EX-99.1 2 tm1923971d1_ex99-1.htm EXHIBIT 99.1

Exhibit 99.1

 

 

Developing Innovative Therapies to Address Rare Diseases and Underserved NASDAQ: OCGN Eye Corporate Deck Exhibit 99.1

 
 

 

 

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 operations and financial position, prospective products, product approvals, research and development costs, timing and likelihood of success, estimated market size or growth, and plans and objectives of management for future operations, are forward-looking statements. When used in this presentation, the words “anticipate,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements involve known and unknown risks, uncertainties and other factors, including those risks set forth in the Company’s filings with the Securities and Exchange Commission, which are available at www.sec.gov, that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Forward-looking statements are based on our management’s beliefs and assumptions and on information available to management as of the date of this presentation. Our actual future results may be materially different from what we expect. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. This presentation includes estimates by us of statistical data relating to market size and growth and other estimated data about our industry. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. This presentation also includes statistical and other industry and market data that we obtained from industry publications and research, surveys and studies conducted by third parties. Industry publications and third-party research, surveys and studies generally indicate that their information has been obtained from sources believed to be reliable, although they do not guarantee the accuracy or completeness of such information. While we believe these industry publications and third-party research, surveys and studies are reliable, we have not independently verified such data. This communication shall not constitute an offer to sell or the solicitation of an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such jurisdiction. No offering of securities shall be made except by means of a prospectus meeting the requirements of Section 10 of the Securities Act of 1933, as amended. 2 ©2019 Ocugen. All Rights Reserved.

 
 

 

 

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 Reserved. SMALL MOLECULE PHASE 3 RARE DISEASE ASSET OCU300 for ocular Graft Versus Host Disease (oGVHD) Orphan Drug Designation MODIFIER GENE THERAPY PLATFORM OCU400 for Inherited Retinal Diseases – Orphan Drug Designations (NR2E3 and CEP290 Mutation-Associated Retinal Diseases) OCU410 for Dry AMD NOVEL BIOLOGIC THERAPIES FOR RETINAL DISEASES OCU200 for Wet AMD, Diabetic Macular Edema, Diabetic Retinopathy OCU100 for Retinitis Pigmentosa

 
 

 

 

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 President, Regulatory & Quality Kelly Beck, MBA Vice President, Investor Relations & Administration Daniel Jorgensen, MD, MPH, MBA Chief Medical Officer Sanjay Subramanian, MBA Chief Financial Officer Shankar Musunuri, PhD, MBA Chairman, CEO and Co-Founder

 
 

 

 

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 Tumslatin-Transferrin 745,000 Diabetic Macular Edema Diabetic Retinopathy OCU100 Retinitis Pigmentosa Gene Therapy Pricing Reference: Novartis gene therapy Zo/gensma® = $2.1M per patient Source: The Wall Street Journal, October 22, 2019 ®, ocugen 5 @2019 Ocusen.AII Rishts Reserved. RETINAL DISEASES (novel biologics) MODIFIER GENE THERAPY PLATFORM OCULAR SURFACE DISEASE (small molecule) Phase 2 Phase 3 Phase 1 Preclinical Prevalence (US) Indication

 
 

 

 

OCU300: oGVHD 6

 
 

 

 

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 recipient ocular tissue Patients encounter dry, tearless eyes, vision issues, severe pain, discomfort, and potential ocular scarring May lead to significant vision loss and irreparable ocular surface damage ~63,000 patients in the US by 2020 • • ~3-6 months from transplant is when patients will develop oGVHD Top 30 BMT centers treat majority of patients with oGVHD 7 ©2019 Ocugen. All Rights Reserved. Source: Prevalence of Hematopoietic Cell Transplant Survivors in the United States, Majhail N et al Oct 2014 Source: https://bethematch.org/tcdirectory/search/advanced/#-/-/-/false/-/TotalTransplants-Ocugen is the first and only company to receive orphan drug designation from FDA for treatment of oGVHD

 
 

 

 

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 Consensus Group revised diagnostic criteria (Ogawa, 2013) Patients referred to specialty BMT centers; 10+ centers are active in this study • 2:1 randomization (OCU300 n=40; Placebo n=20) Co-primary endpoints include: •Symptom: Ocular discomfort based on Visual Analog Scale (VAS) On a scale from 0-10, what was the intensity of your Ocular Discomfort, at its worst, over the past 24 hours? • Sign: Ocular redness based on Validated Bulbar Redness (VBR) Score 8 ©2019 Ocugen. All Rights Reserved. 60 PATIENTS Planned statistical review OCUO3C0U0300 (Brimonidine 0.18% OcuNanoE™) PLAPCLEABCOE*BO (Ophthalmic Buffered Saline)

 
 

 

 

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 preservative-free (no BAK) • Brimonidine approved for chronic treatment in glaucoma • BAK (benzalkonium chloride) is damaging to the cornea 9 ©2019 Ocugen. All Rights Reserved. Generic substitution prohibited • Generic brimonidine (0.2%) not approved for oGVHD • Concentration/formulation different from OCU300 • Contains BAK preservative • OCU300 completing controlled studies in oGVHD patients • AB criteria not applicable

 
 

 

 

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 C U 3 0 0 B r i m o n i d i n e T M O C U 3 0 0 ( 0 . 1 8 % ) O c u N a n o E • • • OCU300 = Brimonidine (0.18%) OcuNanoE™ Brimonidine = Commercial 0.2% solution Figure shows median, interquartile range & min/max fluorescein score **p<0.01 B r i m o n i d i n e s o l u t i o n ( 0 . 2 % ) • 10 ©2019 Ocugen. All Rights Reserved. M e a n ( S D ) T i s s u e C o n c e n t r a t i o n ( n g / g ) C o r n e a l F l u o r e s c e i n S c o r e OcuNanoE™ increases brimonidine in lacrimal gland and improves overall efficacy of OCU300

