Luxa Biotechnology Announces Publication in Cell Stem Cell Describing Positive Clinical Results for First-in-Human Clinical Trial for Dry AMD Therapy

FORT LEE, N.J.--(BUSINESS WIRE)--Luxa Biotechnology LLC (Luxa), a clinical-stage biotechnology company developing novel adult retinal pigment epithelial stem cell (RPESC) therapies for dry age-related macular degeneration (dry AMD), today announced the publication of Phase 1/2a clinical trial results in a paper titled "Safety and Tolerability of RPE Stem Cell-derived RPE Transplantation in Patients with Dry Age-related Macular Degeneration: Low Dose Clinical Outcomes" in the journal Cell Stem Cell. The publication is available via this link.

This publication reports results from the first low-dose cohort (Cohort 1) of Luxa’s ongoing Phase 1/2a first-in-human clinical trial (NCT04627428) evaluating its proprietary RPESC-RPE-4W therapy. In Cohort 1, six participants received a subretinal injection of 50,000 RPESC-RPE-4W cells. Over the follow-up period, patients with severe vision loss gained an average of +21.67 ETDRS letters maintained through 12 months, representing the largest vision improvement reported to date in clinical trials of RPE cell therapies for dry AMD. Patients with better baseline vision showed more modest gains (+3.0 letters at 6 months). No tumor formation, severe inflammation, or therapy-related serious adverse events were observed.

“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,” said Dr. Keith Dionne, CEO of Luxa Biotechnology. “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.”

About Dry AMD

Age-related macular degeneration (AMD) is the leading cause of adult-onset blindness in the Western world, affecting approximately 20 million Americans and 200 million people globally, with over 80% of cases classified as the dry form. Dry AMD causes diminished vision following loss of central retinal pigmented epithelium (RPE) cells in individuals aged 55 and older. There are no approved vision-improving treatments for dry AMD.

About Luxa Biotechnology

Luxa Biotechnology is a joint venture of the Korean technology company Y2 Solution and the Neural Stem Cell Institute (NSCI) in Albany, New York. Luxa licensed RPESC-RPE-4W from the NSCI and maintains a robust research program at NSCI to advance RPESC-RPE-4W as an effective, commercially viable therapy to restore the lost vision of AMD patients. Luxa acknowledges the support and critical contributions of the clinical team at the University of Michigan Kellogg Eye Center, the manufacturing team at the Cedars Sinai Biomanufacturing Center, the California Institute for Regenerative Medicine, the U.S. National Institutes of Health, and the National Eye Institute. For more information, visit our website and follow us on LinkedIn.