Luxa Biotech Shares Early Dry AMD Trial Results

Luxa Biotechnology LLC has announced encouraging results from the first low-dose cohort of its ongoing Phase 1/2a clinical trial evaluating RPESC-RPE-4W, a proprietary stem cell-derived therapy for dry age-related macular degeneration (AMD). The findings, published in Cell Stem Cell, mark a significant step forward in the development of regenerative treatments for a condition that currently lacks vision-restoring options.

Published Clinical Data Highlights Vision Improvement in Advanced AMD

The trial results are detailed in a peer-reviewed paper titled "Safety and Tolerability of RPE Stem Cell-derived RPE Transplantation in Patients with Dry Age-related Macular Degeneration: Low Dose Clinical Outcomes," now available in the journal Cell Stem Cell.

The publication focuses on Cohort 1, the first low-dose group in the ongoing clinical trial (NCT04627428), in which six participants with dry AMD received a subretinal injection of 50,000 RPESC-RPE-4W cells.

Key findings from the study include:

• Patients with severe baseline vision loss experienced a mean improvement of +21.67 ETDRS letters, sustained through 12 months, the largest vision gain reported to date in clinical trials of RPE cell therapies for dry AMD.

• Patients with better initial visual acuity saw more modest improvements, averaging +3.0 letters at 6 months.

• Importantly, there were no reports of tumor formation, severe inflammation, or therapy-related serious adverse events, supporting the early safety profile of the treatment.

Overview of RPESC-RPE-4W Therapy for Dry AMD

RPESC-RPE-4W is Luxa Biotechnology’s investigational retinal pigment epithelium (RPE) stem cell-derived therapy, designed to replace damaged or lost RPE cells in patients with dry AMD, a progressive retinal disease and leading cause of irreversible vision loss among older adults.

“The results from Cohort 1 demonstrate the potential of RPESC-RPE-4W not only to restore vision in patients with severe vision loss, but also to benefit those at earlier stages of disease. We are excited to continue advancing our clinical program, which recently received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, as a potential new regenerative therapy for dry AMD, a leading cause of blindness with no currently approved vision-improving treatments.”, said Dr. Keith Dionne, CEO of Luxa Biotechnology.

Reference:

Rao RC, Arduini BL, Borden S, et al. Safety and tolerability of RPESC-RPE transplantation in patients with dry age-related macular degeneration: Low-dose clinical outcomes. Cell Stem Cell.