OCUL JPM Presentation (Jan 2025).pdf

1、Ocular Therapeutix Corporate Overview43rdAnnual J.P.Morgan Healthcare Conference|January 2025Retina experience redefined.2Forward Looking Statements and Disclaimers2Any statements in this presentation about future expectations,plans,and prospects for the Company,including the development and regulat

2、ory status of the Companys product candidates,including the design of,and the timing of the enrollment of the Companys SOL-1 and SOL-R Phase 3 clinical trials of AXPAXLI(also called OTX-TKI)for the treatment of wet AMD;the Companys plans to advance the development of AXPAXLI and its other product ca

3、ndidates,including in additional indications such as NPDR;the size of potential markets for the Companys product candidates;the potential utility or adoption,if approved,of any of the Companys product candidates;the sufficiency of the Companys cash resources;and other statements containing the words

4、 anticipate,believe”,estimate”,expect”,intend,“designed”,goal”,may,might”,plan”,predict”,project”,target”,potential”,will”,would”,could”,should”,continue”,and similar expressions,constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995.Actual r

5、esults may differ materially from those indicated by such forward-looking statements as a result of various important factors.Such forward-looking statements involve substantial risks and uncertainties that could cause the Companys preclinical and clinical development programs,future results,perform

6、ance,or achievements to differ significantly from those expressed or implied by the forward-looking statements.Such risks and uncertainties include,among others,the timing and costs involved in commercializing DEXTENZA or any product or product candidate that receives regulatory approval;the ability

7、 to retain regulatory approval of DEXTENZA or any product or product candidate that receives regulatory approval;the initiation,design,timing,conduct,and outcomes of clinical trials,including the SOL-1 trial,the SOL-R trial,and the Companys other ongoing clinical trials;the risk that the U.S.Food an

8、d Drug Administration,or FDA,will not agree with the Companys interpretation of the written agreement under the SPA for the SOL-1 trial and the FDAs other written guidance for the SOL clinical program;the risk that even though the FDA has agreed with the overall design of the SOL clinical program,th

9、e FDA may not agree that protocol and statistical analysis plan or the data generated by the SOL clinical program supports potential marketing approval,even if both SOL-1 and SOL-R are successful and meet their primary endpoints;the risk that the Company and the FDA may not agree on the registration

10、al pathway for AXPAXLI for NPDR or any other indication;uncertainty as to whether the data from earlier clinical trials will be predictive of the data of later clinical trials,particularly later clinical trials that have a different design or utilize a different formulation than the earlier trials,w

11、hether preliminary or interim data from a clinical trial will be predictive of final data from such trial,or whether data from a clinical trial assessing a product candidate for one indication will be predictive of results in other indications;the timing of availability of data from clinical trials

12、and expectations regarding the timing and sufficiency of regulatory submissions and approvals;the Companys scientific approach and general development progress;the availability or commercial potential of the Companys product candidates;uncertainties inherent in estimating the Companys cash runway,fu

13、ture expenses,and other financial results,including its ability to fund future operations,including clinical trials;the Companys existing indebtedness and the ability of the Companys creditors to accelerate the maturity of such indebtedness upon the occurrence of certain events of default;the Compan

14、ys ability to enter into strategic alliances or generate additional funding on a timely basis,on favorable terms,or at all;and other factors discussed in the“Risk Factors”section contained in the Companys quarterly and annual reports on file with the Securities and Exchange Commission.In addition,th

15、e forward-looking statements included in this presentation represent the Companys views as of the date of this presentation.The Company anticipates that subsequent events and developments will cause the Companys views to change.However,while the Company may elect to update these forward-looking stat

16、ements at some point in the future,the Company specifically disclaims any obligation to do so,whether as a result of new information,future events or otherwise,except as required by law.These forward-looking statements should not be relied upon as representing the Companys views as of any date subse

17、quent to the date of this presentation.This presentation discusses investigational product candidates in development.Their efficacy and safety profiles have not been established,and they have not been approved for marketing by the FDA.This presentation contains references to the Companys trademarks

