#United States #Rockville #Duchenne #REGENXBIO #RGX-202

REGENXBIO Reveals Positive Interim Data for Duchenne Muscular Dystrophy Trial

REGENXBIO Inc. has shared optimistic interim data from its Phase I/II AFFINITY DUCHENNE® trial focused on RGX-202, a groundbreaking gene therapy aimed at treating Duchenne muscular dystrophy (DMD). The announced data, which will be presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference by Dr. Carolina Tesi-Rocha from Stanford University, showcases RGX-202’s potential to shift the disease's trajectory positively.

In the study, pivotal dose participants exhibited improvements in functional measures across the board when compared to external controls over a period of one year. Impressively, cardiac MRI results indicated that heart health remained stable among participants, reinforcing RGX-202’s promise as a viable treatment option.

Trial Highlights

• - Participant Background: The trial included patients aged between 6 to 12 years, with an evident positive impact on their physical capabilities as determined by various assessments like the North Star Ambulatory Assessment (NSAA) and timed functional tests such as 'Time to Stand' and '10 Meter Walk-run'. Notably, five out of seven pivotal dose participants were aged 8 or older at the time of dosing, a crucial age where functional decline is typically anticipated.

• - Functional Outcomes: In this interim analysis, subjects improved remarkably. Seven pivotal dose participants averaged an increase of +4.9 points on the NSAA compared to expected trajectories without treatment—an impressive result considering the usual progression of DMD in such age groups. The oldest group of patients (aged 8+) noted an even larger average improvement of +5.2 points.

• - Cardiac Health Stability: The study employed MRI technology to assess cardiac function, wherein metrics like the mean left ventricular ejection fraction were closely monitored. The analysis revealed a monitorable stability at the one-year mark, suggesting a favorable cardiac profile for RGX-202.

Safety Profile

The safety profile exhibited during the Phase I/II trial remained favorable, with no severe adverse events or safety concerns raised thus far. Initial biomarkers suggest RGX-202 has a high expression rate, supporting its localization to muscle tissue without accompanying liver harm. Representative measures like gamma-glutamyl transferase levels remained within normal limits up to two years post-treatment, providing added confidence in RGX-202’s safety for young patients.

Forward-Looking Statements

Dr. Steve Pakola, Chief Medical Officer of REGENXBIO, expressed optimism regarding the future of RGX-202, emphasizing the team's dedicated and comprehensive approach toward patient safety and overall efficacy demonstrated thus far. With a topline data release anticipated in early Q2 2026, REGENXBIO plans to engage with the FDA as it works toward a Biologics License Application (BLA).

Understanding Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a severe degenerative condition, primarily affecting boys and leading to progressive muscle degeneration. It’s caused by mutations in the dystrophin gene, resulting in weakened muscle fibers. Without effective treatments, those affected face a loss of mobility and independence, and in severe cases, premature death.

As a leader in gene therapy advancements, REGENXBIO’s RGX-202 may represent a significant leap forward for those grappling with the challenges posed by Duchenne muscular dystrophy. The potential of this investigational therapy could drastically improve the quality of life for many young patients and contribute significantly to ongoing clinical advancements in genetic therapies.

In conclusion, the promising data revealed further solidifies RGX-202’s potential as a transformative treatment for Duchenne muscular dystrophy, reinforcing hope for better management of this challenging condition among affected families.