Q2 2026 Ocugen Inc Earnings and Business Update Call
Speaker #1: Good morning and welcome to Ocugen's second quarter 2026 financial results and business update. All participants are in listen-only mode. Following the speaker's commentary, there will be a question-and-answer session.
Operator: Good morning, and welcome to Ocugen's Q2 2026 financial results and business update. All participants are in listen-only mode. Following the speakers' commentary, there will be a question-and-answer session. I will now turn the call over to Chris Clark, Ocugen's Head of Corporate Communications. You may begin.
Operator: Good morning, and welcome to Ocugen's Q2 2026 Financial Results and Business Update. All participants are in listen-only mode. Following the speakers' commentary, there will be a question-and-answer session. I will now turn the call over to Chris Clark, Ocugen's Head of Corporate Communications. You may begin.
Speaker #1: I will now turn the call over to Chris Clark, Ocugen's Head of Communications. You may begin.
Speaker #2: 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.
Chris Clark: 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 30 June 2026. Abhi Gupta, Executive Vice President of Commercial and Business Development, and Dr. Mohamed Genead, 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 Q2 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.
Chris Clark: 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 30 June 2026. Abhi Gupta, Executive Vice President of Commercial and Business Development, and Dr. Mohamed Genead, 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 Q2 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.
Speaker #2: Rita Johnson-Green, our Chief Financial Officer, is also on the call to provide a financial update for the quarter ended June 30, 2026. Avi Gupta, Executive Vice President of Commercial and Business Development, and Dr. Mohammad Janeed, who joined Ocugen as Chief Medical Officer in June, will be available to answer questions following the presentation.
Speaker #2: 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.
Speaker #2: 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.
Chris Clark: 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 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 Q2 2026, will be filed today. I will now turn the call over to Dr. Musunuri.
Chris Clark: 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 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 Q2 2026, will be filed today. I will now turn the call over to Dr. Musunuri.
Speaker #2: 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.
Speaker #2: We encourage you to review our filings with the securities and exchange commission, including the risk factors detailed therein, for more comprehensive understanding of these potential risks.
Speaker #2: 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.
Speaker #3: Thank you, Chris, and good morning, everyone. The second quarter was a defining one for Ocugen. The FDA cleared our Phase 3 trial for OCU410 to initiate dosing in geographic atrophy patients and granted RMAT designation for the program.
Shankar Musunuri: Thank you, Chris, good morning, everyone. The Q2 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 $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 in 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.
Shankar Musunuri: Thank you, Chris, good morning, everyone. The Q2 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 $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 in 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.
Speaker #3: 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.
Speaker #3: And 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.
Speaker #3: 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 in ophthalmology has been confined to a single gene, a single mutation, and a single small patient population.
Speaker #3: 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 mastoregulators, nuclear hormone receptors, that govern multiple gene networks, the platform is gene agnostic, inherently benefit from a single one-time subretinal injection.
Shankar Musunuri: 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 United States and Europe, a combined patient population, and a commercial opportunity far larger than anything currently served by approved gene therapies in ophthalmology.
Shankar Musunuri: 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 United States and Europe, a combined patient population, and a commercial opportunity far larger than anything currently served by approved gene therapies in ophthalmology.
Speaker #3: What this means in practice is that Ocugen is not building three separate drugs, where 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.
Speaker #3: Retinitis pigmentosa, RRP, star guard disease, and geographic atrophy, RGA, together affect approximately 3 million people across the United States and Europe, a combined patient population, and the commercial opportunity for larger than anything currently served by approved gene therapies in ophthalmology.
Speaker #3: Across our pipeline, spanning Phase 1 through Phase 3, we have treated more than 325 patients, including EAP, through multiple doses and indications, and we have not observed a drug-related serious adverse event.
Shankar Musunuri: Across our pipeline, spanning phase I through phase III, we have treated more than 325 patients, including EAP, 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 H1 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, a secondary to dry age-related macular degeneration or dry AMD. GA represents our largest commercial opportunity, with approximately 2 to 3 million patients in the US and Europe combined. There are currently no approved treatments for GA in Europe.
Shankar Musunuri: Across our pipeline, spanning phase I through phase III, we have treated more than 325 patients, including EAP, 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 H1 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, a secondary to dry age-related macular degeneration or dry AMD. GA represents our largest commercial opportunity, with approximately 2 to 3 million patients in the US and Europe combined. There are currently no approved treatments for GA in Europe.
Speaker #3: We remain on track to file three BLAs by 2028. This positions the first half of 2027 as a catalyst-rich window for Ocugen, with the top-line data for Ocu400 and Ocu410ST, and our planned BLA submissions following over a short period.
Speaker #3: Let me walk you through how each program is advancing. Then I will hand over the call to Rita for financials. Starting with Ocu410 for GA, a secondary-to-dry age-related macular degeneration, or dry AMD, GA represents our largest commercial opportunity with approximately 2 to 3 million patients in the US and Europe combined, that are currently no approved treatments for GA in Europe.
Speaker #3: Current approved therapies in the US target only one complement pathway and require frequent intravitreal injections which has been associated with treatment discontinuation in clinical practice.
Shankar Musunuri: Current approved therapies in the US target only one complement pathway and require frequent intraocular injections, which has been associated with treatment discontinuation in clinical practice. GA is a multifactorial disease driven by four 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 US address only one of these four 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 four 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.
Shankar Musunuri: Current approved therapies in the US target only one complement pathway and require frequent intraocular injections, which has been associated with treatment discontinuation in clinical practice. GA is a multifactorial disease driven by four 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 US address only one of these four 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 four 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.
Speaker #3: GA is a multifactorial disease, driven by four distinct pathways that contribute to the progressive degeneration, of the macula. Drusen, inflammation, oxidative stress, and complement ward activation.
Speaker #3: The currently approved therapies in the US address only one of these four pathways, the complement system, which is partly why there have been unable to demonstrate meaningful functional outcomes for patients.
Speaker #3: Ocu410 operates differently, by delivering RORA, a nuclear hormone receptor, that acts as a mastoregulator of retinal homeostasis, Ocu410 is designed to address all four disease pathways simultaneously, with a single subretinal injection has the potential to redefine the standard of care in this indication.
Speaker #3: We recently received FDA clearance for Ocu410 Phase 3 registration trial for GA, the trial Armada 3 is planned to be a global study of approximately 237 subjects, using an adaptive design covered at 95% for the primary endpoint, with the BLA and market authorization application filings targeted for 2028.
Shankar Musunuri: 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 have to be used in our phase III pivotal trial versus control. Approximately twice the benefit of approved complement inhibitors and from a single injection.
Shankar Musunuri: 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 have to be used in our phase III pivotal trial versus control. Approximately twice the benefit of approved complement inhibitors and from a single injection.
Speaker #3: We plan to initiate Phase 3 by September 2026. This design is anchored by positive 12-month data from our Phase 2 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 mm2, and 17.5 mm2.
Speaker #3: The criteria to be used in our Phase 3 portal trial versus control: approximately twice the benefit of approved complement inhibitors, and from a single injection.
Speaker #3: We also saw a 27% preservation of the ellipsoid zone within the same patient population in no drug-related serious adverse events reported to date. Importantly, these Phase 2 data help support the FDA's decision to grant ARMA designation for Ocu410.
