Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (May 2023)
23 slides from Ocugen's investor presentation, May 5, 2023
Slide 1: Courageous Innovation May 2023
Courageous Innovation May 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 Gene therapies OCU400 * AAV-hNR2E3 Gene mutation-associated retinal degeneration* Retinitis pigmentosa (RP)--NR2E3 Mutation • Phase 1/2 • Favorable safety and tolerability profile • Initial clinical data from low- and medium-dose cohorts in
Slide 5: Modifier Gene Therapy Platform
5 Modifier Gene Therapy Platform Breakthrough technology designed to address many rare diseases as well as complex diseases that affect millions
Slide 6: Modifier Gene Therapy: A Broader Reach
6 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 7: 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 8: Study Overview
Study Overview Clinical Trials.gov Identifier: NCT05203939 Safety of subretinal administration of OCU400 Best Corrected Visual Acuity (BCVA) Multi-Luminance Mobility Test (MLMT) Primary Endpoint: Safety Exploratory Endpoint: Efficacy 38 Enrollment Status
Slide 9: Multi-Luminescence Mobility Test
Multi-Luminescence Mobility Test Total Subjects for analyses (N=7); pooled analyses Subjects with 9-months follow-up : Cohort 1, N=3 Subjects with 6 months follow-up: N=1 from Cohort 1 and N=3 from Cohort 2 Total Subject for analyses (N=3) Cohort 1 with 9 month follow-up Improvement ≥ 1 Lux Improvem
Slide 10: Best Corrected Visual Acuity (BCVA) Score
Best Corrected Visual Acuity (BCVA) Score Total Subjects for analyses (N=7) Subjects with 9-months follow-up : Cohort 1, N=3 Subjects with 6 months follow-up: N=1 from Cohort 1 and N=3 from Cohort 2 Improvement ≥ 8 Letters Treated Eye 42.9% Untreated Eye 0.0% 10
Slide 11: OCU400: Expected Pathway to Clinical Development & Potential Approval
OCU400: Expected Pathway to Clinical Development & Potential Approval Both FDA & EMA granted broad orphan drug designation for RP & LCA • Ocugen plans to meet with regulatory agencies in 3Q to potentially finalize Phase 3 clinical program and overall package • Continuing to enroll LCA and pediatric
Slide 12: OCU410ST: Received ODD for ABCA4-Associated Retinopathies
12 OCU410ST: Received ODD for the Treatment of ABCA4-Associated Retinopathies Including Stargardt, Retinitis Pigmentosa 19 (RP19) and Cone-rod Dystrophy 3 (CORD3) ABCA4-associated retinopathies—Genetic Rare Disease • ABCA4 gene produces an ATP-binding cassette (ABC) superfamily transmembrane protein
Slide 13: OCU410 for the Treatment of Dry Age-related Macular Degeneration
13 OCU410 for the Treatment of Dry Age-related Macular Degeneration (dAMD) dAMD Limited options, presenting significant unmet medical need • US: 10M • Worldwide: condition affects more than 266M people Recently approved therapy for geographic atrophy (GA)— advanced form of dAMD–has limitations • Fre
Slide 14: 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 15: 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 16: OCU200 Novel biologic for DME, DR and Wet AMD
16 OCU200 Novel biologic for treating Diabetic Macular Edema (DME), Diabetic Retinopathy (DR) and Wet Age-Related Macular Degeneration (Wet AMD)
Slide 17: OCU200 is our novel biologics candidate for sight-threatening conditions
17 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 18: NeoCart
18 NeoCart® (Autologous chondrocyte-derived neocartilage)
Slide 19: NeoCart is a regenerative cell therapy technology
19 *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 auto
Slide 20: OCU500 Series
20 OCU500 Series: OCU500: COVID-19 Mucosal Vaccine OCU510: Flu OCU520: COVID-19/Flu
Slide 21: OCU500 Series: Next-Generation Vaccine Candidates Using Inhalation Technology
21 OCU500 Series: Next-Generation Vaccine Candidates Using Inhalation Technology OCU500 A bivalent COVID-19 vaccine OCU510 A seasonal quadrivalent flu vaccine OCU520 A combination quadrivalent flu and bivalent COVID19 vaccine Current focus solely on the development of our inhaled mucosal vaccine pla
Slide 22: Ocugen Vision
22 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 23: ocugen
ocugen. ~~ May 2023 NASDAQ: OCGN