Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Day (February 2024)
27 slides from Ocugen's investor day presentation, Feb 21, 2024
Slide 1: Courageous Innovation Clinical Showcase
Courageous Innovation Clinical Showcase February 21, 2024 Dedicated to Bringing Game-Changing Gene & Cell Therapies and Vaccines to Market and Working Even Harder to Provide Access to Patients Globally
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 subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “cont
Slide 3: Agenda
3 Agenda Opening: Dr. Shankar Musunuri, Chairman, CEO & Co-founder, Ocugen Modifier Gene Therapy Platform & Program Update: Dr. Arun Upadhyay, CSO & Head of R&D, Ocugen Modifier Gene Therapy Market Potential: Mike Shine, SVP Commercial, Ocugen Q&A 10-minute Break Panel: Modifier Gene Therapy & OCU40
Slide 4: We're Here to Make an Impact Through Courageous Innovation
4 We’re Here to Make an Impact Through Courageous Innovation Founded 2013 Headquarters Malvern, PA Employees ~61 (U.S.) Ticker Symbol OCGN R&D Center Hyderabad, India Company Overview Our Values • Respect • Integrity • Teamwork • Accountability
Slide 5: Leveraging First-in-Class Platforms to Address Serious Unmet Medical Needs
Through Courageous Innovation, We are Leveraging Our First-in-Class Platforms to Address Serious Unmet Medical Needs Modifier Gene Therapy Platform First-in-Class • Therapeutic Focus: inherited retinal diseases and larger blindness diseases with unmet need • Differentiator: master gene regulator; ge
Slide 6: Entering a Year of Execution; New $7 PT; Reiterate Buy
6 Entering a Year of Execution; New $7 PT; Reiterate Buy Raising Price Target to $8 Based On Clinical Progress Execution of High Value Gene Therapies Will Increase Valuation Key Gene Therapy Milestones Achieved in 2023 • First gene therapy program to get alignment with FDA for broad RP indication in
Slide 7: Ocugen's Gene Therapy – Fundamentally Changing Gene Therapy
Ocugen’s Gene Therapy – Fundamentally Changing Gene Therapy 20th Century Disruptive Biotechnologies • Penicillin • Polio vaccine • Recombinant DNA • Production of monoclonal antibodies • Genetically engineered bacteria 21st Century Biotechnology Innovations • Human Genome fully sequenced • Gene and
Slide 8: Modifier Gene Therapy is a First-in-Class Platform Technology
Ocugen’s Modifier Gene Therapy is a First-in-Class Platform Technology Designed to Restore Homeostasis and Preserve Vision Modifier Gene Therapy Platform First-in-Class • Therapeutic Focus: Inherited retinal diseases and larger blindness diseases with unmet need • Differentiator: “Master Gene Regula
Slide 9: Retinitis Pigmentosa: Inherited Retinal Disease with a High Unmet Need
Retinitis Pigmentosa: Inherited Retinal Disease with a High Unmet Need 110,000 RP Retinitis Pigmentosa (RP) • RP is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina • RP is associated with mutations in more than 100 genes Leber Congenital Amaurosis (LCA) •
Slide 10: OCU400: Novel Gene-Agnostic Modifier Gene Therapy for RP and LCA
OCU400: A Novel Gene-Agnostic Modifier Gene Therapy for RP and LCA 1 FDA & EMA granted expanded Orphan Drug Designations for all RP and LCA mutations FDA granted RMAT designation for RP indication associated with NR2E3 and RHO mutations OCU400 addresses shortcomings of current gene therapy approache
Slide 11: Ph 1/2 Safety and Efficacy Summary
• OCU400 continued to be generally safe and well-tolerated in subjects across different mutations and dose levels • Efficacy measurements suggest positive trends in Best-Corrected Visual Acuity (BCVA) and Multi-Luminance Mobility Testing (MLMT), and Low-Luminance Visual Acuity (LLVA) among treated e
Slide 12: Phase 3 Study of Subretinal OCU400 Gene Therapy for Retinitis Pigmentosa
A PHASE 3, MULTI-CENTER, RANDOMIZED STUDY TO ASSESS THE EFFICACY, SAFETY AND TOLERABILITY OF SUBRETINAL OCU400 GENE THERAPY FOR THE TREATMENT OF RETINITIS PIGMENTOSA
Slide 13: RP and LCA—Unmet need and Treatment Benefit Target
RP and LCA—Unmet need and Treatment Benefit Target 8 • IRDs, such as RP and LCA, are a group of heterogenous genetic disorders that affect the retina, the light-sensitive tissue at the back of the eye • They often lead to a gradual loss of vision over time and can ultimately result in blindness • St
