UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, DC 20549
___________________________________________________________
FORM 8–K
___________________________________________________________
CURRENT REPORT
Pursuant to Section 13 OR 15 (d)
of the Securities Exchange Act of 1934
Date of Report (Date of Earliest Event Reported): February 14, 2020
___________________________________________________________
OCUGEN, INC.
(Exact Name of Registrant as Specified in its Charter)
___________________________________________________________
| Delaware | 001-36751 | 04-3522315 | ||||||||||||
| (State or Other Jurisdiction of Incorporation) | (Commission File Number) | (I.R.S. Employer Identification Number) | ||||||||||||
5 Great Valley Parkway, Suite 160
Malvern, Pennsylvania 19355
(484) 328-4701
(Addresses, including zip code, and telephone numbers, including area code, of principal executive offices)
N/A
(Former Name or Former Address, if Changed Since Last Report)
Check the appropriate box below if the Form 8–K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
o Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
o Soliciting material pursuant to Rule 14a–12 under the Exchange Act (17 CFR 240.14a–12)
o Pre–commencement communications pursuant to Rule 14d–2(b) under the Exchange Act (17 CFR 240.14d–2(b))
o Pre–commencement communications pursuant to Rule 13e–4(c) under the Exchange Act (17 CFR 240.13e–4(c))
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading Symbol(s) | Name of each exchange on which registered | ||||||||||||
| Common Stock, $0.01 par value per share | OCGN | The Nasdaq Stock Market LLC (The Nasdaq Capital Market) | ||||||||||||
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company o
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. o
Item 7.01 Regulation FD Disclosure
Attached as Exhibit 99.1 and furnished for purposes of Regulation FD is a presentation that Ocugen, Inc. (“Ocugen”) will post on its website on February 14, 2020, and may use from time to time in presentations or discussions with investors, analysts and other parties.
The information in this Item 7.01 (including Exhibit 99.1) is being furnished solely to satisfy the requirements of Regulation FD and shall not be deemed to be “filed” for purposes of Section 18 of the Securities Exchange Act of 1934 (the “Exchange Act”) or otherwise subject to the liabilities of that Section, nor shall it be deemed to be incorporated by reference in any filing under the Securities Act of 1933 or the Exchange Act.
Item 9.01 Financial Statements and Exhibits
The following exhibit is being filed herewith:
(d) Exhibits
| Exhibit No. | Document | |||||||
| 99.1 | ||||||||
SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
Date: February 14, 2020
| OCUGEN, INC. | ||||||||
| By: | /s/ Shankar Musunuri | |||||||
| Name: Shankar Musunuri | ||||||||
| Title: Chief Executive Officer and Chairman | ||||||||
Developing Transformative Therapies to Treat the Whole Eye NASDAQ: OCGN Corporate Deck
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. ©2020 Ocugen. All Rights Reserved. 2
Company Highlights OCGN Strong Diversified (NASDAQ) Global IP Portfolio THREE WAVES OF TECHNOLOGICAL INNOVATION Strategic Gene Therapy SMALL MOLECULE PHASE 3 RARE DISEASE ASSET Manufacturing Partnership OCU300 for ocular Graft Versus Host Disease (oGVHD) MODIFIER GENE THERAPY PLATFORM OCU400 for Inherited Retinal Diseases OCU410 for Dry AMD NOVEL BIOLOGIC THERAPIES FOR RETINAL DISEASES OCU200 for DME, Diabetic Retinopathy, Wet AMD CanSinoBIO ©2020 Ocugen. All Rights Reserved. 3
Experienced Leadership Team • Diverse experience in large pharma, signature biotech, and small companies • Development, Manufacturing and Commercialization expertise provides know-how to take pipeline from preclinical to market Shankar Musunuri, PhD, MBA Sanjay Subramanian, MBA Chairman, CEO and Co-Founder Chief Financial Officer Daniel Jorgensen, MD, MPH, MBA Kelly Beck, MBA Chief Medical Officer Vice President, Investor Relations & Administration Rasappa Arumugham, PhD Vijay Tammara, PhD Chief Scientific Officer Senior Vice President, Regulatory & Quality ©2020 Ocugen. All Rights Reserved. 4
Three Waves of Technological Innovation ©2020 Ocugen. All Rights Reserved. 5
OCU300: oGVHD Near-term Commercialization Opportunity Potential to be First FDA Approved Treatment 6
