Revakinagene Taroretcel Reduces Progression of Macular Telangiectasia Type 2

Revakinagene taroretcel (NT-501) significantly reduces anatomical progression of macular telangiectasia type 2 (MacTel) through 24 months, according to research presented at the American Society of Retina Specialists 41st Annual Scientific Meeting, held in Seattle, Washington, July 28-August 1, 2023. NT-501 treatment-related serious adverse events (SAES) are uncommon, mostly transient, and related to surgical complications, according to the report.  

“MacTel is a neurodegenerative disease resulting in photoreceptor atrophy and loss of vision. There is currently no proven treatment,” according to the report. “NT-501 is a first-in class encapsulated cell therapy inserted surgically into the vitreous that produces sustained levels of ciliary neurotrophic factor (CNTF).”

This research was led by Emily Chew, MD, the deputy director of the Division of Epidemiology and Clinical Applications and the Deputy Clinical Director for the National Eye Institute at the National Institutes of Health.

The study sought to evaluate the efficacy and safety of revakinagene taroretcel in patients with MacTel. 

Participants diagnosed with MacTel with ellipsoid zone (EZ; inner segment/outer segment [IS/OS]) loss between 0.16 and 2.00 mm² were randomly assigned 1:1 to NT-501 or sham treatment in the study eye (120 participants in NTMT-03-A and 119 in NTMT-03-B were included). Both the evaluators and participants were masked to the treatment assignments, while the ophthalmologists and clinic coordinators were unmasked.

The researcher found that the rate of EZ IS/OS area loss from baseline through month 24 was 56.4% lower in the group treated with revakinagene taroretcel compared with the sham groups in NTMT-03-A (NT-501, 0.074 mm² [95% CI, 0.049-0.099]; sham, 0.170 mm² [0.145-0.195]; P <.0001) and 29.2% lower in NTMT-03-B (NT-501, 0.116 mm² [0.088-0.144]; sham, 0.164 mm² [0.134-0.193]; P =.0210). 

The proportion of participants with a reduction of 15 early treatment diabetic retinopathy study (ETDRS) letters was similar between treatment groups in both NTMT-03-A (NT-501,13.8% [8/58]; sham, 8.8% [5/57]; P =.558) and NTMT-03-B (NT-501, 3.4% [2/59]; sham, 5.6% [3/54]; P =.669). 

Overall, ocular SAEs were low, but higher in the revakinagene taroretcel group compared with the sham groups in NTMT-03-A (NT-501, 5.2% [3/58]; sham, 1.8% [1/57]) and NTMT03-B (NT-501, 6.8% [4/59]; sham, 0) and mainly transient and related to surgical complications, the report shows. 

“Treatment with NT-501 significantly reduced anatomical disease progression through 24 months in both phase 3 studies,” according to the research. “These findings demonstrate that intravitreal delivery of CNTF via encapsulated cell therapy NT-501 is safe and effective for the treatment of MacTel.”