Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (December 2021)
36 slides from Ocugen's investor presentation, Dec 15, 2021
Slide 1: Taking Science to New Heights for Patients
Taking Science to New Heights for Patients December 2021 NASDAQ: OCGN 1
Slide 2: Forward Looking Statement
2 Forward Looking Statement This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “conti
Slide 3: Ocugen: A Diversified Portfolio Designed to Serve Unmet Needs
Ocugen: A Diversified Portfolio Designed to Serve Unmet Needs 3 Vaccine development with a COVID-19 vaccine candidate. Modifier gene therapies designed to cure multiple rare and broad diseases with one product. Novel biologic treatment targeting diabetic macular edema, diabetic retinopathy, and wet
Slide 4: Pipeline Overview
Pipeline Overview 4 * Bharat Biotech-sponsored clinical trial ** No approved therapies exist https://www.aao.org/eye-health/diseases/retinitis-pigmentosa-treatment | https://www.aao.org/eye-health/diseases/amd-treatment Asset/Program Indication Phase Vaccine COVAXIN™ (BBV152) Whole-Virion Inactivate
Slide 5: COVAXIN (BBV152) A Whole-Virion Inactivated COVID-19 Vaccine Candidate
5 COVAXIN™ (BBV152) A Whole-Virion Inactivated COVID-19 Vaccine Candidate Licensed from Bharat Biotech (BBIL) for the US and Canadian Markets
Slide 6: Forward Momentum for COVAXIN (BBV152)
Forward Momentum for COVAXIN™ (BBV152) 6 01 02 03 Results of Phase 3 clinical trial for COVAXIN™ published in The Lancet; Emergency Use Authorization submitted to FDA for pediatric (2-18) indication for the prevention of COVID-19 IND filed with FDA for Phase 3 bridging study in support of a BLA sub
Slide 7: Product Profile
Product Profile 7 Dose Level and Regimen 0.5mL per dose suspension 2 Doses: Day 0 & Day 28 Presentation Ten doses per vial Potential Shelf Life Approximately two years at 2°- 8°C and three months at room temp (25°C) Proposed indication Prevention of COVID-19 caused by SARS-CoV-2 Target population Pe
Slide 8: Why COVAXIN (BBV152)?
Why COVAXIN™ (BBV152)? Designed to augment our North American arsenal of vaccines against COVID-19 8 • Data suggest both humoral & cellular responses generated against multiple viral proteins • Data support that the vaccine induces a Th1 response (cell-mediated immunity) which can be vital for durab
Slide 9: Why COVAXIN (BBV152)? Broad Spectrum Response
Why COVAXIN™ (BBV152)? Broad Spectrum Response 9 Research suggests COVAXIN™ elicits a strong IgG responses against spike protein, receptor-binding domain, and the nucleocapsid protein of SARS-CoV-2 along with strong cellular responses Current mRNA and adenovirus-based vaccines only elicit responses
Slide 10: The Only COVID-19 Vaccine Candidate with Clinical Results Against Delta Variant
Why COVAXIN™ (BBV152)? The Only COVID-19 Vaccine Candidate with Clinical Results Against Delta Variant 10 77.8% 93.4% 65.2% Overall efficacy Efficacy vs severe disease Efficacy vs B.1.617.2 (Delta) Serious Adverse Events Adverse Events Placebo Arm Adverse Events COVAXIN™ Arm 12.4% <0.5% n = 25,800 p
Slide 11: Summary of Efficacy and Safety Results from Phase 3 Clinical Trial
Summary of Efficacy and Safety Results from Phase 3 Clinical Trial 11 Source: Ella, Reddy, Blackwelder, Potdar, Yadav, Sarangi et al. (2021) Efficacy, safety, and lot-to-lot immunogenicity of an inactivated SARS-CoV-2 vaccine (BBV152): interim results of a randomised, double-blind, controlled, phase
Slide 12: The Role of the Adjuvant in COVAXIN (BBV152)
