Eyestem Receives CDSCO Approval for Phase 2 Trial of Stem Cell Therapy for Dry AMD
Key Insights
Eyestem Research (search) received regulatory clearance from India's CDSCO to initiate a randomized, controlled Phase 2 clinical trial of Eyecyte-RPE for dry age-related macular degeneration (search).
The Phase 1 trial demonstrated excellent safety and clinically meaningful visual improvements, with patients showing an average 14.8-letter improvement at six months.
The therapy targets geographic atrophy (search) affecting 8 million patients worldwide, with no current treatments available to reverse blindness from this condition.
Biotech innovator Eyestem Research (search) has secured regulatory clearance from India's Central Drugs Standard Control Organisation (search) (CDSCO) to advance its investigational retinal pigment epithelium (search) (RPE) cell therapy into Phase 2 clinical trials. The approval represents a significant milestone for treating blindness caused by dry age-related macular degeneration (search) (AMD), marking a major step forward for India's cell therapy landscape.
Phase 2 Trial Design and Objectives
The CDSCO has approved initiation of a randomized, controlled Phase 2 clinical trial of Eyecyte-RPE, which will assess the safety and efficacy of the therapy in a larger cohort of patients with moderate geographic atrophy (search) (GA) and associated vision loss. This advancement follows encouraging results from Eyestem's completed Phase 1 clinical study.
Promising Phase 1 Results
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.
Addressing Unmet Medical Need
"There is no available therapy that reverses blindness caused by geographic atrophy (search) (GA), affecting an estimated 8 million patients worldwide," said Dr. Jogin Desai, Founder and CEO of Eyestem. "Eyestem is among a few companies globally pursuing a scalable cell therapy approach toward a potential cure."
Dr. Desai emphasized the global significance of this development, noting that "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."
Innovative Cell Therapy Platform
Eyecyte-RPE is derived from human induced pluripotent stem cells (hiPSCs) and represents Eyestem's proprietary retinal cell therapy product with a novel composition, developed and patented by Eyestem Research (search). The product is designed to replace damaged or lost retinal pigment epithelial (RPE) cells and potentially enable tissue regeneration in the diseased retina. This unique formulation is allogeneic, scalable, and can be stored as a frozen vial for extended periods.
Global Expansion Plans
The progression 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 (search) (FDA), which would pave the way for planned clinical studies in the United States.