The single global Phase 3 trial, ArMaDa3 (NCT07770828), is the first pivotal gene therapy trial in geographic atrophy (GA)U.S. Food and Drug Administration (FDA) granted OCU410 Regenerative Medicine Advanced Therapy (RMAT) designation, providing enhanced agency engagement throughout development and eligibility for accelerated approval and priority reviewPhase 3 design fully aligned with FDA; Biologics License Application (BLA) filing anticipated in 2028OCU410 is designed as a one-time subretinal gene therapy that addresses multiple disease pathways, offering a differentiated approach from approved complement inhibitors in the U.S. which address individual disease pathways and require ongoing intravitreal injections MALVERN, Pa., Sept. 01, 2026 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the first patient was dosed in the global Phase 3 registrational trial of OCU410 (AAV5-hRORA), its first-in-class modifier gene therapy candidate for GA secondary to dry age-related macular degeneration (dAMD). The Company also highlighted the recent FDA RMAT designation for OCU410, which supports a potential accelerated development and review pathway for the program.
“Dosing the first patient in our global Phase 3 trial, just weeks after receiving RMAT designation, marks a defining moment for the OCU410 program – and for the millions of people living with geographic atrophy. Outside the U.S., there are currently no approved treatments for GA, while in the U.S., available treatment options address only one of the four disease pathways and require ongoing, repeated eye injections,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “This is our third modifier gene therapy program to advance into late-stage development, demonstrating the strength of our platform and our vision for potentially delivering a one-time treatment for life.”
The initiation of dosing follows the successful completion of a Type B End-of-Phase 2 (EOP2) meeting with FDA’s Center for Biologics Evaluation and Research (CBER) in July 2026, resulting in alignment on all critical Phase 3 design elements, including primary and secondary endpoints, dose, adaptive design, and a single pivotal trial pathway to support a BLA.
“We are entering a global single Phase 3 with a well-defined program: a dose validated in a randomized, controlled Phase 2 study; an FDA-endorsed primary endpoint measuring the rate of lesion growth; and a secondary endpoint assessing functional vision,” said Mohamed Genead, MD, Chief Medical Officer of Ocugen. “The Phase 3 program builds on compelling 12-month Phase 2 data, which demonstrated a statistically significant 31% reduction in lesion growth with the optimal dose compared with control following a single subretinal injection, along with concordant preservation of the ellipsoid zone and no drug-related serious adverse events (SAEs) or adverse events of special interest (AESIs).”
OCU410 delivers the human retinoid-related orphan receptor alpha (RORA) modifier gene via a single subretinal injection of an AAV5 vector. Unlike therapies targeting a single pathway, OCU410 is designed to simultaneously address multiple pathophysiological drivers of GA-complement overactivation, chronic inflammation, oxidative stress, and lipid dysregulation. GA affects approximately 2–3 million people in the U.S. and Europe and is a leading cause of irreversible central vision loss in older adults, with prevalence expected to rise as the population ages.
According to study investigator Victor Gonzalez, MD, “Patients with geographic atrophy continue to face irreversible structural and functional loss of the retina, along with limited treatment options. The OCU410 Phase 3 study provides an important opportunity to evaluate a novel, potential one-time gene therapy approach that could lessen the burden of current treatments in the U.S., which require patients to undergo multiple injections every year.”
Global Phase 3 Registrational Trial Design
The Phase 3 trial is a global, multicenter, randomized, controlled study enrolling 237 subjects with GA secondary to dAMD, randomized 2:1 to a single 200 µL subretinal injection of OCU410 (5×1010 vg/mL) or an untreated control arm, with sites in the United States, Canada, Europe, and Latin America.
