Q2 2024 Ocugen Inc Earnings Call

August 8, 2024

Tiffany Hamilton: This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainty. We may, in some cases, use terms such as predicts, believes, potential, proposed, continue, estimate, anticipates, expects, plans, intends, may, could, might, will, should, or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements include, but are not limited to, statements regarding our clinical development activities and related anticipated timelines.

Operator: Good morning and welcome to Ocugen's second quarter, 2024 financial result and business update. Please note that this call is being recorded at this time. All participant lines are in a listen-only mode.

Good morning and welcome to Ocugen's 2nd quarter 2024 Financial Results and Business Update. Please note that this call is being recorded at this time.

Operator: Following the speaker commentary, there will be a question-and-answer session.

Tiffany Hamilton: All participant lines are in a listen-only mode. Following the speaker commentary, there will be a question and answer session. I will now turn the call over to Tiffany Hamilton, Ocugen's Head of Corporate Communication. You may begin.

Tiffany Hamilton: I will now turn the call over to Tiffany Hamilton, Ocugen's Head of Corporate Communication. You may begin. Thank you, operator, and good morning, everyone.

Tiffany Hamilton: Such statements are subject to numerous important risk factors and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filing with the Security and Exchange Commission, SEC, including the risk factors described in the section entitled risk, and the quarterly and annual reports that we file with the S. The Swayampakula Ramakanth, Swayampakula Ramakan, and forward-looking statements that we make in this presentation speak only as of the date of this presentation, except as required by law.

Tiffany Hamilton: Joining me on today's call in 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. Michael Breininger, our corporate controller, is also on the call to provide a financial update for the quarter-ended June 30, 2024. Dr. Huma Qamar, Chief Medical Officer, will be available to answer questions following the presentation.

Tiffany Hamilton: We assume no obligation to update forward-looking statements contained in this presentation, whether as a result of new information, future events, or otherwise after the date of this presentation. Finally, Ocugen's quarterly report on Form 10-Q, covering the second quarter of 2024, has been filed. I will now turn the call over to Dr. Musunuri.

Tiffany Hamilton: Thank you, Operator, and good morning, everyone.

Shankar Musunuri: Joining me on today's call and webcast is Dr. Shankar Musunuri, Oxygen's Chairman, CEO, and Co-Founder, who will provide a business update and an overview of our clinical and operational progress.

Speaker Change: michael brianer our corporate controller is also on the call to provide a financial update for the quarter ended june thirty two thousand and twenty four dr whuma amar chief medical officer will be available to answer questions following the presentation

Tiffany Hamilton: This morning, we issued a press release detailing associated business and operational highlights for the second quarter of 2024. We encourage listeners to review the press release, which is available on our website at Ocugen.com.

Speaker Change: This morning, we issued a press release detailing associated business and operational highlights for the second quarter of 2024.

Shankar Musunuri: we encourage listeners to review the press release which is available on our website and oxygen dot com this call is being recorded and a replay with the accompany'side presentation will be available on the investor sections of the ocxygen website were approximately forty-five days

Tiffany Hamilton: This call is being recorded, and a replay with the accompanying slide presentations will be available on the investor section of the Ocugen website for approximately 45 days.

Tiffany Hamilton: This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as predicts, believes, potential proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should, or other words that can say uncertainty or future events or outcomes to identify these forward-looking statements. But statements include, but are not limited to, statements regarding our clinical development activity and related anticipated timelines. Such statements are subject to numerous important risk factors and uncertainties that may cause actual events or results to differ materially from our current expectations.

Speaker Change: this presentation contains forward-looking statementsment within the meaning of the private securities litigation reform act one thousandnine and inety five which are subject to risks and uncertainties

Shankar Musunuri: We may, in some cases, use terms such as, projects believe potential proposed continue estimates

Shankar Musunuri: Anticipates, expects, plans, intends, may, could, might, will, should, or other words that can say uncertainty or future events or outcomes to identify these forward-looking statements.

Shankar Musunuri: Such statements include, but are not limited to, statements regarding our clinical development activities and related anticipated timelines. Such statements are subject to numerous important risk factors and uncertainties that may cause actual events or results.

Tiffany Hamilton: These and other risks and uncertainties are more fully described in our periodic filing with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled "Risk Factors" in the quarterly and annual reports that be filed with the SEC. And in forward-looking statements that we make in this presentation speak only as of the date of this presentation, except as required by law. We assume no obligation to update forward-looking statements contained in this presentation, whether as a result of new information, future events, or otherwise, after the date of this presentation.

Shankar Musunuri: These and other risks and uncertainties are more fully described in our periodic filing with the Securities and Exchange Commission, SEC, including the risk factors described in the section entitled Risk Factors, in the quarterly and annual reports that we file with the SEC.

Shankar Musunuri: Any forward-looking statements that we make in this presentation speak only as of the date of this presentation, except as required by law. We assume no obligation to update forward-looking statements contained in this presentation, whether as a result of new information, future events, or otherwise, after the date of this presentation.

Tiffany Hamilton: Finally, applicants' quarterly reports on Form 10-Q, covering the second quarter of 2024, have been filed.

Dr. Musunuri: Finally, Ocugen's quarterly report on Form 10-Q covering the second quarter of 2024 has been filed. I will now turn the call over to Dr. Musunuri.

Shankar Musunuri: I will now turn the call over to Dr. Mousenari. Thank you, Tiffany. And thank you all for joining us today.

Shankar Musunuri: Thank you, Tiffany, and thank you all for joining us today. We're excited to discuss the substantial progress of our Modified Gene Therapy platform across all three clinical programs. And to continue driving these programs, we recently completed a successful fundraising effort with net proceeds of $32.6 million, extending our runway into the third quarter of 2025. Our scientific advances and the strategic growth of the company were further acknowledged by our inclusion in the Russell Index in June.

Shankar Musunuri: We're excited to discuss a substantial progress of our modified inter-p-platform across all three clinical programs. And to continue driving these programs, we recently completed a successful fundraising effort that net proceeds of $32.6 million, extending your runway into the third quarter of 2025. Our scientific advances and the strategic growth of company were further acknowledged by our inclusion in the Russell Index in June. This ranking demonstrates the value of a pipeline and supports Occidence's dedication to creating long-term shareholder value. Additionally, the recent offering was led by a premier mutual fund, along with participation from leading life sciences investors, which further strengthens our shareholder base.

Speaker Change: thank you disney and thank you all for joining us today we're exhiited to discuss a substantial progress for martdifer enttherapy platform across all three clinical programs

Speaker Change: And to continue driving these programs, we recently completed a successful fundraising effort with net proceeds of $32.6 million, extending our runway into the third quarter of 2025.

Speaker Change: our scientific advances and the strategic growth of company were further acknowledged by our inclusion in the russell index in june is ranking demonstration the value of pipeline and supports arygen dedication to createating long-term shareholder value

Shankar Musunuri: This ranking demonstrates the value of the pipeline and supports Ocugen's dedication to creating long-term shareholder value. Additionally, the recent offering was led by a premier mutual fund, along with participation from leading life sciences investors, which further strengthens our shareholder base.

Dr. Musunuri: Additionally, the recent offering was led by a premier mutual fund, along with participation from leading life sciences investors, which further strengthens our shareholder base.

Shankar Musunuri: We are actually recruiting patients in our Q400-based-relimelight clinical trial for the treatment of Agonitis Pigmentosa RP. And just this week, we announced a DA approval for an expanded access program (EAP) for the treatment of adult patients aged 18 and older with RP with Q400. This is the first-ever gene therapy candidate to treat patients with RP, regardless of mutation, approved for EAP. We also progressed into the Q400-based-to-armada clinical trial for the treatment of geographic atrophy, an advanced stage of triage-related macular degeneration.

