Regenxbio’s RGX-202 shows positive Phase 1/2 results in Duchenne

Regenxbio reported new, positive interim safety and efficacy data in the Phase I/II AFFINITY DUCHENNE trial of RGX-202 in patients with Duchenne muscular dystrophy ages 1 to 11 years old. RGX-202 is an investigational one-time AAV Therapeutic. In patients aged 5.8 and 8.5 who received RGX-202 at dose level 2, RGX-202 microdystrophin expression was measured to be 77.2%, and 46.5%, respectively, compared to control at three months. As of July 8, 2024, RGX-202 has been well tolerated with no serious adverse events. All seven patients who completed three-month trial assessments indicate meaningful increases in expression of RGX-202 microdystrophin and reduction from baseline in serum creatinine kinase levels, supporting evidence of clinical improvement. Regenxbio expects to complete enrollment in the dose level 2 expansion cohort early in Q3 2024 and has initiated enrollment in the cohort for boys aged 1-3. Initiation of the pivotal trial is expected in Q4 2024. Regenxbio expects to share initial strength and functional assessment data for both dose levels of the AFFINITY DUCHENNE trial in the second half of 2024.