Q4 2025 Ocugen Inc Earnings Call

March 4, 2026

Speaker #2: Following these speakers' commentary, there will be a question-and-answer session. I will now turn the call over to Tiffany Hamilton, Ocugen's head of corporate communications.

Speaker #2: 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.

Speaker #2: He will provide a business update and an overview of our clinical and operational progress. Rita Johnson-Green, our chief financial officer, is also on the call to provide a financial update for the quarter and full year ended December 31, 2025.

Speaker #2: Dr. Huma Qamar, chief medical 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 fourth quarter and full year 2025.

Speaker #2: 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.

Speaker #2: Before we begin, 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, and financial information and our anticipated cash runway.

Tiffany Hamilton: 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 and full year ended 31 December 2025. Dr. Huma Qamar, Chief Medical 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 Q4 and full year 2025. 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 investor section of the Ocugen website.

Speaker #2: These statements reflect management's current expectations and are inherently subject to risks, uncertainties, and assumptions that may cause actual outcomes to differ materially from those expressed or implied.

Speaker #2: 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.

Speaker #2: Finally, Ocugen's annual report on Form 10-K, covering the full year 2025, will be filed today. I will now turn the call over to Dr. Musunuri.

Speaker #2: Thank you, Tiffany. And thank you all for joining us today. I'm pleased to share an update on what were the transformative years for Ocugen.

Speaker #2: Considerable development across all of our modified gene therapy programs, including licensing and financing agreements to strengthen our financial position, and meaningful appointments to our leadership team were made in 2025—a year of real momentum for Ocugen.

Tiffany Hamilton: Before we begin, 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, and 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 outcomes to differ materially from those expressed or implied. We encourage you to review our filings with Securities and Exchange Commission, including the risk factors detailed therein, for a more comprehensive understanding of these potential risks. Finally, Ocugen's annual report on Form 10-K covering the full year 2025 will be filed today. I will now turn the call over to Dr. Lucinari.

Speaker #2: We are now poised to leverage upcoming catalysts and advanced business as we near the first of our three BLA filings. I'm proud of what this team has accomplished, and I'm confident that with the full range of experienced leadership across the organization, we have the resources and the know-how to drive Ocugen's transition into a commercial-stage company.

Speaker #2: Let me walk you through each program. Starting with OQ400, and right now it is is Pigmentosa, which I will refer to as RP going forward.

Speaker #2: It is important to note that the Phase 3 Limelight clinical trial is the only broad RP gene agnostic trial and the largest known Phase 3 orphan gene therapy trial.

Shankar Musunuri: Thank you, Tiffany. Thank you all for joining us today. I'm pleased to share an update on what was a transformative year for Ocugen. Considerable development across all of our modifier gene therapy programs, including licensing and financing agreements to strengthen our financial position and meaningful appointments to our leadership team, made in 2025 a year of real momentum for Ocugen. We are now poised to leverage upcoming catalysts and advance the business as we near the first of our three BLA filings. I'm proud of what this team has accomplished, and I'm confident that with a full bench of experienced leadership across the organization, we have the resources and the know-how to drive Ocugen's transition into a commercial stage company. Let me walk you through each program, starting with OCU400 and retinitis pigmentosa, which I will refer to as RP going forward.

Speaker #2: Approximately 300,000 people in the US and Europe are living with RP, is caused by mutations in more than 100 genes. OQ400 is designed as a modified gene therapy utilizing MRTP3, a central transcriptional regulator of retinal-specific pathways to address multiple genetic mutations with a single one-time treatment.

Speaker #2: The only approved gene therapy for RP today targets a single gene RP65, which accounts for just 1 to 2 percent of the total RP patient population.

Shankar Musunuri: I'm proud of what this team has accomplished, and I'm confident that with a full bench of experienced leadership across the organization, we have the resources and the know-how to drive Ocugen's transition into a commercial stage company. Let me walk you through each program, starting with OCU400 and retinitis pigmentosa, which I will refer to as RP going forward.

Speaker #2: We believe OQ400 has significantly wider commercial potential as it is intended to provide a therapeutic option for 98 to 99 percent of all RP patients.

Speaker #2: I'm pleased to report that enrollment is now complete for the OQ400 Phase 3 Limelight trial. As a one-year clinical trial, top-line data will be available in the first quarter of 2027.

Shankar Musunuri: It is important to note that the Phase 3 liMeliGhT clinical trial is the only broad RP gene agnostic trial and the largest known Phase 3 orphan gene therapy trial. Approximately 300,000 people in the US and Europe are living with RP is caused by mutations in more than 100 genes. OCU400 is designed as a modified gene therapy utilizing NR2E3, a central transcriptional regulator of retinal specific pathways, to address multiple genetic mutations with a single one-time treatment. The only approved gene therapy for RP today targets a single gene, RPE65, which accounts for just 1% to 2% of the total RP patient population. We believe OCU400 has significantly wider commercial potential as it is intended to provide a therapeutic option for 98% to 99% of all RP patients.

Speaker #2: These data are anticipated to support the biologics license application, BLA, filing for OQ400, and potential approval in 2027. The Limelight clinical trial enrolled 140 patients who were randomized 2 to 1 into the treatment group and untreated control group across mutations, including row and gene agnostic arms.

Speaker #2: The gene agnostic arm includes many genetic mutations, including those most prevalent. Tulip, Cross, XLRS, USHA, XLRP, and PD6B. The target population included patients with early to late-stage disease among a broad RP population, including pediatrics.

Speaker #2: The primary endpoint is 12-month change in visual function assessed by LDNA luminance-dependent navigation assessment, with improvement in lux level from baseline to 12 months.

Shankar Musunuri: I'm pleased to report that enrollment is now complete for the OCU400 Phase 3 liMeliGhT trial. As a 1-year clinical trial, top-line data will be available in Q1 2027. These data are anticipated to support the Biologics License Application, BLA, filing for OCU400 and potential approval in 2027. The liMeliGhT clinical trial enrolled 140 patients who were randomized 2 to 1 into the treatment group and untreated control group across mutations, including RHO and gene agnostic arms. The gene agnostic arm includes many genetic mutations, including those most prevalent, TULP1, CRX, XLRS, USH2A, XLRP, and PDE6B. The target population included patients with early to late stage disease among a broad RP population, including pediatrics. The primary endpoint is 12-month change in visual function assessed by LDNA, Luminance Dependent Navigation Assessment, with improvement in LUX level from baseline to 12 months.

Speaker #2: We also released positive long-term three-year Phase 1/2 data for OQ400 that builds on our prior two-year results. The data demonstrates sustained clinically meaningful approximately two-line LLVA gain reinforcing durable gene agnostic benefit.

Speaker #2: OQ400 maintained a favorable durability safety and tolerability profile with the no-new treatment-related serious adverse events or adverse events of interest emerged. With enrollment complete and these strong long-term data in hand, we are on track to begin the rolling BLA submission in the third quarter of 2026.

Speaker #2: Process validation and manufacturing activities are progressing well in support of the timeline, and blind planning and marketing initiatives are scaling up as well. We anticipate commercialization in 2027 in line with our commitments.

Speaker #2: As we prepare for what will ultimately be the global rollout of OQ400, we are pursuing regional partnerships that preserve Ocugen's right to larger geographies, while also generating near-term value for our shareholders.

