Luxa Biotechnology presents clinical data from the phase 1/2a trial evaluating RPESC-RPE-4W

(Image Credit: AdobeStock/The Little Hut)

Clinical data from the Phase 1/2a clinical trial evaluating RPESC-RPE-4W was presented at the 77th Annual Wills Eye Conference in Philadelphia, Pennsylvania on March 6, 2025.

The findings were from 6 patients in the first low-dose cohort of the ongoing first-in-human trial (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.¹

According to the Luxa Biotechnology’s press release¹, the 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 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.¹

Jeffrey Stern, MD, PhD, Chief Medical Officer of Luxa is quoted on these results in the press release¹, saying, "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, 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.”

Previously, Luxa announced that the US Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) Designation for RPESC-RPE-4W in the treatment of dry AMD.

Reference:

1. Luxa Biotechnology Presents Clinical Data from First-in-Human Retinal Pigment Epithelium Cell Therapy Trial for Dry Age-Related Macular Degeneration at the 77th Annual Wills Eye Conference. Luxa Biotechnology LLC. March 6, 2025. Accessed March 7, 2025. https://www.businesswire.com/news/home/20250306998366/en/Luxa-Biotechnology-Presents-Clinical-Data-from-First-in-Human-Retinal-Pigment-Epithelium-Cell-Therapy-Trial-for-Dry-Age-Related-Macular-Degeneration-at-the-77th-Annual-Wills-Eye-Conference