APLS_JPM2025_presentation.pdf

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1、Boldly delivering life-changing medicinesCedric Francois,M.D.,Ph.D.Co-Founder,Chief Executive Officer and PresidentJanuary 13,20252Forward-looking statementsStatements in this presentation about future expectations,plans and prospects,as well as any other statements regarding matters that are not hi

2、storical facts,may constitute“forward-looking statements”within the meaning of The Private Securities Litigation Reform Act of 1995.Such forward-looking statements include the Companys plans,strategies and expectations for its preclinical,clinical and commercial development of its products and produ

3、ct candidates,its expectations regarding the sNDA for pegcetacoplan for the treatment of for C3G and primary IC-MPGN and the potential commercialization thereof,its plans to initiate Phase 3 studies of pegcetacoplan in FSGS and DGF and the Companys expectations regarding achieving profitability and

4、the timing thereof.The words“anticipate,”“believe,”“continue,”“could,”“estimate,”“expect,”“intend,”“may,”“plan,”“potential,”“predict,”“project,”“should,”“target,”“will,”“would”and similar expressions are intended to identify forward-looking statements,although not all forward-looking statements cont

5、ain these identifying words.Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors,including adjustments to the Companys preliminary revenue figures resulting from,among other things,the completion of financial closing an

6、d review procedures for the quarter and year ended December 31,2024;whether the results of the Companys clinical trials for EMPAVELI,SYFOVRE,or any of its future products will warrant regulatory submissions to the FDA or equivalent foreign regulatory agencies;whether pegcetacoplan will receive appro

7、val from the FDA or equivalent foreign regulatory agencies for C3G and IC-MPGN or any other indication when expected or at all;rate and degree of market acceptance and clinical utility of EMPAVELI,SYFOVRE and any future products for which we receive marketing approval will impact our commercializati

8、on efforts;whether SYFOVRE will receive approval from foreign regulatory agencies for GA when expected or at all;whether the Companys clinical trials will be completed when anticipated;whether results obtained in clinical trials will be indicative of results that will be generated in future clinical

9、 trials or in the real world setting;whether the period for which the Company believes that its cash resources will be sufficient to fund its operations;and other factors discussed in the“Risk Factors”section of Apellis Annual Report on Form 10-K with the Securities and Exchange Commission(SEC)on Fe

10、bruary 27,2024,in Apelliss Quarterly Report on Form 10-Q filed with the SEC on August 1,2024 and the risks described in other filings that Apellis may make with the SEC.Any forward-looking statements contained in this presentation speak only as of the date hereof,and Apellis specifically disclaims a

11、ny obligation to update any forward-looking statement,whether as a result of new information,future events or otherwise3Our missionWe combine courageous science and compassion to develop life-changing medicines for some of the most challenging diseases patients faceROBLiving with GA4Apellis is posit

12、ioned for sustainable value creationMaximizing two blockbuster opportunities with SYFOVRE and EMPAVELILeveraging expertise in complement science to advance our pipeline and drive the next wave of therapeutic innovationOn a path to profitability5Building a top-tier biotech company through clinical an

13、d commercial execution2009 2021Pioneered a new class of complement medicinesAdvanced therapies that target C3 to provide comprehensive control of complement2021 2024Became leaders in C3 therapiesDelivered first new class of complement medicines in 15 years2025+Unlocking blockbuster potential&driving

14、 the next wave of innovationReach more patients with SYFOVRE&EMPAVELIDeliver multiple kidney launchesAdvance innovative pipeline leveraging complement expertiseOur ambitions:6On track to deliver long-term profitable growthCash and cash equivalents of$410 million as of December 31,20241Expect existin

15、g cash and projected revenues to fund operations to profitability1.Estimated 2024 cash and cash equivalents 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 and review procedures.2.Non-GAAP

16、 Operating Expenses are adjusted for stock-based compensation,depreciation and amortization.Nine months ending September 30,2023Nine months ending September 30,2024Total RevenueNon-GAAP OpEx+Cost of Sales128%YOY Total Revenue growth$620M$250M$570M$600M27Our three strategic pillarsTransform the treat

17、ment of GA with SYFOVREMaximize EMPAVELIs impact in rare diseasesAdvance innovative pipeline,leveraging our expertise in complement8Strategic pillar#1:transform the treatment of GA with SYFOVRE Strengthen SYFOVREs position as standard of care Develop leading next-gen therapy:SYFOVRE+APL-3007(siRNA)B

18、ecome a leader in nephrology,starting with C3G/IC-MPGN Expand into additional rare,nephrology indications Develop novel,gene-edited FcRn therapyTransform the treatment of GA with SYFOVREMaximize EMPAVELIs impact in rare diseasesAdvance innovative pipeline leveraging expertise in complement9has incre

19、ased efficacy over timeis approved for use beyond 12 monthsSYFOVRE is the market-leading treatment for GA in the U.S.is approved for as few as 6 doses per yearis in a preferred position with many payersONLY SYFOVRE 10SYFOVRE maintains U.S.leadership in GA category with 120%annual sales growth in FY

