Ocugen, Inc.

Good morning, and welcome to the Ocugen conference call. At this time, all participants are in a listen-only mode. A question and answer session will follow the presentation. If anyone should require operator assistance during the conference, please press star then zero on your telephone keypad. Please note this conference is being recorded. I will now turn the conference over to Lisa DeCenza, Vice President of Integrated Communications at Lavoie Health Science, to introduce the Ocugen team. You may begin.

Thank you, operator. I'd like to welcome you to our conference call. With me today are Ocugen's chairman and CEO, Dr. Shankar Musunuri, and our CFO and head of corporate development, Sanjay Subramanian. Earlier this morning, Ocugen issued a press release including a business update and full year 2020 financial results. We encourage listeners to review the press release, which is available on the Ocugen website, at www.ocugen.com. This call is also being recorded and a replay will be available on the investor section of the Ocugen website for approximately 45 days. Before we begin our formal comments, I'll remind you that various remarks we make today constitute 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, regulatory submissions, and regulatory authorizations or approvals. 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, 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 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 US Food and Drug Administration, the FDA, will be satisfied with the design of and results from preclinical and clinical studies of Covaxin, which have been conducted by Bharat Biotech in India, whether and when any biologics license and or emergency use authorization applications may be filed in the United States for Covaxin, whether and when any such applications may be approved by the FDA, decisions by the FDA impacting labeling, manufacturing processes, safety, and or other matters that could affect the availability or commercial potential of Covaxin in the United States, 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. Except as required by law, we assume 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 webcast. You should read carefully the risks and uncertainties described in today's press release, as well as the risk factors included in our filings with the SEC. Note that we intend to file our Form 10-K with the SEC tomorrow. Any information we provide on this conference call is provided only as of the day of this call, March 18, 2021, and we undertake no obligation to update any forward-looking statements as we may make on this call on account of new information, future events, or otherwise. I will now turn the call over to Ocogen's Chairman and CEO, Dr. Shankar Musunuri.

