MALVERN, Pa. — March 04, 2026 — Leads & Copy — Ocugen, Inc. (NASDAQ: OCGN) has completed enrollment for the OCU400 Phase 3 liMeliGhT clinical trial, the first and largest gene therapy registrational trial for broad retinitis pigmentosa patients. The company expects topline Phase 3 data in the first quarter 2027, advancing OCU400 towards potential approval in 2027.
The OCU410ST Phase 2/3 pivotal confirmatory trial is nearing enrollment completion. Interim data is expected in the third quarter 2026, followed by topline Phase 2/3 data in the second quarter 2027, in advance of the BLA submission.
Ocugen reported fourth quarter and full year 2025 financial results along with a general business update. 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.
The company 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. 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.
The OCU400 Phase 3 liMeliGhT clinical trial enrolled 140 patients who were randomized 2:1 into the treatment group (2.5× vg per eye 250 µL) 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.
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.
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 mm²—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.
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.
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.
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.
Ocugen has scheduled a conference call and webcast for 8:30 a.m. ET today to discuss the financial results and recent business highlights.
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.
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.
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.
Source: Ocugen, Inc.
