Regenxbio said on Wednesday that interim results from an early- to mid-stage clinical study of its experimental gene therapy RGX-202 in patients with Duchenne muscular dystrophy (DMD) showed continued improvement in muscle function along with a clean safety profile. The company is testing the therapy in boys between the ages of 1 and 12 and expects to release pivotal top-line data early in the second quarter of this year.
Duchenne muscular dystrophy is a rare inherited disorder that primarily affects boys. The condition is caused by the absence of dystrophin, a protein needed to protect muscle cells. As a result, muscles weaken steadily over time. Children with the disease typically lose the ability to walk in their early teens and may later develop heart and breathing problems.
The phase 1/2 study included several children receiving RGX-202, with interim data highlighting functional outcomes after treatment. Seven children aged about 6 to 12 years who received the therapy showed functional improvement after one year across standard tests used to track how quickly Duchenne progresses.
Five of those seven participants were eight years old or older, an age at which muscle function generally declines in Duchenne patients. According to the company, all seven children gained an average of 4.9 points on a motor function scale used in Duchenne clinical studies. Among the older group, the average improvement was 5.2 points after one year of treatment.
Safety results were reported from 13 children included in the study. Regenxbio said the therapy showed no signs of liver damage or injury among participants. Concerns about liver toxicity have been associated with some gene therapies used in Duchenne muscular dystrophy.
“None of the 13 patients in the interim phase 1/2 safety dataset had serious adverse events or adverse events of special interest—including drug-induced liver injury—up to 24 months after treatment,” the company reported.
Among the 10 patients who received the pivotal dose of RGX-202, mean levels of two biomarkers associated with liver damage remained below the upper limit of normal for as long as 24 months after treatment.
The company released the updated safety findings together with additional efficacy results from the study. The data included one-year disease trajectory information measured using the North Star Ambulatory Assessment (NSAA), a scale commonly used to evaluate motor function in Duchenne muscular dystrophy. Regenxbio said the expanded dataset provided more evidence that patients who receive RGX-202 outperform external controls and the expected disease trajectory on the NSAA. One-year data also favored RGX-202 on other functional tests, such as time to stand.
The company also reported results from three patients who received a lower dose of the therapy. In those individuals, NSAA performance exceeded the expected disease trajectory two years after treatment.
RGX-202 is designed to deliver microdystrophin, a shortened form of dystrophin, the protein missing in patients with Duchenne muscular dystrophy. Regenxbio reported microdystrophin expression levels ranging from 39.7% to 97.3% across three age-based groups that received the pivotal dose. All patients recorded expression levels above 10% at Week 12.
The pivotal trial’s primary endpoint evaluates the proportion of participants whose microdystrophin expression reaches at least 10% at Week 12. Regenxbio will meet the endpoint if it replicates the result in the pivotal study.
Currently, Elevidys, developed by Sarepta Therapeutics and Roche, is the only gene therapy approved for Duchenne muscular dystrophy. Sarepta has faced increased scrutiny over the safety and effectiveness of the treatment after two non-ambulatory teenage boys died due to acute liver failure linked to the therapy.
Regenxbio said it plans to request a meeting in mid-2026 with the U.S. Food and Drug Administration to discuss a potential filing for accelerated approval of RGX-202. By the time the FDA is reviewing the therapy, the company could have 12-month functional data from most patients in the pivotal trial and have largely enrolled a 30-participant confirmatory study.
Regenxbio Shares Positive Phase 1/2 Findings
Regenxbio has released interim Phase 1/2 clinical data demonstrating encouraging functional improvements in patients receiving its investigational gene therapy RGX-202 for Duchenne muscular dystrophy (DMD). The update highlights Regenxbio’s continued progress in developing innovative gene therapies for severe genetic diseases.
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