OCU300 for oGVHD: Unmet Need for Patients with Rare Ocular Disease Ocular Graft vs Host Disease (oGVHD) ~60% of allogeneic bone marrow transplant • Autoimmune disease that occurs in patients will develop oGVHD allogeneic bone marrow transplant (BMT) patients patients in the US – Donor derived leukocytes attack recipient ocular ~63,000 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 ~3-6 months from transplant is when patients will develop oGVHD Ocugen is the first and only company to receive orphan drug designation from FDA Top 30 BMT centers treat majority for treatment of oGVHD of patients with oGVHD ©2020 Ocugen. All Rights Reserved. 7 Source: Prevalence of Hematopoietic Cell Transplant Survivors in the United States, Majhail N et al Oct 2014 7 Source: https://bethematch.org/tcdirectory/search/advanced/#-/-/-/false/-/TotalTransplants-
Phase 3 Study With Topline Results Expected 2H2020 Indication: Treatment of ocular discomfort and ocular redness in patients with oGVHD 60 PATIENTS • 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 OCU300OCU300 PLACEBO*PLACEBO (Brimonidine 0.18% OcuNanoE™) (Ophthalmic Buffered Saline) 2:1 randomization Co-primary endpoints include: (OCU300 n=40; Placebo n=20) • Symptom: Ocular discomfort based on Visual • Sign: Ocular redness based on Validated Analog Scale (VAS) Bulbar Redness (VBR) Score On a scale from 0-10, what was the intensity of your Ocular Discomfort, at its worst, over the past 24 hours? ©2020 Ocugen. All Rights Reserved. 8
Active Ingredient Proven Safe and Effective Safety Efficacy Existing Molecule (Brimonidine) Early stage clinical studies led to Phase 3 design • 505(b)(2) regulatory pathway allows use of OcuNanoE™ drug delivery system improves overall efficacy safety data already available for brimonidine • Brimonidine approved for chronic treatment in OCU300 is preservative-free (no BAK) glaucoma • BAK (benzalkonium chloride) is damaging to the cornea 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 ©2020 Ocugen. All Rights Reserved. 9
OcuNanoETM Drug Delivery System Improves Overall Efficacy Brimonidine Level in Lacrimal Gland Mouse DED Model (Preclinical) (Preclinical) ** 1000 4 100 3 2 M Tissue ean (SD) Concentration (ng/g) Concentration 10 1 0 3 6 9 12 0 Corneal Fluorescein Score Fluorescein Corneal Drug distribution to lacrimal gland T im e (h ) O CU300 Brimonidine TM from traditional eye drops is low O CU300 (0.18%) OcuNanoE Brimonidine solution (0.2% ) • OCU300 = Brimonidine (0.18%) OcuNanoE™ relative to other target tissues • Brimonidine = Commercial 0.2% solution • Figure shows median, interquartile range & min/max fluorescein score • **p<0.01 OcuNanoE™ increases brimonidine in lacrimal gland and improves overall efficacy of OCU300 ©2020 Ocugen. All Rights Reserved. 10
OCU300 has Compelling Value Proposition Patients Physicians Market Market Access Potential • Spend 3 months in hospital after • Hematologists/Oncologists are first • No approved therapy • Potential to be first approved receiving bone marrow transplant prescribers, then referred to product in US market specialized ophthalmologists • Seek to establish ICD-10 diagnostic • Most exhibit symptoms while still code • First and only company to receive under hematologist/ • Hematologists looking for approved Orphan Drug Designation from FDA oncologist care therapy; no knowledge of off-label • Analysis supports premium pricing for oGVHD options • Opportunities to partner for • Advances in hematopoietic cell commercialization transplantation leading to increasing number of transplant survivors ~63,000 Patients Targeted BMT Centers Premium Pricing Orphan Drug ©2020 Ocugen. All Rights Reserved. 11
Breakthrough Modifier Gene Therapy Platform Addressing Multiple Diseases with One Product 12
Potential to Treat Many Diseases with One Product Gene Augmentation: Transfer functional version of a Modifier Gene Therapy: Introduce a functional gene to modify the non-functional gene into the target cells. expression of many genes, gene-networks and reset homeostasis. We can address a number Modifier of diseases using the same gene M Modifier Gene product. Normal gene X GENE M Cell GENE X Cell Cell Cell Cell Cell GENE X GENE X GENE M GENE X GENE X GENE X GENE X GENE X Cell with mutated/ nonfunctioning gene X Cell with mutated/nonfunctioning Cell with gene(s) other than modifier gene normal function Traditional NR2E3 Mutation-Associated Retinal Disease Gene ONE Disease OCU400 CEP290 Mutation-Associated Retinal Disease Therapy Rhodopsin Mutation-Associated Retinal Disease Traditional approach that targets one individual gene mutation at a time Novel approach that targets nuclear hormone genes (NHRs), which regulate Regulatory pathway focused on specific product for one disease multiple functions within the retina Longer time to recoup development costs Smoother regulatory pathway due to ability to target multiple diseases with one product Ability to recoup development costs over multiple therapeutic indications ©2020 Ocugen. All Rights Reserved. 13
