Ocugen Announces Publication of Phase 1 GARDian1 Trial Results for OCU410ST Modifier Gene Therapy

Rhea-AI Summary

Ocugen (NASDAQ: OCGN) published peer‑reviewed Phase 1 GARDian1 results for OCU410ST, a modifier gene therapy for Stargardt disease, reporting 12‑month safety, tolerability, and exploratory efficacy data.

Key findings in small Phase 1 cohort: 54% reduction in atrophic lesion growth in treated eyes versus fellow eyes, lesion expansion ~50% slower, treated eyes gained an average of +6 letters BCVA versus −1.5 letters in untreated eyes, 100% of treated eyes stabilized or improved, and no drug‑related serious adverse events. Phase 2/3 GARDian3 enrollment is expected to complete in Q1 2026 with a potential BLA filing in H1 2027.

Positive

• Atrophic lesion growth reduced by 54% in treated eyes over 12 months
• Lesion expansion was 50% slower in treated eyes versus untreated eyes
• Treated eyes gained an average of +6 letters BCVA versus −1.5 letters in controls at 12 months
• 100% of treated eyes stabilized or improved in visual acuity
• No drug‑related serious adverse events reported in the 12‑month follow‑up
• Phase 2/3 enrollment progressing; potential BLA filing H1 2027

Negative

• Efficacy and safety data derive from a very small Phase 1 cohort (metrics reported for as few as six patients), limiting generalizability
• Results are 12‑month exploratory outcomes; long‑term durability and larger‑cohort safety remain unproven

News Market Reaction

+18.67% 2.3x vol

50 alerts

+18.67% News Effect

+24.2% Peak in 26 hr 10 min

+$88M Valuation Impact

$559M Market Cap

2.3x Rel. Volume

On the day this news was published, OCGN gained 18.67%, reflecting a significant positive market reaction. Argus tracked a peak move of +24.2% during that session. Our momentum scanner triggered 50 alerts that day, indicating high trading interest and price volatility. This price movement added approximately $88M to the company's valuation, bringing the market cap to $559M at that time. Trading volume was elevated at 2.3x the daily average, suggesting notable buying interest.

Data tracked by StockTitan Argus on the day of publication.

Key Figures

Lesion growth reduction: 54% reduction (0.55 mm² vs 1.19 mm²) Lesion expansion rate: 0.10 mm/year vs 0.19 mm/year Natural history rates: 0.14–0.18 mm/year +5 more

8 metrics

Lesion growth reduction 54% reduction (0.55 mm² vs 1.19 mm²) Atrophic lesion growth over 12 months in six gradable patients

Lesion expansion rate 0.10 mm/year vs 0.19 mm/year Lesion expansion over 12 months in treated vs untreated eyes

Natural history rates 0.14–0.18 mm/year Published natural history lesion expansion range for comparison

BCVA change +6 letters vs -1.5 letters BCVA change at 12 months in six evaluable patients, treated vs fellow eyes

Visual acuity stability 100% of treated eyes Treated eyes stabilized (±4 letters) or improved (≥5 letters) in visual acuity

Trial duration 12 months Follow-up period for Phase 1 GARDian1 safety and efficacy assessment

GARDian3 enrollment timing Q1 2026 Anticipated Phase 2/3 GARDian3 enrollment completion

Planned BLA timing 1H 2027 Target Biologics License Application filing for OCU410ST

Market Reality Check

Price: $1.41 Vol: Volume 2,804,463 vs 20-da...

low vol

$1.41 Last Close

Volume Volume 2,804,463 vs 20-day average 4,150,713 (relative volume 0.68), showing subdued trading ahead of this news. low

Technical Price 1.50 is trading above the 200-day MA at 1.13, indicating an established uptrend into this announcement.

Peers on Argus

OCGN was up 0.67% pre-news while key biotech peers showed mixed moves: FDMT +1.2...

