Regenxbio shares drop 31 percent on trial safety data
Regenxbio shares fell 31% as safety concerns hit its muscle disorder trial. While 93% of patients met the goal, two adverse events weighed on the results.
REGENXBIO INC shares fell 31% in morning trading on May 14 despite its Duchenne Muscular Dystrophy therapy meeting primary trial goals. The late-stage study for RGX-202 reported two serious adverse events that triggered investor concerns over the drug's safety profile. The market reaction underscores the high sensitivity to safety risks in the gene therapy sector, where rival treatments have faced similar hurdles.
### Safety Concerns Overshadow Protein Expression Success The trial data showed RGX-202 produced microdystrophin, a critical muscle protein, in 93% of the 31 participants after 12 weeks. Average protein expression reached 71.1% across the group, though this figure dropped to 41.6% in boys older than 8 years. These results initially met the study's main objective, as the protein is typically missing or defective in patients with the genetic disorder.
