Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (May 2021)
35 slides from Ocugen's investor presentation, May 7, 2021
Slide 1: Corporate Deck: May 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: May 2021
Slide 2: Forward Looking Statement
2©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: Modifier Gene Therapy Platform and COVID-19 Vaccine
3©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
4©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 VP, Head of Research & Development Mohame
Slide 5: Scientific Advisory Boards
5©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: Pipeline Overview
6©2021 Ocugen. All Rights Reserved. Pipeline Overview ** No approved therapies exist https://www.aao.org/eye-health/diseases/retinitis-pigmentosa-treatment https://www.aao.org/eye-health/diseases/amd-treatment *Orphan medicinal product designation for the treatment of both retinitis pigmentosa (RP)
Slide 7: COVAXIN Whole-Virion Inactivated COVID-19 Vaccine Licensed from Bharat Biotech
7 COVAXIN™ Whole-Virion Inactivated COVID-19 Vaccine Licensed from Bharat Biotech (BBIL) for the US Market
Slide 8: COVAXIN - Product Profile
8©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
9©2021 Ocugen. All Rights Reserved. B Both humoral & cellular responses generated against multiple viral proteins Induces a Th1 response (cell-mediated immunity) Broad Spectrum Immune Response E Highly effective in neutralizing UK, BZ P2, and India “double mutant” variants Potentially serve as a uni
Slide 10: COVAXIN Presents Multiple Protein Targets to the Immune System
10©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 Developed and Manufactured by Bharat Biotech
11©2021 Ocugen. All Rights Reserved. COVAXIN™ Developed and Manufactured by Bharat Biotech Established Robust Manufacturing Process for COVAXIN Ocugen licensed COVAXIN™ on the back of Bharat’s strong track record of developing & commercializing vaccines globally Inactivated Vero cell derived vaccine
Slide 12: COVAXIN Amongst Leading COVID-19 Vaccines in the United States
12©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 13: Technology Comparisons: Target Product Profile
13©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 14: COVAXIN Progress and Planned Milestones for U.S. Development
14©2021 Ocugen. All Rights Reserved. COVAXIN™ Progress and Planned Milestones for U.S. Dev. Ocugen signs Definitive Agreement for COVAXIN™ in US 1H 2021 2H 2021 EUA in India Start of mass immunization Planned EUA Filing with FDA Phase 3 Interim Analysis Results COVAXIN™ Vaccination Available in US E
Slide 15: Phase 2: Study Results
15©2021 Ocugen. All Rights Reserved. Phase 2: Study Results 6µg +Algel-IMDG demonstrated high neutralizing Abs responses compared to 3µg + Algel-IMDG group Mean GMT (95% CI) higher than human convalescent serum (HCS) 6µg +Algel-IMDG (Covaxin™) selected for Phase 3 study
Slide 16: Phase 2: Study Results
16©2021 Ocugen. All Rights Reserved. Phase 2: Study Results High Seroconversion rates (>96%) in both MNT50 and PRNT50 measured up to day 56 Induction of Th1 cell mediated immunity as measured by IFN-ƴ, IL-2, TNF-α No vaccine-related severe or life- threatening adverse events reported to date E
Slide 17: Phase 3: Study Outline
17©2021 Ocugen. All Rights Reserved. Phase 3: Study Outline
Slide 18: Phase 3: Efficacy - Second Interim Analysis
18©2021 Ocugen. All Rights Reserved. Phase 3: Efficacy - Second Interim Analysis Phase 3 clinical trial enrolled 25,800 participants including 2,433 in the age group 60 to 98 4,500 with comorbidities First interim analysis at 43 cases Phase 1/2 enrolled 755 participants 12+ years of age 78
Slide 19: Effective Against at least Key Variants
19©2021 Ocugen. All Rights Reserved. Effective Against at least 3 Key Variants B.1.617 (India - double mutant) B.1.1.7 (United Kingdom) B.1.1.28 (Brazil P2) COVAXIN-vaccinated sera effectively neutralized several SARS-CoV-2 variants in an in-vitro plaque reduction neutralization assay The
Slide 20: Ocugen's Modifier Gene Therapy Platform
20 Ocugen’s Modifier Gene Therapy Platform Breakthrough Technology Designed to Address Multiple Diseases with One Product Approach Complex Diseases Through Multiple Factors
Slide 21: Modifier Gene Therapy vs Gene Augmentation
21©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 22: Why Target Nuclear Hormone Receptor Genes (NHRs)?
22©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 23: Preclinical POC Data for Nr2e3 Published in Nature Gene Therapy
23©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 24: OCU400 - Rescue in Early & Advanced Stage of Disease
24©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 25: OCU400 - Demonstrates Improved Vision Signals in Retina
25©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 26: OCU400 - Demonstrated Safety in Mouse Model
26©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 27: OCU400 - Clinical and Regulatory Strategy
27©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 28: OCU400 - Competitive Overview
28©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 29: OCU410 (AAV-RORA) - Dry Age-Related Macular Degeneration
29©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 30: OCU200: DME, DR and Wet AMD - Novel Biologic Offering Benefits Beyond Anti-VEGF
30 OCU200: Diabetic Macular Edema (DME) Diabetic Retinopathy (DR) Wet Age-Related Macular Degeneration (Wet AMD) Novel Biologic Offering Benefits Beyond Anti-VEGF
Slide 31: OCU200 - Potential to Treat DME, DR & Wet AMD
31©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 32: OCU200 Demonstrated Efficacy in Preclinical Models
32©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 33: OCU200 - Distinct Mechanism of Action
33©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 34: Key Inflection Points
34©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 35: 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