Shankar Musunuri: 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, these phase II data help 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 US 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 1 April, we announced the completion of enrollment and dosing in our phase II/III Guardian-3 pivotal confirmatory trial, enrolling 63 participants.
Shankar Musunuri: 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, these phase II data help 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 US 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 1 April, we announced the completion of enrollment and dosing in our phase II/III Guardian-3 pivotal confirmatory trial, enrolling 63 participants.
Speaker #3: Turning to Ocu410ST for star guard disease, star guard is a pediatric onset retinal disorder affecting approximately 100,000 patients in the US and Europe, and roughly 1 million people globally.
Speaker #3: There are no approved therapies available for these patients today. Ocu410ST is designed to address both 1,200 pathogenic mutations in the ABCA4 gene with a single one-time treatment.
Speaker #3: On April 1, we announced the completion of enrollment and dosing in our Phase 2/3 Guardian 3 portal confirmatory trial, enrolling 63 participants. We expect the interim outcome decision for the first 50% of subjects at 8 months in the third quarter of 2026, and top-line Phase 2/3 data in the second quarter of 2027, with our BLA submission followed mid-2027.
Shankar Musunuri: We expect the interim outcome decision for the first 50% of subjects at 8 months in Q3 2026, and top line phase II/III data in Q2 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 US 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.
Shankar Musunuri: We expect the interim outcome decision for the first 50% of subjects at 8 months in Q3 2026, and top line phase II/III data in Q2 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 US 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.
Speaker #3: Moving to Ocu400 for RP, the Phase 3 limelight trial is the first and largest genetic medicine registration trial for broad RP, spanning more than 30 genetic Approximately 300,000 people in the US and Europe are living with RP, which is caused by mutations in more than 100 genes.
Speaker #3: 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 fundamentally different commercial opportunity.
Speaker #3: Enrollment in the limelight is complete, with 140 patients randomized 2 to 1, treated versus control, across the row, and gene-agnostic arms spanning more than 30 genetic mutations, associated with early to late-stage RP, including pediatrics.
Shankar Musunuri: Enrollment in the liMeliGhT is complete, with 140 patients randomized 2 to 1, treated versus control, across the RORA 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 1 year post-DVC for the primary endpoint analysis. Topline phase III data is expected in Q1 2027, advancing OCU400 to a potential approval in Q4 2027. FDA feedback confirmed that the path to rolling BLA submission remains tied to topline data expected in Q1 2027.
Shankar Musunuri: Enrollment in the liMeliGhT is complete, with 140 patients randomized 2 to 1, treated versus control, across the RORA 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 1 year post-DVC for the primary endpoint analysis. Topline phase III data is expected in Q1 2027, advancing OCU400 to a potential approval in Q4 2027. FDA feedback confirmed that the path to rolling BLA submission remains tied to topline data expected in Q1 2027.
Speaker #3: 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.
Speaker #3: Subjects are followed for one year post-dosing for the primary endpoint analysis. Top-line Phase 3 data is expected in the first quarter of 2027, advancing OCU400 to a potential approval in the fourth quarter of 2027.
Speaker #3: FDA feedback confirmed that the path to ruling BLA submission remains tied to top-line data expected in the first quarter of 2027. On the manufacturing side, our process performance qualification, PPQs, badges, are complete, supporting BLA and commercial launch supplies.
Shankar Musunuri: 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 are 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.
Shankar Musunuri: 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 are 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.
Speaker #3: Brand planning and marketing initiatives led by Abhigupta 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, since July we signed a binding term sheet with root pharmaceutical and its strategic partner, LDAO International Holding, for exclusive rights to Ocu400 in the Middle East and North Africa, where active on the BD front to find other global partners for regional commercialization partnerships where RP is most prevalent.
Speaker #3: 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.
Speaker #3: 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.
Shankar Musunuri: 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. Rita?
Shankar Musunuri: 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. Rita?
Speaker #3: 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.
Speaker #3: Third, we're mapping the patient journey from diagnosis through treatment and long-term follow-up, with the goal of facilitating a seamless experience for patients and caregivers and healthcare providers.
Speaker #3: Fourth, we're 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.
Speaker #3: With that, I'll turn the call over to Rita, for the financial update. Rita?
Speaker #1: Thank you, Shankar. Good morning, everyone. Total operating expenses for the three months ended June 30, 2026, were $17.9 million. And included research and development expenses of $10.7 million.
Rita Johnson-Greene: Thank you, Shankar. Good morning, everyone. Total operating expenses for the three months ended 30 June 2026, were $17.9 million, and included research and development expenses of $10.7 million, and general and administrative expenses of $7.2 million. This compares to total operating expenses for the three months ended 30 June 2025, of $15.2 million, which included research and development expenses of $8.4 million, and general and administrative expenses of $6.8 million. Total operating expenses for the six months ended 30 June 2026, were $37.3 million, and included research and development expenses of $21.9 million, and general and administrative expenses of $15.4 million. This compares to the total operating expenses for the six months ended 30 June 2025, of $31.2 million, which included research and development expenses of $17.9 million, and general and administrative expenses of $13.2 million.
Rita Johnson-Greene: Thank you, Shankar. Good morning, everyone. Total operating expenses for the three months ended 30 June 2026, were $17.9 million, and included research and development expenses of $10.7 million, and general and administrative expenses of $7.2 million. This compares to total operating expenses for the three months ended 30 June 2025, of $15.2 million, which included research and development expenses of $8.4 million, and general and administrative expenses of $6.8 million. Total operating expenses for the six months ended 30 June 2026, were $37.3 million, and included research and development expenses of $21.9 million, and general and administrative expenses of $15.4 million. This compares to the total operating expenses for the six months ended 30 June 2025, of $31.2 million, which included research and development expenses of $17.9 million, and general and administrative expenses of $13.2 million.
Speaker #1: And general and administrative expenses of $7.2 million. This compares to total operating expenses for the three months ended June 30, 2025, of $15.2 million.
Speaker #1: Which included research and development expenses of $8.4 million, and general and administrative expenses of $6.8 million. Total operating expenses for the six months ended June 30, 2026, were $37.3 million.
Speaker #1: and included research and development expenses of $21.9 million, and general and administrative expenses of $15.4 million. This compares to total operating expenses for the six months ended June 30, 2025, of $31.2 million.
Speaker #1: Which included research and development expenses of $17.9 million, and general and administrative expenses of $13.2 million. OcuGen reported a 7-cent net loss per common share for the three months ended June 30, 2026, compared to a 5-cent net loss per common share for the three months ended June 30, 2025.
Rita Johnson-Greene: Ocugen reported a $0.07 net loss per common share for the three months ended 30 June 2026, compared to a $0.05 net loss per common share for the three months ended 30 June 2025. On our capital position, following the closing of the $130 million convertible notes financing, the company's cash equivalents, and restricted cash totaled $100.4 million as of 30 June 2026, extending our cash runway into 2028. The company had 339 million shares of common stock outstanding as of 30 June 2026. That concludes my financial update. Shankar, back to you.
Rita Johnson-Greene: Ocugen reported a $0.07 net loss per common share for the three months ended 30 June 2026, compared to a $0.05 net loss per common share for the three months ended 30 June 2025. On our capital position, following the closing of the $130 million convertible notes financing, the company's cash equivalents, and restricted cash totaled $100.4 million as of 30 June 2026, extending our cash runway into 2028. The company had 339 million shares of common stock outstanding as of 30 June 2026. That concludes my financial update. Shankar, back to you.
