Luxa Biotechnology announced trial results showing substantial improvements in best corrected visual acuity (BCVA) for patients receiving its novel adult retinal pigment epithelial stem cell therapy (RPESC-RPE-4W) for dry age-related macular degeneration (AMD).  

Six patients in the low-dose cohort of the ongoing first-in-human phase 1/2a clinical trial each received a 50,000-cell suspension of RPESC-RPE-4W implanted under the macula, said Luxa. Post-implant clinical outcomes were assessed over 12 months for the worse-seeing group I and over three months for the better-seeing group II. No serious adverse events related to the investigational therapy were reported. 

Group I experienced an average gain of +21.67 ETDRS letters at 12 months and group II showed a +3.3-letter improvement at three months, said Luxa chief medical officer Dr Jeffery Stern, presenting data at the Annual Wills Eye Conference in Philadelphia. “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 US Food and Drug Administration has granted RPESC-RPE-4W Regenerative Medicine Advanced Therapy (RMAT) designation, which is designed to accelerate the development and review of promising regenerative medicine therapies which treat serious or life-threatening diseases. The company said it will continue its phase 1/2a trial and evaluate higher dose cohorts.