Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (October 2020)
24 slides from Ocugen's investor presentation, Oct 20, 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: Oct 2020
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: Ocugen Overview
Ocugen Overview Developing transformative therapies with the potential to cure blindness diseases Modifier Gene Therapy Platform – potential for one product to treat many diseases & multi-factor approach OCUGEN’S Technology developed at Dr. Neena Haider’s Lab, Harvard Medical School (POC study r
Slide 4: Experienced Leadership Team
Experienced Leadership Team Leadership Team Shankar Musunuri, PhD, MBA Rasappa Arumugham, PhD Vijay Tammara, PhD Sanjay Subramanian, MBA Jessica Crespo, CPA Arun Upadhyay, PhD Chairman, CEO and Co-Founder Chief Scientific Officer SVP, Regulatory & Quality Chief Financial Officer Corporate Controller
Slide 5: Pipeline Overview
Pipeline Overview Program Indication Prevalence (US) Discovery Preclinical IND-Enabling Phase 1/2 NR2E3 Mutation - Associated Retinal Orphan 500 - 600 Degeneration* US RHO Mutation - Associated Retinal Orphan 10,400 - 12,700 Modifier Gene OCU400 Degeneration* US AAV-hNR2E3 CEP290 Mutation - Associat
Slide 6: Ocugen's Modifier Gene Therapy Platform
Ocugen’s Modifier Gene Therapy Platform Breakthrough Technology Designed to Address Multiple Diseases with One Product Approach Complex Diseases Through Multiple Factors 5
Slide 7: Traditional Approach vs. Ocugen's Novel Platform
Traditional Approach vs. Ocugen’s Novel Platform 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 regulate basic biological processes in retina W
Slide 8: Why Target Nuclear Hormone Receptor Genes (NHRs)?
Why Target Nuclear Hormone Receptor Genes (NHRs)? Modulators of retinal development & function Act as “master genes” in the retina Photoreceptor Inflammation & Development NR2E3 Cell Survival Key Mutations: RGR, RHO, PDE6 NR1D1 Key Mutations: PRP16, OTX Molecular reset of key transcription fac
Slide 9: Nature Gene Therapy Publication
Nature Gene Therapy Publication Preclinical POC Data for Nr2e3 Published in Nature Gene Therapy Efficacy results shown in 5 unique mouse models of RP Technology developed at Harvard Medical School, Dr. Neena Haider’s Lab Study demonstrates potency of modifier gene therapy to elicit broad-spect
Slide 10: OCU400 – Rescue in Early & Advanced Stage of Disease
OCU400 – Rescue in Early & Advanced Stage of Disease Early Stage Rescue Advanced Stage Rescue • P0 single subretinal injection, evaluation 3-4 months post injection • P21 subretinal injection, evaluation 2–3 months post injection • rd1 evaluated one-month post injection • Restored ONL photoreceptors
Slide 11: OCU400 – Demonstrates Improved Vision Signals in Retina
OCU400 – Demonstrates Improved Vision Signals in Retina Electroretinogram (ERG) Response Reveals Rescue under Both Scotopic (dim-lit) as well as Photopic (well-lit) Conditions ERG response: P0 single subretinal injection, evaluation 3-4 months post injection Human vision is enabled by three primary
Slide 12: 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 – No Off-Target Effects ©2020 Ocugen. All Rights Reserved. https://www.nature.com/articles/s41434-020-0134-z 11
Slide 13: OCU400 – Clinical and Regulatory Strategy
OCU400 – Clinical and Regulatory Strategy Planned Timeline 2021 - 2022 2023 - 2025 2025/26 Proposed Phase 1/2a Phase 2b/3 Potential Approval Broad RP Indication NR2E3 NR2E3 Phase 4 Commitments RHO RHO NR2E3 Mutation-Enhanced S-cone Syndrome Broad OCU400 Rhodopsin Mutation-Associated Retinal Disease
