Regenxbio's (RGNX) Duchenne muscular dystrophy gene therapy appears approvable, with efficacy comparable to Sarepta Therapeutics (SRPT) and a more favorable safety profile, as the company moves toward submitting its rolling biologics license application, RBC Capital Markets said Thursday in a report.
Regenxbio is on track to file the first module of the BLA this quarter and expects to complete the submission in Q1 2027, RBC said. By that time, the company anticipates having functional data from roughly half of the 30 patients in its fully enrolled pivotal trial, the report said.
RBC said the confirmatory study is also fully enrolled and designed to compare two-year functional outcomes against established benchmarks for disease progression, while a larger placebo-controlled trial recommended by European regulators is planned for 2027 to support global filings.
RBC reiterated its outperform rating on Regenxbio stock and $19 price target.
Price: $10.47, Change: $-0.81, Percent Change: -7.18%