By Chris Wack
Regenxbio shares were 13% higher at $8.79, after the company said it saw positive interim data from two new patients in a Phase I/II trial of RGX-202, a differentiated investigational gene therapy for Duchenne muscular dystrophy.
The company said the new data from the ages 1 to 3 cohort builds on the favorable safety and efficacy profile seen in ages 4 and older.
Regenxbio said it plans to continue to advance the trial toward completing enrollment this year and BLA submission mid-2026.
In a patient aged 3 at dosing, microdystrophin expression was measured to be 122.3% compared to control. In a patient aged 7 years old, RGX-202 microdystrophin expression was measured to be 31.5% compared to control.
The company said that as of Feb. 21, RGX-202 was well tolerated with no serious adverse events and no adverse events of special interest. Common drug-related adverse events included nausea, vomiting and fatigue. All were resolved and are typically anticipated with gene therapy administration.
The stock hit its 52-week low of $5.62 on March 4, and is down 61% in the past 12 months.
Write to Chris Wack at chris.wack@wsj.com
(END) Dow Jones Newswires
March 19, 2025 10:15 ET (14:15 GMT)
Copyright (c) 2025 Dow Jones & Company, Inc.
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