Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (February 2020)
22 slides from Ocugen's investor presentation, Feb 14, 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 Sanjay Subramanian, MBA Chairman, CEO and Co-
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 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’ oGVHD using the International Chronic Oc
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: Potential to Treat Many Diseases with One Product
Potential to Treat Many Diseases with One Product Gene Augmentation: Transfer functional version of a Modifier Gene Therapy: Introduce a functional gene to modify the non-functional gene into the target cells. expression of many genes, gene-networks and reset homeostasis. We can address a number Mod
Slide 14: OCU400 (NR2E3-AAV) Rescues Vision Loss in Multiple Inherited Retinal Diseases
OCU400 (NR2E3-AAV) Rescues Vision Loss in Multiple Inherited Retinal Diseases (IRDs) Human Disease: PDE6β PDE Rhodopsin Rhodopsin Leber associated associated associated associated Congenital RP arRP adRP adRP Amaurosis Mouse Models: rd1-/- rd10-/- Rho-/- RhoP23H Cep290 (rd16) Untreated Treated* ^ ^
Slide 15: OCU400: Orphan Drug Designation
OCU400: Orphan Drug Designation NR2E3 Mutation-Associated Retinal Degeneration Early stage disease 1 mo Baseline (Control) 1 mo after treatment OCU400 Single Injection (Intravitreal) Advanced stage disease 3 mo Baseline (Control) 1 mo after treatment OCU400 Single Injection (Intravitreal) 3 mo Basel
Slide 16: 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 17: 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 18: OCU410 (RORA-AAV): Dry AMD
OCU410 (RORA-AAV): 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 19: OCU200: DME, Diabetic Retinopathy, Wet AMD
OCU200: Diabetic Macular Edema (DME) Diabetic Retinopathy (DR) Wet AMD Novel Biologic Offering Benefits Beyond Anti-VEGF 19
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 OCU300 OCU400 (NR2E3-AAV) OCU200 ocular GVHD Retinal Degenerative Diseases DME, DR, Wet AMD (Phase 3 small molecule) (gene therapy) (novel biologic) Dec 2019: 50% Enrollment Achieved Feb 2019: Pre-IND Meeting • 2019-2020: Continue IND-Enabling Studies • 1H2020: Ex
Slide 22: 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