NT-501

Unassigned

New Medicines

Renexus Macular telangiectasia, type 2

Information

Renexus
New molecular entity
Neurotech Pharmaceuticals
Neurotech Pharmaceuticals

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Feb 19Granted fast track status in US [4].
Jul 12NT 501 granted orphan drug status for retinal (macular) telangiectasia type 2 by the FDA [4].

Category

An intraocular polymer implant that contains genetically modified human retinal epithelial cells to continuously secrete ciliary neurotrophic factor (CNTF) into the back of the eye for sustained periods of time.
Macular telangiectasia (MacTel) is a disease affecting the macula, causing loss of central vision. MacTel develops when there are problems with the tiny blood vessels around the fovea. The most common form is Type 2 MacTel. The blood vessels around the fovea leak, become dilated (widen), or both. In some cases, new blood vessels form under the retina and they can also break or leak. Fluid from leaking blood vessels causes the macula to swell or thicken [1]. Prevalence of MacTel 2 is 0.1% [2].
Macular telangiectasia, type 2
Intraocular

Trial or other data

Sep 20PIII (NCT03319849) study is no longer recruiting; collection of primary outcome data due to complete Aug 22 [5].
Mar 20PIII (NCT03319849) study is recruiting [3].
Dec 19PIII (NCT03316300) study is recruiting [3].
Feb 18PIII study to determine the safety and efficacy of NT 501 in patients with macular telangiectasia type 2 starts (NTMT-03-B; NCT03319849). The double-blind, randomised, sham-controlled study is enrolling 112 patients in Australia, Germany and the US. Primary outcome is rate of change in ellipsoid zone (EZ) area loss as measured by en face imaging by Spectral-Domain Optical Coherence Tomography (SD-OCT) in the study eye; collection of these data is due to complete Mar 22 [3].
Nov 17PIII study to determine the safety and efficacy of NT 501 in macular telangiectasia type 2 starts (NTMT-03-A; NCT03316300). The double-blind, randomised, sham-controlled study is enrolling approximately 112 patients in the US, the UK (Oxford and Moorfields Eye Hospitals), France and Australia. Primary outcome is rate of change in ellipsoid zone (EZ) area loss as measured by en face imaging by Spectral-Domain Optical Coherence Tomography (SD-OCT) in the study eye; collection of these data is due to complete Mar 22 [3].