Core Viewpoint - Ocugen is discussing data from the first half of patients who have completed one year since treatment in the OCU410 Phase II ArMaDa clinical trial for geographic atrophy [1] Group 1: Company Overview - The webcast features key executives including Dr. Shankar Musunuri (Chairman, CEO, and Co-Founder), Dr. Huma Qamar (Chief Medical Officer), and Dr. Arun Upadhyay (Chief Scientific Officer) [1] - Distinguished clinical trial investigators are also participating in the discussion, including Dr. Jay Chhablani, Dr. Arshad Khanani, and Dr. Lejla Vajzovic [1] Group 2: Clinical Trial Insights - The focus of the webcast is on the clinical trial data related to the OCU410 treatment for geographic atrophy, highlighting the significance of the one-year patient data [1]

Core Insights - Ocugen, Inc. has shared preliminary data from its Phase 2 clinical trial for OCU410, a gene therapy targeting geographic atrophy, showing promising results [1][3] Clinical Trial Results - The Phase 2 ArMaDa clinical trial indicated a 46% reduction in lesion growth compared to the control group after 12 months, with no serious adverse events reported [3] - The medium dose of OCU410 achieved a 54% reduction in lesion size, while the high dose showed a 36% reduction, highlighting the therapy's potential efficacy [3] Future Plans - The company plans to report full data from the Phase 2 trial later this quarter and initiate a Phase 3 trial in 2026 [4] - Ocugen is on track for a Biologics License Application (BLA) filing for OCU410 in 2028 [4] Other Programs - Ocugen announced the publication of Phase 1 GARDian1 trial results for OCU410ST, a novel modifier gene therapy for Stargardt disease, in the peer-reviewed journal Nature Eye [5] - The Phase 1 GARDian1 trial demonstrated robust efficacy and safety outcomes supporting the clinical development of OCU410ST [6] - In September 2025, Ocugen entered a licensing agreement with Kwangdong Pharmaceutical for the exclusive Korean rights to OCU400, its modifier gene therapy for retinitis pigmentosa [6] Stock Performance - Ocugen shares were down 9.08% at $1.71 at the time of publication, despite the broader market gains [2][9] - The stock is currently trading 15.2% above its 20-day simple moving average (SMA) and 28% above its 50-day SMA, indicating short-term strength [7] - Over the past 12 months, shares have increased 138.56% and are closer to their 52-week highs than lows [7]

Summary of Ocugen's OCU410 Phase 2 ARMADA Clinical Trial Conference Call Company and Industry Overview - **Company**: Ocugen - **Industry**: Biotechnology, specifically focusing on gene therapy for geographic atrophy (GA), a form of age-related macular degeneration (AMD) Key Points and Arguments Clinical Updates - The conference call provided updates on the OCU410 program, focusing on the phase 2 ARMADA trial and new data from phase 1 [2][3] - OCU410 is a gene therapy designed to address multiple pathways involved in GA through a single subretinal injection, contrasting with existing therapies that require frequent injections [5][30] Unmet Medical Need - Approximately 2 to 3 million patients in the U.S. and Europe suffer from GA, highlighting a significant unmet medical need [4] - Recent approvals of therapies like Syfovre and Izervay have not fully addressed the disease, as they target only one of the four major pathways involved in GA progression [4] OCU410's Mechanism of Action - OCU410 targets RORA, a nuclear receptor that regulates multiple pathways in retinal cells, providing four therapeutic benefits: 1. Anti-drusen activity, reducing drusen burden [8] 2. Anti-oxidative protection, enhancing retinal cell defenses against oxidative stress [9] 3. Anti-inflammatory modulation, suppressing harmful inflammation [9] 4. Anti-complement activity, preventing complement-mediated cell death [9] Phase 1 Trial Results - The phase 1 trial demonstrated a 20.2% reduction in GA lesion growth at 12 months compared to untreated eyes [15] - Preservation of photoreceptor structure was observed, with a 60% slower loss of the ellipsoid zone in treated eyes [17] Phase 2 Trial Design and Results - The phase 2 trial enrolled 51 patients, with a randomized control design [20] - OCU410 treatment resulted in a 46% statistically significant reduction in GA lesion growth compared to control eyes, with a P-value of 0.015 [24] - The medium dose group showed a 54% reduction in lesion growth, while the high-dose group showed a 36% reduction [25] Comparison with Approved Therapies - OCU410's 46% reduction in lesion growth at 12 months is significantly higher than the approximately 22% reduction seen with approved therapies at 24 months [27][28] - Up to 50% of treated patients achieved greater than 50% lesion size reduction compared to control [29] Safety Profile - OCU410 demonstrated a favorable safety profile with no serious adverse events reported [30] - The one-time administration of OCU410 eliminates the cumulative risks associated with frequent injections required by current therapies [23] Market Potential - OCU410 addresses a massive unmet medical need with the potential for a one-time definitive treatment, contrasting with the ongoing treatment burden of current therapies [31] Other Important Content - The call included insights from clinical trial investigators emphasizing the importance of the ellipsoid zone as a biomarker for photoreceptor health and its correlation with functional vision [40][74] - Future milestones include the full data set release and initiation of the phase 3 trial anticipated in 2026 [43][88] This summary encapsulates the critical aspects of Ocugen's OCU410 program and its potential impact on the treatment landscape for geographic atrophy.

