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): May 6, 2022
___________________________________________________________
(Exact Name of Registrant as Specified in its Charter)
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| (State or Other Jurisdiction of Incorporation) | (Commission File Number) | (I.R.S. Employer Identification Number) | ||||||||||||
(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 (see General Instruction A.2. below):
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading Symbol(s) | Name of each exchange on which registered | ||||||||||||
(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 ☐
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. ☐
Item 2.02 Results of Operations and Financial Condition.
On May 6, 2022, Ocugen, Inc. (the "Company") issued a press release announcing its financial results for the quarter ended March 31, 2022. The Company has scheduled a conference call and webcast for 8:30 a.m. eastern time on May 6, 2022 to discuss these financial results and business updates. The Company will use presentation materials in connection with the conference call and webcast, which presentation materials will be posted on the Company's website at www.ocugen.com. Copies of the press release and presentation materials are furnished herewith as Exhibit 99.1 and Exhibit 99.2, respectively, to this Current Report on Form 8-K and incorporated herein by reference.
The information disclosed under Item 2.02 of this Current Report on Form 8-K, including Exhibit 99.1 and Exhibit 99.2, is being furnished and shall not be deemed "filed" for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the "Exchange Act"), or otherwise subject to the liabilities of that section, and shall not be deemed to be incorporated by reference in any Company filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing
Item 8.01 Other Events.
Attached as Exhibit 99.3 and incorporated herein by reference is a presentation that the Company will post on its website on May 6, 2022 and may use from time to time in presentations or discussions with investors, analysts, and other parties.
Item 9.01 Financial Statements and Exhibits.
The following exhibits are being filed or furnished (as applicable) herewith:
(d) Exhibits
| Exhibit No. | Document | |||||||
| 99.1 | ||||||||
| 99.2 | ||||||||
| 99.3 | ||||||||
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document). | |||||||
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: May 6, 2022
| OCUGEN, INC. | ||||||||
| By: | /s/ Shankar Musunuri | |||||||
| Name: Shankar Musunuri | ||||||||
| Title: Chief Executive Officer and Chairman | ||||||||
2
Exhibit 99.1
Ocugen Provides Business Update and First Quarter 2022 Financial Results
Conference Call and Webcast Today at 8:30 a.m. ET
•OCU400 Phase 1/2 clinical trial for groundbreaking modifier gene therapy for the treatment of NR2E3 and RHO-related retinitis pigmentosa is advancing after DSMB Review
•Ocugen’s exclusive territory for COVAXIN™ (BBV152) marketing expanded to include all of North America
MALVERN, Pa. — May 6, 2022 (GLOBE NEWSWIRE) — Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene therapies, biologicals, and vaccines, today reported first quarter 2022 financial results along with a general business update.
“We’ve made significant progress this quarter across multiple areas and we remain confident in the long-term opportunities and growth that we believe our pipeline will unlock” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “I am very proud of our dedicated team for moving our breakthrough gene therapy into the clinic and for their commitment to advancing COVAXIN™ to fight against COVID-19.”
Business Updates
•OCU400 Clinical Trial — The Company achieved a key milestone in its Phase 1/2 clinical trial for OCU400 of “first patient, first dose” in late March 2022. The Data and Safety Monitoring Board for the clinical trial reviewed safety data based on dosing to date and recommended that the study proceed with enrolling the remaining study subjects in the current cohort at the target dose level. A second patient was dosed in May 2022.
•COVAXIN™ Rights Expanded to include Mexico — In April 2022, the Company expanded its rights to develop, manufacture, and commercialize COVAXIN™ to include Mexico, where the vaccine is already authorized for emergency use in adults and is currently under review by local regulators for emergency pediatric use. The company is now working on commercializing the vaccine in Mexico. The Company’s exclusive territory for COVAXIN™ now encompasses the entire North American region.
