Luxa Biotechnology Presents Data from First-in-Human Retinal Pigment Epithelium Cell Therapy for Dry AMD

Luxa Biotechnology 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 (AMD). The data were presented at the 77th Annual Wills Eye Conference in Philadelphia.

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, according to Luxa, 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. 

The patients with the most severe baseline vision impairment and the greatest clinical need achieved the most significant vision restoration. Individuals who began with better baseline vision saw smaller, yet still clinically relevant, improvements that counteracted the natural decline expected in dry AMD progression. 

"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,” Jeffrey Stern, MD, PhD, Chief Medical Officer of Luxa, said in a company news release. “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 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.