Slide 14: OCU400 – Competitive Overview
OCU400 – Competitive Overview OCU400 Traditional Gene Therapy Cell Therapy Features One product for many IRDs Limited (including broad RP indication) Technology established in the ocular disease space POC data in RP models with different genetic mutations Potentially longer benefit due Potentially l
Slide 15: OCU410 (AAV-RORA) – Dry Age-Related Macular Degeneration
OCU410 (AAV-RORA) – Dry Age-Related Macular Degeneration We Believe OCU410 Has the Potential to Address this Disease through its Multi-Factor Approach Dry AMD Leads to irreversible blindness due to degeneration of Inflammation the retina ~9-10M patients in the U.S. Currently no approved treatm
Slide 16: Gene Therapy Manufacturing Partnership with CanSinoBIO
Gene Therapy Manufacturing Partnership Helps Advance OCU400 into the Clinic with Significantly Reduced Capital and Resources Ocugen Partnership with CanSino Biologics Inc. (CanSinoBIO) CanSinoBIO to perform CMC development & manufacturing of clinical supplies for OCU400 Publicly-listed (6185.HK) w
Slide 17: OCU200: DME, DR and Wet AMD Novel Biologic
OCU200: Diabetic Macular Edema (DME) Diabetic Retinopathy (DR) Wet Age-Related Macular Degeneration (Wet AMD) Novel Biologic Offering Benefits Beyond Anti-VEGF 16
Slide 18: OCU200 – Potential to Treat DME, DR & Wet AMD
OCU200 – Potential to Treat DME, DR & Wet AMD OCU200 Provides Hope to All patients with DME, DR or Wet AMD DME ~0.7M patients in the US* ~50% of Patients DO NOT Respond DR ~7.7M patients in the US* to Anti-VEGF/Corticosteroids Wet AMD ~1.1M patients in the US* Therapies OCU200 is a Transferr
Slide 19: OCU200 – Transferrin-Tumstatin Fusion Protein
OCU200 –Transferrin-Tumstatin Fusion Protein OCU200 Demonstrated Superior Efficacy Compared to Existing Anti-VEGF Therapies • 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 Mod
Slide 20: OCU200 – Distinct Mechanism of Action
OCU200 – Distinct Mechanism of Action We believe OCU200 has the potential to become a disease modifying therapeutic for broader patient population OCU200 Anti-VEGF Anti-Integrin Features (1) (1) (1) Reduces VEGF level/Fluid Selectively works on active endothelial cells (Neovascular) Activates native
Slide 21: Near & Mid-Term Milestones: Planned Timeline
Near & Mid-Term Milestones: Planned Timeline OCU400 IND Filing Safety & Phase 1/2a Publication Orphan-Drug Designation for (AAV-NR2E3) Phase 1/2a Efficacy signal Topline results in Nature RHO Expected start-US (US) IRDs Orphan-Drug Designation for -Orphan Drug Safety read Phase 1/2a (gene therapy) T
Slide 22: Capital Structure Summary
Capital Structure Summary Capital Structure Summary Cash & Cash Equivalents (9/30/20) $19.1M Debt Principal O/S (9/30/20) • EB5 Loan : 1.5M $3.2M • PPP Loan : 0.4M • Apr ‘20 Notes: 1.3M Common Stock O/S (10/15/20) 162.0M Warrants O/S (9/30/20) 0.9M Options O/S (9/30/20) 4.3M ©2020 Ocugen. All Rights
Slide 23: Investment Highlights
Investment Highlights Modifier Gene Therapy Platform has the potential for one product to treat many diseases Strategic manufacturing partnership with CanSinoBio sets clear path for critical manufacturing OCU200 novel biologic has the potential to treat anti- VEGF/corticosteroids non responder
Slide 24: A Bold Vision to Cure Blindness Diseases with Gene Therapies
A Bold Vision to Cure Blindness Diseases with Gene Therapies For more information, contact: IR@ocugen.com