20、20241.Estimated 2024 SYFOVRE net product revenue 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 and review procedures.$886 millionSince launch through December 31,202462K510K injectionsth

21、rough December 31,2024,including clinical trials20232024SYFOVRE Injections(incl.clinical trials)160K510K20232024SYFOVRE U.S.net product revenue$611M$275M11Strengthening SYFOVREs position as standard of careSecure SYFOVRE as the market leaderGrow the GA marketExecuting on two key priorities:51.5M GA

22、patientsestimated in U.S.1GA patientscurrently in ECP office170congresses200peer-to-peer education programs2,300sites of care have ordered SYFOVRE95%of Medicare lives with broad coverage0Payers with a 12-month restrictionOnly preferred GA therapy30%PE-backed practices13Key initiatives to bring SYFOV

23、RE to more GA patientsBroaden reach to eyecare communityAmplify real-world datawith clinical analyses&third-party evidenceConnect with patientsthrough new DTC campaignEducate payerson SYFOVREs differentiated value proposition14New SYFOVRE DTC campaign launching this month!15SYFOVRE+APL-3007:potentia

24、l next generation treatment aimed at comprehensively blocking complement activity in the retina and choroidAPL-3007(siRNA)Phase 1 study in healthy volunteersMean change from baseline in C3Strategic goal:evaluate potential for SYFOVRE+APL-3007 to show superiority vs SYFOVRE aloneAPL-3007 reduced circ

25、ulating C3 concentration by up to 90%in a single dose,Phase 1 healthy volunteer studyExpect to initiate Phase 1b/2 multi-dose study in 2Q 20251.Phase 1b/2 study will evaluate safety,biologic activity,and pharmacodynamics of multiple doses of APL-3007 in combination with SYFOVRE.-100-80-60-40-200Mean

26、(SE)%Change from Baseline in Complement C3(mg/dL)Time Point(Day)Dose 1Dose 2Dose 3Dose 4Pooled DosePooled Placebo16Strategic pillar#2:maximize EMPAVELIs impact in rare diseases Strengthen SYFOVREs position as standard of care Develop leading next-gen therapy:SYFOVRE+APL-3007(siRNA)Become a leader in

27、 nephrology,starting with C3G/IC-MPGN Expand into additional rare,nephrology indications Develop novel,gene-edited FcRn therapyTransform the treatment of GA with SYFOVREMaximize EMPAVELIs impact in rare diseasesAdvance innovative pipeline leveraging expertise in complement17EMPAVELI on track to beco

28、me a blockbuster opportunity1.Estimate based on available epidemiological literature and claims data.2021PNHHematology2025C3G&IC-MPGNRare Nephrology2028+DGF&FSGSRare Nephrology1.5K patients15K patients130K patients118As of December 31,2024:$23 million in 4Q 2024 U.S.net product revenue 97%patient co

29、mpliance rate Continued strong safety profile with zero meningococcal infections due to encapsulated bacteria Robust efficacy sustained long-termEMPAVELI continues to elevate the standard of care in PNH1.Estimated 2024 EMPAVELI net product revenue are unaudited,preliminary and based on managements e

30、stimate as of the date of this presentation and are subject to completion of the Companys financial closing and review procedures.19C3G and IC-MPGN:two debilitating kidney diseases Rare kidney diseases with no approved therapiesProgress to kidney failure in 50%of patients within 5-10 years of diagno

31、sis Leads to kidney transplant or lifelong dialysis,neither curative5,0001 people with C3G/IC-MPGN in U.S.1.Based on moderate&severe patient population.C3G:ClearView Analysis using physician and literature consensus20C3G/IC-MPGN:EMPAVELI showed positive effects on 3 key disease markers in 6 Months i

32、n Phase 3 VALIANT study68.1%relative reduction1(95%CI)in pegcetacoplan vs placebo arms(57.3,76.2)P.00011.Percentages calculated by converting the ratio of geometric means to percentages71.4%of pegcetacoplan-treated patients achieved zero intensity staining at Week 26 vs 8.8%for placeboClearance of C

33、3c StainingDemonstrated stabilization of kidney function based on 6-month eGFRStabilization of eGFRReduction in ProteinuriaBaselineWeek 26+6.3mL/min/1.73m2 pegcetacoplan vs placeboP=.03(nominal)Consistent effects across subgroups based on disease type,age,and transplant statusFavorable safety and to

34、lerability,consistent with established profile21Feedback from C3G patient in VALIANT studyJoining the VALIANT trial and receiving pegcetacoplan has changed my and my familys lives.I am back to my old self again.I have the energy not only to walk around the block,but to climb mountains and to run mar

35、athons.I have been able to reduce the number of medications I was taking to less than half.Simply put,you have given me hope,and that is the best thing patients like myself can ask for.1.Individual patient results may vary.22Anticipate broad EMPAVELI adoption in C3G and IC-MPGN1.2024 claims data ana

