Shots:

• The P-II (ALTITUDE) trial evaluating RGX-314 in patients with NPDR or mild PDR. Patients in Cohort 1 received RGX-314 at a dose level of 2.5x10¹¹ GC/eye (D1) & 5x10¹¹ GC/eye (D2) in Cohorts 2 & 3
• The results showed an improvement in disease severity & less disease worsening over observation control @6mos. in Cohorts 1-3, ≥2-step DRSS improvement 20% (D1: 40%, D2: 11%) vs 10% in control; any DRSS improvement 54% (D1: 60%, D2: 51%) vs 20%; worsened ≥2 steps 0% (D1: 0%, D2: 0%) vs 20%
• No differences in safety outcomes who are Nab+, BCVA remained stable in Cohorts 1-3 @6mos., was well tolerated in 50 patients from Cohorts 1-3 with no drug-related serious AEs. The trial has been expanded to include higher third-dose levels with patients stratified by DRSS levels

Ref: PRNewswire | Image: REGENXBIO