Results of NT-501 Phase 2 Study Published

January 16, 2019, Cumberland, RI and La Jolla, CA. The results of the NT-501 Phase 2 study sponsored by Neurotech Pharmaceuticals, Inc. (Neurotech) in collaboration with the Lowy Medical Research Institute (LMRI), have been published in the journal of the American Academy of Ophthalmology, Ophthalmology1. The study, a Phase 2 multi-center randomized clinical trial, tested the effect of encapsulated cell-based delivery of ciliary neurotrophic factor (CNTF) on retinal degeneration in patients with macular telangiectasia type 2 (MacTel). The paper was authored by a group of clinicians and scientists who collaborated on the project through the Macular Telangiectasia Type 2-Phase 2 CNTF Research Group.

MacTel type 2 is a rare macular degenerative disease typically diagnosed in middle age. It is primarily a neurodegenerative disease, but also affects Müller glia and blood vessels. Patients rarely experience total vision loss, but the disease nonetheless has a significant impact, through visual loss, on a patient’s quality of life. LMRI supports MacTel type 2 clinical and laboratory research and collaborated with Neurotech to test NT-501 in MacTel type 2 patients.

The NT-501, or Renexus® device, is manufactured by Neurotech. It is a novel cell-based drug delivery system. Cells encapsulated in a semipermeable hollow fiber membrane release ciliary neurotrophic factor (CNTF); CNTF has been shown to reduce photoreceptor cell loss in animal models of retinal degeneration. The implanted Renexus® device results in sustained delivery of CNTF localized to the retina, the light-sensing tissue in the back of the eye.

The Ophthalmology report describes the results of a single-masked, randomized clinical trial that tested the effects of CNTF delivered by NT-501 in participants with MacTel type 2. Participants were randomized 1:1 to surgical implantation of the NT-501 device, or sham procedure. Measures of photoreceptor health and function were assessed 24 months from patient enrollment. The study showed that NT-501 treatment slowed the progression of retinal degeneration compared with participants who were given the sham treatment. In addition, NT-501 provided functional benefit; reading speed was stabilized in patients receiving the implant.

Patients from the Phase 1 and 2 studies continue to be followed to evaluate the long-term effect of CNTF on retinal degeneration.

Dr. Martin Friedlander, President of Lowy Medical Research Institute, noted that “the mission of LMRI is to understand MacTel and bring therapies to market for this disease. The Phase 2 NT-501 clinical trial and extension study results are very encouraging, and bring us closer to the important goal of providing efficacious treatment options for people diagnosed with MacTel, a disease for which we currently have no treatments.”

Renexus® is also being studied by Neurotech to test the neuroprotective effects of CNTF in treating glaucoma. Renexus® is currently being tested in a randomized, sham controlled, masked Phase 2 glaucoma study in 54 patients. The glaucoma study is independent of the MacTel research project.

“There is currently no effective therapy available to MacTel type 2 patients”, said Richard Small, CEO of Neurotech. “We are encouraged by the neuroprotective effect of NT-501 observed in the MacTel phase 2 trial and associated extension study. A Phase 3 study to determine the safety and efficacy of Renexus ® in MacTel type 2 is currently enrolling patients in the United States, Australia, and Europe.” ( #NCT03319849/NCT03316300)

The press release is available here.