Ocugen Announces Positive Preliminary Phase 2 Data from OCU410 Modifier Gene Therapy for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration

Rhea-AI Summary

Ocugen (NASDAQ: OCGN) reported positive preliminary 12-month data from the Phase 2 ArMaDa trial of OCU410 (AAV5-RORA) for geographic atrophy (GA) secondary to dry AMD.

Key 12-month results: 46% lesion growth reduction (medium+high dose vs control; p=0.015; N=23), 54% reduction for medium dose (p=0.02; N=10), a 50% responder rate (>50% lesion reduction vs control), and subgroup (N=14) showing 57% greater reduction. Phase 1 evaluable eyes (N=7) showed 60% slower EZ loss versus fellow eyes. No OCU410-related serious adverse events reported across Phase 1 and Phase 2 (60 patients). Company aims to start Phase 3 in 2026 and pursue a BLA in 2028.

Positive

• 46% lesion growth reduction vs control at 12 months (p=0.015; N=23)
• 54% lesion reduction with medium dose at 12 months (p=0.02; N=10)
• 60% slower ellipsoid zone loss in Phase 1 evaluable eyes (N=7)
• No OCU410-related serious adverse events reported across Phase 1/2 (60 patients)

Negative

• Efficacy analysis based on small cohorts (e.g., N=23 overall)
• Results preliminary (~50% of patients evaluated at 12 months)
• Control group remained untreated (no sham control reported)

News Market Reaction

-13.83% 2.3x vol

27 alerts

-13.83% News Effect

+4.6% Peak Tracked

-9.9% Trough Tracked

-$92M Valuation Impact

$575M Market Cap

2.3x Rel. Volume

On the day this news was published, OCGN declined 13.83%, reflecting a significant negative market reaction. Argus tracked a peak move of +4.6% during that session. Argus tracked a trough of -9.9% from its starting point during tracking. Our momentum scanner triggered 27 alerts that day, indicating elevated trading interest and price volatility. This price movement removed approximately $92M from the company's valuation, bringing the market cap to $575M at that time. Trading volume was elevated at 2.3x the daily average, suggesting increased selling activity.

Data tracked by StockTitan Argus on the day of publication.

Key Figures

Lesion growth reduction: 46% Medium-dose lesion reduction: 54% High-dose lesion reduction: 36% +5 more

8 metrics

Lesion growth reduction 46% Phase 2, 12‑month lesion growth reduction vs. control (medium + high dose; N=23; p=0.015)

Medium-dose lesion reduction 54% Phase 2, medium dose lesion reduction vs. control (p=0.02; N=10)

High-dose lesion reduction 36% Phase 2, high dose lesion reduction vs. control (p=0.05; N=8)

Responder rate 50% Phase 2 patients achieving >50% lesion size reduction vs. control

Subgroup lesion reduction 57% Phase 2 subgroup (N=14, baseline ≥7.5 mm²) greater lesion-size reduction vs. control

Slower EZ loss 60% Phase 1 evaluable subjects (N=7) slower ellipsoid zone loss vs. fellow eyes at 12 months

ArMaDa randomization 51 patients Phase 2 GA trial randomized 1:1:1 to medium dose, high dose, or control

Patients in Phase 1/2 60 patients Phase 1 and Phase 2 clinical trials with no OCU410-related serious adverse events to date

Market Reality Check

Price: $1.49 Vol: Volume 6,964,922 is 1.36x...

normal vol

$1.49 Last Close

Volume Volume 6,964,922 is 1.36x the 20-day average of 5,129,173, indicating elevated pre-news activity. normal

Technical Price $1.88 is trading above the 200-day MA at $1.14, near the 52-week high of $1.96 and well above the $0.515 low.

Peers on Argus

OCGN was down 1.57% while key peers showed mostly gains: FDMT +10.89%, MBX +18.4...

OCGN was down 1.57% while key peers showed mostly gains: FDMT +10.89%, MBX +18.4%, DSGN +2.4%, LRMR +3.02%, with only BNTC -0.82%. This points to stock-specific dynamics rather than a broad sector move.

