Company: RGNX
Filing Date: 2025-03-13
Form Type: 10-K
Source: 0000950170-25-038770
Chunk: 79

Company: REGENXBIO Inc.
Filing Date: 2025-03-13
Form: 10-K
Item: Item 1
Chunk 79
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 eye. As of September 11, 2024, ABBV-RGX-314 has been well tolerated in the treated second eye with no drug-related serious adverse events and no cases of intraocular inflammation observed. At nine months post-administration, patients saw a 97% reduction in anti-VEGF treatment burden and sustained vision and anatomy. We believe these data demonstrate the potential of ABBV-RGX-314 to preserve vision long-term for patients with wet AMD as a one-time treatment for both eyes. Bilateral disease impacts a significant number of patients with wet AMD.

In February 2024, we presented updated interim data at the Angiogenesis, Exudation and Degeneration annual meeting from the Phase II pharmacodynamic study designed to evaluate the same dose levels being used in the two pivotal trials. Data demonstrated that ABBV-RGX-314 manufactured using REGENXBIO's NAVXpress platform process was well tolerated at both dose levels. Through month 6, ABBV-RGX-314 in all cohorts exhibited similar and expected protein levels along with stable to improved best corrected visual acuity and central retinal thickness, as well as meaningful reductions in anti-VEGF burden, with the majority of subjects remaining injection-free.

In October 2022, we announced data from the Phase I/IIa long-term follow-up study of ABBV-RGX-314 for the treatment of wet AMD using subretinal delivery. As of August 29, 2022, ABBV-RGX-314 continued to be generally well-tolerated in the long-term follow-up study (n=37). Nine SAEs were reported in four patients, none of which were considered related to ABBV-RGX-314. Patients treated with ABBV-RGX-314 continued to demonstrate a long-term, durable treatment effect in Cohort 3 up to four years and in Cohort 4 up to three years. Stable to improved visual acuity was observed, with a mean BCVA of +12 letters from baseline at four years for Cohort 3 patients and -5 letters from baseline at three years for Cohort 4 patients following ABBV-RGX-314 administration. 

Two-year findings from this study were published in The Lancet in March 2024, in a paper titled "Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study."

Clinical Development