Primary endpoint: Rate of change of square root-transformed GA lesion area (√mm²/year) by fundus autofluorescence (FAF) at baseline, Month 4, Month 8, and Month 12, analyzed by MMRM.Secondary endpoints: Proportion of subjects with Low-Luminance Visual Acuity (LLVA) loss ≥15 ETDRS letters at two consecutive visits through Month 12, providing a functional vision anchor to the primary anatomic endpoint; and rate of change of ellipsoid zone (EZ) area loss by SD-OCT.Regulatory path: A single, adequate and well-controlled Phase 3 trial, aligned with FDA feedback, is intended to support a BLA filing anticipated in 2028. Discussions are ongoing with the European Medicines Agency (EMA) regarding alignment to potentially support a marketing authorization application (MAA) in Europe with this single Phase 3 trial.
RMAT Designation: Regulatory and Strategic Significance
On July 29, 2026, FDA granted RMAT designation to OCU410 based on Phase 2 clinical data demonstrating clinically meaningful efficacy and a favorable safety profile, with no serious adverse events related to OCU410 reported. RMAT designation is granted to regenerative medicine therapies intended to treat serious or life-threatening conditions where preliminary clinical evidence indicates the potential to address an unmet medical need.
For the OCU410 program, RMAT designation provides:
Eligibility for accelerated approval and priority review, which may compress the time from BLA submission to potential market entry.All benefits of Breakthrough Therapy designation, including intensive FDA guidance on efficient development and organizational commitment involving senior FDA leadership.Early and frequent FDA interactions on the use of surrogate and intermediate clinical endpoints reasonably likely to predict long-term clinical benefit-directly relevant to OCU410’s FAF-based anatomic primary endpoint.Potential flexibility in satisfying post-approval requirements, including through expanded patient registries or real-world evidence. Taken together with FDA alignment on the Phase 3 design and the initiation of dosing, RMAT designation further de-risks the regulatory pathway for OCU410 and reinforces the differentiation of a one-time, multi-pathway gene therapy in a GA market currently served only by chronically administered intravitreal complement inhibitors.
Supporting Phase 2 ArMaDa Data
The Phase 3 trial and the RMAT designation are supported by 12-month data from the Phase 2 ArMaDa trial (NCT06018558), a multicenter, randomized, controlled study of 51 subjects with GA secondary to dAMD.
Lesion growth (FAF): 31% reduction in GA lesion area growth rate in the medium dose group versus control at 12 months (p < 0.05) in the pivotal phase 3 population (lesion size of ≥2.5 mm2 and ≤17.5 mm2), a potential 2× treatment benefit relative to the 15% and 22% reductions reported for currently approved therapies in the U.S. at 12 and 24 months, respectively.EZ preservation (SD-OCT): 27% reduction in ellipsoid zone area loss in the medium dose group versus control, a structural correlate of visual function.Responder analysis: In the medium dose group, approximately 20% of treated subjects showed no disease progression; 75% demonstrated >30% reduction in lesion growth at 12 months.Safety: No OCU410-related serious adverse events (SAEs) or adverse events of special interest (AESIs) reported to date. About OCU410
OCU410 (AAV5-hRORA) is Ocugen’s investigational first-in-class modifier gene therapy, delivering the RORA gene via a single unilateral subretinal injection to regulate complement activation, neuroinflammation, oxidative stress, and lipid metabolism – multiple pathways implicated in the pathogenesis of GA. OCU410 has received RMAT designation from the FDA and Advanced Therapy Medicinal Product classification from the European Medicines Agency's Committee for Advanced Therapies.
About Ocugen, Inc.
Ocugen, Inc. is a pioneering biotechnology company developing gene therapies for blindness diseases. The Company’s breakthrough modifier gene therapy platform has the potential to address significant unmet medical needs across large patient populations through a gene-agnostic approach. Unlike traditional gene therapies and gene-editing technologies that target a single gene mutation, Ocugen’s modifier gene therapies are designed to address the underlying disease biology by restoring balance across multiple gene networks. The Company is currently advancing programs for inherited retinal diseases and other causes of blindness that affect millions worldwide, including retinitis pigmentosa, Stargardt disease, and geographic atrophy, an advanced form of dry age-related macular degeneration. Discover more at www.ocugen.com and follow us on LinkedIn and X.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, including the timing of enrollment and data readouts, the ability to initiate new clinical programs, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, statements regarding potential market size and commercial possibilities of Ocugen’s product candidates, 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 convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that receipt of RMAT designation may not lead to faster development or accelerated regulatory review or approval; that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing may not be predictive of the results or success of later clinical trials; and that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our annual and quarterly filings 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. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.