Shankar Musunuri: We also released positive long-term 3-year Phase 1/2 data for OCU400 that builds on our prior 2-year results. The data demonstrates sustained, clinically meaningful approximately 2-line LLVA gain, reinforcing durable gene agnostic benefit. OCU400 maintained a favorable durability, safety, and tolerability profile with the no new treatment-related serious adverse events or adverse events of interest emerged. With enrollment complete and these strong long-term data in hand, we're on track to begin the rolling BLA submission in Q3 2026. Process validation and manufacture activities are progressing well in support of the timeline, and plan planning and marketing initiatives are scaling up as well. We anticipate commercialization in 2027 in line with our commitments. As we prepare for what will ultimately be global rollout of OCU400, we are pursuing regional partnerships that preserve Ocugen's right to larger geographies while also generating near-term value for our shareholders.

Speaker #2: In 2025, we executed our first regional licensing agreement with Guangdong Pharmaceutical Company Limited. For the exclusive Korean rights, OQ400. With upfront fees and near-term development milestone payments, along with royalties, this was a valuable collaboration for Ocugen and a critical step in the company's business development strategy.

Speaker #2: There are an estimated 7,000 individuals in the Republic of Korea with RP, 7% of addressable US RP market. This approach allows us to maximize total patient reach while retaining full commercial rights in the US and Europe.

Shankar Musunuri: Process validation and manufacture activities are progressing well in support of the timeline, and plan planning and marketing initiatives are scaling up as well. We anticipate commercialization in 2027 in line with our commitments. As we prepare for what will ultimately be global rollout of OCU400, we are pursuing regional partnerships that preserve Ocugen's right to larger geographies while also generating near-term value for our shareholders.

Speaker #2: Now, let's move on to OQ410ST for Stargardt disease. OQ410ST hosts the potential to target for 1,200 pathogenic mutations in the ABCA4 gene-associated with the Stargardt disease, and other ABCA4-related retinopathies with a single one-time treatment.

Shankar Musunuri: In 2025, we executed our first regional licensing agreement with Kwangdong Pharmaceutical Co., Ltd. for the exclusive Korean rights OCU400. With upfront fees and near-term development milestone payments along with royalties, this was a valuable collaboration for Ocugen and a critical step in the company's business development strategy. There are an estimated 7,000 individuals in the Republic of Korea with RP, equal to approximately 7% of addressable US RP market. This approach allows us to maximize total patient reach while retaining full commercial rights in the US and Europe. Now, let's move on to OCU410ST for Stargardt disease. OCU410ST holds the potential to target over 1,200 pathogenic mutations in the ABCA4 gene-associated with the Stargardt disease and other ABCA4-related retinopathies with a single one-time treatment.

Speaker #2: Stargardt disease affects approximately 100,000 patients in the US and Europe combined, and approximately 1 million people globally, with no approved treatment options available. The Phase 2/3 Guardian 3 total confirmatory trial remains ahead of schedule.

Speaker #2: We anticipate top-line Phase 2/3 data in the second quarter of 2027, followed by the BLA submission. In January, we announced the peer-reviewed publication of our Phase 1 Guardian trial results in Nature Eye, which supports a favorable safety, tolerability, and efficacy profile of OQ410ST and its potential to provide clinically meaningful functional and structural benefits in Stargardt patients.

Speaker #2: This independence validation further strengthens the scientific foundation supporting the ongoing Total trial. Importantly, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency confirmed that data from our single US-based trial can also support an EMA application.

Shankar Musunuri: Stargardt disease affects approximately 100,000 patients in the US and Europe combined, and approximately 1 million people globally, with no approved treatment options available. The Phase 2/3 GARDian3 pivotal confirmatory trial remains ahead of schedule. We anticipate top line Phase 2/3 data in Q2 2027, followed by the BLA submission. In January, we announced the peer-reviewed publication of our Phase 1 GARDian trial results in Eye, which supports the favorable safety, tolerability, and efficacy profile of OCU410ST, and its potential to provide clinically meaningful functional and structural benefits in Stargardt patients. This independent validation further strengthens the scientific foundation supporting the ongoing total trial. Importantly, the Committee for Medicinal Products for Human Use, CHMP, of the European Medicines Agency confirmed that data from our single US-based trial can also support an EMA application.

Speaker #2: This alignment allows us to maintain the same timeline and budget efficiencies in Europe as we have with this OQ400 total trial streamlining our development efforts and bringing OQ410ST to patients in Europe sooner than originally anticipated.

Speaker #2: The program has also received great pediatric disease designation. Further strengthening its regulatory positioning. I would like to explain ellipsoid zone. Easy analysis in greater detail.

Speaker #2: As this is now an exploratory endpoint for both the Guardian 3 and Armora clinical trials. The ellipsoid zone is a hyper-reflective band representing photoreceptor inner and outer layer segment junction.

Speaker #2: It indicates photoreceptor health line, and is a biomarker for photoreceptor structural integrity and metabolic health. Easy disruption precedes RPE loss and visible atrophy in geographic atrophy and Stargardt disease.

Shankar Musunuri: This alignment allows us to maintain the same timeline and budget efficiencies in Europe as we have with this OCU400 total trial, streamlining our development efforts and bringing OCU410ST to patients in Europe sooner than originally anticipated. The program has also received Rare Pediatric Disease Designation, further strengthening its regulatory positioning. I would like to explain Ellipsoid Zone, EZ analysis, in greater detail as this is now an exploratory endpoint for both the GARDian3 and ArMaDa clinical trials. The Ellipsoid Zone is a hyperreflective band representing photoreceptor inner and outer layer segment junction. It indicates photoreceptor health line and is a biomarker for photoreceptor structural integrity and metabolic health. EZ disruption precedes RPE loss and visible atrophy in geographic atrophy and Stargardt disease. EZ measurement is important because it provides early and sensitive detection.

Speaker #2: Easy measurement is important because it provides early and sensitive detection. Easy changes occur before visible RPE atrophy expansion in GA and Stargardt progression. It also enables earlier intervention and more sensitive treatment effect detection in GA and Stargardt.

Speaker #2: Finally, easy correlates to earlier functional therapeutic benefit with an effect as early as one year, compared to other measures such as visual equity with clinically meaningful effect at two years or more.

Speaker #2: Since all of our clinical trials aim to demonstrate benefit at one year, and we are targeting significant unmet medical needs, easy is a relevant measure to show functional outcome in these trials.

Speaker #2: As easy analysis has been established as a clinically relevant endpoint for dry AMD clinical trials, it was critical to incorporate this measure for both our Stargardt and GA trials.

Speaker #2: As shown in this bar graph, change from baseline at 12 months in treated fellow eyes across doses excluding two subjects last to follow up and one subject with retinal detachment demonstrated a mean of 116% in lesion reduction in a valuable treated eyes relative to untreated eyes.

Shankar Musunuri: EZ changes occur before visible RPE atrophy expansion in GA and Stargardt progression. It also enables earlier intervention and more sensitive treatment effect detection in GA and Stargardt. Finally, EZ correlates to earlier functional therapeutic benefit with an effect as early as one year compared to other measures such as visual acuity, with clinically meaningful effect at two years or more. Since all of our clinical trials aim to demonstrate benefit at one year and we are targeting significant unmet medical needs, EZ is a relevant measure to show functional outcome in these trials. As EZ analysis has been established as a clinically relevant endpoint for dry AMD clinical trials, it was critical to incorporate this measure for both our Stargardt and GA trials.

Speaker #2: Specifically, 50% of OQ410ST treated eyes achieved easy preservation exceeding expected disease decline or atrophy progression at 12 months. This change from baseline structural preservation on spectral domain OCT quantified as 116% lesion reduction and ellipsoid zone integrity highlights meaningful photoreceptor protection and functional therapeutic benefit in Stargardt disease.

