-- Enrollment for the OCU400 Phase 3 liMeliGhT clinical trial--the first and
largest gene therapy registrational trial for broad retinitis pigmentosa
patients--was completed. Topline Phase 3 data expected in the first
quarter 2027, advancing OCU400 towards potential approval in 2027.
-- OCU410ST Phase 2/3 pivotal confirmatory trial nearing enrollment
completion. Interim data expected in the third quarter 2026, followed by
topline Phase 2/3 data in the second quarter 2027 in advance of the BLA
submission.
-- OCU410 positive preliminary Phase 2 data announced in January. Full Phase
2 data expected in March 2026.
-- First regional licensing agreement for OCU400 in 2025 initiates strategic
partnership strategy ahead of commercialization
-- Rounded out executive leadership team with top talent in business
development, commercial, finance, and operations to encompass all
required expertise for upcoming growth
MALVERN, Pa., March 04, 2026 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) $(OCGN)$, a pioneering biotechnology leader in gene therapies for blindness diseases, today reported fourth quarter and full year 2025 financial results along with a general business update.
"Considerable development across all our modifier gene therapy programs, notable licensing and financing agreements to strengthen our financial position, and meaningful appointments to our leadership team made 2025 a transformative year for Ocugen," said Dr. Shankar Musunuri, Chairman, CEO, Co-founder of Ocugen. "We are poised to leverage upcoming catalysts and advance the business as we near the first of our three BLA filings."
Enrollment is now complete for the OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP). As a one-year clinical trial, topline data will be available in the first quarter of 2027. These data are anticipated to support the Biologics License Application (BLA) filing for OCU400 and potential approval in 2027. The liMeliGhT clinical trial enrolled 140 patients who were randomized 2:1 into the treatment group (2.5× vg per eye 250 uL) and untreated control group across mutations (RHO and gene-agnostic arms). The target population included patients with early- to late-stage disease among a broad RP population, including pediatrics (3+ years). The primary endpoint is 12-month change in visual function assessed by LDNA (luminance dependent navigation assessment) with improvement in Lux Level from baseline to 12 months. The OCU400 Phase 3 liMeliGhT clinical trial is the only broad RP gene-agnostic trial and the largest known Phase 3 orphan gene therapy trial.
The OCU410ST Phase 2/3 GARDian clinical trial for Stargardt disease (ST) remains ahead of schedule in preparation for the 2027 BLA filing. In January, the Company announced publication of Phase 1 GARDian1 trial results for OCU410ST in EYE. The study supports the favorable safety, tolerability and efficacy profile of OCU410ST and its potential to provide clinically meaningful functional and structural benefits in ST patients. OCU410ST holds the potential to address the unmet medical need that remains for approximately 100,000 Stargardt patients in the U.S. and Europe who have no treatment option available.
Recently, Ocugen 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). Key findings from Phase 2 include 46% lesion growth reduction (medium + high dose vs. control; p=0.015; N=23) at 12 months and 50% responder rate with patients achieving >50% lesion size reduction vs. control. A subgroup analysis of patients with a baseline GA size >=7.5 mm(2)--representing advanced atrophy--demonstrated a 57% reduction in lesion growth in treated eyes for medium dose and a 56% reduction in high dose compared with control eyes. This reduction in lesion size in medium and high doses suggests OCU410 may be more effective in patients with substantial disease burden.
The latest OCU410 data set also included encouraging 12-month Phase 1 findings where OCU410-treated eyes demonstrated 60% slower loss of the ellipsoid zone (a structural and functional exploratory endpoint) compared to untreated fellow eyes. The 60% reduction in ellipsoid zone (EZ) loss rate indicates that OCU410 treatment is substantially slowing the rate of photoreceptor degeneration compared to the natural history observed in the untreated fellow eye of the same patient.
"With approximately 2 to 3 million GA patients in the U.S. and Europe combined, OCU410 represents a significant market opportunity. Current therapies have notable limitations, and there are no treatments approved for GA in Europe, as existing FDA-approved options fail to demonstrate meaningful functional outcomes," said Dr. Musunuri. "OCU410 is therefore well-positioned to address this critical unmet need, and we look forward to reporting full data from the OCU410 Phase 2 clinical trial this month and initiating Phase 3 in 2026."
