- Patients with the most severe baseline vision impairment and the greatest clinical need achieved the most significant vision restoration
- Individuals with better baseline vision saw smaller, yet still clinically relevant, improvements that counteracted the natural decline expected in dry AMD progression
FORT LEE, N.J.--(BUSINESS WIRE)--Luxa Biotechnology LLC (Luxa), a clinical-stage biotechnology company developing a novel adult retinal pigment epithelial stem cell (RPESC) therapy for dry age-related macular degeneration (AMD), today announced clinical data from its Phase 1/2a clinical trial evaluating RPESC-RPE-4W, a proprietary retinal pigment epithelium (RPE) cell therapy for patients with dry age-related macular degeneration (dry AMD). The data were presented today at the 77th Annual Wills Eye Conference in Philadelphia, Pennsylvania.
Findings from six patients in the first low-dose cohort of the ongoing first-in-human trial were presented (NCT04627428). Each patient received a 50,000-cell suspension of RPESC-RPE-4W implanted under the macula. Post-implant clinical outcomes were assessed over 12 months for the worse-seeing Group I and over 3 months for the better-seeing Group II. No serious adverse events (SAEs) related to the investigational therapy were observed.
Best Corrected Visual Acuity (BCVA) improvements were substantial, with Group I experiencing an average gain of +21.67 ETDRS letters at 12 months and Group II showing a +3.3-letter improvement at 3 months. These gains are particularly meaningful given that even a 5- to 10-letter improvement can translate into a real-world functional benefit for patients, such as improved reading ability, better recognition of faces, and enhanced mobility.
The patients with the most severe baseline vision impairment and the greatest clinical need achieved the most significant vision restoration, reinforcing the potential of this therapeutic approach to address the substantial unmet need in advanced dry AMD. Meanwhile, individuals who began with better baseline vision saw smaller, yet still clinically relevant, improvements that counteracted the natural decline expected in dry AMD progression. This reversal of disease trajectory underscores the potential for preserving vision in earlier-stage patients, possibly delaying or even preventing progression to severe visual impairment.
“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,” said Dr. Jeffrey Stern, Chief Medical Officer of Luxa. “The substantial BCVA gains, particularly in worse-seeing patients, highlight the promise of our approach in restoring vision, while the modest improvements in better-seeing individuals suggest a beneficial impact in slowing disease progression. This reinforces our commitment to advancing innovative treatments that address the urgent unmet needs in dry AMD.”
Last month, Luxa announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) Designation for RPESC-RPE-4W in the treatment of dry AMD. This designation will further facilitate the development and regulatory progress of the novel adult stem cell therapy.
“We are thrilled with these promising clinical outcomes, which mark a significant step forward in our progress towards addressing a major unmet need in dry AMD,” said Dr. Keith Dionne, CEO of Luxa. “This achievement underscores our commitment to reversing vision loss for millions of adults affected by dry AMD and geographic atrophy. We look forward to advancing our Phase 1/2a trial and evaluating higher dose cohorts.”
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 accompanied by 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 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 and the U.S. National Institutes of Health and the National Eye Institute. For more information, visit our website and follow us on LinkedIn.
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Christine Quern
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