RegenXBio's DMD Gene Therapy Shows Promise in Phase 1/2 Trial

RegenXBio, a biotechnology company focused on developing gene therapies, has released updated data from its phase 1/2 trial of RGX-202, a potential treatment for Duchenne muscular dystrophy (DMD). The results demonstrate improved functional outcomes in older boys with the disease, positioning the company as a strong competitor in the race to bring effective DMD treatments to market.

Improved Functional Outcomes in Older Patients

The latest data update focused on five patients aged 6 to 12 years who received RGX-202. At both 9 and 12 months post-treatment, mean scores improved across multiple functional tests, including time to stand, 10-meter walk-run, and time to climb. Additionally, the company reported enhancements in a scale measuring functional motor abilities specific to children with DMD.

Dr. Steve Pakola, RegenXBio's chief medical officer, emphasized the significance of these results, stating, "All the patients are doing better than external natural history and also doing better than baseline, even when you look across a broad range of functional endpoints. These are boys that you would definitely be anticipating would be declining. Not only are we seeing stabilization, but we're actually seeing improvement."

Safety Profile and Competitive Landscape

The safety profile of RGX-202 remains favorable, with no reports of serious adverse events, central or peripheral neurotoxicity, drug-induced liver injury, or thrombocytopenia in either dose cohort. This safety record gains particular importance in light of recent concerns surrounding DMD gene therapies, following a patient death associated with Sarepta Therapeutics' Elevidys earlier this year.

RegenXBio views the safety profile of RGX-202 as a potential differentiator in the competitive DMD treatment landscape. The company is now accelerating towards pivotal data, with plans to complete enrollment in its pivotal trial by the end of this year. This timeline puts RegenXBio on track for a potential regulatory filing in 2026 and market entry in 2027.

Looking Ahead

As RegenXBio continues to advance its DMD gene therapy program, the pharmaceutical industry watches closely. The positive functional outcomes and promising safety profile of RGX-202 could potentially reshape the treatment paradigm for DMD, a devastating genetic disorder that affects thousands of children worldwide. With pivotal trial data on the horizon, RegenXBio is positioning itself as a key player in the ongoing effort to develop effective therapies for this challenging condition.