Roger A. Goldberg, MD: Pooled Visual Function Data of NT-501 for MacTel

NT-501 ocular implant achieved functional and structural benefit in eyes with macular telangiectasia type 2 (MacTel), according to late-breaking data presented at the 128th Annual American Academy of Ophthalmology (AAO) Meeting.

In an interview with Roger Goldberg, MD, MBA, Bay Area Retina Associates, described the analysis of pooled visual function data from 3 randomized, controlled clinical trials evaluating NT-501 for patients with MacTel, including a Phase 2 study and two Phase 3 studies

“We know MacTel tends to start paracentrally and expand into the fovea, so most of the patients who enrolled had good vision to start, and we saw that the vision was stable over the course of the two years,” Goldberg told HCPLive.

MacTel is a progressive, neurodegenerative disease of the retina leading to the deterioation of central vision, significantly impacting quality of life. NT-501 delivers CNTF, a neuroprotective protein that promotes the survival and maintenance of photoreceptors, for the treatment of chronic retinal diseases, including MacTel.

With the provision of sustained delivery of CNTF, NT-501 seeks to slow retinal degeneration and potentially improve long-term visual outcomes. As MacTel is a rare disease, Goldberg indicated the pooled data set allows for the evaluation of a correlation between structural observations and functional outcomes in these patients to determine how to better protect vision.

Baseline characters were similar in each clinical trial, with only small differences between methodologies, with assessment of the effect of NT-501 over a 2-year period. Upon analysis, NT-501 achieved a 36% reduction in the primary structural endpoint, ellipsoid zone (EZ) area over 2 years in patients with MacTel.

Regarding the functional endpoints, the pooled data showed a 68% reduction in monocular reading speed loss over 2 years, favoring NT-501-treated eyes. Microperimetry data showed a nearly 35% reduction in aggregate sensitivity loss.

Best-corrected visual acuity (BCVA) data revealed a 1 letter difference favoring NT-501-treated eyes, but Goldberg indicated most patients entered the trials with very good vision. The application for NT-501 has a Prescription Drug User Fee Act (PDUFA) target date of December 17, 2024.

“We’re hopeful to have this technology available to our patients, hopefully as soon as early next year, and it’s nice that we’re able to correlate structure and function in terms of a protective benefit in both of these categories with NT-501 for our patients with MacTel,” Goldberg told HCPLive.

References

1. _{Goldberg RA. Pooled Visual Function Data of Neurotrophic Factor–Producing Revakinagene Taroretcel in People With Macular Telangiectasia Type 2. Presented at the American Academy of Ophthalmology (AAO) 2024 Meeting. Chicago, Illinois. October 18-21, 2024.}
2. _{Neurotech receives priority review of Biologics License Application (BLA) for NT-501 (Revakinagene Taroretcel) as a treatment for Macular Telangiectasia Type 2 (MacTel) . neurotech. June 20, 2024. Accessed October 20, 2024. https://www.neurotechpharmaceuticals.com/receives-priority-review-for-bla-for-nt-501/.}