Business
REGENXBIO ANNOUNCES NEW POSITIVE INITIAL EFFICACY DATA FROM AFFINITY DUCHENNE® TRIAL
New three-month assessment in first patient at dose level 2 demonstrates robust microdystrophin expression Patient aged 12 years at dosing had expression
About this update from Regenxbio Inc.
[{"type":"text","content":"New three-month assessment in first patient at dose level 2 demonstrates robust microdystrophin expression Patient aged 12 years at dosing had expression level at 75.7% of controlEarly evidence of strength and motor function improvement observedOn track to initiate pivotal trial in second half of 2024 Webcast this morning, Tuesday, March 5, 8:30 a.m. ET, with principal investigatorROCKVILLE, Md., March 5, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported additional interim safety and efficacy data in the Phase I/II AFFINITY DUCHENNE® trial of RGX-202 in patients with Duchenne muscular dystrophy (Duchenne) ages 4 to11 years old, including RGX-202 microdystrophin expression from dose level 2 and video of trial clinic assessments demonstrating initial evidence of strength and functional improvement.\n\"RGX-202 at dose level 2 is demonstrating significantly increased microdystrophin expression in a 12-year-old patient,\" said Kenneth T. Mills, President and CEO, REGENXBIO. \"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.\"\n\"There is a need for treatment options for boys with Duchenne that have the potential to alter the disease trajectory,\" said Aravindhan Veerapandiyan, M.D., Arkansas Children's Hospital. \"I am very pleased with the new microdystrophin expression data from RGX-202 dose level 2. It is encouraging to see that patients are safely progressing through their trial protocol strength and motor function assessments with early observations of improvement, including in older boys.\"\nSafety UpdateAs of February 28, 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 (1x1014 genome copies (GC)/kg body weight) and dose level 2 (2x1014 GC/kg body w...