Core Insights - Ocugen, Inc. announced positive preliminary 12-month data from the Phase 2 ArMaDa clinical trial for OCU410, a gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD) [1][4] - The global prevalence of dAMD is 266 million, with GA affecting approximately 2-3 million people in the U.S. and Europe [1][8] Clinical Trial Findings - Phase 2 results show a 46% reduction in lesion growth compared to control, with a 50% responder rate achieving over 50% lesion size reduction [5][6] - No serious adverse events related to OCU410 were reported in both Phase 1 and Phase 2 trials [2][5] - The medium dose achieved a 54% lesion reduction, while the high dose achieved a 36% reduction compared to control [6] Treatment Landscape - Current treatment options for dAMD in the U.S. are limited and involve frequent injections with side effects, leaving approximately 2 million patients in Europe without approved treatments [2][8] - OCU410 is a multifunctional gene therapy targeting multiple pathways associated with GA, contrasting with existing therapies that target a single mechanism [3][9] Future Developments - Ocugen plans to file a Biologics License Application (BLA) for OCU410 in 2028, aiming for three regulatory submissions for marketing authorization within three years [7][10] - Full data from the Phase 2 clinical trial is expected to be reported later this quarter, with Phase 3 trials anticipated to begin in 2026 [4][7]

Core Insights - Ocugen, Inc. is hosting a conference call and live webcast to discuss data from the OCU410 Phase 2 ArMaDa clinical trial, focusing on patients who have completed one year since treatment [1] - The conference call is scheduled for January 15, 2026, at 8:30 a.m. ET, featuring key opinion leaders and Ocugen's executive leadership [1][2] Company Overview - Ocugen, Inc. is a biotechnology leader specializing in gene therapies for blindness diseases, utilizing a modifier gene therapy platform that addresses complex diseases caused by imbalances in multiple gene networks [3] - The company is developing therapies for inherited retinal diseases and blindness diseases, including retinitis pigmentosa, Stargardt disease, and geographic atrophy, which affect millions globally [3]

