Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (December 2024)
25 slides from Ocugen's investor presentation, Dec 3, 2024
Slide 1: Courageous Innovation
—_ ~ ocugen. > ee Courageous Innovation Dedicated to Bringing Game-Changing Gene & Cell Therapies and Vaccines to Market and Working Even Harder to Provide Access to Patients Globally OCGN December 2024
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical
Slide 3: Leveraging Our 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 Nadifi 7 4 wry) erapeutic Focus: Inherited retinal diseases & larger blindness Coates ‘ Gene Thera py diseases with unmet need Elation + Differentiator: Master gene regulator; gene-a
Slide 4: We're Here to Make an Impact Through Courageous Innovation
We’re Here to Make an Impact Through Courageous Innovation Company Overview Founded 2013 HQ & Manufacturing Malvern, PA Employees ~100 Ticker Symbol OCGN India Business Center Hyderabad Market Cap $300 million Our Values ocugen Respect 5 ayaa Integrity Teamwork Accountability ocugen. <<
Slide 5: Modifier Gene Therapy Platform
Modifier Gene Therapy Platform Breakthrough technology designed to address many rare diseases as well as complex diseases that affect millions — ocugen. 5 —
Slide 6: Traditional Gene Therapy is Highly Inefficient for Retinal Diseases
Traditional Gene Therapy is Highly Inefficient for Retinal Diseases Red-green colour _First Retinitis Pigmentosa Human Whole exome and whole vision deficit gene (RHO) discovered - Genome ‘genome sequencing gene Southern Blotting Linkage and Sanger sequence discovery structural sequencing of candidat
Slide 7: Control Untreated Mutant Treated Mutant
• • Control Untreated Mutant Treated Mutant
Slide 8: The Photoreceptor is Dependent on Cellular Homeostasis Regulated by Key Transcription Factors
The Photoreceptor is Dependent on Cellular Homeostasis Regulated by Key Transcription Factors (TFs) such as NR2E3(OCU400) Untreated — RP Treated -OCU400 (Rhomutation) os ONL ONL INL INL RP Impact on the Photoreceptor Recovery after treatment with OCU400 Normal Photoreceptor + Immunolabeling of rod p
Slide 9: OCU400 Modifier Gene Therapy — A Paradigm Shift in Gene Therapy
OCU400 Modifier Gene Therapy — A Paradigm Shift in Gene Therapy Prevalence of RP: + Nearly 310,000 patients in the U.S., EU, & Canada * ~1.6M patients globally rrently approved treatment for RP: * Luxturna® was FDA approved based on a mobility test and has achieved the following responder rate in Ph
Slide 10: Mobility Test (LuxIDNA) is Primary Endpoint for Phase 3
Mobility Test (LDNA*) is Primary Endpoint for Phase 3 * Primary endpoint (Efficacy): Mobility test has been used for an approved product * Alignment with FDA to demonstrate clinical efficacy (Responder 2 2 Lux level improvement) - Validated for RP patients * Phase 1/2 results: More than 60% of the I
Slide 11: OCU410: Addressing Shortcomings of Current Approaches
OCU410: Addressing Shortcomings of Current Approaches evalence of G, ~2-3 million people in the U.S. & EU + ~8M patients globally Currently approved treatment for GA: * Focused only on the complement system * Requires frequent intravitreal injections (~6-12 doses per year) * Structural effect only,
Slide 12: OCU410 Phase 1/2 Study: Design and Key Criteria
OCU410 Phase 1/2 Study : Design and Key Criteria Patients with GA secondary to Dry AMD = — Up to 63 subjects Key Inclusion Criteria * Age 250 years + BCVA 221 letters ETDRS + GAlesion requirements: Phase 2 + Total GAarea 22.0 mm2and $20.5 mm?; if multifocal, Dose expansion at least one lesion must b
Slide 13: 0.96 ± 0.25 0.76 ± 0.53 Inset • • •
