Neurotech's vision loss cell therapy implant hits primary goals in phase 3 trials

A pair of phase 3 trials of Neurotech Pharmaceuticals’ encapsulated cell therapy implant have met their primary endpoints, positioning the biotech to talk to the FDA about bringing the treatment for a cause of central vision loss to market.

The trials compared NT-501 to a sham procedure. NT-501 consists of a hollow cylindrical semi-permeable membrane that contains human epithelial cells genetically engineered to produce ciliary neurotrophic factor, a protein that promotes survival of neuronal cell types. After NT-501 is implanted into the vitreous cavity of the eye, the proteins cross the membrane and act on the patient.

In one phase 3 trial, Neurotech linked NT-501 to a 56.4% rate of change in ellipsoid zone area loss from baseline through 24 months in patients with macular telangiectasia type 2 (MacTel). The rate of change in the second study was 29.2%. Both primary endpoint results were statistically significant.

Neurotech, which changed the primary endpoints from mean change to rate of area loss in 2019, sees the effect on ellipsoid zone area loss as evidence of a change in the rate of photoreceptor loss. Patients with MacTel can suffer photoreceptor loss that affects their vision. 

“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,” Neurotech CEO Richard Small said in a statement. 

Neurotech is yet to share results from secondary endpoints that looked at measures such as reading speed and self-reported vision health status.