BUZZ-Regenxbio falls after late-stage data for muscle disorder therapy
Regenxbio RGNX | 0.00 |
** Shares of drug developer Regenxbio RGNX.O fall 18.7% to $8.16 premarket
** Co says its experimental gene therapy for duchenne muscular dystrophy met the main goal of a late-stage study
** Co says 93% of participants reached at least 10% microdystrophin expression, the muscle-protecting protein missing or defective in people with Duchenne, at 12 weeks
** The trial evaluated RGX-202 in 31 ambulatory boys aged 1 year and older; two serious adverse events were reported, both were easily managed and resolved within weeks, co says
**"Two serious adverse events muddy the update and FDA feedback will be debated as we await additional clarity from RGNX’s upcoming meeting with FDA prior to BLA filing" - Leerink Partners
** Duchenne muscular dystrophy is a rare neuromuscular disease characterized by worsening muscle weakness and loss of function
** Co plans to discuss this data with the FDA at a future meeting and pursue accelerated approval for RGX-202
** Up to last close, stock down 30.3% YTD