18、and trade names and to those belonging to other entities.Solely for convenience,trademarks and trade names referred to in this presentation may appear without the or symbols,but such references are not intended to indicate,in any way,that the Company will not assert,to the fullest extent under appli

19、cable law,its rights or the rights of the applicable licensor to these trademarks and trade names.The Company does not intend its use or display of other companies trademarks or trade names to imply a relationship with,or endorsement or sponsorship of it by,any other companies.AXPAXLI is a trade nam

20、e which the Company uses to refer to its OTX-TKI product candidate.The FDA has not approved AXPAXLI or OTX-TKI as product names.345Retina Experience RedefinedRedefining developmentRedefining outcomesRedefining treatmentOur retina experience is redefining your retina experience6Our retina experience

21、is redefining your retina experienceRetina Experience RedefinedRedefining treatment7Wet AMD:Significant Unmet Need90%of patients require injection every 1-3 months2 up to12 injections/yr for patientsup to12 PTO days/yr for caregivers INJECTION BURDEN172839410511612Poor long-term visual outcomesPulsa

22、tile dosing leads to fibrosis and atrophy4,5FIBROSIS&ATROPHY1.6M people with wet AMD in U.S.140%discontinue by one year3Illustrative OCTAMD(age-related macular degeneration);OCT(optical coherence tomography).1.Downs P.2023 Retinal Pharmaceuticals Market Report:Global analysis for 2022 to 2028.Market

23、 Scope;2023.2.Ophthalmic Market Trends:Quarterly US Retina Edition:Q3-2024 Analysis of Historical Trends and Latest Developments.Market Scope;2024.3.MacCumber et al.Canadian Ophthalmology Society.2021;10.1016/j.jcjo.2021.10.008.4.Llorente-Gonzalez S,et al.Acta Ophthalmol.2022;100(2):e521-e531.5.Evan

24、s RN,et al.JAMA Ophthalmol.2020;138(10):1109.8Incremental Durability Improvements Deliver Significant Market Opportunity2006 2008 2010 2012 2014 2016 2018 2020 2022 2024VabysmoEyleaLucentisEst.$0.4B$15BGLOBAL BRANDED ANTI-VEGF REVENUE11.Data aggregated from company reports.Captures revenue across al

25、l labeled indications.Figure excludes BEOVU.Q3 24 Revenues carried forward to Q4 24 to curate FY 24 estimate.EYLEA HD FDA approved in August 2023 captured in EYLEA sales figures.2.LUCENTIS package insert.San Francisco,CA:Genentech,Inc.2018.3.Real-World Evidence(TRUCKEE Study):VABYSMO offers 1-2 week

26、 extension over EYLEA in switch patients.Study Results presented at Roche Ophthalmology Day,July 2024.9$15BIncremental Durability Improvements Deliver Significant Market OpportunityLUCENTIS30 Days2200620072008$1.8BEst.2006 2008 2010 2012 2014 2016 2018 2020 2022 2024VabysmoEyleaLucentisLUCENTISGLOBA

27、L BRANDED ANTI-VEGF REVENUE11.Data aggregated from company reports.Captures revenue across all labeled indications.Figure excludes BEOVU.Q3 24 Revenues carried forward to Q4 24 to curate FY 24 estimate.EYLEA HD FDA approved in August 2023 captured in EYLEA sales figures.2.LUCENTIS package insert.San

28、 Francisco,CA:Genentech,Inc.2018.3.Real-World Evidence(TRUCKEE Study):VABYSMO offers 1-2 week extension over EYLEA in switch patients.Study Results presented at Roche Ophthalmology Day,July 2024.10201220132014Incremental Durability Improvements Deliver Significant Market OpportunityLUCENTIS v EYLEA+

29、2 weeksLUCENTIS44 Days330 Days2$2.8B200620072008$1.8B$15BEst.2006 2008 2010 2012 2014 2016 2018 2020 2022 2024VabysmoEyleaLucentisEYLEALUCENTISGLOBAL BRANDED ANTI-VEGF REVENUE11.Data aggregated from company reports.Captures revenue across all labeled indications.Figure excludes BEOVU.Q3 24 Revenues

