Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (July 2020)
23 slides from Ocugen's investor presentation, Jul 16, 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: July 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 ©2020 Ocugen. All Rights Reserved. Confidential. 5
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 6
Slide 7: Ocugen's Modifier Gene Therapy Platform
Ocugen’s Modifier Gene Therapy 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 We can add
Slide 8: Nuclear Hormone Receptor Genes (NHRs)
Nuclear Hormone Receptor Genes (NHRs) Modulators of retinal development & function Photoreceptor Inflammation & Act as “master genes” in the retina Development NR2E3 Cell Survival Key Mutations: RGR, RHO, PDE6 NR1D1 Key Mutations: PRP16, OTX RORA Molecular reset of key transcription factors an
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. Confidential. https://www.nature.com/articles/s41434-020-0134-z 12
Slide 13: OCU400 – Clinical and Regulatory Strategy
OCU400 – Clinical and Regulatory Strategy Planned Timeline 2021 2022 - 2024 2025 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 Spectrum T
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 AMD
OCU410 (AAV-RORA) – Dry AMD 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 the retina ~9-10M patients in the U.S.* Inflammation Currently no approved treatment for Dry AMD Normal Retin
Slide 16: Gene Therapy Manufacturing Partnership
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 17
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% patients do not respond to DR ~7.7M patients in the US* Anti-VEGF/corticosteroids Therapies Wet AMD ~1.1M patients in the US* OCU200 is a Transferrin-
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 OCU 200 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 nativ
Slide 21: Near & Mid-Term Milestones: Planned Timeline
Near & Mid-Term Milestones: Planned Timeline OCU400 IND Filing Expected Safety & Phase 1/2a Complete Publication Phase 1/2a (AAV-NR2E3) -Orphan Drug Designation Efficacy signal Topline results - US in Nature start-US IRDs for Rho Expected -Orphan Drug Phase 1/2a Phase 2b/3 (gene therapy) Tox study S
Slide 22: Capital Structure Summary
Capital Structure Summary Capital Structure Summary Cash & Cash Equivalents (6/30/20) $15.0M Debt Principal O/S (6/30/20) • EB5 Loan : 1.5M $6.4M • PPP Loan : 0.4M • Apr ‘20 Notes: 4.5M Common Stock O/S (7/15/20) 135.0M Warrants O/S (6/30/20) 0.9M Options O/S (6/30/20) 4.5M ©2020 Ocugen. All Rights
Slide 23: 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