Regenxbio

REGENXBIO has initiated dosing in the pivotal phase of its AFFINITY DUCHENNE trial, testing RGX-202 for Duchenne muscular dystrophy (DMD), with positive efficacy and safety data from the phase 1/2 portion. The trial includes two cohorts, with the pivotal trial assessing RGX-202 at 2x1014 GC/kg in 30 ambulatory patients aged 1 and older. RGX-202, encoding key regions of dystrophin, showed stable or improved function on the North Star Ambulatory Assessment (NSAA) and timed function tests. The company anticipates submitting a biologics license application (BLA) in 2026 for accelerated approval.

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REGENXBIO has initiated the pivotal phase 3 of its AFFINITY DUCHENNE trial, evaluating RGX-202 gene therapy for Duchenne muscular dystrophy (DMD), with a planned BLA submission in 2026. The trial will enroll 30 ambulatory DMD patients aged 1 and older, using microdystrophin expression as the primary endpoint. Positive functional data from earlier phases show stability or improved function in treated patients, with no serious adverse events reported.

Arshad Khanani presented positive phase 2 fellow eye sub-study data on ABBV-RGX-314 for bilateral wet AMD at AAO 2024, highlighting a 97% reduction in anti-VEGF treatment burden and potential as a one-time treatment. The sub-study showed no serious adverse events and sustained visual acuity and retinal thickness.

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Regenxbio has initiated the pivotal phase of its clinical trial for RGX-202 gene therapy in boys with Duchenne muscular dystrophy (DMD), aiming for accelerated FDA approval. New data from the Phase 1/2 study shows RGX-202 led to physical function gains, with microdystrophin expression levels at 10% or more of normal dystrophin in all participants. The pivotal study will enroll 30 DMD patients, including those as young as 1 year old, and is actively recruiting participants in multiple U.S. states.

REGENXBIO has initiated a pivotal Phase I/II/III trial for its gene therapy RGX-202 for Duchenne muscular dystrophy (DMD), following positive Phase I/II results. RGX-202, using a novel microdystrophin construct, demonstrated functional improvements and a favorable safety profile. The therapy is expected to file a Biologics License Application (BLA) in 2026 under the accelerated approval pathway, positioning REGENXBIO as a significant competitor to Sarepta Therapeutics.

Regenxbio aligns with FDA for an abbreviated approval path for its Duchenne muscular dystrophy gene therapy, expanding a Phase 1/2 study into a pivotal trial. The therapy aims to produce at least 10% normal levels of microdystrophin, with early data showing higher microdystrophin levels than Elevidys. The trial will not include an active placebo, focusing on microdystrophin production as the main goal, unlike previous studies by Pfizer and Sarepta.

REGENXBIO reports Q3 2024 financials and operational updates: RGX-202 for Duchenne Muscular Dystrophy advances in Phase I/II AFFINITY DUCHENNE trial; BLA submission for RGX-121 initiated, expected Q1 2025; positive Phase II data supports bilateral administration of subretinal ABBV-RGX-314; End-of-Phase II meeting for ABBV-RGX-314 in diabetic retinopathy accelerated to Q4 2024; conference call at 4:30 p.m. ET.