MALVERN, Pa., April 08, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-founder of Ocugen will present at the 2025 Cell & Gene Meeting on the Mediterranean being held April 15-17, 2025 at the Rome Cavalieri in Rome, Italy. “I look forward to sharing more on how Ocugen’s modifier gene therapy platform can potentially cha ...

OCU200 has a very favorable safety and tolerability profileNo serious adverse events related to the study drug have been reportedDosing of the second cohort has been approved MALVERN, Pa., March 18, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the Data and Safety Monitoring Board (DSMB) for the OCU200 clinical trial recently convened and reviewed safety data following dosing ...

Financial Data and Key Metrics Changes - Research and development expenses for Q4 2024 were $8.3 million, up from $7.8 million in Q4 2023. For the full year, R&D expenses decreased to $32.1 million from $39.6 million in 2023 [34] - General and administrative expenses for Q4 2024 were $6.3 million, compared to $5.2 million in Q4 2023. For the full year, G&A expenses decreased to $26.7 million from $32.0 million in 2023 [34] - Net loss for Q4 2024 was approximately $13.9 million or $0.05 per share, compared to a net loss of approximately $11 million or $0.04 per share in Q4 2023. Full year net loss was $54.1 million or $0.20 per share, compared to a net loss of $63.1 million in 2023 or $0.26 per share [35] - Cash and restricted cash totaled $58.8 million as of December 31, 2024, compared to $39.5 million at the end of 2023, providing a cash runway into Q1 2026 [36] Business Line Data and Key Metrics Changes - OCU400, targeting retinitis pigmentosa, has shown a statistically significant improvement in low-luminance visual acuity (LLVA) with a p-value of 0.005% at two years, demonstrating long-term durability [14][15] - OCU410ST received orphan medicinal product designation from the EMA and demonstrated a 100% stabilization or improvement in visual function in treated eyes, with a p-value of 0.02% [17][18] - OCU410, targeting geographic atrophy, showed a 44% slower lesion growth at nine months compared to untreated eyes, with no serious adverse events reported [24][26] Market Data and Key Metrics Changes - Approximately 300,000 people in the US and EU are affected by retinitis pigmentosa, with OCU400 addressing a significant unmet medical need [13] - There are no approved treatments for Stargardt disease, affecting about 100,000 patients in the US and Europe, highlighting the potential market for OCU410ST [17] - Geographic atrophy affects approximately 2 to 3 million patients in the US and EU, with 8 million patients globally, indicating a large market opportunity for OCU410 [23] Company Strategy and Development Direction - The company aims to file three Biologics License Applications (BLAs) in the next three years: OCU400 in 2026, OCU410ST in 2027, and OCU410 in 2028, reflecting a bold ambition supported by FDA alignment [11] - The company is focused on advancing its gene therapy platform and has secured $65 million in financing to support its clinical programs [12] - The strategy includes leveraging the ATMP classification for OCU400 and OCU410ST to expedite regulatory review and enhance interactions with the EMA [14][17] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving BLA targets due to a strong track record and ongoing clinical progress across all programs [56] - The company is optimistic about the potential of its therapies to address significant unmet medical needs and improve patient outcomes [11][27] - Management highlighted the importance of safety and tolerability profiles in differentiating their products from existing therapies [64] Other Important Information - The company is actively enrolling patients in clinical trials for its therapies, with plans to complete enrollment for OCU400 in the first half of 2025 [16] - The investigational new drug application for OCU500, an inhaled mucosal vaccine for COVID-19, has been cleared by the FDA, with a Phase 1 trial expected to start in Q2 2025 [30][31] Q&A Session Summary Question: Does the runway factor in newly launching studies like OCU410ST Phase 2/3? - Yes, it is already budgeted [42] Question: When can we expect data from the DME program? - Safety and efficacy reports for OCU200 are expected towards the end of the year, focusing on BCVA and dose response [44][47] Question: What gives confidence in achieving the aggressive BLA filing targets? - The track record of starting gene therapy programs and the ongoing progress across all three programs provide confidence [54][56] Question: How will OCU410 compete with existing therapies? - The safety and tolerability profile of OCU410 is a significant advantage, as current therapies have concerning safety profiles [61][64] Question: Will there be grant revenue associated with the OCU500 Phase 1 trial? - NIAID is sponsoring the program, and the company has completed its obligations; funding will not be recorded as revenue [78][81] Question: What is the manufacturing strategy for OCU410ST? - Commercial-scale manufacturing will be used for both Phase 1 and Phase 2/3 studies [85] Question: Will the study include both US and ex-US sites? - The study will primarily be conducted in the US, with Canada as the only ex-US site [87]

