CUMBERLAND, R.I.– Neurotech Pharmaceuticals, Inc., a clinical stage biotech company, today announced positive topline results in two replicative Phase 3 clinical trials with their investigational encapsulated cell therapy (ECT) for the treatment of Macular telangiectasia type 2 (MacTel), an orphan, slowly progressive degenerative disease of the macula that results in gradual deterioration of central vision. The multicenter, randomized, sham-controlled studies were designed to evaluate the safety and efficacy of the investigational NT-501 implant. The studies demonstrated statistical significance for the pre-specified primary endpoint, rate of change in ellipsoid zone (EZ) area loss from baseline through 24 months. The rate of change in area loss translates to and demonstrates a change in the rate of photoreceptor loss.
“We are extremely pleased to see these positive topline data for MacTel since there has been no pharmacological treatment to date,” said Richard Small, Chief Executive Officer of Neurotech. “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.”
“After a long period of intensive research, development, and clinical trials, we are extremely gratified with our Phase 3 results,” stated Thomas Aaberg Jr., M.D., Chief Medical Officer of Neurotech. “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.”
Macular telangiectasia type 2 (MacTel), or idiopathic juxtafoveal macular telangiectasia type 2, is a rare neurodegenerative disease with characteristic alterations of the retinal vasculature and localized retinal degeneration.1 The prevalence of MacTel ranges from 0.02% – 0.06% 2,3 with most patients diagnosed in their 40s and 50s.4 MacTel typically affects both eyes and causes a gradual deterioration in central vision.
Designed to be implanted into the vitreous cavity of the eye, the investigational NT-501 implant is a tiny hollow cylindrical membrane which encapsulates human epithelial cells genetically engineered to produce ciliary neurotropic factor (CNTF) continuously, a protein now clinically validated in Phase 3 clinical trials to slow the progression of MacTel.
Encapsulated Cell Therapy (ECT) is an investigational, first in class, platform technology that promotes continuous production of therapeutic proteins to the eye with the potential to treat a broad array of ocular diseases. It utilizes a proprietary, well-characterized retinal pigment epithelial cell line that has been genetically engineered to produce therapeutically active biologics. The cells are encapsulated in a semi-permeable membrane that allows for selective passage of therapeutic proteins. The ECT platform is inserted during a single outpatient surgical procedure through a small scleral incision, and can also be removed through the same incision, if desired. ECT has the potential to address the current limitations of intraocular drug delivery by allowing for and ensuring patient compliance and reducing treatment burden with one surgical procedure that can deliver drug for at least 2 years.
References:
- Charbel Issa P, Gillies MC, Chew EY, Heeren TFC, Bird AC, Peto T, Holz FG, Scholl HPN (2013) Macular Telangiectasia Type 2. Prog. Retin. Eye Res. 34: 49-77.
- Aung, K.Z., Wickremasinghe, S.S., Makeyeva, G., Robman, L. Guymer, R.H., 2010. The prevalence estimates of macular telangiectasia type 2: The Melbourne Collaborative Cohort Study. Retina 30, 473-478.
- Sallo, F.B., Leung, I., Mathenge, W., Kyari, F., Kuper, H. Gilbert, C.E., Bird, A.C., Peto, T., 2012. The prevalence of type 2 idiopathic macular telangiectasia in two African populations. Ophthalmic Epidemiol. 19, 185-189.
- Clemons, T.E., Gillies, M.C., Chew, E.Y., Bird, A.C., Peto, T., Figueroa, M.J., Harrington, M.W., 2010. Baseline characteristics of participants in the natural history study of macular telangiectasia (MacTel) MacTel Project Report No. 2. Ophthalmic Epidemiol. 17, 66-73.
