Eyestem Receives CDSCO Approval to Initiate Phase 2 Randomized, Controlled Human Trials of Eyecyte-RPE™

Eyestem Research Pvt Ltd, a pioneering biotechnology company developing advanced scalable cell therapy solutions for blindness caused by dry age-related macular degeneration, has received approval from the Central Drugs Standard Control Organisation (CDSCO) to initiate the clinical trial of its investigational retinal pigment epithelium (RPE) cell therapy, Eyecyte-RPE™ in India. The randomized, controlled phase 2 clinical trials will evaluate the safety and efficacy of Eyecyte-RPE™ in a larger group of subjects with moderate geographic atrophy (GA) and associated vision loss.

The approval follows the successful completion of Eyestem’s Phase 1 clinical study of Eyecyte-RPE™, being developed for patients with geographic atrophy (GA) secondary to dry age-related macular degeneration (dry AMD). The study demonstrated an excellent safety profile with clinically meaningful improvements in visual acuity. In subjects with advanced GA and severe vision loss, patients recorded an average improvement of 14.8 letters at six months in the first eight subjects, with sustained gains of an average 14.6 letters observed at one year in five subjects. Importantly, six different surgeons performed the surgery in the Phase 1 study with excellent safety outcomes which validated Eyestem’s unique approach towards delivery of Eyecyte-RPE™ to the intended site of action.

Commenting on the upcoming Phase 2 trial and potential global market entry of Eyecyte-RPE™, Dr Jogin Desai, Founder and CEO, 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.”

The advancement to Phase 2 of Eyestem’s lead therapy is supported by capital raised in a recent oversubscribed funding round, which is enabling continued clinical development and preparations for global regulatory filings. 

Eyestem is also preparing to file an Investigational New Drug (IND) application with the US Food and Drug Administration (FDA), enabling the planned initiation of clinical studies in the United States.