Shankar Musunuri: We're actually recruiting patients in our up to 400 pastry lime-light clinical trial for the treatment of, And just this week, we announced FDA approval for an Expanded Access Program, EAP, for the treatment of adult patients aged 18 and older with RP with RT-400. This is the first ever gene therapy candidate to treat patients with RP, regardless of mutation, approved for EAP. We also progressed into the OCU410 Phase 2 ARMADA clinical trial for the Treatment of Geographic Atrophy, an Advanced Stage of Tri-Age-Related Macular Degeneration, following completion of dosing and patients. Fishwan, I will discuss these pivotal milestones and their significance in greater depth later in the presentation.

Dr. Musunuri: We are actively recruiting patients in our OPQ400 Phase 3 Limelight clinical trial for the treatment of retinitis pigmentosa, or P.

Speaker Change: and just this week we announced a d approval an expanded access program eapp of the treatment of adult patients aged eighteen and order with rp with archqfour hundred

Dr. Musunuri: This is the first ever gene therapy candidate to treat patients with RP, regardless of mutation, approved for EAP.

Dr. Musunuri: we also progressed into the archq fourten pase to our moa clinical trial or the treatment of geographic atroacy and advanced stage of tryry agated macularision ration

Shankar Musunuri: Following completion of dosing in patients in phase 1, I will discuss these pivotal milestones in greater depth later in the presentation. Additionally, we are about to conclude phase 1 of the ACU-4-10ST, phase 1-2 Guardian clinical trial for the treatment of stroke or disease. ACU-400 is making remarkable strides in clinical development, and we are actually those same patients in the phase 3 Limelight clinical trial. As announced earlier, ACU-400 has received key regulatory approvals, including expanded orphan drug designations for RP from the FDA and the European Medicines Agency, as well as region-reduced medicine-advanced therapy or matte designation from the FDA.

Shankar Musunuri: following completion of dosing and patients. I will discuss these pivotal milestones in greater depth later in the presentation, and we are actively dosing patients in the Phase 3 Limelight clinical trial from the European Medicines Agency. We are very encouraged that more than 60% of the intent-to-treat patients from the Phase 1-2 clinical trial, including patients with Rho mutations, meet the responder criteria established for Phase 3. The Phase 3 mobility test responder rate for the only FDA-approved product to treat one mutation in RP was 52%. The OPIEU 400 Phase 3 study includes pediatric patients eight years of age or older and adults with early, intermediate, to advanced stages of RP. The study has a sample size of 150 participants.

Dr. Musunuri: following completion of dosing and patients.

Dr. Musunuri: in pas one

Dr. Musunuri: i will discuss this pital milestones and greater depth laterer in the presentation

Shankar Musunuri: Additionally, we're about to conclude Phase 1 of the RQ-410ST Phase 1-2 Guardian Clinical Trial for the treatment of Stargardt. RQ400 is making remarkable strides in clinical development, and we are actively dosing patients in the Phase 3 Limelight clinical trial. As announced earlier, RQ400 has received key regulatory approvals, including expanded orphan drug designations for RP from the FDA and the European Medicines Agency, as well as regional redo-medicine-advanced therapy or math designations from the FDA. With face-free dosing, RQ400 remains on track to meet the 2026 approval targets for a biological licensing application, BLA, from the FDA and for a market authorization application, from the European Medicines Agency.

Dr. Musunuri: Additionally, we are about to conclude Phase 1 of the RQ-410ST, Phase 1-2 Guardian Clinical Trial for the treatment of Stargardt disease.

Dr. Musunuri: RQ400 is making remarkable strides in clinical development, and we are actively dosing patients in the Phase III Limelight clinical trial.

Dr. Musunuri: As announced earlier, Parkview Fort 100

Dr. Musunuri: as receive key regulatory approvals including extanded or phantrop designations for rp

Dr. Musunuri: from the FDA and the European Medicines Agency, as well as Regenerative Medicine Advanced Therapy, or MAT, designation from the FDA.

Shankar Musunuri: With phase 3 dosing, ACU-400 remains on track to meet the 2026 approval targets for a Biological Licensing Application (BLA) from the FDA and for a Market Authorization Application (MAA) from the European Medicines Agency. We are very encouraged that more than 60 percent of the intent to treat patients from the phase 1-2 clinical trial, including patients with row mutation, meet the responder criteria established for phase 3. The phase 3 mobility tests responder rate for the only FDA approved product, a treat 1 mutation in RP, was 52 percent. The phase 3 study is powered where the 95 percent is using a 50 percent responder rate.

Speaker Change: it's fachas dosing occq four hundred remains on track to meet the two thousand and twenty six approval targets by a biological licensing application bla from the fda and for a market authorization application ma

Shankar Musunuri: We are very encouraged that more than 60% of the intent-to-treat patients from the Phase 1-2 clinical trial, including patients with Rho mutations, meet the responder criteria established for Phase 3. The Phase 3 mobility test responder rate for the only FDA-approved product to treat one mutation in RP was 52%. The phase 3 study has power greater than 95%, assuming a 50% responder rate. The OCU-400 phase 3 study includes pediatric patients eight years of age or older and adults with early, intermediate, to advanced stages of RP. The study has a sample size of 150 participants.

Dr. Musunuri: from the european mediticizens agency

Dr. Musunuri: we are very anchorate at more than sixty percent of the intend to treat patients from the pface one to clinical trial includy patients with romutation meet the respond criteria established for phas three

Speaker Change: the phas three mobility test is ponder rate of the only if we approved product triatved one mutation in rp was fifty two percent the phase e study is power thirty than ninety- five percent is suming fifty percent responder rate

Shankar Musunuri: The ACU-400 Phase 3 study includes pediatric patients, eight years of age or older, and adults with early intermediate to advanced stages of RP. The study has a sample size of 150 participants; one arm has 75 participants with the row gene mutations, and the other arm has 75 participants with mutations in any of several other genes. Ramakanth, Swayampakula Ramakanth, Arun Upadhyay, Michael Breininger, Michael Breininger, and focus on the proportion of responders and treated groups who achieve an improvement of at least two last levels from baseline.

Speaker Change: the occup four hundred fifth study includes pediatric patients eighty years of age or order and adults with early intermediate to advan stages of rp

Shankar Musunuri: One arm has 75 participants, the control group, and the other arm has 75 participants with mutations in any of several other genes. Randomized 2-to-1, A mobility test with luminance-dependent navigation assessment, LDNA, is the primary endpoint of the study. In this assessment, a participant navigates an obstacle course that constitutes a more sensitive and specific measurement of visual function than the mobility measurement used in previous phase 3 clinical trials. The Phase 3 Limelight Study will focus on the proportion of responders in treated and untreated groups who achieve an improvement of at least two lux levels from baseline.

Speaker Change: The study has a sample size of 150 participants.

Shankar Musunuri: One arm has 75 participants with the row gene mutations, and the other arm has 75 participants with the mutations in any of several other genes, randomized 2 to 1.

Speaker Change: a mobility says luminants

Shankar Musunuri: Dependent Navigation Assessment, LDNA, is the primary endpoint for the study.

Speaker Change: In this assessment, a participant navigates an obstacle course that constitutes a more sensitive and specific measurement of visual function than the mobility measurement used in previous phase 3 clinical trials.

Shankar Musunuri: The Phase 3 Limelight Study will focus on the proportion of responders in treated and untreated groups who achieve an improvement of at least 2 lux levels from baseline, to discuss the unmet need and underserved market for RT patients. Since the only other treatment currently on the market addresses mutations in one gene associated with RP, patients may be eligible to receive treatment prior to approval of the BLA. We look forward to working with clinicians, patients, and the RP community to provide access to RP-400 for eligible patients through our EAP.

Shankar Musunuri: the facaststre linelight study will focus on the proportion of responders in treated and untreated groups who achieve an improvement of at least two-l levels from baseline

Shankar Musunuri: Let me take a moment to discuss the unmet need and unreserved market for our patients. There are approximately 300,000 patients in the virus and EU that are affected by the disease, which is caused by mutations in any of approximately 100 different genes. The only other treatment currently on the market addresses mutations in one gene associated with Rp. Rp400 is a potential to treat multiple gene mutations because of its gene-agnostic mechanism of action, and in this way it will fulfill a significant unmet medical need.

