Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (March 2023)
25 slides from Ocugen's investor presentation, Mar 13, 2023
Slide 1: Courageous Innovation March 2023
Courageous Innovation March 2023 NASDAQ: OCGN
Slide 2: Forward Looking Statements
Forward Looking Statements 2 This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are based on the beliefs and assumptions of Ocugen, Inc. and on information currently available to management. All statements contained
Slide 3: We're Here to Make an Impact Through Courageous Innovation
We’re Here to Make an Impact Through Courageous Innovation 3 Mission: Developing cutting-edge innovations for people facing serious disease and conditions with a commitment to ensuring global market access Pioneering modifier gene therapy for inherited retinal diseases, as well as larger blindness d
Slide 4: Pipeline Overview
4 Pipeline Overview Asset/Program Indication Current Status Cell therapies (Regenerative Medicine) NeoCart® (Autologous chondrocyte-derived neocartilage) RMAT* Treatment of Articular Cartilage Defects in the Knee • Phase 3 clinical trial is planned for 1H 2024 Gene therapies OCU400 ** AAV-hNR2E3 Gen
Slide 5: NeoCart (Autologous chondrocyte-derived neocartilage)
5 NeoCart® (Autologous chondrocyte-derived neocartilage)
Slide 6: NeoCart regenerative cell therapy for knee cartilage damage
6 *The Journal of Bone & Joint Surgery: June 1, 2011 - Volume 93 - Issue 11 - p 994-1000 **https://www.biospace.com/article/cell-therapy-market-size-cagr-trends-forecast-report-2022-2030/ NeoCart is a regenerative cell therapy technology • Combines bioengineering and cell processing to enhance autol
Slide 7: Modifier Gene Therapy Platform
7 Modifier Gene Therapy Platform Breakthrough technology designed to address many rare diseases as well as complex diseases that affect millions
Slide 8: Modifier Gene Therapy: A Broader Reach
8 Modifier Gene Therapy: A Broader Reach Gene modifier therapy can potentially address multiple genetic defects with a single product utilizing a gene agnostic approach. In patients with IRDs, this could mean: Improved visual outcomes and quality of life GENE X GENE M cell NR2E3 Delivery “Molecular
Slide 9: FDA granted expanded Orphan Drug Designations for RP and LCA
FDA granted expanded Orphan Drug Designations for all retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) mutations Despite its prevalence, RP and LCA patients have limited treatment options • US: RP & LCA affect 110,000 and 15,000 people, respectively • Worldwide: conditions affect appro
Slide 10: OCU400-101 Clinical Program
A Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa associated with NR2E3 and RHO mutations and Leber Congenital Amaurosis with mutation(s) in CEP290 gene Clinical Trials.gov Identifier: NCT05203939 Safety of subretinal administration of OCU400 Best Corrected Visua
Slide 11: OCU400: Expected Pathway to Clinical Development & Potential Approval
11 OCU400: Expected Pathway to Clinical Development & Potential Approval RP LCA Proposed Indication RP & LCA Both FDA & EMA granted broad orphan drug designation for RP & LCA
Slide 12: OCU410 for Dry AMD and Stargardt disease
Macular Degeneration Age related GA Drusen Deposit Old age STGD ABCA4 mutation Fatty Deposits Early Age Retina Degeneration Vision Loss Genetic Drusen Fatty Deposits 12 Dry AMD Limited options, presenting significant unmet medical need • US: 10M • Worldwide: condition affects more than 266M people S
Slide 13: AMD: Risk Factors, Treatment Options and Unmet Needs