Speaker #2: Underscoring the key differentiator of modified gene therapy. Now, let's turn to OQ410 and GA secondary to late-stage dry AMD. With approximately 2 to 3 million GA patients in the US and Europe combined, OQ410 represents a significant market opportunity.

Shankar Musunuri: As shown in this bar graph, change from baseline at 12 months in treated fellow eyes across doses, excluding two subjects lost to follow-up and one subject with retinal detachment, demonstrated a mean of 116% in lesion reduction in evaluable treated eyes relative to untreated eyes. Specifically, 50% of OCU410ST treated eyes achieved EZ preservation exceeding expected disease decline or atrophy progression at 12 months. This change from baseline structural preservation on spectral domain OCT, quantified as 116% lesion reduction and Ellipsoid Zone integrity, highlights meaningful photoreceptor protection and functional therapeutic benefit in Stargardt disease, underscoring the key differentiator of modified gene therapy. Now, let's turn to OCU410 in GA secondary to late stage dry AMD.

Speaker #2: OQ410 is specifically designed to address multiple pathways implicated in the pathogenesis of dry age-related macular degeneration and offers a promising advantage over current treatment options that target only one pathway, the complement system.

Speaker #2: Currently approved treatment options require frequent intravitreal injections about 6 to 12 doses per year. And are accompanied by various safety risks. For example, roughly 12% of patients develop wet AMD following treatment.

Speaker #2: There are no treatment approved for GA in Europe, and existing FDA-approved options have failed to demonstrate meaningful functional outcomes. OQ410 is therefore well-positioned to address this critical unmet need.

Speaker #2: In January, we announced positive preliminary 12-month data from approximately 50% of patients evaluated to date in the Phase 2 Armora clinical trial evaluating OQ410.

Shankar Musunuri: With approximately $2 to 3 million GA patients in the US and Europe combined, OCU410 represents a significant market opportunity. OCU410 is specifically designed to address multiple pathways implicated in the pathogenesis of dry isolated macular degeneration, and offers a promising advantage over current treatment options that target only one pathway, the complement system. Currently approved treatment options require frequent intravitreal injections, about 6 to 12 doses per year, and are accompanied by various safety risks. For example, roughly 12% of patients develop wet AMD following treatment. There are no treatment approved for GA in Europe, and existing FDA-approved options have failed to demonstrate meaningful functional outcomes. OCU410 is therefore well-positioned to address this critical unmet need. In January, we announced positive preliminary 12-month data from approximately 50% of patients evaluated to date in the phase 2 ArMaDa clinical trial evaluating OCU410.

Speaker #2: The key findings were compelling. We observed a 46% reduction in lesion growth at 12 months across the medium and high-dose groups combined versus control, with the statistical significance at P of 0.015 in a cohort of 23 patients.

Speaker #2: We also saw a 50% responder rate with patients achieving greater than 50% lesion size reduction versus control. To put this in context, currently market products have demonstrated only a 22% lesion reduction at two years.

Speaker #2: So at one year, OQ410 is already delivering more than double the benefit seen with existing therapies at twice the time. A subgroup analysis of patients with baseline GA size of 7.5 mm² or greater, representing advanced atrophy, demonstrated a 57% reduction in lesion growth in treated eyes for the medium dose and a 56% reduction for the high dose compared with control.

Shankar Musunuri: OCU410 is therefore well-positioned to address this critical unmet need. In January, we announced positive preliminary 12-month data from approximately 50% of patients evaluated to date in the phase 2 ArMaDa clinical trial evaluating OCU410.

Shankar Musunuri: The key findings were compelling. We observed a 46% reduction in lesion growth at 12 months across the medium and high dose groups combined versus control, with statistical significance at P of 0.015 in a cohort of 23 patients. We also saw a 50% responder rate, with patients achieving greater than 50% lesion size reduction versus control. To put this in context, currently market products have demonstrated only a 22% lesion reduction at 2 years. At 1 year, OCU410 is already delivering more than double the benefit seen with existing therapies at twice the time.

Speaker #2: This suggests OQ410 may be even more effective in patients with substantial disease burden. The data set also included encouraging 12-month Phase 1 findings where OQ410 treated eyes demonstrated 60% slower loss of the ellipsoid zone or easy compared to untreated fellow eyes.

Speaker #2: The 60% reduction in easy loss rate indicates that OQ410 treatment is substantially slowing the rate of photoreceptor degeneration compared to the natural history observed in the untreated fellow eyes.

Speaker #2: We look forward to reporting the complete data set from the OQ410 Phase 2 Armora trial this month. And anticipate initiating Phase 3 in 2026.

Shankar Musunuri: The subgroup analysis of patients with baseline GA size of 7.5 mm² or greater, representing advanced atrophy, demonstrated a 57% reduction in lesion growth in treated eyes for the medium dose and a 56% reduction for the high dose compared with control. This suggests OCU410 may be even more effective in patients with substantial disease burden. The data set also included encouraging 12-month Phase 1 findings, where OCU410 treated eyes demonstrated 60% slower loss of the ellipsoid zone or EZ compared to untreated fellow eyes. The 60% reduction in EZ loss rate indicates that OCU410 treatment is substantially slowing the rate of photoreceptor degeneration compared to the natural history observed in the untreated fellow eyes. We look forward to reporting the complete data set from the OCU410 Phase 2 ArMaDa trial this month and anticipate initiating Phase 3 in 2026.

Speaker #2: Let me also provide a brief update on our other programs. For OQ200, no serious adverse events or adverse events related to OQ200 have been reported to date across the Phase 1 dose escalation cohorts and trial enrollment is expected to be completed in the first quarter of 2026.

Speaker #2: Regarding our inhaled vaccine candidate, OQ500, NIAID intends to initiate the Phase 1 clinical trial in the second quarter of 2026. Finally, we created OrthoCelix as a wholly owned subsidiary for our regenerative cell therapy assets, including Neocart, with the goal to be independent through financing that will maximize value for oxygen shareholders and patients.

Speaker #2: We will provide further details as the process progresses. Across the portfolio, 2026 represents multiple defined inflection points. These include completion of enrollment for OQ410ST in early 2026, full Phase 2 data for OQ410 this month, interim pivotal data for OQ410ST in the third quarter, initiation of Phase 3 for OQ410 in 2026, and start of rolling BLA submission for OQ400 in the third quarter.

Shankar Musunuri: Let me also provide a brief update on our other programs. For OCU200, no serious adverse events or adverse events related to OCU200 have been reported to date across the phase 1 dose escalation cohorts. Trial enrollment is expected to be completed in Q1 2026. Regarding our inhaled vaccine candidate, OCU400, NIAID intends to initiate the phase 1 clinical trial in Q2 2026. Finally, we created NeoCelix as a wholly-owned subsidiary for our regenerative cell therapy assets, including NeoCart, with the goal to be independent through financing that will maximize value for Ocugen shareholders and patients. We will provide further details as the process progresses. Across the portfolio, 2026 represents multiple defined inflection points.

Speaker #2: Each of these milestones builds towards longer-term regulatory and commercialization objectives and reinforces our commitment to file three BLAs in the next three years. Operationally, we also strengthened our executive leadership team with the several appointments, including Abhigupta, to Executive Vice President, Commercial and Business Development, bringing more than 20 years of experience across commercial strategy, gene therapy, and corporate development in the biopharmaceutical industry.