Shankar Musunuri: Let me take a moment to discuss the unmet need and underserved market for RT patients. There are approximately 300,000 patients in the U.S. and E.U. that are affected by the disease, which is caused by mutations in any of approximately 100 different genes.

Shankar Musunuri: Let me take a moment to discuss the unmet need and underserved market for RP patients.

Speaker Change: there were approximately three hundred thousand patients than the u s and eu that are affected by the disease which is caused by limmutations in any of approximately one hundred different gs

Shankar Musunuri: The only other treatment currently on the market addresses mutations in one gene associated with RP. Archive 400 has the potential to treat multiple gene mutations because of its gene agnostic mechanism of action, and in this way, it will fulfill a significant unmet medical need. We continue our extensive campaign to educate the ophthalmology community about the concept of modified gene therapy, and we recently presented supporting data at a variety of conferences, such as the annual meeting of the American Society of Retina Specialists, which convened in Stockholm, Sweden last month.

Shankar Musunuri: The only other treatment currently on the market addresses mutations in one gene associated with RP.

Shankar Musunuri: RQ400 has the potential to treat multiple gene mutations because of its gene agnostic mechanism of action, and in this way, it will fulfill a significant unmet medical need.

Shankar Musunuri: We continue our extensive campaign to educate the ophthalmology community about the concept of modified gene therapy, and we recently presented supporting data at a variety of conferences, such as the annual meeting of the American Society of Redness Specialists, which convened in Stockholm, Sweden, last month. At the conference, Dr. Benjamin Pukal, who's housed at the Director of Clinical Research at Associated Regional Consultant and as Clinical Assistant Professor at the University of Arizona's College of Medicine and Phoenix, presented face onto data on Rp400. With the initiation of our EAP or Rp400, Rp patients with early intermediate to advanced Rp with at least minimal retinal preservation and who may benefit from the mechanism of action of Rp400 may be eligible to receive treatment prior to approval of the BLA.

Speaker Change: we continue our extensive campaign to educate the optimmology community about the concept of modifiesing in turkey

Speaker Change: and we recently presented supporting data at a variety of conferences such as annual meeting of the american socisaety of written specialists which conveed in starckown sweden pllast month

Shankar Musunuri: At the conference, Dr. Benjamin Bacall, who serves as the Director of Clinical Research at Associated Retina Consultants and as Clinical Assistant Professor at the University of Arizona's College of Medicine in Phoenix, presented Phase I-II data on RQ400. With the initiation of our EAP for RQ400, patients with early intermediate to advanced RP with at least minimal retinal preservation who may benefit from the mechanism of action of RQ400 may be eligible to receive treatment prior to approval of the BLA.

Speaker Change: At the conference, Dr. Benjamin Bacall, who serves as the Director of Clinical Research at Associated Retina Consultant and as Clinical Assistant Professor at the University of Arizona's College of Medicine in Phoenix, presented Phase I-II data on ARCHIE 400.

Shankar Musunuri: With the initiation of our EAP for RQ400, RP patients with early, intermediate to advanced RP with at least minimal retinal preservation, and who may benefit from the mechanism of action of RQ400.

Shankar Musunuri: may be eligible to receive treatment prior to approval of the BLA.

Shankar Musunuri: The decision by the FDA to endorse the use of Rp400 in any patients with Rp reflects the agency's position on the safety, tolerability, and benefit profile of Rp400 for any mutations relative to any risk of treatment. The approval of an expanded access program for Rp400 further supports the gene-agnostic mechanism of action for this novel modifier gene therapy. We look forward to working with clinicians, patients, and the Rp community to provide access to Rp400 for eligible patients through our EAP.

Shankar Musunuri: The decision by the FDA to endorse the use of OPQ-400 in any patient with RP reflects the agency's position on the safety, tolerability, and benefit profile of OPQ-400 for any mutations relative to any risk of recurrence. The approval of an expanded access program for OPTI 400 further supports the gene agnostic Mechanism of action for this novel modifier gene therapy. We look forward to working with clinicians, patients, and the RP community to provide access to RP-400 for eligible patients through our EAP.

Speaker Change: the decision by fda to endorse the use of ourq four hundred in any patients with rp refs the agency's position on the safety tolerability and benefit provides a off occq four hundred for any immutations relative to any risk of treatment

Speaker Change: the approval of an expanded access program for occe four hundred further supports the gene agnostic mechanisof action for this novel modifier gin therapkey

Shankar Musunuri: We look forward to working with clinicians, patients, and the RP community to provide access to RP-400 for eligible patients through our EAP.

Shankar Musunuri: Now let's move on to our developments in Rp400 and Rp400, which aim to treat geographic atrophy secondary to DAMD and start our disease respectively. These modifier gene therapies leverage a nuclear receptor gene called RORA, which stands for RORA related Awesome Receptor A as a potential one-time therapy for life with a single sub-brick milk injection. Q410, physically designed to address multiple pathways implicated in the pathogenesis of D-A-M-D, offers a distinct advantage for current treatment options that target only one pathway, a complement system, and require frequent interrogatory injection, about 60-12 doses per year, accompanied by various safety concerns that has roughly 12% of patients developed by D-A-M-D.

Shankar Musunuri: Now, let's move on to our developments in RQ410 and RQ410-SD, which aim to treat geographic atrophy secondary to DAMD and start our disease prospectively. These modifier gene therapies leverage a nuclear receptor gene called RORA, which stands for RAR-related Orphan Receptor A, as a potential one-time therapy for life with a single subretinal injection. RQ410, specifically designed to address multiple pathways implicated and the pathogenesis of D-AMD, offers a distinct advantage for current treatment options that target only one pathway, the complement system, and require frequent intrauterine injections, about 6 to 12 doses per year.

Speaker Change: now let's move on to our developments in occq fourten and ourcqptennesity which aim to treat geographic atrophy secondary to dmd and start our disease spectity

Shankar Musunuri: These modifier gene therapies leverage a nuclear receptor gene called RORA, which stands for RAR-related Orphan Receptor A, as a potential one-time therapy for life with a single subretinal injection.

Speaker Change: RQ-410, specifically designed to address multiple pathways implicated in the

Speaker Change: and the pathogenesis of D-AMD offers a distinct advantage for current treatment options that target only one pathway, the complement system, and require frequent intrauterine injection about 6 to 12 doses per year.

Shankar Musunuri: Accompanying various safety concerns, such as roughly 12% of patients developed with AMD, thereby addressing the underlying causes of the disease with a single original injection. Our approach with RK410 is to provide a comprehensive and durable solution with a potential one-time treatment. In July, we announced the completion of dosing in the third cohort of the RQ410 Phase I-II Armada clinical trial for the treatment of geographic atrophy. The Phase 2 dose expansion, SSR-Blinded clinical trial has been initiated and will assess the safety and efficacy of RQ410 in a larger group of patients who will be randomized into one of three groups and the dose escalation portion of the study. Targor affects approximately 100,000 people in the U.S. and Europe, and there are no approved therapies available.

Shankar Musunuri: Although accompanied by various safety concerns, such as roughly 12% of patients developing AMD, RQ410 has the potential to regulate all four pathways related to disease progression, lipid metabolism, inflammation, oxidative stress, and the complement system, thereby addressing the underlying causes of the disease with a single subretinal injection. An Armada Clinical Trial Update, providing further insights into the safety and efficacy of RQ410, is anticipated later this year Our approach with RK410 is to provide a comprehensive and durable solution with a potential one-time treatment.

Speaker Change: accompany by various ety concerns that has roughly twelve percent off patients developed pretty le

Shankar Musunuri: Ocugen Q410 has a potential to regulate all four pathways related to disease progression, lipid metabolism, inflammation, oxidative stress, and the complement system, thereby addressing the underlying causes of the disease with a single, aboriginal injection. An Armada clinical trial update, providing further insights into the safety and efficacy of Ocugen Q410, is anticipated later this year. Our approach with Ocugen Q410 is to provide a comprehensive and durable solution with a potential one-time treatment. There are 2-3 million geographic atrophy patients among the 19 million people affected by D-A-M-D in the U.S. and Europe, demonstrating a considerable market opportunity.

