REGENXBIO Inc. (Nasdaq: RGNX), a clinical-stage biotechnology company has announced that the first patient has been dosed in the “AAVIATE” trial, a Phase II trial to evaluate the suprachoroidal delivery of RGX-314 for the treatment of wet age-related macular degeneration (wet AMD). The study uses a randomized, dose-escalation trial designed to evaluate the efficacy, safety and tolerability of RGX-314 gene therapy in subjects with nAMD. Approximately 40 participants who meet the inclusion/exclusion criteria, will be enrolled into one of 2 dose cohorts. Efficacy will be the primary focus of the study and participants will be evaluated for safety and tolerability.The company expects to report interim data from the first cohort of this trial by the end of 2020.
RGX-314 is being developed for wet AMD and diabetic retinopathy using an AAV8 vector encoding an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). According to the company, two separate routes of administration of RGX-314 use a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. A microinjector medical device from Clearside Biomedical, Inc. is aimed to deliver gene therapy treatment to the suprachoroidal space of the eye. The primary outcome of the study across 5 US centres is aimed to evaluate the mean change in BCVA for RGX-314 compared with ranibizumab monthly. The trial is expected to enrol approximately 40 patients with severe wet AMD across two cohorts. Patients in each cohort will be randomized to receive RGX-314 versus monthly 0.5 mg ranibizumab intravitreal injection at a 3:1 ratio, and two dose levels of RGX-314 will be evaluated: 2.5×1011 GC/eye and 5×1011 GC/eye. Patients will not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314.
Following the announcement of the achieved milestone, Dr. Steve Pakola, of the company Chief Medical Officer, stated that, “the initiation of this trial is an important milestone for REGENXBIO, as it is the first clinical trial to evaluate the delivery of any gene therapy, including RGX-314, to the suprachoroidal space. RGX-314 has shown evidence of potentially meaningful treatment effect in patients with wet AMD when delivered subretinally in the ongoing Phase I/IIa trial, and we are excited about the potential of the targeted, in-office suprachoroidal approach, which may provide additional RGX-314 delivery options for patients”.