MALVERN, PA — Ocugen, Inc. (Nasdaq: OCGN) announced it has completed enrollment for the Phase 3 liMeliGhT clinical trial evaluating OCU400, a gene therapy candidate for retinitis pigmentosa, a group of inherited retinal disorders that can lead to vision loss.
The one-year trial enrolled 140 patients and is expected to produce topline results in the first quarter of 2027, which the company said could support a biologics license application with the U.S. Food and Drug Administration.
Ocugen said the study includes patients with early- to late-stage disease across multiple genetic mutations associated with retinitis pigmentosa.
Participants were randomized in a 2-to-1 ratio to receive the treatment or remain in an untreated control group.
The trial’s primary endpoint measures changes in visual function using luminance dependent navigation assessment, a mobility test that evaluates a patient’s ability to navigate environments under varying light levels.
Chief Executive Officer Shankar Musunuri said the completion of enrollment marks a key step in the company’s development program.
“With enrollment complete for OCU400, we enter into a very significant time as a company,” Musunuri said.
Chief Medical Officer Huma Qamar said the trial includes a broad range of patients with different genetic mutations linked to retinitis pigmentosa.
“The Phase 3 liMeliGhT clinical trial includes representation of a wide range of gene mutations associated with early to advanced stages of RP,” Qamar said.
Ocugen also reported long-term data from an earlier Phase 1/2 study showing that treated patients maintained improvements in visual function over three years.
According to the company, approximately 88 percent of evaluable patients in the study showed improvement or preservation of vision compared with untreated eyes.
The treatment also showed a sustained improvement of roughly two lines on a low-luminance visual acuity chart across multiple mutation types.
The company said no new treatment-related serious adverse events were reported in the long-term follow-up.
Ocugen said it plans to begin a rolling biologics license application submission in the third quarter of 2026.
The European Medicines Agency has also indicated the U.S.-based trial could be used to support a marketing authorization application in Europe, the company said.
Retinitis pigmentosa is associated with mutations in more than 100 genes and currently has limited treatment options for most patients.
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