Slide 1: Courageous Innovation 3Q 2023 Business Update
Courageous Innovation 3Q 2023 Business Update November 9, 2023 NASDAQ: OCGN
Slide 2: 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,” “continue,” “estimates,” “antici
Slide 3: 2023/2024: Updated Anticipated Timing on Milestones
3 2023/2024: Updated Anticipated Timing on Milestones OCU500 NeoCart® OCU400 OCU410ST OCU200 3Q 2H244Q NIAID Selection Initiate Phase 3 trial Initiate Phase 3 trial for RP* Initiate Phase 1 trial Clinical trial update Initiate Phase 1/2 trial OCU410 *The Company will be expanding the OCU400 Phase 3
Slide 4: OCU400 Clinical Program
OCU400 Clinical Program A Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa associated with NR2E3 and RHO mutations and Leber Congenital Amaurosis with mutation(s) in CEP290 gene Clinical Trials.gov Identifier: NCT05203939 Safety of subretinal administration of OCU
Slide 5: Retinitis Pigmentosa and Leber Congenital Amaurosis: Unmet Need and Treatment Benefit Target
Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA)— Unmet need and Treatment Benefit Target • Inherited retinal diseases (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 • IRDs often lead t
Slide 6: Responder Analysis Stabilization/Improvement from Baseline
Responder Analysis Stabilization/Improvement from Baseline [Treated Eyes] Showed stabilization or improvement on all three parametersStabilization: BCVA: 4 letters change LLVA: 4 letters change MLMT: 0 change in Lux Level Improvement: BCVA: ≥ 5 letters change LLVA: ≥ 5 letters change MLMT: ≥1 chan
Slide 7: Encouraging Results from OCU400 Phase 1/2 Clinical Trial Update
Encouraging Results from OCU400 Phase 1/2 Clinical Trial Update Results suggest gene modifier gene therapy has potential benefits in broader RP and LCA patients • OCU400 continues to demonstrate a favorable safety and tolerability profile • Clinical study update suggests continued positive trends in
Slide 8: Continued Momentum in Progressing OCU400 Phase 1/2 Trial
Continued Momentum in Progressing OCU400 Phase 1/2 Trial • Completed dosing adult RP patients in the dose-escalation and dose-expansion portions of the trial • Completed dosing three LCA patients including a pediatric patient • Planning to initiate Phase 3 trial for the treatment of RP in early 2024
Slide 9: OCU410 Advancement from Recently Approved Therapies for GA
* As demonstrated in Peak scotopic B-wave amplitudes. N≥5 biological replicates Advancement from recently approved therapies for GA: Potential to address limitations of recently approved therapies for GA focused only on the complement system, including: • Patient Compliance Frequent intravitreal i
Slide 10: OCU410 (RORA): A Potential Modifier Therapeutic for Dry-AMD and STGD
50 60 70 80 90 1e61E51E41E31E2Cells N or m al iz ed C el l S ur vi va l 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
Slide 11: OCU410 Program Overview
OCU410 Program Overview https://clinicaltrials.gov/study/NCT06018558?cond=Geographic%20atrophy&rank=2 11 • Assess the safety and efficacy of OCU410 in subjects with Geographic Atrophy (GA) secondary to dry Age-Related Macular Degeneration (AMD) • Single, unilateral subretinal injection using a modif
Slide 12: OCU410ST: Received ODD for ABCA4-Associated Retinopathies
OCU410ST: Received ODD for ABCA4-Associated Retinopathies: Stargardt, Retinitis Pigmentosa 19(RP19) & Cone-rod Dystrophy 3(CORD3) ABCA4-associated retinopathies—Genetic Rare Disease • ABCA4 gene produces an ATP-binding cassette (ABC) superfamily transmembrane protein involved in clearance of all-tra
Slide 13: OCU410ST Phase 1/2 Study Design
• Assess the safety and efficacy of OCU410ST in subjects with Stargardt disease • Single, unilateral subretinal injection using a modifier gene therapy platform • 42 subjects (30 adults and 12 pediatrics) • Mild to moderate disease presentation 2 3 41 Dose Escalation: • 3+3 design with a low, medium
Slide 14: OCU500 Selected for NIAID's Project NextGen Covid-19 Clinical Trial
OCU500 Selected for NIAID’s Project NextGen Covid-19 Clinical Trial OCU500 to expand vaccine options in fighting COVID-19 • NIAID Phase 1 clinical trials will evaluate the safety and immunogenicity of OCU500 using inhaled and intranasal routes of delivery • Collaborating with NIAID to initiate the c
Slide 15: Pipeline Updates
Pipeline Updates NeoCart® Regenerative Cell Therapy • Phase 3 clinical trial on track to be initiated in 2H2024 • Renovations to develop a cGMP facility for manufacturing NeoCart clinical trial material continues with expected completion at the end of 2023 OCU200 Ophthalmic Biologic Product • Contin
Slide 16: Financial Update
FinancialUpdate 16
Slide 17: Financial Update
Financial Update Nine months ended September 30, Three months ended September 30,Statement of Operations 2022202320222023 $32.5$30.1$15.6$6.3Research and development expense 28.226.87.59.1General and administrative expense 1.33.31.21.3Other income (expense), net $(59.4)$(53.6)$(21.9)$(14.2)Net loss
Slide 18: Questions & Answers
Questions & Answers 18
Slide 19: Ocugen Vision
19 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