Speaker #2: Recently, Rita Johnson Green was named Chief Financial Officer. Rita's experience in financial strategy and capital planning supports our continued focus on disciplined resource allocation as our programs advance toward late-stage development and potential commercialization.

Shankar Musunuri: These include completion of enrollment for OCU410ST in early 2026, full phase 2 data for OCU410 this month, interim pivotal data for OCU410ST in Q3, initiation of phase 3 for OCU410 in 2026, and start of rolling BLA submission for OCU400 in Q3. Each of these milestones builds towards longer-term regulatory and commercialization objectives and reinforces our commitment to file 3 BLAs in the next 3 years. Operationally, we also strengthened our executive leadership team with the several appointments, including Abhi Gupta to Executive Vice President, Commercial and Business Development, bringing more than 20 years of experience across commercial strategy, gene therapy, and corporate development in the biopharmaceutical industry. Recently, Rita Johnson-Greene was named Chief Financial Officer. Rita's experience in financial strategy and capital planning supports our continued focus on disciplined resource allocation as our programs advance toward late-stage development and potential commercialization.

Speaker #2: And just this week, Paul State joined us as Executive Vice President Operations. Paul has more than 20 years of leadership experience in biologics, and tel and gene therapy technical operations.

Speaker #2: He joins from Bristol-Myers Squibb, where for over 16 years he held leadership roles in manufacturing, launch, scale-up, and orchestration of reliable global supply chains, with a clarity focus for the last five years.

Speaker #2: He will lead operations to strengthen execution and support the company's transition toward regulatory approvals and commercialization. I will now turn the call over to Rita Johnson Green to provide an update on our financial results for the quarter and full year ended December 31, 2025.

Shankar Musunuri: Recently, Rita Johnson-Greene was named Chief Financial Officer. Rita's experience in financial strategy and capital planning supports our continued focus on disciplined resource allocation as our programs advance toward late-stage development and potential commercialization.

Speaker #2: Rita, thank you, Shankar. I am thrilled to join the OccuGen team and support the company through its eminent transitions into a commercial enterprise. Starting with our fourth-quarter results, research and development expenses for the three months ended December 31st 2025 were $10.7 million, compared to $8.3 million for the three months ended December 31st, 2024.

Shankar Musunuri: Just this week, Paul Slade joined us as Executive Vice President, Operations. Paul has more than 20 years of leadership experience in biologics and cell and gene therapy technical operations. He joins from Bristol Myers Squibb, where for over 16 years, he held leadership roles in manufacturing launch, scale-up, orchestration of reliable global supply chains with a CAR-T focus for the last 5 years. He will lead operations to strengthen execution and support the company's transition toward regulatory approvals and commercialization. I will now turn the call over to Rita Johnson-Greene to provide an update on our financial results for the quarter and full year ended 31 December 2025. Rita.

Speaker #2: General and administrative expenses for the three months ended December 31st, 2025 were $6.1 million, compared to $6.3 million for the three months ended December 31st, 2024.

Speaker #2: Ocugen reported a $0.06 net loss per common share for the three months ended December 31, compared to a $0.05 net loss per common share for the three months ended December 31, 2024.

Speaker #2: For the full year ended December 31, 2025, research and development expenses were $39.8 million, compared to $32.1 million for the full year ended December 31, 2024.

Rita Johnson-Greene: Thank you, Shankar. I am thrilled to join the Ocugen team and support the company through its imminent transitions into a commercial enterprise. Starting with our Q4 results, research and development expenses for the three months ended 31 December 2025 were $10.7 million compared to $8.3 million for the three months ended 31 December 2024. General and administrative expenses for the three months ended 31 December 2025 were $6.1 million compared to $6.3 million for the three months ended 31 December 2024.

Speaker #2: General and administrative expenses were $27.6 million, compared to $26.7 million for the prior year. OccuGen reported a $23 cent net loss per common share for the year ended December 31st, 2025, compared to a $20 cent net loss per common share for the year ended December 31st, 2024.

Speaker #2: Our current cash and cash equivalents extend our runway into the fourth quarter of 2026. This includes the recent raise of $22.5 million through an underwritten registered direct offering of common stock led by RTW Investments.

Rita Johnson-Greene: Ocugen reported a $0.06 net loss per common share for the 3 months ended 31 December 2025, compared to a $0.05 net loss per common share for the 3 months ended 31 December 2024. For the full year ended 31 December 2025, research and development expenses were $39.8 million compared to $32.1 million for the full year ended 31 December 2024. General and administrative expenses were $27.6 million compared to $26.7 million for the prior year. Ocugen reported a $0.23 net loss per common share for the year ended 31 December 2025, compared to a $0.20 net loss per common share for the year ended 31 December 2024. Our current cash and cash equivalents extend our runway into the Q4 of 2026.

Speaker #2: In addition, if the $30 million in warrants from the prior Janice Henderson raise are exercised in full, it will extend cash runway into the second quarter of 2027.

Speaker #2: That concludes my financial update. Shankar, back to you.

Speaker #1: Thank you, Rita. We'll now open the call for questions. Operator, thank you. If you'd like to ask a question, please press star and the number one on your telephone keypad to raise your hand and enter the queue.

Speaker #1: And if you'd like to withdraw your question or your question has been answered, please press star and the number one again. And we will be taking our first question from Michael, Ocunovich from Maxim Group.

Speaker #1: Please go ahead.

Speaker #3: Hey guys, thank you so much for taking my questions today. Wrapping all the great progress you've made. I guess to start off, just given that it is a 12-month primary endpoint for the Limelight study, how confident are you in the ability to turn around the data from that from when you hit on that top-line endpoint to actually releasing the top-line data?

Rita Johnson-Greene: This includes the recent raise of $22.5 million through an underwritten registered direct offering of common stock led by RTW Investments. In addition, if the $30 million in warrants from the prior Janus Henderson raise are exercised in full, it will extend cash runway into Q2 2027. That concludes my financial update. Shankar, back to you.

Speaker #3: Within first quarter 27.

Speaker #1: Two months.

Speaker #4: Thank you for the question. We are very confident that we will be able to hit our timeline.

Shankar Musunuri: Thank you, Rita. We'll now open the call for questions. Operator?

Operator: Thank you. If you'd like to ask a question, please press star and the number 1 on your telephone keypad to raise your hand and enter the queue. If you'd like to withdraw your question or your question has been answered, please press star and the number 1 again. We will be taking our first question from Michael Okunewitch from Maxim Group. Please go ahead.

Speaker #3: All right. And then just for that endpoint, could you remind us of some of the modifications that you made for that particular navigation assessment course, and why you decided to go with a primary metric for RP?

Speaker #4: Okay. So in terms of the mobility test that we are using, proprietary to Ocugen, that's luminance-dependent navigation assessment. It's the mobility test that was approved as the primary endpoint for Luxturna, that was called MLMT at that time.

Michael Okunewitch: Hey, guys. Thank you so much for taking my questions today. Congrats on all the great progress you've made. I guess to start off, just given that it is a 12-month primary endpoint for the liMeliGhT study, how confident are you in the ability to turn around the data from that, from when you hit on that top line endpoint to actually releasing the top line data within Q1 of 2027?

Speaker #4: That was only designed for RPE 65 mutation that covers only 1 to 2 percent of the RP landscape. This is a very sensitive and specific test.

Speaker #4: As you can see, this is the broad RP indication trial covering all clinical syndromic, non-syndromic, all the genetic mutations that cause RP are included.