Ocugen míří na podání BLA pro OCU400 pro retinitis pigmentosa během několika měsíců po zveřejnění dat z 1. čtvrtletí příštího roku. Firma zároveň čeká top-line data ze studie Stargardtovy choroby ve 2. čtvrtletí příštího roku.
Ocugen NASDAQ: OCGN is positioning its ophthalmology gene therapy portfolio around treatments for inherited retinal diseases and dry age-related macular degeneration, with pivotal-stage programs in retinitis pigmentosa, Stargardt disease and geographic atrophy, Chairman, CEO and Co-Founder Shankar Musunuri said during a Canaccord discussion.
Musunuri said the company is targeting biologics license applications, or BLAs, next year for its retinitis pigmentosa and Stargardt programs, while its geographic atrophy program is expected to follow on a longer timeline. He said the company’s objective is to pursue approvals in major markets, including the U.S., Europe and Japan, and to improve patient access in parallel with regulatory work.
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Modifier Gene Therapy Platform
Ocugen’s approach differs from gene-specific therapies by using “modifier genes,” which Musunuri described as genes that regulate networks of functions in retinal cells. The company’s technology originated from work by Dr. Neena Haider at Harvard, according to Musunuri.
For retinitis pigmentosa, Ocugen is developing OCU400, which uses the Nr2e3 modifier gene. Musunuri said Nr2e3 affects phototransduction pathways, which are particularly relevant in retinitis pigmentosa as patients can lose peripheral and night vision before central vision.
For Stargardt disease and geographic atrophy, Ocugen is developing treatments using the RORA modifier gene. Musunuri said RORA is intended to regulate multiple disease-related pathways, including oxidative stress, lipid metabolism, inflammation and the complement system. He said the company believes the therapy may help restore cellular homeostasis and create a healthier environment for retinal cells.
Musunuri said Ocugen’s intellectual property extends beyond ophthalmology into neurological applications, though he characterized that area as a future opportunity.
Stargardt and Geographic Atrophy Programs
OCU410ST, the company’s Stargardt disease candidate, is in a Phase II/III study enrolling patients ages 3 and older, from early through advanced stages of disease. Musunuri said the study was endorsed by the European Medicines Agency and that the FDA allowed Ocugen to convert its Phase II study into a combined Phase II/III trial following a small Phase I study.
The company expects top-line data from the Stargardt study in the second quarter of next year, Musunuri said. Ocugen then plans to file a BLA and pursue market authorization in parallel within several months of the results. If development proceeds according to plan, he said approval and launch could occur in 2028.
Musunuri contrasted the program with oral therapies that may target individual disease pathways and require ongoing dosing. He said Ocugen’s subretinal gene therapy is designed as a one-time administration, while emphasizing that efficacy and safety data will determine its potential differentiation.
OCU410, Ocugen’s geographic atrophy candidate, uses the same RORA construct as OCU410ST but at a different dose, Musunuri said. The geographic atrophy program has received the FDA’s Regenerative Medicine Advanced Therapy, or RMAT, designation. He said the company has received FDA clearance to begin a single Phase III trial and is working with the EMA to align the study as a global trial.
Retinitis Pigmentosa Readout and Filing Plans
Ocugen expects first-quarter top-line data next year from its Phase III trial of OCU400 in retinitis pigmentosa. The study includes 140 patients, uses a 2:1 treatment-to-control ratio and covers more than 30 mutations, according to Musunuri.
