Eyestem moves closer to blindness breakthrough with phase 2 trial green light

The early-stage trial demonstrated an excellent safety profile alongside clinically meaningful improvements in visual acuity

Biotech innovator Eyestem Research has secured regulatory clearance to advance its bid to treat blindness caused by dry age-related macular degeneration, marking a significant step forward for India’s cell therapy landscape.

The Central Drugs Standard Control Organisation (CDSCO) has approved the initiation of a randomized, controlled Phase 2 clinical trial of Eyestem’s investigational retinal pigment epithelium (RPE) cell therapy, Eyecyte-RPE. The study will assess the safety and efficacy of the therapy in a larger cohort of patients with moderate geographic atrophy (GA) and associated vision loss.

The decision follows encouraging results from Eyestem’s completed Phase 1 clinical study of Eyecyte-RPE, developed for patients with GA secondary to dry age-related macular degeneration.

The early-stage trial demonstrated an excellent safety profile alongside clinically meaningful improvements in visual acuity. Among patients with advanced GA and severe vision loss, the first eight subjects recorded an average improvement of 14.8 letters at six months, with sustained gains averaging 14.6 letters at one year in five subjects.

Notably, the Phase 1 trial involved six different surgeons, all of whom reported strong safety outcomes—underscoring the robustness and reproducibility of Eyestem’s proprietary delivery approach to targeting the intended site of action in the retina.

Commenting on the Phase 2 approval and the therapy’s global potential, Dr Jogin Desai, Founder and CEO of Eyestem, said, “There is no available therapy that reverses blindness caused by geographic atrophy (GA), affecting an estimated 8 million patients worldwide."

"Eyestem is among a few companies globally pursuing a scalable cell therapy approach toward a potential cure. CDSCO approval enables us to advance Eyecyte-RPE into Phase 2 clinical evaluation, allowing broader, randomized validation of our best-in-class Phase 1 outcomes. With AMD projected to affect 288 million people by 2040, this marks a major milestone in our mission to transform retinal care and patient lives worldwide.”

Progression of Eyestem’s lead program into Phase 2 is being supported by capital raised in a recent oversubscribed funding round, strengthening the company’s ability to advance clinical development and prepare for global regulatory engagement.

Alongside its India trials, Eyestem is also preparing to file an Investigational New Drug (IND) application with the US Food and Drug Administration (FDA), a move that would pave the way for planned clinical studies in the United States.