OCGN was up 0.67% pre-news while key biotech peers showed mixed moves: FDMT +1.21%, MBX +2.26%, DSGN +1.08%, BNTC -3.11%, LRMR -2.06%. No coordinated sector momentum signal.

Historical Context

5 past events · Latest: Dec 10 (Neutral)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Dec 10	Conference presentation	Neutral	+1.6%	CEO presentation at rare disease summit and investor meetings on gene therapy.
Nov 20	Investor conference	Neutral	-2.6%	NobleCon21 appearance and discussions of strategy and three BLAs in three years.
Nov 05	Earnings update	Neutral	-1.4%	Q3 2025 results, financing, cash position, and pipeline progress, including OCU410ST.
Oct 23	Earnings call setup	Neutral	+1.3%	Announcement of webcast to discuss Q3 2025 results and business updates.
Oct 02	Conference participation	Positive	+5.4%	Multiple October 2025 conferences and reiterated goal of three BLAs in three years.

Pattern Detected

Recent non-clinical news (conferences, updates, earnings) tended to produce modest, directionally aligned price moves, with one stronger positive reaction to conference participation.

Recent Company History

Over the past few months, Ocugen highlighted conferences, investor outreach, and Q3 2025 results, alongside financing and progress toward multiple BLAs. Price reactions to these events ranged from about -2.63% to +5.36%, generally aligning with the informational, non-transformational nature of the updates. Today’s Phase 1 GARDian1 publication adds detailed efficacy and safety support for OCU410ST, reinforcing the clinical narrative that previously underpinned the pivotal GARDian3 trial and 1H 2027 BLA timing guidance.

Market Pulse Summary

The stock surged +18.7% in the session following this news. A strong positive reaction aligns with t...

Analysis

The stock surged +18.7% in the session following this news. A strong positive reaction aligns with the clearly favorable Phase 1 GARDian1 results, which showed a 54% reduction in atrophic lesion growth and consistent BCVA gains. Historical clinical updates have produced volatile moves, including sizable drops, so traders may reassess risk once initial enthusiasm fades. The move would be occurring against a backdrop of an uptrend above the 200-day MA and a pipeline strategy targeting a BLA for OCU410ST in 1H 2027.

Key Terms

stargardt disease, fundus autofluorescence, bcva, retinal pigment epithelium, +4 more

8 terms

stargardt disease medical

"modifier gene therapy for Stargardt disease, in the peer-reviewed journal"

A hereditary eye disorder that gradually destroys the macula, the small central part of the retina responsible for sharp, straight‑ahead vision; imagine the center of a camera sensor losing pixels so faces and words become blurred while peripheral sight stays. It matters to investors because it defines the size and urgency of the market for new drugs, gene therapies and diagnostics, shapes clinical trial design and regulatory risk, and influences potential future revenue and valuation for companies developing treatments.

fundus autofluorescence medical

"Among six patients with gradable Fundus Auto Fluorescence images, atrophic lesion"

Fundus autofluorescence is a noninvasive eye imaging method that captures natural glow from molecules in the retina to create a map of tissue health and disease-related changes. For investors, it matters because this objective, repeatable scan is often used in clinical trials and medical monitoring to show whether an eye therapy is slowing damage or improving tissue, much like a before-and-after photo that reveals hidden changes over time.

bcva medical

"Among six BCVA-evaluable patients without confounders, treated eyes gained"

Best corrected visual acuity (BCVA) is the sharpest level of vision a person can achieve when using the optimal prescription lenses during an eye exam, measured by reading standardized letters on a chart. Investors care because BCVA is a common, standardized clinical endpoint in eye‑disease trials and regulatory reviews; improvements in BCVA are used like a speedometer to gauge how well a treatment works and therefore influence a product’s market potential and valuation.

retinal pigment epithelium medical

"lipofuscin accumulation in the retinal pigment epithelium. Currently, no approved"

A thin layer of cells at the back of the eye that acts like a maintenance crew for the light-sensing cells, supplying nutrients, clearing waste, and keeping the retina stable so vision works. It matters to investors because damage or dysfunction of this layer underlies major eye diseases and is a common drug and device target; treatments that protect or restore it can drive clinical trial outcomes, regulatory approvals, and market value.