Core Insights - Ocugen (OCGN) is advancing its gene therapy programs for retinal diseases, with plans to file three regulatory applications in the next three years, resulting in a stock increase of 75.1% in 2025 compared to the industry's 20.9% rise [1] Group 1: OCU400 for Retinitis Pigmentosa (RP) - Enrollment is nearing completion in the phase III liMeliGhT study for OCU400, targeting retinitis pigmentosa, with top-line data expected in Q4 2026 [2][8] - Ocugen plans to file a biologics license application (BLA) for OCU400 in 2026, with a rolling submission starting in the first half of 2026 [2] - OCU400 has the potential to treat multiple gene mutations associated with RP with a single subretinal injection, unlike the only approved gene therapy that targets a specific mutation [3] Group 2: OCU410 for Stargardt Disease - The phase II/III GARDian3 study for OCU410ST, aimed at treating Stargardt disease, is ongoing, with interim data expected in mid-2026 and a BLA filing planned for the first half of 2027 [4][8] - There are currently no FDA-approved treatments for Stargardt disease, highlighting the potential market opportunity for OCU410ST [4] Group 3: OCU410 for Geographic Atrophy (GA) - OCU410 is in phase II development as a one-time gene therapy for geographic atrophy, with data expected in Q1 2026 and phase III starting in mid-2026 [5][8] - Currently, patients with GA have only one treatment option, which requires multiple injections and addresses only one aspect of the disease, while OCU410 aims to address multiple aspects [6]

Financial Data and Key Metrics Changes - The company's cash, cash equivalents, and restricted cash totaled $32.9 million as of September 30, 2025, down from $58.8 million as of December 31, 2024 [13] - Total operating expenses for the three months ended September 30, 2025, were $19.4 million, compared to $14.4 million for the same period in 2024 [14] Business Line Data and Key Metrics Changes - The OCU400 phase three LI M EL IGHT clinical trial is on track for BLA and MAA submissions in 2026, addressing multiple genetic mutations with a single therapeutic approach [7] - OCU410 is designed to address multiple pathways implicated in dry age-related macular degeneration, showing a 23% reduction in lesion growth at 12 months [12] Market Data and Key Metrics Changes - Approximately 300,000 people in the U.S. and Europe combined live with Retinitis Pigmentosa (RP), with OCU400 targeting the remaining 98-99% of RP patients [7][8] - There are an estimated 7,000 individuals in South Korea with RP, representing about 7% of the U.S. market [9] Company Strategy and Development Direction - The company aims to file three BLAs in the next three years, with a focus on addressing unmet medical needs for patients facing vision loss [5] - Ocugen is pursuing regional partnerships to maximize patient reach while generating returns for shareholders, including an exclusive licensing agreement with Kwang Dong Pharmaceutical for OCU400 in South Korea [9] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the progress of clinical trials and the potential for OCU400 to provide a therapeutic option for a large patient population [8] - The company anticipates commercialization of OCU400 in 2027, with brand planning and marketing initiatives already underway [8] Other Important Information - The company closed a registered direct offering with Janus Henderson in August, raising approximately $20 million to extend its runway through the second quarter of 2026 [6] - The interim data from the ongoing phase two three study for OCU14ST is expected mid-2026, further advancing the goal of bringing the treatment to patients [11] Q&A Session Summary Question: Timing of BLA completion for OCU400 - Management indicated that resources are ready to turn around pivotal data to the FDA in weeks [15] Question: Manufacturing readiness for commercial production - The company confirmed that process validation runs are on target and materials for registration can be commercialized [16] Question: Endpoints for OCU410ST interim readout - The primary endpoint is lesion growth compared to untreated controls, with visual acuity as a secondary endpoint [17] Question: Statistical design of the LIMELIGHT study for OCU400 - The study includes a control arm and has a planned enrollment of 150 subjects, with a 97% power for the study [19] Question: Enrollment progress for OCU410ST - Enrollment is on track with no geographical restrictions, covering a pediatric population [24] Question: Commercial strategy for OCU400 - The company is targeting a broad indication for RP, including various mutations to maximize treatment potential [25] Question: Regulatory path for OCU400 in South Korea - Approval in South Korea is expected to follow FDA approval without the need for additional clinical trials [29] Question: Manufacturing capacity for larger indications - The company has a facility in Pennsylvania and plans to produce all U.S. supply from this site by 2027 [30]

