Eyestem Reports Interim Phase 2 Data for Eyecyte-RPE in Geographic Atrophy
Indian cell therapy company reports sustained visual-acuity gains and favorable safety profile at EURETINA 2026
Indian regenerative medicine company Eyestem has presented interim results from its randomized, controlled Phase 2 study of Eyecyte-RPE™, an investigational retinal pigment epithelium (RPE) cell therapy for geographic atrophy (GA) associated with dry age-related macular degeneration (AMD).
The company presented the data at the 2026 EURETINA conference, highlighting continued safety, improvements in best-corrected visual acuity (BCVA), and the reproducibility of its surgical delivery approach.
Eyestem says the findings strengthen its confidence in the potential of Eyecyte-RPE to address vision loss associated with GA and plans to begin preparations for a U.S. IND filing in early 2027.
A Cell-Replacement Approach for Geographic Atrophy
Geographic atrophy is an advanced form of dry AMD in which progressive loss of retinal pigment epithelium and associated retinal structures causes irreversible deterioration of central vision.
The RPE plays a critical role in maintaining photoreceptor health and retinal function. Eyestem’s approach is therefore based on cell replacement rather than simply slowing disease progression.
Eyecyte-RPE is an allogeneic RPE cell suspension designed to replace damaged or lost RPE cells. The company’s platform uses induced pluripotent stem cell (iPSC)-derived RPE cells as a potentially scalable source for transplantation.
The company previously reported completion of its Phase 1 study, with no treatment-related serious adverse events and encouraging improvements in visual acuity among the initial participants.
Interim Phase 2 Findings
According to Eyestem’s presentation of the interim data:
- 24 subjects have been recruited into the Phase 2 randomized controlled trial.
- Three Phase 2 participants have completed six months of follow-up, showing an average 16-letter improvement in BCVA.
- Across Phase 1 and Phase 2, 12 participants have completed six months, with an average 13.8-letter BCVA gain.
- Four Phase 1 participants have reached 18 months of follow-up, with an average 15.3-letter gain.
- The company reports that no product-related serious adverse events have been observed across the two phases.
- Seven surgeons have performed the procedures, providing early evidence that the company’s investigational surgical technique can be reproduced across surgeons.
Eyestem also reports a characteristic pattern in visual acuity: an initial improvement during approximately 4–8 weeks, followed by stabilization around month three and maintenance thereafter.
These results remain interim findings from a relatively small number of patients. Larger numbers and longer follow-up will be needed to determine the durability and reproducibility of the observed visual improvements.
Why the Surgical Delivery Matters
Cell replacement therapies for the retina depend not only on the quality and survival of the transplanted cells but also on where and how those cells are delivered.
Eyestem is developing Eyecyte-RPE as a cell suspension delivered through a proprietary surgical approach intended to place the cells in the appropriate retinal location.
The participation of seven surgeons is therefore an important operational milestone for the program. If the technique can be reproduced consistently across surgeons and clinical centers, it could support broader clinical development.
The company describes its approach as a patent-pending surgical technique.
From Phase 1 to Phase 2
Eyestem received approval from India’s Central Drugs Standard Control Organisation (CDSCO) in January 2026 to initiate a randomized, controlled Phase 2 trial of Eyecyte-RPE in patients with moderate geographic atrophy and associated vision loss.
The company’s earlier Phase 1/2a study involved patients with GA secondary to dry AMD. Eyestem reported that the study demonstrated an encouraging safety profile and sustained visual improvements during follow-up.
The transition to a randomized, controlled Phase 2 study is an important step because it provides a more rigorous framework for determining whether the observed visual gains are attributable to the cell therapy.
A Crowded and Evolving GA Landscape
The development of Eyecyte-RPE comes as the therapeutic landscape for geographic atrophy is rapidly changing.
Existing approaches have largely focused on slowing complement-mediated disease progression rather than replacing cells that have already been lost. Cell replacement therefore represents a fundamentally different strategy: restore retinal cellular function rather than only slow further degeneration.
The challenge is demonstrating that transplanted cells survive, integrate appropriately, remain functional over the long term and translate into meaningful improvements in vision.
EURETINA’s 2026 programming reflects the broader momentum in GA research, with sessions covering current and emerging therapies, retinal implants, gene therapies, and new approaches to measuring functional outcomes.
The Next Milestones
For Eyestem, the immediate priorities will be completing the Phase 2 study and establishing whether the early visual-acuity signal remains consistent as more participants reach longer follow-up.
The company also plans to prepare for a U.S. IND submission in early 2027, potentially opening the way for clinical development outside India.
The most important questions will be whether the visual gains are reproducible in a larger randomized population, how long they persist, and whether the transplanted RPE cells can provide durable functional benefits.
For now, the interim results provide an encouraging clinical signal for an Indian-developed cell replacement approach targeting one of the major causes of irreversible vision loss in older adults.
Key Takeaway
Eyecyte-RPE remains an investigational therapy, and the reported Phase 2 results are preliminary. The combination of an encouraging safety profile, substantial visual-acuity gains in early follow-up, and reproducibility of the surgical procedure makes the program one to watch as Eyestem moves toward larger clinical datasets and potential international development.
Website: https://eyestem.com/