The primary assessment uses a mobility test designed to measure patients’ ability to navigate under low-light conditions. Musunuri said the company refined the test with FDA input and also plans to track low-luminance visual acuity as a secondary and longer-term measure. He said the company observed approximately two lines of low-luminance visual acuity improvement in treated eyes among Phase I/II patients over three years.
Musunuri said Ocugen has treated more than 325 patients across its clinical trials and expanded-access program, including more than 200 patients with retinitis pigmentosa. He said the company has not observed serious adverse events related to its gene therapy programs in those populations.
On manufacturing, Musunuri said Ocugen has completed process performance qualification validation runs needed for an OCU400 BLA submission and has commercial-scale material that could be used for supply. The company expects to complete the BLA within months after the Phase III data. Under RMAT, it may be eligible for a rolling submission, although Musunuri said the final clinical module would start the FDA review clock.
Capital Runway and Commercialization
Musunuri said a recently announced $130 million convertible note financing is expected to fund Ocugen into 2028. He said overall spending is not expected to increase substantially next year because recruitment for two Phase III programs was completed this year, while the geographic atrophy study begins.
The company is also evaluating regional partnerships and non-dilutive funding opportunities, particularly outside the U.S., Musunuri said. He said Ocugen intends to remain opportunistic regarding U.S. commercialization and may consider additional equity financing if needed to support a U.S. launch.
About Ocugen (NASDAQ:OCGN)Ocugen Inc is a clinical-stage biopharmaceutical company focused on discovering, developing and commercializing gene therapies to treat rare inherited retinal diseases, as well as vaccines designed to address unmet needs in infectious diseases. Headquartered in Malvern, Pennsylvania, the company applies its proprietary gene therapy platform to create novel treatments aimed at preserving and restoring vision, while leveraging strategic partnerships to broaden its vaccine pipeline.
In its gene therapy portfolio, Ocugen is advancing multiple programs targeting retinal disorders.
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Ocugen (OCGN - Free Report) came out with a quarterly loss of $0.07 per share versus the Zacks Consensus Estimate of a loss of $0.05. This compares to a loss of $0.05 per share a year ago. These figures are adjusted for non-recurring items.
This quarterly report represents an earnings surprise of -40.00%. A quarter ago, it was expected that this biotech knee implant developer would post a loss of $0.05 per share when it actually produced a loss of $0.06, delivering a surprise of -20%.
Over the last four quarters, the company has not been able to surpass consensus EPS estimates.
Ocugen, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted revenues of $1.49 million for the quarter ended June 2026, surpassing the Zacks Consensus Estimate by 6.29%. This compares to year-ago revenues of $1.37 million. The company has topped consensus revenue estimates three times over the last four quarters.
The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call.
Ocugen shares have lost about 5.2% since the beginning of the year versus the S&P 500's gain of 12.8%.
What's Next for Ocugen?While Ocugen has underperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock?
There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately.
Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions.
Ahead of this earnings release, the estimate revisions trend for Ocugen was mixed. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #3 (Hold) for the stock. So, the shares are expected to perform in line with the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.
It will be interesting to see how estimates for the coming quarters and the current fiscal year change in the days ahead. The current consensus EPS estimate is -$0.05 on $1.3 million in revenues for the coming quarter and -$0.22 on $4.14 million in revenues for the current fiscal year.
Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the top 44% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1.
One other stock from the same industry, CAMP4 Therapeutics Corporation (CAMP - Free Report) , is yet to report results for the quarter ended June 2026.
This company is expected to post quarterly loss of $0.24 per share in its upcoming report, which represents a year-over-year change of +61.3%. The consensus EPS estimate for the quarter has remained unchanged over the last 30 days.
CAMP4 Therapeutics Corporation's revenues are expected to be $1.35 million, down 10% from the year-ago quarter.
Ocugen získal od FDA povolení zahájit fázi 3 studie OCU410 pro geografickou atrofii a získal také označení RMAT. Firma zároveň uzavřela financování v hodnotě 130,0 milionu USD, které prodlužuje hotovostní runway do roku 2028.