lipofuscin medical

"vision loss due to photoreceptor degeneration caused by toxic lipofuscin accumulation"

A brownish cellular pigment made of leftover, broken-down fats and proteins that builds up over time inside long-lived cells, acting like microscopic 'rust' from normal wear and tear. For investors, lipofuscin matters because its accumulation is a marker of aging and certain diseases, can interfere with cell function, and influences the development of diagnostics and therapies aimed at age‑related or degenerative conditions.

modifier gene therapy medical

"OCU410ST, its novel modifier gene therapy for Stargardt disease, in the"

Modifier gene therapy is a treatment that changes the activity of genes that influence how severe or how a disease progresses, rather than directly fixing the original faulty gene. Think of it as turning a volume knob on disease symptoms: it can make conditions milder or slow progression, which matters to investors because it can expand the number of patients who benefit, affect how quickly and cheaply a product can reach the market, and change commercial and regulatory risk and reward.

phase 2/3 medical

"These results provide important support for our ongoing Phase 2/3 GARDian3 trial."

A phase 2/3 trial is a combined clinical study that first evaluates how well a treatment works and the best dose, then expands into a larger test to confirm those results and safety. For investors, it matters because moving into a phase 2/3 signals that an experimental therapy has shown initial promise and will be tested at scale, which can materially change the odds and timeline for regulatory approval and commercial potential.

biologics license application (bla) regulatory

"positioned for Biologics License Application (BLA) filing in the first half of 2027"

A biologics license application (BLA) is a formal request to a government agency seeking approval to sell a biological medicine, such as vaccines or gene therapies, in the market. It is similar to a detailed report that proves the product is safe, effective, and manufactured properly. For investors, a BLA signifies a critical step toward commercial availability, often impacting a company's valuation and market prospects.

AI-generated analysis. Not financial advice.

• Study supports favorable safety and tolerability profile and clinically meaningful functional and structural benefits in Stargardt disease patients
  

MALVERN, Pa., Jan. 12, 2026 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced the publication of positive Phase 1 GARDian1 trial results for OCU410ST, its novel modifier gene therapy for Stargardt disease, in the peer-reviewed journal Nature Eye—published by the Royal College of Ophthalmologists.

The publication, A novel modifier gene therapy to treat Stargardt disease: Phase 1 GARDian1 Trial Insights, was authored by Arshad M. Khanani, MD, MA, FASRS, Director of Clinical Research, and Director of Fellowship at Sierra Eye Associates and Clinical Professor at the University of Nevada, Reno School of Medicine; Lejla Vajzovic, MD, FASRS, Professor of Ophthalmology, Director of CME-Ophthalmology, Duke University School of Medicine; Benjamin A. Bakall, MD, PhD. Associated Retina Consultants, President and Founder, Retina Research Foundation of America, Clinical Assistant Professor, University of Arizona College of Medicine; and Ocugen researchers Dr. Murthy Chavali and Dr. Huma Qamar. The publication reports comprehensive 12-month safety, tolerability, and exploratory efficacy data from the first-in-human Phase 1 trial evaluating OCU410ST in patients with early to advanced Stargardt disease.

The Phase 1 GARDian1 trial demonstrated robust efficacy and safety outcomes supporting the clinical development of OCU410ST:

Key Findings include:

• Among six patients with gradable Fundus Auto Fluorescence images, atrophic lesion growth was reduced by 54% (0.55 ± 0.27 mm²) in treated eyes, compared to untreated fellow eyes (1.19 ± 0.31 mm²) over 12 months​
• Lesion expansion was 50% slower (0.10 ± 0.039 mm/year) in treated eyes versus untreated eyes (0.19 ± 0.026 mm/year), below published natural history rates​ (0.14–0.18 mm/year)
• Among six BCVA-evaluable patients without confounders, treated eyes gained +6 letters in BCVA (+4.5 letters) compared to a −1.5 letter decline in untreated fellow eyes at 12​ months
• 100% of treated eyes either stabilized (±4 letters) or improved (≥5 letters) in visual acuity​
• No drug-related serious adverse events or adverse events of special interest were observed​
  

