Primary-source record
Decision-gradeTopline results
OCGN: OCU400 Phase 3 liMeliGhT enrollment complete; topline Q1 2027
Enrollment is now complete for the OCU400 Phase 3 liMeliGhT trial in broad retinitis pigmentosa. Topline data are expected in Q1 2027, supporting a rolling BLA filing targeted for Q3 2026 and potential approval in 2027. The trial is the largest known Phase 3 orphan gene therapy study and the only broad RP gene-agnostic registrational trial.
Key facts
- Filed
- Mar 4, 2026, 04:07 PM
- Event
- Topline results
- Direction
- Neutral
- Company
OCGNOcugen- Asset
- OCU400
- Source
- SEC 8-K
- Confidence
- Sourced
- dose
- 2.5×10^10 vg per eye 250 µL
- randomization
- 2:1
- primary endpoint
- 12-month change in visual function assessed by LDNA
- phase3 enrollment
- 140 patients
Source excerpt
EX-99.1 2 tm267731d1_ex99-1.htm EXHIBIT 99.1 Exhibit 99.1 Ocugen Provides Business Update with Fourth Quarter and Full Year 2025 Financial Results Conference Call and Webcast Today at 8:30 a.m. ET · 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 4, 2026 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: 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 financi
Oct 15, 2026
12:01 PM
OCGNOcugenOcugen Updates OCU400 Phase 3 RP Trial Design, Endpoints and Timelines