Financial Performance and Capital Requirements - The company incurred net losses of approximately $63.1 million and $54.1 million for the years ended December 31, 2023, and 2024, respectively, with an accumulated deficit of $340.2 million as of December 31, 2024[229]. - The cash balance as of December 31, 2024, was approximately $58.5 million, which is insufficient to meet capital requirements over the next 12 months[229]. - The company estimates that its cash and cash equivalents will fund operations into the first quarter of 2026, indicating a need for significant additional capital[229]. - The company anticipates an increase in expenses for fiscal year 2025 compared to fiscal year 2024 due to ongoing clinical activities and increased headcount[232]. - The company may need to raise additional capital to support its product development activities, which could lead to dilution for existing shareholders[230]. - The company has $30.0 million of outstanding principal borrowings under the Loan and Security Agreement as of December 31, 2024[247]. - The company has incurred significant net operating losses, with United States federal net operating loss carryforwards of approximately $263.9 million as of December 31, 2024[254]. - The company is actively seeking funding through various means, including public and private placements, government grants, and collaborations[244]. Product Development and Regulatory Challenges - The company has not generated any revenue from product sales to date and may never attain profitability without sufficient revenues from product candidates[231]. - The company faces significant risks related to the success of its product candidates, including regulatory approval and market acceptance[225]. - The company relies on third parties for conducting clinical trials, which may not perform satisfactorily, potentially delaying development[225]. - The company is focused on developing product candidates from its modifier gene therapy platform, which is subject to an uncertain regulatory environment[263]. - Regulatory approval processes for gene therapy products are lengthy and unpredictable, potentially delaying commercialization and revenue generation[268]. - The company is at risk of delays in clinical trials due to various factors, including regulatory approvals and patient enrollment challenges[270]. - The company must navigate changing regulatory requirements that could lengthen the approval process and increase development costs[265]. - The company must establish agreements with distributors and maintain patent protections to ensure successful commercialization of its product candidates[265]. - The company currently has no product candidates approved for sale in any jurisdiction, including international markets, and lacks experience in obtaining regulatory approval outside the United States[298]. Market and Competitive Landscape - The biotechnology industry is characterized by intense competition, with numerous companies focusing on gene therapies and regenerative medicine[331]. - Competitors may obtain regulatory approval more rapidly, potentially establishing a strong market position before the company can enter the market[336]. - Acceptance of product candidates among physicians and patients may be limited, affecting revenue generation even if approved[339]. - Market acceptance of product candidates is crucial for generating significant revenues and achieving profitability[341]. - Potential market opportunities for product candidates may be smaller than estimated, adversely affecting revenue and business sustainability[342]. - Successful commercialization depends on timely coverage and adequate reimbursement from third-party payors, which is influenced by various factors including efficacy and cost-effectiveness[343]. Manufacturing and Supply Chain Risks - The company does not own manufacturing facilities and faces risks related to production delays, quality control, and compliance with stringent regulations, which could impact revenue generation[363]. - The company relies on third-party manufacturers for clinical and commercial supplies, which may affect future profit margins if agreements are not established on commercially favorable terms[361]. - The number of available, qualified third-party manufacturers is limited, and finding alternatives could lead to delays and increased expenses in product development and commercialization[372]. - The company may encounter shortages in materials necessary for production, which could hinder the ability to meet demand for approved products and result in lost revenues[371]. Legal and Compliance Issues - The company is currently facing numerous securities class action lawsuits, which could divert management's attention and potentially harm its business[383]. - The company may be subject to substantial penalties if it fails to comply with federal and state healthcare laws, adversely affecting its financial condition and results of operations[385]. - Legislative changes, including cost-containment programs and price controls, could negatively impact the company's business and results of operations[387]. - The company may face criminal and civil penalties if it fails to comply with anti-corruption laws, which could adversely impact its financial condition and operations[403]. Intellectual Property and Patent Risks - The patent prosecution process is expensive and time-consuming, and the company may not be able to maintain necessary patent protections for its technologies[404]. - The company may face challenges in identifying patentable aspects of its research and development output, impacting its ability to secure patent protection[406]. - The company’s patent rights may be challenged in courts or patent offices, leading to potential loss of exclusivity or narrowing of patent claims[409]. - The company’s ability to develop and market its product candidates is contingent upon not infringing third-party intellectual property rights, which poses a significant risk[417].