Stargardt disease is the most common form of inherited macular degeneration, affecting more than 100,000 people in the United States and Europe combined. The disease is characterized by progressive central vision loss due to photoreceptor degeneration caused by toxic lipofuscin accumulation in the retinal pigment epithelium. Currently, no approved treatment exists for this devastating condition, representing a critical unmet medical need.

"This publication in Eye validates the scientific approach and clinical promise of OCU410ST as a modifier gene therapy for Stargardt disease," said Dr. Huma Qamar, Chief Medical Officer at Ocugen. "The Phase 1 GARDian1 trial demonstrated convergent functional and structural benefits. This represents a paradigm shift from any other approaches, including oral or mutation-constrained replacement approaches, to an agnostic modification strategy that can potentially benefit patients regardless of their underlying ABCA4 mutation with a potential single gene therapy for life. These results provide important support for our ongoing Phase 2/3 GARDian3 trial."

"The consistent benefits observed across both structural and functional endpoints including slowing atrophic lesion progression and stabilization or improvement in visual acuity highlight the potential of this modifier gene therapy platform approach to transform treatment outcomes for patients with Stargardt disease, who currently have no disease-modifying options available,” said Dr. Arshad M. Khanani, lead author of the publication, Director of Clinical Research at Sierra Eye Associates, and Ocugen Scientific Advisory Board member. “I am looking forward to the data read out from the ongoing Phase 2/3 GARDian3 trial.”

The Phase 2/3 GARDian3 trial is progressing ahead of schedule with anticipated enrollment completion in the first quarter of 2026. The Company remains positioned for Biologics License Application (BLA) filing in the first half of 2027, aligned with its strategy to advance three regulatory submissions in three years.

About OCU410ST
OCU410ST utilizes an AAV5 delivery platform to deliver the RORA (RAR-Related Orphan Receptor A) gene to the retina. By restoring nuclear hormone receptor signaling, OCU410ST addresses pathophysiological pathways linked to Stargardt disease, including lipofuscin formation, oxidative stress, complement activation, inflammation, and photoreceptor survival networks independent of the underlying ABCA4 genotype.

About Stargardt Disease
Stargardt disease type 1 is a genetic eye disorder caused by biallelic mutations in the ABCA4 gene. The condition leads to progressive macular degeneration, with onset typically occurring during childhood or adolescence. Affected patients experience progressive central vision loss while peripheral vision is usually preserved. There are currently no FDA-approved treatments for this orphan indication.

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 OCU410ST 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 did Ocugen (OCGN) report for OCU410ST in the Phase 1 GARDian1 trial?

The Phase 1 trial reported 12‑month data showing 54% reduced atrophic lesion growth, lesion expansion ~50% slower, average +6 letters BCVA gain in treated eyes, and no drug‑related serious adverse events.

How many patients were included in the published OCU410ST Phase 1 efficacy analyses?

Key imaging and visual acuity metrics were reported for small subsets, with efficacy figures referenced for as few as six patients.

When is Ocugen expecting Phase 2/3 GARDian3 enrollment and a potential BLA for OCU410ST?

Phase 2/3 enrollment is anticipated to complete in Q1 2026, with the company positioned for a possible BLA filing in H1 2027.

Did the Phase 1 trial for OCU410ST report any drug‑related serious adverse events?

No drug‑related serious adverse events or adverse events of special interest were observed in the 12‑month follow‑up.

What clinical benefits did OCU410ST show for Stargardt disease patients at 12 months?

Treated eyes showed both structural benefits (slower lesion growth and expansion) and functional benefits (visual acuity stabilization or improvement, including an average +6‑letter gain in evaluable treated eyes).