Conference Call and Webcast Today at 8:30 a.m. ET Received U.S. Food and Drug Administration (FDA) clearance to initiate OCU410 Phase 3 trial for geographic atrophy (GA), secondary to dry age-related macular degeneration (AMD); on track to initiate Phase 3 this quarter Granted Regenerative Medicine Advanced Therapy (RMAT) designation by FDA for OCU410, enabling eligibility for priority review and accelerated approval Signed a binding term sheet with Roots Pharmaceutical, and its strategic partner Al-Dhow International Holding, to negotiate an exclusive license for OCU400 in retinitis pigmentosa (RP) across the Middle East and North Africa (MENA) region Successfully completed OCU400 Process Performance Qualification (PPQ) batches, supporting Biologics License Application (BLA) and commercial launch supplies Closed $130.0 million convertible senior notes financing, extending cash runway into 2028 Remain on track to announce top-line results for two late-stage clinical programs, OCU400 for RP and OCU410ST for Stargardt disease in 1Q 2027 and 2Q 2027, respectively Strengthened leadership team with the appointments of Mohamed Genead, M.D.
Ocugen oznámila, že FDA udělila OCU410 status RMAT pro léčbu geografické atrofie u suché formy věkem podmíněné makulární degenerace. Tento status může urychlit vývoj i regulační cestu.
RMAT designation is intended to help expedite development of new regenerative medicines that have potential to treat serious medical conditions with significant unmet needThe designation could accelerate OCU410’s regulatory path while further differentiating its one-time treatment for life approach from current therapies requiring chronic administration MALVERN, Pa, July 29, 2026 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted RMAT designation to Ocugen’s investigational product OCU410 for the treatment of geographic atrophy (GA), secondary to dry age-related macular degeneration (dAMD).
"RMAT designation for OCU410 is a significant accomplishment that recognizes both the potential of our ‘one treatment for life’ novel gene therapy platform and the substantial unmet medical need for geographic atrophy,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “With an estimated 2 to 3 million people in the U.S. and Europe affected, a number expected to grow as the population ages, geographic atrophy is a leading cause of irreversible blindness in older adults, highlighting the urgent need for new treatment options. We look forward to continuing to work closely with the FDA to responsibly and efficiently advance the development program for patients suffering from this devastating disease."
The RMAT designation for OCU410 was supported by Phase 2 clinical data demonstrating clinically meaningful efficacy and a favorable safety profile, with no reported serious adverse events related to drug. Based on these data, the FDA determined that OCU410 met the criteria for RMAT designation by providing preliminary clinical evidence that the therapy has the potential to address a serious condition with significant unmet medical need. This designation recognizes the promise of OCU410 as a regenerative medicine therapy and supports an expedited development pathway.
In early July 2026, Ocugen reached alignment with the FDA on the design of the OCU410 Phase 3 registrational trial, with study initiation expected in the third quarter of 2026, and a Biologics License Application (BLA) filing anticipated in 2028.
About Regenerative Medicine Advanced Therapy (RMAT) Designation
The FDA established the RMAT designation to expedite the development and review of regenerative medicine therapies intended to treat, modify, reverse, or cure serious or life-threatening diseases or conditions. RMAT designation is granted to investigational regenerative medicine therapies supported by preliminary clinical evidence indicating the potential to address unmet medical needs. Products receiving RMAT designation are eligible for all the benefits of the Fast Track and Breakthrough Therapy programs, including increased interactions with the FDA to support efficient development, eligibility for rolling BLA review, and the potential for accelerated approval and Priority Review, where appropriate.
