REGENXBIO Inc. announced additional positive interim data from the ongoing Phase II ALTITUDE™ trial of RGX-314 for the treatment of diabetic retinopathy (DR) without center-involved diabetic macular edema (CI-DME) using in-office suprachoroidal delivery. The data is being presented at the Angiogenesis, Exudation, and Degeneration 2022 conference by Michael A. Klufas, M.D., Retina Service, Wills Eye Hospital, Assistant Professor of Ophthalmology, Thomas Jefferson University. RGX-314 is being investigated as a potential one-time gene therapy for the treatment of wet age-related macular degeneration and DR.
Also Read: Arkuda Raises $64 Million Series B Financing to Advance Pipeline of Programs
“We are pleased to see that RGX-314 continues to be well tolerated at six months following a one-time, in-office injection, with nearly 50 percent of patients dosed with RGX-314 in Cohort 1 demonstrating a clinically meaningful improvement from baseline” said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. “We are continuing to enroll patients in Cohorts 2 and 3 and look forward to sharing additional updates from this trial”
“I am encouraged by the clinical improvement of disease severity observed in the ALTITUDE trial of RGX-314,” said Dr. Klufas. “Globally, DR is the leading cause of blindness in working-age adults, and these patients are in need of new treatment options. I look forward to the further investigation of RGX-314 as a potentially compelling treatment option for patients with DR.”
Study Design and Safety Update from Phase II ALTITUDE Trial of RGX-314 for the Treatment of DR Using Suprachoroidal Delivery
ALTITUDE is a multi-center, open-label, randomized, controlled dose-escalation trial evaluating the efficacy, safety and tolerability of suprachoroidal delivery of RGX-314 using the SCS Microinjector® in patients with a DR diagnosis of moderately severe or severe nonproliferative diabetic retinopathy (NPDR) or mild proliferative diabetic retinopathy (PDR). Twenty patients in Cohort 1 were randomized to receive RGX-314 at a dose level of 2.5×1011 genomic copies per eye (GC/eye) versus observational control at a 3:1 ratio. Cohort 2 will include 20 patients randomized to receive RGX-314 at an increased dose level of 5×1011 GC/eye versus observational control at a 3:1 ratio. Cohort 3 is designed to evaluate RGX-314 at the same dose level as Cohort 2 in 20 patients who are neutralizing antibody (NAb) positive. Enrollment is ongoing in Cohorts 2 and 3. Patients in this trial do not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314.