March 11 (Reuters) – Regenxbio said on Wednesday that interim data from a early-to-mid stage study of its experimental gene therapy in patients with Duchenne muscular dystrophy showed continued improvement in muscle function and a clean safety profile.
The company was testing the treatment, called RGX-202, in boys aged 1 to 12.
Duchenne is a rare, inherited disease that mostly affects boys and causes muscles to weaken steadily over time. Children typically lose the ability to walk in their early teens and later develop heart and breathing problems.
The condition is caused by the absence of dystrophin, a protein needed to protect muscle cells.
In the study, seven children aged about 6 to 12 years who received the therapy showed meaningful functional improvement in one year across standard tests used to track how quickly Duchenne progresses.
Five of these seven boys were eight or older, an age when muscle function typically declines, the company said. After one year of being on the treatment, all seven gained an average of 4.9 points on a scale that measures motor function, while the older group improved by 5.2 points.
The therapy showed no signs of liver damage or injury, which is a known concern for gene therapies, among the 13 children in the study.
Currently, Sarepta Therapeutics and Roche’s Elevidys is the only approved gene therapy for the condition.
Sarepta has faced heightened scrutiny over the safety and effectiveness of Elevidys after two non-ambulatory teenage boys died due to acute liver failure linked to the therapy.
Regenxbio expects to release further trial results early in the second quarter this year.
(Reporting by Kamal Choudhury and Siddhi Mahatole in Bengaluru; Editing by Leroy Leo and Alan Barona)
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