Shankar Musunuri: Opium-410 has the potential to regulate all four pathways related to disease progression, lipid metabolism, inflammation, oxidative stress, and the complement system, thereby addressing the underlying causes of the disease with a single aboriginal injection.

Shankar Musunuri: and our moder clinical trial update providing further insights into the safety and efficcuracy of ocq four ten is antisipated later this year

Shankar Musunuri: Our approach with RK410 is to provide a comprehensive and durable solution with a potential one-time treatment.

Shankar Musunuri: There are 2 to 3 million geographic atrophy patients among the 19 million people affected by DAMD in the U.S. and Europe, demonstrating a considerable market opportunity. In July, we announced the completion of dosing in the third cohort of the RQ410 Phase I-II Armada clinical trial for the treatment of geographic atrophy. Today, nine patients with geographic atrophy have been treated with a low, medium, and high dose. The Phase 2 dose expansion, SSR-blinded clinical trial has been initiated and will assess the safety and efficacy of RQ410 in a larger group of patients who will be randomized into one of three groups, a medium-dose treatment group, a high-dose treatment group, or a control group.

Shankar Musunuri: there are trudtolythree million geographic atrophy patients among the nineteen million people affected by d aemd in the u s and europe demonstrating a considerable market opportunity

Shankar Musunuri: In July, we announced the completion of those in the third cohort of the Ocugen Q410 Phase 1-2 Armada clinical trial for the treatment of geographic atrophy. Today, 9 patients, geographic atrophy, have been treated with low, medium, and high doses. The Phase 2 dose expansion, SSR blinded clinical trial, has been initiated and will assess the safety and efficacy of Ocugen in a larger group of patients who will be randomized into one of three groups: a medium dose treatment group, a high dose treatment group, or a control group. Participants must be aged 50 or older, be able to identify 24 letters or more on the BCVA, which is like those charts you read at the optometrist office, and I have a total geographic atrophy area between 2.5 and 20.5 square millimeters.

Shankar Musunuri: in july we announced the completion of dosing in the third cohort of the ocq fourten phase one to our moa clentical trial for the treatment of geographic entrophy

Shankar Musunuri: today nine patients the geographic atrophy have been treated with the low medium hdoses

Shankar Musunuri: The Phase 2 dose expansion, SSR blinded clinical trial has been initiated and will assess the safety and efficacy of RQ410 in a larger group of patients who will be randomized into one of three groups.

Shankar Musunuri: a medium-dose treatment group, a high-dose treatment group, or a control group.

Shankar Musunuri: Participants must be aged 50 or older, be able to identify 24 letters or more on a BCVA, which is like those charts you read at the optometrist's office, and I have a total geographic atrophy area between 2.5 and 20.5 square millimeters. Turning now to RQ-14SD, which has received an orphan drug designation from the FDA for the treatment of ABCA-4-associated retinopathies, including Stargard The Phase I-II Guardian clinical trial for the treatment of Stargardt disease is actively enrolling patients in the high-dose cohort and the dose escalation portion of the study.

Speaker Change: particper must be aged fifty or older be able to identify twenty-four letters or more

Speaker Change: on a bcva which is like those charts youe read at the oto mattric office and i have a total geographic rophy area between two point five and twenty point five square n measures

Shankar Musunuri: Turning now to Ocugen SD, which has received an all-front drug designation from the FDA for the treatment of ABCA-4 associated netna fatis, including starcard disease. The Phase 1-2 Guardian clinical trial for the treatment of Starcard disease is actually enrolling patients in the high dose cohort and the dose escalation portion of the study. Starcard FX approximately 100,000 people in the US and Europe, and there is no approved therapies available.

Shankar Musunuri: Turning now to OCU410SD, which has received an orphan drug designation from the FDA for the treatment of ABCA4-associated retinopathies, including Stargardt disease. The Phase I to Guardian clinical trial for the treatment of Stargardt disease is actively enrolling patients in the high-dose cohort.

Shankar Musunuri: Targor affects approximately 100,000 people in the U.S. and Europe, and there are no approved therapies available. These efforts represent our commitment to advancing treatments for blindness, focusing on innovative gene therapy solutions that aim to provide lasting benefits to patients. We look forward to sharing further updates as we continue to advance these promising therapies for clinical development. With that, I will now turn the call over to Corporate Comptroller Michael Breininger to provide an update on our financial results for the second quarter ended June 30, 2024. Michael? Thank you.

Speaker Change: and the dose escalation portion of the thir charger efpexs approximatelyone hundred thousand people and the u s and europe and there is no approved therapy available

Shankar Musunuri: These efforts represent our commitment to advancing treatments for blindness, focusing on innovative gene therapy solutions that aim to provide lasting benefits to patients.

Shankar Musunuri: We look forward to sharing further updates as we continue to advance these promising therapies to clinical development.

Shankar Musunuri: We look forward to sharing further updates as we continue to advance these promising therapies for clinical development.

Michael Breininger: With that, I will now turn the call over to Corporate Controller Michael Bringer to provide an update on our financial results for the second quarter in the June 30, 2024. Thank you, Shankar. The company's cash, cash equivalent center should the cash told $16 million as of June 30, 2024, compared to $39.5 million as of December 31, 2023. The company had $257.4 million shares of common stock outstanding as of June 30, 2024. Total operating expenses for three months ended June 30, 2024 were $16.6 million and included research and development expenses of $8.9 million and general and administrative expenses of $7.7 million.

Shankar Musunuri: With that, I will now turn the call over to Corporate Comptroller Michael Breininger to provide an update on our financial results for the second quarter ended June 30, 2024.

Michael Breininger: Thank you, Shankar. The company's cash, cash equivalents, and restricted cash totaled $16 million as of June 30, 2024, compared to $39.5 million as of December 31, 2023. The company had 257.4 million shares of common stock outstanding as of June 30, 2024. Toll operating expenses for the three months ended June 30, 2024 were $16.6 million and included research and development expenses of $8.9 million and general and administrative expenses of $7.7 million. This compares to total operating expenses for the three months ended June 30, 2023 of $24 million, which included research and development expenses of $14.5 million and general and administrative expenses of $9.5 million.

Michael Breininger: With that, I will now turn the call over to Corporate Comptroller Michael Breininger to provide an update on our financial results for the second quarter ended June 30, 2024. Michael.

Michael Breininger: Thank you, Shankar. The company's cash, cash equivalents, and restricted cash totaled $16 million as of June 30, 2024, compared to $39.5 million as of December 31, 2023. This compares to total operating expenses for the three months ended June 30, 2023 of $24 million, which included research and development expenses of $14.5 million and general and administrative expenses of $9.5 million. As stated earlier, we recently completed a successful fundraising effort with net proceeds of $32.6 million, extending our runway into the third quarter of 2025.

Michael Breininger: Thank you, Shankar. The company's cash equivalents and restricted cash totals $16 million as of June 30, 2024, compared to $39.5 million as of December 31, 2023.

Michael Breininger: The company had 257.4 million shares of common stock, outstanding as of June 30, 2024.

Michael Breininger: till operating expenses for three months ended june thirty two thousand and twenty four were sixteen point six million included research and development expenses of eight point nine million and general and administrative expenses of seven point seven million

Michael Breininger: This compares to total operating expenses for three months ended June 30, 2023, of $24 million that included research and development expenses of $14.5 million and general and administrative expenses of $9.5 million. As stated earlier, we recently completed a successful fundraising effort with net proceeds of $32.6 million, extending our runway into the third quarter of 2025. As always, we are constantly exploring strategic and shareholder-friendly opportunities to increase our working capital and continue to pursue strategic partnerships that will drive long-term strategy.

Michael Breininger: This compares to total operating expenses for the three months ended June 30, 2023, of $24 million that included research and development expenses of $14.5 million and general and administrative expenses of $9.5 million.

Michael Breininger: As stated earlier, we recently completed a successful fundraising effort with net proceeds of $32.6 million, extending our runway into the third quarter of 2025. As always, we are constantly exploring strategic and shareholder-friendly opportunities to increase our working capital and continue to pursue strategic partnerships that will drive long-term strategy. That concludes my update for the quarter. Tiffany, back to you. Thank you, Mike. We will now open the call for questions.

