▎Edited by the WuXi AppTec content team

Neurotech Pharmaceuticals today announced that the U.S. FDA has approved Encelto (revakinagene taroretcel) for marketing, to treat type 2 macular telangiectasia (MacTel). The press release noted that this isFDA Approval for TreatmentThe First Therapy for MacTel。

Phase 2 clinical trial results show,Compared with the untreated eye, the treated eyeEnceltoTreatment significantly reduces the progression of photoreceptor loss in the eye.This Phase 2 study enrolled a total of 67 patients (99 eyes), and the eligible eyes were randomly assigned to receive treatment containing ciliary neurotrophic factor (CNTF).EnceltoImplant or sham surgery.The primary endpoint of the study was the change in the ellipsoid zone from baseline to Month 24.In patients with MacTel, an increase in the ellipsoid zone area is an indicator of disease progression and is associated with photoreceptor loss, which can lead to vision decline.

At 24 months,AcceptEnceltoCompared with the eyes that received sham surgery, the treated eyes showed a significant reduction in photoreceptor loss. The ellipsoid zone area of the sham surgery eyes increased by 0.213 square millimeters, while the treated eyes only increased by 0.148 square millimeters.The difference in the increase of MacTel lesion area (0.065 mm²) was statistically significant (p=0.030).Compared with the baseline, the proportion of eyes with an increase in ellipsoid zone area of 35% or more was significantly lower in the treatment group (p=0.045), and compared with the control group, the macular thickness in the treatment group was significantly increased (p=0.007).. Secondary clinical endpoints showed,The reading speed of the treated eye was maintained, while the reading speed of the sham-operated eye decreased (p=0.016).

EnceltoGenerally well tolerated, consistent with previous findings in patients with retinitis pigmentosa and age-related macular degeneration.EnceltoThe results are consistent. During the study, no subjects removed the implant for any reason. Most adverse events were related to surgical procedures, and in all cases, these surgery-related events were resolved within 3 months.

EnceltoIt is an ocular implant designed to deliver CNTF directly to the retina at a sustained therapeutic dose to slow disease progression.

References:

[1] Neurotech’s ENCELTO™ (revakinagene taroretcel-lwey) Approved by the FDA for the Treatment of Macular Telangiectasia Type 2 (MacTel). Retrieved March 6, 2025, from https://www.businesswire.com/news/home/20250306180680/en

[2] Neurotech Pharmaceuticals, Inc. Receives Priority Review of Biologics License Application (BLA) for NT-501 (revakinagene taroretcel) as a Treatment for Macular Telangiectasia Type 2 (MacTel). Retrieved March 4, 2025 from https://www.businesswire.com/news/home/20240620620734/en/