Slide 1: Courageous Innovation August 2022
Courageous Innovation August 2022 NASDAQ: OCGN
Slide 2: Forward Looking Statements
Forward Looking Statements 2 This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are based on the beliefs and assumptions of Ocugen, Inc. and on information currently available to management. All statements contained
Slide 3: We're Here to Make an Impact Through Courageous Innovation
We’re Here to Make an Impact Through Courageous Innovation 3 Mission: At Ocugen, we are developing novel solutions to medical challenges, approaching healthcare innovation with purpose and agility to deliver new options for people facing serious disease and conditions Pioneering a breakthrough modif
Slide 4: Pipeline Overview
4 Pipeline Overview Asset/Program Indication Status Vaccine COVAXIN™ (BBV152) SARS-CoV-2 virus COVID-19 • EUA for adults in Mexico; EUA for 2 to 18-year-olds pending* • U.S. Phase 2/3 Immuno-bridging and broadening clinical trial in-progress • Health Canada NDS under review* Cell therapy NeoCart® (A
Slide 5: Modifier Gene Therapy Platform
5 MODIFIER GENE THERAPY PLATFORM Breakthrough technology designed to address many rare diseases as well as complex diseases that affect millions
Slide 6: Prevalence of IRDs and Associated Genes
~2 million >250 genes IRD ~1.5 million >100 genes RP ~100,000 25 genes LCA ~100,000 ~8,200~400,000 • Only one approved gene therapy for LCA associated with RPE65 mutation: Luxturna® (Voretigene Neparvovec) • Electronic Smart Glasses are used to enable low vision aid in retinitis pigmentosa (RP) pati
Slide 7: Our Vision: Inherited Retinal Diseases Modifier Gene Therapy vs Traditional Gene Augmentation
7 Our Vision: Inherited Retinal Diseases Modifier Gene Therapy vs Traditional Gene Augmentation Normal gene X Gene Augmentation: Transfer functional version of a non-functional gene into the target cells Modifier Gene Therapy: Designed to introduce a functional gene to modify the expression of many
Slide 8: Our Focus: Nuclear Hormone Receptor Genes (NHRs)
8 Our Focus: Nuclear Hormone Receptor Genes (NHRs) *References: https://pubmed.ncbi.nlm.nih.gov/28556246/ | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5409218/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4339951/ | https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0183526 NHRs in
Slide 9: Ocugen's Modifier Gene Therapy Regulates Gene Networks
OCU400, NR2E3 gene within the AAV5 capsid RP /L C A E ye H ea lth y Ey e N R2E3 NR2E3 modifier gene therapy to restore retinal homeostasis Ocugen’s Modifier Gene Therapy Regulates Gene Networks 9
Slide 10: Proof of Principle: Published in Nature Gene Therapy
10 Proof of Principle: Published in Nature Gene Therapy OCU400 NR2E3 Mutation-Associated Retinal Disease CEP290 Mutation-Associated Retinal Disease ONE Disease GENE X GNE X https://www.nature.com/articles/s41434-020-0134-z Important milestone for development of therapy; demonstrated proof of princip
Slide 11: OCU400 Pharmacological Studies
OCU400 Pharmacological Studies Wert et al., J. Vis. Exp. (69), 2012 OCU400 administered subretinally Mouse Model RhoP23HRho-/-rd1 Untreated rd7rd16B6 AAV8-Nr2e3 Treated Human Disease Control PDE6β associated RP Rhodopsin associated adRP Rhodopsin associated adRP Leber Congenital Amaurosis (CEP290) N
Slide 12: OCU400 Biodistribution, Safety and Toxicity Studies
OCU400 Biodistribution, Safety and Toxicity Studies NR2E3 protein expression in the retina following OCU400 administration Retina Vitreous Humor RPE/Choroid Aqueous Humor Iris/Ciliary Body Sclera Lateral Geniculate Nucleus Optic Nerve Optic Tract Relative tissue distribution Image: Charles River Lab