Speaker #1: On our capital position, following the closing of the $130 million convertible note financing, the company's cash cash equivalence and restricted cash totaled $100.4 million.
Speaker #1: As of June 30, 2026, extending our cash runway into 2028. The company had $339 million shares of common stock outstanding as of June 30, 2026.
Speaker #1: That concludes my financial update, Shankar, back to you.
Speaker #2: Thank you, Rita. The second quarter is a quarter of execution. The remainder of 2026 is poised to be impactful. We expect the Ocu410 SD interim outcome decision in the third quarter, and we expect to initiate the Ocu410 Phase 3 trial in this quarter.
Shankar Musunuri: Thank you, Rita. The Q2 was a quarter of execution. The remainder of 2026 is poised to be impactful. We expect the OCU410ST interim outcome decision in the Q3, and we expect to initiate the OCU410 phase III trial in this quarter. Looking to 2027, we expect topline data from both OCU400 and OCU410ST in the H1 of the year, followed by our planned BLA submissions. Each of these milestones brings us a step closer to delivering on our commitment to three BLAs by 2028, offering potentially life-altering improvement to patients coping with blindness-causing diseases. I want to thank our investigators and patients who have trusted us with their participation, and our shareholders for their continued belief in our mission to advance cures for blindness. We'll now open the call for questions. Operator?
Shankar Musunuri: Thank you, Rita. The Q2 was a quarter of execution. The remainder of 2026 is poised to be impactful. We expect the OCU410ST interim outcome decision in the Q3, and we expect to initiate the OCU410 phase III trial in this quarter. Looking to 2027, we expect topline data from both OCU400 and OCU410ST in the H1 of the year, followed by our planned BLA submissions. Each of these milestones brings us a step closer to delivering on our commitment to three BLAs by 2028, offering potentially life-altering improvement to patients coping with blindness-causing diseases. I want to thank our investigators and patients who have trusted us with their participation, and our shareholders for their continued belief in our mission to advance cures for blindness. We'll now open the call for questions. Operator?
Speaker #2: Looking to 2027, we expect top-line data from both Ocu400 and Ocu410 SD in the first half of the year. Followed by our planned BLA submissions.
Speaker #2: Each of these milestones brings us a step closer to delivering on our commitment to three BLAs by 2028, offering potentially life-altering improvement to patients coping with blindness causing diseases.
Speaker #2: I want to thank our investigators, and patients who have trusted us with their participation and our shareholders for their continued belief in our mission to advance cures for blindness.
Speaker #2: We'll now open the call for questions. Operator?
Speaker #4: Thank you. Ladies and gentlemen, we will now begin the question-and-answer session. And at this time, I would like to remind everyone in order to ask a question, please press star, followed by the number one on your telephone keypad.
Operator: Thank you. Ladies and gentlemen, we will now begin the question and answer session. At this time, I would like to remind everyone, in order to ask a question, please press star followed by the number 1 on your telephone keypad. If you would like to withdraw your question, simply press star 1 again. Again, if you would like to ask a question, press star 1 on your telephone keypad. Our first question comes from the line of Michael Okunewitch with Maxim Group. Please go ahead.
Operator: Thank you. Ladies and gentlemen, we will now begin the question and answer session. At this time, I would like to remind everyone, in order to ask a question, please press star followed by the number 1 on your telephone keypad. If you would like to withdraw your question, simply press star 1 again. Again, if you would like to ask a question, press star 1 on your telephone keypad. Our first question comes from the line of Michael Okunewitch with Maxim Group. Please go ahead.
Speaker #4: And if you would like to withdraw your question, simply press star one again. Again, if you would like to ask a question, press star one on your telephone keypad.
Speaker #4: Our first question comes from the line of Michael Oconwich with Maxim Group. Please go ahead.
Speaker #5: Hey there. Thank you so much for taking my questions, and congrats on all the great progress.
Michael Okunewitch: Hey there. Thank you so much for taking my questions, congrats on all the great progress.
Michael Okunewitch: Hey there. Thank you so much for taking my questions, congrats on all the great progress.
Speaker #6: Thank you, Michael.
Shankar Musunuri: Thank you, Michael.
Shankar Musunuri: Thank you, Michael.
Speaker #5: So I wanted to ask you, now have a handful of international partnerships, which makes Ocu400 a truly international program at this point. So I just wanted to see if you could share the regulatory plans in particular for XUS jurisdictions, what's required there, and how those timelines could vary versus your BLA path.
Michael Okunewitch: I wanted to ask, you now have a handful of international partnerships, which makes OCU400 a truly international program at this point. I just wanted to see if you could share the regulatory plans, in particular for ex-US jurisdictions, what's required there, and how those timelines could vary versus your BLA path.
Michael Okunewitch: I wanted to ask, you now have a handful of international partnerships, which makes OCU400 a truly international program at this point. I just wanted to see if you could share the regulatory plans, in particular for ex-US jurisdictions, what's required there, and how those timelines could vary versus your BLA path.
Speaker #2: Michael, what we have with Ocu400, we got alignment from EMA in addition to FDA. With the same that single trial we're doing in the US is good for approvals.
Shankar Musunuri: Michael, what we have with OCU400, we got alignment from EMA in addition to FDA with the same, that single trial we're doing in US is good for approvals. Across the globe for orphan gene therapies, typically, they get approval based on US approval. Everything will be linked to our US FDA approval in MENA and other regions.
Shankar Musunuri: Michael, what we have with OCU400, we got alignment from EMA in addition to FDA with the same, that single trial we're doing in US is good for approvals. Across the globe for orphan gene therapies, typically, they get approval based on US approval. Everything will be linked to our US FDA approval in MENA and other regions.
Speaker #2: And across the globe, for orphan gene therapies, typically they get approval based on US approval. So everything will be linked to our US FDA approval.
Speaker #2: In MENA and other regions.
Speaker #5: All right. Thank you. And then I wanted to see also if you could just highlight some of the key differences in the trial design between our Modic 3 and the Phase 2 Armada trial.
Michael Okunewitch: All right. Thank you. I wanted to see if also if you could just highlight some of the key differences in the trial design between ARMADA-3 and the phase II ArMaDa trial.
Michael Okunewitch: All right. Thank you. I wanted to see if also if you could just highlight some of the key differences in the trial design between ARMADA-3 and the phase II ArMaDa trial.
Speaker #2: Yeah, I let our CMO, Dr. Jeanine, answer that.
Shankar Musunuri: I let our CMO, Dr. Genit, answer that.
Shankar Musunuri: I let our CMO, Dr. Genit, answer that.
Speaker #6: Thank you, Michael. Regarding Armada R3, which is our global Phase 2 trial for GA, we just received approval from the FDA recently to initiate it this quarter.
Mohamed Genead: Thank you, Michael. Regarding ARMADA-3, which is our global, phase III trial for GA, which we just got the approval from FDA just recently to be initiated this quarter. The phase III trial design for ARMADA-3 will be one treatment arm with OCU410 versus a control, will be 2 to 1 randomization allocation. The idea is to follow each subject up to 12 months, and this is where we're going to be looking at the primary efficacy endpoint plus other key functional endpoint. The ArMaDa, the earlier phase I/II GA trials was similar on the efficacy, we should expect similar outcome here. The numbers obviously is different. We're going to be enrolling in ARMADA-3 close to 237 subject into one allocation. It's going to be global. We're going to go ex-US.
