REGENXBIO Inc. (NASDAQ:RGNX) on Thursday released topline and interim functional data from the pivotal Phase 1/2/3 AFFINITY DUCHENNE trial evaluating RGX-202, its investigational gene therapy for Duchenne muscular dystrophy.



The company said the study met its primary endpoint with high statistical significance, as 93% of participants achieved at least 10% microdystrophin expression at week 12.



REGENXBIO also reported a statistically significant correlation between microdystrophin expression and functional improvement.



 • Regenxbio stock is testing key support levels. Why are RGNX shares at support?



<h2>Functional Improvement Supports Therapy Potential</h2>



The pivotal portion of the study evaluated 31 ambulatory boys aged one year and older treated with RGX-202 at 2×1014 GC/kg.



According to the company, average microdystrophin expression reached 71.1% across participants and 41.6% among boys older than eight years.



In addition, 80% of participants achieved greater than 40% microdystrophin expression.



Interim one-year functional data from nine participants aged about five to 12 years showed improvements across North Star Ambulatory Assessment (functional motor abilities) scores and timed function tests, including time to stand, walk-run performance, and stair climbing assessments.



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<h2>Safety Profile Remains Favorable</h2>



REGENXBIO said RGX-202 was generally well tolerated and maintained a favorable safety profile as of the latest data cutoff.



The company reported two serious adverse events that resolved within weeks without long-term effects. One participant experienced subacute myocarditis, while another developed asymptomatic liver injury.



Probably, investors are reacting to two serious adverse events and the fact that the drug showed better efficacy in younger patients.



<h2>FDA Discussions and Commercial Plans</h2>



REGENXBIO said recent discussions with the FDA indicated the agency would evaluate RGX-202 microdystrophin expression as a surrogate endpoint based on its correlation with clinical outcomes.



The company plans to pursue accelerated approval for RGX-202 and is preparing for a potential commercial launch in 2027.



The gene therapy company has enrolled over 20 additional participants in the confirmatory trial of RGX-202 (n=30), and the company expects to complete dosing in all 60 patients across the pivotal and confirmatory trials by mid-year.



REGENXBIO expects its balance in cash, cash equivalents, and marketable securities of $150.5 million as of March 31, 2026, to fund its operations into early 2027.



RGNX Price Action: Regenxbio shares were down 36.06% at $6.42 at the time of publication on Thursday, according to Benzinga Pro. 



Over the past month, RGNX has declined about 30.97% versus a 8.6% rise in the S&amp;P 500 and is down roughly 55% year-to-date compared to the index’s 9% gain.



Photo via Shutterstock



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REGENXBIO Inc. announced positive results from its Phase 1/2/3 AFFINITY DUCHENNE trial for RGX-202, a gene therapy for Duchenne muscular dystrophy, achieving its primary endpoint with 93% of participants showing at least 10% microdystrophin expression. The therapy demonstrated a favorable safety profile, with two serious adverse events reported. The company plans to seek accelerated FDA approval and aims for a commercial launch in 2027. However, REGENXBIO's stock has seen a significant decline, down 36.06% to $6.42, amid broader market gains.

REGENXBIO Advances Duchenne Gene Therapy Toward Accelerated Approval