Thank you, Lisa. Good morning, everyone, and thank you for joining. Since this time last year, when we hadn't grasped the full impact of the pandemic and what it would mean in our daily lives, we at Ockogen have made it our personal mission to be part of the solution to help end COVID-19. Through our partnership with Park Biotech, a leading vaccine developer based in India, we are working to potentially bring the COVID-19 vaccine to the United States. Covaxin was developed in partnership between the government of India and Park Biotech and was granted approval for emergency use authorization by the Indian authorities and is currently part of the largest vaccine distribution program in the world. Several million people have received this vaccine in India. It has an excellent safety track record to date. Leading journals such as Nature and Lancet have published the rigorous scientific research conducted on Covaxin to date, which includes preclinical and human phase 1 and phase 2. Covaxin provides an excellent opportunity for oxygen to enter the infectious disease market. It is different from other COVID-19 vaccine options currently authorized for emergency use in the United States. These vaccines rely on new mRNA or adenovirus technology, while Covaxin utilizes a traditional approach as an advanced stage whole variant inactivated vaccine using a viral cell manufacturing platform. This is the same technology that has been successful in producing the polio vaccine for infants globally for decades. Another key differentiator is that Covaxin induces the immune system to target the whole virus, which we believe reduces the possibility of mutant virus escape. The other currently available options in the United States target just the spike antigen to elicit an antibody response, but Covaxin has been shown to induce immune responses against multiple protein antigens, including the spike protein, receptor binding domain, and the nucleocapsid protein of the SARS-CoV-2 virus, along with the strong cellular responses. In fact, data demonstrating Covaxin's ability to neutralize the UK variant of SARS-CoV-2 was published recently. On the data front on March 3rd, we announced that the Covaxin was found to be 81% effective in an interim analysis of the Phase III clinical trial of nearly 26,000 participants in India. Not only did Covaxin demonstrate a high clinical efficacy trend against COVID-19, but it also showed significant immunogenicity against the rapidly emerging UK variant. and has potential to be effective against other emerging variants, too. The interim analysis included a preliminary review of the safety database, which showed that severe, serious, and medically-attended adverse events occurred at low levels and were balanced between vaccine and placebo groups. The Phase III trial, which is being conducted by Bharat Biotech, will result in the final efficacy analysis at 130 confirmed cases. Currently three EUAs for the other COVID-19 vaccine products in the United States do not authorize use in children under age 16. Covaxin's phase two results cover adolescents ages 12 plus. There are over 16 million children in the US between ages of 12 and 16, and most of them must attend middle school or high school. We are planning to use our existing data to potentially cover this age group in our EUA offering a potential significant immediate benefit to vaccinate children. We will consider initiating US clinical trials of Covaxin in additional patient populations, including pediatric and high-risk studies. Oxygen is in active discussions with the FDA to continue to develop the regulatory pathway for Covaxin vaccine candidate EUA application. Based on our discussions, we are planning to file the EUA application in April on additional efficacy and safety data from Phase III clinical trial. We are also in discussions with BARDA, the Biomedical Advanced Research and Development Authority, regarding their role in procuring medical countermeasures for the Strategic National Stockpile. With regard to manufacturing, under our agreement with Barth Biotech, they will supply the initial doses in the United States upon EUA approval. We are already working with an FDA-approved local CRO to establish release testing of the product for the U.S. market. Overall, we are aiming to make up to 100 million doses available this year to support the U.S. COVID-19 immunization program. We are in discussions with potential U.S. manufacturers and planning to work with Bharat Biotech on technology transfer once the initial doses are supplied. Importantly, Covaxin offers additional benefits for stockpiling, storing, and distribution. Because it will be shipped in a ready-to-use liquid formulation, we'll be able to use existing vaccine supply chain channels. Covaxin has an expected shelf life of up to two years in a normal refrigerator temperature and up to three months at room temperature. In summary, we are excited to collaborate with Park Biotech to develop the Covaxin vaccine candidate for the U.S. market. Our management team, as well as the Vaccine Scientific Advisory Board and highly experienced vaccine advisors we assembled at Ockigen, have a strong relationship with the leadership at Bharat Biotech. This collaboration leverages Ockigen's vaccine expertise and R&D, manufacturing, regulatory, and commercialization capabilities in the United States. Under the terms of the agreement, Ockigen will have the US rights to the vaccine candidate and will be responsible for clinical development regulatory approval, including EUA, and commercialization in the United States, and will retain 45% of the net profits. Now, to shift our discussion to ophthalmology pipeline, we're on track to enter the clinic with our first gene therapy candidate, OQ400. Based on our modified gene therapy platform, which provides the potential to address multiple diseases with one product, we're planning to file an I&D to initiate two Phase 1-2 clinical trials later this year for gene mutation-associated retinal diseases. In February, we announced that the European Commission granted Orphan Medicinal Product designation for RQ400 for retinitis pigmentosa and Leber congenital hemorrhosis, which we believe further supports the potential of our modified gene therapy platform to treat many inherited diseases. Inherited retinal diseases associated with retinitis pigmentosa and Leber congenital hemorrhosis diseases are caused by mutations in over 175 genes. And it is impractical to develop therapies that are specific to each gene. ARQ400, a single product, has potential to treat both RP and LCA, including all mutations associated with 175 genes. On the manufacturing side, for ARQ400, we have completed our 200 liter commercial scale-up process. Finally, the development of our novel biologic program, Occu200, and gene therapy candidate, Occupotent, are both progressing well, and we are on track to be in the clinic for both programs next year. Since October 1st last year, we have raised gross proceeds of around $39 million through offerings of our common stock, and now we have a healthy balance sheet. We plan to continue to raise capital as needed to fund our co-vaccine development and ophthalmology pipeline development. We're also seeking potential funding from the U.S. government for co-vaccine development and supply. We made strong progress during 2020 toward our goals of developing a differentiated vaccine to save lives from COVID-19 and developing gene therapies for blindness diseases. We look forward to continuing our momentum in 2021 with the planned U.S. rollout of Covaxin, as well as filing an IND for RQ400 to move our first gene therapy program into the clinic. I will now turn the call over to Sanjay to provide our year-end 2020 financial update. Sanjay?