#United States #Wet AMD #Rockville #REGENXBIO #surabgene

REGENXBIO Completes Enrollment in Pivotal Trials for Wet AMD Treatment

REGENXBIO Inc. (Nasdaq: RGNX) has announced the successful completion of participant enrollment in its groundbreaking ATMOSPHERE® and ASCENT® studies. These pivotal trials are crucial in evaluating the safety and efficacy of subretinal surabgene lomparvovec, also known as sura-vec (ABBV-RGX-314), aimed at treating wet age-related macular degeneration (wet AMD). This achievement not only highlights the commitment of REGENXBIO to innovate in the field of gene therapies but also offers hope to millions affected by vision loss.

A New Era for Wet AMD Treatment

Wet AMD is a leading cause of severe vision impairment in older adults worldwide, characterized by the formation of abnormal blood vessels that leak fluid into the retina, causing damage. Current treatment protocols often involve frequent and invasive intraocular injections, which can be burdensome for patients. The completion of enrollment in these pivotal trials, which boasts over 1,200 participants across more than 200 sites, signifies a significant advance toward developing an effective and less burdensome therapy.

Dr. Steve Pakola, REGENXBIO's Chief Medical Officer, emphasized the importance of this milestone in delivering potential long-term vision preservation solutions. He remarked, “The millions of patients worldwide with wet AMD are in dire need of options that can preserve their eyesight and reduce the burden of frequent injections.” The positive results from earlier-stage trials have given researchers optimism regarding the safety and long-term durability of sura-vec.

The Pivotal Trials: ATMOSPHERE® and ASCENT®

Both trials are designed as multi-center, randomized, controlled studies to demonstrate the therapeutic efficacy of sura-vec compared to standard care options, such as ranibizumab and aflibercept. The trials focus on evaluating non-inferiority based on changes from baseline in Best Corrected Visual Acuity (BCVA) over specific periods. Key secondary objectives include assessing safety, tolerability, and the necessity for additional anti-VEGF injections, which are often required with current standard therapies.

The ATMOSPHERE study is conducted exclusively within the United States, while ASCENT extends its reach to 13 additional countries, showcasing the global interest and investment in finding new solutions to combat wet AMD.

Expectations and Future Steps

REGENXBIO anticipates that topline data from these trials will be available in the fourth quarter of 2026, marking a pivotal moment in what could become a transformative treatment option for patients suffering from wet AMD as well as diabetic retinopathy. In long-term follow-up studies, sura-vec has demonstrated durability and a well-tolerated profile, with patients benefitting from stable or improved vision outcomes for up to four years and a reduction in the need for additional injections.

A Closer Look at Sura-vec

Sura-vec operates using the NAV® AAV8 vector, designed to encode an antibody fragment that inhibits vascular endothelial growth factor (VEGF). By targeting the VEGF pathway, this therapy aims to combat the formation of the leaky blood vessels responsible for the accumulation of fluid in the retina, which leads to vision loss.

Conclusion: A Hopeful Future

The completion of enrollment in these landmark clinical trials represents a critical step in REGENXBIO's ongoing mission to develop innovative gene therapies that improve patient outcomes. With the potential of surabgene lomparvovec to reduce the treatment burden associated with wet AMD, there is hope that a new era in managing this devastating condition is on the horizon. As the company prepares to share pivotal data in the coming years, the anticipation continues to grow around the future of gene therapy in ophthalmology.

For further updates and information on REGENXBIO and its pioneering work, please visit their official website.