Core Insights - Ocugen, Inc. reported its fourth quarter and full year 2024 financial results, highlighting advancements in its clinical programs and regulatory achievements [1][2][12] Clinical Developments - The company is advancing its clinical programs, including a Phase 2/3 trial for OCU410ST targeting Stargardt disease, which affects approximately 100,000 patients in the U.S. and Europe [2][3] - OCU410ST showed a 52% reduction in lesion growth and a statistically significant improvement in visual function in treated eyes during the Phase 1 trial [3] - The OCU410 clinical trial for geographic atrophy (GA) reported a 44% reduction in lesion growth and a meaningful improvement in visual function [5] - Two-year data from the OCU400 trial for retinitis pigmentosa (RP) demonstrated a 100% improvement in visual function among treated subjects [6] Regulatory Achievements - Ocugen received orphan medicinal product designation from the EMA for OCU410ST and positive opinions for ATMP classification for OCU400, OCU410, and OCU410ST [7][8] - The FDA has aligned with Ocugen on the Phase 2/3 trial for OCU410ST, potentially expediting clinical development by two to three years [7] Financial Performance - For Q4 2024, research and development expenses were $8.3 million, up from $7.8 million in Q4 2023, while general and administrative expenses increased to $6.3 million from $5.2 million [14] - The company reported a net loss of $0.05 per share for Q4 2024, compared to a loss of $0.04 per share in Q4 2023 [14] - Full year 2024 research and development expenses totaled $32.1 million, down from $39.6 million in 2023, with a net loss of $0.20 per share compared to $0.26 in 2023 [15] Cash Position - As of December 31, 2024, Ocugen had cash and restricted cash totaling $58.8 million, an increase from $39.5 million in 2023, which is expected to fund operations into Q1 2026 [15]

