Regenxbio Announces Initiation of Second Pivotal Trial in RGX-314 Clinical Program for the Treatment of Wet AMD Using Subretinal Delivery

Regenxbio announced the initiation of ASCENT, the second of two phase 3 pivotal trials to evaluate the efficacy and safety of subretinal delivery of RGX-314 in patients with wet age-related macular degeneration (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 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 company news release. "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 1/2a trial of RGX-314. We look forward to advancing both trials to support our goal of a BLA filing in 2024."

"The initiation of this phase 3 study, a first under our 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," said Michael Robinson, MD, vice president, clinical development, ophthalmology, AbbVie. "We look forward to identifying the full potential of RGX-314 as part of our commitment to advancing vision care."

ASCENT is a multicenter, 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 noninferiority to aflibercept based on the change from baseline in Best Corrected Visual Acuity (BCVA) at 1 year. The trial will enroll approximately 465 patients across the two dose arms and the aflibercept control arm.