Company: RGNX
Filing Date: 2025-03-13
Form Type: 10-K
Source: 0000950170-25-038770
Chunk: 81

Company: REGENXBIO Inc.
Filing Date: 2025-03-13
Form: 10-K
Item: Item 1
Chunk 81
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 of which were considered drug related. For patients in dose levels 1 and 2 (n=50), common ocular treatment-emergent adverse events in the study eye through one year included conjunctival hemorrhage and conjunctival hyperemia. Three patients had mild intraocular inflammation, which resolved on topical corticosteroids. Six patients had mild to moderate episcleritis and have resolved on topical corticosteroids. At one year, dose level 2 in NPDR patients prevented disease progression as measured by the Early Treatment Diabetic Retinopathy Study-Diabetic Retinopathy Severity Scale. Dose level 2 reduced the risk of developing vision-threatening events by 89% in these patients. 

NPDR patients treated with ABBV-RGX-314 at dose levels 1 and 2 demonstrated clinically meaningful improvements in disease severity and reduction of vision-threatening events. In dose level 2, patients with baseline NPDR:

•100% demonstrated stable to improved disease severity

o70.8% achieved ≥1 step improvement vs. 25.0% in control 

o0% worsened ≥2 steps vs. 37.5% in control

•4.2% developed vision-threatening events vs. 37.5% in control

In August 2024, we announced the ALTITUDE trial is enrolling a new cohort of patients with center-involved DME. DME is a vision-threatening complication of DR. Patients will receive a one-time, in-office injection of ABBV-RGX-314 at dose level 4 (1.5x10e12 GC/eye) with short course prophylactic steroid eye drops.

A successful end-of-Phase II meeting with the FDA occurred in the fourth quarter of 2024. In January 2025, AbbVie and REGENXBIO announced they will plan a Phase III program. The program is expected to support global regulatory submissions.

RGX-202 for the Treatment of Duchenne

We believe that RGX-202 has the potential to serve as a second-to-market, differentiated gene therapy treatment for Duchenne. 

RGX-202 is our investigational AAV Therapeutic for the treatment of Duchenne, a rare disease caused by mutations in the gene responsible for making dystrophin, a protein of central importance for muscle cell structure and function. Without dystrophin, muscles throughout the body degenerate and become weak, eventually leading to loss of movement and independence, required support for breathing, cardiomyopathy