Ocugen, Inc.

Good morning, and welcome to the Ocugen's first quarter 2023 financial results and business update call. Please note that this call is being recorded at this time. All participant lines are in a listen-only mode. Following the speaker's commentary, there will be a question-answer session. I will now turn the call over to Sharon Cho, Ocugen's Head of Investor Relations. You may begin.

Thank you, Monteith. Joining me today are Ocugen's chairman, CEO, and co-founder, Dr. Shankar Nusinuri, who will provide a business update, and our chief financial officer and chief business officer, Quan Vu, who will provide a financial update. Earlier this morning, we issued a press release detailing business and operational highlights for the first quarter of 2023. We encourage listeners to review the press release which is available on our website at www.ocugen.com. This call is being recorded, and a replay with the accompanying slide presentation will be available on the Investors section of the Ocugen website for approximately 45 days. This presentation contains forward-looking statements within the meaning of the Private Security Litigation Reform Act of 1995, which are subject to risks and uncertainty. We may, in some cases, use terms such as predict, believe, potential, propose, continue, estimate, anticipate, expect, plan, intend, may, could, might, will, should, or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties and may cause actual events or results to differ materially from our current expectations. Investors should familiarize themselves with the company's filings for complete details. Except as required by law, we assume 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. Finally, OccuGen's quarterly report on Form 10Q, covering the first quarter of 2023, will be filed soon after today's call. I will now turn the call over to Dr. Musuneri.