•COVAXIN™ in the United States — The Company is actively engaged in discussions with the U.S. Food and Drug Administration (the “FDA”) to address its questions and resume the Company’s Phase 2/3 immuno-bridging and broadening clinical trial for COVAXIN™, OCU-002. In addition, the Company intends to continue working with the FDA to finalize the additional studies required for a Biologics License Application (“BLA”).
•COVAXIN™ in Canada — Discussions with Health Canada regarding COVAXIN™ are ongoing. The Company is in discussions with Canadian officials regarding financial support for the potential acquisition of a manufacturing facility in Belleville, Ontario, Canada that would be transformed to become a manufacturing and research and development hub to support both the Company’s current and future product candidate pipeline.
•Organizational Growth — Nearly 20 employees joined Ocugen in the first quarter to fill key roles that support operational needs, including clinical trials and regulatory milestones. The number of employees now totals 79.
•Addition to the Board of Directors — In March 2022, Marna C. Whittington, Ph.D., former Chief Executive Officer of Allianz Global Investors Capital, joined the Company’s Board of Directors. Dr. Whittington is a renowned financial sector leader and her experience and expertise will be exceedingly important to the Company’s growth strategy.
First Quarter 2022 Financial Results
•The Company’s cash, cash equivalents, and restricted cash totaled $129.9 million as of March 31, 2022, compared to $95.1 million as of December 31, 2021. The Company had 215.6 million shares of common stock outstanding as of March 31, 2022.
•Research and development expenses for the three months ended March 31, 2022 were $7.9 million compared to $2.9 million for the three months ended March 31, 2021. General and administrative expenses for the three months ended March 31, 2022 were $10.1 million compared to $4.2 million for the three months ended March 31, 2021. Ocugen reported a $0.09 net loss per share for the three months ended March 31, 2022 compared to a $0.04 net loss per share for the three months ended March 31, 2021.
Conference Call and Webcast Details
Ocugen has scheduled a conference call and webcast for 8:30 a.m. eastern time today to discuss the financial results and recent business updates. Ocugen's senior management team will host the call, which will be open to all listeners. There will also be a question-and-answer session following the prepared remarks.
The call can be accessed by dialing (844) 873-7330 (U.S.) or (602) 563-8473 (international) and providing the conference ID 6995784. To access a live audio webcast of the call on the “Investors” section of the Ocugen website, please click here. A replay of the webcast will be archived on Ocugen’s website for approximately 45 days following the call.
About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene therapies, biologicals and vaccines that improve health and offer hope for people and global communities. We are making an impact through courageous innovation, taking science in new directions in service of patients. Our breakthrough modifier gene therapy platform has the potential to treat multiple diseases with one drug and we are advancing research in other therapeutic areas to offer new options for people with unmet medical needs. Discover more at www.ocugen.com and follow us on Twitter and LinkedIn.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene therapies, biologicals and vaccines that improve health and offer hope for people and global communities. We are making an impact through courageous innovation, taking science in new directions in service of patients. Our breakthrough modifier gene therapy platform has the potential to treat multiple diseases with one drug and we are advancing research in other therapeutic areas to offer new options for people with unmet medical needs. Discover more at www.ocugen.com and follow us on Twitter and LinkedIn.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. Ocugen may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such forward-looking statements include, but are not limited to, statements about Ocugen’s progress in advancing the review of COVAXIN™ and its other product candidates with the FDA, and are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements, including, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, ability to timely enroll clinical trial participants, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates; the risk that Health Canada does not accept its New Drug Submission (“NDS”) for COVAXIN™ or that Ocugen may not be able to adequately resolve the deficiencies noted by Health Canada with respect to its NDS, for which Ocugen has provided responses that are currently under review by Health Canada; the risk that Ocugen may not be able to successfully commercialize COVAXIN™ in Mexico for adults over the age of 18 pursuant to Ocugen’s agreement with Bharat Biotech and the risk that Ocugen does not obtain emergency pediatric use for COVAXIN™ in Mexico for children between two