 
 

 

 

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 only company to receive Orphan Drug Designation from FDA for oGVHD • Advances in hematopoietic cell transplantation leading to increasing number of transplant survivors ~63,000 Patients by 2020 Orphan Drug 11 ©2019 Ocugen. All Rights Reserved. • Hematologists/Oncologists are first prescribers, then referred to specialized ophthalmologists • Hematologists looking for approved therapy; no knowledge of off-label options • No approved therapy • Seek to establish ICD-10 diagnostic code • Analysis supports premium pricing • Opportunities to partner for commercialization Targeted BMT Centers Premium Pricing

 
 

 

 

Modifier Gene Therapy Platform 12

 
 

 

 

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. ModifierWe can address a number gene MModifier Gene product. Cell Cell Cell Cell with mutated/nonfunctioningCell with OCU400 Novel approach that targets nuclear hormone genes (NHRs), which regulate multiple functions within the retina Smoother regulatory pathway due to ability to target multiple diseases with one Ability to recoup development costs over multiple therapeutic indications NR2E3 Mutation-Associated Retinal Disease CEP290 Mutation-Associated Retinal Disease Rhodopsin Mutation-Associated Retinal Disease GENE X GENE X GEN E M GENE M Gene Augmentation: Transfer functional version of a non-functional gene into the target cells. Normal gene X Cell Cell Cell Cell with mutated/ nonfunctioning gene X Traditional Gene Therapy Traditional approach that targets one individual gene mutation at a time Regulatory pathway focused on specific product for one disease Longer time to recoup development costs ONE Disease GENE X GENE X GENE X GEN E X GEN E X GENE X

 
 

 

 

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: subretinal single injection Control Retinal Degeneration Mice Models 30 days post-injection ^rd16 is another name for CEP290 model 14 ©2019 Ocugen. All Rights Reserved. Untreated Treated* One Product Rescues Multiple IRDs after onset

 
 

 

 

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 Baseline (Control) 1 mo after treatment OCU400 Single Injection (Subretinal) rd7 mouse model 15 ©2019 Ocugen. All Rights Reserved. Our First Gene Therapy Product to Clinic

 
 

 

 

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 Bamboo; Celgene acquired Juno Others being acquired by major CMOs to establish their presence in the gene therapy – eg: Thermo Fisher acquired Brammer Bio; Catalent acquired Paragon • Gene therapy companies facing manufacturing bottleneck & costs • • • Long wait in the queue for CMO while large pharma can bypass (due to scope and financial power) Traditional CMO model not appropriate for implementing specialized process optimization steps High cost for the CMC development and clinical supplies; approximately: – – – $7M - $10M for Phase 1 $8M - $10M for late stage $10M - $15M for scale-up development for commercialization/BLA filing 16 ©2019 Ocugen. All Rights Reserved. Source: Manufacturing Cures: Infrastructure Challenges Facing Cell And Gene Therapy Developers In Vivo June 2019 invivo.pharmaintelligence.informa.com Critical to find a Strategic and Reliable Partner that also shares costs

 
 

 

 

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 culture adopted) for commercial manufacturing CanSinoBIO to perform CMC development & manufacturing of clinical supplies • CanSinoBIO responsible for all associated costs • Option for commercial manufacturing agreement CanSinoBIO has rights to develop, manufacture and commercialize OCU400 for Greater China market 17 ©2019 Ocugen. All Rights Reserved. Partnership paves a path for Ocugen to advance OCU400 into the clinic with significantly reduced capital and resources

 
 

 

 

OCU200: Wet AMD/DME/DR New therapies Anti-VEGF needed beyond 18

 
 

 

 

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 compared to PBS and/or tumstatin treatment. † indicates p<0.05 when compared to bevacizumab; CNV lesions measured on day 14 after treatment. Data expressed as percentage of CNV lesions on Day 10 after treatment. Laser induction & treatment start on Day 0. 19 ©2019 Ocugen. All Rights Reserved. L e a k y C N V l e s i o n s ( % ) OCU200 Demonstrated Superior Efficacy with Potentially Fewer Injections in Head-to-Head Studies ✓50% of patients are non-responders to Anti-VEGF ✓Selectively works on active endothelial cells ✓Activates native anti-angiogenic response ✓Targeting element enhances effective concentration ✓Pro-apoptotic and anti-oxidative ✓Inhibits new blood vessel formation ✓Reduces damage to retina

 
 

 

 

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 Study Note: Check mark (✓) denotes completed milestone. All other milestones are anticipated future milestones. 20 ©2019 Ocugen. All Rights Reserved. ✓Feb 2019: Pre-IND Meeting ✓Feb 2019: ODD for NR2E3 Mutation-Associated Retinal Diseases ✓Aug 2019: ODD for CEP290 Mutation-Associated Retinal Diseases ✓Sept 2019: CanSinoBIO Co-Development & Manufacturing Partnership • 2019-2020: Continue IND-Enabling Studies • 2021: Target Phase 1/2a Clinical Trial OCU400 (NR2E3-AAV) Retinal Degenerative Diseases (gene therapy)

 
 

 

 

Contact Kelly Beck Vice President, Investor Relations & Administration kelly.beck@ocugen.com 21 ©2019 Ocugen. All Rights Reserved.