Historical Context

5 past events · Latest: Jan 13 (Positive)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Jan 13	OCU410 data webcast	Positive	+7.3%	Announced webcast to discuss upcoming OCU410 Phase 2 one-year data.
Jan 12	OCU410ST Phase 1 data	Positive	+18.7%	Published peer-reviewed Phase 1 GARDian1 results with strong efficacy and safety.
Jan 09	JPM conference plans	Positive	+0.7%	Detailed JPM Healthcare Conference presentation and path to three BLAs.
Dec 10	Oppenheimer summit talk	Neutral	+1.6%	CEO presentation on gene-modifier platform and 2026 catalysts at summit.
Nov 20	NobleCon presentation	Neutral	-2.6%	Conference appearance and strategy update on three BLAs in three years.

Pattern Detected

Recent clinical and corporate updates have more often seen positive price alignment, with only one notable divergence in the last five events.

Recent Company History

Over the last six months, Ocugen has steadily highlighted its modifier gene therapy platform. On Feb 12, 2025, completion of dosing in the Phase 2 ArMaDa trial for OCU410 and a 44% slower lesion growth metric preceded today’s more mature OCU410 data. Subsequent clinical readouts for OCU200 and OCU410ST, plus plans for three BLAs in three years, underscored a broad retinal pipeline. Two recent clinical‑trial headlines in Jan 2026 produced moves of 7.3% and 18.67%, framing today’s Phase 2 OCU410 update as part of a continuing data‑driven story.

Market Pulse Summary

The stock dropped -13.8% in the session following this news. A negative reaction despite positive pr...

Analysis

The stock dropped -13.8% in the session following this news. A negative reaction despite positive preliminary data would fit a pattern where balance sheet risk tempers enthusiasm. The Phase 2 results show a 46% lesion growth reduction and no drug‑related serious adverse events in 60 patients, but prior filings highlighted funding needs and going‑concern language. Historically, most clinical updates have aligned with gains, so a sharp decline could reflect profit‑taking or renewed focus on capital requirements rather than the trial outcomes alone.

Key Terms

geographic atrophy, dry age-related macular degeneration, ellipsoid zone, intravitreal injections, +4 more

8 terms

geographic atrophy medical

"modifier gene therapy for geographic atrophy (GA) secondary to dry age-related"

Geographic atrophy is a progressive eye condition in which patches of light-sensing cells in the retina die, causing growing blind spots and ultimately significant central vision loss. For investors, it matters because the condition defines the market size and urgency for drugs, devices, and diagnostics — like a spreading hole in a photograph that companies aim to stop or repair — so clinical results, approvals, and reimbursement determine potential revenue and risk.

dry age-related macular degeneration medical

"modifier gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD)"

Dry age-related macular degeneration is a progressive eye disease in which the central part of the retina (the macula) slowly loses light-sensing cells, leading to gradual blurring or loss of central vision like a camera with a worn-out sensor. It matters to investors because the condition affects a large and growing population, creates demand for diagnostics, treatments and long-term care, and clinical or regulatory developments can significantly influence companies' revenues and stock prices.

ellipsoid zone medical

"ellipsoid zone (EZ) loss was 60% slower in OCU410-treated eyes compared"

A bright, thin layer seen on a retinal scan (OCT) that represents part of the light-sensing cells in the eye; it acts like an indicator light for the cells that convert light into vision. Its integrity correlates with how well someone can see, so changes are used as objective biomarkers in clinical trials and regulatory assessments and matter to investors because they help predict treatment effectiveness and market value of eye therapies.

intravitreal injections medical

"current therapies involve frequent (monthly or every other month) injections and have unwanted"

An intravitreal injection is a medical procedure that delivers medication directly into the eye’s vitreous, the clear gel at the back of the eyeball, to treat retinal and other internal eye conditions. For investors it matters because these injections create recurring demand for specialty drugs, delivery tools and clinic services; treatment frequency, safety and reimbursement determine revenue size and predictability, similar to a subscription product for vision care.

subretinal medical

"subjects received a single subretinal 200-µL administration of 5 x 1010 vector genomes"