Slide 14: Phase 3 Study Design
14 Phase 3 Study Design LDNA: Luminance Dependent Navigation Assessment Study Design Population • Patient with ≥8 years of age with Clinical and Molecular Diagnosis of Retinitis Pigmentosa Key Eligibility Criteria • BCVA ≤ 75 letters and ≥35 letters (ETDRS Chart) • Able to perform LDNA at ≤ 500 Lux
Slide 15: Phase 3 Study: Statistical Considerations
15 Phase 3 Study: Statistical Considerations Sample Size (N=150); Two Arm ( RHO and Gene Agnostic) Population • Each arm will enroll 75 participants • In each arm, participants (N=75) will be randomized 2:1 to the Treatment group (2.5 x 1010 vg/eye of OCU400) and Untreated Control group • This sampl
Slide 16: Phase 1/2 Patients Meeting Intent to Treat Criteria for Phase 3
Phase 1/2 Patients Population Meeting “Intent to Treat Population” Criteria for Phase 3 Study* *RHO +AR-NR2E3 Subjects (- Adverse Events, Sentinel); and Ceiling Effect (RHO) Subjects; ^ ceiling effect (AR-NR2E3) # AR-NR2E3 Subjects: Baseline MLMT at 5 Lux level; 1Lux level improvement resulted in ce
Slide 17: OCU400: Demonstrate Gene Agnostic Effect in RHO Patients
OCU400: Demonstrate Gene Agnostic Effect in RHO Patients Showed stabilization or improvement on both the parameters from Baseline Stabilization: LLVA: ± 4 letters change MLMT: 0 change in Lux Level Improvement: LLVA: ≥ 5 letters change MLMT: ≥1 change in Lux Level 17*Primary Endpoint: Proportion of
Slide 18: OCU400: Expected Pathway to Clinical Development & Potential Approval
RP LCA Adult & Pediatric • Both FDA & EMA granted broad orphan drug designation for RP & LCA • Received RMAT designation from FDA RHO NR2E3 CEP290 Indication Expect to pursue LCA indication in 2H 2024 Treatment of retinitis pigmentosa (RP) In December 2023, received alignment from FDA on key aspects
Slide 19: OCU400 RP – U.S. & EU Market Potential
OCU400 RP – U.S. & EU Market Potential • First five years projected penetration 3% - 15% • 5-year revenue: $47B projection* 0 2 4 6 8 10 12 14 16 18 20 2026 2027 2028 2029 2030 $ Bi lli on s Year Revenue 19 *Internal modelling based on: 1) Wong, C.H., Li, D., Wang, N. et al. The estimated annual fin
Slide 20: OCU400 RP – U.S. & EU Market Potential (Downside)
OCU400 RP – U.S. & EU Market Potential • First five years projected penetration 3% - 15% • 5-year revenue: $47B projection • Downside: $30B* 0 2 4 6 8 10 12 14 16 18 20 2026 2027 2028 2029 2030 $ Bi lli on s YearRevenue Revenue Downside 20 Downside assumptions: • Price assumption U.S.: $425K per dos
Slide 21: OCU410 GA Market Potential
OCU410 GA Market Potential • First five years projected penetration U.S.: 2% - 8% and EU: 1% - 4% • First 5-years cumulative revenue projection: $75B* 0 5 10 15 20 25 2027 2028 2029 2030 2031 $ Bi lli on s Year Revenue• Price assumption U.S.: $225K per dose ($450k per patient) • Price assumption EU:
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: Dr. Ramakanth, H.C. Wainwright
23 Dr. Ramakanth is a Managing Director of Equity Research at H.C. Wainwright whose research focuses on the healthcare sector. His research covers companies operating in oncology, wound health, medical devices, spine health and AI Drug Discovery. Dr. Ramakanth possesses over twenty years of experien
Slide 24: Neena Haider, PhD
Neena Haider, PhD Dr. Haider is the founder of Shifa Precision, a world-renowned geneticist, and visionary scientist who has brought multiple gene therapies to the clinic. She has served/serves in numerous leadership roles and steering committees at institutions including Harvard Medical School, Nat
Slide 25: Byron L. Lam, MD
Byron L. Lam, MD Dr. Lam is the Mark J. Daily Professor at the Bascom Palmer Eye Institute, University of Miami Miller School of Medicine. Dr. Lam is a clinical scientist with expertise and translational research experience in the areas of hereditary retinal disease, neuro- ophthalmology, and ophtha
Slide 26: Lejla Vajzovic, MD, FASRS
Lejla Vajzovic, MD, FASRS Dr. Vajzovic is a vitreoretinal surgeon and tenured Associate Professor of Ophthalmology at Duke University School of Medicine with expertise in adult and pediatric retinal diseases and surgery. She serves as a principal investigator for numerous national clinical trials in
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