Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Day (November 2024)
49 slides from Ocugen's investor day presentation, Nov 12, 2024
Slide 1: Courageous Innovation: Clinical Showcase
—_ ~ ocugen. > ee Courageous Innovation Dedicated to Bringing Game-Changing Gene & Cell Therapies and Vaccines to Market and Working Even Harder to Provide Access to Patients Globally Clinical Showcase November 12,2024
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical
Slide 3: Agenda
Agenda Welcome Opening Remarks The Potential of Modifier Gene Therapy Clinical Trial Update Investigator Presentations Patient Panel Commercial Update Q&A Conclusion — ocugen. — Tiffany Hamilton, Head of Corporate Communications Dr. Shankar Musunuri, Chairman, CEO & Co-founder, Ocugen Dr. Arun Upadh
Slide 4: Placeholder for video
Placeholder for video — ocugen. —
Slide 5: We're Here to Make an Impact Through Courageous Innovation
We’re Here to Make an Impact Through Courageous Innovation Company Overview Founded 2013 Our Values Headquarters Malvern, PA Respect Manufacturing Facility Malvern, PA Integrity Teamwork Accountability India Business Center Hyderabad, India Ticker Symbol OCGN Market Cap $300 Million = . ocugen. <——
Slide 6: First-In-Class Modifier Gene Therapy
ocugen. ~<a First-In-Class Modifier Gene Therapy: * OCU400 Phase 3 liMeliGhT Clinical Trial Retinitis pigmentosa * OCU410 Phase 2 ArMaDa Clinical Trial Geographic atrophy ¢ OCU410ST Phase 1/2 GARDian Clinical Trial Stargardt disease
Slide 7: The Potential of Modifier Gene Therapy
The Potential of Modifier Gene Therapy Arun Upadhyay, PhD Chief Scientific Officer and Head of R&D — ocugen. 7 —
Slide 8: Retinal Diseases: Leading Cause of Blindness with Significant Unmet Medical Needs
Retinal Diseases: Leading Cause of Blindness with Significant Unmet Medical Needs Key Retinal Diseases Leading to Blindness Forms of IRD and their underlying mutations * Inherited retinal diseases (e.g. RP, LCA, Stargardt) * Multifactorial (Dry AMD; geographic atrophy) Prevalence * RP-U.S. and EU: 3
Slide 9: Barriers to Effective Treatment in Genetically Diverse & Multifactorial Diseases
Barriers to Effective Treatment in Genetically Diverse & Multifactorial Diseases Gene specific strategy not be applicable for multifactorial/multigenic diseases Significant cost and effort required to develop and manufacture individual gene therapies la \ Focusing ona single factor in multifactorial
Slide 10: Modifier Gene Therapy: A Revolutionary Approach to Treat Disease at Roots
Modifier Gene Therapy: A Revolutionary Approach to Treat Disease at Roots Modifier Genes Main control system for how cells in the retina survive and function Adisease-modifying treatment for inherited and aging related retinal diseases + Gene-Agnostic - Targets multiple genotypes and broader patient
Slide 11: Modifier Gene Therapy (OCU400): Multiple IRDs with ONE Powerful Solution
Modifier Gene Therapy (OCU400): Multiple IRDs with ONE Powerful Solution Therapeutic Development Approaches for IRDs + Gene Augmentation o Delivers normal copy of affected gene OCU400: Modifier Gene Therapy o Limited to treat one gene; monogenic conditions; and recessive gene * GeneAgnostic + Gene E
Slide 12: OCU400: A Novel Modifier Gene Therapy Targeting RP and LCA
OCU400: A Novel Modifier Gene Therapy Targeting RP and LCA OCU400 is designed to address shortcomings of current gene therapy Cytoplasm approaches ocu400 * Broad-spectrum, gene-agnostic approach to genetically diverse IRDs * Potential curative therapy with a single sub-retinal injection po / 4 Cell
Slide 13: OCU400 Preclinical Animal Model Data
