RegenxBio has reported preliminary results from the ongoing Phase II ALTITUDE clinical trial of RGX-314 to treat diabetic retinopathy (DR) without centre-involved diabetic macular oedema (CI-DME).
A potential one-time gene therapy, RGX-314 is currently being developed to treat wet age-related macular degeneration, as well as various other chronic retinal ailments.
The multicentre, randomised, open-label, controlled dose-escalation Phase II trial is analysing the efficacy, safety and tolerability of an in-office suprachoroidal delivery of RGX-314 given with the help of SCS Microinjector in DR patients.
Subjects included in the trial comprised people with moderately severe or severe non-proliferative diabetic retinopathy (NPDR) or mild proliferative diabetic retinopathy (PDR).
Findings showed that RGX-314 was demonstrated to be well tolerated in the 15 subjects dosed with RGX-314 in cohort 1.
A patient receiving RGX-314 experienced a serious adverse event, which happened in the untreated fellow eye and is deemed unrelated to the treatment.
No intraocular inflammation was reported on slit-lamp examination in subjects in Cohort 1 receiving RGX-314.
One subject had mild episcleritis, which was resolved by using topical corticosteroids.
Common ocular treatment-emergent adverse events in the study eye were reported to be unrelated to the treatment and were mostly mild in nature.
A two-step or higher improvement from baseline on the Early Treatment Diabetic Retinopathy Study-Diabetic Retinopathy Severity Scale (ETDRS-DRSS) were experienced by 33% of subjects in Cohort 1 at three months against 0% in the observational control arm.
Nearly 43% of the NPDR patients had a two-step or greater improvement following three months of RGX-314 administration.
Approximately 25% of the subjects with PDR showed a two-step or greater improvement at three months on receiving treatment, the company noted.
Regenxbio chief medical officer Steve Pakola said: “We are pleased to share initial data from the Phase II ALTITUDE trial, and we are encouraged to see treatment effect particularly at this early time point of three months after the one-time, in-office administration of RGX-314.”
“RGX-314 is the first gene therapy in clinical trials for DR using suprachoroidal delivery, and has the potential to provide sustainable, long-term anti-VEGF protein production in the eye for the treatment of DR, which affects approximately eight million people in the US alone.”
Cell & Gene Therapy Coverage on Clinical Trials Arena supported by Cytiva.
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