30、carried forward to Q4 24 to curate FY 24 estimate.EYLEA HD FDA approved in August 2023 captured in EYLEA sales figures.2.LUCENTIS package insert.San Francisco,CA:Genentech,Inc.2018.3.Real-World Evidence(TRUCKEE Study):VABYSMO offers 1-2 week extension over EYLEA in switch patients.Study Results pres

31、ented at Roche Ophthalmology Day,July 2024.11$15B201220132014202220232024Incremental Durability Improvements Deliver Significant Market OpportunityEYLEA v VABYSMOLUCENTIS v EYLEA+2 weeks+2 weeksLUCENTISEst.44 Days357 Days330 Days2$2.8B$4.3B200620072008$1.8BEst.2006 2008 2010 2012 2014 2016 2018 2020

32、 2022 2024VabysmoEyleaLucentisVABYSMOEYLEALUCENTISGLOBAL BRANDED ANTI-VEGF REVENUE11.Data aggregated from company reports.Captures revenue across all labeled indications.Figure excludes BEOVU.Q3 24 Revenues carried forward to Q4 24 to curate FY 24 estimate.EYLEA HD FDA approved in August 2023 captur

33、ed in EYLEA sales figures.2.LUCENTIS package insert.San Francisco,CA:Genentech,Inc.2018.3.Real-World Evidence(TRUCKEE Study):VABYSMO offers 1-2 week extension over EYLEA in switch patients.Study Results presented at Roche Ophthalmology Day,July 2024.126-12 Months Incremental Durability Improvements

34、Deliver Significant Market Opportunity+MONTHSFuture201220132014EYLEA v VABYSMOLUCENTIS v EYLEA+2 weeks+2 weeksLUCENTIS44 Days257 Days230 Days1$2.8B200620072008$1.8B202220232024Est.$4.3BAXPAXLIVABYSMOEYLEALUCENTISData aggregated from company reports.Captures revenue across all labeled indications.Fig

35、ure excludes BEOVU.Q3 24 Revenues carried forward to Q4 24 to curate FY 24 estimate.EYLEA HD FDA approved in August 2023 captured in EYLEA sales figures.1.LUCENTIS package insert.San Francisco,CA:Genentech,Inc.2018.2.Real-World Evidence(TRUCKEE Study):VABYSMO offers 1-2 week extension over EYLEA in

36、switch patients.Study Results presented at Roche Ophthalmology Day,July 2024.13AXPAXLI is Designed to Redefine the MarketAXPAXLIContinuous and consistent delivery up to 12 months3Single injection,single hydrogel3Complete and predictablebioresorption3Highly selective pan VEGF inhibitor1Most potent TK

37、I2AXITINIBMulti-target Tyrosine Kinase Inhibitor(TKI)ELUTYX TECHNOLOGYProprietary hydrogelVersatile,biocompatible,tunable platform3 Bioresorbable,Sustained Drug DeliveryVEGF(vascular endothelial growth factor).1.Zhao Y,et al.Oncologist.2015;20(6):660-673.2.Gross-Goupil M,et al.Clin Med Insights Onco

38、l.2013;7:269-277;Liang C,et al.Mol Ther Oncolytics.2022;24:577-584.3.Blizzard CD,Driscoll A,El-Hayek R,et al.Ocular implant containing a tyrosine kinase inhibitor.Published online September 13,2022.Accessed September 26,2022.14Our retina experience is redefining your retina experienceRetina Experien

39、ce RedefinedRedefining development 15AXPAXLI Wet AMD Clinical Program SummaryU.S.1100%rescue free per protocol at 6 months;80%at 10 monthsAustralia2 Monotherapy activity in treatment-naive wAMDDemonstratedActivity and DurabilityDesigned to Show SuperiorityDesigned to ShowNon-Inferiority to SoCProof