Michael Breininger: As stated earlier, we recently completed a successful fundraising effort with net proceeds of $32.6 million, extending our runway into the third quarter of 2025.

Michael Breininger: As always, we are constantly exploring strategic and shareholder-friendly opportunities to increase our working capital and continue to pursue strategic partnerships that will drive long-term strategy.

Michael Breininger: That concludes my update for the quarter.

Tiffany Hamilton: Tiffany, back to you. Thank you, Mike.

Michael Breininger: That concludes my update for the quarter. Tiffany, back to you.

Tiffany Hamilton: Thank you, Mike. We will now open the call for questions. Operator?

Operator: We will now open the call for questions. Operator? This time, I would like to remind everyone in order to ask a question. Press star, then the number one on your telephone keypad. We will pause for just a moment to compile a question and answer roster.

Michael Breininger: thank you mike we will now open the call for questions operator

Operator: This time, I would like to remind everyone in order to ask a question. Press star then the number 1 on your telephone keypad. We will pause for just a moment to compile the question and answer list. Our first question comes from the line of Sean Lee.

Speaker Change: This time, I would like to remind everyone, in order to ask a question,

Speaker Change: Press star then the number 1 on your telephone keypad. We will pause for just a moment to compile the question and answer roster.

Sean Lee: Our first question comes from the line up, Chan Lee. Please go ahead. Hi. Good morning. This is Sean from HCWingright, standing in for RK. How are you? Good morning, Sean. Great. Thanks for taking my questions. My first one is on the ALQ400 Expanded Access Program. So I was wondering what is the AEP primarily targeted towards since I'm sure you are still actively recruiting a lot of patients into the phase three study. Do you want? Yes, thank you for the question. So the expanded access program is targeting the populations that do not meet the inclusion exclusion criteria of our phase three, or they would have to have an option meeting at least a bit more flexibility based on what we did because that is mandated by FDA regulatory process.

Speaker Change: V.C. College of Nursing Coordination and Enrollment Vanthu Bharadwaj S.M. Mohit G. Sinha M.D. S.M. Ravipa J.M. Srinivasan S.M. V.C. College of Nursing Coordination and Enrollment Vanthu Bharadwaj S.M. Mohit G. Sinha M.D. S.M. Ravipa J.M. Srinivasan S.M. Ravipa J.M. Srinivasan

Speaker Change: Our first question comes from the line up, Chan Lee.

Sean Lee: Hi, good morning. This is Sean from HC Wainwright sending in for RK. How are you? Good morning, Sean. Okay. My first question is about the OCU-400 Expanded Access Program. So I was wondering, what is the EAP primarily targeted towards since I'm sure you are still actively recruiting a lot of patients into the Phase III study?

Speaker Change: Please go ahead.

Michael Breininger: Hi, good morning, this is Sean from HCC Wainwright, sending in for RK, how are you?

Shankar Musunuri: Yes, thank you for the question. So the Expanded Access Program is targeting the population that does not meet the inclusion and exclusion criteria of our Phase 3, or they would have to have an option with a little bit more flexibility based on what we have not offered in our Phase 3 because that is mandated by the FDA regulatory process. So, in this trial, our inclusion criteria would be 18 years of age, anyone that has a clear certified genetic diagnosis of RP and those who have photos, and also discretionary by the treating physician. This is a decision that will be individually taken by the treating physician and the patient.

Sean: Good morning, Sean.

Speaker Change: Great. Thanks for taking my questions. My first one is on the RQ400 Extended Access Program.

Speaker Change: so i was wondering what is the ap primarily targeted towards since i'm sure you are so actively recruiting a lot of patients into the phas three study

Michael Breininger: umma

Speaker Change: yes thank you for the question so the expanded access program is targeting the population that do not meet the inclusion exclusion criteria for phase three

Speaker Change: Or they would have to have an option meeting a little bit more flexibility based on what we have not offered in our Phase 3 because that is mandated by FDA regulatory process.

Shankar Musunuri: So, in this trial, our inclusion criteria would be 18 years of age. Anyone that has a clear certified genetic diagnosis of RP and those who, and also discretionary by the treating position. This is the decision that individually will be taken by the treating position and the patient. I see. Thanks for that.

Speaker Change: so in this trial our ininclusion criteria would be aging years of h anyone that has a clear certified genetic diagnosis of r p and those who have pto receptors

Speaker Change: and also discretionary by the treating physician. This is the decision that individually will be taken by the treating physician and the patient.

Shankar Musunuri: I see. Thanks for that. On to the OCU-400 phase 3, I was wondering what the expected difference is between the treatment and untreated arms and how is the study powered to detect it?

Shankar Musunuri: On to the Oculus 400 Phase series. I was wondering, have you disclosed what six acted the difference between the treatment and untreated arms, and how is the study powered to detect it? Yeah, so the treated and untreated untreated is not truly untreated because it's an SSR blinded study. It's a sub-retinal surgery. So that's the way you actually blinded the study. So the second, so the study is powered to one ratio. That means out of 150 patients, 50 patients are going to be the untreated group. And the study is powered at greater than 95%, assuming there is a 50% response rate.

Speaker Change: I see. Thanks for that. On to the OCU-400 Phase 3. I was wondering, have you disclosed what's the expected difference between the treatment and untreated arms, and how is the study powered to detect it?

Shankar Musunuri: Yeah, so the treated and untreated. The untreated is not truly untreated because it's an SSR blinded study. It's subretinal surgery, so that's the way you actually blind a study. So, the second, so the study is powered by a two to one ratio. That means that out of 150 patients, 50 patients are going to be in the untreated group. And the study is powered at greater than 95%, assuming there is a 50% response rate. So responders are defined as those who can reach either two levels or higher on the mobility test, which is our mobility test is proprietary LDNA.

Sean Lee: Okay, understood. So 95% to detect a 50% difference. Got it.

Speaker Change: Yeah, so the treated and untreated. Untreated is not truly untreated because it's an SSR blinded study. It's a subretinal surgery so that's the that's the way you actually blind a study.

Speaker Change: So the study is powered at 2 to 1 ratio, that means...

Speaker Change: Out of 150 patients, 50 patients are going to be in the untreated group.

Speaker Change: And the study is powered at greater than 95%, assuming there is a 50% response rate. So responders are defined as who can reach either two levels or higher on the mobility test, which is, or mobility test is proprietary LDNA.

Shankar Musunuri: So responders are defined as who can reach either two levels or higher on the mobility test. Which is our mobility test is proprietary LDNA. Okay, understood. So 95% to detect the 50% difference. Got it.

Shankar Musunuri: And then finally, for the OCU 410 study update expected later this year, could you elaborate a little more on what we can expect at the update? What will you provide? What kind of data will you provide?

Speaker Change: okcan understood so ninety-five percent to detect the fifty percent difference got and then finally for the

Shankar Musunuri: And then finally, for the, for 10, for the Oculus 410 study update, excited later this year, could you elaborate a little more on what can we expect at the update? What will you provide? What kind of data will you provide? So, yes, for the 410 geographic entropy secondary to dry age-related microdegeneration study, we are hoping to provide preliminary safety and efficacy updates later this year. Okay, so we think that this is both safety and some efficacy results. Yes. Great. Thanks. That's all the questions I have. Thanks again for taking my questions. Thank you.

Speaker Change: For the OCU 410 study update expected later this year, could you elaborate a little more on what can we expect at the update? What will you provide? What kind of data will you provide?

Shankar Musunuri: So yes, for the 410 Geographic Atrophy Secondary to Dry Age-Related Microrotation Study, we are hoping to provide preliminary safety and efficacy updates later this year. Okay, so we're getting back to the...

Operator: So yes, for the 410 Geographic Atrophy Secondary to Dry Age-Related Microdegeneration Study, we are hoping to provide preliminary safety and efficacy updates later this year. Okay, so we can expect to...

