The investigational gene therapy RGX-202 exhibited a favorable safety profile with no serious adverse events and no evidence of liver injury, the company said.
- The only drug-related adverse events observed are typically anticipated with gene therapy administration, the company noted.
- It added that a proactive, short-course immune modulation regimen in combination with a differentiated construct and industry-leading product purity levels may contribute to a favorable safety profile for RGX-202.
- Sarepta Therapeutics’s FDA-approved DMD gene therapy Elevidys came under scrutiny last year over concerns of acute liver failure and associated patient deaths.
REGENXBIO Inc. (RGNX) on Wednesday announced positive interim data from the company’s early-to-mid stage trial of its gene therapy for Duchenne muscular dystrophy.
The investigational gene therapy RGX-202 demonstrated evidence of positively changing disease trajectory for the disease in a few patients enrolled in the trial. It further exhibited a favorable safety profile with no serious adverse events and no evidence of liver injury, the company said.
The only drug-related adverse events observed are typically anticipated with gene therapy administration, the company noted, while adding that a proactive, short-course immune modulation regimen in combination with a differentiated construct and industry-leading product purity levels may contribute to a favorable safety profile for RGX-202.
Sarepta Therapeutics’s (SRPT) FDA-approved DMD gene therapy, Elevidys, came under scrutiny last year over concerns of acute liver failure and associated patient deaths. Duchenne muscular dystrophy (DMD) is a severe, progressive genetic muscle disease affecting mostly boys.
Application Timeline
REGENXBIO now expects to share topline data from the trial in early second quarter and request a meeting with the FDA in mid-2026, ahead of filing an application seeking approval for the gene therapy.
In the meantime, the company will continue to enroll participants for its confirmatory trial, which is expected to have about 30 participants. The company said on Wednesday that it expects to have the majority of the trial enrolled at the time of the application submission.
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RGNX stock has gained nearly 54% over the past 12 months.
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