Regenxbio Reports Positive Phase 2 Data on ABBV-RGX-314 for Bilateral Wet AMD

Regenxbio announced results from the phase 2 fellow eye sub-study evaluating the subretinal delivery of ABBV-RGX-314, a gene therapy candidate, in patients with bilateral wet age-related macular degeneration (AMD). The data was presented at the American Academy of Ophthalmology (AAO) meeting by Arshad Khanani, MD, of Sierra Eye Associates.

ABBV-RGX-314, developed in partnership with AbbVie, is under investigation as a one-time gene therapy treatment for wet AMD. This study is the first to explore gene therapy in the second eye of patients with bilateral wet AMD.

"The data from the phase 2 sub-study show ABBV-RGX-314's potential to reduce the treatment burden for patients with bilateral disease," Curran Simpson, CEO of Regenxbio, said in a company news release. "We are excited to see the durable effects of the therapy, which could preserve vision long-term for millions globally."

The 9-month results from the phase 2 fellow eye sub-study included nine patients who had previously received ABBV-RGX-314 in one eye and later underwent treatment in the second eye. Key findings included:

• 97% reduction in annualized anti-VEGF treatment burden
• 100% of patients required zero or only one supplemental injection
• 78% of patients were completely injection-free
• Sustained improvements in best-corrected visual acuity (BCVA) and central retinal thickness (CRT)

Additionally, ABBV-RGX-314 demonstrated similar protein levels in both treated eyes. The therapy was well tolerated, with no serious adverse events reported.

"This is the first bilateral gene therapy treatment for wet AMD. The durability of ABBV-RGX-314, combined with its potential to drastically reduce injection frequency, offers hope for better long-term outcomes," said Dr. Khanani.