Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (November 2019)
21 slides from Ocugen's investor presentation, Nov 26, 2019
Slide 1: Developing Innovative Therapies to Address Rare Diseases and Underserved Eye Diseases
Developing Innovative Therapies to Address Rare Diseases and Underserved NASDAQ: OCGN Eye Corporate Deck Exhibit 99.1
Slide 2: Forward Looking Statement
Forward Looking Statement This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this presentation, including statements regarding our business strategy, future results of operati
Slide 3: Company Highlights
Company Highlights Nasdaq-listed company as of September 30, 2019 Robust IP portfolio with 31 issued U.S. and foreign patents and 29 U.S. and foreign patent applications Strategic Partnership with CanSinoBIO for OCU400 Gene Therapy Co-Development & Manufacturing • • • 3 ©2019 Ocugen. All Rights Rese
Slide 4: Experienced Leadership Team
Experienced Leadership Team • • • Diverse experience in large pharma, signature biotech, and small companies Track record of success Brings large and small company learnings to Ocugen 4 ©2019 Ocugen. All Rights Reserved. Rasappa Arumugham, PhD Chief Scientific Officer Vijay Tammara, PhD Vice Preside
Slide 5: Orphan us oGVHD 60,000 OCU300 NR2E3 Mutation-Associated RetinalDegeneration Orphan us 500-600 CEP290 Mutation-Associa…
Orphan us oGVHD 60,000 OCU300 NR2E3 Mutation-Associated RetinalDegeneration Orphan us 500-600 CEP290 Mutation-Associated RetinalDegeneration Orphan us OCU400 NR2E3-MV 2,500-3,000 Rhodopsin Mutation-Associated RetinalDegeneration 10,400-12,700 OCU410 RORA-MV 9-10M Dry AMD > WetAMD 1.1M > > OCU200 Tum
Slide 6: OCU300: oGVHD
OCU300: oGVHD 6
Slide 7: OCU300 for oGVHD: Unmet Need for Patients with Ocular Graft vs Host Disease
OCU300 for oGVHD: Unmet Need for Patients with Ocular Graft vs Host Disease (oGVHD) Rare Ocular Diseases ~60% of allogeneic bone marrow transplant patients will develop oGVHD • Autoimmune disease that occurs in allogeneic bone marrow transplant (BMT) patients –Donor derived leukocytes attack recipie
Slide 8: First Phase 3 Study With Orphan Drug Designation
First Phase 3 Study With Orphan Drug Designation Indication: Treatment of ocular discomfort and ocular redness in patients with oGVHD • • 84-day, Randomized, Double-Masked, Placebo-Controlled Study Key inclusion criteria: diagnosis of ‘definite’ oGVHD using the International Chronic Ocular GVHD Cons
Slide 9: Brimonidine Safety and Efficacy
Brimonidine Safety Existing Molecule is already proven safe and effective Efficacy Early stage clinical studies led to Phase 3 design OcuNanoE™ drug delivery system improves overall efficacy • 505(b)(2) regulatory pathway allows use of safety data already available for brimonidine OCU300 is preserva
Slide 10: OcuNanoE Drug Delivery System Improves Overall Efficacy
OcuNanoETM Drug Delivery System Improves Overall Efficacy Brimonidine Level in Lacrimal Gland (Preclinical) Mouse DED Model (Preclinical) ** 1 0 0 0 4 3 1 0 0 2 1 0 1 0 3 6 9 1 2 0 Drug distribution to lacrimal gland from traditional eye drops is low relative to other target tissues T i m e ( h ) O
Slide 11: OCU300 has Compelling Value Proposition
OCU300 has Compelling Value Proposition Market Access Market Potential Physicians Patients • Spend 3 months in hospital after receiving bone marrow transplant • Potential to be first approved product in US market • Most exhibit symptoms while still under hematologist/ oncologist care • First and onl
Slide 12: Modifier Gene Therapy Platform
Modifier Gene Therapy Platform 12
Slide 13: Modifier Gene Therapy Has a Broader Impact of Diseases
Modifier Gene Therapy Has a Broader Impact of diseases using the same gene(s) other than modifier gene normal function product 13 ©2019 Ocugen. All Rights Reserved. Modifier Gene Therapy: Introduce a functional gene to modify the expression of many genes, gene-networks and reset homeostasis. Modifie
Slide 14: OCU400 (NR2E3) Rescues Vision Loss in Multiple Inherited Retinal Diseases
OCU400 (NR2E3) Rescues Vision Loss in Multiple Inherited Human Disease: Retinal Diseases (IRDs) Rhodopsin associated adRP Rho-/-Rhodopsin associated adRP RhoP23 H PDE6B associated RP rd1-/-PDE associated arRP rd10-/-Leber Congenital Amaurosis Cep290 (rd16) Mouse Models: ^ *Treated fundus photos: sub
Slide 15: OCU400: Orphan Drug Designation NR2E3 Mutation-Associated Retinal Degeneration
OCU400: Orphan Drug Designation NR2E3 Mutation-Associated Retinal Degeneration Early stage disease OCU400 Single Injection (Intravitreal) 1 mo Baseline (Control) 1 mo after treatment Advanced stage disease OCU400 Single Injection (Intravitreal) 3 mo Baseline (Control) 1 mo after treatment 3 mo Basel
Slide 16: Gene Therapy Manufacturing: A Major Bottleneck Faced by All
Gene Therapy Manufacturing: A Major Bottleneck Faced by All Increased demand for cell & gene therapy manufacturing • • 1,060 clinical trials globally; 80 cell and gene therapy trials in Phase 3 Large pharma acquiring companies to support internal programs – eg: Roche acquired Spark; Pfizer acquired
Slide 17: OCU400 Gene Therapy Manufacturing: Strategic Partnership with CanSinoBIO
OCU400 Gene Therapy Manufacturing: Strategic Partnership with CanSinoBIO CanSinoBIO • • • Biotech company publicly-listed on Hong Kong exchange (6185.HK) with market cap of ~$1 Billion State-of-the-art facilities with world class team Provides scalable GMP cell lines (such as HEK293 suspension cultu
Slide 18: OCU200: Wet AMD/DME/DR
OCU200: Wet AMD/DME/DR New therapies Anti-VEGF needed beyond 18
Slide 19: Lead Biologic OCU200: Tumstatin-Transferrin Fusion Protein
Lead Biologic OCU200: Tumstatin-Transferrin Fusion Protein Offering Benefits Beyond Anti-VEGF In-Vivo Laser-Induced Rat CNV Model In-Vivo Laser-Induced Mouse CNV Model 8 0 6 0 4 0 PBS Tumstatin OCU200 Avastin V e h i c l e E y l e a O C U 2 0 0 O C U 2 0 0 + E y l e a * indicates p<0.05 when compare
Slide 20: Summary of Near-Term Milestones
Summary of Near-Term Milestones OCU300 ocular GVHD (small molecule) OCU200 Wet AMD, DME, DR (novel biologic) • ✓ Dec 2018: First Patient Dosed 2019-2020: Continue IND-Enabling Studies • 1H2020: Statistical Review • 2021: Target Phase 1/2a Clinical Trial • 2H2020: Estimated Topline Results of First S
Slide 21: Contact
Contact Kelly Beck Vice President, Investor Relations & Administration kelly.beck@ocugen.com 21 ©2019 Ocugen. All Rights Reserved.