Mohamed Genead: Thank you, Michael. Regarding ARMADA-3, which is our global, phase III trial for GA, which we just got the approval from FDA just recently to be initiated this quarter. The phase III trial design for ARMADA-3 will be one treatment arm with OCU410 versus a control, will be 2 to 1 randomization allocation. The idea is to follow each subject up to 12 months, and this is where we're going to be looking at the primary efficacy endpoint plus other key functional endpoint. The ArMaDa, the earlier phase I/II GA trials was similar on the efficacy, we should expect similar outcome here. The numbers obviously is different. We're going to be enrolling in ARMADA-3 close to 237 subject into one allocation. It's going to be global. We're going to go ex-US.
Speaker #6: The Phase 3 trial designed for Armada 3 will be one treatment arm with Ocu410 versus a control will be 2 to 1 randomization allocation.
Speaker #6: And that data to follow each subject up to 12 months. And this is where we're going to be looking at the primary efficacy endpoint, plus other key functional endpoint.
Speaker #6: The Armada one, the earlier Phase 1, 2 GA trials was similar. On the efficacy, so we should expect, you know, similar outcome here. We're going to look the numbers obviously is different.
Speaker #6: We're going to be enrolling in Armada 3 close to 237 subjects in 2-1 allocation. It's going to be global. We're going to go XUS.
Speaker #6: We're going to go to Europe and other territorial part in the world. But the primary endpoint will be very similar so we should expect to see similar trend what we saw from Armada 1, the Phase 1, 2 AGA trial.
Mohamed Genead: We're going to go to Europe and other territorial part in the world. The primary endpoint will be very similar, so we should expect to see similar trend, what we saw from ArMaDa 1, the phase I/II GA trial.
Mohamed Genead: We're going to go to Europe and other territorial part in the world. The primary endpoint will be very similar, so we should expect to see similar trend, what we saw from ArMaDa 1, the phase I/II GA trial.
Speaker #5: All right, thank you. And then just one last one for me before I hop back into the queue. So, it looks like, in Stargardt, there is a chance that we'll have an approved therapy sometime around when you'll be completing your own BLA filing.
Michael Okunewitch: All right. Thank you. Just one last one from me before I hop back into the queue. It looks like in Stargardt, there is a chance that we'll have an approved therapy sometime around when you'll be completing your own BLA filing. It'd be a chronic therapy versus a one-time. I wanted to ask how important the pricing on other therapies, since we don't have any pricing comps, would be to inform your own pricing strategy, and if there's any way that we can think about how to translate pricing between a chronic ongoing therapy and a one-time therapy.
Michael Okunewitch: All right. Thank you. Just one last one from me before I hop back into the queue. It looks like in Stargardt, there is a chance that we'll have an approved therapy sometime around when you'll be completing your own BLA filing. It'd be a chronic therapy versus a one-time. I wanted to ask how important the pricing on other therapies, since we don't have any pricing comps, would be to inform your own pricing strategy, and if there's any way that we can think about how to translate pricing between a chronic ongoing therapy and a one-time therapy.
Speaker #5: So, it'll be a chronic therapy versus a one-time. But I wanted to ask how important the pricing on other therapies is, since we don't have any pricing comps, to inform your own pricing strategy.
Speaker #5: And if there's any way that we can think about how to translate pricing between a chronic ongoing therapy and a one-time therapy.
Speaker #2: Yeah. Good question, Michael. I think I think the way you look at it is our treatments are one-and-done treatments potentially. So that'll have a different pricing structure than ongoing chronic therapies.
Shankar Musunuri: Yeah. Good question, Michael. I think the way you look at it is, our treatments are one and done treatments potentially, so that'll have a different pricing structure than ongoing chronic therapies. Number two, everything will be dictated by data, and if you have a safe one-time treatment, I think our gene therapies, once again, we're still collecting data, as you can see in some of the patients in RP, and as they approach like second year, third year, they're improving further. The current therapies, if the oral therapy comes to the market, what patients, providers are going to look for is the therapy just reducing the degeneration of the disease? Or in some patients, is it stalling it? Is it has potential to reverse it in some patients? At least, with our modifier gene therapy, in some of the patients, we're seeing all those trends.
Shankar Musunuri: Yeah. Good question, Michael. I think the way you look at it is, our treatments are one and done treatments potentially, so that'll have a different pricing structure than ongoing chronic therapies. Number two, everything will be dictated by data, and if you have a safe one-time treatment, I think our gene therapies, once again, we're still collecting data, as you can see in some of the patients in RP, and as they approach like second year, third year, they're improving further. The current therapies, if the oral therapy comes to the market, what patients, providers are going to look for is the therapy just reducing the degeneration of the disease? Or in some patients, is it stalling it? Is it has potential to reverse it in some patients? At least, with our modifier gene therapy, in some of the patients, we're seeing all those trends.
Speaker #2: Number two, everything will be dictated by data. And if you have a safe one-time treatment, I think our gene therapies, once again, you know, we're still collecting data.
Speaker #2: As you can see in some of the patients in RP, and as they approach like second year, third year, they're improving further. So the current therapies, if the oral therapy comes to the market, what patients providers are going to look for is the therapy just reducing the degeneration of the disease, or in some patients is it stalling it, is it has potential to reverse it in some patients.
Speaker #2: At least, you know, with our modified gene therapy, in some of the patients we're seeing all those trends. So that could be a big differentiating factor.
Shankar Musunuri: That could be a big differentiating factor. Also, as you know, Stargardt impacts a lot of pediatric patients, and the current clinical trial they're conducting focuses on 12 plus, and our clinical trial focuses on three plus. There are a lot of differentiators. Whenever we come for pricing, because of the differentiated disruptive technology platform we have, and obviously everybody will focus on safety, efficacy, and one and done treatment, that'll be more compliant for anyone. I think all those factors will be rolled in. I don't think we'll be truly comparing any pricing what the other chronic therapies are doing. If you have a me-too products, the answer is yes. If you have truly a differentiated disruptive technology, just completely different, we can price it on its own merits.
Shankar Musunuri: That could be a big differentiating factor. Also, as you know, Stargardt impacts a lot of pediatric patients, and the current clinical trial they're conducting focuses on 12 plus, and our clinical trial focuses on three plus. There are a lot of differentiators. Whenever we come for pricing, because of the differentiated disruptive technology platform we have, and obviously everybody will focus on safety, efficacy, and one and done treatment, that'll be more compliant for anyone. I think all those factors will be rolled in. I don't think we'll be truly comparing any pricing what the other chronic therapies are doing. If you have a me-too products, the answer is yes. If you have truly a differentiated disruptive technology, just completely different, we can price it on its own merits.
Speaker #2: And also, as you know, Stargardt impacts a lot of pediatric patients. And the current clinical trial, they're conducting focuses on 12 plus. And our clinical trial focuses on 3 plus.
Speaker #2: So there are a lot of differentiators. So whenever we come for pricing, because of the differentiated disruptive technology platform we have, and obviously everybody will focus on safety, efficacy, and one-and-done treatment that'll be more compliant for anyone.
