Regenxbio announces initial efficacy data from phase 1/2 AFFINITY DUCHENNE trial

Betsy Goodfellow | March 6, 2024 | News story | Research and Development |  Duchenne Muscular Dystrophy, Musculo-skeletal disorder, Regenxbio, clinical trial 

Regenxbio has announced additional interim safety data from its phase 1/2 AFFINITY DUCHENNE trial of RGX-202 in patients with Duchenne muscular dystrophy (Duchenne) between the ages of four and 11.

The data showed that all four patients, across both dosage levels, who completed three-month trial assessments indicated encouraging increases in expression of RGX-202 microdystrophin and reduction from baseline in serum CK levels. This data supports evidence of clinical improvement.

Kenneth T Mills, president and chief executive officer of Regenxbio, commented: “RGX-202 at dose level 2 is demonstrating significantly increased microdystrophin expression in a 12-year-old patient. We know there is an insufficient level of data available to the community for boys older than 7 years, and we are committed to being transparent with our data for a Duchenne community in need of new treatment options that can meaningfully impact disease. In addition, we are encouraged by the safety data at both dose levels and initial caregiver observations of strength and motor function improvement in boys treated with RGX-202. We look forward to following these patients to establish durability and greater separation from baseline, which we hope will further establish RGX-202 as an important option among treatments in development.”

Betsy Goodfellow