Speaker #4: So this has uniform lux levels and intensity, and lux levels from 0 to 9. And that has the ability to capture the change in real time, which is from the baseline up to 52 weeks.

Shankar Musunuri: Neema?

[Company Representative] (Ocugen): Thank you for the question. We are very confident that we will be able to hit our timeline.

Michael Okunewitch: All right. Then just for that endpoint, could you just remind us some of the modifications that you made for that particular navigation assessment course and why you decided to go with a primary metric for RP?

Speaker #4: So this was also aligned with the FDA. It's a validated test, as well as this was approved by the FDA, and it's the only test that can capture the real change with functional outcome—improving the functional outcome or demonstrating the functional outcome in these mutations. And just to let you know, we are the only trial globally that is covering all, or the majority, of the gene-agnostic mutations, as we have covered this morning in one of our slides as well.

[Company Representative] (Ocugen): Okay. In terms of the mobility test that we are using proprietary to Ocugen, that's Luminance Dependent Navigation Assessment. It's the mobility test that was approved as the primary endpoint for Luxturna, that was called MLMT at that time. That was only designed for RPE65 mutation that covers only 1 to 2% of the RP landscape. This is a very sensitive and specific test. As you can see that this is the broad RP indication trial covering all clinical, syndromic, non-syndromic, all the genetic mutations that cause RP are included. This has uniform lux levels and intensity and lux levels from 0 to 9, and that has the ability to capture the change in real time, which is from the baseline up to 52 weeks. This was also aligned with FDA.

Speaker #4: Thank you.

Speaker #3: Thank you. Certainly very helpful. And then, last one before I jump back into the queue. For the Stargardt program, it's looking like there could be an approval from another company for a chronic therapy by the time you file for 410ST.

Speaker #3: So I wanted to know how this might impact the opportunity or pricing potential, and if there's any reason that 410 ST couldn't be complementary with other therapies as they come to market.

Speaker #1: Yeah, I'll take that. So there are other therapies out there. Obviously, what we have shown, if you look at the data, we published an eye.

[Company Representative] (Ocugen): It's a validated test as well as this was approved by FDA and the only test that can capture the real change with functional outcome, improving the functional outcome or demonstrating the functional outcome in these mutations. Just to let you know, we are the only trial globally that is covering all the majority of the gene agnostic mutations as we have covered this morning in one of our slides as well. Thank you.

Speaker #1: In one year, again, I wanted to restate all our trials were able to show treatment benefit in one year. Unlike other trials out there, two years or more.

Speaker #1: And so the data we showed in one year is compelling. It looks superior. And also, our goal is to show functional benefit as well. With a gene therapy, we're targeting the major pathways, which are complex in Stargardt and GA.

Michael Okunewitch: Thank you. Certainly very helpful. Last one before I jump back into the queue. For the Stargardt program, it's looking like there could be an approval from another company for a chronic therapy by the time you file for OCU410ST. I wanted to know how this might impact the opportunity or pricing potential and if there's any reason that OCU410ST couldn't be complementary with other therapies as they come to market.

Speaker #1: With the RORA gene, and also, we have the ability to reset the homeostasis and make sure we create a healthy environment for retinal cells to survive.

Speaker #1: That's very important, factor. We're not just trying to slow down the disease progression. We're working on the in our genes have ability to control the entire network.

Speaker #1: So there is a big difference. And also, this is one and done. And if you have a one and done therapy, this will set up the standard of care.

Speaker #1: So we're not worried about other therapies if they come to the market first. It's good. Those therapies will educate the market and they'll create a market education and everything else.

Shankar Musunuri: Yeah, I'll take that. There, there are other therapies out there. Obviously what we have shown, if you look at the data we published in Eye, in one year, again, I wanted to restate all our trials, we're able to show treatment benefit in one year, unlike other trials out there, two years or more. The data we showed in one year is compelling. It looks superior. Our goal is to show also functional benefit. With the gene therapy, we're targeting the major pathways which are complex in Stargardt and GA with the RORA gene. We have ability to reset the homeostasis and bring, you know, make sure we create a healthy environment for retinal cells to survive. That's very important factor. We're not just trying to slow down the disease progression.

Speaker #1: We will come back and then we maybe behind them, but it's okay. Because what we believe we are going to set the standard of care for Stargard patients globally.

Speaker #4: Yes, I would like to thank you, Shankar—very well said. To that, I would like to add that this is the trial where we are also including the population three years of age and above, versus the other trials that are very limited in the age population.

Speaker #4: Also, the inclusion-exclusion criteria is very globally representing. Other than that, this is the Oc410 ST is targeting early to advanced cases of Stargard disease.

Speaker #4: If you look at in the comparison the safety and tolerability and efficacy that we have seen in terms of lesion growth reduction and also the functional and structural outcomes, has been trending in the right direction and promising from the clinical standpoint as well.

Shankar Musunuri: We're working in our genes have ability to control the entire network. There's a big difference. Also this is one and done. If you have a one and done therapy, this will set up the standard of care. We're not worried about other therapies if they come to the market first. It's good. Those therapies will educate the market, and they'll create a market, education, and everything else. We will come back and then we may be, you know, behind them, but it's okay because what we believe, we are going to set the standard of care for Stargardt patients globally. Yes, I would like. Thank you, Shankar. very well said.

Speaker #3: Thank you. It's certainly an exciting time for the space. I'm looking forward to any further updates that you have.

Speaker #1: Thank you.

Speaker #3: Thank you. Our next question comes from the line of Boris Peaker from Titan Partners. Please go ahead.

Shankar Musunuri: That I would like to add that this is the trial that we are also having the population three years of age and above, versus the other trials that are very limited in the age population. Also, the inclusion/exclusion criteria is very globally representing. Other than that, this is the OCU410ST is targeting early to advanced cases of Stargardt disease. If you look at in the comparison, the safety and tolerability and efficacy that we have seen in terms of lesion growth reduction and also the functional and structural outcomes, has been trending in the right direction and promising from the clinical standpoint as well.

Speaker #1: Boris.

Speaker #3: Mr. Peaker, you might be on mute.

Speaker #5: Hear me? Sorry.

Speaker #3: Perfect. So for the RP400, for the rolling BLA, when would we get the FDA feedback on your CMC part of the filing?

Speaker #1: Typically, the CMC will be—also, we're planning to file this year. Obviously, I mean, FDA has the right to request comments before, or they will wait for the entire section to be filed. Even though they're internally reviewing, you may not expect anything before that.

Michael Okunewitch: Thank you. It's certainly an exciting time for the space and looking forward to any further updates that you have.

Shankar Musunuri: Thank you.

Operator: Thank you. Our next question comes from the line of Boris Peaker from Titan Partners. Please go ahead.

Speaker #1: Actual final clinical module is filed.

Speaker #3: Got it. And speaking of the FDA, have you discussed the ellipsoid zone as an endpoint with the agency? I'm just curious, what their thoughts about it as maybe kind of a secondary endpoint?

Shankar Musunuri: Boris?

Operator: Mr. Peaker, you might be on mute.

Speaker #3: Is it something that could be incorporated into a label claim? Would that make any kind of a difference from the commercial perspective? Just kind of general thoughts on that endpoint.

Speaker #1: Yeah, ellipsoid zone—I mean, obviously, as we stated before, all our clinical trials, we're trying to show a benefit because the diseases we're targeting have significant unmet medical needs.

Boris Peaker: Hear me? Sorry.

