Eyestem Completes Phase 1 Study of Eyecyte-RPE for Geographic Atrophy

Eyestem has announced the successful completion of its Phase 1 clinical trial for Eyecyte-RPE, an investigational retinal pigment epithelium (RPE) cell therapy targeting geographic atrophy (GA) secondary to dry age-related macular degeneration (dry AMD). The trial results have been submitted to the Central Drugs Standard Control Organization (CDSCO)—India’s regulatory authority for drugs and medical devices—for clearance to initiate Phase 2.

Promising Results in Early-Stage Evaluation

The Phase 1/2a study was designed to assess both safety and efficacy of the therapy. According to the company, Eyecyte-RPE has demonstrated “promising outcomes in the initial phase,” with a strong safety profile and measurable visual gains.

“Our Phase 1/2a trial demonstrated an excellent safety profile with no serious adverse events while delivering clinically meaningful vision improvement—an average of 15.8 letters in the first six patients over six months,” stated Rajani Battu, PhD, Chief Medical Officer at Eyestem.

Dosing and Treatment Details

The study involved nine patients, treated in three sequential, ascending dose-level (DL) cohorts:

       • DL1: 100,000 cells

       • DL2: 200,000 cells

       • DL3: 300,000 cells

Eyecyte-RPE is a suspension of human induced pluripotent stem cell (hiPSC)-derived retinal pigment epithelial (RPE) cells, designed to restore RPE function and slow progression in GA associated with dry AMD.

Pending regulatory approval, Eyestem is set to move forward with Phase 2 to further evaluate efficacy and expand the safety dataset in a broader patient population.