May 14 (Reuters) – Regenxbio said on Thursday that its experimental gene therapy to treat a muscle-wasting disorder met the main goal in a late-stage study.
The Rockville, Maryland-based company’s shares, however, were down 15% in premarket trading.
Regenxbio said 93% of patients with Duchenne muscular dystrophy treated with the therapy RGX-202 reached the expected level of a key protein known as microdystrophin 12 weeks after treatment.
The company said the drug was well-tolerated. It plans to seek accelerated approval in 2027.
Microdystrophin is a shortened version of dystrophin, the muscle-protecting protein missing or defective in people with the disorder.
Wall Street has been monitoring the safety profile of RGX-202, which could help it stand apart from FDA-approved competitors. The U.S. Food and Drug Administration, in November, added its most serious safety warning to the label of Sarepta’s gene therapy, Elevidys, over liver toxicity risks.
Duchenne is a rare inherited condition that mostly affects boys and weakens muscles, causing restricted mobility, breathing problems, heart disease and early death.
Two serious adverse events were reported in the study; one case of subacute myocarditis or heart inflammation was reported in an 8-year-old, and one case of asymptomatic liver injury in a 10-year-old. Both were resolved within weeks without lasting effects, the company said.
(Reporting by Padmanabhan Ananthan in Bengaluru; Editing by Sahal Muhammed)
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