RegenxBio has completed dosing of the third cohort in a Phase I clinical trial that seeks to examine RGX-314 for the treatment of patients suffering from wet age-related macular degeneration (wet AMD).
The cohort includes six patients, while topline data from the study is expected to be released later this year.
REGENXBIO chief medical officer Stephen Yoo said: “Completing enrolment of the third cohort in the Phase I clinical trial brings us one step closer toward delivering on the promise of a one-time treatment with rapid and sustained therapeutic effects for patients with wet AMD, one of the largest indications for which gene therapy is being developed.”
The Phase I trial is a multi-centre, open-label, multiple-cohort, dose-escalation study and aims to evaluate the safety and tolerability of RGX-314 as a one-time therapy for patients with previously treated wet AMD.
It intends to enrol about 18 previously treated wet AMD patients who are responsive to anti-vascular endothelial growth factor (anti-VEGF) therapy and are 50 years of age or older.
As part of the trial, three doses of RGX-314 will be evaluated.
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The trial’s primary objective is to examine the safety and tolerability of RGX-314 at 24 weeks after a single dose administered by subretinal delivery.
Its primary endpoints are safety and tolerability, while secondary endpoints are ocular examinations and imaging as well as the need for additional anti-VEGF therapy.
Once the primary study period concludes, the subjects will enter a follow-up period and will continue to be evaluated until week 106 to determine long-term safety and durability of effect of RGX-314.
The trial is being conducted at six retinal surgery centres across the US.