Ap pr oa ch es b ei ng in ve st ig at ed Antioxidants Complement cascade inhibitors Neuroprotective Visual cycle inhibitors Gene therapy Cell-based therapies A strong role of inflammation, complement, and oxidative pathways in AMD Pegcetacoplan (Syfovre) - approved Lampalizumab Zimura sCD59 AREDS AR
Slide 14: OCU410 (RORA): A Potential Modifier Therapeutic for Dry-AMD and STGD
50 60 70 80 90 1e61E51E41E31E2Cells N or m al ize d Ce ll Su rv iv al RORA NR2E3 NR1D1 Anti-oxidative: Improves ARPE19 cells survival Anti-complement: Increased anti-compliment (Cd59) protein Anti-inflammatory: Suppresses inflammation in HMC3 cellsAnti-drusen activity and improves retinal function 0
Slide 15: OCU200 Novel biologic for DME, DR and Wet AMD
15 OCU200 Novel biologic for treating Diabetic Macular Edema (DME), Diabetic Retinopathy (DR) and Wet Age-Related Macular Degeneration (Wet AMD)
Slide 16: OCU200 novel biologics candidate for sight-threatening conditions
16 OCU200 is our novel biologics candidate for sight-threatening conditions • A recombinant fusion protein of transferrin and tumstatin • Potential to address diabetic macular edema (DME), diabetic retinopathy (DR), wet AMD High prevalence of DME, DR and wet AMD patients • DME: 21M worldwide • DR: 1
Slide 17: OCU500 Series: COVID-19 Mucosal Vaccine, Flu, COVID-19/Flu
17 OCU500 Series: OCU500: COVID-19 Mucosal Vaccine OCU510: Flu OCU520: COVID-19/Flu
Slide 18: Next-Generation Vaccine Candidates Using Inhalation Technology
18 OCU500 A bivalent COVID-19 vaccine OCU510 A seasonal quadrivalent flu vaccine OCU520 A combination quadrivalent flu and bivalent COVID19 vaccine Next-Generation Vaccine Candidates Using Inhalation Technology to Potentially Overcome Durability and Transmission Challenges COVID-19 and flu infection
Slide 19: Factors Responsible for the Rapid Decline in Vaccine Efficacy
• Multiple RBD mutations > less cross-protection • Change in infection and pathogenesis pattern > reduced latency period • Reduce cellular memory > limited tissue-resident memory T-cells Limited local immunity at site of infection • Decrease in neutralizing IgG antibody levels • Lack of a robust sec
Slide 20: Strategies to Improve COVID-19 and Flu Vaccines
20 • Maximizing the area of vaccine delivery to the respiratory tract (Intranasal vs. Inhalation) • Formulations to facilitate the adsorption and prolong the retention time on respiratory epithelial cells • Delivery and uptake of antigens to target APC cells such as M-cells, DCs, and macrophages Enh
Slide 21: Approved (Ex-U.S.) Mucosal COVID-19 Vaccines
21 Approved (Ex-U.S.) Mucosal COVID-19 Vaccines: Demonstrated Safety & Efficacy as a Heterologous Booster Studies demonstrating the benefit of AAV Improved Immunogenicity in Ph3: iNCOVACC vs. COVAXIN • Superior GMT ratio of nAbfor Wuhan (1.45) • Superior GMT ratio of nAbfor OmicronBA.5 (2.1) • GMT r
Slide 22: COVAXIN (BBV152)
22 COVAXIN™ (BBV152) A whole-virion inactivated COVID-19 vaccine candidate licensed from Bharat Biotech (BBIL) for North American Markets
Slide 23: COVAXIN: Final Data and Analysis Expected Mid-Year 2023
23 COVAXIN™(BBV152): Final Data and Analysis Expected Mid-Year 2023 for Our Injectable COVID-19 Vaccine Enrollment completed for Phase 2/3 immuno-bridging and broadening clinical trial in December 2022 Topline data highlights, reported in January 2023, include the following: • Safety: well-tolerated
Slide 24: Ocugen Vision
24 Ocugen™ Vision Fully integrated, patient-centric biotech company focused on vaccines in support of public health and gene and cell therapies targeting unmet medical needs through Courageous Innovation
Slide 25: ocugen
————_ ~ ocugen. > ee March 2023, NASDAQ: OCGN