Pooled data from three randomized, controlled clinical trials indicate that the NT-501 ocular implant provides both functional and structural benefits for individuals with macular telangiectasia type 2 (MacTel). The analysis, presented at the 128th Annual American Academy of Ophthalmology (AAO) Meeting, offers hope for a disease that leads to progressive deterioration of central vision.
NT-501's Impact on MacTel Progression
MacTel, a neurodegenerative disease affecting the retina, currently has limited treatment options. NT-501 delivers ciliary neurotrophic factor (CNTF), a neuroprotective protein, directly to the eye, aiming to slow retinal degeneration by supporting photoreceptor survival. The pooled data set allowed researchers to correlate structural observations with functional outcomes, offering insights into how to better protect vision in these patients.
Key Findings from the Pooled Analysis
The pooled analysis included data from a Phase 2 study and two Phase 3 studies, assessing the effect of NT-501 over a 2-year period. Baseline characteristics were similar across the trials. According to Roger A. Goldberg, MD, MBA, Bay Area Retina Associates, most patients enrolled in the trials had good vision at the start, reflecting the disease's initial paracentral impact before expanding into the fovea.
Structurally, NT-501 achieved a 36% reduction in the primary structural endpoint, ellipsoid zone (EZ) area loss, over two years in MacTel patients. Functionally, the data demonstrated a 68% reduction in monocular reading speed loss and a nearly 35% reduction in aggregate sensitivity loss, as measured by microperimetry, in NT-501-treated eyes.
Visual Acuity and Future Prospects
Best-corrected visual acuity (BCVA) data showed a marginal one-letter difference favoring NT-501-treated eyes. However, Dr. Goldberg noted that most patients entered the trials with relatively good vision, suggesting the treatment's primary benefit may be in stabilizing vision rather than significantly improving it.
Neurotech Pharmaceuticals has received priority review for the Biologics License Application (BLA) for NT-501 (Revakinagene Taroretcel), with 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," Dr. Goldberg told HCPLive.