Operator: So yes, for the 410 Geographic Atrophy Secondary to Dry Age-Related Microdegeneration Study, we are hoping to provide preliminary safety and efficacy updates later this year.

Sean Lee: So we can expect both safety and some efficacy results then. Yes. Great. Thanks. That's all the questions I have. Thanks again for taking my questions.

Speaker Change: so in weking sectors both safety and some adficacy results that great thanks that's all the questions i have thanks again taking my questions

Robert Leboyer: For next questions comes from the line of Robert Leboyer with Noble Capital Market. Please go ahead. Good morning. My question has to do with OCU 400, and you'd mention that you're on track for the 2026 BLA. So, I was wondering if you could give any details on upcoming milestones or data presentations for the trial.

Operator: Our next question comes from the line of Robert LeBoyer with Noble Capital Markets. Please go ahead.

Robert Leboyer: Our next question comes from the line of Robert LeBoyer with Noble Capital Markets. Please go ahead.

Speaker Change: Our next question comes from the line of Robert LeBoyer with Noble Capital Markets. Please go ahead.

Robert Leboyer: Good morning. My question has to do with OCU 400. And you mentioned that you're on track for the 2026 BLA. So I was wondering if you could give any details on upcoming milestones or data presentations for the trial.

Robert Leboyer: Good morning. My question has to do with OCU 400, and you had mentioned that you're on track for the 2026 BLA.

Robert Leboyer: So, I was wondering if you could give any details on upcoming milestones or data presentations for the trial? Thank you. Thank you. Thank you.

Shankar Musunuri: Robert, good morning, Robert. Since it's an SSL blinded study, updates will be provided on the recruitment rates, and how we are meeting the BLA timeline. Since we do have RMAT designation, as well as orphan designations in the US and EU, that will allow us to do a rolling submission of our BLA and MAA. So that's the process we potentially are going to take starting late next year. And when the clinical recruitment is done early next year, it will take one year for us to complete the last patient, which is the duration of the trial.

Shankar Musunuri: Robert, good morning, Robert. Since it's an SSL blinded study, updates will be providing on the recruitment rates, how we are meeting the BLA timeline. Since we do have our map designation as well as orphaned designations in the US and the EU, that will allow us to do a rolling submission of our BLA and MAA. So, that's the process potentially we're going to take starting from late next year, and then the clinical recruitment is done early next year. That will take one year for us to complete the last patient, which is a duration of the trial.

Speaker Change: Good morning, Robert. Since it's an SSL-blinded study, updates will be providing on the recruitment rates, how we are meeting the BLA timeline.

Shankar Musunuri: Since we do have RMAT designation, as well as orphan designations in the US and EU, that will allow us to do a rolling submission of our BLA and MAA. So that's the process we potentially are going to take starting late next year. And when the clinical recruitment is done early next year, it will take one year for us to complete the last patient, which is the duration of the trial. And when the data comes out, we'll close the clinical sections. And then that will trigger an accelerated path of six months in 2020.

Shankar Musunuri: Since we do have RMAT designation.

Shankar Musunuri: as well as orphtdesignations in u us u

Speaker Change: let will allow us to do a ruling submission of er blla and the may so that's the process potentialally we' going to take starting from late next year

Shankar Musunuri: And then when the clinical recruitment is done early next year, that will take one year for us to complete the last patient, which is the duration of the trial. And when the data comes out, we'll close the clinical sections. And then that will trigger the accelerated path of six months.

Shankar Musunuri: And when the data comes out, we'll close the clinical sections, and then that will trigger the accelerated path of six months. in 2026. So that allow us to potentially get approval in both the US and EU late 2026.

Shankar Musunuri: And when the data comes out, we'll close the clinical sections, and then that will trigger the accelerated path of six months in 2020. So that will allow us to potentially get approvals in both the U.S. and EU late in 20.

Shankar Musunuri: intwo thousand and twenty six

Shankar Musunuri: So that will allow us to potentially get approvals in both US and EU late 2026.

Robert Leboyer: Okay, great. Thank you very much. Thank you, Robert.

Robert Leboyer: Okay, great. Thank you very much. Thank you.

Speaker Change: Okay, great. Thank you very much.

Daniil Gataulin: Our next question comes from the line of Daniel Gataulin with Chaden Capital Market. Please go ahead.

Operator: Our next question comes from the line of Daniil Gataulin with Shazan Capital Markets. Please go ahead.

Daniil Gataulin: Our next question comes from the line of Daniil Gataulin with Shazan Capital Markets. Please go ahead.

Operator: Our next question comes from the line of Daniil Gataulin with Shazan Capital Markets. Please go ahead.

Shankar Musunuri: Hi, this is Janani on behalf of Daniil. So my first question is on Q200. Can you tell us where you are in the process for getting the clinical hold lifted for Q200, and once the hold is lifted, will you be launching the trial right away, or are you focusing on the gene therapy programs at this point? Thank you. We are still working with FDA to get the submit the information that requested and try to get the clinical hold lifted, and we designed a very simple phase one study. After a BS decision lifting the clinical hold, we will define the path forward for the program.

Janani Sundararajan: Hi, this is Janani on behalf of Daniil. So my first question is on OCU-200. Can you tell us where you are in the process of getting the clinical hold lifted for OCU-200? And once the hold is lifted, will you be launching the trial right away, or are you focusing on the gene therapy programs at this point? Thank you.

Operator: Good morning and welcome to Ocugen's second quarter, 2024 Financial Result, and Business Update. Please note that this call is being recorded at this time. All participant lines are in a listen-only mode.

Speaker Change: hithis is journey on behalf of denu

Daniil Gataulin: So my first question is on OCU-200. Can you tell us where you are in the process for getting the clinical hold lifted for OCU-200? And once the hold is lifted, will you be launching the trial right away, or are you focusing on the gene therapy programs at this point? Thank you.

Tiffany Hamilton: Following the speaker commentary, there will be a question and answer session. I will now turn the call over to Tiffany Hamilton, Ocugen's head of corporate communication. You may begin. Thank you, operator, and good morning, everyone.

Shankar Musunuri: We're still working with FDA to get the information they requested and try to get the clinical hold lifted. And we designed a very simple phase one study. And after FDA's decision on lifting the clinical hold, we will define the path.

Speaker Change: We're still working with FDA to submit the information they requested and try to get the clinical hold lifted.

Speaker Change: and we designed a very simple phase on study and after a ds station lifting the clinical ho we will define the path forward for theprogram

Tiffany Hamilton: Joining me on today's call in 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.

Shankar Musunuri: OK, and I mean, again, I just want to reiterate our focus has been primarily gene therapy, but the 200 is a good program. As soon as they deal with the clinical hold, we'll provide direction on that.

Shankar Musunuri: I just want to reiterate how focus has been primarily gene therapy, but the 200 is a good program. As soon as they deal with the clinical hold, we will provide a direction on that program. Okay, thank you.

Tiffany Hamilton: Michael Breininger, our corporate controller, is also on the call to provide a financial update for the quarter-ended June 30, 2024.

Speaker Change: okay and i mean again as i just want to reitrate her focus has been primarily leg therapies but to two hundred a good program as soon as a deaily the clinical hole will provide a direction on the program

Tiffany Hamilton: 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 second quarter of 2024. We encourage listeners to review the press release, which is available on our website at Ocugen.com.

Janani Sundararajan: Okay, thank you. So, are there meaningful differences in achieving responder criteria with the LDNA compared to the mobility assessments used in previous phase 3 trials? Yes. As we stated in

Shankar Musunuri: So I have another question, and our Q400. So are there meaningful differences in achieving responder criteria with the LDNA compared to the mobility assessments used in previous phase three trials? Yes, as we stated and showed today, intent to treat population data we analyzed from the phase one to that means patients who will qualify for phase three based on our criteria, and we clearly showed 62% response rate based on people who can reach two levels or more, and in the approved product they are 52% response rate. I think one of the questions earlier we addressed, we powered the study at 50% response rate; that means we actually powered it lower than what we achieved in phase two.