• • • • OCU400 (AAV encoding NR2E3) Rho-/- RhoP23H+/- Rd7 (NR2E3) Rd1 (PDE6B) Rd16 (CEP290) Adapted from: § Li et al., Nature Gene Therapy, 2021 § McNamee et al., Nature Gene Therapy, 2024 Fundus Imaging Improvement in Retinal Structure OCU400 Untreated Treated Histology Slow/Stop Retina Degeneratio
Slide 14: OCU410/OCU410ST (ROFRA) for Geographic Atrophy and Stargardt Diseases
OCU410/OCU410ST (ROFRA) for the Treatment for Geographic Atrophy and Stargardt Diseases ocugen. — 14
Slide 15: Regulation of Multiple Pathways by RORA
Regulation of Multiple Pathways by RORA RORA: a nuclear hormone receptor (NHR) and important modulator oe + Plays arole in development and function of rod and cone photoreceptor cells pid de tiepoctte/ + Regulates genes and pathways involved in onset and progression of AMD a xe RORAregulates genes i
Slide 16: OCU410 (RORA): Proof of Concept Summary
OCU410 (RORA): Proof of Concept Summary Anti-drusen activity and improves retinal function 7 Months 12981 Untreated F Abcas! (OCU410 High Dose Treated Abcad” much AbCaK® bcos Ceontot pected OCUNO Macular Degeneration Model Anti-oxidative: Improves ARPE19 cell survival RORA NHR Re — ocugen — Anti-inf
Slide 17: OCU410 (RORA): A Single-Injection Approach to Addressing Unmet Needs in dAMD
OCU410 (RORA): A Single-Injection Approach to Addressing Unmet Needs in dAMD BEYOND the Complement System * Phase 2 OCU410 ArMaDa study for GA currently underway + Advancement from recently approved therapies for GA: Potential to address limitations of recently approved therapies for GA focused only
Slide 18: OCU410: The Opportunity for a One-Time Procedure Beyond the Complement System
OCU410: The Opportunity for a One-Time Procedure Designed to Address All Mechanistic Factors Driving Geographic Atrophy BEYOND the Complement System Anti-Complement Anti-Inflammatory Anti-Drusen Activity Anti-Oxidative v v Vv Vv Anti-complement: Increased Anti-inflammatory: Suppresses Anti-drusen ac
Slide 19: OCU410ST: Received ODD for ABCA4-Associated Retinopathies
OCU410ST: Received ODD for ABCA4-Associated Retinopathies: Stargardt, Retinitis Pigmentosa 19 & Cone-rod Dystrophy 3 ABCA4-associated retinopathies—Genetic Rare Disease * ABCA4gene produces an ATP-binding cassette (ABC) superfamily transmembrane protein involved in clearance of all-trans-retinal ald
Slide 20: Clinical Trial Updates
Clinical Trial Updates Huma Qamar, MD, MPH Chief Medical Officer — ocugen. we
Slide 21: OCU400 Modifier Gene Therapy - A Paradigm Shift in Gene Therapy
OCU400 Modifier Gene Therapy - A Paradigm Shift in Gene Therapy v Health Canadaapproved initiation of OCU400 Phase3 liMeliGhT trial for the treatment of retinitis pigmentosa (RP) v Received FDA approval of expanded access program (EAP) for adult patients with RP Upcoming anticipated catalysts: * Cli
Slide 22: OCU410: A Single-Injection Approach to Address Unmet Need
OCU410: A Single-Injection Approach to Address Unmet Need Y Dosing is underway in the OCU410 Phase 2 ArMaDa clinical trial, following the completion of Phase 1 low, medium, and high dose cohorts, which involved nine patients with geographic atrophy (GA) * The Phase 2 trialis actively recruiting a la
Slide 23: OCU410ST: Modifier Gene Therapy Addressing Shortcomings of Current Approaches
OCU410ST: Modifier Gene Therapy Addressing Shortcomings of Current Approaches v Dataand Safety Monitoring Board (DSMB) approved enrollment for the Phase 2 of the OCU410ST Phase 1/2 clinical trial Upcoming anticipated catalysts: * Preliminary safety and efficacy update trom ongoing Phase 1/2 clinical
Slide 24: Ocular Program
Ocular Prograrn f=} OCU400 for OCU400 for Retinitis Pigmentosa Leber Congenital Amaurosis * Enrollment complete * Treated eyes showed: * Stabilization or preservation (67%) in visual function (BCVA) in peds (9M) and adults (12M) Stabilization or improvement (100%) in overall cone response (FST) in p