40、of ConceptPhase 3 Registrational TrialsSingle injection AXPAXLI Repeat dosingPHASE 1SOL-1SOL-RComplementary studies designed to showdurability,repeatability,and flexibilitywAMD(wet age-related macular degeneration);SoC(standard of care).1.U.S.Phase I wet AMD trial(NCT06223958).AXPAXLI-treated N=15.P

41、revious reported numbers(80%at 6 months,73%at 10 months)include investigator discretion rescues.2.Australia Phase I wet AMD Trial(NCT03630315).Treatment-nave AXPAXLI monotherapy N=11.16Redefining DevelopmentDe-risking Registrational ProgramCompelling Phase 1 dataDe-risking Phase 3 designsDe-risking

42、regulatory path SOL-1+SOL-RDe-risking Complementary TrialsAXPAXLI Registrational Program in Wet AMDAMD(age-related macular degeneration).17R 1:1SOL-1 Design:AXPAXLI First Registration Study in Wet AMDSuperiority Study Comparing a Single AXPAXLI Dose to a Single Aflibercept(2 mg)DosePRIMARYENDPOINTPo

43、st-week 52 safety follow-upWeekLoading failures enter SOL-RRandomization failures enter SOL-R03653-104-4-852Two-arm trial with 150 subjects per groupDemonstrate that a single AXPAXLI dose is superior to a single aflibercept 2 mg dose based on proportion of subjects who maintained visual acuity,defin

44、ed as 15 ETDRS letters of BCVA loss at Week 36PRIMARY ENDPOINT(36 WEEKS)DESIGNTRIAL SCHEMATICSOL-1Monthly Study VisitsMonthly Study VisitsAXPAXLIAflibercept(2mg)AMD(age-related macular degeneration);BCVA(best-corrected visual acuity);ETDRS(Early Treatment Diabetic Retinopathy Study).18SOL-1 Design:D

45、e-riskingR 1:1PRIMARYENDPOINTPost-week 52 safety follow-upWeekLoading failures enter SOL-RRandomization failures enter SOL-R03653-104-4-852Two-arm trial with 150 subjects per groupDemonstrate that a single AXPAXLI dose is superior to a single aflibercept 2 mg based on proportion of subjects who main

46、tained visual acuity,defined as 300subjects randomizedDec.2024Ocular Therapeutix data on file as of January 10,2025.AMD(age-related macular degeneration).23Clinical Execution Exceeds ExpectationsRetina Leadership Drives AXPAXLI Program for Wet AMD and Beyond311subjects enrolledacross various stages

47、of loading and randomizationJan.10,202520242025MAYConcept and DesignJUL1st Patient EnrolledAUGFDA Type C Supports as 2nd PivotalJAN 2025Enrolling RapidlySOL-RSOL-ROcular Therapeutix data on file as of January 10,2025.AMD(age-related macular degeneration).24Diabetic Retinopathy(DR):Large and Unrealiz

48、ed Market Opportunity HIGH BURDEN,LOW TREATMENT RATEDR is the leading cause of blindnessin the working-age population1NPDR=72%of total DR population50Kadditional patients could avoid vision loss in the U.S.alone31Redefining Outcomes in Wet AMD:Keeping Patients on TreatmentIf 25%discontinued treatmen

49、tcontinuetreatment150Kadditional patients could avoid vision loss in the U.S.aloneOcular Therapeutix company estimates.AMD(age-related macular degeneration).32Redefining Outcomes in Wet AMD:Keeping Patients on TreatmentIf 15%discontinued treatmentcontinuetreatment250Kadditional patients could avoid

50、vision loss in the U.S.aloneOcular Therapeutix company estimates.AMD(age-related macular degeneration).33Redefining Outcomes in Wet AMD:Addressing Pulsatile DosingCurrent SoCfrequent,pulsatile dosingcontinuetreatmentpulsatiletreatmentAMD(age-related macular degeneration);SoC(standard of care).34Rede

