Ocugen, Inc. Common Stock 2026 Q2 Earnings Call

You may begin. Thank you, operator, 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. Rita Johnson-Greene, our Chief Financial Officer, is also on the call to provide a financial update for the quarter ended June 30th, 2026. Abhi Gupta, Executive Vice President of Commercial and Business Development, and Dr. Mohammed Jameel, who joined Ocugen as Chief Medical Officer in June, will be available to answer questions following the presentation. This morning we issued a press release covering our business and operational highlights for the second quarter of 2026. We encourage listeners to review the press release, which is available on our website at ocugen.com. A replay of this call, along with the accompanying slide presentation, will be available on the investors section of the Ocugen website.

Please note that certain statements made during today's discussion may be forward-looking in nature, including those related to our clinical development pipeline, regulatory timelines, commercialization strategy, financial information, and our anticipated cash runway. These statements reflect management's current expectations and are inherently subject to risks, uncertainties, and assumptions that may cause actual results to differ materially from those expressed or implied. We encourage you to review our filings with the Securities and Exchange Commission, including the risk factors detailed therein, for a more comprehensive understanding of these potential risks. Finally, Ocugen's quarterly report on Form 10-Q, covering the second quarter of 2026, will be filed today. I will now turn the call over to Dr. Musunuri.

Thank you, Chris, good morning, everyone. The second quarter was a defining one for Ocugen. The FDA cleared our phase III trial for OCU410 to initiate dosing in geographic atrophy patients and granted RMAT designation for the program. We signed a binding term sheet with Roots Pharmaceutical to negotiate an exclusive license for OCU400 in retinitis pigmentosa across the Middle East and North Africa, MENA region. From the closing of a $130 million convertible notes financing, we extended our cash runway into 2028, now able to support all three of our late-stage programs. Before I walk through the quarter, I want to step back because Ocugen's potential is worth putting into context. For more than a decade, gene therapy and ophthalmology has been confined to a single gene, a single mutation, and a single small patient population. Our modified gene therapy platform takes a fundamentally different approach.

Rather than targeting individual mutations, it is designed to address the root cause of complex retinal diseases by modulating master regulators, nuclear hormone receptors that govern multiple gene networks. The platform is gene agnostic, inherently multifactorial, and designed to deliver a durable benefit from a single one-time subretinal injection. What this means in practice is that Ocugen is not building three separate drugs. We are advancing one platform across three late-stage programs, each targeting a major cause of blindness for which patients today have either no approved treatment whatsoever or therapies that demand chronic injections and carry meaningful safety burdens. Retinitis pigmentosa, or RP, Stargardt disease, and geographic atrophy, or GA, together affect approximately 3 million people across the U.S. and Europe, a combined patient population, and the commercial opportunity for larger than anything currently served by approved gene therapies in ophthalmology.

Across our pipeline, spanning phase I through phase III, we have treated more than 325 patients, including AAP, through multiple doses and indications, and we have not observed a drug-related serious adverse event. We remain on track to file 3 BLAs by 2028. This positions the first half of 2027 as a catalyst reach window for Ocugen with the top-line data for OCU400 and OCU410ST and our planned BLA submissions following over a short period. Let me walk you through how each program is advancing. I will hand over the call to Rita for financials. Starting with OCU410 for GA, secondary to dry age-related macular degeneration, or dry AMD, GA represents our largest commercial opportunity with approximately 2 million-3 million patients in the U.S. and Europe combined. There are currently no approved treatments for GA in Europe.

Current approved therapies in the U.S. target only one complement pathway and require frequent intravitreal injections, which has been associated with treatment discontinuation in clinical practice. GA is a multifactorial disease driven by 4 distinct pathways that contribute to the progressive degeneration of the macula. Drusen, inflammation, oxidative stress, and complement or activation. The currently approved therapies in the U.S. address only one of these 4 pathways, the complement system, which is partly why they have been unable to demonstrate meaningful functional outcomes for patients. OCU410 operates differently by delivering RORA, a nuclear hormone receptor that acts as a master regulator of retinal homeostasis. OCU410 is designed to address all 4 disease pathways simultaneously with a single subretinal injection, has the potential to redefine the standard of care in this indication. We recently received FDA clearance for OCU410 phase III registrational trial for GA.

