REGENXBIO Inc. Presents Additional Positive Interim Data from Trials of RGX-314 in Wet AMD and Diabetic Retinopathy Using Suprachoroidal Delivery at AAO 2021
November 12, 2021 at 04:32 pm EST
Share
REGENXBIO Inc. announced additional positive interim data from the ongoing Phase II AAVIATE? trial and the ongoing Phase II ALTITUDE? trial of RGX-314 using in-office suprachoroidal delivery for the treatment of wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR) without center-involved diabetic macular edema (CI-DME), respectively. The results were presented at the American Academy of Ophthalmology (AAO) 2021 Annual Meeting by Robert L. Avery, M.D., Founder of California Retina Consultants and Research Foundation. AAVIATE is a multi-center, open-label, randomized, active-controlled, dose-escalation trial that will evaluate the efficacy, safety and tolerability of suprachoroidal delivery of RGX-314 using the SCS Microinjector?. Twenty patients in Cohort 1 were randomized to receive RGX-314 at a dose level of 2.5x1011 genomic copies per eye (GC/eye) versus monthly 0.5 mg ranibizumab intravitreal injection at a 3:1 ratio. Twenty patients in Cohort 2 were randomized to receive RGX-314 at a dose level of 5x1011 GC/eye through two injections versus monthly 0.5 mg ranibizumab intravitreal injection at a 3:1 ratio. Cohort 3 is evaluating RGX-314 at the same dose level as Cohort 2 in 20 patients who are neutralizing antibody (NAb) positive. Enrollment is ongoing in two additional cohorts (Cohorts 4 and 5) to evaluate RGX-314 at a third dose level of 1x1012 GC/eye. Cohort 4 will enroll 15 patients who will be dosed with RGX-314 and Cohort 5 will evaluate the same dose level of RGX-314 in 20 patients who are NAb positive. Patients do not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314. In Cohort 2, patients dosed with RGX-314 demonstrated stable Best Corrected Visual Acuity (BCVA) and central retinal thickness (CRT) at 6 months. Fifteen patients in Cohort 2 dosed with RGX-314 had a mean BCVA change of -0.1 letters (95% Confidence Interval: -3.8, 3.6) when measured from Day 1 (at Screening) and +0.2 letters (-2.7, 3.1) when measured from Week 1 (prior to Randomization). These patients also demonstrated stable central retinal thickness (CRT), with a mean change of -33 ?m (-71, 5) at six months from Day 1. Ten control patients receiving monthly injections of ranibizumab in Cohorts 1 and 2 had a mean BCVA change at six months of +4.0 letters (-0.5, 8.5) when measured from Day 1 and +1.3 letters (-2.2, 4.8) when measured from Week 1. Patients receiving monthly injections of ranibizumab had a mean change of CRT of -12 ?m (-33, 8) at six months from Day 1. There was a meaningful reduction in anti-vascular endothelial growth factor (anti-VEGF) treatment burden in patients following administration of RGX-314 compared to the mean annualized injection rate during the 12 months prior to administration. Patients in Cohort 2 received a mean of 1.3 injections over six months following administration of RGX-314, which represents a 71.8% reduction in anti-VEGF treatment burden. Six out of 15 patients (40%) in Cohort 2 received no anti-VEGF injections over six months following RGX-314 administration. In these patients, visual acuity and CRT was observed to be stable from Day 1 over six months, with a mean change of BCVA of +1.0 letters (-3.8, 5.8), and a mean change of CRT of +8 ?m (-9.2, 24.2). REGENXBIO shared additional data from Cohort 1 of the ongoing ALTITUDE trial for the treatment of DR without CI-DME using in-office suprachoroidal delivery, supporting the positive initial data previously reported at the American Society of Retina Specialists (ASRS) Annual Meeting in October 2021. At three months, 15 patients dosed with 2.5x1011 GC/eye of RGX-314 demonstrated stable BCVA of +2.6 letters, while five patients in the observational control arm demonstrated stable BCVA of -0.4 letters. RGX-314 is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina. REGENXBIO is advancing research in two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector? from Clearside Biomedical Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye.
REGENXBIO Inc. is a clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. The Company has developed a pipeline of gene therapy programs using its proprietary adeno-associated virus (AAV) gene therapy delivery platform (NAV Technology Platform) to address genetic diseases. The Company is focused on its internal development pipeline in three areas: retinal, neuromuscular and neurodegenerative diseases. Its investigational AAV therapeutics include ABBV-RGX-314, RGX-202 and RGX-121. It is developing ABBV-RGX-314 in collaboration with AbbVie to treat large patient populations impacted by wet age-related macular degeneration, diabetic retinopathy (DR) and other chronic retinal diseases characterized by loss of vision. It is developing RGX-202 to treat Duchenne muscular dystrophy (Duchenne). The Company is developing RGX-121 to treat Mucopolysaccharidosis type II (MPS II), a progressive, neurodegenerative lysosomal storage disorder.
REGENXBIO Inc. Presents Additional Positive Interim Data from Trials of RGX-314 in Wet AMD and Diabetic Retinopathy Using Suprachoroidal Delivery at AAO 2021