ASCENT, RegenXBio’s Phase III clinical trial conducted in partnership with AbbVie, is expected to enroll patients in the United States and Canada, with pivotal trials expected to support BLA submission for RGX-314 in 2024.
RegenXBio Inc is kicking off the second pivotal trial in the clinical program for RGX-314, a subretinal gene therapy being tested for wet age-related macular degeneration (AMD).
ASCENT is the second of two Phase III pivotal trials to evaluate the efficacy and safety of subretinal delivery of RGX-314 in patients with wet age-related macular degeneration (wet AMD). ASCENT, the first trial to be initiated by RegenXBio under the eye care collaboration with AbbVie, is currently active and screening patients. RGX-314 is being investigated as a potential one-time gene therapy for the treatment of wet AMD.
A Biologics License Application (BLA) is expected to be submitted to the United States Food and Drug Administration (FDA) in 2024 based on two pivotal trials, ASCENT and the ongoing ATMOSPHERE trial.
"The initiation of ASCENT is an important milestone for the pivotal program for subretinal delivery of RGX-314 in patients with wet AMD, and it is the first trial to be started under our partnership with AbbVie," Steve Pakola, MD, chief medical officer of RegenXBio, said in a statement. "ASCENT is designed similarly to our ongoing ATMOSPHERE trial, and key design elements for both pivotal studies are based on the positive long-term data from our dose-escalation Phase I/IIa trial of RGX-314. We look forward to advancing both trials to support our goal of a BLA filing in 2024."
According to Michael Robinson, MD, vice president, clinical development, ophthalmology, AbbVie, the initiation of this Phase III study, a first under the company’s collaboration with RegenXBio, “is an important advancement in our continued pursuit of innovative treatments for patients living with difficult-to-treat retinal diseases, visual impairment, and devastating vision loss."
ASCENT is a multi-center, randomized, active-controlled trial evaluating the efficacy and safety of subretinal delivery of RGX-314 across two dose arms, 6.4x1010 genomic copies per eye (GC/eye) and 1.3x1011 GC/eye, versus intravitreal injections of aflibercept, per label instructions. The primary endpoint of the trial is non-inferiority to aflibercept based on the change from baseline in Best Corrected Visual Acuity (BCVA) at one year. The trial will enroll approximately 465 patients across the two dose arms and the aflibercept control arm.
RegenXBio is investigating RGX-314 in collaboration with AbbVie as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 includes the NAV AAV8 vector containing a gene encoding for a monoclonal 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.1