The trial, ARMADA-3, is planned to be a global study of approximately 237 subjects, using an adaptive design powered at 95% for the primary endpoint, with the BLA and market authorization application filings targeted for 2028. We plan to initiate phase III by September 2026. This design is anchored by positive 12-month data from our phase II ARMADA trial. At the optimal dose, OCU410 delivered a statistically significant 31% reduction in GA lesion growth within the patient population of lesion size 2.5 millimeters square and 17.5 millimeters square. The criteria to be used in our phase III pivotal trial versus control, approximately twice the benefit of approved complement inhibitors and from a single injection. We also saw a 27% preservation of the ellipsoid zone within the same patient population, and no drug-related serious adverse events reported to date.

Importantly, this phase II data helps support the FDA's decision to grant RMAT designation for OCU410. Turning to OCU410ST for Stargardt disease. Stargardt is a pediatric-onset retinal disorder affecting approximately 100,000 patients in the U.S. and Europe, and roughly 1 million people globally. There are no approved therapies available for these patients today. OCU410ST is designed to address over 1,200 pathogenic mutations in the ABCA4 gene with a single one-time treatment. On April 1st, we announced the completion of enrollment and dosing in our phase II/III GUARDIAN-3 pivotal confirmatory trial, enrolling 63 participants. We expect the interim outcome decision for the first 50% of subjects at eight months in the third quarter of 2026, and top-line phase II/III data in the second quarter of 2027, with our BLA submission to follow mid-2027. Moving to OCU400 for RP.

The phase III liMeliGhT trial is the first and largest genetic medicine registration trial for broad RP, spanning more than 30 genetic mutations. Approximately 300,000 people in the U.S. and Europe are living with RP, which is caused by mutations in more than 100 genes. The only approved gene therapy for RP today targets a single gene, RPE65, which accounts for less than 2% of all RP cases. OCU400 is designed to provide a therapeutic option for all RP patients, and that is a fundamentally different commercial opportunity. Enrollment in the liMeliGhT is complete, with 140 patients randomized two to one, treated versus control across the RHO and gene-agnostic arms, spanning more than 30 genetic mutations associated with early to late-stage RP, including pediatrics. The breadth of the population intended to validate the gene-agnostic mechanism of action of our novel modified gene therapy platform.

The primary endpoint is 12-month change in visual function assessed by luminance dependent navigation assessment, or LDNA. Subjects are followed for one year post-dosing for the primary endpoint analysis. Top-line phase III data is expected in the first quarter of 2027, advancing OCU400 to a potential approval in the fourth quarter of 2027. FDA feedback confirmed that the path to rolling BLA submission remains tied to top-line data expected in the first quarter of 2027. On the manufacturing side, our process performance qualification, PPQs, batches are complete, supporting BLA and commercial launch supplies. Brand planning and marketing initiatives led by Abhi Gupta, our EVP of Commercial and Business Development, continue to scale in preparation for launch. We also advanced our global commercialization strategy for OCU400 during the quarter.

In July, we signed a binding term sheet with Roots Pharmaceutical and its strategic partner, Al-Dhow International Holding, for exclusive rights to OCU400 in the Middle East and North Africa. We're active on the BD front to find other global partners for regional commercialization partnerships where RP is most prevalent. Here is a snapshot of the market opportunity across our three late-stage development programs. While OCU410 for GA represents our largest commercial opportunity, we believe all three programs have the potential to generate significant revenue while addressing areas of substantial unmet medical need. As we continue advancing our pipeline, we're also building the foundational commercial capabilities to support future global access. Our efforts are focused on five key areas. First, we're in discussions with CMS and payers to establish early market access and reimbursement strategies.

Second, we continue to identify and evaluate specialized centers of excellence with expertise in subretinal surgical procedures that could support future treatment delivery. Third, we are mapping the patient journey from diagnosis through treatment and long-term follow-up with the goal of facilitating a seamless experience for patients, caregivers, and healthcare providers. Fourth, we are assessing manufacturing, supply chain, and distribution requirements to help ensure operational readiness. Finally, we are beginning to build out our commercial infrastructure, including our marketing and sales capabilities, as we ramp up for launch. With that, I'll turn the call over to Rita for the financial update.