Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (March 2021)
31 slides from Ocugen's investor presentation, Mar 5, 2021
Slide 1: Corporate Deck: March 2021
Our Mission is to Develop Gene Therapies to Cure Blindness Diseases and Develop a Vaccine to Save Lives from COVID-19 NASDAQ: OCGN Corporate Deck: March 2021
Slide 2: Forward Looking Statement
1©2021 Ocugen. All Rights Reserved. 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
Slide 3: Ocugen Overview
2©2021 Ocugen. All Rights Reserved. OCUGEN’S BREAKTHROUGH MODIFIER GENE THERAPY PLATFORM NOVEL BIOLOGIC Potential for one product to treat many diseases & multi-factor approach (POC study results published in Nature) OCU400 (AAV-NR2E3): Orphan medicinal product designation for the treatment of b
Slide 4: Leadership Team
3©2021 Ocugen. All Rights Reserved. Shankar Musunuri, PhD, MBA Chairman, CEO and Co-Founder Sanjay Subramanian, MBA CFO and Head of Corporate Development Vijay Tammara, PhD SVP, Regulatory & Quality Jessica Crespo, CPA Corporate Controller Arun Upadhyay, PhD Head of Research & Development Mohamed Ge
Slide 5: Scientific Advisory Boards
4©2021 Ocugen. All Rights Reserved. Mohamed Genead, MD Chair Retina Scientific Advisory Board David Boyer, MD Carl D. Regillo, MD, FACS Mark Pennesi, MD, PhD Geeta Lalwani, MD Scientific Advisory Boards Vaccine Scientific Advisory Board Satish Chandran PhD David Fajgenbaum, MD, MBA, MSc, FCPP Bruce
Slide 6: R&D Pipeline
5©2021 Ocugen. All Rights Reserved. • 81% efficacy Phase-3 interim analysis Active Immunization to Prevent COVID-19 caused by SARS-CoV-2 • EUA in India for development partner • US EUA pathway in development Program Indication Prevalence (US) Discovery Preclinical IND-Enabling Phase 1/2 NR2E3 Mutati
Slide 7: COVAXIN Whole-Virion Inactivated COVID-19 Vaccine Licensed from Bharat Biotech for the US Market
6 COVAXIN™ Whole-Virion Inactivated COVID-19 Vaccine Licensed from Bharat Biotech (BBIL) for the US Market
Slide 8: COVAXIN - Product Profile
7©2021 Ocugen. All Rights Reserved. COVAXIN™ - Product Profile DOSE LEVEL and REGIMEN PRESENTATION STORAGE 0.5mL per dose suspension 2 Doses: Day 0 & Day 28 2°- 8°C (Expected shelf-life ~2 Yrs.) Room temp (25ºC): 3 Months 10 doses per vial PROPOSED INDICATION TARGET POPULATION 12 Years of age and ol
Slide 9: Why COVAXIN
8©2021 Ocugen. All Rights Reserved. Why COVAXIN™ Designed to fill a significant unmet need in our national arsenal of vaccines against COVID-19 COVAXIN™ elicits broad spectrum immune response 98.3% Seroconversion • Both humoral & cellular responses generated against multiple viral proteins • Effe
Slide 10: COVAXIN Presents Multiple Protein Targets to the Immune System
9©2021 Ocugen. All Rights Reserved. Spike (S) Nucleocapsid (N) Membrane (M) Small membrane Protein (E) COVAXIN™ Presents Multiple Protein Targets to the Immune System Resulting in Broad Spectrum Response Spike (S) COVAXIN™ mRNA and Adenovirus-Based Vaccines COVAXIN™, an adjuvanted inactivated virus
Slide 11: COVAXIN Amongst Leading COVID-19 Vaccines and Candidates in the US
10©2021 Ocugen. All Rights Reserved. COVAXIN™ is Distinct Amongst Leading COVID-19 Vaccines and Select Vaccine Candidates in the United States Company Technology Antigen Stage COVAXIN™ Inactivated SARS CoV-2 Virus, Aluminum hydroxide, TLR agonist Whole virus (Including S & N Proteins) EUA in India;
Slide 12: Technology Comparisons: Target Product Profile
11©2021 Ocugen. All Rights Reserved. Technology Comparisons: Target Product Profile Characteristic mRNA Adeno- Based COVAXIN™ Acceptable Safety ✓ ✓ ✓ Neutralizing antibody response ✓ ✓ ✓+ Cellular responses against multiple viral antigens ✓ ✓ ✓+ Efficacy ✓ ✓ ✓+ Stability at 2-8oC X ✓ ✓ Multiple Vira
Slide 13: COVAXIN: Safety, Efficacy, and Immunogenicity
12©2021 Ocugen. All Rights Reserved. COVAXIN™: Safety, Efficacy, and Immunogenicity *: https://www.medrxiv.org/content/10.1101/2020.12.21.20248643v1 **: https://www.fda.gov/media/144325/download Safety* No vaccine-related severe or life-threatening adverse events reported to date Mild to moder
Slide 14: COVAXIN Progress and Planned Milestones for U.S. Development
13©2021 Ocugen. All Rights Reserved. COVAXIN™ Progress and Planned Milestones for U.S. Dev. 1H 2020 2H 2020 Start of Phase 3 by BBIL Completion of Animal Studies by BBIL Ocugen signs Definitive Agreement for COVAXIN™ in US 1H 2021 2H 2021 EUA in India Start of mass immunization Planned EUA Filing wi
Slide 15: Ocugen's Modifier Gene Therapy Platform
14 Ocugen’s Modifier Gene Therapy Platform Breakthrough Technology Designed to Address Multiple Diseases with One Product Approach Complex Diseases Through Multiple Factors
Slide 16: Modifier Gene Therapy vs Gene Augmentation Approach
15©2021 Ocugen. All Rights Reserved. Novel approach that targets nuclear hormone genes (NHRs), which regulate multiple functions within the retina Smoother regulatory pathway due to ability to target multiple diseases with one product Ability to recoup development costs over multiple therapeutic ind
Slide 17: Why Target Nuclear Hormone Receptor Genes (NHRs)?
