Slide 1: Topline Data for Phase 2 ArMaDa Trial of OCU410 for Geographic Atrophy
Courageous Innovation Topline Data for Phase 2 ArMaDa Trial A Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy (late - stage dry AMD)
Slide 2: Forward-Looking Statements
2 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 fo r t he preclinical and clinical develop
Slide 3: OCU410 GA Webcast Meeting Agenda
3 • Introduction – Shankar Musunuri , PhD, MBA, Chairman, Chief Executive Officer, and Co - founder of Ocugen • Phase 2 GA Data Updates • Lejla Vajzovic , MD, FASRS, Professor of Ophthalmology, CME Program Director, Duke Ophthalmology, Duke University School of Medicine & Chairman, Ocugen Scientific
Slide 4: Ocugen's Bold Mission
Ocugen’s Bold Mission Leader in ophthalmic gene therapies Address major blindness diseases with novel modifier gene therapy Bring transformative one - time treatments to market Work even harder to provide access to patients globally 1 2 3 4
Slide 5: OCU410 Aims to Disrupt GA Treatment Driven by a Novel MOA
5 1 Akula et al. Gene Ther 2024; MOA= Mechanism of Action: a nti - drusen activity (improves retinal function), anti - inflammatory (suppresses inflammation in HMC3 cells), anti - oxidative (impr oves ARPE19 cell survival), anti - complement (increases Cd59 protein) OCU410 Aims to Disrupt GA Treatme
Slide 6: OCU410 Phase 2 Data Show Significant Reduction in Lesion Size at 12 Months
OCU410 Phase 2 Data Show Significant Reduction in Lesion Size at 12 Months References: Apellis OAKS/DERBY (Heier et al, Lancet), Iveric GATHER 2 (Liao 2023, Khanani 2024, Lancet/Ophthalmology), Natural History Meta - analysis (Fleckenstein 2018, Ophthalmology). Cha nge from Baseline for OCU410 was a
Slide 7: Phase 2 ArMaDa Trial: To Assess Safety and Efficacy of OCU410 in GA
Endpoints Randomization 1:1:1 EZ preservation (correlates to visual function ) 17 Control Group No Treatment Primary: Exploratory: 17 Medium Dose 1x 10 10 vg per eye, 200 µL 17 High Dose 3x 10 10 vg per eye, 200 µL Phase 2 ArMaDa Trial: To Assess Safety and Efficacy of OCU410 in GA Target Population
Slide 8: Phase 2 Baseline Demographics and Characteristics
Phase 2 Baseline Demographics and Characteristics 8 All participants >60 yrs, all White except 1 (Control Group) which was not reported, * Criteria for approved trials (for Syfovre TM ) include baseline lesion of ≥2.5mm 2 and ≤18.5m m 2 Nonfoveal lesions becoming foveal by M12: 1 in Control Group, 1
Slide 9: Phase 2 Safety and Tolerability Data
9 Retinal Vasculitis and/or Retinal Vascular Occlusion Choroidal Neovascularization (CNV) Intraocular Inflammation I schemic Optic Neuropathy Treatment Emergent Serious Adverse Events Treatment Emergent Adverse Events C onsidered S evere OCU410 Med Dose (N=16) Endophthalmitis and Retinal Detachments
Slide 10: OCU410 Provides Treatment Benefit at 12 Months
Lesion Size 31%, 16% reduction in medium, high dose group, respectively compared to control 10 OCU410 Provides Treatment Benefit at 12 Months For lesion criteria (≥2.5 mm 2 and ≤17.5mm 2 ) evaluable subjects (N=39)include controls (N=12), medium dose (N=16), and high dose (N=11) and for lesion crite
Slide 11: Treatment Benefit with Optimal Dose (Medium) in Phase 2 ArMaDa Trial
0 10 20 30 12 Months Control Medium Dose Lesion Size 31% reduction in medium dose group compared to control Treatment Benefit with Optimal Dose (Medium) in Phase 2 ArMaDa trial References for Natural History: Mones and Biarnes, 2018, TVST, N =117; For Primary Endpoint analysis evaluable subjects inc
Slide 12: Clinical Effects Observed with OCU410 Treatment in GA Participants
Clinical Effects Observed with OCU410 Treatment in GA Participants 12 Efficacy Highlights (N=42) • Robust response – 55% achieved ≥30% reduction • Central therapeutic benefit – median 33% reduction • Superior to natural history (p<0.05 vs natural history/control) Fisher's exact p<0.05 vs. Natural Hi
Slide 13: Ocugen Hopes to Deliver on Its Promise to Transform the Treatment Landscape
Ocugen Hopes to Deliver on Its Promise to Transform the Treatment Landscape for Patients with GA 13 OCU410 potentially creates a new standard of care • First - in - class RORA MOA designed to support central retina and photoreceptor integrity • Promising Phase 2 results indicate 31% reduction in les
Slide 14: Advancing cures for blindness
Advancing cures for blindness IR @ocugen.com