Ocugen, Inc.

Good morning and welcome to Ocugen's second quarter 2025 financial results and business update. Please note that this call is being recorded at this time. All participants' lines are in listen-only mode. Following the speaker's commentary, there will be a question and answer session. I'll now turn the call over to Tiffany Hamilton, Ocugen's head of corporate communications. You may begin.

Thank you, Operator, and good morning, everyone. Joining me on today's call and webcast is Dr. Shankar Musunuri, Ocugen's chairman, CEO, and co-founder, who will provide a business update and an overview of our clinical and operational progress. Ramesh Ramachandran, our chief accounting officer, is also on the call to provide a financial update for the quarter ended June 30, 2025. Dr. Huma Kumar, chief medical officer, and Dr. Arun Upadhyay, chief scientific officer, will be available to answer questions following the presentation. This morning, we issued a press release detailing associated business and operational highlights for the second quarter of 2025. We encourage listeners to review the press release, which is available on our website at Ocugen.com. This call is being recorded, and a replay with the accompanying slide presentation will be available on the investor section of the Ocugen website for approximately 45 days. 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 statements include but are not limited to statements regarding our clinical development activities and related anticipated timelines. Such statements are subject to numerous important risk factors, risk and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission, the 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 the date of this presentation. Except it's 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. I will now turn the call over to Dr. Musunuri.