16©2021 Ocugen. All Rights Reserved. Why Target Nuclear Hormone Receptor Genes (NHRs)? Modulators of retinal development & function Act as “master genes” in the retina Molecular reset of key transcription factors and associated gene networks – retinal homeostasis Gene modifier concept includ
Slide 18: Preclinical POC Data for Nr2e3 Published in Nature Gene Therapy
17©2021 Ocugen. All Rights Reserved. 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-spectrum therapeutic benefits in early and advanced stages of RP
Slide 19: OCU400 – Rescue in Early & Advanced Stage of Disease
18©2021 Ocugen. All Rights Reserved. Human Diseases: Control PDE6β associated RP Rhodopsin associated adRP Leber Congenital Amaurosis Enhanced S-cone Syndrome • P0 single subretinal injection, evaluation 3-4 months post injection • rd1 evaluated one-month post injection ONL: Outer Nuclear Layer • P2
Slide 20: OCU400 – Demonstrates Improved Vision Signals in Retina
19©2021 Ocugen. All Rights Reserved. ERG response: P0 single subretinal injection, evaluation 3-4 months post injection Human vision is enabled by three primary modes: • Photopic vision: Vision under well-lit conditions, which provides for color perception and functions primarily due to cone cells i
Slide 21: OCU400 – Demonstrated Safety in Mouse Model
20©2021 Ocugen. All Rights Reserved. https://www.nature.com/articles/s41434-020-0134-z Study Results Confirm Overexpression of Nr2e3 by subretinal AAV8-Nr2e3 Injection Is Not Detrimental to Retina – No Off-Target Effects OCU400 – Demonstrated Safety in Mouse Model
Slide 22: OCU400 – Clinical and Regulatory Strategy
21©2021 Ocugen. All Rights Reserved. Phase 2b/3Phase 1/2a NR2E3 RHO NR2E3 RHO CEP290 Potential Approval Proposed Broad RP Indication Phase 4 Commitments OCU400 NR2E3 Mutation-Enhanced S-cone Syndrome Rhodopsin Mutation-Associated Retinal Disease CEP290 Mutation-Leber Congenital Amaurosis Broad Spect
Slide 23: OCU400 – Competitive Overview
22©2021 Ocugen. All Rights Reserved. Features OCU400 Traditional Gene Therapy Cell Therapy One product for many IRDs (including broad RP indication) Limited Technology established in the ocular disease space POC data in RP models with different genetic mutations Expected long-term outcome Potentiall
Slide 24: OCU410 (AAV-RORA) – Dry Age-Related Macular Degeneration
23©2021 Ocugen. All Rights Reserved. Dry AMD Leads to irreversible blindness due to degeneration of the retina ~9-10M patients in the U.S. Currently no approved treatment for Dry AMD Contributing Factors Aging Genetics Environmental Factors Normal Retina Dry AMD Oxidative Stress RORA Inf
Slide 25: CanSinoBIO Manufacturing Partnership
24©2021 Ocugen. All Rights Reserved. Ocugen Partnership with CanSino Biologics Inc. (CanSinoBIO) CanSinoBIO to perform CMC development & manufacturing of clinical supplies for OCU400 Publicly-listed (6185.HK) with market cap of ~$7B State-of-the-art facilities with world class team Provides sc
Slide 26: OCU200: Novel Biologic Offering Benefits Beyond Anti-VEGF
25 OCU200: Diabetic Macular Edema (DME) Diabetic Retinopathy (DR) Wet Age-Related Macular Degeneration (Wet AMD) Novel Biologic Offering Benefits Beyond Anti-VEGF
Slide 27: OCU200 – Potential to Treat DME, DR & Wet AMD
26©2021 Ocugen. All Rights Reserved. DME ~0.7M patients in the US* DR ~7.7M patients in the US* Wet AMD ~1.1M patients in the US* OCU200 is a Transferrin-Tumstatin Fusion Protein • Tumstatin: Multiple MOAs for treatment and prevention of macular degeneration and neovascularization • Transfer
Slide 28: OCU200 – Transferrin-Tumstatin Fusion Protein
27©2021 Ocugen. All Rights Reserved. Data expressed as percentage of CNV lesions on Day 10 after treatment. Laser induction & treatment start on Day 0 Wet AMD In-Vivo Laser-Induced Mouse CNV Model * indicates p<0.05 when compared to PBS and/or tumstatin treatment † indicates p<0.05 when compared to
Slide 29: OCU200 – Distinct Mechanism of Action
28©2021 Ocugen. All Rights Reserved. Features OCU200 Anti-VEGF Anti-Integrin Reduces VEGF level/Fluid Selectively works on active endothelial cells (Neovascular) Activates native anti-angiogenic response Enhanced effective delivery through Transferrin Pro-apoptotic and anti-oxidative Dosing Frequenc
Slide 30: Key Inflection Points
29©2021 Ocugen. All Rights Reserved. Key Inflection Points COVAXIN™ - Vaccine candidate for the US market with potential for significant revenues this year Ophthalmology • Modifier Gene Therapy Platform has the potential for one product to treat many diseases • Novel biologic has the potential to
Slide 31: A Bold Vision to Cure Blindness Diseases and Offer a Differentiated Vaccine
A Bold Vision to Cure Blindness Diseases and Offer a Differentiated Vaccine to Save Lives from COVID-19 For more information, contact: IR@ocugen.com