and 18 years of age on a timely basis, if at all; the risk that the FDA does not lift the clinical hold on Ocugen’s Phase 2/3 immuno-bridging and broadening clinical trial for COVAXIN™ on a timely basis, if at all; risks associated with preliminary and interim data, including the possibility of unfavorable new clinical trial data and further analyses of existing clinical trial data; the risk that the results of in-vitro studies will not be duplicated in human clinical trials; the risk that clinical trial data is subject to differing interpretations and assessments, including during the peer review/publication process, in the scientific community generally, and by regulatory authorities; whether and when data from Bharat Biotech's clinical trials will be published in scientific journal publications and, if so, when and with what modifications; whether the data and results from the preclinical and clinical studies of COVAXIN™, which have been conducted by Bharat Biotech in India, will be accepted by regulatory authorities or otherwise sufficient to support Ocugen’s submissions for regulatory approvals or authorizations in the United States, Canada or Mexico; the size, scope, timing and outcome of any additional clinical trials or studies that Ocugen may be required to conduct to support regulatory approvals or authorizations; any additional chemistry, manufacturing, and controls information that Ocugen may be required to submit to regulatory authorities ; whether and when a BLA for COVAXIN™ will be submitted to or approved by the FDA; the risk that Ocugen may not be able to successfully negotiate and execute definitive transaction agreements for the acquisition of the manufacturing site on acceptable terms, if at all, and the ultimate terms and timing for closing of the transactions contemplated thereby; the risk that Ocugen will not be able to successfully close the acquisition of the manufacturing site; risks associated with the planned development and refurbishing of the manufacturing site, including that the expected costs for such development may be greater than currently contemplated or that the planned development may take longer than expected or fail to be completed on a timely basis, if at all; and the risk that Ocugen will not be able to scale production for such manufacturing site to adequately support manufacturing of its product candidates or the other products that may in the future be manufactured at such manufacturing site; whether developments with respect to the COVID-19 pandemic will affect the regulatory pathway available for vaccines in the United States, Canada, Mexico or other jurisdictions; market demand for COVAXIN™ in the United States, Canada or Mexico; decisions by the regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of COVAXIN™ in the United States, Canada or Mexico, including development of products or therapies by other companies. These and other risks and uncertainties are more fully described in Ocugen’s periodic filings with the Securities and Exchange Commission (“SEC”), including the risk factors described in the section entitled “Risk Factors” in
the quarterly and annual reports that Ocugen files with the SEC. Any forward-looking statements that Ocugen makes in this press release speak only as of the date of this press release. Except as required by law, Ocugen assumes no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.
Ocugen Contact:
Ken Inchausti
Head, Investor Relations & Communications
+1 484 237 3398
ken.inchausti@ocugen.com
Please submit investor-related inquiries to: IR@ocugen.com
(tables to follow)
OCUGEN, INC.
CONSOLIDATED BALANCE SHEETS
(in thousands)
(Unaudited)
March 31, 2022 | December 31, 2021 | ||||||||||
| Assets | |||||||||||
| Current assets | |||||||||||
| Cash and cash equivalents | $ | 129,771 | $ | 94,958 | |||||||
| Prepaid expenses and other current assets | 8,256 | 7,688 | |||||||||
| Total current assets | 138,027 | 102,646 | |||||||||
| Property and equipment, net | 1,921 | 1,164 | |||||||||
| Restricted cash | 151 | 151 | |||||||||
| Other assets | 1,628 | 1,800 | |||||||||
| Total assets | $ | 141,727 | $ | 105,761 | |||||||
| Liabilities and stockholders' equity | |||||||||||
| Current liabilities | |||||||||||
| Accounts payable | $ | 3,896 | $ | 2,312 | |||||||
| Accrued expenses | 3,537 | 4,325 | |||||||||
| Operating lease obligations | 254 | 363 | |||||||||
| Total current liabilities | 7,687 | 7,000 | |||||||||
| Non-current liabilities | |||||||||||
| Operating lease obligations, less current portion | 1,180 | 1,231 | |||||||||
| Long term debt, net | 1,731 | 1,712 | |||||||||
| Total liabilities | 10,598 | 9,943 | |||||||||
| Stockholders' equity | |||||||||||
| Convertible preferred stock | 1 | 1 | |||||||||
| Common stock | 2,158 | 1,995 | |||||||||
| Treasury stock | (48) | (48) | |||||||||
| Additional paid-in capital | 278,704 | 225,537 | |||||||||
| Accumulated deficit | (149,686) | (131,667) | |||||||||
| Total stockholders' equity | 131,129 | 95,818 | |||||||||
| Total liabilities and stockholders' equity | $ | 141,727 | $ | 105,761 | |||||||
OCUGEN, INC.
CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except share and per share amounts)
(Unaudited)
| Three months ended March 31, | |||||||||||
| 2022 | 2021 | ||||||||||
| Operating expenses | |||||||||||
| Research and development | $ | 7,915 | $ | 2,872 | |||||||
| General and administrative | 10,119 | 4,185 | |||||||||
| Total operating expenses | 18,034 | 7,057 | |||||||||
| Loss from operations | (18,034) | (7,057) | |||||||||
| Other income (expense), net | 15 | (20) | |||||||||
| Net loss and comprehensive loss | $ | (18,019) | $ | (7,077) | |||||||
| Shares used in calculating net loss per common share — basic and diluted | 205,693,498 | 186,298,122 | |||||||||
| Net loss per share of common stock — basic and diluted | $ | (0.09) | $ | (0.04) | |||||||
Courageous Innovation Q1 2022 Update May 2022 NASDAQ: OCGN
Forward Looking Statement 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. Ocugen may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such forward-looking statements include, but are not limited to, statements about Ocugen’s progress in advancing the review of COVAXIN™ and its other product candidates with the FDA, and are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements, including, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, ability to timely enroll clinical trial participants, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates; the risk that Health Canada does not accept its New Drug Submission (“NDS”) for COVAXIN™ or that Ocugen may not be able to adequately resolve the deficiencies noted by Health Canada with respect to its NDS, for which Ocugen has provided responses that are currently under review by Health Canada; the risk that Ocugen may not be able to successfully commercialize COVAXIN™ in Mexico for adults over the age of 18 pursuant to Ocugen’s agreement with Bharat Biotech and the risk that Ocugen does not obtain emergency pediatric use for COVAXIN™ in Mexico for children between two and 18 years of age on a timely basis, if at all; the risk that the FDA does not lift the clinical hold on Ocugen’s Phase 2/3 immuno-bridging and broadening clinical trial for COVAXIN™ on a timely basis, if at all; risks associated with preliminary and interim data, including the possibility of unfavorable new clinical trial data and further analyses of existing clinical trial data; the risk that the results of in-vitro studies will not be duplicated in human clinical trials; the risk that clinical trial data is subject to differing interpretations and assessments, including during the peer review/publication process, in the scientific community generally, and by regulatory authorities; whether and when data from Bharat Biotech's clinical trials will be published in scientific journal publications and, if so, when and with what modifications; whether the data and results from the preclinical and clinical studies of COVAXIN™, which have been conducted by Bharat Biotech in India, will be accepted by regulatory authorities or otherwise sufficient to support Ocugen’s submissions for regulatory approvals or authorizations in the United States, Canada or Mexico; the size, scope, timing and outcome of any additional clinical trials or studies that Ocugen may be required to conduct to support regulatory approvals or authorizations; any additional chemistry, manufacturing, and controls information that Ocugen may be required to submit to regulatory authorities ; whether and when a BLA for COVAXIN™ will be submitted to or approved by the FDA; the risk that Ocugen may not be able to successfully negotiate and execute definitive transaction agreements for the acquisition of the manufacturing site on acceptable terms, if at all, and the ultimate terms and timing for closing of the transactions contemplated thereby; the risk that Ocugen will not be able to successfully close the acquisition of the manufacturing site; risks associated with the planned development and refurbishing of the manufacturing site, including that the expected costs for such development may be greater than currently contemplated or that the planned development may take longer than expected or fail to be completed on a timely basis, if at all; and the risk that Ocugen will not be able to scale production for such manufacturing site to adequately support manufacturing of its product candidates or the other products that may in the future be manufactured at such manufacturing site; whether developments with respect to the COVID-19 pandemic will affect the regulatory pathway available for vaccines in the United States, Canada, Mexico or other jurisdictions; market demand for COVAXIN™ in the United States, Canada or Mexico; decisions by the regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of COVAXIN™ in the United States, Canada or Mexico, including development of products or therapies by other companies. These and other risks and uncertainties are more fully described in Ocugen’s periodic filings with the Securities and Exchange Commission (“SEC”), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that Ocugen files with the SEC. Any forward-looking statements that Ocugen makes in this presentation speak only as of the date of this presentation. Except as required by law, Ocugen assumes no obligation to update forward-looking statements contained in this presentation whether as a result of new information, future events, or otherwise, after the date of this presentation.
We’re Here to Make an Impact Through Courageous Innovation 3
4 Business update on COVAXIN™ (BBV152) The Company is working with the FDA to address questions arising from WHO’s inspection of BBIL’s manufacturing facility in order to continue with OCU- 002 clinical trial Planning to commercialize COVAXIN™ in Mexico in 2022, following the broadening of the Ocugen Territory which now covers all of North America Teams are poised for continuing OCU-002 once path forward is clarified with regulators
5 Business update on the Ophthalmology Portfolio First and second patients dosed in Phase 1/2 clinical trial studying OCU400 for the treatment of retinitis pigmentosa resulting from genetic mutations of NR2E3 and RHO Data Monitoring and Safety Board recommended OCU400-101 clinical trial to proceed with enrolling the remaining study subjects in the current cohort at the target dose level OCU410 initiating IND-enabling studies to support a future Phase 1/2 clinical trial
Financial Update 6
7 Financial Update Statement of Operations Three months ended March 31, 2022 2021 Research and development expense $7.9 $2.9 General and administrative expense 10.1 4.2 Net loss $(18.0) $(7.1) Net loss per share of common stock — basic and diluted $(0.09) $(0.04) Balance Sheet Data March 31, 2022 December 31, 2021 Cash, cash equivalents, and restricted cash $129.9 $95.1 Debt $1.7 $1.7 Shares outstanding 215.6 199.4 Unaudited; in millions, except per share amounts
Questions & Answers 8
May 2022 NASDAQ: OCGN Thank you!
