1、42ndAnnual J.P.Morgan Healthcare Conference 2024Cedric Francois,M.D.,Ph.D.Chief Executive OfficerJanuary 8,20242Forward-looking statementsStatements in this presentation about future expectations,plans and prospects,as well as any other statements regarding matters that are not historical facts,may

2、constitute“forward-looking statements”within the meaning of The Private Securities Litigation Reform Act of 1995.These statements include,but are not limited to,statements regarding preliminary financial information for the fourth quarter and full year ended December 31,2023 and the appeal and re-ex

3、amination of the MAA if CHMP issues a negative opinion for intravitreal pegcetacoplan and the safety profile of intravitreal pegcetacoplan.The words“anticipate,”“believe,”“continue,”“could,”“estimate,”“expect,”“intend,”“may,”“plan,”“potential,”“predict,”“project,”“should,”“target,”“will,”“would”and

4、similar expressions are intended to identify forward-looking statements,although not all forward-looking statements contain these identifying words.Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors,including adjustme

5、nts to Apellis preliminary revenue figures resulting from,among other things,the completion of financial closing and review procedures for the quarter and year ended December 31,2023;whether the benefit/risk profile of SYFOVRE following the reported events of retinal vasculitis will impact our comme

6、rcialization efforts;whether SYFOVRE will receive approval from foreign regulatory agencies for GA when expected or at all,including the impact on the likelihood and timing of such approvals;and other factors discussed in the“Risk Factors”section of Apellis Annual Report on Form 10-K with the Securi

7、ties and Exchange Commission on February 21,2023 and Quarterly Report on Form 10-Q filed on November 1,2023 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission.Any forward-looking statements contained in this presentation speak only as of the da

8、te hereof,and Apellis specifically disclaims any obligation to update any forward-looking statement,whether as a result of new information,future events or otherwise.3ROBLiving with GAWe combine courageous science and compassion to develop life-changing medicines for some of the most challenging dis

9、eases patients face4Developing life-changing medicines for patients700+employeesglobally2 FDA-approved medicinesFIRST C3 THERAPYFocused on PATIENTSof retinal,rare and CNS programsPIPELINEMARKET-LEADING treatment for GATargeting C3 for comprehensive control of complement 52024 priorities support mult

10、iple drivers of future growthAdvance early pipeline and Beam collaborationBring SYFOVRE to patients with GA worldwideMaximize EMPAVELI in PNH&C3G/IC-MPGN with compassion and commitment to patientsReach more U.S.patients with GA123462024 priorities support multiple drivers of future growthAdvance ear

11、ly pipeline and Beam collaborationBring SYFOVRE to patients with GA worldwideMaximize EMPAVELI in PNH&C3G/IC-MPGN with compassion and commitment to patientsReach more U.S.patients with GA12347GA is a leading cause of blindness around the worldNORMAL VISIONVISION WITH ADVANCED GA1 million people in U

12、S1Nearly 7 in 10 people with GA(68%)believe impact on independence and quality of life due to visual decline is worse than expected275 million people worldwide1.Boyer DS et al.,Retina 2017.2.The Geographic Atrophy Insights Survey(GAINS)was conducted by The Harris Poll and was sponsored by Apellis8St

13、rong launch to date:$114 million U.S.net product sales in 4Q23Q1(March)2023Q2 2023Q3 2023Q4 2023U.S.Net Product Sales$18.4M$67.3M$75.3M$114M1Q1(March)2023Q2 2023Q3 2023Q4 2023Total Vials Distributed(incl.samples)9K42K47K62K1.Estimated 2023 SYFOVRE and EMPAVELI U.S.net product sales are unaudited,pre

14、liminary and based on managements estimate as of the date of this presentation and are subject to completion of the Companys financial closing procedures.$275 million2023 U.S.Net Product Sales160K doses(vials distributed)as of December 31,20239SYFOVRE is the market-leading treatment for GA in the U.

15、S.More experience1.Contextualization and support for the statements on this slide are included in subsequent slides.This slide is not intended to make direct or indirect comparative statements against competitor products.More efficacy over timeMore vision for patients10More efficacy over time:contin

16、ued to demonstrate increasing effects,with up to 42%in year 3 in nonsubfoveal patients0.01.02.03.04.05.06.07.08.00122436GA lesion change from baselineLS mean(SE),mmPegcetacoplan Monthly(n=95)Pegcetacoplan EOM(n=104)Sham(n=87)Projected sham Year 1 27%28%Year 2 28%21%Year 3 42%28%All p-values are nomi

17、nalStudy MonthReduction vs sham(Months 0-36)PM:32%;p150K SYFOVRE injections estimated since launch1 SYFOVRE holds an estimated 95%of total patient share2 Double-digit number of new sites each week;70%of demand from non-PE3 Strong access and reimbursement;permanent J-code as of 10/1/23 Estimated rate

