Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (February 2023)
25 slides from Ocugen's investor presentation, Feb 6, 2023
Slide 1: Courageous Innovation February 2023
Courageous Innovation February 2023 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: Developing cutting-edge innovations for people facing serious disease and conditions with a commitment to ensuring global market access Pioneering modifier gene therapy for inherited retinal diseases, as well as larger blindness d
Slide 4: Pipeline Overview
4 Pipeline Overview Asset/Program Indication Current Status Vaccines COVAXIN™ (BBV152) SARS-CoV-2 virus COVID-19 • EUA for adults in Mexico; EUA for 5 to 18-year-olds submitted • Top line results show that the U.S. Phase 2/3 immuno-bridging and broadening clinical trial met both primary endpoints. A
Slide 5: OCU500 COVID-19 Mucosal Vaccine
5 OCU500 COVID-19 Mucosal Vaccine Exclusive license agreement with Washington University to develop, manufacture and commercialize its proprietary vaccine in the United States, Europe, Japan and other major markets
Slide 6: OCU500 Intranasal Mucosal Vaccine for COVID-19 & Seasonal Flu
OCU500 Intranasal Mucosal Vaccine for COVID-19 & Seasonal Flu • Potential to generate rapid local immunity in the upper airways, and lungs -where SARS-CoV-2 and seasonal flu enters and infects the body • Shown to generate neutralizing IgG, mucosal IgA, and T cell responses to help reduce transmissio
Slide 7: Licensed Mucosal COVID-19 Vaccines Well-Tolerated and Effective as Booster
Licensed Mucosal COVID-19 Vaccines Have Been Well-Tolerated and Demonstrated Efficacy as a Heterologous Booster in Phase 3 Trials Studies demonstrating the benefit of AAV 7 Improved Immunogenicity in Ph3: iNCOVACC vs. COVAXIN • Superior GMT ratio of nAbfor Wuhan (1.45) • Superior GMT ratio of nAbfor
Slide 8: COVAXIN (BBV152)
8 COVAXIN™ (BBV152) A whole-virion inactivated COVID-19 vaccine candidate licensed from Bharat Biotech (BBIL) for North American Markets
Slide 9: Why COVAXIN (BBV152)?
9 Why COVAXIN™ (BBV152)? Designed to augment our North American arsenal of vaccines against COVID-19 • Adult and pediatric phase 2/3 data suggest both humoral & cellular responses generated against multiple viral proteins • Data support that the vaccine induces a Th1 response (cell-mediated immunity
Slide 10: Pathway for COVAXIN (BBV152) Development
10 Pathway for COVAXIN™ (BBV152) Development NCT: 05258669 Immuno-bridging and broadening Safety Top line results BLA Submission Window Phase 2/3 Trial A Phase 2/3, Observer-Blind, Immuno-bridging, and Broadening Study of a Whole, Inactivated Severe Acute Respiratory Syndrome Coronavirus (SARS-CoV-2
Slide 11: Modifier Gene Therapy Platform
11 MODIFIER GENE THERAPY PLATFORM Breakthrough technology designed to address many rare diseases as well as complex diseases that affect millions
Slide 12: Modifier Gene Therapy: A Broader Reach
12 Modifier Gene Therapy: A Broader Reach Gene modifier therapy can potentially address multiple genetic defects with a single product utilizing a gene agnostic approach. In patients with IRDs, this could mean: Improved visual outcomes and quality of life GENE X GENE M cell NR2E3 Delivery “Molecular
Slide 13: Proof of Principle: Published in Nature Gene Therapy
13 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 14: OCU400 Phase 1/2 U.S. Clinical Trial Progress
14 OCU400 Phase 1/2 U.S. Clinical Trial Progress OCU400 PHASE 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa Associated with NR2E3 and RHO Mutations and Leber Congenital Amaurosis with Mutations(s) in CEP290 Gene NCT: 05203939 Study Type: Interventional (Clinical Tria
Slide 15: OCU400 Expected Pathway to Clinical Development & Potential Approval
15 OCU400 Expected Pathway to Clinical Development & Potential Approval RP LCA Proposed Indication RP & LCA Both FDA & EMA granted broad orphan drug designation for RP & LCA
Slide 16: Age-related Macular Degeneration and Stargardt Disease
16 Age-related Macular Degeneration(AMD) Stargardt Disease (STGD) Acquired Age-related Inherited Macular Degeneration AMD starts as a “dry,” non- neovascular form Progresses to geographic atrophy Most prevalent (≈90%) form Can progress to a more rapid “wet,” neovascular form Wet AMD may cau
Slide 17: RORA Regulated Gene Networks Relevant in AMD & Stargardt Disease
17 ABCA4 RORA CD59 Inflammatory response RORA regulated gene networks are relevant in AMD and Stargardt disease RORA Regulated Gene Networks are Relevant in AMD & Stargardt Disease
Slide 18: OCU410 Restores Anti-Complement CD59 Expression
18 • Gene variants of ABCA4 are associated with both Stargardt disease and AMD • Very low CD59 (anti- complement) expression in ABCA4 -/- mice retinas • OCU410 restored CD59 expression in the RPE cells • CD59 prevents the formation of the complement membrane attack complex (MAC) and subsequent retin
Slide 19: OCU410 Restoring Retinal Function in ABCA4 -/- Mice
19 OCU410 Restoring Retinal Function in ABCA4 -/- Mice Blue Autofluorescence (Lipofuscin) Wild type Control 1M 7M Abca4-/- OCU410 Abca4-/- Electroretinogram (Retina Function) p-value: **<0.01; *** <0.001; **** <0.0001 • OCU410 treatment reduces the age-dependent increase in lipofuscin and improves r
Slide 20: OCU200 Novel Biologic for DME, DR and Wet AMD
20 OCU200 Novel biologic for treating Diabetic Macular Edema (DME), Diabetic Retinopathy (DR) and Wet Age-Related Macular Degeneration (Wet AMD)
Slide 21: OCU200 Potential to Treat DME, DR & Wet AMD
21 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 22: NeoCart (Autologous chondrocyte-derived neocartilage)
22 NeoCart® (Autologous chondrocyte-derived neocartilage)
Slide 23: NeoCart Regenerative Cell Therapy
23 NeoCart® Regenerative Cell Therapy Attributes: • Designated by FDA as “Regenerative Medicine Advanced Therapy” • Combines breakthroughs in bio-engineering and cell processing to enhance the autologous cartilage repair process • Merges a patient’s own cells with a fortified 3-D scaffold designed t
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: Ocugen
———— ~ ocugen. > ee February 2023 NASDAQ: OCGN