Luxa Biotech’s RPESC-RPE-4W Boosts Vision in AMD Trial

Luxa Biotechnology has presented clinical data from its Phase 1/2a trial evaluating RPESC-RPE-4W at the 77th Annual Wills Eye Conference in Philadelphia on March 6, 2025. The investigational therapy demonstrated promising vision improvements in patients with advanced dry age-related macular degeneration (AMD) without any serious adverse events, marking a significant step forward in regenerative medicine for retinal diseases.

Key Findings from the Phase 1/2a Trial

The trial (NCT04627428) involved six patients in the first low-dose cohort, each receiving a 50,000-cell suspension of RPESC-RPE-4W implanted under the macula. The clinical outcomes were monitored over 12 months for the worse-seeing Group I and over three months for the better-seeing Group II.

Notable Results:

• No serious adverse events (SAEs) related to the therapy were observed.

• Group I (worse-seeing patients): Achieved an average gain of +21.67 ETDRS letters at 12 months.

• Group II (better-seeing patients): Showed a +3.3-letter improvement at 3 months.

• Patients with severe baseline vision impairment exhibited the most significant vision restoration.

• Individuals with better baseline vision saw modest yet clinically meaningful improvements, countering expected disease progression.

Expert Insights

Jeffrey Stern, MD, PhD, Chief Medical Officer of Luxa, emphasized the significance of these findings:
"These findings are encouraging, as they demonstrate not only meaningful vision improvements in patients with the greatest need but also the potential to alter the trajectory of dry AMD in those with better baseline vision. The substantial BCVA gains highlight the promise of our approach in restoring vision, while the modest improvements suggest a beneficial impact in slowing disease progression."

Regulatory Recognition

Luxa Biotechnology previously announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) Designation for RPESC-RPE-4W, reinforcing the potential of this innovative therapy in addressing the unmet needs of dry AMD patients.

As the trial progresses, Luxa Biotechnology remains committed to advancing cutting-edge treatments that offer hope for vision restoration and disease modification in dry AMD.