Operator: I'm-

Boris Peaker: Perfect. For the OCU400, for the rolling BLA, when would we get the FDA feedback on your CMC part of the filing?

Speaker #1: So more delays and doing longer trial trials will take not only the resources from our perspective, it's not doing benefit to the patients. If you're able to show a benefit using primary endpoints, what we picked, which are acceptable, obviously, the easy will be a secondary and some other analysis just to support further that demonstrating disease, showing a good functional outcome or related to functional outcome.

Shankar Musunuri: Typically, the CMC will be, also we're planning to file this year. I mean, FDA has right to, you know, request comments before, or they will wait for entire section to be filed. Even though they're internally reviewing, you may not expect anything before the actual final clinical module is filed.

Speaker #1: That's important. So, if you do longer trials, like two or three years, sure, we can look into multiple options. So, obviously, the agency's perspective—from the FDA's perspective—they really focus on the primary endpoint.

Boris Peaker: Got it. You know, speaking of the FDA, have you discussed the ellipsoid zone as an endpoint with the agency? I'm just curious what their thoughts about it as maybe, you know, kind of a secondary endpoint. Is it something that could be incorporated into a label claim? Would that make any kind of a difference from the commercial perspective? Just kind of general thoughts on that endpoint.

Speaker #1: If you hit the primary endpoint, you'll get the approval. If you hit the secondary, yes, you can include it in the product insert and the label.

Speaker #1: And however, remember, all our clinical trials, we have obligation to continue them for five years, for safety monitoring. And so that means one year data is needed for filing.

Shankar Musunuri: Yeah. Ellipsoid Zone, I mean, obviously, as we stated before, all our clinical trials, we're trying to show a benefit because diseases we are targeting have significant unmet medical needs. More delays and doing longer trials will take not only the resources from our perspective, it's not doing benefit to the patients. If you're able to show a benefit using primary endpoints, what we picked, which are acceptable, obviously, the EZ will be a secondary and some other analysis just to support further that, you know, demonstrating this is showing a good functional outcome or related to functional outcome. That's important. If you do longer trials, like two or three years, sure, we can look into multiple options. Obviously, the agency's perspective from FDA's perspective, they really focus on primary endpoint. If you hit the primary endpoint, you'll get the approval.

Speaker #1: After that, second year, third year, fourth year, and fifth year, we monitor the patients. Even we'll continue to monitor them with these secondary endpoints at any point.

Speaker #1: The data is looking at we can always add it to the label. And so from FDA's perspective, you have to hit the primary endpoint to get the product approved.

Speaker #1: Secondary is not necessary for approval. I mean, if you hit it, it's good. You can put it in the label.

Speaker #3: Got it. But I just want to understand also, have you spoken to docs? What's the commercial value? Let's say you could get an ellipsoid zone label claim.

Speaker #3: Obviously, not the primary endpoint, but still mentioned as positive in the label. Would that really make a difference? Is this something that the docs actually care about?

Shankar Musunuri: That's important. If you do longer trials, like two or three years, sure, we can look into multiple options. Obviously, the agency's perspective from FDA's perspective, they really focus on primary endpoint. If you hit the primary endpoint, you'll get the approval.

Speaker #3: Or is it just kind of scientifically nice and a curiosity more than anything else?

Speaker #1: Yeah, go ahead, Jim.

Speaker #4: Boris, this is Huma. So, in terms of your question, with FDA alignment—yes, all the protocols are approved with exploratory secondary endpoints. And yes, in terms of EAS—it's the new hot topic for the clinicians in terms of functional outcomes and structural integrity for photoreceptor and retinal pigment epithelium.

Shankar Musunuri: If you hit the secondary, yes, you can include it in the product insert and the label. However, remember, all our clinical trials, we have obligation to continue them for five years for safety monitoring. That means one year data is needed for filing. After that second year, third year, fourth year, and fifth year, we monitor the patients. Even we'll continue to monitor them with the secondary endpoints. At any point, the data is looking good, we can always add it to the label. From FDA's perspective, you have to hit the primary endpoint to get the product approved. Secondary is not necessary for approval. I mean, if you hit it's good. You can put it in the label.

Speaker #4: This is where actually FDA is leaning a lot based on these particular conditions such as Stargard disease and geographic atrophy, secondary to age-related macular degeneration.

Speaker #4: In fact, there has been a buy-in consensus from the IRD physicians, as well as the geographic atrophy AMD surgeons, as well. And there is a real benefit to it—not only from the structural perspective, but also from the functional.

Boris Peaker: Got it. I just want to understand also, have you spoken to docs? Like, what's the commercial value? Let's say you could get an Ellipsoid Zone label claim, you know, obviously not the primary endpoint, but still mentioned as positive in the label. Would that really make a difference? Is this something that the docs actually care about, or is it just kind of scientifically nice and a curiosity more than anything else?

Speaker #4: But yes, this is now being taken not only nationally in the US, but also from Europe as well. And of course, there is a clinical meaningfulness in terms of functional outcome for EYS611, and that's what we are seeing—that that could have potentially meaningful information when we are going to file our claim commercially.

Shankar Musunuri: Yeah. Go ahead, Huma. Yeah. Boris, this is Huma. So in terms of your question, with FDA alignment, yes, all the protocols are approved with exploratory secondary endpoints. Yes, in terms of EZ, it's the new hot topic for the clinicians, in terms of functional outcomes and structural integrity for photoreceptor and retinal pigment epithelium. This is where actually FDA is leaning a lot, based on, you know, these particular conditions such as Stargardt disease and geographic atrophy secondary to age-related macular degeneration. In fact, there has been a buying in consensus from the IRD physicians as well as the geographic atrophy AMD surgeons as well. There is a real benefit to it, not only from the structural perspective, but also from functional.

Speaker #1: And also, geographic atrophy, phase three didn't start yet. That's why we're going to look at entire phase two data set this month. And then we're going to propose the endpoints with FDA and DMA.

Speaker #1: So we do have an opportunity to introduce EZ as a secondary endpoint if the data is trending the way we anticipate.

Speaker #3: Great. Thank you very much for taking my questions.

Speaker #1: Thank you.

Speaker #3: Thank you. Our next question comes from Velas Wayampahula Ramakhan from HC Wainwright. Please go ahead.

Speaker #5: Thank you. Good morning, Shankarita and Huma. A couple of questions from me. Looking into the RQ410 program, in the phase two study, the medium dose showed a 54% reduction versus the high dose which showed a 36% reduction.

Shankar Musunuri: Yes, this is now being taken not only, you know, nationally in US, but also from Europe as well. Of course, there is a clinically meaningfulness in terms of functional outcome for EZ. That's what we are seeing, that, you know, that could have a potential meaningful information when we are going to file our claim commercially. Also geographic atrophy phase III didn't start yet. That's why, we're going to look at entire phase III data set this month, and then we're going to propose the endpoints with FDA and EMA. We do have an opportunity to introduce EZ as a secondary endpoint if the data is trending the way we anticipate.

Speaker #5: So I'm just trying to understand when as you go into your phase three study, what is going to impact your decision for dose selection?

Speaker #5: And also, do you think that between these doses, you are actually seeing some sort of a plateau effect in the transgene expression?

Boris Peaker: Great. Thank you very much for taking my questions.

Shankar Musunuri: Thank you.

Speaker #1: Okay, good morning. Yeah, I think the data we released—obviously, the high dose had fewer numbers in there. I would wait until we get the complete data set this month.

Operator: Thank you. Our next question comes from the line of Swayampakula Ramakanth from H.C. Wainwright. Please go ahead.