0.96 ± 0.25 0.76 ± 0.53 Inset • • •
Slide 14: OCU410 Treatment Preserves Retinal Tissue in GA Lesions
OCU410 Treatment Preserves Retinal Tissue in GA Lesions — ocugen. — 024 ° 2 2 a oe x i ° 3 8 Difference in Mean GA Lesion Size Between Treated and Untreated Eyes (mm?) ° ro) iS 8 3 ° 3 8 OCU410 low dose treatment ia i preserves more retinal tissue around the GA lesions of treated eyes at 6M compared
Slide 15: -55 -50 -45 -40 -35 -30 -25 -20 -15 -10 -5 SCREENING 3 MONTHS
-55 -50 -45 -40 -35 -30 -25 -20 -15 -10 -5 0 5 10 SCREENING 3 MONTHS 6 MONTHS LL VA c ha ng e (∆ )f ro m b as el in e Study Duration (Months) 006-001 Treated 006-001 Untreated 007-001 Treated 007-001 Untreated 006-004 Treated 006-004 Untreated ±
Slide 16: Conclusions
Conclusions OCU410 demonstrated a favorable safety and tolerability profile * NoSAEs or AEs deemed related to study drug or study procedure were reported including ischemic optic neuropathy, vasculitis, endophthalmitis, and choroidal neovascularization * OCU410 treated eyes showed a 21.4% decrease i
Slide 17: OCU410ST: A Single-Injection Approach to Address Unmet Need
OCU410ST: A Single-Injection Approach to Address Unmet Need Prevalence of Stargardt disease: ~100,000 people in the U.S. and Europe OCU410ST status/upcoming anticipated catalysts: Data and Safety Monitoring Board (DSMB) approved enrollment for Phase 2 of the OCU410ST Phase 1/2 clinical trial 9- and
Slide 18: Overall Safety — Phase 1 Trial
Overall Safety — Phase 1 Trial * OCU4I10ST demonstrated a favorable safety and tolerability profile ¢ Nostudy drug or study procedure related SAEs (deemed related to study drug or study procedure) were reported including: * Nodevelopment of exudation * Noinfectious endophthalmitis * Nointraocular In
Slide 19: M e a n C h a n g e in T o ta
M e a n C h a n g e in T o ta l A tr o p h y A re a f ro m B a s el in e (m m 2 ) 84% 0.12 ± 0.36 0.73 ± 0.26
Slide 20: OCU410ST Stabilizes or Improves Retinal Structure and Visual Function
OCU410ST Stabilizes or Improves Retinal Structure and Visual Function Parameters for Assessment Low1 Low2 Low3. Med 2 Med 3 Overall Measures Atrophic lesion growth (mm?) compared 6 N.D & e@ N.D to untreated eyes Visual Function Improvement (BCVA) - - e@ e e@ 3/5 (60%) Total Retinal Thickness (Change
Slide 21: Conclusions
Conclusions * Demonstrated a favorable safety and durability profile. High dose is the Maximum Tolerated Dose. * Demonstrated stabilization or improvement in visual function and retinal structure outcomes * Aligning with FDA for Phase 2 clinical trial design — ocugen. ames a
Slide 22: OCU200
OCU200 Novel biologic for treating Diabetic Macular Edema (DME), Diabetic Retinopathy (DR) and Wet Age-Related Macular Degeneration (Wet AMD) eee 7 ocugen. 22 —
Slide 23: OCU200: Phase 1 Clinical Trial Targeting Diabetic Macular Edema (DME)
OCU200: Phase 1 Clinical Trial Targeting Diabetic Macular Edema (DME) Novel biologics candidate for sight-threatening conditions * Arecombinantfusion protein of transferrinand tumstatin * Potentialtoaddress diabetic macularedema (DME), diabetic retinopathy (DR), and wet AMD High prevalence of DME, D
Slide 24: Near-Term Targeted Milestones
Near-Term Targeted Milestones Complete OCU400 Phase 3 recruitment and continue to monitor patients to ensure BLA/MAA filings are complete in 1H2026 Complete OCU410 Phase 2 recruitment and continue to provide clinical trial updates—Phase 1 patients up to 1 year Continue to provide clinical trial upda
Slide 25: Ocugen Vision
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 — ocugen. aes Ey