51、fining Outcomes in Wet AMD:Addressing Pulsatile DosingAXPAXLInon-pulsatile treatmentoptimizedtreatmentcontinuetreatmentAMD(age-related macular degeneration).35AXPAXLI Estimated U.S.Market Potential:9.2M Patients1wAMD:1.65MRegistrational Trials Ongoing41x5Mod-Severe NPDR:2.7M*Future OpportunitiesDME:

52、1.7MRVO:1.4MPDR:1.7M*Over half(52%)of anti-VEGF injections today are for wet AMD2Registrational Trials in PlanningwAMD(wet age-related macular degeneration);DME(diabetic macular edema);NPDR(non-proliferative diabetic retinopathy);PDR(proliferative diabetic retinopathy);RVO(retinal vein occlusion).*E

53、xcludes patients with DME as some patients have both NPDR/PDR and DME.1.Downs P.2023 Retinal Pharmaceuticals Market Report:Global analysis for 2022 to 2028.Market Scope;2023.2.Ophthalmic Market Trends:Quarterly US Retina Edition:Q3-2024 Analysis of Historical Trends and Latest Developments.Market Sc

54、ope;2024.36Retina Experience RedefinedRedefining developmentRedefining outcomesRedefining treatmentOur retina experience is redefining your retina experience37Redefining Treatment:AXPAXLICompelling Early ResultsProven MoA/Proven DeliveryPotential for up to 12-month dosing across retinal diseasesAXIT

55、INIBELUTYX TECHNOLOGYwAMDNPDR0%vision threatening complications at one year3Improvement in DME3Multi-targeted Highly selectiveMost potent TKIBioresorbableTunableHydrogel100%rescue free per protocol at 6 months180%rescue free per protocol at 10 months1Monotherapy activity in treatment-naive wAMD2 MoA

56、(mechanism of action);TKI(tyrosine kinase inhibitor);wAMD(wet age-related macular degeneration);NPDR(non-proliferative diabetic retinopathy);DME(diabetic macular edema).1.U.S.Phase I wet AMD trial(NCT06223958).AXPAXLI-treated N=15.Previous reported numbers(80%at 6 months,73%at 10 months)include inve

57、stigator discretion rescues.2.Australia Phase I wet AMD Trial(NCT03630315).Treatment-nave AXPAXLI monotherapy N=11.3.HELIOS Phase I NPDR Trial(NCT05695417).AXPAXLI-treated N=14.38Redefining Development:Risk-Off Approach to Registrational ProgramRandomizing reliable anti-VEGF respondersDesigned to en

58、able Q6M dosing on labelProvides commercially relevant dataFDA alignment with Type C written responseSOL-R Non-Inferiority TrialRandomizing strong anti-VEGF respondersDesigned to establish AXPAXLI durabilityDesigned to enable superiority claim on labelFDA alignment through SPASOL-1 Superiority Trial

59、 Complementary trials with measures taken to de-risk outcomes VEGF(vascular endothelial growth factor);SPA(Special Protocol Agreement).39Redefining the Market:AXPAXLI Designed with Ease of Adoption In MindTo succeed in the retinal vascular disease market,new products MUST meet three key criteria:Saf

60、eEffective Durable AdoptableFlexibleTo drive utilization QUICKLY,new products should also be:With AXPAXLI,we intend to check all these boxes,and more.Our retina experience tells us 40Resourced for Success:Infrastructure,Capital,and Expertise to ExecuteWorld-class team played key roles in the approva

61、ls of Lucentis,Eylea,and VabysmoExpertise InfrastructureClinical and commercialcapabilities to execute successfully Capital Strong cash position($427M at 9/30/24)expected to fund operations into 202811.As of September 30,2024.Current cash expected to fund operations through topline data for both wet

62、 AMD pivotal trials,with support from continued DEXTENZA growth;does not include expenses related to future clinical trials.41Our retina experience is redefining your retina experienceRetina Experience RedefinedRedefining treatmentRedefining developmentRedefining outcomes THANK YOU.Retina experience redefined.Investor R