Thank you, Tiffany. And thank you all for joining us today. We're very enthusiastic about the progress of our novel modified gene therapy platform. All three candidates are advancing through the clinic, two in late stage, and we are on track to meet our goal of three biological licensing applications and market authorization application filings in the next three years. We remain steadfast in our mission to provide a one-time therapy for life to address considerable unmet medical needs that exist for millions of patients facing the terrifying prospect of losing their vision. Patients are actively being recruited in the United States and Canada for the novel modifier gene therapy OQ400 phase 3 limelight clinical trial for retinitis pigmentosa. And we are on target for BLA and MA filings in 2026. You may have seen our recent social media collaboration with Molly Burke, an outspoken RP patient and advocate to raise awareness for the limelight clinical trial. It's gratifying to see the increased visibility for the work we are doing, and the response has been very positive. Notable accomplishments this quarter included the FDA's agreement to proceed with the Phase 2-3 Guardian-3 pivotal confirmatory trial for RQ410ST for Stargardt disease following rare pediatric disease designation RPDD, and dosing of first patient earlier this month. Additionally, phase one 12-month preliminary data for RQ410ST and RQ410 is positive, demonstrating favorable safety and efficacy with improved structural and functional outcomes. The RQ400 phase three limelight clinical trial is the only global broad RP gene agnostic trial to address multiple genetic mutations with a single therapeutic approach. RQ400 has obtained multiple designations to assist with regulatory review, including regenerative medicine advanced therapy designation and orphan drug designation from the U.S. Food and Drug Administration, as well as orphan medicinal product designation, OMPD, and advanced therapy medicinal product, ATMP, classification from the European Medicines Agency, EMA. In the second quarter, the EMA granted eligibility to submit the RQ400 market authorization application, MAA, through the centralized procedure based on the current study design and statistical analysis plan. Due to its novel gene agnostic mechanism for action, RQ400 has potential to address more than 100 different mutations associated with RP. In contrast, a traditional gene therapy approach would require development of over 100 products to treat each individual mutation, which is commercially not physical. Currently, the only approved gene therapy for RP targets a single gene, RPE65, which accounts for 1 to 2% of RP patient population. As a result, approximately 298,000 people in the U.S. and Europe with other forms of RP remain without any approved treatment options. In anticipation of the planned BLA filing in 2026, we're actively ramping up preparation for commercialization, process validation, and manufacturing activities remain on track for completion this year. The Data and Safety Monitoring Board recently convened and reported no serious adverse events related to OQ400 and recommended continuing the study dosing as planned. Stargardt disease is an inherited retinal disorder primarily caused by mutations in the ABCA4 gene with over 1,200 different mutations in this gene identified to date. It typically presents in childhood or adolescence and affects approximately 100,000 individuals in the United States and Europe combined and an estimated 1 million people globally. Despite its prevalence, there is currently no FDA-approved treatment available for Stargardt disease. The RQ410ST clinical trial is progressing well, having achieved key milestones, receipt of rare pediatric disease designation in May, IND amendment clearance in June, and first patient dosing in July in registration trial. There is a clear sense of urgency from the agency to bring treatment options to patients who currently have no approved therapies. As we initiate the Phase 2-3 registration trial, by potentially accelerating clinical development of RQ410ST by two to three years, potentially delivering an innovative gene therapy to patients in critical need even sooner than originally anticipated. The Guardian 3 clinical trial builds upon encouraging results and positive data from the Phase 1 Guardian trial, which included a dose range and dose escalation study to evaluate the safety and preliminary efficacy of RQ410. In the phase one trial, evaluable treated eyes demonstrated a 48% slower lesion growth at 12-month follow-up compared to untreated eyes. Additionally, treated eyes showed a statistically significant with a p-value of 0.031 and critically meaningful improvement of nearly two line or nine-letter gain and best corrected visual equity, BCVA, a 12-month follow-up when compared to untreated eyes. Our third novel modified gene therapy candidate, OCU410, is specifically designed to treat geographic atrophy, secondary to dry age-related macular degeneration, TAMD, a leading cause of irreversible vision loss in the elderly. Unlike current treatment options, Though the target only one pathway, the complement system required frequent intravitreal injections, typically 6 to 12 per year, with associated safety concerns. Example, approximately 12% of patients developed with AMD. Ocu410 offers a more comprehensive and one-time therapeutic approach. Ocu410 has the potential to regulate all four pathways related to disease progression, lipid metabolism, inflammation, oxidative stress, and the complement system, thereby addressing the underlying causes of disease with a single subretinal injection. The phase 1 data at 12 months demonstrate 23% slower lesion growth in the treated eye when compared to the untreated fellow eye after a single subretinal injection. In addition to the structural improvement, treated eyes also demonstrated a stabilization or gain in visual function, low light, low luminescence visual equity with a two-line or 10-letter gain when compared to untreated fellow eyes. Preliminary results from six-month interim analysis in 31 subjects demonstrated a 27% lesion growth and the preservation of retinal tissue in the treated eyes when compared to untreated control eyes. The 27% reduction in lesion growth at six months is more pronounced compared to currently approved intravitreal therapies. Monthly and every other month, PEG, cetechol plan injections with demonstrated reductions of 13% and 12% respectively. In addition to improved lesion reduction, a single subretinal injection of RQ410 demonstrates greater efficacy in preserving retinal tissue surrounding GA lesions compared to monthly and every other month PACT-Certico plan treatments. We plan to provide full 12-month data from the Phase II study in the first quarter of 2026 and initiate Phase III in 2026. During the second quarter, we signed a binding term sheet for our first regional partnership for Occu400, announced the spin-off of Neocart into OrthoCellix, two significant strategic events that we expect will make a considerable impact on OccuGen's financial position. Aligned with OccuGen's business development strategy for Occu400, the company signed a term sheet to negotiate and enter into a licensing agreement with a well-established leader in the pharmaceutical and healthcare sector in Korea for exclusive Korean rights to Aukio 400. We are expecting to close the definitive agreement by September. This regional partnership approach will allow Aukio to retain rights in larger geographies, maximizing global patient reach while generating value for shareholders. We are actively engaged in discussions to explore a range of strategic partnership opportunities for Octi400 and all of our gene therapy candidates. The proposed reverse merger with OrthoCellix, a wholly-owned subsidiary, and Charisma Therapeutics will create a NASDAQ-listed late clinical stage regenerative cell therapy company with a first-in-class technology platform focused on orthopedic diseases. The combined company will focus on the development of Orthosilix's neocot technology for the treatment of knee articular cartilage defects. This strategic move is intended to create value for oxygen stockholders without dilution of oxygen stock. As Orthosilix is planned to be funded with $25 million in private financing under the exchange ratio formula in the merger agreement, Orthosilix's value is estimated at $135 million. We believe there's a significant valuation increase opportunity for orthocellics upon initiation of Phase III neocard clinical trial, given the more than $2 billion market cap of the only other autologous cartilage implant on the market today. Ocogen and industry experts believe this market is significantly underpenetrated, and neocard could represent a true step forward in the innovation for ACRs. With our business rapidly evolving, it was imperative to make new appointments to our board of directors to round out the skill set needed at this pivotal time. During the annual meeting in June, we announced appointments of Blaise Coleman and Dr. Satish Chandran. Blaise, a pharmaceutical executive, brings deep financial expertise and a proven track record of leading successful transformations, most notably at Endo. Blaise also held senior financial roles at AstraZeneca, including CFO of the U.S. diabetes business. Satish brings more than 30 years of leadership experience across academic research, early and mid-stage biotechs, and large pharmaceutical companies, including Wyeth and Pfizer. Satish has his name on countless patents and in recent years considers himself as a serial entrepreneur leading biotech companies focused on developing products across a variety of therapeutic indications. We also made updates to the Retina Scientific Advisory Board to reflect the absolute best guidance in this space as we move closer to commercialization. Three renowned retinal surgeons who are at the forefront of research and cutting-edge advancements for retinal disease, Drs. Jeff Heyer, Peter Kaiser, and Arshad Kanani, have joined the SAB to help us bring meaningful, innovative therapeutic options for patients living with serious retinal diseases. Finally, to optimize OxyGens R&D and clinical efforts and build upon positive momentum, pursuing strategic partnerships and developing commercial strategy, we strengthen the company's internal expertise in critical functions. Vijay Tamara joined Ocigen in the newly created position of Chief Development Officer and brings over 32 years of global regulatory leadership with deep expertise in biotechnology, biosimilars, and complex regulatory submissions. Abhi Gupta has been named Executive Vice President in Commercial and Business Development following the retirement of Mike Schein. Abby has more than 20 years of experience across commercial strategy, gene therapy, and corporate development in the biopharmaceutical industry. And Michael Blackton is our new VP of manufacturing and supply, bringing over 30 years of experience in quality, operations, and manufacturing across the biotechnology and pharmaceutical industries. I am delighted with these leadership changes and confident that we have the best people at the helm to take Oxygen to the next level of our success. With that, now I will turn the call over to Chief Accounting Officer Ramesh Ramachandran to provide an update on our financial results for the second quarter ended June 30, 2025. Ramesh. Thank you, Shankar.