18、 of vasculitis remains rare at 0.01%per injection;last confirmed case in Sept 20231.Apellis data on file;2.This is an estimate based on ECP injection demand data on file as of December 31,2023.This dataset may not represent the entire patient population;3.ECP vial shipment data on file.12More vision

19、 for patients:preserved vision longer in multiple post hoc analysesMicroperimetry 10-2 MAIA grid with the 4 central scotomatous points corresponding to the foveal area#64-1,-9#46-5,-7#45-3,-7#44-1,-7#43 1,-7#42 3,-7#41 5,-7#25-5,-5#47-7,-5#40 7,-5#24-3,-5#23-1,-5#22 1,-5#21 3,-5#20 5,-5#26-5,-3#48-7

20、,-3#39 7,-3#10-3,-3#9-1,-3#8 1,-3#7 3,-3#19 5,-3#29 5,3#51-7,3#60 7,3#13-3,3#14-1,3#15 1,3#16 3,3#36 5,3#30-5,5#52-7,5#59 7,5#31-3,5#32-1,5#33 1,5#34 3,5#35 5,5#53-5,7#54-3,7#55-1,7#56 1,7#67-1,9#68 1,-9#57 3,7#58 5,7#27-5,-1#49-7,-1#65-9,-1#38 7,-1#62 9,-1#11-3,-1#6 3,-1#18 5,-1#28-5,1#50-7,1#66-9,

21、1#61 7,1#69 9,1#12-3,1#17 3,1#37 5,1#63 1,-9Central macular light sensitivity analysis using 4 central points,as measured by microperimetry13Pegcetacoplan preserved light sensitivity in central foveaHazard ratio estimated from Cox Proportional Hazards model including patients in the mITT population

22、at-risk for the event with at least one post-baseline assessment.Model includes Treatment+baseline GA lesion area(=7.5 mm2)+baseline presence of choroidal neovascularization in the fellow eye(Yes or No)+baseline number of central 16 scotomatous points.All P values are nominal.Microperimetry only per

23、formed in OAKS.GA,geographic atrophy;mITT,modified intent to treat,BCVA,best-corrected visual acuityMaintenance of light sensitivity in foveal center shown to be associated with less vision loss over 2 years,as measured by BCVALoss of central 4 pointsMonth 24Mean(SD)change inBCVA,ETDRS letters Yes(n

24、=149)-11.7(17.33)No(n=183)-4.9(11.16)0.02.00.51.01.5Favors shamFavors pegcetacoplanMonthlyEOMHazard Ratio34%risk reduction over 2 years(95%CI:0.46,0.96;p=0.0282)36%risk reduction over 2 years(95%CI:0.44,0.92;p=0.0164)0.660.64Loss of central 4 points,as measured by microperimetry14Positive physician

25、feedbackSource:Apellis market research on file.Physician feedback is based on surveyed physicians and may not reflect the opinions and views of all treating physicians.1.This is an estimated rate per injection based on data on file.SANTILiving with GA“The GALE data are promising and continue to sugg

26、est SYFOVREs efficacy increases over time.”“Right now,100%of my patients who receive treatment for GA are getting SYFOVRE.”“I have only used SYFOVRE and continue to use it on my new and existing patients.I feel comfortable with a 1/10,000 risk of vasculitis at this time.”1152024 priorities support m

27、ultiple drivers of future growthAdvance early pipeline and Beam collaborationBring SYFOVRE to patients with GA worldwideMaximize EMPAVELI in PNH&C3G/IC-MPGN with compassion and commitment to patientsReach more U.S.patients with GA123416Approximately 5 million people living with GA worldwide1No appro

28、ved treatments available outside of the U.S.2.5M people with GA in Europe184%of European ECPs are convinced that early GA treatment is essential21.Apellis data on file reflecting analysis of Colijin et al.,2016;Wong et al.2014;Rudnicka et al.,2014;Korb et al.,2014;Piermarocchi et al.,2011;Fernandez-

29、Arias et al.,2011;Augood et al.,2006.2.Europe HCP ATU insights Nov22Add new world17Committed to bringing SYFOVRE to patients in EUQ1 2024Q2 2024Q3 2024CHMP to issue formal opinion.If negative,Apellis to appealCHMP designates new rapporteurs and initiates formal re-examination processCHMP to issue fi

30、nal opinionEuropean Commission decisionAnticipated EU timeline182024 priorities support multiple drivers of future growthAdvance early pipeline and Beam collaborationBring SYFOVRE to patients with GA worldwideMaximize EMPAVELI in PNH&C3G/IC-MPGN with compassion and commitment to patientsReach more U

31、.S.patients with GA123419EMPAVELI is elevating the standard of care in PNH1.Estimated 2023 EMPAVELI U.S.net product sales are unaudited,preliminary and based on managements estimate as of the date of this presentation and are subject to completion of the Companys financial closing procedures.Ashley,

32、living with PNH ASHLEYLiving with PNHAs of December 31,2023:$91 million1 in FY 2023 U.S.net product sales$24 million1 in 4Q 2023 U.S.net product sales 10%of demand in 2023 was from treatment-nave patients 97%patient compliance rate 20Unprecedented safety profile demonstrated through real-world exper

