Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (January 2021)
26 slides from Ocugen's investor presentation, Jan 5, 2021
Slide 1: Our Mission is to Develop Gene Therapies to Cure Blindness Diseases and a COVID-19 Vaccine
Our Mission is to Develop Gene Therapies to Cure Blindness Diseases and Develop a Vaccine to fight COVID-19 NASDAQ: OCGN Corporate Deck: January 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. Developing transformative therapies for blindness diseases and a vaccine for COVID-19 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 Nat
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: Pipeline Overview
5©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 * * * * * Program Indication Prevalence (US) Discovery Preclinical IND-Enabling Phase 1/2 N
Slide 7: COVAXIN Whole-Virion Inactivated COVID-19 Vaccine Being Co-Developed for the US Market
6 COVAXIN™ Whole-Virion Inactivated COVID-19 Vaccine Being Co-Developed for the US Market
Slide 8: COVID-19 Vaccine in Development for US Market
7©2021 Ocugen. All Rights Reserved. COVID-19 Vaccine in Development for US Market Evaluated in about 1,000 subjects in Phase 1 & 2 clinical trials in India. Phase 3 trial underway in India involving about 26,000 volunteers Promising safety and immunogenicity demonstrated by the Phase 1 and Phase
Slide 9: 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
Slide 10: Modifier gene therapy approach targeting nuclear hormone receptor genes
9©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 indi
Slide 11: Why Target Nuclear Hormone Receptor Genes (NHRs)?
10©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 12: Preclinical POC Data for Nr2e3 Published in Nature Gene Therapy
11©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 13: OCU400 – Rescue in Early & Advanced Stage of Disease
12©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 14: OCU400 – Demonstrates Improved Vision Signals in Retina
13©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 15: OCU400 – Demonstrated Safety in Mouse Model
14©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 16: OCU400 – Clinical and Regulatory Strategy
15©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 17: OCU400 – Competitive Overview
16©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 18: OCU410 (AAV-RORA) – Dry Age-Related Macular Degeneration
17©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 19: CanSinoBIO Manufacturing Partnership
18©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 20: OCU200: Novel Biologic Offering Benefits Beyond Anti-VEGF
19 OCU200: Diabetic Macular Edema (DME) Diabetic Retinopathy (DR) Wet Age-Related Macular Degeneration (Wet AMD) Novel Biologic Offering Benefits Beyond Anti-VEGF
Slide 21: OCU200 – Potential to Treat DME, DR & Wet AMD
20©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 22: OCU200 – Transferrin-Tumstatin Fusion Protein
21©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 23: OCU200 – Distinct Mechanism of Action
22©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 24: Near & Mid-Term Milestones
23©2021 Ocugen. All Rights Reserved. Near & Mid-Term Milestones: Planned Timeline 1H 2021 2H 2021 1H 2022 2H 2022 Expected -Orphan Drug Designation in EMA IND Filing Phase 1/2a start-US Phase 1/2a start-EU Safety & Efficacy signal Tox study initiation IND Filing Start Ph 1/2a Safety & Efficacy signa
Slide 25: Investment Highlights
24©2021 Ocugen. All Rights Reserved. Investment Highlights Modifier Gene Therapy Platform has the potential for one product to treat many diseases OCU200 novel biologic has the potential to treat anti-VEGF /corticosteroids non-responders (~50% of the patients) Multiple near and mid-term milestone
Slide 26: A Bold Vision to Cure Blindness Diseases and Offer a Differentiated Vaccine for COVID-19
A Bold Vision to Cure Blindness Diseases and Offer a Differentiated Vaccine for COVID-19 in the US For more information, contact: IR@ocugen.com