Courageous Innovation May 2022 NASDAQ: OCGN
Forward Looking Statement 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,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such forward-looking statements include information about qualitative assessments of available data, potential benefits, expectations for clinical trials, and anticipated timing of clinical trial readouts and regulatory submissions. This information involves risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, including the risk that such dates are not met due to impacts from the ongoing COVID-19 pandemic, as well as risks associated with preliminary and interim data, including the possibility of unfavorable new clinical trial data and further analyses of existing clinical trial data; the risk that the results of in-vitro studies will not be duplicated in human clinical trials; the risk that clinical trial data are subject to differing interpretations and assessments, including during the peer review/publication process, in the scientific community generally, and by regulatory authorities; whether and when data from Bharat Biotech’s clinical trials will be published in scientific journal publications and, if so, when and with what modifications; whether the data and results from preclinical and clinical studies of COVAXIN™, which have been conducted by Bharat Biotech in India, will be accepted by the U.S. Food and Drug Administration (“FDA”) or otherwise sufficient to support our Investigational New Drug applications (“IND”) or planned Biologics License Applications (“BLA”), as applicable; whether and when we are able to resolve the clinical hold on our Phase 2/3 immuno-bridging and broadening clinical trial for COVAXIN ™; the size, scope, timing and outcome of any additional trials or studies that we may be required to conduct to support a for COVAXIN™, including our Phase 2/3 immuno-bridging and broadening clinical trial and planned safety-bridging clinical trial; any additional chemistry, manufacturing, and controls information that we may be required to submit; whether and when a BLA for COVAXIN™ will be submitted to the FDA; whether and when a BLA may be approved by the FDA, whether a New Drug Submission application may be approved by Health Canada, and whether the additional information that we provide to Health Canada will be sufficient to support an approval by Health Canada of COVAXIN™ and any delays associated therewith; our ability to successfully commercialize COVAXIN™ in Mexico for adults over the age of 18 pursuant to our agreement with Bharat Biotech, and whether and when we will obtain Emergency Use Authorization approval for COVAXIN™ in Mexico for children between 2 and 18 years of age; the authorizations or approvals will depend on myriad factors, including making a determination as to whether the vaccine candidate’s benefits outweigh its known risks and determination of the vaccine candidate’s efficacy and, if authorized or approved, whether it will be commercially successful; whether developments with respect to the COVID-19 pandemic will affect the regulatory pathway available for vaccines in the United States, Canada, Mexico or other jurisdictions; manufacturing capabilities, manufacturing capacity, and supply restrictions, including whether sufficient doses of COVAXIN™ can be manufactured or supplied within our projected time periods; market demand for COVAXIN™ in the United States, Canada or Mexico; decisions by the FDA, Health Canada or the Federal Commission for Protection against Sanitary Risks in Mexico impacting labeling, manufacturing processes, safety, and/or other matters that could affect the availability or commercial potential of COVAXIN™ in the United States, Canada or Mexico, including development of products or therapies by other companies. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (“SEC”), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this presentation speak only as of the date of this presentation. Except as required by law, we assume no obligation to update forward-looking statements contained in presentation whether as a result of new information, future events, or otherwise, after the date of this presentation. .
