Neurotech Granted Fast Track Designations From the FDA for NT-501 in Two Indications – Retinitis Pigmentosa and Dry Age-Related Macular Degeneration
LINCOLN, R.I., Sept. 3 /PRNewswire/ — Neurotech Pharmaceuticals, Inc., a privately-held biotechnology company focused on the development of sight-saving therapeutics for chronic retinal diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designations for NT-501 for the treatment of visual loss in two indications — retinitis pigmentosa (RP) and the dry form of age-related macular degeneration (dry AMD). NT-501 is an intraocular, cell containing polymer implant designed to provide continuous, long-term release of the therapeutic protein Ciliary Neurotrophic Factor (CNTF) directly into the back of the eye by means of the Company’s proprietary Encapsulated Cell Technology. CNTF, a well established neurotrophic factor, rescues dying retinal photoreceptors and protects them from degeneration.
“We remain on track to announce top line results from our two Phase 2/3 studies in RP and our Phase 2 study in dry AMD by early 2009,” said Ted Danse, President and CEO of Neurotech. “The receipt of Fast Track designations for NT-501 in these indications is an important component in our ongoing product development strategy, allowing us to potentially accelerate our two clinical development programs as we seek to provide much needed treatment options for patients facing these devastating diseases.”
The Fast Track program, established under the FDA Modernization Act of 1997, provides for expedited regulatory review of a drug that demonstrates the potential to address an unmet medical need for the treatment of serious or life-threatening conditions. Under the program, the FDA will take such actions as are appropriate to expedite the development and review of the application for approval of such product, including potentially accepting, on a rolling basis, portions of the marketing application for review prior to the completion of the final registration package.
About Neurotech's Clinical Programs Phase 2/3 Studies in Retinitis Pigmentosa (RP)
Neurotech is conducting two Phase 2/3 trials of NT-501 for the treatment of visual loss associated with RP-one consisting of patients with earlier stage disease (60 patients) and the second consisting of patients with later stage disease (60 patients). Both trials are randomized, multi-centered, double-masked, sham-controlled dose ranging studies. Each patient receives either a high or low dose NT-501 implant in one eye and a sham treatment in the fellow eye. The primary efficacy endpoint is visual field sensitivity for the early-stage RP study and best corrected visual acuity for the late-stage RP study.
Phase 2 Study — Dry Age-related Macular Degeneration (Dry AMD)
This randomized, multi-centered, double-masked, sham-controlled study is evaluating NT-501 in 48 subjects with an advanced stage of dry AMD called geographic atrophy. Each subject receives either a high or low dose NT-501 implant or a sham treatment in one eye only. Best corrected visual acuity is the primary efficacy endpoint of this study.
About the Diseases
Retinitis Pigmentosa (RP) is an inherited disease that causes the retina’s rod and cone photoreceptors to gradually degenerate leading to loss of vision and blindness. The symptoms of RP predominately appear in young adults and affect approximately 100,000 people in the United States and over 1 million people worldwide. At this time there is no known cure or effective treatment for RP.
Age-related macular degeneration (AMD) is a chronic progressive disease of the macula that results in the loss of central vision. It is the leading cause of blindness in elderly people in the developed world. There are two forms of AMD — dry and wet. Dry AMD is the most common form of AMD representing approximately 90% of all AMD cases. In its later stages dry AMD can lead to the degeneration of photoreceptors and retinal pigment epithelial cells, a chronic condition called geographic atrophy (GA). GA affects approximately 1 million people in the United States for which there currently are no effective treatments.
About Encapsulated Cell Technology
Neurotech’s core technology platform is Encapsulated Cell Technology (ECT), a unique technology that allows for the long term, sustained delivery of therapeutic factors to the back of the eye. ECT implants consist of cells that have been genetically modified to produce a specific therapeutic protein and are encapsulated in a semi-permeable hollow fiber membrane. The diffusive characteristics of the hollow fiber membrane are designed to promote long-term cell survival by allowing the influx of oxygen and nutrients while simultaneously preventing direct contact of the encapsulated cells with the cellular and molecular elements of the immune system. The cells continuously produce the therapeutic protein which diffuses out of the implant at the target site. ECT therefore enables the controlled, continuous delivery of therapeutic factors directly to the retina, bypassing the blood-retina barrier.
About Neurotech Pharmaceuticals, Inc.
Neurotech is developing sight-saving therapeutics for the treatment of chronic retinal diseases. The Company’s lead product candidate, NT-501, is currently in late-stage clinical development for retinitis pigmentosa (RP) and dry age-related macular degeneration (dry AMD). RP is the leading inherited cause of blindness for which there is no current treatment. The Company’s portfolio of product candidates also includes treatments for wet AMD and diabetic macular edema. All of Neurotech’s development programs are based on the Company’s proprietary Encapsulated Cell Technology (ECT). ECT uniquely enables the controlled, continuous delivery of biologics directly to the back of the eye, overcoming a major obstacle in the treatment of retinal disease.
To learn more about Neurotech and the clinical studies of NT-501, please visit our web site at http://www.neurotechusa.com/.
Neurotech Pharmaceuticals, Inc.
CONTACT: Rich Small, Vice President, CFO of Neurotech Pharmaceuticals,Inc., +1-401-495-2403, email@example.com
Web site: http://www.neurotechusa.com/