Slide 13: OCU400 Well Tolerated and Not Immunogenic
OCU400 Well Tolerated and Not Immunogenic No toxic effects related to OCU400 No ADA response in serum or aqueous humor No immunogenic response in PBMCs or lymphocytes Center Image: Enrico et al., Clinical Cancer Research 26(4), 2020 13
Slide 14: OCU400 Phase 1/2 Clinical Trial Progress
14 OCU400 Phase 1/2 Clinical Trial Progress ⌛ OCU400 A Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa Associated with NR2E3 (Nuclear Receptor Subfamily 2 Group E Member 3) and RHO (Rhodopsin) Mutations NCT: 05203939 Study Type: Interventional (Clinical Trial) Es
Slide 15: OCU400 Pathway to Phase Clinical Trials
15 OCU400 Pathway to Phase 3 Clinical Trials RP LCA Proposed Indication RP & LCA
Slide 16: OCU410 Modifier Gene Therapy Platform
16 OCU410 Modifier Gene Therapy Platform
Slide 17: OCU410 (AAV-RORA) Dry Age-Related Macular Degeneration
Phase 1/2 clinical trial 17 OCU410 (AAV-RORA) Dry Age-Related Macular Degeneration 30 Dry AMD • Leads to irreversible blindness due to degeneration of the retina • ~9-10M patients in the U.S. • Currently no approved treatment for Dry AMD • Contributing factors: aging, genetics, environmental factors
Slide 18: OCU410 (RORA) A Potential Modifier Therapeutic for Dry-AMD
OCU410 (RORA) A Potential Modifier Therapeutic for Dry-AMD • Genetic modifiers are genes like Nuclear Hormone Receptors (NHR) that can: a) Significantly affect disease outcomes such as onset, rate of progression, and severity b) Enhance or suppress disease phenotypes c) Regulate cellular homeostasis
Slide 19: OCU410 Reduces Drusen in Abca4 -/- Mice, Improves Function
OCU410 Reduces Drusen in Abca4 -/- Mice, Improves Function • ABCA4 is a retina-specific protein localized in outer segment disk edges of rod photoreceptors • Mutations in ABCA have been linked to: a) Age-related macular degeneration (AMD) b) Stargardt macular dystrophy (STGD) c) Recessive RP d) Rece
Slide 20: OCU410 Restores Cd59 Expression in Abca4-/- mice
OCU410 Restores Cd59 Expression in Abca4-/- mice IV - Intravitreal; SR-Subretinal; B6 - C57BL/6mice INL - Inner nuclear layer, ONL - outer nuclear layer RPE – Retinal Pigment Epithelium ONL RPE INL • Abca -/- mice show very low CD59 expression in their retinas • CD59 prevents the formation of the co
Slide 21: OCU200 Novel biologic for DME, DR and Wet AMD
21 OCU200 Novel biologic for treating Diabetic Macular Edema (DME), Diabetic Retinopathy (DR) and Wet Age-Related Macular Degeneration (Wet AMD)
Slide 22: OCU200 Potential to Treat DME, DR & Wet AMD
22 OCU200 Potential to Treat DME, DR & Wet AMD 30 DME DR Wet AMD OCU200 is a Transferrin-Tumstatin Fusion Protein • Tumstatin: Multiple Mechanisms of Action (MOAs) for treatment and prevention of macular edema and neovascularization • Transferrin: Targets the site of action and improves uptake (bett
Slide 23: OCU200 Demonstrated Superior Efficacy Compared to Existing Anti-VEGF Therapies
23 OCU200 Demonstrated Superior Efficacy Compared to Existing Anti-VEGF Therapies 30 DME DR 2 Effect of OCU200 intravitreal treatments on Neovascularization (NV). Data are presented as mean± SD. Filled circles represent data points from individual eyes * P < 0.05, ** P < 0.01 (n = 9-10 eyes per grou
Slide 24: Ocugen Vision
24 Ocugen™ Vision Fully integrated, patient-centric biotech company focused on vaccines in support of public health and gene and cell therapies targeting unmet medical needs through Courageous Innovation
Slide 25: Thank you!
August 2022 NASDAQ: OCGN Thank you!