Company: RGNX
Filing Date: 2025-03-13
Form Type: 10-K
Source: 0000950170-25-038770
Chunk: 83

Company: REGENXBIO Inc.
Filing Date: 2025-03-13
Form: 10-K
Item: Item 1
Chunk 83
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-202 at dose level 2 (DL2), the pivotal dose. 

In November 2024, we reported additional interim data from the Phase I/II portion of the AFFINITY DUCHENNE trial, including functional outcomes from the first five Phase I/II participants. Results include 12-month data from three dose level 1 patients aged 4-10 and nine-month data from two dose level 2 (pivotal dose) patients aged 8 and 12. In all five participants, across both dose levels, RGX-202 demonstrated evidence of positively impacting disease trajectory, with patients demonstrating stable or improved function on NSAA and timed function tests including time to stand, 10 meter walk/run and time to climb. Results were measured against external natural history controls matched for age and baseline function. 

We also reported new biomarker and safety data in November 2024. The biomarker data continues to support consistent, high expression and transduction of RGX-202 microdystrophin. RGX-202 was appropriately localized to the sarcolemma, demonstrating the differentiated construct with the CT-Domain is appropriately targeting the muscle. In the 11 patients dosed as of November 1, 2024, RGX-202 was well tolerated with no SAEs or AESIs reported. Common drug-related AEs included nausea, vomiting and fatigue. All resolved and are typically anticipated with gene therapy administration. 

Also in November 2024, we announced that a pivotal (Phase III) trial of AFFINITY DUCHENNE had been initiated and that the first patient had been dosed under the pivotal protocol. The trial will recruit approximately 30 ambulatory patients, aged 1 and above. In March 2025, we announced that the pivotal study was nearly 50% enrolled, and we expect to complete enrollment in the study in 2025, share top line data in the first half of 2026 and submit a BLA under the accelerated approval pathway in mid-2026.

We expect to share additional Phase I/II biomarker data at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, including the first biomarker data from the cohort of patients aged 1-3. The company expects to share additional efficacy and safety data, including additional functional data, in the first half of 2025.

RGX-202 is manufactured using our proprietary, high-yielding NAVXpress platform process. This suspension-based manufacturing process has