Speaker #2: So I think all those factors will be rolled in. So I don't think we'll be truly comparing any pricing what the other chronic therapies are doing.
Speaker #2: If you have a MeToo product, the answer is yes. But if you have truly a differentiated, disruptive technology—just completely different—it'll come; we can price it on its own merits.
Speaker #5: All right. Thank you. I appreciate the additional color.
Michael Okunewitch: All right. Thank you. I appreciate the additional color.
Michael Okunewitch: All right. Thank you. I appreciate the additional color.
Speaker #4: Our next question comes from the line with the IGM Mechanical Genuity. Please go ahead.
Operator: Our next question comes from the line of Whitney Aitken with Canaccord Genuity. Please go ahead.
Operator: Our next question comes from the line of Whitney Aitken with Canaccord Genuity. Please go ahead.
Speaker #7: Hey guys, Mike, congrats on all the progress as well. Just to keep going in the Stargardt discussion, Shankar, since you mentioned it, can you talk about a little bit more, I guess, around the TPP here and the potential to show kind of reversal of disease and improvement in visual acuity?
Whitney Aitken: Hey, guys. Mike, congrats on all the progress as well. Just to keep going on the Stargardt discussion, Shankar, since you mentioned it, can you talk about a little bit more, I guess, around the TPP here and the potential to show kind of reversal of disease and improvement in visual acuity? Is that something that is reasonable to expect given the duration of follow-up in the ongoing phase II/III study? I guess if so, is there anything that was done in terms of entry criteria to maybe enrich for that outcome as far as patient baseline characteristics?
Whitney Ijem: Hey, guys. Mike, congrats on all the progress as well. Just to keep going on the Stargardt discussion, Shankar, since you mentioned it, can you talk about a little bit more, I guess, around the TPP here and the potential to show kind of reversal of disease and improvement in visual acuity? Is that something that is reasonable to expect given the duration of follow-up in the ongoing phase II/III study? I guess if so, is there anything that was done in terms of entry criteria to maybe enrich for that outcome as far as patient baseline characteristics?
Speaker #7: Is that something that is reasonable to expect given the duration of follow-up in the ongoing Phase 2, 3 study? And I guess if so, is there anything that was done in terms of entry criteria to maybe enrich for that outcome as far as patient baseline characteristics?
Shankar Musunuri: I will ask Dr. Genit to talk about a little bit about baseline characteristics, I'll answer the other question. Go ahead.
Shankar Musunuri: I will ask Dr. Genit to talk about a little bit about baseline characteristics, I'll answer the other question. Go ahead.
Speaker #2: I will ask Dr. Jeanine to talk about a little bit about baseline characteristics, then I'll answer the other question. Go ahead.
Speaker #6: Thank you, Shankar. Hi, Whitney. Yes, happy to answer. So our population was definitely broader than other competitors. Just to highlight first, we included patients from early to late stage target disease.
Mohamed Genead: Thank you, Shankar. Hi, Whitney. Yes, happy to answer. Our population was definitely broader than other competitor. Just to highlight first, we included patients from early to late stage Stargardt disease. That's number 1. As Shankar just mentioned, 2, we included subjects are younger than young adults. We included subjects 3 plus years of age, so that's a very broad population. As you know, for Stargardt, the earlier, the better, especially it's a progressive retinal degeneration disease. The lesion size we also included in our trial, the phase II/III GARDian trial, was more broader than what we saw with others. Our lesion size includes smaller lesion, also larger lesion. We have a broad spectrum, and that's also going to be aligned with our early late-stage strategy for the disease.
Mohamed Genead: Thank you, Shankar. Hi, Whitney. Yes, happy to answer. Our population was definitely broader than other competitor. Just to highlight first, we included patients from early to late stage Stargardt disease. That's number 1. As Shankar just mentioned, 2, we included subjects are younger than young adults. We included subjects 3 plus years of age, so that's a very broad population. As you know, for Stargardt, the earlier, the better, especially it's a progressive retinal degeneration disease. The lesion size we also included in our trial, the phase II/III GARDian trial, was more broader than what we saw with others. Our lesion size includes smaller lesion, also larger lesion. We have a broad spectrum, and that's also going to be aligned with our early late-stage strategy for the disease.
Speaker #6: That's number one. As Shankar just mentioned, too, we included subjects, you know, our younger than, you know, young adults. We included, you know, subject, you know, 3 plus, years of age.
Speaker #6: So that's a very broad population. As you know, for Stargardt, the earlier the better, especially it's a progressive retinal degeneration disease. The lesion size, we also included in our trials, the Phase 2, 3 Guardian trial was more broader than what we saw with others.
Speaker #6: And our lesion size and include, you know, smaller lesion and also larger lesion. So we have, you know, broad spectrum. And that's also going to be aligned with our early, late stage strategy.
Speaker #6: For the disease, we already included, you know, some of the subject in our, you know, Phase 2, 3 trials. So we will be excited to see the data.
Mohamed Genead: We already included some of the subjects in our phase II/III trials, we will be excited to see the data. In addition to the gene mutation, specifically, we include all the variants and all the other specific mutation included in the ABCA4-related retinopathy, it includes Stargardt and others as well. This is also on the disease indication as well, overall. Based on your point about the functional, I think this is going to be critical. We saw from our phase I data that we just published at the Eye Nature early in the year. We saw a very clear slowing in the structural progression in those patients. Also we saw functional benefit in those patients.
Mohamed Genead: We already included some of the subjects in our phase II/III trials, we will be excited to see the data. In addition to the gene mutation, specifically, we include all the variants and all the other specific mutation included in the ABCA4-related retinopathy, it includes Stargardt and others as well. This is also on the disease indication as well, overall. Based on your point about the functional, I think this is going to be critical. We saw from our phase I data that we just published at the Eye Nature early in the year. We saw a very clear slowing in the structural progression in those patients. Also we saw functional benefit in those patients.
Speaker #6: In addition to the gene mutation, specifically we include all the variants and all the other specific mutation included in the ABCA4 related retinopathy. So to include Stargardt and others as well.
Speaker #6: So this is also on the disease indication it overall. Based on your point about the functional, I think, you know, this is going to be a critical.
Speaker #6: So we saw from our Phase 1 data, that we just published at the eye, nature, you know, early in the year, we saw, you know, very clear, you know, structure slowing in the structure progression in these patients.
Speaker #6: And also we saw functional benefit in these patient. And as you remember, as you know, in Stargardt disease, the first target is to hold that progression, to stop, you know, losing more retinal structure and function, which we achieved in our prior trial.
Mohamed Genead: As you remember, as you know, in Stargardt disease, the first target is to hold that progression, to stop losing more retinal structure and function, which we achieved in our prior trial. The second goal, which will be the upside here, and the ultimate goal, to reverse that tide. Try to improve on the progress or improve on the disease outcome. We saw that in our phase I too. We saw some of the patients did improve in visual function. The gain was 6 letters, close to 1 line between the treated versus the untreated eye. We felt also very excited about the functional gain in those patient population. That's where we think the big differentiation, the broader application of our molecule.
Mohamed Genead: As you remember, as you know, in Stargardt disease, the first target is to hold that progression, to stop losing more retinal structure and function, which we achieved in our prior trial. The second goal, which will be the upside here, and the ultimate goal, to reverse that tide. Try to improve on the progress or improve on the disease outcome. We saw that in our phase I too. We saw some of the patients did improve in visual function. The gain was 6 letters, close to 1 line between the treated versus the untreated eye. We felt also very excited about the functional gain in those patient population. That's where we think the big differentiation, the broader application of our molecule.
