Pooled Analyses of Anatomic and Visual Function Data of Revakinagene Taroretcel-lwey in Participants with Macular Telangiectasia Type 2

PURPOSE: To assess anatomic and visual function data from 3 randomized, sham-controlled clinical trials of revakinagene taroretcel-lwey in participants with macular telangiectasia type 2 (MacTel). DESIGN: This study pooled data from 1 Phase 2 (NTMT-02) clinical trial and 2 identically designed Phase 3 (NTMT-03-A and NTMT-03-B) clinical trials of revakinagene taroretcel-lwey in MacTel. PARTICIPANTS: In total, 67 participants (99 eyes) from NTMT-02, 115 participants (115 eyes) from NTMT-03-A, and 113 participants (113 eyes) from NTMT-03-B were included in the study. The pooled population included 327 eyes from 295 participants (165 participants who received revakinagene taroretcel-lwey and 162 who underwent the sham surgical procedure). METHODS: Assessments were conducted on data from the individual trials as well as on a single pooled population from all 3 trials. MAIN OUTCOME MEASURES: The rate of ellipsoid zone (EZ) area loss over 24 months and changes from baseline in monocular reading speed loss, aggregate retinal sensitivity loss measured by microperimetry, and best-corrected visual acuity (BCVA) were assessed. RESULTS: /24 months; 95% CI, 0.154, 0.217) (nominal P = 0.003). A 68.2% reduction in monocular reading speed loss at 24 months from baseline was observed with revakinagene taroretcel-lwey (-4.65 words per minute [WPM]; 95% CI, -9.58, 0.27) versus sham (-14.60 WPM; 95% CI, -20.41, -8.79) (nominal P = 0.01). Across the 3 trials, a 36.6% reduction in aggregate retinal sensitivity loss was reported over 24 months with revakinagene taroretcel-lwey (28.47 decibels; 95% CI, 6.62, 50.31) compared with sham (44.85 decibels; 95% CI, 36.99, 52.72) (nominal P < 0.001). Both treatment groups experienced minimal changes in BCVA over 24 months. CONCLUSIONS: In this pooled analysis, revakinagene taroretcel-lwey reduced the rate of MacTel disease progression and preserved visual function over 24 months as shown by reductions in the rate of EZ area loss, reading speed loss, and aggregate retinal sensitivity loss.