Regenxbio released positive new data from a Phase 2 trial investigating its experimental one-time gene therapy for bilateral wet age-related macular degeneration (wet AMD).
In the sub-study, the AbbVie-partnered treatment, called ABBV-RGX-314, was administered using a subretinal delivery in the second eye of nine previously treated patients who received the first dose in the Phase 1/2a or bridging studies. Nine months after the second treatment, patients saw a 97% reduction in annualized anti-VEGF treatment burden; 100% of patients required either no supplemental injection or just one; and 78% of patients were completely injection-free. There were no drug-related serious adverse events.
The data were presented Monday at the American Academy of Ophthalmology’s annual meeting.
“This is the first time we have performed bilateral treatment for wet AMD patients,” Arshad Khanani, director of clinical research at Sierra Eye Associates and study investigator, said in a news release. He added that the results “highlight the potential of ABBV-RGX-314 as a one-time effective treatment option for patients with wet AMD.”
Stifel analysts wrote in a note on Monday that although this is an open-label study with “a relatively small sample size, these data provide strong early support” that the treatment “can be dosed bilaterally in wAMD without loss of efficacy or an elevated safety risk.”
These data follow interim results released in January from another Phase 2 trial in patients with wet AMD. In that study, patients saw an 80% reduction in annualized injection rate of anti-VEGF treatments, and 50% of patients remained injection-free at the highest dose. The treatment also showed “stable” best corrected visual acuity, the primary endpoint.
AbbVie and Regenxbio first partnered in 2021, with the pharma company paying $370 million upfront with another $1.38 billion in milestones on the table. AbbVie is leading development and global commercialization.