Subretinal describes the space or actions beneath the retina, the thin light-sensing tissue at the back of the eye. It is often used to describe injections, implants, or surgical procedures that place medicine or devices directly under that layer — imagine slipping a tiny patch beneath wallpaper to treat the wall. For investors, subretinal approaches matter because they influence a treatment’s effectiveness, safety profile, surgical complexity and regulatory scrutiny, all of which affect commercial potential.

vector genomes medical

"single subretinal 200-µL administration of 5 x 1010 vector genomes (vg)/mL (medium dose)"

Vector genomes are the genetic payload carried inside a delivery vehicle (often a harmless virus) used to insert specific genes into cells for therapies or vaccines. Think of the vector as a delivery truck and the genome as the package it drops off; the exact contents determine how well a treatment works, how long it lasts, and the safety profile. Investors watch vector genomes because their design and quality drive clinical success, manufacturing complexity, regulatory approval, and therefore the commercial value of biotech programs.

biologics license application regulatory

"The Company remains on track for a Biologics License Application (BLA) filing for OCU410 in 2028"

A biologics license application is a formal request submitted to regulatory authorities seeking approval to market a new biological medicine, such as vaccines or treatments made from living organisms. It is a comprehensive review process that evaluates the safety, effectiveness, and manufacturing quality of the product. For investors, receiving approval signals that a biological therapy can be sold to the public, potentially leading to revenue growth and market success.

vasculitis medical

"no cases of endophthalmitis, retinal detachment, vasculitis, choroidal neovascularization, or optic"

Vasculitis is inflammation and injury of blood vessels that can narrow, block, or cause leaks in the body's circulation; think of blood vessels like pipes that swell or crack, reducing flow and damaging nearby organs. It matters to investors because the condition drives demand for specific treatments, shapes clinical trial success and regulatory decisions, and can influence a healthcare company's revenue, liability risk, and valuation.

AI-generated analysis. Not financial advice.

• Phase 2 (~50% of patients evaluated to date at 12 months) shows 46% lesion growth reduction vs. control
• There are no OCU410-related serious adverse events reported across the Phase 1 and Phase 2 clinical trials to date

MALVERN, Pa., Jan. 15, 2026 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced positive preliminary 12-month data (~50% of patients evaluated to date) from the Phase 2 ArMaDa clinical trial evaluating OCU410 (AAV5-RORA), its novel modifier gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). The global prevalence of dAMD is 266 million worldwide, and GA – the late stage of dAMD – affects approximately 2-3 million people in the United States (U.S.) and Europe.

There are limited options for patients with dAMD in the U.S. and current therapies involve frequent (monthly or every other month) injections and have unwanted side effects that can affect vision. Outside of the U.S., there are no approved products available, leaving approximately 2 million patients in Europe without a treatment option.

Key findings from Phase 2 include:

• 46% lesion growth reduction (medium + high dose vs. control; p=0.015; N=23) at 12 months
• Medium dose achieved 54% lesion reduction (p=0.02; N=10) vs. high dose 36% (p=0.05; N=8) compared to control
• 50% responder rate with patients achieving >50% lesion size reduction vs. control
• Subgroup (N=14, subjects with ≥7.5 mm² at baseline) showed 57% greater reduction in lesion size compared to control

New findings from Phase 1 (N=9) include:

• In evaluable subjects (N=7) ellipsoid zone (EZ) loss was 60% slower in OCU410-treated eyes compared to untreated fellow eyes at 12 months
• EZ-RPE complex loss reduced in treated eyes versus fellow eyes, demonstrating photoreceptor + RPE preservation

In both the Phase 1 and Phase 2 clinical trials no OCU410-related serious adverse events were observed and no cases of endophthalmitis, retinal detachment, vasculitis, choroidal neovascularization, or optic ischemic neuropathy have been reported to date.

GA is a multifactorial disease with a complex etiology that involves genetic and environmental factors. The current treatment options for GA in the U.S. are limited to those targeting a single mechanism—the complement pathway—requiring frequent intravitreal injections, either monthly or every other month. By contrast, OCU410 is a multifunctional modifier gene therapy, which targets multiple pathways associated with GA.

