Cell-Based Ciliary Neurotrophic Factor Therapy for Macular Telangiectasia Type 2
Emily Y. Chew, Mark Gillies, Glenn J. Jaffe, Alain Gaudric, Cathy Egan, Ian J. Constable, Traci E. Clemons, Thomas M. Aaberg, D Manning, Thomas C. Hohman, Alan C. Bird, Martin Friedlander
Abstract
BACKGROUND: Revakinagene taroretcel (NT-501) is an encapsulated cell therapy producing ciliary neurotrophic factor that slowed retinal degeneration in patients with macular telangiectasia type 2 (MacTel) in phase 2 trials. METHODS: /24 months). Secondary outcomes included changes in retinal sensitivity, reading speed, and National Eye Institute Visual Function Questionnaire 25 (NEI VFQ-25) scores (range, 0 to 100; higher scores indicate better function). Safety end points included the proportion of participants experiencing one or more treatment-emergent serious adverse event(s) and loss of 15 or more letters in best-corrected visual acuity (BCVA). Delayed dark adaptation and miosis were among the monitored adverse events. RESULTS: /24 months (95% CI, -0.089 to -0.008; P=0.02). Retinal sensitivity and reading-speed changes between groups were inconsistent in the trials. NEI VFQ-25 scores, BCVA loss, and treatment-emergent serious adverse events did not differ between treatment groups. Miosis was experienced by 17% and 14% of participants receiving NT-501 in NTMT-03-A and NTMT-03-B, respectively, and by none of the participants in sham groups. Delayed dark adaptation was experienced by 17% and 24% of participants receiving NT-501 in NTMT-03-A and NTMT-03-B, respectively, by none in the NTMT-03-A sham group, and by 2% in the NTMT-03-B sham group. CONCLUSIONS: NT-501 for MacTel resulted in statistically significantly reduced EZA loss compared with sham procedures. (Funded by Neurotech Pharmaceuticals; ClinicalTrials.gov numbers, NCT03316300 and NCT03319849.).