Earlier on 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.
As of February 6, 2024, RGX-202 has been well tolerated with no drug-related serious adverse events in five patients aged 4.4 to 12.1 at dose level 1 (1×1014 genome copies (GC)/kg body weight) and dose level 2 (2×1014 GC/kg body weight).
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.
A reduction from baseline in serum creatinine kinase (CK) levels of 93% was observed at ten weeks.
All three patients, at dose level 1, who completed three-month trial ...