The licensing agreement with Kwangdong Pharmaceutical, Co., Ltd. for the exclusive Korean rights to OCU400--with upfront fees and near-term development milestone payments, along with royalties--was a critical step in Ocugen's business development strategy, affirming a regional partnership approach for OCU400 that preserves the Company's rights to larger geographies while also generating a potential return for shareholders.
To extend the cash runway into the fourth quarter of 2026, in January 2026 the Company secured $22.5 million in gross proceeds through an underwritten registered direct offering of common stock led by RTW Investments, with additional participation from new and existing investors. This raise follows the $20 million registered direct offering of common stock and warrants with Janus Henderson Investors in August 2025. The Company may receive up to $30 million of additional gross proceeds from the August 2025 registered direct offering if the warrants are exercised in full.
"I am proud of our accomplishments in 2025, as they accelerate our drive to achieve even more significant clinical and pre-commercial objectives in 2026," said Dr. Musunuri. "With a full bench of experienced leadership across the organization, I am confident that we have the resources and know-how to take Ocugen to the next level."
Business Updates
Novel Modifier Gene Therapy Platform--Targeting Three BLA Filings in the Next Three Years
-- OCU400 -- Completed enrollment in the Phase 3 liMeliGhT clinical trial
for OCU400 and are on track to file the rolling BLA in the third quarter
of 2026. Subjects will be followed for a year after dosing for primary
endpoint analyses. Positive long-term, 3-year Phase 1/2 durable, safety
and tolerability data demonstrates sustained clinically meaningful,
approximately 2-line LLVA gain, reinforcing durable gene-agnostic
benefit.
-- OCU410ST -- The Phase 2/3 GARDian3 pivotal confirmatory trial is
progressing ahead of schedule with anticipated enrollment completion in
the first quarter of 2026. Interim data is expected in the third quarter
of 2026.
-- OCU410 -- In January 2026, Ocugen announced positive preliminary 12-month
data for Phase 2 subjects from the ArMaDa clinical trial for GA secondary
to dAMD. The complete data set for the ArMaDa trial is expected to be
available in March 2026.
Other Programs
-- OCU200 -- No serious adverse events (SAEs) or adverse events (AEs)
related to OCU200 reported to date across the dose-escalation cohorts and
trial enrollment is expected to be completed by the first quarter of
2026.
-- OCU500 -- NIAID intends to initiate the OCU500 Phase 1 clinical trial in
the second quarter of 2026.
-- NeoCart -- Created OrthoCellix as a wholly-owned subsidiary of Ocugen for
the regenerative cell therapy assets with a goal of obtaining independent
financing.
Financial Results
-- Fourth quarter -- Research and development expenses for the three months
ended December 31, 2025, were $10.7 million compared to $8.3 million for
the three months ended December 31, 2024. General and administrative
expenses for the three months ended December 31, 2025, were $6.1 million
compared to $6.3 million for the three months ended December 31, 2024.
Ocugen reported a $0.06 net loss per common share for the three months
ended December 31, 2025, compared to a $0.05 net loss per common share
for the three months ended December 31, 2024.
-- Full year -- Research and development expenses for the year ended
December 31, 2025, were $39.8 million compared to $32.1 million for the
year ended December 31, 2024. General and administrative expenses for the
year ended December 31, 2025, were $27.6 million compared to $26.7
million for the year ended December 31, 2024. Ocugen reported a $0.23 net
loss per common share for the year ended December 31, 2025, compared to a
$0.20 net loss per common share for the year ended December 31, 2024.
-- Ocugen's cash and restricted cash, totalled $18.9 million as of December
31, 2025, compared to $58.8 million as of December 31, 2024. The Company
estimates that additional proceeds from the $22.5 million financing in
January 2026 will enable it to fund its operations into the fourth
quarter of 2026. If the Janus Henderson warrants are fully exercised this
year, it is expected that cash runway will be extended into the second
quarter of 2027. The Company had 312.4 million shares of common stock
outstanding as of December 31, 2025.
Conference Call and Webcast Details
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