Thank you, Sharon. Good morning, and thank you all for joining us today. Looking at what we have achieved, Since we reported our 2022 fourth quarter and full year results, I'm kicking off today's call with a high sense of accomplishment and optimism for the future of oncogen. At the top of our list of highlights is the recent announcement of positive preliminary safety and efficacy results from the Phase 1-2 trial of our OQ400 program and the FDA's Orphan Drug Designation for our OQ410ST program to potentially treat ABCA4-associated retinopathies such as Stargardt disease. We also forged ahead in our pursuit of non-dilutive government funding to support our inhaled vaccines pipeline and submitted multiple proposals to various federal agencies. And we will begin seeking corporate partnerships for our gene therapies. I will also share updates on our ARCA-200 and NEOCAR programs later in my commentary. 2023 is off to a strong start, and you can see we remain on track to achieve the significant milestones for the year that we first shared with you during the last business update webcast. Our modified gene therapy approach continues to be the leading differentiator for oxygen. Unlike single gene replacement therapies, which only target one genetic mutation, we believe that our modifier gene therapy platform through its use of nuclear hormone receptors, represents a novel approach that has the potential to both address multiple retinal diseases caused by mutations in multiple genes with one product, and to address complex diseases that are potentially caused by imbalances in multiple gene networks. Currently, Ocugen has three modifier gene therapy programs. Ocu400 retinitis pigmentosa, and Leber congenital amaurosis, which affects approximately 125,000 patients in the U.S. living with any of more than 125 associated mutated genes. Ocu410 for dry age-related macular degeneration, a disease affecting approximately 10 million people in the U.S. alone. And Ocu410ST, for the treatment of ABCA4-associated retinopathies, including Stargardt, retinitis pigmentosa 19, RP19, and cone-rot dystrophy 3, CORD3, diseases affecting 44,000 Americans. We recently announced positive preliminary safety and efficacy results from the Phase I-II trial of RQ400 for the treatment of retinitis pigmentosa and Leber congenital neurosis. These preliminary positive results serve as the first clinical validation of the platform where patient responses across various genetic mutations support that RQ400 has the potential to transform the lives of many patients who are struggling with debilitating blindness diseases. This Phase I-II trial is a multicenter, open-label, dose-ranging study. We have enrolled a total of 18 RP patients in this study. with 10 subjects in the dose escalation and eight subjects in the expansion phase. The age of subjects enrolled to date ranges from 18 to 77 years across rhodopsin and NR2A3 gene mutations. We further expanded this Phase I-II trial to enroll LCA patients with the CEP290 gene mutation and pediatric patients with NR2A3, Rho, and CEP290 mutations. In cohort one, which is low dose, and cohort two, which is medium dose, a total of seven subjects with moderate to advanced vision impairment due to RP associated with Rho and NR2E3 gene mutations received a unilateral subretinal injection of either a low dose, which is 1.66 times 10 to the 10 VGs per mil, and cohort one, or medium dose, which is 3.33 times 10 to the 10 pgs per ml of RQ400 and cohort two respectively. In the preliminary data analysis, nine month follow up data for three subjects in cohort one and six month follow up data for one subject from cohort one and three subjects from cohort two were assessed. Overall preliminary results showed a favorable safety and tolerability profile for RQ400. Regarding efficacy, we looked at multi-luminescence mobility test, or MLMT, a primary efficacy endpoint used in clinical trials for an FDA-approved product in this disease area, and best corrected visual equity, or BCVA. Key efficacy outcomes from seven subjects demonstrated four key points, 100% of treated eyes, showed a stable or improved MLMT score trend. 71% of OQ400 treated eyes demonstrated a one or more lux level improvement in MLMT score compared to 29% of untreated eyes. 67% of OQ400 treated eyes in cohort one with a nine month follow up demonstrated a two or more lux level improvement in MLMT score compared to none of the untreated eyes. And 43% of RQ400 treated eyes demonstrated 8 to 11 letters of improvements in BCVA score compared to none of the untreated eyes. The early results from patients treated in the Phase I-II trial are encouraging and support the paradigm-changing potential of our modified gene therapy technology to address unmet medical needs for patients with RP and LCA. With this favorable safety profile and positive trend in efficacy signals, we are very eager to see longer-term data and to potentially initiate Phase III clinical trials in the US and EU. As I mentioned earlier, we received exciting news last week that the FDA granted Orphan Drug Designation for OCU410ST, AAV5, Aurora, for the treatment of ABCA4-associated retinopathies, including Stargardt, RP19, and CORD3 diseases. As a refresher, orphan drug designation is granted by the FDA to certain products that show promise in the treatment, prevention, or diagnosis of rare and serious diseases affecting fewer than 200,000 people in the United States. Additionally, the orphan drug designation status allows for a potential seven-year market exclusivity specifically to the designated orphan use following FDA approval. Other development incentives include the clinical protocol, design assistance, and potentially accelerated review times. This designation represents a noteworthy milestone in our effort to develop innovative treatments for inherited retinal diseases. And while OQ410-ST is intended to treat rare diseases, OQ410, also targeting the RORA gene network, is aimed at treating dry age-related macular degeneration that affects hundreds of millions of people across the globe. Using our modified gene therapy, we believe OQ410 potentially addresses shortcomings of current treatments for geographic atrophy that affects about 1 million people in the U.S., because it is a broad-spectrum approach that has potential as a one-time curative therapy with a single subretinal injection. Now, turning to vaccines. The OQ500 series of vaccines in development grants oxygen a distinct product candidate profile status that could significantly impact major global health obstacles and maximize our opportunity to serve broader patient markets. Parent COVID-19 vaccines are limited by a lack of durability and inability to stop transmission. As part of our commitment to address current gaps in the fight against COVID-19, we are developing a novel inhalation vaccine platform that includes OQ500, a bivalent COVID-19 inhaled vaccine, OQ510, a seasonal quadrivalent flu inhaled vaccine, and OCU520, a combination quadrivalent seasonal flu and bivalent COVID-19 inhaled vaccine. The OCU500 vaccine series is based on a novel chat platform designed to reduce transmission and protect against new variants with a potential durability up to one year. We decided to develop the flu vaccine in addition to addressing COVID-19 because flu will always be a health concern. There is also longer-term business potential as Americans continue to be regularly vaccinated against the flu. For the 2022 to 2023 flu season, over 50% of the U.S. population above six months of age received a seasonal flu shot, representing a market size of more than 170 million doses. To optimize resources across our diverse and critically needed development programs, To maintain shareholder value, our team has been busy in D.C. speaking with the government agencies to pursue non-dilutive funding opportunities for our RQ400 vaccine series. We have submitted multiple comprehensive proposals for review and consideration and maintain an ongoing dialogue with the respective agencies regarding the development of the inhaled vaccines platform. We look forward to updating you as we hear more. Last quarter, we submitted an investigation of new drug application, IND, with the U.S. Food and Drug Administration to initiate a Phase I trial of OCU-200 for treating diabetic macular edema, DME. The IND was placed on clinical hold by the FDA as part of its request for additional information related to chemistry, manufacturing, and controls prior to initiating the Phase I trial. The company plans to respond to the FDA promptly, to get FDA clearance to initiate the phase one clinical trial. We believe RQ200 works with a distinct mechanism of action compared to existing therapies and targets multiple causative pathways, such as angiogenesis, oxidation, and inflammation, and has the potential to offer better treatment to all patients. Neocot is our phase three ready regenerative cell therapy technology that combines novel advancement in bioengineering and cell processing to enhance the autologous cartilage repair process. We are in the process of renovating our facility to accommodate CGMP manufacturing for neocot and plan to complete construction in the fourth quarter of 2023 with a phase three randomized control study in subjects with articulate cartilage defect commencing in 2024. As you can see, we're highly dedicated to completing our stated objective with sound strategies that we believe will enable Okogen to reach several value enhancing milestones over the course of 2023 and beyond. With that, I will now turn the call over to our Chief Financial Officer and Chief Business Officer, Kwan Wu, to review our first quarter financial update.