Financial Performance - Ocugen reported a net loss per common share of $0.05 for Q4 2024, compared to a loss of $0.04 in Q4 2023; for the full year, the net loss per share was $0.20, down from $0.26 in 2023[12][18] - Total revenue for the three months ended December 31, 2024, was $764 million, a decrease of 45.7% compared to $1,409 million in the same period of 2023[23] - Net loss for the three months ended December 31, 2024, was $13,880 million, compared to a net loss of $10,970 million in the same period of 2023, indicating a 26.5% increase in losses[23] - Net loss per share attributable to common shareholders was $0.05 in Q4 2024, compared to $0.04 in Q4 2023, indicating a worsening loss per share[23] Expenses - Fourth quarter 2024 research and development expenses were $8.3 million, up from $7.8 million in Q4 2023; general and administrative expenses increased to $6.3 million from $5.2 million[12] - Full year 2024 research and development expenses totaled $32.1 million, a decrease from $39.6 million in 2023; general and administrative expenses fell to $26.7 million from $32.0 million[18] - Research and development expenses rose to $8,290 million in Q4 2024, up 6.5% from $7,779 million in Q4 2023[23] - Total operating expenses for the year ended December 31, 2024, were $58,812 million, down 17.8% from $71,567 million in 2023[23] Financing and Cash Position - The company has secured $65 million in equity/debt financing in the second half of 2024 to extend its cash runway into Q1 2026[10] - Ocugen's cash and restricted cash totaled $58.8 million as of December 31, 2024, compared to $39.5 million a year earlier[18] Clinical Trials and Product Development - The Phase 2/3 clinical trial for OCU410ST for Stargardt disease will randomize 51 subjects, with a BLA filing planned for 2027[3] - Preliminary 9-month efficacy data for OCU410 in geographic atrophy patients showed a 44% slower lesion growth from baseline and a clinically meaningful 2-line improvement in visual function[4] - Two-year data from the Phase 1/2 OCU400 clinical trial for retinitis pigmentosa demonstrated a statistically significant (p=0.005) improvement in visual function in all evaluable treated subjects[5] - The company aims for three potential BLA submissions in 2026, 2027, and 2028 for treatments targeting retinitis pigmentosa, Stargardt disease, and geographic atrophy, respectively[11] - OCU200 is currently in Phase 1 clinical trials for diabetic macular edema, targeting the 30-40% of patients who do not respond to current anti-VEGF therapies[8] Assets and Liabilities - Total assets increased to $82,442 million in 2024 from $64,547 million in 2023, representing a growth of 27.5%[21] - Long-term debt increased significantly to $27,345 million in 2024 from $2,800 million in 2023, reflecting a rise of 875.5%[21] - Total current liabilities rose to $21,588 million in 2024, up 26.5% from $17,089 million in 2023[21] - Stockholders' equity decreased to $29,632 million in 2024 from $40,564 million in 2023, a decline of 27.0%[21] Shareholder Information - The weighted average shares used in calculating net loss per common share increased to 290,924,531 in Q4 2024 from 256,506,387 in Q4 2023[23]

Core Insights - Ocugen, Inc. has received a positive opinion from the European Medicines Agency's Committee for Advanced Therapies for its gene therapy candidates OCU410 and OCU410ST, aimed at treating vision loss due to geographic atrophy and Stargardt disease respectively [1][3] Group 1: Product Development and Clinical Trials - OCU410 is designed for patients with geographic atrophy, affecting 2-3 million people in the U.S. and Europe, while OCU410ST targets Stargardt disease, which affects approximately 100,000 people in the same regions [2] - The Phase 2 clinical trial for OCU410 has been completed, with plans to initiate a Phase 3 trial next year, targeting Marketing Authorization Application and Biologics License Application filings in 2028 [3] - Preliminary data from the Phase 1 trial of OCU410 showed a 44% reduction in lesion growth and a clinically meaningful improvement in visual function [4] - The Phase 1 trial data for OCU410ST indicated a 52% reduction in lesion growth and a statistically significant improvement in visual function [5] Group 2: Mechanism and Technology - Both OCU410 and OCU410ST utilize an adeno-associated virus platform to deliver the RORA gene, which plays a crucial role in lipid metabolism and has anti-inflammatory properties [6] - The gene therapy is administered through a single subretinal injection, offering a potential one-time treatment solution [6] Group 3: Company Overview - Ocugen, Inc. focuses on developing innovative gene and cell therapies, biologics, and vaccines to address unmet medical needs globally [7]

