Enrollment Completed in Pivotal Trials for AMD Treatment
REGENXBIO Inc. (Nasdaq: RGNX) has successfully announced the completion of participant enrollment in its pivotal trials, ATMOSPHERE and ASCENT, which focus on the innovative gene therapy known as surabgene lomparvovec for the treatment of wet age-related macular degeneration (wet AMD).
Milestone Achieved for Gene Therapy
This achievement marks a groundbreaking moment in the field, as it represents the largest global gene therapy program ever conducted in relation to wet AMD, involving over 1,200 participants. These trials are designed to evaluate the effectiveness of surabgene lomparvovec delivered via subretinal injection, a method aimed at providing a long-lasting treatment for this debilitating eye condition.
Dr. Steve Pakola, Chief Medical Officer at REGENXBIO, shared his enthusiasm regarding the enrollment phase, stating, "Completing enrollment in this large, global pivotal program is an exciting milestone in our efforts to deliver sura-vec to patients as the potential first gene therapy for wet AMD." He expresses confidence in the necessity for new treatment options that can offer vision preservation, prevent disease progression, and alleviate the burden of ongoing intraocular injections characteristic of current treatments.
Understanding the Pivotal Trials
The ATMOSPHERE and ASCENT trials are meticulously designed as multi-center, randomized, active-controlled studies. The ATMOSPHERE trial, conducted in the United States, compares surabgene lomparvovec against ranibizumab. Simultaneously, the ASCENT trial spans across the U.S. and 13 other countries, against aflibercept. Together, these studies are crucial for supporting global regulatory submissions aimed at securing approval for this innovative therapy.
Details on Study Parameters
The primary objective of these trials is to assess non-inferiority based on the changes in Best Corrected Visual Acuity (BCVA) after 54 weeks and one year, respectively. Secondary endpoints will focus on the safety and tolerability of the treatment, changes in central retinal thickness, and the need for additional anti-VEGF injections during the study.
Promising Results from Earlier Studies
Previously conducted studies have demonstrated that surabgene lomparvovec is generally well tolerated and shows a durable treatment effect, with stable or improved vision observed in participants after up to four years. The positive outcomes from a Phase II pharmacodynamic study have further contributed to the growing anticipation for the topline data expected in the fourth quarter of 2026.
About Surabgene Lomparvovec
Surabgene lomparvovec, also identified as ABBV-RGX-314, is under evaluation as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal diseases. This innovative therapy employs the NAV AAV8 vector to encode an antibody fragment that targets vascular endothelial growth factor (VEGF), a significant contributor to the pathogenesis of wet AMD.
Exploring Wet AMD
Wet AMD leads to severe vision loss associated with abnormal blood vessel growth in the retina. Millions suffer from this condition, making it a pressing health concern in many developed countries. Current anti-VEGF therapies have transformed the landscape of treatment, showcasing their efficacy in slowing disease progression. However, they typically necessitate continuous, frequent intraocular injections, which can discourage patient adherence.
Company Overview
Founded in 2009, REGENXBIO is a biotechnology company dedicated to improving lives through gene therapy. The company is advancing a late-stage pipeline of one-time treatments aimed at addressing various diseases such as Duchenne muscular dystrophy and more. Their commitment to creating impactful treatments has positioned REGENXBIO at the forefront of gene therapy innovation.
Frequently Asked Questions
What is surabgene lomparvovec?
Surabgene lomparvovec is a gene therapy under development for wet AMD, designed to be delivered via a one-time subretinal injection to inhibit the growth of leaky blood vessels.
What are the ATMOSPHERE and ASCENT trials?
These are pivotal clinical trials aimed at evaluating the safety and efficacy of surabgene lomparvovec in treating wet AMD, involving over 1,200 participants globally.
When is topline data expected?
Topline data from these studies is expected to be released in the fourth quarter of 2026.
Why is there a need for new treatments for wet AMD?
Existing treatments require life-long frequent injections, which many patients find burdensome. New therapies such as surabgene lomparvovec aim to alleviate this burden by offering longer-lasting effects.
How does REGENXBIO contribute to the field of gene therapy?
REGENXBIO is pioneering innovative gene therapies using AAV vectors, advancing several promising treatments for various genetic and retinal conditions, significantly impacting patient care.
