REGENXBIO Launches Pivotal Trial for Gene Therapy in Diabetic Retinopathy
- REGENXBIO launches a pivotal trial for surabgene lomparvovec, targeting diabetic retinopathy with a novel delivery method.
- The trial follows promising Phase II results, highlighting sura-vec's safety and efficacy over two years post-injection.
- REGENXBIO aims to advance its gene therapy platform, focusing on retinal diseases and improving patient vision outcomes.
REGENXBIO Initiates Pivotal Trial for Gene Therapy Targeting Diabetic Retinopathy
REGENXBIO Inc. announces the launch of a pivotal Phase IIb/III clinical trial for its investigational gene therapy, surabgene lomparvovec (sura-vec, ABBV-RGX-314), targeting diabetic retinopathy (DR) through a novel suprachoroidal delivery method. This decision comes on the heels of encouraging results from the Phase II ALTITUDE® trial, which showcased the treatment's durable safety and efficacy profile over a two-year period following a single in-office injection. Notably, no serious drug-related adverse events were reported, and intraocular inflammation was absent at the highest dose level, underscoring the therapy's favorable safety profile. This pivotal trial represents a significant step in REGENXBIO's commitment to addressing the unmet medical needs of patients suffering from DR, a condition that poses a substantial risk of vision loss.
The upcoming trial aims to validate the efficacy of sura-vec, with the primary endpoint being a greater than two-step improvement in the Diabetic Retinopathy Severity Scale (DRSS) at one year. The collaboration between REGENXBIO and AbbVie is reinforced by a revised milestone payment structure, with REGENXBIO poised to receive $100 million upon the first subject being dosed in the Phase IIb/III trial, and an additional $100 million for the first subject in a subsequent Phase III trial. This financial backing highlights the confidence both companies have in sura-vec as a transformative treatment option for millions affected by diabetic retinopathy, which is a leading cause of blindness among working-age adults.
Steve Pakola, M.D., Chief Medical Officer of REGENXBIO, articulates the potential of sura-vec to revolutionize the management of diabetic retinopathy, aiming to improve patients’ quality of life and vision outcomes. The encouraging results from the Phase II ALTITUDE trial are scheduled to be presented at future medical conferences, promoting additional dialogue and interest in the therapeutic approach. Meanwhile, the site selection process for this new pivotal trial is currently underway, positioning REGENXBIO to advance its investigational therapy toward potential regulatory approval and wider patient access.
In addition to this trial, REGENXBIO continues to focus on enhancing its gene therapy platform, which aims to address various genetic disorders. By leveraging innovative delivery mechanisms and robust clinical data, the company seeks to establish itself as a leader in the gene therapy landscape, particularly in treating retinal diseases, which remain a significant area of unmet medical need. The progress of sura-vec marks a pivotal moment in REGENXBIO’s journey, setting the stage for potential breakthroughs in vision-saving therapies.