The Role of the Adjuvant in COVAXIN™ (BBV152) 12 Expert commentary suggests adjuvant provides additional enhancement to elicit immune responses supporting broad protection Source: National Institutes of Health; June 29, 2021, https://www.nih.gov/news-events/news-releases/adjuvant-developed-nih-fundi
Slide 13: Data Suggest Th1 Mediated Response Boosted by Novel Adjuvant
13 Source: Lancet Infect Dis 2021; 21: 950–61 Published Online March 8, 2021 https://doi.org/10.1016/S1473-3099(21)00070-0 Data Suggest Th1 Mediated Response Boosted by Novel Adjuvant Induction of Th1 cell mediated immunity as measured by IFN-ƴ, IL-2, TNF-α
Slide 14: COVAXIN Phase 3 Trial: 90% of Infections by Variants
COVAXIN™ (BBV152) Phase 3 Trial: 90% of Infections by Variants 14 Active surveillance every 15 days begins 14 days after the 2nd dose N=85 Swab Samples Genome Sequenced in Central Lab 59% 5% 13% 16% 7% Delta Alpha Kappa Other Genome Not Retrieved Data on file Sequencing in central laboratory RTPCR i
Slide 15: COVAXIN Efficacy Against Variants in Phase 3 Trial
COVAXIN™ (BBV152) Efficacy Against Variants in Phase 3 Trial 15 Source: Lancet Infect Dis 2021; 21: 950–61 Published Online March 8, 2021 https://doi.org/10.1016/S1473-3099(21)00070-0 Data include per protocol population only. Efficacy estimates were only reported for at least 10 symptomatic cases.
Slide 16: COVAXIN May Help Reduce Transmission Rate from Breakthrough Infections
COVAXIN™ (BBV152) May Help Reduce Transmission Rate from Breakthrough Infections 16 Ct values All cases BBV152 Placebo mean Mean ratio of BBV152/ Placebo (95% CI) B.1.617.2 (Delta) – E gene 20.11 25.55 18.20 1.42 (1.28, 1.57) B.1.617.2 (Delta) – ORF gene 22.97 28.29 21.09 1.35 (1.24, 1.46) ~150-fold
Slide 17: Extensive Publication Portfolio of the COVAXIN Clinical Development Journey
17 Publications found at ocugen.com Extensive Publication Portfolio of the COVAXIN™ (BBV152) Clinical Development Journey
Slide 18: Modifier Gene Therapy Platform
18 MODIFIER GENE THERAPY PLATFORM Breakthrough technology designed to address many rare diseases as well as complex diseases that affect millions
Slide 19: Forward Momentum for OCU400/OCU410
Forward Momentum for OCU400/OCU410 19 01 02 03 Successfully completed manufacturing at commercial scale (200L) at CanSinoBio to support OCU400 clinical studies IND accepted by FDA for clinical trials studying OCU400 for the treatment of retinitis pigmentosa resulting from genetic mutations of NR2E3
Slide 20: Our Focus: Nuclear Hormone Receptor Genes (NHRs)
Our Focus: Nuclear Hormone Receptor Genes (NHRs) 20 *References: https://pubmed.ncbi.nlm.nih.gov/28556246/ | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5409218/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4339951/ | https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0183526 Photore
Slide 21: Our Vision: Modifier Gene Therapy vs Traditional Gene Augmentation
Our Vision: Modifier Gene Therapy vs Traditional Gene Augmentation 21 Normal gene X Gene Augmentation: Transfer functional version of a non-functional gene into the target cells. Modifier Gene Therapy: Designed to introduce a functional gene to modify the expression of many genes, gene-networks and
Slide 22: Our Proof of Principle: Published in Nature Gene Therapy
Our Proof of Principle: Published in Nature Gene Therapy 22 https://www.nature.com/articles/s41434-020-0134-z Important milestone for development of therapy; demonstrated proof of principle Protection elicited in multiple animal models of degeneration caused by different mutations Potential to repre
Slide 23: Data Show How OCU400 Stops Disease Progression and Rescues Vision
