Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (June 2020)
22 slides from Ocugen's investor presentation, Jun 3, 2020
Slide 1: Our Mission is to develop Gene Therapies to Cure Blindness Diseases
Our Mission is to develop Gene Therapies to Cure Blindness Diseases NASDAQ: OCGN Corporate Deck
Slide 2: Forward Looking Statement
Forward Looking Statement This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this presentation, including statements regarding our business strategy, future results of operati
Slide 3: Company Highlights
Company Highlights OCGN Strong Diversified (NASDAQ) Global IP Portfolio CUTTING-EDGE TECHNOLOGY PLATFORM Strategic Gene Therapy MODIFIER GENE THERAPY PLATFORM Manufacturing Partnership OCU400 for Inherited Retinal Diseases OCU410 for Dry AMD NOVEL BIOLOGIC THERAPY FOR RETINAL DISEASES OCU200 for DME
Slide 4: Experienced Leadership Team
Experienced Leadership Team • Diverse experience in large pharma, signature biotech, and small companies • Development, Manufacturing and Commercialization expertise provides know-how to take pipeline from preclinical to market Shankar Musunuri, PhD, MBA Chairman, CEO and Co-Founder Sanjay Subramani
Slide 5: Pipeline Overview
Pipeline Overview ©2020 Ocugen. All Rights Reserved. 5
Slide 6: Breakthrough Modifier Gene Therapy Platform Addressing Multiple Diseases with One Product
Breakthrough Modifier Gene Therapy Platform Addressing Multiple Diseases with One Product 6
Slide 7: Nuclear Hormone Receptor Genes are Important Modulators of Retinal Development & Function
Nuclear Hormone Receptor Genes (NHRs) are Important Modulators of Retinal Development & Function Metabolism = Inflammation and energy Cone cell Cell Survival development = daylight and color vision Phototransduction = translates light into NR2E3 an electrical signal NR1D1 Photoreceptor RORA developm
Slide 8: Potential to Treat Many Diseases with One Product
Potential to Treat Many Diseases with One Product Modifier Gene Therapy: Introduce a functional gene to modify the expression of many genes and gene-networks, and regulate basic biological processes in retina Potential to address a number of diseases Modifier Gene M using the same Modifier Gene prod
Slide 9: Preclinical Data Published in Nature Gene Therapy
Preclinical Data Published in Nature Gene Therapy Efficacy results in 5 unique mouse models of retinitis pigmentosa (RP) Underwent administration of AAV-NR2E3 by subretinal injection Study demonstrates potency of novel modifier gene therapy to elicit broad-spectrum therapeutic benefits in early and
Slide 10: OCU400 Preserves Photoreceptors in Early Stage of Disease
OCU400 Preserves Photoreceptors in Early Stage of Disease Control PDE6β Rhodopsin Leber Congenital Enhanced S-cone associated RP associated adRP Amaurosis Syndrome Retina histology • P0 single subretinal injection, evaluation 3-4 months post injection • rd1 evaluated 1-month post injection • Restore
Slide 11: OCU400 Preserves Photoreceptors in Advanced Stage of Disease
OCU400 Preserves Photoreceptors in Advanced Stage of Disease Rhodopsin Leber Congenital Enhanced S-cone Control associated adRP Amaurosis Syndrome Retina histology • Animals injected at P21 and evaluated 2–3 months post injection • Restored ONL photoreceptors morphology in rd7 • ONL cell layer chang
Slide 12: OCU400 Demonstrates Improved Vision Signals in Retina
OCU400 Demonstrates Improved Vision Signals in Retina Scotopic ERG B-wave Percentage Increase Photopic ERG B-wave Percentage Increase • ERG response: P0 single subretinal injection, evaluation 3-4 months post injection • rd1 evaluated 1- month post injection ©2020 Ocugen. All Rights Reserved. https:
Slide 13: OCU400 Demonstrated Safety in Mouse Model
OCU400 Demonstrated Safety in Mouse Model Study results confirm overexpression of Nr2e3 by subretinal AAV8-Nr2e3 injection is not detrimental to retina ©2020 Ocugen. All Rights Reserved. https://www.nature.com/articles/s41434-020-0134-z 13
Slide 14: OCU400 Regulatory Strategy
OCU400 Regulatory Strategy Proposed Phase 1/2a Phase 2b/3 Potential Approval Broad RP Indication NR2E3 NR2E3 Phase 4 Commitments RHO RHO CEP290 ©2020 Ocugen. All Rights Reserved. 14
Slide 15: Gene Therapy Manufacturing: Plagued by Backlog and Timing Delays
Gene Therapy Manufacturing: Plagued by Backlog and Timing Delays Cell & gene therapy manufacturing demand continues to increase • 1,060 clinical trials globally; 80 cell and gene therapy trials in Phase 3 • Large pharma acquiring companies to support internal programs – eg: Roche acquired Spark; Pfi
Slide 16: OCU400 Gene Therapy Manufacturing: Strategic Partnership with CanSinoBIO
OCU400 Gene Therapy Manufacturing: Strategic Partnership with CanSinoBIO CanSinoBIO • Biotech company publicly-listed on Hong Kong exchange (6185.HK) with market cap of ~$7 Billion USD • State-of-the-art facilities with world class team • Provides scalable GMP cell lines (such as HEK293 suspension c
Slide 17: OCU410 (AAV-RORA): Dry AMD
OCU410 (AAV-RORA): Dry AMD Dry Age-Related Macular Degeneration (AMD) • Leads to irreversible blindness due to degeneration of the retina ~9-10M patients in the US Inflammation Contributing Factors • Aging • Genetics Oxidative RORA Stress Normal Retina • Environmental Factors Lipid Peroxidation Curr
Slide 18: OCU200: DME, Diabetic Retinopathy, Wet AMD Novel Biologic Offering Benefits Beyond Anti-VEGF
OCU200: Diabetic Macular Edema (DME) Diabetic Retinopathy (DR) Wet AMD Novel Biologic Offering Benefits Beyond Anti-VEGF 18
Slide 19: DME, DR & Wet AMD are Leading Causes of Blindness
DME, DR & Wet AMD are Leading Causes of Blindness Diabetic Macular Edema (DME) Diabetic Retinopathy (DR) ~745,000 DME patients in the US Wet AMD • Most common causes of vision loss in patients with diabetes and aging ~7.7M DR patients in the US population • Anti-VEGF & corticosteroids therapies not
Slide 20: OCU200: Tumstatin-Transferrin Fusion Protein
OCU200: Tumstatin-Transferrin Fusion Protein • Inhibits new blood vessel formation • Anti-inflammatory • Anti-oxidative DME/DR Wet AMD Wet AMD Oxygen-Induced Retinopathy (OIR) Mouse Model In-Vivo Laser-Induced Rat CNV Model In-Vivo Laser-Induced Mouse CNV Model 80 Neovascular area (normalized) * 150
Slide 21: Summary of Near-Term Milestones
Summary of Near-Term Milestones OCU400 (AAV-NR2E3) OCU410 (AAV-RORA) OCU200 Retinal Degenerative Diseases Dry AMD DME, DR, Wet AMD (gene therapy) (gene therapy) (novel biologic) Mar 2020: Preclinical Data Published • 2020-2021: Continue IND-Enabling • 2020-2021: Continue IND-Enabling in Nature Gen
Slide 22: A Bold Vision to Cure Blindness Diseases with Gene Therapies
A Bold Vision to Cure Blindness Diseases with Gene Therapies For more information, contact: Kelly Beck Vice President, Investor Relations & Administration kelly.beck@ocugen.com