SRPT - Gene Therapy Player REGENXBIO's RGX-121 Marks Success In Trial Yet Faces Challenges In A Crowded Market: Analyst | Benzinga
Last Wednesday, REGENXBIO Inc (NASDAQ:RGNX) reported additional interim safety and efficacy in the Phase 1/2 AFFINITY DUCHENNE trial of RGX-202 in patients with Duchenne muscular dystrophy ages 4 to11 years old.
In new data from the third patient, aged 6.6 years, who received RGX-202 at dose level 1, RGX-202 microdystrophin expression was measured to be 83.4% compared to control at three months.
REGENXBIO also announced topline results from the Phase 1/2/3 CAMPSIITE trial of RGX-121 in patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome.
The trial met its primary endpoint with statistical significance.
Wedbush writes that the supplementary data from the Ph1/2 AFFINITY DUCHENNE trial provides additional confirmation of RGX-202’s ...