Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (November 2023)
23 slides from Ocugen's investor presentation, Nov 9, 2023
Slide 1: Courageous Innovation: Gene & Cell Therapies and Vaccines
Courageous Innovation Dedicated to Bringing Game-Changing Gene & Cell Therapies and Vaccines to Market and Working Even Harder to Provide Access to Patients Globally November 2023 NASDAQ: OCGN
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: 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”;
Slide 4: Pipeline Overview
4 Pipeline Overview Current StatusIndicationAsset/Program • Phase 1/2 • Completed dosing of RP and LCA patients • Phase 3 trial for the treatment of RP expected to be initiated in early 2024 Retinitis pigmentosa (RP)--NR2E3 MutationOCU400 * AAV-hNR2E3 Gene mutation-associated retinal degeneration Ge
Slide 5: Modifier Gene Therapy Platform
5 Modifier Gene Therapy Platform Breakthrough technology designed to address many rare diseases as well as complex diseases that affect millions
Slide 6: Modifier Gene Therapy: A Broader Reach
6 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
Slide 7: FDA granted expanded Orphan Drug Designations for RP and LCA
FDA granted expanded Orphan Drug Designations for all retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) mutations Despite its prevalence, RP and LCA patients have limited treatment options • US: RP & LCA affect 110,000 and 15,000 people, respectively • Worldwide: conditions affect appro
Slide 8: 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 9: Safety Summary for OCU400—Clinical Study Update
Safety Summary for OCU400—Clinical Study Update • 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
Slide 10: RP and LCA—Unmet need and Treatment Benefit
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 11: Responder Analysis
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 12: Conclusions from Latest Clinical Study Results
Conclusions from Latest Clinical Study Results • OCU400 continues to demonstrate a favorable safety and tolerability profile • Clinical study update suggests continued positive trends in Best-Corrected Visual Acuity (BCVA) and Multi-Luminance Mobility Testing (MLMT), as well as positive trends in Lo
Slide 13: OCU400: Expected Pathway to Clinical Development & Potential Approval
OCU400: Expected Pathway to Clinical Development & Potential Approval Both FDA & EMA granted broad orphan drug designation for RP & LCA • Ocugen plans to meet with regulatory agencies in 4Q to potentially finalize Phase 3 clinical program and overall package • Continue to dose adult LCA patients and
Slide 14: OCU410 for Dry AMD: Advancement from Approved GA Therapies
* As demonstrated in Peak scotopic B-wave amplitudes. N≥5 biological replicates 14 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 intravitr
Slide 15: OCU410 (RORA): 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 16: OCU410ST: Received ODD for ABCA4-Associated Retinopathies
16 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-
Slide 17: OCU200: Novel biologic for DME, DR and Wet AMD
17 OCU200 Novel biologic for treating Diabetic Macular Edema (DME), Diabetic Retinopathy (DR) and Wet Age-Related Macular Degeneration (Wet AMD)
Slide 18: OCU200 is our novel biologics candidate for sight-threatening conditions
18 OCU200 is our novel biologics candidate for sight-threatening conditions • A recombinant fusion protein of transferrin and tumstatin • Potential to address diabetic macular edema (DME), diabetic retinopathy (DR), wet AMD High prevalence of DME, DR and wet AMD patients • DME: 21M worldwide • DR: 1
Slide 19: NeoCart (Autologous chondrocyte-derived neocartilage)
19 NeoCart® (Autologous chondrocyte-derived neocartilage)
Slide 20: NeoCart is a regenerative cell therapy
20 *The Journal of Bone & Joint Surgery: June 1, 2011 - Volume 93 - Issue 11 - p 994-1000 **https://www.biospace.com/article/cell-therapy-market-size-cagr-trends-forecast-report-2022-2030/ NeoCart is a regenerative cell therapy • Received RMAT designation • Combines bioengineering and cell processin
Slide 21: OCU500 Series: COVID-19 Mucosal Vaccine, Flu, COVID-19/Flu
21 OCU500 Series: OCU500: COVID-19 Mucosal Vaccine OCU510: Flu OCU520: COVID-19/Flu
Slide 22: OCU500 Series: Next-Generation Vaccine Technology
22 OCU500 Series: Next-Generation Vaccine Technology Inhaled mucosal vaccine platform based on ChAd vector OCU500 A bivalent COVID-19 vaccine OCU510 A seasonal quadrivalent flu vaccine OCU520 A combination quadrivalent flu and bivalent COVID19 vaccine Inhalation technology as a differentiator • Mult
Slide 23: Ocugen Vision
23 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