Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Corporate Update (April 2023)
12 slides from Ocugen's corporate update, Apr 14, 2023
Slide 1: Ocular Modifier Gene Therapy Program Update
Ocular Modifier Gene T herapy Program Update A PHASE 1/2 STUDY TO ASSESS THE SAFETY AND EFFICACY OF OCU400 (MODIFIER GENE THERAPY) FOR RETINITIS PIGMENTOSA ASSOCIATED WITH NR2E3 AND RHO MUTATIONS AND LEBER CONGENITAL AMAUROSIS WITH MUTATION(S) IN CEP290 GENE
Slide 2: Forward Looking Statements
2 Forward Looking Statements 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, including, but not limited to, statements regarding the development of OCU400 and the interpr
Slide 3: Today's Agenda
Today’s Agenda Opening Shankar Musunuri, PhD, MBA Chairman, CEO and Co - founder, Ocugen Overview of Preliminary Arun Upadhyay, PhD Safety and Efficacy Results Chief Scientific Officer, Head of Research, Development and Medical, Ocugen Closing Shankar Musunuri, PhD, MBA Q&A • Huma Qamar, MD, MPH, He
Slide 4: Modifier Gene Therapy: A Broader Reach
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 re
Slide 5: Study Overview
Study Overview Clinical Trials.gov Identifier: NCT05203939 Safety of s ubretinal a dministration of OCU400 Best Corrected Visual Acuity (BCVA) Multi - Luminance Mobility Test (MLMT) Primary Endpoint : Safety Exploratory Endpoint : Efficacy 3 5 Enrollment Status
Slide 6: Multi-Luminance Mobility Test
Multi - Luminance Mobility Test 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 Cohor1 and N=3 from Cohort 2 Improvement ≥ 1 Lux Improvement ≥ 2 Lux Treated Eye 71.4% 28.6% Untreat ed Eye 28.6% 0.0% • 100% of treated
Slide 7: 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 Cohor1 and N=3 from Cohort 2 Improvement ≥ 8 Letters Treated Eye 42.9% Untreat ed Eye 0.0% 7
Slide 8: OCU410: 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 OCU410: Dry Age - related Macular Degeneration ( dAMD ) and Stargardt Disease (STGD) Dry AMD Limited options, presenting significant u
Slide 9: AMD: Risk Factors, Treatment Options and Unmet Needs
Approaches being investigated 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 AREDS2 (f
Slide 10: OCU410 (RORA): A Potential Modifier Therapeutic for Dry-AMD and STGD
50 60 70 80 90 1e6 1E5 1E4 1E3 1E2 Cells Normalized Cell Survival RORA NR2E3 NR1D1 Anti - oxidative: Improves ARPE19 cells survival Anti - complement: Increased anti - compliment (Cd59) protein Anti - inflammatory: Suppresses inflammation in HMC3 cells Anti - drusen activity and improves retinal fun
Slide 11: Q&A
Q&A
Slide 12: Ocugen Vision
12 Ocugen Ρ Vision Fully integrated, patient - centric b iotech company focused on vaccines in support of public health and gene and cell therapies targeting unmet medical needs through Courageous Innovation