About Dry Age-Related Macular Degeneration (dAMD) and Geographic Atrophy (GA)
Geographic atrophy is an advanced form of dAMD characterized by progressive degeneration of the macula, leading to irreversible central vision loss. Millions of patients worldwide are affected by GA, with a particularly high burden in aging populations in the United States and Europe. Despite recent approvals, treatment options remain limited and require chronic intravitreal injections, underscoring the need for innovative, durable therapies that address multiple disease mechanisms. dAMD affects approximately 10 million Americans and more than 266 million people worldwide. It is characterized by the thinning of the macula, the portion of the retina responsible for clear vision in one’s direct line of sight. dAMD involves the slow deterioration of the retina with submacular drusen (small white or yellow dots on the retina), atrophy, loss of macular function, and central vision impairment. dAMD accounts for 85-90% of all AMD cases.
About OCU410
OCU410 is an investigational, subretinal injection, AAV5-based gene therapy that delivers RORA (retinoid-related orphan receptor alpha), a nuclear receptor that regulates key pathways involved in retinal homeostasis, including oxidative stress response, complement regulation, inflammation, and lipid metabolism. OCU410 is being developed as a one-time gene therapy for patients with GA secondary to dAMD. OCU410 received Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency.
About Ocugen, Inc.
Ocugen, Inc. is a pioneering biotechnology company developing gene therapies for blindness. The Company’s breakthrough modifier gene therapy platform has the potential to address significant unmet medical needs across large patient populations through a gene-agnostic approach. Unlike traditional gene therapies and gene-editing technologies that target a single gene mutation, Ocugen’s modifier gene therapies are designed to address the underlying disease biology by restoring balance across multiple gene networks. The Company is currently advancing programs for inherited retinal diseases and other causes of blindness that affect millions worldwide, including retinitis pigmentosa, Stargardt disease, and geographic atrophy, an advanced form of dry age-related macular degeneration. Discover more at www.ocugen.com and follow us on LinkedIn and X.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, including the timing of enrollment and data readouts, the ability to initiate new clinical programs, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, statements regarding potential market size and commercial possibilities of Ocugen’s product candidates, 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 convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that receipt of RMAT designation may not lead to faster development or regulatory review; that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing may not be predictive of the results or success of later clinical trials; and that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our annual and quarterly filings 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. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.
Ocugen podepsal závazný term sheet s Roots Pharmaceutical pro exkluzivní licenci na OCU400 v regionu MENA. Firma může získat až 4 miliony USD předem a krátkodobě, plus až 255 milionů USD na milnících a 22% royalty z čistých tržeb.
Cumulative sales milestones up to $255 million and modest upfront/near-term development milestonesRoyalties equaling 22% of net salesOcugen to manufacture and supply OCU400 MALVERN, Pa., July 13, 2026 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced the signing of a binding term sheet to negotiate and enter into a license agreement with Roots Pharmaceutical, and its strategic partner Al-Dhow International Holding, for the exclusive rights to OCU400, Ocugen's novel modifier gene therapy for Retinitis Pigmentosa (RP), in the Middle East and North Africa (MENA) region.
Pursuant to the term sheet, under the license agreement, Ocugen is expected to receive upfront license fees and near-term development milestone payments totaling up to $4 million. The Company would be entitled to sales milestone payments up to $255 million, in addition to a 22% royalty on net sales of OCU400 generated by Ocugen's partner. Additionally, Ocugen would manufacture commercial supply of OCU400 under the terms of a related supply agreement.
RP is a leading cause of inherited vision loss globally, with notable prevalence across the MENA region, underscoring the significant unmet need OCU400 is positioned to address through this partnership.
"This step forward represents an important milestone in our effort to advance OCU400 regional partnership strategy," said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. "By partnering with an established leader with strong reach across the Middle East and North Africa, we are expanding our ability to bring this one-time potential treatment for life to a region where RP is highly prevalent with a significant unmet medical need where patients are desperately looking for rescue from blindness. This agreement underscores the momentum behind OCU400 and our continued commitment to patients."
