Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (April 2020)
28 slides from Ocugen's investor presentation, Apr 13, 2020
Slide 1: Developing Transformative Therapies to Treat the Whole Eye
Developing Transformative Therapies to Treat the Whole Eye 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 THREE WAVES OF TECHNOLOGICAL INNOVATION Strategic Gene Therapy SMALL MOLECULE PHASE 3 RARE DISEASE ASSET Manufacturing Partnership OCU300 for ocular Graft Versus Host Disease (oGVHD) MODIFIER GENE THERAPY PLATFORM OCU400 for Inh
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: Three Waves of Technological Innovation
Three Waves of Technological Innovation ©2020 Ocugen. All Rights Reserved. 5
Slide 6: OCU300: oGVHD Near-term Commercialization Opportunity
OCU300: oGVHD Near-term Commercialization Opportunity Potential to be First FDA Approved Treatment 6
Slide 7: OCU300 for oGVHD: Unmet Need for Patients with Rare Ocular Disease
OCU300 for oGVHD: Unmet Need for Patients with Rare Ocular Disease Ocular Graft vs Host Disease (oGVHD) ~60% of allogeneic bone marrow transplant • Autoimmune disease that occurs in patients will develop oGVHD allogeneic bone marrow transplant (BMT) patients patients in the US – Donor derived leukoc
Slide 8: Phase 3 Study With Topline Results Expected 2H2020
Phase 3 Study With Topline Results Expected 2H2020 Over 95% planned enrollment completed Indication: Treatment of ocular discomfort and ocular redness in patients with oGVHD ~60 PATIENTS • 84-day, Randomized, Double-Masked, Placebo-Controlled Study • Key inclusion criteria: diagnosis of ‘definite’ o
Slide 9: Active Ingredient Proven Safe and Effective
Active Ingredient Proven Safe and Effective Safety Efficacy Existing Molecule (Brimonidine) Early stage clinical studies led to Phase 3 design • 505(b)(2) regulatory pathway allows use of OcuNanoE™ drug delivery system improves overall efficacy safety data already available for brimonidine • Brimoni
Slide 10: OcuNanoE Drug Delivery System Improves Overall Efficacy
OcuNanoETM Drug Delivery System Improves Overall Efficacy Brimonidine Level in Lacrimal Gland Mouse DED Model (Preclinical) (Preclinical) ** 1000 4 100 3 2 M Tissue ean (SD) Concentration (ng/g) Concentration 10 1 0 3 6 9 12 0 Corneal Fluorescein Score Fluorescein Corneal Drug distribution to lacrim
Slide 11: OCU300 has Compelling Value Proposition
OCU300 has Compelling Value Proposition Patients Physicians Market Market Access Potential • Spend 3 months in hospital after • Hematologists/Oncologists are first • No approved therapy • Potential to be first approved receiving bone marrow transplant prescribers, then referred to product in US mark
Slide 12: Breakthrough Modifier Gene Therapy Platform Addressing Multiple Diseases with One Product
Breakthrough Modifier Gene Therapy Platform Addressing Multiple Diseases with One Product 12
Slide 13: NHRs as Modifier Gene Therapeutics
NHRs as Modifier Gene Therapeutics Nuclear Hormone Receptors (NHRs) NHRs such as NR2E3 play a critical role in modulating cellular homeostasis by regulating basic biological processes • Development • Metabolism • Circadian cycle • Energy homeostasis NHR • Steroidogenesis • Lipid metabolism • Xenobio
Slide 14: 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 15: 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 16: 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 17: 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 18: 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 19: 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 19
Slide 20: 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. 20
Slide 21: 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 22: 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 ~$2 Billion USD • State-of-the-art facilities with world class team • Provides scalable GMP cell lines (such as HEK293 suspension c
Slide 23: 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 24: OCU200: DME, Diabetic Retinopathy, Wet AMD
OCU200: Diabetic Macular Edema (DME) Diabetic Retinopathy (DR) Wet AMD Novel Biologic Offering Benefits Beyond Anti-VEGF 24
Slide 25: 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 26: 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 27: Summary of Near-Term Milestones
Summary of Near-Term Milestones OCU300 OCU400 (AAV-NR2E3) OCU200 ocular GVHD Retinal Degenerative Diseases DME, DR, Wet AMD (Phase 3 small molecule) (gene therapy) (novel biologic) Mar 2020: Over 95% Planned Mar 2020: Preclinical Data Published • 2020-2021: Continue IND-Enabling Enrollment Achie
Slide 28: A Bold Vision to Treat the Whole Eye
A Bold Vision to Treat the Whole Eye For more information, contact: Kelly Beck Vice President, Investor Relations & Administration kelly.beck@ocugen.com