Slide 1: Study Update: OCU400 Phase 1/2 for RP and LCA
Study Update: OCU400 Phase 1/2 for RP and LCA 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
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 qualitative assessments of ava ilable da
Slide 3: OCU400: A Novel Gene-Agnostic Modifier Gene Therapy for RP and LCA
OCU400: A Novel Gene - Agnostic Modifier Gene Therapy for RP and LCA FDA granted expanded Orphan Drug Designations for all retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) mutations Despite its high prevalence, RP and LCA patients have limited treatment options • US: RP & LCA affect 11
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: 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 6: Key Inclusion/Exclusion Criteria
Key Inclusion/Exclusion Criteria Clinical Trials.gov Identifier: NCT05203939 For RP Subjects: BCVA ≤ 20/50 or visual field less than 20 ° in any meridian, as measured by a III4e isopter or equivalent in study eye Inclusion Exclusion 4 Confirmed genetic diagnosis of AR - NR2E3 mutations or AD - NR2E3
Slide 7: Enrollment Status
Enrollment Status 5 Adult RP AR NR2E3 n=5 AD NR2E3 n=3 AD RHO n=10 AR=AutosomalRecessive,AD=AutosomalDominant COMPLETED Adult LCA ARCEP290 n=3 Pediatric RP/LCA AR CEP290, AD RHO, AR/ADNR2E3 n=3 ENROLLING 5 • Subjects (N=18) with ages ranging 18 - 77 years enrolled in this study to date • Consists of
Slide 8: Safety Summary for OCU400
Safety Summary for OCU400 The Phase 1/2 clinical trial demonstrated that OCU400 continued to be generally safe and well - tolerated in subjects across different mutations and dose levels There were no serious adverse events (SAEs) related to the investigational product in the low and medium dose coh
Slide 9: RP and LCA - Unmet need and Treatment Benefit Target
RP and LCA - Unmet need and Treatment Benefit Target 7 • 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
Slide 10: Responder Analyses
Responder Analyses Assessed for subjects who have completed a minimum of 6 - months follow - up post - OCU400 dosing • Improvement in Best - Corrected Visual Acuity (BCVA) from Baseline ≥ 4 Letters, and ≥ 7 Letters • Improvement in (Low Luminance Visual Acuity) LLVA from Baseline ≥ 5 Letters and ≥ 1
Slide 11: Responder Analysis Stabilization/Improvement from Baseline [Treated Eyes]
Responder Analysis Stabilization/Improvement from Baseline [Treated Eyes] * * * * Non responder 9 Confidential - Preliminary Showed stabilization or improvement on all three parameters
Slide 12: Responder Analysis BCVA LLVA MLMT - Preliminary
Responder Analysis BCVA LLVA MLMT 10 Confidential - Preliminary
Slide 13: Conclusions
Conclusions • OCU400 continues to demonstrate favorable safety and tolerability profile of OCU400 investigational drug product in Phase 1/2 study • Clinical study update suggests continued positive trends in Best - Corrected Visual Acuity (BCVA) and Multi - Luminance Mobility Testing (MLMT), as well
Slide 14: Key Takeaways
Key Takeaways The Phase 1/2 clinical trial demonstrated that OCU400 continued to be generally safe and well - tolerated in subjects across different mutations and dose levels Our study supports the gene - agnostic mechanism of action of the drug and demonstrates positive trends for key efficacy outc
Slide 15: Acknowledgements
Acknowledgements We would like to thank the study investigators, study participants, site coordinators, and research teams. 13
Slide 16: Ocugen
ocugen