Benzinga | Feb 16, 2021 09:09AM EST

Regenxbio Gene Therapy Shows Treatment Benefit In Wet AMD Study

* Regenxbio Inc (NASDAQ: RGNX) has reported additional interim data from Cohorts 4 and 5 of its Phase 1/2a trial evaluating RGX-314 for the treatment of wet age-related macular degeneration (wet AMD) and Cohort 3 of its Long-Term Follow-Up (LTFU) study.

* As of January 22, RGX-314 continued to be generally well-tolerated across all cohorts, with 20 serious adverse events (SAEs) reported in 13 patients, including one possibly drug-related SAE of a significant decrease in vision in Cohort 5.

* Patients in Cohorts 4 and 5 at 1.5 years after administration of RGX-314 demonstrated stable visual acuity and decreased central retinal thickness (CRT).

* A meaningful reduction in anti-VEGF treatment burden was observed in both Cohorts 4 and 5 compared to the mean annualized injection rate during the 12 months before RGX-314 administration.

* Patients in Cohort 4 and Cohort 5 received a mean of 4.4 injections and 1.7 injections over 1.5 years following administration of RGX-314, a 58.3% and 81.2% reduction in anti-VEGF treatment burden, respectively.

* Following the Phase 1/2a trial, patients can enroll in a long-term study to assess the safety and efficacy up to five years after RGX-314 administration.

* All six patients from Cohort 3 of the Phase 1/2a trial enrolled in the LTFU study and demonstrated a mean BCVA improvement of +12 letters from baseline at three years.

* Patients also demonstrated long-term reductions in anti-VEGF treatment burden over three years with a mean annualized rate of 2.4 anti-VEGF injections, a 66.7% reduction.

* Price Action: RGNX stock closed 0.8% lower at $46.96 on Friday.