Speaker #6: The second goal which will be, you know, the upside here, you know, and the ultimate goal to reverse, you know, that tight, right, you know, to improve on the progress or improve on the disease outcome.
Speaker #6: And we saw that in our Phase 1, 2. We saw some of the patient did improve in visual function, the gain was 6 letters close to one line between the treated versus the untreated eye.
Speaker #6: So we felt also very excited about the functional gain in these patient population. So that's kind of where we think, you know, the big differentiation, the broader application of our molecule.
Speaker #2: Whitney, just to clarify the primary endpoint, because it's a one-year trial, it's not a two-year trial. It's still a lesion. Then there are secondary visual function will be monitoring.
Shankar Musunuri: Whitney, just to clarify, the primary endpoint, because it's a 1-year trial, it's not a 2-year trial, it's still a lesion. There are secondary visual function we'll be monitoring. In addition to that, at the time of filing, we continue to monitor our early-stage phase I patients, we'll have long-term data in those patients, too.
Shankar Musunuri: Whitney, just to clarify, the primary endpoint, because it's a 1-year trial, it's not a 2-year trial, it's still a lesion. There are secondary visual function we'll be monitoring. In addition to that, at the time of filing, we continue to monitor our early-stage phase I patients, we'll have long-term data in those patients, too.
Speaker #2: In addition to that, at the time of filing, we continue to monitor our early stage Phase 1 patients. And so we'll have a long-term data in those patients too.
Speaker #7: Got it. Really helpful. And then just last question, and maybe Rita, this one's for you. Just can you help us understand how you're thinking about cash given the exciting progress with the GA study and the ability to start that study in September?
Whitney Aitken: Got it. Really helpful. Just last question, and maybe Rita, this one's for you. Just can you help us understand how you're thinking about cash, given the exciting progress with the GA study and the ability to start that study in September, I think you said. If there is a need to pull levers to extend cash runway further, how should we think about maybe the startup of GA versus commercial prep for RP or Stargardt, just how you guys are thinking about those different levers if needed? Thanks.
Whitney Ijem: Got it. Really helpful. Just last question, and maybe Rita, this one's for you. Just can you help us understand how you're thinking about cash, given the exciting progress with the GA study and the ability to start that study in September, I think you said. If there is a need to pull levers to extend cash runway further, how should we think about maybe the startup of GA versus commercial prep for RP or Stargardt, just how you guys are thinking about those different levers if needed? Thanks.
Speaker #7: I think you said if there is a need to kind of pull levers to extend cash runway further, how should we think about maybe the startup of GA versus commercial prep for RP or Stargardt and just kind of how you guys are thinking about those different levers if needed?
Speaker #7: Thanks.
Speaker #3: Yeah, thank you, Whitney. So, first of all, our primary goal is to make sure that we are minimizing shareholder dilution, but also evaluating our opportunities to raise capital, just as you said, in order to bring these novel products to patients.
Rita Johnson-Greene: Yeah. Thank you, Whitney. First of all, our primary goal is to make sure that we are minimizing shareholder dilution, but evaluating our opportunities in order to raise capital, just as you said, in order to bring these novel products to patients. Just first of all, we have cash runway into 2028, I just want to remind everyone of that, which gives us the confidence to execute our clinical, our late-stage products that we have. Progress to BLA submission for both OCU410 and OCU410ST in 2027, with the potential to commercialize OCU400 by the end of the year in 2027. We do have some additional levers that we can pull. One, we have the PRV for OCU410ST, given the RPD designation that we have.
Rita Johnson-Greene: Yeah. Thank you, Whitney. First of all, our primary goal is to make sure that we are minimizing shareholder dilution, but evaluating our opportunities in order to raise capital, just as you said, in order to bring these novel products to patients. Just first of all, we have cash runway into 2028, I just want to remind everyone of that, which gives us the confidence to execute our clinical, our late-stage products that we have. Progress to BLA submission for both OCU410 and OCU410ST in 2027, with the potential to commercialize OCU400 by the end of the year in 2027. We do have some additional levers that we can pull. One, we have the PRV for OCU410ST, given the RPD designation that we have.
Speaker #3: So just first of all, we have cash runway into 2028. And so I just want to remind everyone of that, which gives us the confidence to execute our, you know, our clinical state, our late stage products that we have.
Speaker #3: And then progress to BLA submission for both OCU 410 and OCU 410 ST. In 2027, with the potential to commercialize, OCU 400 by the end of the year in 2027.
Speaker #3: We do have some additional levers that we can pull, one, you know, we have the PRV for OCU 410 ST given the RPD designation that we have.
Speaker #3: And so of course we have the ability to sell that for somewhere between, you know, 100 to 200, even prior to, even prior to approval.
Rita Johnson-Greene: Of course, we have the ability to sell that for somewhere between $100 to $200, even prior to approval, that's something that we are evaluating. We also have various business development deals that we are looking at from a globalization perspective. We're looking at ex-US for both OCU400, OCU410ST, and even GA, right? Just depending upon what that term sheet looks like. Always looking for potential deals that we can make in order to, again, just minimize that dilution. We also have the Janus Henderson warrants, right? There's another 10 million warrants at a $1.50 strike price, which could bring in another $15 million. Those warrants expire in August of 2027.
Rita Johnson-Greene: Of course, we have the ability to sell that for somewhere between $100 to $200, even prior to approval, that's something that we are evaluating. We also have various business development deals that we are looking at from a globalization perspective. We're looking at ex-US for both OCU400, OCU410ST, and even GA, right? Just depending upon what that term sheet looks like. Always looking for potential deals that we can make in order to, again, just minimize that dilution. We also have the Janus Henderson warrants, right? There's another 10 million warrants at a $1.50 strike price, which could bring in another $15 million. Those warrants expire in August of 2027.
Speaker #3: And that's something that we are evaluating. We also have very business development deals that we are looking at, you know, from a globalization perspective.
Speaker #3: We're looking at XUS for both OCU 400, OCU 410 ST, and even GA, right, just depending upon what that term sheet looks like. So always looking for, you know, potential deals that we can make in order to, you know, again, just minimize that dilution.
Speaker #3: We also have the Janis Henderson warrants, right? There's another $10 million warrants at $1.50 strike price, which could bring in another $15 million and those warrants don't expire in August of 2027.
Rita Johnson-Greene: Of course, we anticipate a special meeting in September of this year in order to increase authorized shares, which will give us the ability to raise additional equity if we decide to do so. Again, just looking at both non-dilutive as well as dilutive options in order to make sure that we are able to bring these amazing and novel products to patients, as well as looking at maximizing shareholder value.
Speaker #3: And then, of course, you know, we anticipate a special meeting in September of this year in order to increase authorized shares, which will give us the ability to raise additional equity if we decide to do so.
Rita Johnson-Greene: Of course, we anticipate a special meeting in September of this year in order to increase authorized shares, which will give us the ability to raise additional equity if we decide to do so. Again, just looking at both non-dilutive as well as dilutive options in order to make sure that we are able to bring these amazing and novel products to patients, as well as looking at maximizing shareholder value.