Swayampakula Ramakanth: Thank you. Good morning, Shankar, Rita, and Huma. Couple of questions from me. Looking into the OCU410 program, you know, in the phase 2 study, the medium dose showed a, you know, 54% reduction versus the high dose which showed a 36% reduction. I'm just trying to understand, you know, when as you go into your phase 3 study, what is going to impact your decision for dose selection? Also, do you think that between these doses, you are actually seeing some sort of a plateau effect in the transgene expression?

Speaker #1: To make any inference—and obviously, from the agency's perspective—if a lower dose is showing equal or better effect, I mean, you would take that into phase three.

Speaker #1: That's a standard practice. And so I suggest we wait. Typically, what we look for in our genes and what we have seen in our RP studies too, typically these genes require a threshold.

Speaker #1: Once you hit the threshold, we didn't see any dose response. So we're going to evaluate carefully once you get the full data set.

Speaker #5: Okay, thanks for that. And then in the subpopulations, where the baseline lesions were greater than equal to or greater than seven and a half millimeters square, you saw a 57% reduction in the lesion growth.

Speaker #5: So, as you get into the Phase 3 study, would you have any restrictions in terms of the size of the lesions? Or do you plan to use the same criteria as in Phase 2, which was the all-comers?

Shankar Musunuri: RK, good morning. Yeah. I think the data we released, obviously, the high dose had less numbers in there. I would wait until we get the complete data set this month to make any inference. Obviously, you know, Agency's perspective, if a lower dose is showing equal or better effect, I mean, you would take that into phase 3. That's a standard practice. I suggest, you know, we wait. Typically, what we look for in our genes and what we have seen in our RP studies too, typically these genes require a threshold. Once you hit the threshold, we didn't see any dose response. We're going to evaluate carefully once we get the full data set.

Speaker #1: Let's see. Good question. right? We're going to carefully evaluate and we go from what is the 2.5 to 22. And so we're going to evaluate and see because in the lower side, I mean, as you know, analytically, you'll have more variability.

Speaker #1: Of course, we're going to look at where the average patients fall, even though in the large trials, people are done with a lot of data.

Speaker #1: And we're going to look at all those metrics and see what is the right group to go into the phase three.

Swayampakula Ramakanth: Okay. Thanks for that. You know, in the subpopulations where the baseline lesions were greater than, equal to or greater than 7.5 mm², you saw a 57% reduction in the lesion growth. As you get into the phase 3 study, you know, would you have any restrictions in terms of the size of the lesions? Or do you plan to use the same criteria as in phase 2, which was the all-comers?

Speaker #5: All right. And then the last question from me is on the ST for RQ410 ST program, where you're expecting to get the enrollment done this quarter and put up some interim data?

Speaker #5: In Q3, in that data set, what are we really looking for which can give us some indication of how the 27 BLA filing is going to go?

Speaker #5: Especially I'm thinking about the signals on either on the structural side of things or on the functional side of things. And what do you weigh more and how should we be thinking when the data comes out?

Shankar Musunuri: That's a good question. Yes. This is why we do phase 2, right? We're going to carefully evaluate and we go from, what is it, 2.5 to 22.

Swayampakula Ramakanth: Mm-hmm.

Shankar Musunuri: We're going to evaluate and see. Because on the lower side, I mean, as you know, analytically, it'll have more variability. Of course, we're going to look at, you know, where the average patients fall, even though in the large trials people have done with a lot of data. We're going to look at all those metrics and see what is the right group to go into the phase 3.

Speaker #4: So RK, good morning. Thanks for your question. So in terms of the masked interim analysis that's coming later part of the year, will be for 24 subjects, 16 treatment, and 8 in the control.

Speaker #4: And this is the adaptive design—that's a unique approach we have taken. And what are the, what kind of data points are we going to present, as we have presented this morning as well?

Swayampakula Ramakanth: All right. Then the last question from me is on the ST for, OCU410ST program, where, you know, you're expecting to get the enrollment done this quarter and put up some interim data in Q3. In that data set, you know, what are we really looking for, which can give us some indication of how the 2027 BLA filing is gonna go? Especially I'm thinking about the signals on, either on the structural side of things or on the functional side of things and where, what do you weigh more, and how should we be thinking when the data comes out?

Speaker #4: Of course, the primary endpoint is the lesion growth reduction, as well as structural and functional outcomes, which include the visual acuity. And, last but not least, of course, we are looking into the ellipsoid zone, which is the functional outcome and is very unique.

Speaker #4: And that data was very well received from the Stargard perspective, that we have recently presented at one of the conferences as well. So yeah, we are going to look at all of that.

Speaker #4: And of course, safety and tolerability will be there as well as of right now it is trending in the right direction. And this is what we are looking into to release masked interim analysis for those subjects later part of the year.

Speaker #5: Perfect. Thank you very much. Thanks for taking all my questions.

[Company Representative] (Ocugen): RK, good morning. Thanks for your question. In terms of the masked interim analysis that's coming later part of the year will be for 24 subjects, 16 treatment and 8 in the control. This is the adaptive design that's a unique approach we have taken. What are the what kind of data points we are going to present as we have presented this morning as well, of course, the primary endpoint, the lesion growth reduction, as well as structural, as well as functional, which is the visual acuity. Not last but not the least, of course, we are looking into the Ellipsoid Zone, which is the functional outcome, which is very unique. That data was very well received from Stargardt perspective that we have recently presented at one of the conferences as well.

Speaker #1: Thank you.

Speaker #3: Thank you. Our next question comes from Velan of LMR Piros from Lucid Capital Markets. Please go ahead.

Speaker #6: Yes, good morning. I'd like to ask a question about the primary measure, visual function. In the RP study, what would be a clinically meaningful improvement?

Speaker #6: Where do you draw the threshold for that?

Speaker #4: So thanks for your question. So basically, as we said, that it's a change in Lux level improvement from baseline. And as you know, there is a lot of mutations we are looking into.

Speaker #4: Technically, one lux level and more, because it's a validated protocol—LDNA, luminance dependent navigation assessment. That's what we are aiming for, and that's what our analysis is going to be based off of.

[Company Representative] (Ocugen): Yeah, we are going to look all of that. Of course, safety and tolerability will be there as well. Right now, it is trending in the right direction, and this is what we are looking into to release a masked interim analysis for those subjects, later part in the year.

Speaker #4: And as I have said earlier as well, there is a lot of heterogeneity with clinical diagnosis and roaming non-syndromic forms. So of course, the clinically meaningfulness is greater than or equal to one Lux level.

Swayampakula Ramakanth: Perfect. Thank you very much. Thanks for taking all my questions.

Shankar Musunuri: Thank you.

Operator: Thank you. Our next question comes from the line of Elemer Piros from Lucid Capital Markets. Please go ahead.

Speaker #4: Depending on yes. Greater than or equal to one Lux.

Speaker #1: And I think, Elmer, as you might have stated, we've validated this course during Phase 3 with the real patients. And the course looks very robust.

Elemer Piros: Yes, good morning. I'd like to ask a question about the primary measure visual function in the RP study. What would be a clinically meaningful improvement? Where do you draw the threshold for that?

Speaker #1: And based on our KYL input, they're very extremely happy with this.

Speaker #3: Yeah, thank you. And what are some of the secondary endpoints that you will also look at to support the primary?

