Regenxbio Inc RGNX revealed on Wednesday new interim information from two extra sufferers within the Part 1/2 portion of the AFFINITY DUCHENNE trial of RGX-202, an investigational gene remedy for Duchenne muscular dystrophy.
Outcomes had been offered on the 2025 Muscular Dystrophy Affiliation (MDA) Medical & Scientific Convention.
At dosing, microdystrophin expression was 122.3% increased in a affected person age three in comparison with the management.
In a affected person age seven, RGX-202 microdystrophin expression was 31.5% increased than within the management.
In all sufferers, RGX-202 was appropriately localized to the sarcolemma, demonstrating the differentiated assemble with the CT-Area is appropriately focusing on the muscle.
RGX-202 microdystrophin expression leads to ambulatory sufferers age eight and above are the best reported microdystrophin ranges throughout authorized or investigational gene therapies.
To assist a Biologics License Utility (BLA) utilizing the accelerated approval pathway, the first endpoint within the pivotal section of AFFINITY DUCHENNE is the proportion of members whose RGX-202 microdystrophin expression is ≥10% at Week 12.
RGX-202 additionally demonstrates the best reported vector genome copies (4.9-55.4) measured by qPCR throughout authorized or investigational gene therapies.
As of Feb. 21, 2025, RGX-202 was properly tolerated, with no severe adversarial occasions or AEs of particular curiosity. Widespread drug-related AEs included nausea, vomiting and fatigue. All had been resolved and usually anticipated with gene remedy administration.
Part 3 portion of the AFFINITY DUCHENNE trial is enrolling ambulatory sufferers (who can stroll) aged one and above, on monitor for BLA submission in mid-2026.
Final week, Regenxbio reported a fourth-quarter lack of $1.01 per share, beating the consensus of $1.15.
Gross sales decreased barely from $22.2 million to $21.21 million, lacking the consensus of $23.75 million.
Regenxbio expects its money, money equivalents and marketable securities of $244.9 million as of Dec. 31, 2024, and the $110 million upfront cost to be acquired underneath the Nippon Shinyaku collaboration to fund its operations into the second half of 2026.
RGNX Value Motion: Regenxbio inventory is up 5.90% at $8.26 at publication Wednesday.
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