OCU400 (NR2E3-AAV) Rescues Vision Loss in Multiple Inherited Retinal Diseases (IRDs) Human Disease: PDE6β PDE Rhodopsin Rhodopsin Leber associated associated associated associated Congenital RP arRP adRP adRP Amaurosis Mouse Models: rd1-/- rd10-/- Rho-/- RhoP23H Cep290 (rd16) Untreated Treated* ^ ^ *Treated fundus photos: subretinal single injection Control Retinal Degeneration Mice Models 30 days post-injection ^rd16 is another name for CEP290 model One Product Rescues Multiple IRDs after onset ©2020 Ocugen. All Rights Reserved. 14
OCU400: Orphan Drug Designation NR2E3 Mutation-Associated Retinal Degeneration Early stage disease 1 mo Baseline (Control) 1 mo after treatment OCU400 Single Injection (Intravitreal) Advanced stage disease 3 mo Baseline (Control) 1 mo after treatment OCU400 Single Injection (Intravitreal) 3 mo Baseline (Control) 1 mo after treatment Our First Potentially Transformative OCU400 Gene Therapy Candidate Bringing Hope to Single Injection Patients with No Current Treatment Options (Subretinal) rd7 mouse model ©2020 Ocugen. All Rights Reserved. 15
Gene Therapy Manufacturing: Plagued by Backlog and Timing Delays Cell & gene therapy manufacturing demand continues to increase • 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 Critical to find a Strategic and Reliable Partner that also shares costs ©2020 Ocugen. All Rights Reserved. Source: Manufacturing Cures: Infrastructure Challenges Facing Cell And Gene Therapy Developers 16 In Vivo June 2019 invivo.pharmaintelligence.informa.com
OCU400 Gene Therapy Manufacturing: Strategic Partnership with CanSinoBIO CanSinoBIO • Biotech company publicly-listed on Hong Kong exchange (6185.HK) with market cap of ~$2 Billion USD • 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 Partnership paves a path for Ocugen to advance OCU400 into the clinic with significantly reduced capital and resources ©2020 Ocugen. All Rights Reserved. 17
OCU410 (RORA-AAV): Dry AMD Dry Age-Related Macular Degeneration (AMD) • Leads to irreversible blindness due to degeneration of the retina ~9-10M patients in the US Inflammation Contributing Factors • Aging • Genetics Oxidative RORA Stress Normal Retina • Environmental Factors Lipid Peroxidation Currently no approved treatment for Dry AMD Dry AMD ©2020 Ocugen. All Rights Reserved. Source: https://www.brightfocus.org/macular/article/age-related-macular-facts-figures 18
OCU200: Diabetic Macular Edema (DME) Diabetic Retinopathy (DR) Wet AMD Novel Biologic Offering Benefits Beyond Anti-VEGF 19
OCU200: Tumstatin-Transferrin Fusion Protein • Inhibits new blood vessel formation • Anti-inflammatory • Anti-oxidative DME/DR Wet AMD Wet AMD Oxygen-Induced Retinopathy (OIR) Mouse Model In-Vivo Laser-Induced Rat CNV Model In-Vivo Laser-Induced Mouse CNV Model 80 Neovascular area (normalized) * 150 ** ** 60 100 50 40 0 Leaky CNV (%) lesions -50 Vehicle E y le a OCU200 OCU200 + NV (% of control retina NV) g g g µ µ µ g) Tumstatin OCU200 Avastin Eylea 5 µ PBS Eylea 2.5 10 Vehicle OCU200 (40 Effect of OCU200 intravitreal treatments on Neovascularization (NV). Data are * indicates p<0.05 when compared to PBS and/or tumstatin treatment Data expressed as percentage of CNV lesions on presented as mean± SD. Filled circles represent data points from individual eyes † indicates p<0.05 when compared to Avastin; CNV lesions measured Day 10 after treatment. Laser induction & * P < 0.05, ** P < 0.01 (n = 9-10 eyes per group) on day 14 after treatment treatment start on Day 0 OCU200 Demonstrated Superior Efficacy Compared to Existing Anti-VEGF Therapies ©2020 Ocugen. All Rights Reserved. 20
Summary of Near-Term Milestones OCU300 OCU400 (NR2E3-AAV) OCU200 ocular GVHD Retinal Degenerative Diseases DME, DR, Wet AMD (Phase 3 small molecule) (gene therapy) (novel biologic) Dec 2019: 50% Enrollment Achieved Feb 2019: Pre-IND Meeting • 2019-2020: Continue IND-Enabling Studies • 1H2020: Expected Completion of Feb 2019: ODD for NR2E3 Mutation- Enrollment Associated Retinal Diseases • 2021: Target Phase 1/2a Clinical Trial • 2H2020: Topline Results Expected 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 Note: Check mark () denotes completed milestone. All other milestones are anticipated future milestones. ©2020 Ocugen. All Rights Reserved. 21
A Bold Vision to Treat the Whole Eye For more information, contact: Kelly Beck Vice President, Investor Relations & Administration kelly.beck@ocugen.com