Ocugen's OCU410 gene therapy achieved a 31% reduction in macular lesion size in patients with geographic atrophy secondary to dry age-related macular degeneration (dAMD) at 12 months, meeting its primary endpoint3
The company plans to launch a Phase 3 trial for OCU410 later in 2026, enrolling up to 300 patients with an adaptive design powered at over 95%3
OCU410 showed no serious adverse events or adverse events of special interest in its Phase 2 trial3
Ocugen completed enrollment for its Phase 3 liMeliGhT trial of OCU400, a modifier gene therapy for retinitis pigmentosa (RP), with topline data expected in Q1 20271
OCU400 demonstrated approximately 2-line visual function gain over 3 years in Phase 1/2 studies with 88% of treated subjects showing improvement or preservation versus untreated eyes1
The Phase 3 liMeliGhT trial is the largest known Phase 3 orphan gene therapy trial and the only broad RP gene-agnostic trial, addressing treatment needs for the 98% of RP patients not candidates for existing approved therapies1
Sources:
1. https://www.globenewswire.com/news-release/2026/03/02/3247241/0/en/Ocugen-Announces-Phase-3-liMeliGhT-Enrollment-Completion-for-OCU400-a-Novel-Modifier-Gene-Therapy-for-Broad-Retinitis-Pigmentosa.html
3. https://www.fiercebiotech.com/biotech/ocugen-eye-disease-gene-therapy-nabs-full-ph-2-win-underwhelms-investors