Daniil Gataulin: okay thank you so i have another question and occupa hundred so are there meaningful differences in achieving respond to criteria with the l d na compared to the mobility assessments used in previous space three trials

Tiffany Hamilton: This call is being recorded and a replay with the accompanying slide presentations will be available on the investor section of the Ocugen website for approximately 45 days. This presentation contains forward-looking statements within the meeting of the Private Security's litigation reform act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as, predicts, believes, potential proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should, or other words, that can say uncertainty or future events or outcomes to identify these forward-looking statements.

Shankar Musunuri: As we stated and showed today, in terms of population data, we analyzed from phase one to that means patients who will qualify for phase three based on our criteria. And we clearly showed a 62 percent response rate based on people who can reach two levels or more. And in the approved product, there is a 52 percent response rate. And I think one of the questions earlier we addressed, we powered the study at a 50 percent response rate. That means we actually powered it lower than what we achieved in phase.

Operator: As we stated in,

Operator: yes

Speaker Change: As we stated and showed today, intent-to-treat population data we analyzed.

Speaker Change: from the fish one two that means patients who will qualify for phas three based on our criteria and we clearly showed sixty two percent response rate based on people who can reach

Speaker Change: two levels or more, and in the approved product, they are 52% response rate.

Speaker Change: and i think one of the questions earlier we addressed we powered the study at fifty percent response rate that means we actually powered it lower than what we achieve can face to

Tiffany Hamilton: But statements include, what are not limited to, statements regarding our clinical development activity and related anticipated timelines, such statements are subject to numerous important risk factors and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filing with the Security and Exchange Commission, SEC, including the risk factors described in the section entitled risk factors, in the quarterly and annual reports that be filed with the SEC.

Shankar Musunuri: Thank you.

Speaker Change: Okay, thank you.

Operator: This concludes the question in answer portion.

Operator: This concludes the question and answer portion.

Operator: This concludes the question and answer portion. I will now turn the call back over to Chairman, CEO, and Co-Founder, Dr. Shankar Musunuri. Please go ahead.

Shankar Musunuri: I will now turn the call back over to Chairman, CEO and co-founder Dr. Shankar, moved in order. Please go ahead. Thank you, operator. Thank you, everyone, for joining us today. We appreciate your continued support as we move forward with our groundbreaking scientific and clinical initiatives. We look forward to the second half of 2024 as we continue to solidify Akigen's position as a biotechnology leader. Thank you.

Speaker Change: This concludes the question and answer portion. I will now turn the call back over to Chairman, CEO , and Co-Founder, Dr. Shankar Musunuri. Please go ahead.

Shankar Musunuri: Thank you, operator. Thank you, everyone, for joining us today. We appreciate your continued support as we move forward with our groundbreaking scientific and clinical initiatives. We look forward to the second half of 2024 as we continue to solidify Ocugen's position as a biotechnology leader.

Speaker Change: thank you operator thank you everyone for joining us today we appreciate your our continued support as we move forward with our groundbreaking scientific and clinical initiatives

Operator: We look forward to the second half of 2024 as we continue to solidify Ocugen's position as a biotechnology leader.

Tiffany Hamilton: And, in forward-looking statements that we make in this presentation speak only as of the date of this presentation, except it's required by law. We assume no obligation to update forward-looking statements contained in this presentation, whether as a result of new information, future events, or otherwise, after the date of this presentation.

Operator: We look forward to the second half of 2024 as we continue to solidify Ocugen's position as a biotechnology leader. Thank you.

Operator: Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect. Swayampakula Ramakanth,

Operator: Thank you. Ladies and gentlemen, that concludes today's call. Thank you all for joining us.

Operator: Ladies and gentlemen, that concludes today's call. Thank you all for joining us. You may now disconnect.

Operator: Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect.

Tiffany Hamilton: Finally, applicants quarterly reports on Form 10Q, covering the second quarter of 2024 has been filed.

Shankar Musunuri: I will now turn the call over to Dr. Mousenari. Thank you, Tiffany. And thank you all for joining us today.

Unnamed: Swayampakula Ramakanth

Shankar Musunuri: We're excited to discuss a substantial progress of our modified inter-p-platform across all three clinical programs. And to continue driving these programs, we recently completed a successful fundraising effort that net proceeds of $32.6 million, extending our runway into the third quarter of 2025. Our scientific advances and the strategic growth of company were further acknowledged by our inclusion in the Russell Index in June. This ranking demonstrates the value of a pipeline and supports Occidence Dedication to creating long-term shareholder value. Additionally, the recent offering was led by a premier mutual fund, along with participation from leading Life Sciences Investors, which further strengthens our shareholder base.

Shankar Musunuri: We are actually recruiting patients in our Q400-based-relimelite clinical trial for the treatment of Ignatis Pigmentosa RP. And just this week, we announced a DAF Provo for an expanded access program EAP for the treatment of adult patients aged 18 and older with RP with RQ400. This is the first-ever gene therapy candidate to treat patients with RP, regardless of mutation, approved for EAP.

Shankar Musunuri: We also progressed into the RQ410, face-to-armada clinical trial for the treatment of geographic atrophy, an advanced stage of triage-related macular degeneration. Following completion of dosing in patients and face-one, I will discuss these pivotal milestones in greater depth later in the presentation.

Shankar Musunuri: Additionally, we are about to conclude face-one of the RQ410ST, face-one to guardian clinical trial for the treatment of stroke R disease. RQ400 is making remarkable strides in clinical development, and we are actually those same patients in the face-to-relimelite clinical trial. As announced earlier, RQ400 has received key regulatory approvals, including expanded orphan drug designations for RP from the FDA and the designation from the FDA. With face-to-reducing, RQ400 remains on track to meet the 2026 approval targets for a biological licensing application, BLA from the FDA, and for a market authorization application, MAA from the European Medicines Agency.

Shankar Musunuri: We are very encouraged that more than 60% of the intent to treat patients from the face-one to clinical trial, including patients with room mutation, meet the responder criteria established for face-3. The face-3 mobility tests responder rate for the only FDA approved product, treat one mutation in RP was 52%. The face-3 study is powered by 95%, assuming 50% responder rate. The RQ400 face-3 study includes pediatric patients eight years of age or older, and adults with early intermediate to advanced stages of RP. The study has a sample size of 150 participants, one arm has 75 participants with a row gene mutations, and the other arm has 75 participants with mutations in any of several other genes.

Shankar Musunuri: Ramakula Ramakanth, Arun Upadhyay, David Birch, David Birch, David Birch, Archive 400 is a potential to treat multiple gene mutations because of its gene-agnostic mechanism of action, and in this way it will fulfill its significant unmet medical need.

Shankar Musunuri: At the conference, Dr. Benjamin Pukal, who sells at the Director of Clinical Research, its Associated Regional Consultant, and as clinical assistant professor at the University of Arizona College of Medicine and Phoenix, presented face-on-to-data on AQ400. With the initiation of our EAP for AQ400, RPE patients with early intermediate to advanced RPE with at least minimal retinal preservation and who may benefit from the mechanism of action of AQ400 may be eligible to receive treatment prior to approval of the BLA.

Shankar Musunuri: The decision by the FDA to endorse the use of AQ400 in any patients with RPE reflects the agency's position on the safety, tolerability, and benefit profile of AQ400 for any mutations relative to any risk of treatment. The approval of an expanded access program for AQ400 further supports the gene-agnostic mechanism of action for this novel modifier gene therapy. We look forward to working with clinicians, patients, and the RPE community to provide access to AQ400 for eligible patients through our EAP.

Shankar Musunuri: Now, let's move on to our developments in AQ410 and AQ410STs, which aim to treat geographic atrophy secondary to D-A-M-D and start-up disease respectively. These modifier gene therapies leverage a nuclear receptor gene called RORA, which stands for RAR-related alpha receptor A as a potential one-time therapy for life with a single sub-briqunal Q410, physically designed to address multiple pathways implicated in the pathogenesis of DAMD, offers a distinct advantage for current treatment options, the target only one pathway, the complement system, and requires frequent interrogatory injection, about 60-12 doses per year, accompanied by various safety concerns, which has roughly 12% of patients developed by DAMD.