Core Insights - Ocugen is progressing with its late-stage modifier gene therapies, targeting BLA/MAA filings in 2026 and 2027, with significant enrollment milestones achieved in ongoing clinical trials [2][3][4] Clinical Development - The OCU400 Phase 3 liMeLiGhT clinical trial is nearing completion, with 50% enrollment achieved, and is on track for BLA and MAA submissions in 2026 [3][6] - The OCU410ST Phase 2/3 GARDian3 pivotal confirmatory trial is also progressing well, with the EMA accepting a single U.S.-based trial for MAA submission [4][6] - Ocugen's gene-agnostic approach aims to treat retinitis pigmentosa (RP), which affects approximately 300,000 individuals in the U.S. and Europe, with a single therapeutic method addressing multiple genetic mutations [3][12] Licensing and Financial Agreements - Ocugen has entered an exclusive licensing agreement with Kwangdong Pharmaceutical for OCU400 in South Korea, which includes potential upfront and milestone payments totaling up to $7.5 million, plus sales milestones projected to reach $180 million in the first decade [2][6] - The company will receive a 25% royalty on net sales from Kwangdong and is responsible for manufacturing and supplying OCU400 [2][6] Financial Performance - For Q3 2025, Ocugen reported total revenue of $1.75 million, an increase from $1.14 million in Q3 2024, while total operating expenses rose to $19.4 million from $14.4 million in the same period [10][19] - The net loss for Q3 2025 was $20.05 million, compared to a net loss of $12.97 million in Q3 2024 [19] - As of September 30, 2025, the company had cash and cash equivalents totaling $32.9 million, down from $58.8 million at the end of 2024 [12][19] Future Outlook - Upcoming milestones include the release of full data from the OCU410 Phase 2 trial in Q1 2026, interim data from the OCU410ST trial in mid-2026, and top-line data from the OCU400 Phase 3 trial in Q4 2026 [7][12] - The company is actively pursuing additional financing and strategic partnerships to support its commercialization efforts [7]

Financial Data and Key Metrics Changes - The company's cash, cash equivalents, and restricted cash totaled $27.3 million as of June 30, 2025, down from $58.8 million as of December 31, 2024 [22] - Total operating expenses for the three months ended June 30, 2025, were $15.2 million, including $8.4 million in research and development expenses and $6.8 million in general and administrative expenses, compared to $16.6 million in the same period of 2024 [22][23] Business Line Data and Key Metrics Changes - The OCU400 Phase three Limelight clinical trial for retinitis pigmentosa is actively recruiting patients in the United States and Canada, with a target for BLA and MAA filings in 2026 [7] - The OCU410 ST clinical trial for Stargardt disease has achieved key milestones, including the first patient dosing in July 2025 [12] - Preliminary data for OCU410 ST and OCU410 shows favorable safety and efficacy with improved structural and functional outcomes [8] Market Data and Key Metrics Changes - The OCU400 therapy has the potential to address over 100 different mutations associated with retinitis pigmentosa, which currently lacks approved treatment options for approximately 298,000 patients in the US and Europe [10] - Stargardt disease affects around 100,000 individuals in the US and Europe combined, with an estimated one million people globally, and there is currently no FDA-approved treatment available [11] Company Strategy and Development Direction - The company aims to file three biological licensing applications and market authorization applications in the next three years, focusing on providing one-time therapies for significant unmet medical needs [6] - A regional partnership for OCU400 has been signed with a well-established leader in the pharmaceutical sector in Korea, allowing the company to retain rights in larger geographies [16] - The proposed reverse merger with OrthoCelix is intended to create a NASDAQ-listed late clinical stage regenerative cell therapy company, focusing on orthopedic diseases [17] Management's Comments on Operating Environment and Future Outlook - Management expressed enthusiasm about the progress of their modified gene therapy platform and the positive response from the FDA regarding their clinical trials [6][12] - The company is actively exploring strategic partnerships to enhance its financial position and drive long-term strategy [23] Other Important Information - The Data and Safety Monitoring Board reported no serious adverse events related to OCU400, recommending the continuation of the study [11] - Leadership changes were made to strengthen the company's internal expertise, including the appointment of a new Chief Development Officer and Executive Vice President of Commercial and Business Development [20] Q&A Session Summary Question: Are there any other deals that you might be looking to execute? - The company is continuously looking for potential partnership opportunities, including regional partnerships for all gene therapy programs [27] Question: How many sites are included in the Stargardt Phase three trial? - The trial has 15 centers activated, and the company does not anticipate challenges in enrolling Stargardt patients due to the lack of approved products [32] Question: What does a 27% lesion growth reduction in GA mean for patients? - A 27% reduction in lesion growth is expected to significantly help patients maintain their visual function over time [34] Question: Was there a futility analysis during the DSMB review? - There was no futility analysis; the review was purely for safety [40] Question: When will the interim analysis for geographic atrophy be updated? - The interim analysis data is expected in the fourth quarter, providing structural and functional outcomes [41] Question: What is the regulatory path for OCU400 in Korea? - The company expects to use US FDA approval to gain approval in Korea without needing further clinical trials [63]