33、ience1,400 patient years of systemic pegcetacoplan exposure1Zero reported cases of meningococcal infection1Thrombosis rate of 0.51 events per 100 patient-years21.Includes patients as of November 2023 from all subcutaneous pegcetacoplan clinical trials and post-marketing exposure of EMPAVELI;2.Includ

34、es patients from U.S.post-marketing exposure of EMPAVELI.21EMPAVELI Injector:improving the patient experience21PATIENT QUOTES1There were no skin issues.It took 45 minutes.Virtually painless!It was great.“Everything went great!Ive actually had less swelling at the injection site with this new pump.So

35、 far Im loving it!”“Super simple,no problems at injections site,no problem removing the injector,not a single problem so far!”Since October approval:50%conversion of existing patients 90%of new starts1.Patient quotes may not necessarily reflect the views and opinions of all patients.Individual patie

36、nt experience may vary.221.Based on moderate&severe patient population.C3G:ClearView Analysis using physician and literature consensusHigh unmet need in C3G and IC-MPGNRare kidney diseases with no approved medicinesProgress to kidney failure in 50%of patients within 5-10 years of diagnosis5,0001 pot

37、ential US patients for C3G/IC-MPGNChase,living with C3G23Phase 2 NOBLE data:pegcetacoplan reduced disease activity in only 12 weeksReduction in C3c staining in pegcetacoplan-treated patientsReduction in intensity%of patients(n=10)1+magnitude80%2+magnitude50%Zero staining40%C3c Staining at Week 12(Pa

38、tient 1)Baseline(C3c 3+)Week 12(C3c 0)24Results strengthen our confidence in pegcetacoplan for C3G/IC-MPGNIn only 12 weeks,pegcetacoplan reduced disease activityTreatment effect shown in both C3G/IC-MPGN in patients with kidney transplantsImprovements across key clinical measures,including stable ki

39、dney function and improved proteinuriaPegcetacoplan was well tolerated in the study25Physician feedback is based on surveyed physicians.Physician quotes may not reflect the opinions and views of all physicians.Nephrologists are highly encouraged by the Phase 2 NOBLE dataThis is miraculousI have a pa

40、tient that needs thisA true disease-modifying agentYou dont have to convince me anymoreI didnt think this was possible!This is impactful there is a clear effect26*Subjects entering VALE,the planned long-term extension study,will not complete the follow-up period.VALIANT Phase 3 study:patient enrollm

41、ent complete,with top-line data study expected mid-2024Group 1+2(n=124)Pegcetacoplan+SOCGroup 1(n=62)Pegcetacoplan+SOCGroup 2(n=62)Placebo+SOCStandard of Care(SOC)(n=124)ScreeningSC infusion,twice weeklyRandomized,double-blind controlled periodWash OutPopulation:Patients 12 years+with C3G or primary

42、 IC-MPGN pre-and post-transplant and evidence of active renal diseasePrimary endpoint:Change in proteinuria(uPCR)at week 26 vs.baselineSecondary endpoints:Change in kidney function measured by eGFR.Reduction in C3 staining.Patient reported fatigue and QOLVALE extension studyUp to 8 weeks26 weeksPrim

43、ary endpointreadoutOpen-label period26 weeksFollow-up*8 weeksSC infusion,twice weekly272024 priorities support multiple drivers of future growthAdvance early pipeline and Beam collaborationBring SYFOVRE to patients with GA worldwideMaximize EMPAVELI in PNH&C3G/IC-MPGN with compassion and commitment

44、to patientsReach more U.S.patients with GA123428PRODUCTDISEASEPRECLINICALPHASE 1PHASE 2PHASE 3APPROVEDOPHTHALMOLOGYSYFOVRE(pegcetacoplan injection)GAOral complement inhibitorUndisclosedRARE DISEASEEMPAVELI(pegcetacoplan)*PNHIC-MPGN&C3GCADHSCT-TMANEUROLOGY Gene/RNA therapies Undisclosed MULTIPLE THER

45、APEUTIC AREASAPL-3007(siRNA silencing C3)UndisclosedGene-edited therapies(Beam)UndisclosedPrioritizing high-potential opportunities in retina,rare and central nervous system(CNS)diseases*Sobi has global co-development and ex-U.S.commercialization rights for systemic pegcetacoplan.The safety and effi

46、cacy of the agents for the indications under investigation have not been established.Marketed in the USMarketed in the US292024 priorities support multiple drivers of future growthAdvance early pipeline and Beam collaborationBring SYFOVRE to patients with GA worldwideMaximize EMPAVELI in PNH&C3G/IC-MPGN with compassion and commitment to patientsReach more U.S.patients with GA123442ndAnnual J.P.Morgan Healthcare Conference 2024Cedric Francois,M.D.,Ph.D.Chief Executive OfficerJanuary 8,2024