Shankar Musunuri: Upv410 has a potential to regulate all four pathways related to disease progression, lipid metabolism, inflammation, oxidative stress, and the complement system, thereby addressing the underlying causes of the disease with a single aboriginal injection. An Armada clinical trial update, providing further insights into the safety and efficacy of Ocu410 is anticipated later this year. Our approach with Ocu410 is to provide a comprehensive and durable solution with a potential one-time treatment. There are 2-3 million geographic atrophy patients among the 19 million people affected by DAMD in the US and Europe, demonstrating a considerable market opportunity.

Shankar Musunuri: In July, we announced the completion of those in the 3rd cohort of the Ocu410, Phase 1-2 Armada clinical trial for the treatment of geographic atrophy. Today, 9 patients, geographic atrophy, have been treated with low, medium, and high doses. The Phase 2 dose expansion, SSR blinded clinical trial, has been initiated and will assess the safety and efficacy of Ocu410 in a larger group of patients who will be randomized into one of three groups, a medium dose treatment group, a high dose treatment group, or a control group.

Shankar Musunuri: Participants must be aged 50 or older, be able to identify 24 letters or more on the BCVA, which is like those charts you read at the Optometrist Office and I have a total geographic atrophy area between 2.5 and 20.5 square millimeters.

Shankar Musunuri: Turning now to Ocu410SD, which has received an all-front drug designation from the FDA for the treatment of ABCA4 Associated Netnafatis, including starcard disease. The Phase 1-2 guardian clinical trial for the treatment of starcard disease is actually enrolling patients in the high dose cohort and the dose escalation portion of the study. Starcard FX approximately 100,000 people in the US and Europe and there is no approved therapies available. These efforts represent our commitment to advancing treatments for blindness, focusing on innovative gene therapy solutions that aim to provide lasting benefits to patients.

Shankar Musunuri: We look forward to sharing further updates as we continue to advance these promising therapies to clinical development.

Michael Breininger: With that, I will now turn the call over to Corporate Controller Michael Breninger to provide an update on our financial results for the second quarter in June 30, 2024. Thank you, Shankar. The company's cash, cash equivalent center should the cash told $16 million as a June 30, 2024 compared to $39.5 million as of December 31, 2023. The company had $257.4 million shares of common stock outstanding as of June 30, 2024. Total operating expenses for three months ended June 30, 2024 were $16.6 million and included research and development expenses of $8.9 million and general fandom administrative expenses of $7.7 million. This compares to total operating expenses for three months ended June 30, 2023 up 24 million that included research and development expenses of $14.5 million and general administrative expenses of $9.5 million.

Michael Breininger: As stated earlier, we recently completed a successful fundraising effort with net proceeds of $32.6 million extending our runway into the third quarter of 2025. As always, we are constantly exploring strategic and shareholder friendly opportunities to increase our working capital and continue to pursue strategic partnerships that will drive long-term strategy.

Tiffany Hamilton: That concludes my update for the quarter Tiffany back to you.

Tiffany Hamilton: Thank you, Mike. We will now open the call for questions. Operator? This time, I would like to remind everyone in order to ask a question, press star, then the number one on your telephone keypad. We will pause for just a moment to compile a question and answer roster.

Sean Lee: Our first questions comes from the line up, Chan Lee. Please go ahead. Hi. Good morning. This is Sean from HCWingright, standing in for RK. How are you? Good morning, Sean. Great. Thanks for taking my questions. My first one is on the ALQ400 expanded access program. So I was wondering what is the AEP primarily targeted towards since I'm sure you are still actively recruiting a lot of patients into the phase three study.

Sean Lee: Duma? Yes, thank you for the question. So the expanded access program is targeting the population that do not meet the inclusion exclusion criteria of our phase three or they would have to have an option meeting at least a bit more flexibility based on what we have not offered in our phase three because that is mandated by FDA regulatory process. So in this trial, our inclusion criteria would be 18 years of age and anyone that has a clear certified genetic diagnosis of RP and those who have photos, and also discretionary by the treating position. This is the decision that individually will be taken by the treating position and the patient. I see. Thanks for that.

Sean Lee: On to the Oculus 400 phase 3. I was wondering, have you disclosed what six packet difference between the treatment and untreated arms and what how is the study powered to detect? Yeah, so the treated and untreated untreated is not truly untreated because it's SSR blinded study. It's a sub-retinal surgery. So that's the way you actually blind the study. So the second, so the study is powered to one ratio. That means out of 150 patients, 50 patients are going to be the untreated group.

Sean Lee: And the study is powered at greater than 95% assuming there is a 50% response rate. So responders are defined as who can reach either two levels or higher on the mobility test, which is our mobility test is proprietary LDNA. Okay, understood. So 95% to detect a 50% difference. Got it.

Sean Lee: And then finally, for the, for 10, for the Oculus 410 study update, excited later this year, could you elaborate a little more on what can we expect at the update? What will you provide? What kind of data will you provide? So, yes, for the 410 geographic entropy secondary to dry age related microdegeneration study, we are hoping to provide preliminary safety and efficacy updates later this year. Okay, so we're taking sectors both safety and some efficacy results. Yes, great. Thanks. That's all the questions I have. Thanks again for taking my questions. Thank you.

Robert Leboyer: For next questions comes from the line of Robert Leboyer with noble capital market, please go ahead. Good morning. My question has to do with OCU 400 and you'd mention that you're on track for the 2026 BLA. So I was wondering if there if you could give any details on upcoming milestones or data presentations for the trial. Robert, good morning, Robert. Since it's a SSR blinded study updates will be providing our on the recruitment rates, how we are meeting the BLA timeline.

Robert Leboyer: Since we do have our map designation, as well as orphaned designations in US and EU, that allow us to do a rolling submission offer BLA and MA. So that's the process potentially we're going to take starting from late next year and live in the clinical recruitment is done early next year that will take one year for us to complete the last patient, which is a duration of the trial. And when the data comes out, we'll close the clinical sections and then that will trigger the accelerated path of six months. N2026, so that Leila was to potentially get approval in both US and EU late 2026.

Robert Leboyer: Okay, great, thank you very much. Thank you Robert.

Janani Sundararajan: Our next questions comes from the line of Daniel Gataulin with Chaden Capital Market. Please go ahead. Hi, this is Janani on behalf of Daniel.

Janani Sundararajan: So my first question is on OQ 200. Can you tell us where you are in the process for getting the clinical hold lifted for OQ 200? And once the hold is lifted, will you be launching the trial right away or are you focusing on the gene therapy programs at this point? Thank you. We are still working with FDA to get the submit the information that requested and try to get the clinical hold lifted.

Janani Sundararajan: And we designed a very simple phase one study. And after a BS decision lifting the clinical hold, we will define the path forward for the program. Okay, I mean, again, I just want to reiterate how focused has been primarily gene therapy, but the 200 is a good program. As soon as it daily clinical hold will provide a direction on that program. Okay, thank you.

Janani Sundararajan: So I have another question in OQ 400. So are there meaningful differences in achieving responder criteria with the LDNA compared to the mobility assessments used in previous phase three trials? Yes, as we stated and showed today, um, intent to treat population data we analyzed from the phase one to that means patients who will qualify for phase three based on our criteria. And we clearly showed 62% response rate based on people who can reach two levels or more.

Janani Sundararajan: And in the approved product, they are 52% response rate. And I think one of the questions earlier we addressed, we powered the study at 50% response rate. That means we actually powered it lower than what we achieved in phase two. Thank you.

Operator: This concludes the question in answer portion.

Shankar Musunuri: I will now turn the call back over to chairman CEO and co-pounder, Dr. Shankar. Moving on. Please go ahead. Thank you operator. Thank you everyone for joining us today. We appreciate your continued support as we move forward with our groundbreaking scientific and clinical initiatives. We look forward to the second half of 2024 as we continue to solidify origins position as a biotechnology leader. Thank you.

Operator: Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect. Jennifer Kim, Robert LeBoyer, Li Chen, Arun Upadhyay, David Birch

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