Slide 25: Ocular Programs: Key Highlights
Ocular Programs : Key Highlights OCU410 for Geographic Atrophy * Phase 1 complete and Phase 2 enrollment on track * Excellent safety profile with single sub- retinal injection. Reduces treatment burden and compliance issues. * Slows GA lesion growth in treated eye vs. untreated eye at 6M + Low-dose
Slide 26: Outcomes from Novel Modifier Gene Therapy (OCU400) in LCA Subjects with CEP290 mutation
tcomes from Novel Modifier Gene OCU400) in LCA Subjects with CEP290 mutation Benjamin Bakall, MD, Ph.D. sh at Associated Retina Consultants (ARC) & ant Professor at University of Arizona, allege of Medicine - Phoenix
Slide 27: OCU400 Clinical Program (Phase 1/2 Study—Completed Enrollment)
OCU400 Clinical Program (Phase 1/2 Study—Completed Enrollment) * OCU400 demonstrated an excellent safety and tolerability profile in Phase 1/2 retinitis pigmentosa patients with RHOand NRZE3mutations ¢ Evaluating safety and efficacy of OCU400 in Leber Congenital Amaurosis with mutation(s) in CEPz290
Slide 28: LCA Open Label Study
LCA Open Label Study * OCU40O0 is novel Modifier Gene Therapy — Strong Preclinical Data * Asingle subretinal delivery of OCU400 (Medium Dose, 3.33 x 10"° vg/mL) in LCA subjects to overexpress NR2ZE3 * Safety outcomes evaluated in all LCA subjects * Month 9 efficacy is presented for all subjects * Ad
Slide 29: -10 -8 -6 -4 -2 Screening M3 M6 M9 M12 002-007 (treated) 002-007 (untreated)
-10 -8 -6 -4 -2 0 2 4 6 8 Screening M3 M6 M9 M12 002-007 (treated) 002-007 (untreated) 010-001 (treated) 010-001 (untreated) 003-010 (treated) 003-010 (untreated) ∆ ∆ • • • •
Slide 30: Retinal Sensitivity Assessments with FST Demonstrate Improved Cone Response
Retinal Sensitivity Assessments with FST Demonstrate Improved Cone Response by Dark-Adapted Light Testing Adult LCA, 12M Pediatric LCA, 9M 10: mm Treated Eye [= Untreated Eye 2 4 mm Treated Eye 2 S the 6 Be = Untreated Eye 32 oS 2 bs te 2a ca cS Threshold OE 0 Threshold oe BS a6 ge Improvement se Im
Slide 31: Conclusions
Conclusions * OCU40O0 is generally safe and well tolerated in LCA subjects with CEP290mutation * Noserious adverse events (SAEs) deemed related to study drug reported * Oneadult LCA subject showed: * 1Luxlevelimprovementin the Mobility Test (from 130 lux at BL to 50 lux at 12M) * Stabilization in vi
Slide 32: Outcomes from Novel Modifier Gene Therapy (OCU410) from Phase 1/2 Study in GA
Outcomes from Novel Modifier Gene CU410) from Phase 1/2 Study in GA secondary to Dry AMD Syed M. Shah, MD, FACS iseases and Surgery/Emplify Health, Vice Chair For Research 4ealth, Gundersen Health — La Crosse, Wisconsin
Slide 33: OCU410 Phase 1/2 Study: Design and Key Criteria
OCU410 Phase 1/2 Study : Design and Key Criteria Patients with GA secondary to Dry AMD Up to 63 subjects Phase1 Dose escalation Low Dose (n=3) Medium Dose(N=3) High Dose (N=3) (25x10 vg/ml) (6x 10" vg/ml) (1.5x 10" vg/ml) Phase 2 Dose expansion 1:1:1 design Medium dose (N=15) High Dose (MTD) (N=15)
Slide 34: 0.96 ± 0.25 0.76 ± 0.53 Inset • • •
0.96 ± 0.25 0.76 ± 0.53 Inset • • •
Slide 35: OCU410 Treatment Preserves Retinal Tissue in GA Lesions
OCU410 Treatment Preserves Retinal Tissue in GA Lesions — ocugen. — 024 2 2 C3 i) 2 a i Difference in Mean GA Lesion Size Between Treated and Untreated Eyes (mm?) ° 8 8 8 x 1 L 1 f 1 1 4 1 i 1 1 i] a Ht 12 PEOM (6M) PM (6M) ocuat0 (3M) Published data OAKS and DERBY used Sham controls in lieu of untr
Slide 36: -55 -50 -45 -40 -35 -30 -25 -20 -15 -10 -5 SCREENING 3 MONTHS
-55 -50 -45 -40 -35 -30 -25 -20 -15 -10 -5 0 5 10 SCREENING 3 MONTHS 6 MONTHS LL VA c ha ng e (∆ )f ro m b as el in e Study Duration (Months) 006-001 Treated 006-001 Untreated 007-001 Treated 007-001 Untreated 006-004 Treated 006-004 Untreated ±
