ROCKVILLE, Md. - REGENXBIO Inc. (NASDAQ:RGNX) has shared new interim data from its ongoing Phase I/II AFFINITY DUCHENNE® trial, evaluating RGX-202, an investigational gene therapy for Duchenne muscular dystrophy (Duchenne). The data presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference showed positive biomarker results in patients aged 1-3 years, with one 3-year-old patient demonstrating microdystrophin expression levels at 122.3% compared to control.
These findings add to the consistent and robust microdystrophin and transduction levels observed across all treated age groups. The trial is currently enrolling ambulatory patients aged 1 and above, with plans for a Biologics License Application (BLA) submission by mid-2026. The stock has shown significant momentum recently, gaining nearly 17% in the past week, though it remains well below its 52-week high of $23.14.
The safety profile of RGX-202 remains encouraging, with no serious adverse events (SAEs) or adverse events of special interest (AESIs) reported as of February 21, 2025. Common drug-related adverse events included nausea, vomiting, and fatigue, which were resolved and are typically expected with gene therapy administration.
Dr. Steve Pakola, Chief Medical Officer of REGENXBIO, highlighted that RGX-202 is the only next-generation gene therapy for Duchenne in a pivotal phase trial and expressed optimism about the therapy’s potential to serve a wide age range of patients. Dr. Carolina Tesi-Rocha from Stanford Children’s Hospital also expressed encouragement by RGX-202’s profile based on the microdystrophin expression and biomarker data.
RGX-202 has demonstrated the highest reported vector genome copies across approved or investigational gene therapies, which may be attributed to a differentiated construct with the CT-Domain targeting the muscle appropriately.
Previous reports from November 2024 indicated functional improvements in the first five participants, exceeding external natural history controls. Additional interim functional data is expected to be shared in the first half of 2025, with the next earnings report scheduled for April 30, 2025. InvestingPro analysis suggests the stock is currently fairly valued, with additional insights available in the comprehensive Pro Research Report, which provides detailed analysis of REGENXBIO’s financial health and market position.
Duchenne muscular dystrophy is a severe, progressive disease affecting approximately 1 in 3,500 to 5,000 boys born each year worldwide. It leads to muscle degeneration, loss of movement, and eventually premature death. For investors tracking REGENXBIO’s progress in this crucial market, InvestingPro offers 8 additional exclusive tips and comprehensive financial metrics to better understand the company’s potential and risks.
The data presented is based on a press release statement from REGENXBIO Inc. and provides a glimpse into the ongoing research and development efforts in gene therapy for Duchenne muscular dystrophy.
In other recent news, Regenxbio Inc. reported its fourth-quarter 2024 financial results, with earnings per share (EPS) of -$1.01, outperforming analysts’ expectations of -$1.13. The company’s revenue for the quarter was $21.2 million, falling short of the forecasted $23.47 million. For the full year, Regenxbio’s revenue reached $83.3 million, with a net loss of $227.1 million. H.C. Wainwright adjusted the price target for Regenxbio to $34.00, maintaining a Buy rating due to the potential approval of RGX-121, a gene therapy for Mucopolysaccharidosis II (MPS II).
Regenxbio has completed its Biologics License Application (BLA) submission for RGX-121, seeking accelerated FDA approval in the second half of 2025. Stifel analysts also reiterated a Buy rating with a $40.00 price target, citing the company’s transition to gene therapy commercialization as a key factor. The analysts noted the progress of Regenxbio’s RGX-202 program for Duchenne muscular dystrophy, with Phase 3 enrollment about 50% complete. Additionally, Regenxbio’s partner AbbVie is advancing trials for ABBV-RGX-314 for retinal diseases, with results expected in 2026. These developments highlight Regenxbio’s strategic advancements in its gene therapy pipeline and potential growth opportunities.
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