Neurotech has announced updated data from protocol A and B phase 3 studies.
NT-501, Neurotech Pharmaceuticals’ encapsulated cell therapy implant, has demonstrated a clinically meaningful change in the rate of progression in macular telangiectasia type 2, according to updated data from 2 phase 3 studies (protocol A, NCT03316300; protocol B, NCT03319849).
"We are extremely pleased to see these positive topline data for MacTel since there has been no pharmacological treatment to date," Richard Small, chief executive officer, Neurotech, said in a statement. "These findings are an important step towards giving hope to those currently suffering with unrelenting vision loss and who don’t have a way of stopping the progression of the disease today. We look forward to engaging with the FDA throughout the review process."
NT-501 is a novel ophthalmic neuroprotectant therapy that secretes ciliary neurotropic factor (CNTF). The therapy is a tiny hollow cylindrical membrane designed to be implanted into the vitreous cavity of the eye. It encapsulates human epithelial cells genetically engineered to continuously produce CNTF. Neurotech's encapsulated cell therapy technology uses a genetically engineered, proprietary retinal pigment epithelial NTC-201-6A cell lineencapsulated in a semi-permeable membrane to allow selective passage of therapeutic proteins. The therapy is inserted with a small scleral incision and can be removed from the same incision. Neurotech hopes NT-501 can reduce treatment burden and ensure patient compliance for at least 2 years of therapeutic delivery.
The randomized, sham-controlled, multicenter studies were primarily assessing the rate of change in ellipsoid zone area loss, whichcorrelates to photoreceptor loss, from baseline through 24 months as well as the safety of the implant and procedure.Other secondary outcome measures include the mean change in aggregate sensitivity of microperimetry, mean change in reading speed as measured by Internal Reading Speed Texts cards, mean change in National Eye Institute-Visual Function Questionnaire activities subscale score.
Investigators found that the implant was durable and well-tolerated with a manageable safety profile. In terms of efficacy, they found that the protocol A trial had a 56.4% rate of reduction (P <.0001) in treated patients and protocol B had a 29.2% rate of reduction (P = .021).
"After a long period of intensive research, development, and clinical trials, we are extremely gratified with our Phase 3 results," Thomas Aaberg Jr, MD, chief medical officer, Neurotech, added to the statement. "The positive study results increase the confidence of our innovative ECT platform technology to deliver a novel biological agent over an extended period of time in order to slow the progression of retinal-based diseases."