixekizumab IL-5 mepilizumab 2 Benralizumab 3 FJORD(NCT04612790)2024Q2 Dupilumab 2/3(LIBERTY-BP NCT04206553)2024Q3 BP efgar BP BP Argenx P.Zeng et al.Bullous pemphigoid What do we know about the most recent therapies?46 03.3.3SS47(5%)61/10 40 33 4001000 SS T B AAb(RNA)SOC CNI SS SS SS Brito-Xavier Mariette et al.s Syndrome48 SS SS BTK FcRn CD40 RTX RTX SS IVIg SS efgar Novartis ianalumab BAFF-R 3-SLE ITPMedImmune anifrolumab IFNAR-1 2 2021 Saphnelo SLEGilead tirabrutinib BTK 2 2020(JP)Velexbru Novartis remibrutinib BTK 2-CSUEli Lilly venanprubart BTLA 2-Novartis iscalimab CD40 2-SLE,T1DAbbVie ravagalimab CD40 2-UCHorizon dazodalibep CD40L 2-FSGSanofi frexalimab CD40L 2-MSArgenx efgar FcRn 2 2021 Vyvgart gMG ITPJNJ nipocalimab FcRn 2-RA HDFNNovartis leniolisib PI3K 2 2023 Joenja ADPSResolve RSLV-132 RNA Rnase 2-SLE LCGilead GLPG3970 SIK2/3 2-UC RAGilead lanraplenib Syk 2-AML CLEMedImmune daxdilimab LILRA4 1-SLE AABMS BMS-986325 FPR2 1-SS SS SS clinicaltrial.gov Dominic Ridgewell et al,49 03.3.4MN50/80%20%1.2/10 8 MN 30%1/3 1/3 IC(IgG)(GBM)GBM 80%PLA2R AAb IgG THSD7A NELL1 EXT1/2 SOC CHB CNI CTX RTX ACEi 30%RTX 66%60%12 PLA2R RTX CTX(58%vs 84%)CHB CNI 40%CNI 30%60%PLA2R MN MN/MN Ronco,P.et al.Membranous nephropathy.51 PLA2R RTX MN AAb B Obinutuzumab(CD20 RTX)Belimumab(BLys)60%CD38 daratumumab felzartamab PLA2R C5 B D 2024 MN 2024 MN MN Ronco,P.et al.Membranous nephropathy.52 03.3.5TED53 1.9/10000 10 TED 20%TED TSHR AAb orbital fibroblast THSR fibroblast TSHR cross-talk IGF1R TSHR AAb SOC Horizon Tepezza(2020)TED Tepezza TED TED TED Horizon Amgen 54 SOC TEPEZZA(teprotumumab IGF1R 2020)TED 2022 15 Teprotumumab 3:24 83%3.32mm 10%0.53mm teprotumumab Efgar Tepezza IGF1R FcRn THSR IGF1R AAb Efgar 2023Q4 2025 TED efgar Tepezza TEPEZZA teprotumumab efgar Horizon James F Howard Jr et al.Safety,efficacy,and tolerability of efgartigimod in patients with generalised myasthenia gravis(ADAPT)55 03.3.6AAV56(ANCA)(AAV)AAV 6 60%3042.1/10 10.5 510 ANCA(MPO PR3)ANCA SOC 2021 Tavneos CTX(GBM)FcRn RTX CTX CTX SOC 1)11%16%2)3)Efgar POC 2023Q4 2025 AAV efgar 2025 ANCA ANCA Amgen2022 Holers,V.M.Complement therapeutics are coming of age in rheumatology.SA202057 Tavneos(avacopan C5aR/CD88 2021)Chemocentryx FIC AAV 2022Amgen 40 Chemocentryx Tavneo 2023Q1 2300 QoQ 10%Avacopan 3 RTX CTX avacopan 26 72.3%vs 70.1%52 65.7%vs 54.9%avacopan SAE 37.3%vs 39%10%CTX FcRn(complement cascade classical pathway)IgG IgG FcRn Efgar avacopan efgar AAb CD20 AAV efgar Tavneos efgar efgar AAV AAV avacopan efgar Avacopan Avacopan Holers,V.M.Complement therapeutics are