ROCKVILLE, Md. - REGENXBIO Inc. (NASDAQ:RGNX) announced interim results from its Phase I/II AFFINITY DUCHENNE® trial of RGX-202 for Duchenne muscular dystrophy (Duchenne), showing encouraging signs of efficacy and safety.
A 12-year-old patient at dose level 2 exhibited 75.7% of normal microdystrophin expression levels after three months and a 77% reduction in serum creatinine kinase (CK) levels at ten weeks, indicating potential clinical improvement.
The trial, which includes patients aged 4 to 11, is evaluating the safety and effectiveness of RGX-202, a gene therapy candidate. Thus far, no drug-related serious adverse events have been reported. All patients who reached the three-month follow-up have completed the immunosuppression regimen as per study protocol.
Kenneth T. Mills, President and CEO of REGENXBIO, highlighted the need for treatment options for boys older than 7 years and expressed commitment to transparency with data for the Duchenne community. Initial caregiver observations suggest improvements in strength and motor function in boys treated with RGX-202.
Dr. Aravindhan Veerapandiyan from Arkansas Children's Hospital noted the importance of treatment options that can alter the disease trajectory and expressed optimism about the safety and early signs of improvement in trial participants.
REGENXBIO plans to determine the pivotal dose by mid-2024 and aims to initiate a pivotal trial in the second half of the year. The company intends to use microdystrophin expression as a surrogate endpoint to support a Biologics License Application filing through the accelerated approval pathway.
The AFFINITY DUCHENNE trial is a dose-escalation and expansion study assessing a one-time intravenous dose of RGX-202. The trial design was influenced by community engagement and aims to evaluate various clinical endpoints, including microdystrophin protein levels and strength and functional assessments.
REGENXBIO, founded in 2009, focuses on gene therapy medicines and has a pipeline of AAV Therapeutics for retinal and rare diseases. The company's NAV AAV8 vector, used in RGX-202, is part of an established platform with a history of clinical trials.
The information provided is based on a press release statement.
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