Eyestem Research's phase 1/2a trial results find significant vision improvements in patients treated with Eyecyte-RPE for GA

Eyecyte-RPE is a cryopreserved, allogenic retinal pigment epithelium (RPE) cell suspension. Image credit: AdobeStock/Trsakaoe

Bangalore-based cell therapy company Eyestem Research has announced results from its phase 1 trials of Eyecyte-RPE, an investigational drug, which was evaluated in patients with geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD), according to a news release.¹ The phase 1/2a multicenter, dose-escalation study worked to answer the main questions of safety and tolerability of the novel stem cell formulation, and potential efficacy of the novel stem cell formulation.²

“The substantial vision improvement, sustained over a 4- to 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,” said Jogin Desai, MD, MBBS, founder and CEO of Eyestem, in the release. “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 2 study in India and the US.”

All patient subretinal injections were completed across 3 cohorts without any serious adverse events and reported significant vision improvement, with the first 6 patients having an average improvement of 14.9 letters in early treatment diabetic retinopathy study (ETDRS) standard vision tests within 4 to 6 months post transplantation. Additionally, retinal imaging scans in the trial revealed early signs of disease reversal in a handful of the 9 patients who were enrolled in the study.¹

Inclusion criteria for the trial included men and women aged 50 years or older at screening and those diagnosed with GA secondary to dry AMD. Best corrected visual acuity was equal to or less than 20/200 Snellen (ETDRS letter score at or less than 35) in the study eye at screening for phase 1, whereas phase 2a included patients with greater than or equal to 20/64 (ETDRS letter score 60) in the study eye. Additionally, the GA lesion included criteria determined by the assessment of fundus autofluorescence imaging at screening. This included a total GA area of equal to or greater than 1.25, and equal to or less than 17.5 mm2 (0.5 and 7 disk areas respectively); at least 1 of the lesions had to be subfoveal, and the entire lesion was required to be completely visualized on the macula centered image, able to be imaged in its entirety, and not contiguous with any areas of peripapillary atrophy.²

Eyecyte-RPE is a cryopreserved, allogenic retinal pigment epithelium (RPE) cell suspension designed to replace damaged retinal cells for the restoration of vision.¹ Human induced pluripotent stem cells derived Eyecyte-RPE. These stem cells can perform multiple functions, including immunoregulation, prevention of apoptosis in sensory neurons, and secretion of neurotrophic factors.²

Following the approval from the Central Drug Standards Control Organization, the trial was conducted across 3 centers in India, including AIIMS New Delhi, LV Prasad Eye Institute Hyderabad, and Shri Ganpati Nethralaya, Jalna. Safety monitoring oversight was provided by an independent drug safety monitoring board comprising global experts, the release stated.

“As some of the earliest backers of Eyestem, we are delighted that this pioneering treatment is delivering remarkable outcomes,” said Gullapalli N. Rao, MBBS, founder of LV Prasad Eye Institute and former president of Academia Ophthalmologica Internationalis, and Oppel Greeff, MBChB, FCFP(SA), MPharmMed, FFPM(RCR), MD, former president of Global Clinical Development at Quintiles (now IQVIA), in a joint statement in the release. “The results observed so far in phase 1 not only demonstrate promising efficacy compared to other ongoing global trials but also validate India's potential as a leader in advanced cell therapy innovations. We are privileged to support a breakthrough that has the potential to redefine treatment outcomes for millions suffering from dry AMD.”

Eyestem will be presenting its data at the ARVO 2025 Annual Meeting, as well as at the 10^th Retinal Cell and Gene Therapy Innovation Summit in early May.

References:

1. Eyestem’s Eyecyte-RPE trial shows vision rescue for geographic atrophy patients. News release. Eyestem Research. April 14, 2025. Accessed April 14, 2025.

2. Safety and efficacy of Eyecyte-RPE in patients with geographic atrophy secondary to dry age-related macular degeneration. Eyestem Research Pvt. Ltd. Updated September 24, 2024. Accessed April 14, 2025. https://clinicaltrials.gov/study/NCT06394232