“Bringing innovative gene therapies to patients across the MENA region is a strategic imperative for Roots Pharmaceutical and its strategic partner Al-Dhow International Holding,” said Dr. Islam Zayed, CEO & Co founder of Roots Pharmaceutical. Dr.Zayed emphasized that “ OCU400 built on our legacy of bringing Rare Disease therapies to patients in MENA and enables our combined teams to decrease disease burden in the region. Importantly, Roots is dedicated to bringing OCU400 to patients with Retinitis Pigmentosa and creating a new treatment paradigm. We are excited to partner with the Ocugen team.”
Additional details will be available once the definitive agreement between the parties is executed, which is expected to occur within the next 90 days.
Ocugen continues to advance OCU400 through its Phase 3 liMeliGhT clinical development with a topline readout expected in 1Q 2027 and BLA submission to follow.
About Ocugen, Inc.
Ocugen, Inc. is a pioneering biotechnology leader in gene therapies for blindness diseases. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach. Unlike traditional gene therapies and gene editing, Ocugen’s modifier gene therapies address the entire disease—complex diseases that are potentially caused by imbalances in multiple gene networks. Currently we have programs in development for inherited retinal diseases and blindness diseases affecting millions across the globe, including retinitis pigmentosa, Stargardt disease, and geographic atrophy—late-stage dry age-related macular degeneration. Discover more at www.ocugen.com and follow us on X and LinkedIn.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the terms of the definitive license and supply agreement with Roots Pharmaceutical, the timing of entering into such definitive agreement or whether such definitive agreement will be executed at all, the anticipated benefits to Ocugen of such definitive agreement, qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, 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 convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that the definitive license and supply agreement with Roots Pharmaceutical will be delayed or not executed at all, or that, if executed, it will not be on terms described above, the risk that such definitive agreement, if executed, will not lead to the currently anticipated benefits to Ocugen, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings 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. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.
Ocugen plánuje příští rok podat žádost o BLA pro retinitis pigmentosa a Stargardtovu chorobu, zatímco třetí žádost pro geografickou atrofii cílí na rok 2028.
Firma má všechny tři programy v pozdní fázi vývoje.
Ocugen NASDAQ: OCGN outlined plans to advance three late-stage retinal disease programs, with company speaker Shankar saying the company is targeting biologics license applications for retinitis pigmentosa and Stargardt disease next year and a third BLA for geographic atrophy in 2028.
Speaking during a Piper Sandler discussion with analyst Biren Amin, Shankar said Ocugen is focused on inherited retinal diseases and dry age-related macular degeneration, including retinitis pigmentosa, Stargardt disease and geographic atrophy, the late form of dry AMD. He cited more than 100,000 retinitis pigmentosa patients in the U.S., about 50,000 Stargardt disease patients in the U.S. and 2 million to 3 million geographic atrophy patients in the U.S. and EU in the late stage of disease.
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Shankar said Ocugen has retinitis pigmentosa and Stargardt programs in Phase 3 and expects to begin a global Phase 3 program for geographic atrophy in the third quarter, with alignment from regulators in the U.S. and Europe.
OCU410 Phase 3 Plans for Geographic Atrophy Amin began by asking about OCU410, Ocugen’s gene therapy candidate for geographic atrophy. Shankar said the therapy uses a modified RORA gene, which Ocugen is also using in its Stargardt program. He said RORA is intended to regulate multiple pathways involved in disease progression, including oxidative stress, lipid metabolism, inflammation and the complement system.
Shankar contrasted the approach with currently approved therapies that target the complement system, saying Ocugen believes RORA could help “reset” cellular homeostasis and create a healthier environment for photoreceptors and retinal pigment epithelial cells. He said Phase 2 data showed treatment benefit in one year, while many trials in the disease area run for at least two years.
Discussing Phase 2 results, Shankar said the medium dose showed about a 33% reduction in lesion growth when using the square root measure, while the company observed a bell-shaped dose-response pattern rather than a linear one. He said the high dose did not perform as consistently as the medium dose, leading Ocugen to select the medium dose for Phase 3.