Speaker #3: So again, you know, just looking at both non-dilutive as well as dilutive options in order to make sure that we are able to bring these amazing and novel products to patients as well as looking at maximizing shareholder value.
Speaker #7: Very helpful. Thank you.
Whitney Aitken: Very helpful. Thank you.
Whitney Ijem: Very helpful. Thank you.
Speaker #1: Our next question comes from the line of Charles. While it's with HD Wade Wright, please go ahead.
Operator: Our next question comes from the line of Charles Wallace with H.C. Wainwright. Please go ahead.
Operator: Our next question comes from the line of Charles Wallace with H.C. Wainwright. Please go ahead.
Speaker #5: Hi, this is Charles from HD Wade Wright. I'm for RK. Thanks for taking my question. Maybe a question on Armada 3 design. So it seems like based on the prior earnings call that the study has been a little bit resized.
Charles Wallace: Hi. This is Charles from H.C. Wainwright. I am for RK. Thanks for taking my question. Maybe a question on the ARMADA-3 design. It seems like based on the prior earnings call, the study has been a little bit resized. I think previously you said it would be about 300 patients, and now it is 237 patients. I was just curious if this was something the FDA specifically asked for, or if this was something you proposed. And then also if the assumptions changed based on effect size, variability, dropout, or the narrower lesion size compared to the phase II. Dr. Vinit?
Charles Wallace: Hi. This is Charles from H.C. Wainwright. I am for RK. Thanks for taking my question. Maybe a question on the ARMADA-3 design. It seems like based on the prior earnings call, the study has been a little bit resized. I think previously you said it would be about 300 patients, and now it is 237 patients. I was just curious if this was something the FDA specifically asked for, or if this was something you proposed. And then also if the assumptions changed based on effect size, variability, dropout, or the narrower lesion size compared to the phase II.
Speaker #5: I think previously you said it would be about 300 patients and now it's 237 patients. So I was just curious, if this was something the FDA specifically asked for or if this was something you proposed, and then also if the assumptions changed based on effect size, variability, dropout, or the narrower lesion size compared to the Phase 2.
Speaker #2: Yeah, Dr. Vineeth.
Shankar Musunuri: Dr. Vinit?
Speaker #4: Thank you, Charles. Yeah, so we had a discussion with the agency, the FDA. So all this being aligned and discussed, with the FDA. But to answer your question specifically, it was based on all on the sample size estimation and also the power calculation we did.
Mohamed Genead: Thank you, Charles. We had a discussion with the agency, the FDA. All this being aligned and discussed with the FDA. To answer your question specifically, it was based on the sample size estimation and also the power calculation we did. The estimate you are citing, the 300, was based on estimate. When we saw the effect size based on our ArMaDa, the phase I/II trial, as we discussed today, we saw the 31% reduction in the medium dose, the optimal dose, which is the one we are taking forward. When we did our calculation based on that, we saw the 237 total population to be enrolled will give us 95% power in our pivotal trial. All these pieces have been discussed with the agency. It is based on the rate of change, the slope analysis for the primary efficacy.
Mohamed Genead: Thank you, Charles. We had a discussion with the agency, the FDA. All this being aligned and discussed with the FDA. To answer your question specifically, it was based on the sample size estimation and also the power calculation we did. The estimate you are citing, the 300, was based on estimate. When we saw the effect size based on our ArMaDa, the phase I/II trial, as we discussed today, we saw the 31% reduction in the medium dose, the optimal dose, which is the one we are taking forward. When we did our calculation based on that, we saw the 237 total population to be enrolled will give us 95% power in our pivotal trial. All these pieces have been discussed with the agency. It is based on the rate of change, the slope analysis for the primary efficacy.
Speaker #4: So the estimate you're citing, the 300 was based on estimate. But when we saw the effect size based on our Armada, one, the Phase 1, 2 trial, and as we discussed today, we saw the 31% reduction in the medium dose, the optimal dose, which is the one we are taking forward.
Speaker #4: When we did our calculation based on that, we saw, you know, the 237 total population to be enrolled will give us 95% power in our pivotal trial.
Speaker #4: All these pieces have been discussed with the agency, obviously this based on the rate of change, the slope analysis for the primary efficacy. So the effect size, you know, based on what we saw from earlier trial was very positive and was strong enough that we end up, you know, with 237, 2 to 1 randomization as we mentioned earlier, 158 in the treatment arm, and 79 in the control arm.
Mohamed Genead: The effect size, based on what we saw from earlier trial, was very positive and was strong enough that we end up with 237. Two-to-one randomization, as we mentioned earlier, 158 in the treatment arm and 79 in the control arm. All this has been discussed and aligned, and as we announced today, we got the clearance from the FDA to initiate our phase III trial in the next few weeks.
Mohamed Genead: The effect size, based on what we saw from earlier trial, was very positive and was strong enough that we end up with 237. Two-to-one randomization, as we mentioned earlier, 158 in the treatment arm and 79 in the control arm. All this has been discussed and aligned, and as we announced today, we got the clearance from the FDA to initiate our phase III trial in the next few weeks.
Speaker #4: So all this have been discussed and aligned and as we announced today, we got the clearance from the FDA to initiate our Phase 3 trial in the next few weeks.
Speaker #5: Thank you. Very helpful. And then I guess for on the rolling submission, so I think the originally the guidance was to submit in the third quarter.
Charles Wallace: Thank you. Very helpful. I guess for the rolling submission. I think originally the guidance was to submit in Q3, and now I believe it's Q1 after the liMeliGhT data. I guess my question is, what kind of changed between submitting the non-clinical module earlier compared to after the top line data, the liMeliGhT?
Charles Wallace: Thank you. Very helpful. I guess for the rolling submission. I think originally the guidance was to submit in Q3, and now I believe it's Q1 after the liMeliGhT data. I guess my question is, what kind of changed between submitting the non-clinical module earlier compared to after the top line data, the liMeliGhT?
Speaker #5: And now I believe it's the first quarter after the limelight data and, you know, I think, I guess my question is, what kind of changed between submitting the non-clinical module earlier compared to after the top line data, the limelight?
Speaker #2: I mean, Charles, I think from our perspective, we're ready. I mean, I think we're doing very well with our PPQs, as we mentioned. A lot of gene therapy companies stuck with CMC.
Shankar Musunuri: Charles, I think from our perspective, we are ready. I think we're doing very well with our PPQs, as we've mentioned. A lot of gene therapy companies start with CMC. We're ahead of the game. We used commercial scale large in our phase III. We completed our PPQs on time. We got non-clinical and PPQ are done, so we have CMC non-clinical ready to go. Obviously, this is where we have to work with the agency whenever they're comfortable, and that's the timeline they gave us, and we're going to be fine with that. The reason is, I just want to clarify, it's good to have rolling submission that gives a head start for agency. Okay? It's for their own benefit. If they want to wait until next year, we are ready to file it as soon as the top line comes for the pre-BLA meeting.
Shankar Musunuri: Charles, I think from our perspective, we are ready. I think we're doing very well with our PPQs, as we've mentioned. A lot of gene therapy companies start with CMC. We're ahead of the game. We used commercial scale large in our phase III. We completed our PPQs on time. We got non-clinical and PPQ are done, so we have CMC non-clinical ready to go. Obviously, this is where we have to work with the agency whenever they're comfortable, and that's the timeline they gave us, and we're going to be fine with that. The reason is, I just want to clarify, it's good to have rolling submission that gives a head start for agency. Okay? It's for their own benefit. If they want to wait until next year, we are ready to file it as soon as the top line comes for the pre-BLA meeting.
