Pooled visual function data from 3 clinical trials indicates that an investigational ocular insert that continuously delivers ciliary neurotrophic factor (CNTF) to the retina can improve long-term visual outcomes for patients with the rare neurodegenerative disease macular telangiectasia (MacTel) type 2. The data were presented at the 2024 American Academy of Ophthalmology (AAO) meeting in Chicago.
“There’s tremendous excitement about the prospect of having a treatment for what’s previously been an untreatable, slowly progressive, and ultimately blinding eye disease,” said Roger A. Goldberg, MD, who presented the pooled data during the late-breaking developments session of Saturday’s Retina Subspecialty program at AAO.
Revakinagene taroretcel (NT-501; Neurotech Pharmaceuticals) is an encapsulated cell therapy that is implanted in the vitreous. It is designed to deliver sustained therapeutic doses of CNTF. The NT-501 insert was shown to be effective and safe in the phase 2 study NTMT-02 and a pair of parallel phase 3 studies, NTMT-03A and NTMT-03B. Analyzing the pooled data from all 3 studies is a way to balance out some of the variability, which is particularly important for visual function measures, which have a higher degree of test-retest variability, particularly when compared to structural endpoints, which can be measured down the micron-level. This enables researchers to hone in on the true effect of NT-501 in patients with MacTel, he said.
According to Dr. Goldberg, the NT-501 trials collectively compared 165 patients treated with the ocular insert to 162 sham-treated patients. The pooled data showed that NT-501 patients showed a 36% reduction in ellipsoid zone (EZ) loss over 2 years, as assessed with spectral domain ocular coherence tomography (SD-OCT). The NT-501 patients also showed a 68% reduction in reading speed loss and a 35% reduction in aggregate sensitivity loss detected by microperimetry.
“In the combined population, not only do we see an anatomic benefit in terms of preservation of photoreceptors, but we also see this reflected in some of the visual function endpoints, including monocular reading speed and aggregate retinal sensitivity as measured by microperimetry testing,” explained Dr. Goldberg. “We now have the potential, hopefully, to intervene and slow the disease down to ultimately prevent visual function deterioration in MacTel patients.”
In June, Neurotech Pharmaceuticals submitted a biologic license application to the US Food and Drug Administration, with a PDUFA goal date of December 17, 2024. RP