For the pivotal study, Shankar said Ocugen plans to use a baseline lesion size range of 2.5 mm squared to 17.5 mm squared, consistent with approved products, and will measure the rate of change in lesion size at four-month intervals through 12 months. He said the company expects to enroll fewer than 300 patients and plans an adaptive design in which a data monitoring committee may review the trial after 50% of patients complete one year.
Shankar said Ocugen designed the trial assuming an effect size lower than the Phase 2 result, using roughly 25% to build in a buffer. Secondary endpoints will include ellipsoid zone preservation and low-luminance visual acuity. He said the medium dose showed about 27% preservation of ellipsoid zone loss in Phase 2, and the company hopes to see results in the 20%-plus range.
On safety, Shankar said there were no serious adverse events related to the drug in Phase 2, though some surgery-related issues can occur with vitrectomy and most resolved.
Stargardt Program OCU410ST Amin also asked about OCU410ST, Ocugen’s program for Stargardt disease. Shankar said Phase 1 results from the GARDian1 trial showed a 54% reduction in lesion growth in evaluable subjects compared with untreated eyes, using the contralateral eye as a control. He said the result informed the design of the Phase 2/3 pivotal study, which included 51 planned subjects, with 34 treated and 17 untreated.
Shankar said the company is also monitoring ellipsoid zone and low-luminance visual acuity as secondary measures. He said the program is intended as a one-time therapy and is being studied across a broad range of patients, including pediatric and adult patients aged three and older and patients from early to advanced stages of disease.
The GARDian3 pivotal trial includes an adaptive sample size re-estimation, which Shankar said is expected this quarter. He said the data monitoring committee could recommend no change, an adjustment in sample size or an extension of follow-up from 12 months to 16 months. Ocugen over-recruited the study to 63 patients, he said, potentially reducing the need for additional enrollment if the committee recommends a larger sample.
If no changes are made, Shankar said top-line results are expected in the second quarter of next year, followed by a BLA filing “a few weeks after that” or around mid-next year. If the timeline is extended, he said any delay could be roughly four to six months and still could allow the company to reach year-end next year. He also said no serious adverse events related to the drug have been observed so far in the Stargardt program.
OCU400 for Retinitis Pigmentosa Ocugen’s third program, OCU400, targets retinitis pigmentosa. Shankar said enrollment is complete in the Phase 3 trial, with top-line data expected in the first half of next year. He described the trial as a large genetic medicine study with 140 patients and more than 25 genetic mutations represented.
Shankar said OCU400 uses NR2E3 delivered through an AAV vector and is designed to be gene-agnostic by upregulating key transcription factors and helping reset cellular function. He said the trial includes a 2:1 randomization ratio, with more patients in the treatment arm than the control arm, and covers a broad population that includes syndromic and non-syndromic retinitis pigmentosa.
The primary functional assessment will use a Luminance Dependent Navigation Assessment, which Shankar said is similar to the multi-luminance mobility test used in the approval of Luxturna but modified to be more specific and sensitive. He said the trial will compare the rate of improvement over one year against the control arm.
Upcoming Milestones Shankar said Ocugen is aiming for two top-line readouts next year from its retinitis pigmentosa and Stargardt programs, followed by a third program readout in geographic atrophy in 2028. He reiterated the company’s goal of filing three BLAs over the next two years, followed by marketing authorization applications and potential global approvals.
“We’re going after with our one-time potential treatments, targeting hundreds of thousands to millions,” Shankar said, describing that as a key point of differentiation for the company.
About Ocugen NASDAQ: OCGNOcugen Inc is a clinical-stage biopharmaceutical company focused on discovering, developing and commercializing gene therapies to treat rare inherited retinal diseases, as well as vaccines designed to address unmet needs in infectious diseases. Headquartered in Malvern, Pennsylvania, the company applies its proprietary gene therapy platform to create novel treatments aimed at preserving and restoring vision, while leveraging strategic partnerships to broaden its vaccine pipeline.
In its gene therapy portfolio, Ocugen is advancing multiple programs targeting retinal disorders.
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