36、lysis(patients with confirmed diagnosis).Servais et al.Kidney Int.2012;82(4):454.Iatropoulos et al,Mol Immunol.2016;71:1315K C3G/IC-MPGN patients estimated in U.S.1SeverePost transplantModerateMild2370%of C3G/IC-MPGN patients covered by overlapping PNH targets 1.Apellis internal assessment based on

37、claims data and HCP-HCO affiliation analysis.C3G/IC-MPGN Nephrology Primary HCO Targets1900 overlapping accounts include:70%of C3G/IC-MPGN patients60%of prescribing nephrologistsPNH Primary HCO TargetsOVERLAPPING HCO TARGETS 25%overlap between C3G/IC-MPGN and PNH primary HCO targets24Focused commerc

38、ial footprint covers majority of target nephrologists and patientsHCO accountsEach sales-based employee covers 20 HCO accountsOf the 900 target HCO accounts,commercial footprint requiresless than 50 sales-based employees25Expanding EMPAVELI into new rare,kidney indicationsPRIMARY FOCAL SEGMENTAL GLO

39、MERULOSCLEROSIS(FSGS)DELAYED GRAFT FUNCTION(DGF)Plan to initiate two Phase 3 trials in 2H 202511.FDA interactions are planned in 1H 2025 for both indications.26Primary FSGS is a rare,progressive kidney diseaseApproximately 13,000 patients in the US have primary FSGS1FSGS DISEASE OVERVIEWRare kidney

40、disease that causes scarring in the glomeruli and can lead to ESRD UNMET NEED No FDA-approved therapies 50%of patients progress to end-stage kidney disease(ESKD)within 5-10 years1 Persistent proteinuria is a major predictor of progression and remains difficult to controlRATIONALE FOR EMPAVELI Low le

41、vels of C3 correlate with higher disease activity,poorer outcomes,and higher risk of progression to ESKD Complement proteins are detected in glomeruli of patients 1.Prevalence estimated using claims data and published literature for FSGS incidence and sub-types.2.Bensink,Mark E et al.Kidney Med.2023

42、;6(2):100760(USRDS Analysis).27DGF presents a significant challenge in kidney transplantationDGF DISEASE OVERVIEW A condition where the transplanted kidney fails to function,requiring dialysis 1-week post-transplantUNMET NEED No FDA-approved therapies DGF leads to a higher incidence of transplant re

43、jection including re-transplantation Risk of graft failure and mortality persist after DGF resolutionRATIONALE FOR EMPAVELIComplement plays a significant role in transplantation processComprehensive C3 inhibition may both prevent and treat complement overactivation associated with DGFDGF occurs in a

44、pproximately 30-35%of deceased donor kidneys(21,000 in the US in 2023)11.Organ Procurement and Transplantation Network.28Key EMPAVELI milestones in 2025Early 2025Plan to submit U.S.supplemental NDA for C3G/IC-MPGN2H 2025U.S.launch of EMPAVELI in C3G/IC-MPGN,if approvedEarly 2025Sobi expected to subm

45、it application in EMA for C3G/IC-MPGN2H 2025Anticipate initiation of two Phase 3 trials in nephrology29Strategic pillar#3:advance innovative pipeline Strengthen SYFOVREs position as standard of care Develop leading next-gen therapy:SYFOVRE+APL-3007(siRNA)Become a leader in nephrology,starting with C

46、3G/IC-MPGN Expand into additional rare,nephrology indications Develop novel,gene-edited FcRn therapyTransform the treatment of GA with SYFOVREMaximize EMPAVELIs impact in rare diseasesAdvance innovative pipeline leveraging expertise in complement30Developing first-ever gene editing approach to FcRnO

47、PPORTUNITY FOR:First-ever,gene-edited FcRnNeonatal Fc receptor(FcRn)One-time dosingPre-clinical studies ongoing31PRODUCTDISEASEPRECLINICALPHASE 1PHASE 2PHASE 3APPROVEDOPHTHALMOLOGYSYFOVRE(pegcetacoplan injection)GAAPL-3007+SYFOVREGARARE DISEASEEMPAVELI (pegcetacoplan)*PNHC3G/IC-MPGNHSCT-TMAFSGSDGFNE

48、UROLOGYRNA therapies Undisclosed MULTIPLE THERAPEUTIC AREASGene-edited FcRn therapy(Beam)UndisclosedGene-edited complement therapies(Beam)UndisclosedOral complement inhibitorUndisclosed*Sobi has global co-development and ex-U.S.commercialization rights for systemic pegcetacoplanThe safety and effica

49、cy of the agents for the indications under investigation have not been established.Advancing innovative pipeline leveraging expertise in complementMarketed in the USMarketed in the USPlan to Initiate Ph3 in H2 2025Plan to Initiate Ph1b/2 in Q2 2025Plan to Initiate Ph3 in H2 202532Apellis is positioned for sustainable value creationMaximizing two blockbuster opportunities with SYFOVRE and EMPAVELILeveraging expertise in complement science to advance our pipeline and drive the next wave of therapeutic innovationOn a path to profitability