Financial Data and Key Metrics Changes - Research and development expenses for Q1 2025 were $9.5 million, up from $6.8 million in Q1 2024, indicating a significant increase in investment in R&D [21] - General and administrative expenses for Q1 2025 were $6.5 million, slightly up from $6.4 million in the same period of 2024 [22] - The net loss for Q1 2025 was approximately $15.3 million, or $0.05 net loss per share, compared to a net loss of approximately $11.9 million, or $0.05 net loss per share, in Q1 2024 [22] - Cash and restricted cash totaled $38.1 million as of March 31, 2025, down from $58.8 million as of December 31, 2024, indicating a decrease in liquidity [22] Business Line Data and Key Metrics Changes - The company is advancing three gene therapies through clinical trials, with a focus on modifier gene therapy, which targets diseases with large patient populations [7][9] - OCU400 has shown positive two-year long-term safety and efficacy data, demonstrating a statistically significant improvement in visual function [12] - OCU410 is designed to address multiple pathways in dry AMD, with a goal of providing a one-time therapy for millions suffering from the condition [17] Market Data and Key Metrics Changes - OCU400 has the potential to treat approximately 300,000 RP patients in the U.S. and EU, and 1.6 million patients globally, using a gene-agnostic approach [11] - The company is planning to initiate a Phase III clinical trial for OCU410 in 2026, targeting a BLA submission in 2028 [10] Company Strategy and Development Direction - The company aims to file three biologics license applications (BLA) in the next three years, focusing on modifier gene therapies that offer potential cures for life [7] - The strategy includes building manufacturing capabilities in-house to support future commercial launches, with plans for a second site in the U.S. [31][32] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the progress of their gene therapy programs and the significance of upcoming milestones, emphasizing the mission to address unmet medical needs for patients facing vision loss [58] - The company is actively engaging with regulatory agencies to align clinical trial designs and expedite the approval process for their therapies [39] Other Important Information - The company is in discussions regarding developmental funding for its vaccine technology for influenza [20] - The investigational new drug application for OCU500 is in effect, with plans for a Phase I clinical trial to begin in Q2 2025 [20] Q&A Session Summary Question: Enrollment status for the Limelight study and filing targets - Management confirmed that enrollment for the OCU400 Phase III trial is on track for the first half of 2025, with a BLA submission expected a year from now [28] Question: Manufacturing capabilities for filing and commercialization - Management stated that process validations at commercial scale are on target for completion this year, with plans to bring manufacturing in-house after initial commercial launch [30][31] Question: Update on the OCU200 program - Management indicated that a clinical update, including preliminary efficacy and safety data, is expected before the end of the year [34] Question: Clarification on European regulations for OCU400 - Management clarified that no additional trials are required in Europe if approval is obtained in the U.S., as the trial has sufficient representation for global mutations [55]