[Company Representative] (Ocugen): Thanks for your question. Basically, as we said that it's a change in LUX level improvement from baseline, and as you know, there is a lot of mutations we are looking into. Technically 1 LUX level and more because it's a validated protocol, LDNA Luminance Dependent Navigation Assessment. That's what we are aiming for, and that's what our analysis gonna be based off of. As I've said earlier as well, there is a lot of heterogeneity with clinical diagnosis, syndromic, and non-syndromic forms. Of course, the clinically meaningfulness is greater than or equal to 1 LUX level.

Speaker #4: So, of course, the secondary endpoints are, of course, on the visual acuity, low luminance visual acuity, and also the patient-reported outcome scores. We will be looking into it.

Speaker #4: And that is actually very well agreed and aligned upon with the FTA.

Speaker #6: And one last question. Or both eyes are treated if you could remind us.

Speaker #4: Yes, that is correct. Yes, if they meet the inclusion-exclusion criteria, both the eyes are treated. It's a two into one randomization, a single subretinal injection.

Speaker #4: And the control group will have a crossover after one year.

Elemer Piros: One LUX.

[Company Representative] (Ocugen): Depending on... Yes. Greater than or equal to 1 LUX.

Speaker #1: And yeah, and the study is the worst eye for analysis.

Shankar Musunuri: I think, Elemer, as we, Huma has stated, we validated this course during Phase 3 with the real patients. The course looks very robust. Based on our KOL input, they're extremely happy with this.

Speaker #6: So you would compare Diverse Eye to the control group in Diverse Eye in that group?

Speaker #1: Yeah, it's so there's a study eye. Yeah. Study eye is compared to the control group. Yeah. For the analysis.

Elemer Piros: Yeah. Thank you. What are some of the secondary endpoints that you will also, look at to support the primary?

Speaker #6: Thank you so much. Thank you, Shankar. Thanks, Huma.

[Company Representative] (Ocugen): Of course, the secondary endpoints are of course on the visual acuity, low luminance visual acuity, and also, you know, the patient-reported outcome scores. We will be looking into it, and that is actually very well, you know, agreed and aligned upon with the FDA.

Speaker #3: Thank you. And our next question comes from Velan of Danil Gataolin from Sharden. Please go ahead.

Elemer Piros: One last question. Are both eyes are treated, if you could remind us, in the treatment arm?

Speaker #7: Hi, this is Stephen Onford, Danil. Thanks for taking my question. For dry AMD, you mentioned a 50% responder rate. Were there any underlying characteristics that made a patient more likely to be a responder?

[Company Representative] (Ocugen): Yes, that is correct.

Elemer Piros: Yeah, okay.

[Company Representative] (Ocugen): Yes. If you meet the inclusion/exclusion criteria, both the eyes are treated. It's a 2 into 1 randomization, a single subretinal injection. The control group will have a crossover after 1 year.

Speaker #4: Are you talking about the 410J? Yes. So in terms of that, the responder rate by the way, we have the inclusion-exclusion criteria, which was very well uniform across the groups.

Shankar Musunuri: Yeah.

[Company Representative] (Ocugen): Under standard of care.

Shankar Musunuri: The study eye is the worst eye for analytic analysis. It's set.

Elemer Piros: You would compare the worst eye to the control group and the worst eye in that group.

Speaker #4: And it was not the baseline characteristics were that there was a mean age that we were looking into. And of course, the GA gets diagnosed at a certain age.

Shankar Musunuri: Yeah. It's so the study eye, yeah.

Elemer Piros: Uh-

Shankar Musunuri: Is compared to the control group. Yeah.

Speaker #4: But of course, 70 in the mid-70s was the mean age. We were also looking at the lesion size, which actually is pretty much well worse with the Oaks and Derby trials and Apple has got approval squared.

Elemer Piros: Yeah.

Shankar Musunuri: From the analysis.

Elemer Piros: Thank you so much.

Shankar Musunuri: Primary. Yeah.

Elemer Piros: Thank you, Shankara. Thanks, Huma.

Operator: Thank you. Now our next question comes from the line of Daniil Gataulin from Chardan. Please go ahead.

Speaker #4: That was the mean as well up to 8.03. And in terms of the baseline characteristics, the responders basically responded on the medium dose as well as on the high dose as well.

[Analyst] (Chardan): Hi, this is Stephen on for Daniel. Thanks for taking my question. For dry AMD, you mentioned a 50% responder rate. Were there any underlying characteristics that made a patient more likely to be a responder?

Stephen Urbach: Hi, this is Stephen on for Daniel. Thanks for taking my question. For dry AMD, you mentioned a 50% responder rate. Were there any underlying characteristics that made a patient more likely to be a responder?

Speaker #4: So there was not really any other unique criteria that we would say at this point till we get our final clinical study report at that point.

Speaker #4: But at this point, it seemed like it was uniform across all dose groups.

[Company Representative] (Ocugen): Are you talking about the OCU410 GA?

[Analyst] (Chardan): Yeah.

Stephen Urbach: Yeah.

[Company Representative] (Ocugen): Yes. In terms of that, the responder rate, by the way, we have the inclusion/exclusion criteria, which was very well uniform across the groups. It was, the baseline characteristics were that, you know, there was a mean age that we were looking into. Of course, the GA gets diagnosed at a certain age, but of course 70 in the mid-seventies was the mean age. We were also looking at, you know, the lesion size, which actually is pretty much well worse with the OAKS and DERBY trials, and Apellis has got approval on it, was 7.5 millimeters square. That was the mean as well, up to 8.03.

Speaker #3: Got it. Thank you. Thank you. This concludes the Q&A portion. I will now turn the call back over to Chairman CEO and co-founder Dr. Shankar Musunuri.

Speaker #1: Thank you, operator. 2025 was marked by important clinical progress, strategic business development, and essential financing accomplishments across the organization. We are entering 2026 with a strong momentum and a clear line of sight to multiple catalysts that will further advance Occasion's position as a biotechnology leader in gene therapy for blindness diseases.

[Company Representative] (Ocugen): In terms of the baseline characteristics, the responders basically responded, you know, on the medium dose as well as on the high dose as well. There was not really any other unique criteria that we would say at this point till we get our final, you know, clinical study report at that point. At this point it seemed like it was uniform across all dose groups.

Speaker #1: We expect to deliver full phase two data for OQ410 this month, complete enrollment for OQ410 ST, initiate phase three for OQ410 in geographic atrophy, and begin our rolling BLA submission for OQ400.

Speaker #1: I want to thank our employees, investigators, patients, and shareholders for their continued support. We look forward to updating you on our progress. Have a great day.

[Analyst] (Chardan): Got it. Thank you.

Stephen Urbach: Got it. Thank you.

Operator: Thank you. This concludes the Q&A portion. I will now turn the call back over to Chairman, CEO, and Co-founder, Dr. Shankara Musunuri.

Shankar Musunuri: Thank you, operator. 2025 was marked by important clinical progress, strategic business development, and essential financing accomplishments across the organization. We're entering 2026 with a strong momentum and a clear line of sight to multiple catalysts that will further advance Ocugen's position as a biotechnology leader in gene therapy for blindness diseases. We expect to deliver full phase II data for OCU410 this month, complete enrollment for OCU410ST, initiate phase III for OCU410 in geographic atrophy, and begin our rolling BLA submission for OCU400. I want to thank our employees, investigators, patients, and shareholders for their continued support. We look forward to updating you on our progress. Have a great day.

Operator: The meeting is now concluded. Thank you all for joining. You may now disconnect.

Q4 2025 Ocugen Inc Earnings Call

Request a DemoDemo

OCGN

Ocugen

Earnings