Eyestem reports promising phase 1 trial results in patients with geographic atrophy

Eyestem Research announced positive results from its phase 1 trial (NCT06394232) evaluating its investigational drug product, Eyecyte-RPE, in patients with geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD).

The company previously announced it had completed the first round of dosing in its trial in August 2024. The trial treated 9 patients in 3 sequential, ascending dose-level (DL) cohorts. The 3 dose levels were Eyecyte-RPE at 100,000; 200,000; and 300,000 cells. Eyecyte-RPE is a suspension of hiPSCs (human induced pluripotent stem cells) derived retinal pigment epithelial (RPE) cells.

The company stated that no serious adverse events were reported across all 3 cohorts, and the first 6 patients had an average improvement of 14.9 letters in early treatment diabetic retinopathy study (ETDRS) standard vision tests within 4 to 6 months post-transplantation. The company also noted that “retinal imaging scans revealed early signs of disease reversal in a handful of patients.”

Jogin Desai, founder and CEO of Eyestem commented on the results in a press release from the company.

“The substantial vision improvement, sustained over a 4-6-month period in all 6 patients, combined with an excellent safety profile, has been very encouraging and has the potential to reshape the landscape of GA treatment globally. It is especially rewarding to see the improvement in the quality of life in these patients. We look forward to exploring these early results further in our phase two study in India and the US," said Desai.

Eyestem plans to present data from the trial at the upcoming ARVO 2025 annual meeting.

References:

1. Eyestem’s Eyecyte-RPE trial shows vision rescue for geographic atrophy patients. Published April 14, 2025. Accessed April 14, 2025. https://economictimes.indiatimes.com/small-biz/sme-sector/eyestems-eyecyte-rpe-trial-shows-vision-rescue-for-geographic-atrophy-patients/articleshow/120271413.cms

2. Safety & efficacy of Eyecyte-RPE™ in patients with geographic atrophy secondary to dry age-related macular degeneration. NCT06394232. Accessed April 14, 2025. https://clinicaltrials.gov/study/NCT06394232