Core Insights - Ocugen, Inc. has received a positive opinion from the European Medicines Agency's Committee for Advanced Therapies for its gene therapy candidates OCU410 and OCU410ST, aimed at treating vision loss due to geographic atrophy and Stargardt disease respectively [1][3] Company Developments - OCU410 is designed for patients with geographic atrophy, affecting 2-3 million people in the U.S. and Europe, while OCU410ST targets Stargardt disease, which affects approximately 100,000 individuals in the same regions [2][3] - The company has completed dosing for Phase 2 of the OCU410 ArMaDa clinical trial and plans to initiate Phase 3 trials next year, with potential regulatory submissions expected in 2028 [3] - The FDA has endorsed Ocugen's plan for a Phase 2/3 pivotal trial for OCU410ST, with potential submissions for marketing authorization anticipated in 2027 [3] Clinical Trial Results - Preliminary data from the Phase 1 trial of OCU410 showed a 44% reduction in lesion growth and a clinically meaningful improvement in visual function in treated eyes compared to untreated eyes [4] - Phase 1 data for OCU410ST indicated a 52% reduction in lesion growth and a statistically significant improvement in visual function [5] Technology and Innovation - Both OCU410 and OCU410ST utilize an adeno-associated virus platform for gene delivery, targeting multiple pathways associated with dry age-related macular degeneration and Stargardt disease [6] - The RORA gene, delivered via a single subretinal injection, plays a crucial role in lipid metabolism and has demonstrated anti-inflammatory properties [6] Company Overview - Ocugen, Inc. focuses on developing innovative gene and cell therapies, biologics, and vaccines to address unmet medical needs and improve patient health globally [7]

Core Viewpoint - Ocugen, Inc. has reached alignment with the FDA to proceed with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST, a gene therapy for Stargardt disease, which could lead to a biologics license application (BLA) submission if successful [1][2][5] Company Developments - The GARDian trial for OCU410ST has shown promise, with the FDA recognizing its potential to address a critical unmet medical need for approximately 44,000 Stargardt patients in the U.S. [3] - The company plans to initiate the pivotal trial in the coming months and aims for a potential BLA filing by 2027 [2] - OCU410ST has received orphan drug designations from both the FDA and the European Medicines Agency (EMA) in 2023 and 2024, respectively [2] Clinical Trial Details - The Phase 2/3 trial will involve 51 subjects, with 34 receiving a single subretinal injection of OCU410ST and 17 serving as untreated controls [4] - The primary endpoint is the change in atrophic lesion size, while secondary endpoints include visual acuity measurements [4] - One-year data from the trial will be utilized for the BLA filing [4] Safety and Efficacy - OCU410ST has demonstrated a favorable safety profile with no serious adverse events reported, including no cases of ischemic optic neuropathy or endophthalmitis [7] - The Phase 1 study indicated a 52% slower lesion growth from baseline in treated eyes compared to untreated eyes at the 6-month follow-up [7] - There was a statistically significant 2-line (10-letter) improvement in visual function at the 6-month follow-up in treated eyes [7] About Stargardt Disease - Stargardt disease is a genetic eye disorder leading to retinal degeneration and vision loss, affecting around 100,000 people in the U.S. and Europe combined [2][8] - The disease primarily causes loss of central vision due to the degeneration of photoreceptor cells in the macula [9] About Ocugen, Inc. - Ocugen, Inc. focuses on developing novel gene and cell therapies, biologics, and vaccines aimed at improving health and addressing unmet medical needs [10] - The company is advancing research in multiple areas, including retinal diseases and infectious diseases [10]

Core Viewpoint - Ocugen, Inc. is set to host a conference call and live webcast on March 5, 2025, to discuss its fourth quarter and full year 2024 financial results and provide a business update [1]. Group 1: Company Overview - Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines aimed at improving health and offering hope to patients globally [3]. - The company is advancing a breakthrough modifier gene therapy platform that has the potential to treat multiple retinal diseases with a single product [3]. - Ocugen is also conducting research in infectious diseases and orthopedic diseases to address unmet medical needs [3]. Group 2: Upcoming Events - A pre-market earnings announcement will be issued on the same day as the conference call [2]. - Participants can join the call using specific dial-in numbers for U.S. and international callers, with a conference ID provided [2]. - A replay of the call and archived webcast will be available for approximately 45 days following the event on the Ocugen investor site [2].