coming of age in rheumatology.David R.W.Jayne et al.Avacopan for the Treatment of ANCA-Associated Vasculitis58 03.3.7LN59 LN SLE SLE 4.8%LN 9%SLE 8180/10 241/10 5 LN 2/3 4 LN 3 1 DNA AAb 2 T(Th)B 3 DNA()SLE GBM SOC LN CNI RTX IVIg CD20 LN SOC 10 5%30%10 ESKD LN SOC 10 LN LN Anders,HJ.et al.Lupus nephritis.Mok,C.C.et al.Treatment of lupus nephritis:consensus,evidence and perspectives.60 5 1 CD20 RTX obinotuzumab 2 SLE anifrolumab 3 4 5 AAb 6 2023 Mok,C.C.Nature 50 15(CD20 CD40 IL12/23 JAK)FcRn nipocalimab 2 Efgar 2 2025 POC Efgar Argenx MNC Stelara Tremfya RA(2017 136)SLE(2018 20)biotech Argenx/niche LN 5 LN LN Anders,HJ.,Saxena,R.,Zhao,Mh.et al.Lupus nephritis.Mok,C.C.et al.Treatment of lupus nephritis:consensus,evidence and perspectives.61 03.462 efgar 13 ronzanolixizumab 5 nipocalimab 10 batoclimab 5 4 FcRn vs B FcRn AAb B CD19 CD20 BTK FcRn vs C5 MG FcRn C5 Efgar vs FcRn FcRn Argenx rozanolixizumab UCB EudraCT2019-000969-21 gMG 3 NCT04596995 ITP 3 EudraCT2021-000352-19 MOGAD 3 NCT04875975 AE 2 NCT05643794 FM 2 nipocalimab J&J NCT04951622 gMG 3 NCT05912517 HDN 3 EudraCT2019-000720-17 AIHA 3/2 jRCT2041220037 CIDP 3/2 EudraCT2020-005568-79 LN 2 jRCT2041210058 SLE 2 EudraCT2021-000510-42 RA 2 NCT04883619 CLE 2 EudraCT2021-005202-98 IIM 2 EudraCT2021-000665-32 SS 2 batoclimab Immunovant EudraCT2022-002839-66 TED 3-NCT05039190 gMG 3 NCT04428255 ITP 3/2 NCT05581199 CIDP 2 NCT04227470 NMO 1 FcRn FcRn Efgar efgar FcRn efgar LDL FcRn63 03.5ARGX-11764 100 6(C1-INH Pegcetacoplan,Sutimlimab,Eculizumab,Ravulizumab,Avacopan pozelimab 2023/8/20)Holers,V.M.Complement therapeutics are coming of age in rheumatology.AJMC 65 Argenx 3 C5 C5 C1 C3 B D Argenx C2 C2 C2 C5 eculizumab C2 classic lectin alternative C2 C5 C1 C4 ARGX-117 efgar IgG efgar ARGX-117 C2 ARGX-117 classical lectin C2 Argenx 66 MG ARGX-117 MMN ARGX-117 MG MMN Ricklin,D.,Mastellos,D.C.&Lambris,J.D.Therapeutic targeting of the complement system.Argenx2023 MMN ARGX-117 12/10 6000 Biotech/MMN IgM ALS SOC IVIg ARGX-117 1 ARGX-117 C3 2 CIDP ARGX-117 2 POC 2023 POC ARGX-117 IVIg67 0468 1 AAb AAb 20 ITP immune thrombocytopenia 2 AAV ANCA-vasculitis ANCA 21 IVIg intravenous immunoglobulin IVIg3 AE autoimmune encephalitis 22 LN lupus nephritiss 4 AIHA autoimmune hemolytic anemia 23 MMN multifocal motor neuropathy 5 BP bullous pemphigoid 24 MN membranous nephropathy 6 CF cystic fibrosis 25 MOGAD MOG antibody disease MOG-7 CIDPchronic inflammatory demyelinating polyneuropathy 26 NMO ophthalmoneuromyelitis 8 CLE cutaneous lupus Erythe
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