Data Show How OCU400 Stops Disease Progression and Rescues Vision in Both Early and Advanced Stages 23 Early-Stage Rescue Advanced Stage Rescue P21 subretinal injection, evaluation 2–3 months post injection Restored outer nuclear layer (ONL) photoreceptors morphology in rd7 ONL cell layer change in
Slide 24: OCU400 Demonstrates Improved Vision Signals in Retina
OCU400 Demonstrates Improved Vision Signals in Retina 24 ERG response: P0 single subretinal injection, evaluation 3-4 months post injection How these data matter: Human vision is enabled by three primary modes Electroretinogram (ERG) Response Reveals Rescue under Both Scotopic (dim-lit) as well as P
Slide 25: OCU400 Demonstrated Safety in Mouse Model
OCU400 Demonstrated Safety in Mouse Model 25 Study results confirm overexpression of Nr2e3 by subretinal AAV8-Nr2e3 injection is not detrimental to retina creating no off-target effects https://www.nature.com/articles/s41434-020-0134-z
Slide 26: OCU400 – Clinical and Regulatory Strategy
OCU400 – Clinical and Regulatory Strategy Planned timeline 26 Phase 3 Potential ApprovalPhase 1/2 2021 - 2022 2023 - 2025 2025/26 Proposed Broad RP Indication RHO CEP290 NR2E3NR2E3 RHO Phase 4 Commitments OCU400 NR2E3 Mutation-Associated Retinal Disease Rhodopsin Mutation-Associated Retinal Disease
Slide 27: OCU400 – Competitive Overview
OCU400 – Competitive Overview 27 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 Potentially lo
Slide 28: OCU410 (AAV-RORA): Dry Age-Related Macular Degeneration
OCU410 (AAV-RORA): Dry Age-Related Macular Degeneration 28 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 Normal Retina Contributing Factors • Aging • Genetics • Environmental Factors Dry AMD We
Slide 29: OCU200 Novel biologic for DME, DR and Wet AMD
29 OCU200 Novel biologic for treating Diabetic Macular Edema (DME), Diabetic Retinopathy (DR) and Wet Age-Related Macular Degeneration (Wet AMD)
Slide 30: OCU200: Potential to Treat DME, DR & Wet AMD
DME DR Wet AMD OCU200: Potential to Treat DME, DR & Wet AMD OCU200 is a Transferrin-Tumstatin Fusion Protein • Tumstatin: Multiple MOAs for treatment and prevention of macular degeneration and neovascularization • Transferrin: Targets the site of action and improves uptake (better target engagement)
Slide 31: OCU200 Demonstrated Superior Efficacy Compared to Existing Anti-VEGF Therapies
31 OCU200 Demonstrated Superior Efficacy Compared to Existing Anti-VEGF Therapies Effect of OCU200 intravitreal treatments on Neovascularization (NV). Data are presented as mean± SD. Filled circles represent data points from individual eyes * P < 0.05, ** P < 0.01 (n = 9-10 eyes per group) * indicat
Slide 32: Leadership and Scientific Advisors
32 Leadership and Scientific Advisors
Slide 33: Leadership Team
Leadership Team = Shankar Musunuri, PhD, MBA Sanjay Subramanian, MBA Bruce D. Forrest, MB, BS, MD, MBA 4. Gabriel Chairman, CEO and Co-Founder (CFO and Head of Corporate Development ‘Acting CMO SVP, Manufacturing & Supply Chain bp VijayTammara, PhD Michael Shine, MBA ‘Arun Upadhyay, PhD Jessica Cres
Slide 34: Scientific Advisory Boards
Scientific Advisory Boards 34
Slide 35: Forward Momentum for Ocugen
IND accepted by FDA for clinical trials studying OCU400 for the treatment of retinitis pigmentosa resulting from genetic mutations of NR2E3 and RHO Successfully completed manufacturing at commercial scale (200L) at CanSinoBio to support clinical studies Expanded manufacturing agreement with CanSinoB
Slide 36: Taking Science to New Heights for Patients
Taking Science to New Heights for Patients 36 December 2021 NASDAQ: OCGN