Slide 37: Conclusions
Conclusions * OCU410 demonstrated a favorable safety and tolerability profile * NoSAEs or AEs deemed related to study drug or study procedure were reported including ischemic optic neuropathy, vasculitis, endophthalmitis, and choroidal neovascularization * OCU410 treated eyes showed a 21.4% decrease
Slide 38: Lejla Vajzovic, MD, FASRS
Vajzovic, MD, FASRS al Vitreoretinal Fellowship Program, phthalmology with Tenure, Adult and Pediatric Surgery and Disease, Duke University Eye Center, sientific Advisory Board Chair of Ocugen
Slide 39: OCU410ST Phase 1/2 Study: Design and Key Criteria
OCU410ST Phase 1/2 Study : Design and Key criteria Patients with Stargardt Disease Up to 42 subjects Phase 2 Dose expansion Numbers and design to be finalized Medium dose (N=8) Phase1 Dose escalation / Key Inclusion Criteria for Adults Age 18-75 years BCVA s SO letters ETDRS Atrophic lesion requirem
Slide 40: Overall Safety
Overall Safety * OCU4I0ST demonstrated a favorable safety and tolerability profile ¢ Nostudy drug or study procedure related SAEs (deemed related to study drug or study procedure) were reported including: * Nodevelopment of exudation * Noinfectious endophthalmitis * Nointraocular Inflammation * Noan
Slide 41: M e a n C h a n g e in T o ta
M e a n C h a n g e in T o ta l A tr o p h y A re a f ro m B a s el in e (m m 2 ) 84% 0.12 ± 0.36 0.73 ± 0.26
Slide 42: OCU410ST Stabilizes or Improves Retinal Structure and Visual Function
OCU410ST Stabilizes or Improves Retinal Structure and Visual Function Parameters for Assessment Low1 Low2 Low3. Med 2 Med 3 Overall Measures Atrophic lesion growth (mm2) compared @ ND & e@ ND to untreated eyes Visual Function Improvement (BCVA) - - e@ e@ 3/5 (60%) @ Total Retinal Thickness (Change f
Slide 43: Conclusions
Conclusions * Demonstrated a favorable safety and durability profile. High dose is the Maximum Tolerated Dose. * Demonstrated stabilization or improvement in visual function and retinal structure outcomes * Currently aligning with FDA for Phase 2 confirmatory trial — ocugen. ig 43
Slide 44: Patient Panel
Patient Panel — ocugen. 38 —
Slide 45: Commercial Update
Commercial Update Mike Shine, MBA SVP Commercial — ocugen. 45 —
Slide 46: Retinitis Pigmentosa: Projected U.S. & EU Market Opportunity for OCU400
Retinitis Pigmentosa: Projected U.S. & EU Market Opportunity for OCU400 Projected Market Landscape at Time of Approval/Launch $61B Opportunity Others ocu400 ~260,000 potential patients 1] icipated 2025 - 2026 0@ Johnson&Johnson aks LUXTURNA innovative Medicine namgsgere ocugen OCU400 RPEGS XLRP Seve
Slide 47: Geographic Atrophy: Projected U.S. & EU Market Opportunity for OCU410
Geographic Atrophy: Projected U.S. & EU Market Opportunity for OCU410 ected Market Landscape at Time of Approval/Launch $115B Opportunity Compliment Inhibitors / Others OCU410 ~1.8 million potential patients 2023) @ Anticipated 2025 - 2027 2028) syFovRe” izervay i ss ocugen. 1 ANNEXON (erent xn Sco
Slide 48: Stargardt: Projected US & EU Market Opportunity for OCU410ST
Stargardt: Projected US & EU Market Opportunity for OCU410ST ected Marke ndscape at T Approval/Launch $23B Opportunity Others OCU410ST ~85,000 Potential Patients Anticipated 2025 - 2027 @ Belite* natigstore oaugen, OCU4IOST Be ocug Retinoid Severe ST only Novel Modifier Gene Inhibitor (Legally Blind
Slide 49: Ocugen Vision
Ocugen Vision We're here to make an impact. At Ocugen, we approach drug development with a sense of urgency, resolve, ingenuity, and boldness. We consider patients in everything we do. Courageous innovation means driving science in new directions and breaking new ground. — ocugen. — 49