Macular telangiectasia cell therapy meets primary endpoints in phase 3 trials
Click Here to Manage Email Alerts
WAILEA, Hawaii —NT-501, an encapsulated cell therapy for the treatment of macular telangiectasia type 2, met its primary endpoints across two phase 3 trials, according to a speaker at Retina 2024.
“Treatment with NT-501 vs. sham preserved photoreceptors in patients with MacTel 2, thus meeting the primary endpoint,” Jiong Yan, MD, said.
The NTMT-03-A and NTMT-03-B trials randomized patients 1:1 to treatment with NT-501 (revakinagene taroretcel, Neurotech Pharmaceuticals) or sham to investigate the cell therapy’s efficacy and safety. The trials involved 115 and 113 patients, respectively. Inclusion criteria included an ellipsoid zone (EZ) break between 0.16 mm2 and 2.00 mm2 and a best corrected visual acuity of greater than or equal to 54 EDTRS letters — a Snellen equivalent of 20/80 or better. Only patients aged older than 21 years and younger than 80 years were included.
The cell therapy is implanted into the eye and anchored to the sclera, where it is designed to protect photoreceptor cells with long-term sustained levels of ciliary neurotrophic factor (CNTF).
The primary endpoint of both trials was the rate of change in the area of EZ loss from baseline through 24 months. In a post-hoc analysis, patients with a preservation of EZ area of more than 20% vs. sham were categorized as responders, while those with more than 50% vs. sham were categorized as high responders. Patients treated with NT-501 had a significantly higher chance of receiving responder or high responder status.
“Factors associated with higher odds of achieving responder status with NT-501 vs. sham included lower baseline EZ area loss, younger age and a lack of foveal involvement,” Yan said.
Patients in NTMT-03-A had a smaller median EZ loss when compared with group B at baseline.
According to Yan, NTMT-03-A saw a 55% reduction in EZ zone area loss through 2 years after treatment overall, while NTMT-03-B saw a 31% reduction after treatment through the same time period. NT-501 was well tolerated as most ocular treatment adverse events, including delayed dark adaptation and miosis, were seen as mild or transient.
The few cases of serious ocular treatment emergent events were related to surgery and were resolved with topical steroids, Yan said.
“Treating macular telangiectasia early in disease progression may increase the likelihood of achieving greater reduction in further progression of the disease,” she said.
Collapse
Yan J. Baseline characteristics associated with treatment response across phase 3 studies of ciliary neurotrophic factor — Producing Revakinagene Taroretcel (NT-501) in people with Macular Telangiectasia Type 2. Presented at: Retina 2024; Jan. 13-19, 2024; Wailea, Hawaii.
Read more about
Click Here to Manage Email Alerts