"The OCU410 Phase 1 and Phase 2 results mark a pivotal moment for Ocugen's modifier gene therapy platform and GA patients worldwide," said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. "Delivering 60% slower EZ loss in Phase 1 and 46% lesion growth reduction in the Phase 2 preliminary analysis demonstrates the capability of our multi-pathway RORA approach. We look forward to reporting full data from the OCU410 Phase 2 clinical trial later this quarter and initiating Phase 3 in 2026.”

"The clinical development journey of OCU410 has been remarkable," said Dr. Huma Qamar, Chief Medical Officer of Ocugen. "Our Phase 2 randomized trial delivered robust anatomic efficacy that was statistically significant across multiple analyses. Critically, our safety data across 60 patients has shown no drug-related serious adverse events, no inflammation signals, and no injection complications to date, supporting a favorable risk-benefit profile."

"As a practicing retinal specialist, OCU410's clinical profile is genuinely exciting for geographic atrophy patients—including a reduction in ellipsoid zone loss observed in Phase 1, which may serve as a potential marker of retinal health, and a reduction in lesion growth seen in Phase 2," said Lejla Vajzovic, MD, FASRS, Director, Duke Surgical Vitreoretinal Fellowship Program, Professor of Ophthalmology with Tenure, Adult and Pediatric Vitreoretinal Surgery and Disease, Duke University Eye Center, and Retina Scientific Advisory Board Chair of Ocugen. "With these promising results, I believe OCU410 has the potential to set a new standard of care with a single treatment for life."

In the Phase 2 study, the safety and efficacy of OCU410 in patients with GA secondary to dAMD are being assessed. Fifty-one (51) patients were randomized 1:1:1 into either of two treatment groups (medium or high dose) or a control group. In the treatment groups, subjects received a single subretinal 200-µL administration of 5 x 10¹⁰ vector genomes (vg)/mL (medium dose) or 1.5 x 10¹¹ vg/mL (high dose), while the control group remained untreated. The Company remains on track for a Biologics License Application (BLA) filing for OCU410 in 2028, aligned with its strategy to advance three regulatory submissions for marketing authorization in three years.

About dAMD and Geographic Atrophy
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, intravitreally administered, 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 dry AMD. OCU410 has received Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene therapies to address major blindness diseases and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach. 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 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 preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; the ability of OCU410 to perform in humans in a manner consistent with nonclinical, preclinical or previous clinical study 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 of 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.

Contact:
Tiffany Hamilton
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com

FAQ

What were the primary 12-month efficacy results for OCU410 in the Phase 2 ArMaDa trial (OCGN)?

The Phase 2 preliminary 12-month analysis showed 46% lesion growth reduction for medium+high dose vs control (p=0.015; N=23).

How did the medium and high doses of OCU410 compare at 12 months in the ArMaDa study?

The medium dose showed 54% lesion reduction (p=0.02; N=10) and the high dose 36% reduction (p=0.05; N=8) versus control.

What safety events were reported for OCU410 across Phase 1 and Phase 2?

Across Phase 1 and Phase 2 (60 patients), no OCU410-related serious adverse events and no reported endophthalmitis, retinal detachment, vasculitis, choroidal neovascularization, or optic ischemic neuropathy.

What clinical signal was observed in Phase 1 regarding retinal structure?

In Phase 1 evaluable subjects (N=7), treated eyes had 60% slower ellipsoid zone (EZ) loss versus untreated fellow eyes at 12 months.

When does Ocugen plan to start Phase 3 and file a BLA for OCU410 (OCGN)?

Ocugen plans to initiate Phase 3 in 2026 and remains on track for a BLA filing in 2028.

How many patients were randomized in the Phase 2 ArMaDa trial and what were the doses?

Fifty-one patients were randomized 1:1:1; treatment was a single subretinal 200 µL dose of 5×10^10 vg/mL (medium) or 1.5×10^11 vg/mL (high); control remained untreated.