We’re Here to Make an Impact Through Courageous Innovation 3 Mission At Ocugen, we are developing novel solutions to medical challenges, approaching healthcare innovation with purpose and agility to deliver new options for people facing disease. Vision We are fostering a future where no one feels hopeless in the face of disease. From genetic disorders to new diseases, our expertise and tenacity are creating choices – for people and for global communities. Pioneering a breakthrough modifier gene therapy for several genetic forms of vision impairment Innovating a novel biologic to treat eye diseases that can lead to vision loss for millions of people Co-developing a COVID-19 vaccine
4 Pipeline Overview Asset/Program Indication Status Vaccine COVAXIN™ (BBV152) Whole-Virion Inactivated Vaccine COVID-19 • EUA for adults in Mexico; EUA for 2–18 year olds under review* • US Phase 2/3 clinical trial* (Temporarily paused dosing/clinical hold) • Health Canada NDS under review* Modifier Gene Therapy Platform OCU400 *** AAV-hNR2E3 Gene mutation-associated retinal degeneration** NR2E3 Mutation Phase 1/2 RHO Mutation Phase 1/2 CEP290 Mutation To be submitted PDE6B Mutation To be submitted OCU410 AAV-hRORA Dry Age-Related Macular Degeneration (Dry AMD)** Preclinical Novel Biologic OCU200 Transferrin – Tumstatin Diabetic Macular Edema Preclinical Diabetic Retinopathy Preclinical Wet Age-Related Macular Degeneration (Wet AMD) Preclinical * Based on Bharat Biotech-sponsored clinical trials in India ** No approved therapies exist https://www.aao.org/eye-health/diseases/retinitis-pigmentosa-treatment | https://www.aao.org/eye-health/diseases/amd-treatment *** ORPHAN DRUG DESIGNATION in the US; Broad ORPHAN MEDICINAL PRODUCT DESIGNATION by the EC for the treatment of retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA)
5 COVAXIN™ (BBV152) A Whole-Virion Inactivated COVID-19 Vaccine Candidate Licensed from Bharat Biotech (BBIL) for the US and Canadian Markets
Product Profile 6 COVAXIN™ (BBV152): Whole Virion Inactivated SARS- CoV-2 Antigen & Adjuvant: 6ug/SHD + Algel-IMDG (TLR7/8 Agonist) Dose Level and Regimen 6 ug per 0.5 mL suspension; 2 Doses: Day 0 & Day 28 Presentation Ten doses per vial Expected Shelf Life Two years in storage conditions of 2°- 8°C and stable for six months at room temp (25°C) Proposed indication Prevention of COVID-19 caused by SARS-CoV-2 Target population Pediatric: 2-18 years of age Adult: 18 years of age and older
7 Why COVAXIN™ (BBV152)? Designed to augment our North American arsenal of vaccines against COVID-19 • Adult and pediatric phase 2/3 data suggest both humoral & cellular responses generated against multiple viral proteins • Data support that the vaccine induces a Th1 response (cell-mediated immunity) which can be vital for durable protection • Data demonstrate strong safety profile within adult and pediatric populations • Technology platform used to produce Polio, Influenza and Rabies vaccines KNOWN SAFETY PROFILE USING VERO CELL PLATFORM • Phase 3 data suggest prevention of hospitalizations caused by COVID-19 • Booster dose provides robust neutralizing antibody responses against Omicron and Delta variants DESIGNED FOR BROAD SPECTRUM IMMUNE RESPONSE RESULTS SHOW PREVENTION OF SEVERE COVID-19 DISEASE • 10 dose vial that can be stored and shipped at 2°- 8° C with a 2-year shelf life and 6-month stability at room temperature TRANSPORTATION AND STORAGE EASE Image for illustrative purposes only
8 Why COVAXIN™ (BBV152)? Phase 3 Clinical Trial Highlights 77.8% 93.4% 65.2% Overall efficacy Efficacy vs severe disease Efficacy vs B.1.617.2 (Delta) Serious Adverse Events Adverse Events Placebo Arm Adverse Events COVAXIN™ Arm 12.4% <0.5% n = 25,800 participants Participants recruited between November 2020 and January 2021 across 25 sites Two doses, 28 days apart Source: Ella, Reddy, Blackwelder, Potdar, Yadav, Sarangi et al. (2021) Efficacy, safety, and lot-to-lot immunogenicity of an inactivated SARS-CoV-2 vaccine (BBV152): interim results of a randomised, double-blind, controlled, phase 3 trial; The Lancet. Advanced online publication. https://doi.org/10.1016/S0140-6736(21)02000-6 Accessed November 11, 2021
9 Pathway for COVAXIN™ (BBV152) development NCT: 05258669 Immuno-bridging and broadening (OCU-002) Booster and Safety* Proposed Interim Analysis BLA Submission Window *Protocol to be submitted OCU-002 A Phase 2/3, Observer-Blind, Immuno-bridging, and Broadening Study of a Whole, Inactivated Severe Acute Respiratory Syndrome Coronavirus (SARS-CoV-2) Vaccine (BBV152) in Healthy Adults Study Type Interventional (Clinical Trial) Estimated Enrollment 400 participants Allocation Randomized Intervention Model Parallel assignment Intervention Model Description 1:1 randomization ratio Primary Purpose Prevention
10 MODIFIER GENE THERAPY PLATFORM Breakthrough technology designed to address many rare diseases as well as complex diseases that affect millions
