Interim Phase 2 Data Show Sustained Visual Acuity Gains and Favorable Safety Profile
Eyestem Research, a biotechnology company developing scalable cell therapy solutions for blindness associated with dry age-related macular degeneration (AMD), announced promising interim clinical findings from its randomized Phase 2 trial of Eyecyte-RPE in patients with geographic atrophy (GA) secondary to dry AMD.
The interim findings were presented by Dr. Ritesh Narula, Co-Principal Investigator of the trial, at EURetina 2026 in Vienna, Austria.
With the Phase 2 study progressing, Eyestem is preparing to file a U.S. Investigational New Drug (IND) application in early 2027 for the treatment of geographic atrophy and associated vision loss.
Key Highlights
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24 subjects have been recruited in the randomized Phase 2 trial.
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15 subjects are in the treatment arm and 9 in the natural history control arm.
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Eyecyte-RPE demonstrated an excellent overall safety profile, consistent with findings from the preceding Phase 1 trial.
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Surgeries have been performed by 7 different surgeons using Eyestem’s patent-pending surgical technique.
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Visual acuity improvements observed in Phase 1 have continued with longer follow-up.
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Seven subjects demonstrated a 13-letter improvement at 12 months versus baseline.
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Four subjects completing 18 months maintained an average 15.3-letter improvement.
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In Phase 2, three subjects completing six months demonstrated a 16-letter improvement versus baseline.
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Across both Phase 1 and Phase 2, 12 subjects completing six months showed an average improvement of 13.8 letters.
Phase 2 Clinical Trial Progress
The randomized controlled Phase 2 trial is evaluating Eyecyte-RPE in patients with geographic atrophy secondary to dry AMD.
Trial Enrollment
| Study Group | Subjects |
|---|---|
| Treatment arm | 15 |
| Natural history control arm | 9 |
| Total | 24 |
Safety Profile
Eyecyte-RPE has demonstrated an overall favorable safety profile in the Phase 2 study, consistent with the safety findings reported in the earlier Phase 1 clinical trial.
The surgical procedures have been performed by seven different surgeons, providing additional clinical experience with Eyestem’s patent-pending surgical technique.
Visual Acuity Results
Phase 1 Long-Term Follow-Up
The visual acuity improvements observed during Phase 1 have continued during longer-term follow-up:
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7 subjects showed a 13-letter improvement at 12 months compared with baseline.
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4 subjects who completed 18 months maintained an average 15.3-letter improvement.
Phase 2 Results
Early Phase 2 findings have been consistent with the visual acuity improvements observed in Phase 1:
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3 subjects completing six months demonstrated a 16-letter improvement versus baseline.
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Across both Phase 1 and Phase 2, 12 subjects completing six months showed an average improvement of 13.8 letters.
Next Regulatory Milestone
As the Phase 2 trial progresses, Eyestem is preparing for its next major development milestone: filing a U.S. IND application in early 2027 for Eyecyte-RPE in geographic atrophy and related vision loss.
CEO Commentary
“Early results from the phase 2 clinical study build on the highly promising phase 1 results, achieving an important milestone for Eyestem’s geographic atrophy program. We are encouraged to see the gains sustained over longer follow-up periods coupled with a good safety profile. Together, these findings give us greater confidence in the potential of Eyecyte-RPE to meaningfully change the treatment landscape for patients with geographic atrophy across the world.”
— Dr. Jogin Desai, Founder & CEO, Eyestem Research