Speaker #2: We're ahead of the game. We used to come still scale lots in our Phase 3. We completed our PPQs on time because non-clinical and PPQ are done.
Speaker #2: So we have CMC non-clinical ready to go. I mean, obviously, this is where we have to work with the agency whenever they're comfortable. And that's the timeline they gave us.
Speaker #2: And we're going to be fine with that. The reason is I just want to clarify it's good to have rolling submission that gives a head start for agency, okay?
Speaker #2: It's for their own benefit. And if they want to wait until next year, I mean, we are ready to file it as soon as the top line comes for the 3BLA meeting, we may still give them a head start of maybe a month or two months before we drop the clinical section.
Shankar Musunuri: We may still give them a head start of maybe a month or two months before we drop the clinical section. However, I just want to clarify, until the final BLA is completed with the clinical section, the PDUFA date, the accelerated clock of six months doesn't start. I just want to clarify that. Once again, this is a collaboration between the sponsor and the agency. In this case, of course, we respect their decision, whatever they are, because they have a lot of programs and a lot of workload. Whatever the reasons are, we are fine with it. I think we're ready from our perspective, and we'll work with them closely in a collaborative way. Whenever we have a top line, we'll be ready to file it.
Shankar Musunuri: We may still give them a head start of maybe a month or two months before we drop the clinical section. However, I just want to clarify, until the final BLA is completed with the clinical section, the PDUFA date, the accelerated clock of six months doesn't start. I just want to clarify that. Once again, this is a collaboration between the sponsor and the agency. In this case, of course, we respect their decision, whatever they are, because they have a lot of programs and a lot of workload. Whatever the reasons are, we are fine with it. I think we're ready from our perspective, and we'll work with them closely in a collaborative way. Whenever we have a top line, we'll be ready to file it.
Speaker #2: So however, I just want to clarify until the final BLA is completed with the clinical section, the PDUFA date, the accelerated clock of six months doesn't start.
Speaker #2: I just want to clarify that. So once again, this is a collaboration between the sponsor and the agency. In this case, of course, we respect the decision, whatever they are, because they have a lot of programs and a lot of workload, whatever the reasons are, we are fine with it.
Speaker #2: I think we're ready from our perspective. And we'll work with them closely and collaborative way. And whenever we have a top line, we'll be ready to file it.
Charles Wallace: Great. Very helpful.
Charles Wallace: Great. Very helpful.
Shankar Musunuri: That it doesn't change any filing clock, as we mentioned before. Q2, complete the BLA filing, anticipated approval in Q4, 6 months accelerated clock.
Shankar Musunuri: That it doesn't change any filing clock, as we mentioned before. Q2, complete the BLA filing, anticipated approval in Q4, 6 months accelerated clock.
Speaker #2: So it doesn't change any yeah, filing clock, as we mentioned before, second quarter complete the BLA filing. Anticipated approval in fourth quarter, six months accelerated clock.
Speaker #5: Very helpful. Thanks for taking both questions.
Charles Wallace: Very helpful. Thanks for taking both questions.
Charles Wallace: Very helpful. Thanks for taking both questions.
Speaker #1: Once again, if you would like to ask a question, please press star followed by the number one on your telephone keypad. Our next question comes from the line of Robert LeBoyer with Noble Capital Markets.
Operator: Once again, if you would like to ask a question, please press star followed by 1 on your telephone keypad. Our next question comes from the line of Robert LeBoyer with Noble Capital Markets. Please go ahead.
Operator: Once again, if you would like to ask a question, please press star followed by 1 on your telephone keypad. Our next question comes from the line of Robert LeBoyer with Noble Capital Markets. Please go ahead.
Speaker #1: Please go ahead.
Speaker #5: Good morning and congratulations on the progress. Just to follow up on that last question, my understanding was that the BLA submission will be completed in early 2027 when the clinical module is filed.
Robert LeBoyer: Good morning, and congratulations on the progress. Just to follow up on that last question. My understanding was that the BLA submission will be completed in early 2027 when the clinical module is filed. That's when you get the PDUFA date and the approval launch is based on that. You also have rolling submission and have the option of filing the CMC and the other non-clinical modules before that. Is that still your plan?
Robert LeBoyer: Good morning, and congratulations on the progress. Just to follow up on that last question. My understanding was that the BLA submission will be completed in early 2027 when the clinical module is filed. That's when you get the PDUFA date and the approval launch is based on that. You also have rolling submission and have the option of filing the CMC and the other non-clinical modules before that. Is that still your plan?
Speaker #5: That's when you get the PDUFA date and the approval launches based on that. But you also have rolling submission and have the option of filing the CMC and the other non-clinical modules before that.
Speaker #5: Is that still your plan?
Speaker #2: Yes. Yes, Robert. Absolutely. Because based on agencies' suggestion, recommendation, as soon as the top line comes out, we'll have a pre-BLA meeting. Right after that, we can file the two modules non-clinical and CMC modules.
Shankar Musunuri: Yes. Yes, Robert. Absolutely. Based on agency's suggestion and recommendation, as soon as the top line comes out, we'll have a pre-BLA meeting. Right after that, we can file the two modules, non-clinical and CMC modules. That will still give them a head start. As soon as the clinical module is done, when you file it, the PDUFA date starts.
Shankar Musunuri: Yes. Yes, Robert. Absolutely. Based on agency's suggestion and recommendation, as soon as the top line comes out, we'll have a pre-BLA meeting. Right after that, we can file the two modules, non-clinical and CMC modules. That will still give them a head start. As soon as the clinical module is done, when you file it, the PDUFA date starts.
Speaker #2: So that will still give them a head start. Then as soon as the clinical module is done, when you file it, the PDUFA update starts.
Speaker #2: So that's basically our plan is to file that in second quarter, so six months clock should be fourth quarter approval clock.
Robert LeBoyer: Yes
Robert LeBoyer: Yes
Shankar Musunuri: Our plan is to file that in Q2. 6 months clock should be Q4, approval clock.
Shankar Musunuri: Our plan is to file that in Q2. 6 months clock should be Q4, approval clock.
Speaker #5: Okay, terrific. Thank you for that.
Robert LeBoyer: Okay. Terrific. Thank you for that.
Robert LeBoyer: Okay. Terrific. Thank you for that.
Speaker #1: And at this time, we have no further questions. I would like to turn the call back over to the OCTAGEN team for closing remarks.
Operator: At this time, we have no further questions. I would like to turn the call back over to the Ocugen team for closing remarks.
Operator: At this time, we have no further questions. I would like to turn the call back over to the Ocugen team for closing remarks.
Speaker #2: Thank you all for attending today's webcast. Really appreciate all our investors, shareholders, patients, providers, thank you.
Shankar Musunuri: Thank you all for attending today's webcast. Really appreciate all our investors, shareholders, patients, and providers. Thank you.
Shankar Musunuri: Thank you all for attending today's webcast. Really appreciate all our investors, shareholders, patients, and providers. Thank you.
Operator: This concludes today's conference call. You may now disconnect. Have a good day.
Operator: This concludes today's conference call. You may now disconnect. Have a good day.