11 Our Focus: Nuclear Hormone Receptor Genes (NHRs) *References: https://pubmed.ncbi.nlm.nih.gov/28556246/ | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5409218/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4339951/ | https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0183526 NHRs are modulators of retinal development & function, acting as “master genes” in the retina Molecular reset of key transcription factors and associated gene networks – retinal homeostasis Gene modifier concept including, its impact on clinical phenotypes, is well known in other disease areas, such as cystic fibrosis and spinal muscular atrophy
12 Our Vision: Modifier Gene Therapy vs Traditional Gene Augmentation Normal gene X Gene Augmentation: Transfer functional version of a non-functional gene into the target cells. Modifier Gene Therapy: Designed to introduce a functional gene to modify the expression of many genes, gene-networks and regulate basic biological processes in retina. 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 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 product Ability to recoup development costs over multiple therapeutic indications Traditional Gene Therapy OCU400 NR2E3 Mutation-Associated Retinal Disease Rhodopsin Mutation-Associated Retinal Disease CEP290 Mutation-Associated Retinal Disease PDE6B Mutation-Associated Retinal Disease Broad Spectrum Therapy for RP ONE Disease GENE X cell GENE X Cell with mutated/nonfunctioning gene X GENE X GENE X cell GENE X GENE X cell GENE X cell GENE M Cell with mutated/nonfunctioning gene(s) other than modifier gene GENE X GENE M Modifier gene M Cell with normal function cell We plan to address a number of diseases using the same Modifier Gene product. cell
13 Our Proof of Principle: Published in Nature Gene Therapy Efficacy results shown in 5 unique mouse models of RP Technology developed at Harvard Medical School, Dr. Neena Haider’s Lab Study suggests potency of modifier gene therapy to elicit broad-spectrum therapeutic benefits in early and advanced stages of RP Results suggest evidence of vision rescue in Early & Advanced Stages of disease https://www.nature.com/articles/s41434-020-0134-z Important milestone for development of therapy; demonstrated proof of principle Protection elicited in multiple animal models of degeneration caused by different mutations Potential to represent first broad-spectrum therapy and to provide rescue even after disease onset
14 OCU400 – Pathway to Phase 3 Clinical Trials Just 30 days to receive FDA clearance for Phase 1/2 gene therapy clinical trial • NCT: 05203939 • Seven clinical trial sites being activated • Escalation study involving low, medium, high doses • First patient dosed by end of Q1 2022 • Periodic updates available starting in Q3 2022 • Enrollment concludes by YE 2022 OCU400 A Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa Associated With NR2E3 (Nuclear Receptor Subfamily 2 Group E Member 3) and RHO (Rhodopsin) Mutations Study Type Interventional (Clinical Trial) Estimated Enrollment 18 participants Allocation Non-randomized Intervention Model: Sequential assignment Masking: None (Open Label) Primary Purpose: Treatment
15 First and second patients dosed in Phase 1/2 clinical trial studying OCU400 for the treatment of retinitis pigmentosa resulting from genetic mutations of NR2E3 and RHO Successfully completed manufacturing at commercial scale (200L) at CanSinoBio to support clinical studies Expanded manufacturing agreement with CanSinoBio to include support for OCU410 Summary of activities at Ocugen Planning to commercialize COVAXIN™ in Mexico in 2022, following the broadening of the Ocugen Territory which now covers all of North America The Company is working with the FDA to address questions arising from WHO’s inspection of BBIL’s manufacturing facility in order to continue with OCU-002 clinical trial Health Canada NDS review continues COVAXIN™ (BBV152) OCU400/410 » » » » » »
16 Experienced Leadership Shankar Musunuri, PhD, MBA Chairman, CEO, & Co-Founder Zara Gaudioso, SHRM-CP AVP, Human Resources, Chief of Staff Nirdosh Jagota, PhD SVP, Regulatory Affairs, Compliance and Safety Jessica Crespo, CPA Chief Accounting Officer & SVP, Finance Bruce Forrest, MD Acting Chief Medical Officer J.P. Gabriel SVP, Technical Operations Huma Qamar, MD, MPH, CMI AVP, Clinical Development Mike Shine SVP, Commercial Arun Upadhyay, PhD SVP, Research & Development
May 2022 NASDAQ: OCGN Thank you!