Positive Results from Neurotech's NT-501 Phase 2 Dry AMD (Geographic Atrophy) Study Demonstrate Proof of Concept
Validate Company's ECT Technology and Support Initiation of Pivotal Studies
LINCOLN, R.I.--(BUSINESS WIRE)--Neurotech Pharmaceuticals, Inc., today announced that the Company's lead product candidate, NT-501, substantially slowed the loss of vision in a Phase 2 clinical trial in subjects with dry age-related macular degeneration (AMD) involving geographic atrophy (GA). GA is a condition that destroys sharp central vision, often resulting in serious vision loss to one or both eyes. There are currently no approved treatments for dry AMD. In the study, the high dose of NT-501 stabilized best corrected visual acuity (BCVA) at 12-months, with 96.3% (p=0.078) of treated-patients losing fewer than three lines of vision, or 15 letters, versus 75% of the patients in the sham-treatment group.
NT-501 is an intraocular implant that consists of human cells that have been genetically modified to secrete ciliary neurotrophic factor (CNTF). CNTF is delivered directly to the back of the eye in a controlled, continuous basis by means of the Company's proprietary Encapsulated Cell Technology (ECT) platform, thereby bypassing the blood-retinal barrier and overcoming a major obstacle in the treatment of retinal disease.
The Phase 2 study is a multi-centered, randomized, double-masked, sham-controlled study of 51 subjects with GA. Patients received either a high or low dose NT-501 implant or a sham treatment in one eye only and were assessed for changes in BCVA. BCVA was measured by an Electronic Visual Acuity Tester (EVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Patients were also evaluated for an increase in BCVA. However, no increase was observed, likely due to existing photoreceptor damage. There were no NT-501 associated serious adverse events reported and both NT-501 and the surgical procedure were well-tolerated.
"The favorable functional visual acuity results for patients at this advanced stage of dry AMD are particularly promising due to the significant prevalence of the condition, its serious impact on quality of life and the current unmet medical need for effective therapy," stated Dr. George A. Williams, a study investigator and Professor and Chair of the Department of Ophthalmology at William Beaumont Hospital and the Oakland University William Beaumont School of Medicine in Royal Oak, MI.
The strong trend in visual acuity stabilization at 12 months was preceded by a dose-dependent, statistically significant (p<0.001 and p=0.013 for high and low dose, respectively) increase in retinal thickness as measured by optical coherence tomography (OCT) that was observed as early as 4 months post-implantation. The observed structural change is consistent with preclinical studies of NT-501 in which CNTF was shown to increase the thickness of the retina and the outer nuclear layer of photoreceptors responsible for vision. This increase in retinal thickness may be responsible for photoreceptor rescue and protection as observed in numerous animal models of retinal degeneration.
"Based on the increase in retinal thickness observed in this study it appears that CNTF may be exhibiting a biological effect on retinal photoreceptors as has been observed previously in animal studies," said Dr. Paul Sieving, Director of the National Eye Institute and Principal Investigator of Neurotech's Phase 1 study of NT-501 in retinitis pigmentosa.
"We believe the anatomical changes observed in patients treated with NT-501 have led to the emergence of a clinically meaningful visual acuity benefit for patients with geographic atrophy," commented Ted Danse, President and Chief Executive Officer of Neurotech. "NT-501 may provide a much needed treatment option for these patients and we intend to discuss these data and a pivotal trial design with the FDA."
"We are very pleased that the outcome of this trial has shown such promise for patients with dry AMD involving geographic atrophy and are proud of our long-term support for this unique, breakthrough technology," stated Stephen Rose, PhD, Chief Research Officer, Foundation Fighting Blindness.
Five devices from this trial have been explanted 12 months following implantation and all have been found to have uniformly healthy, viable cells that continue to produce therapeutic levels of CNTF. This is consistent with data from multiple trials of NT-501 in which, to date, 23 devices have been explanted between 12 and 18 months following implantation and all devices have contained healthy, viable CNTF-producing cells.
"We also believe the positive results of this study and long-term cell viability validate our ECT platform and support a breakthrough opportunity to advance long-term, well-tolerated treatments for patients facing chronic sight-stealing retinal diseases. As such, we are developing our second product utilizing the ECT platform to address a well-validated target, anti-VEGF therapy for wet AMD, that has the potential to provide a one-time administration for a 12 to 18 month period versus the current wet AMD treatment regimen that requires monthly injections with routine patient monitoring," concluded Danse.
Data from the Phase 2 dry AMD/GA trial will be presented at the Retinal Physician Symposium on March 27, 2009 in the Bahamas, at IBC's 5th International Ocular Angiogenesis & Retinal Degeneration conference on March 31, 2009 in Las Vegas, at the Advanced Vitreoretinal Techniques and Technologies meeting on April 24, 2009 in Las Vegas and at the Association for Research in Vision and Ophthalmology (ARVO) meeting on May 6, 2009 in Ft. Lauderdale.
About Dry AMD/Geographic Atrophy
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 advanced stages dry AMD can lead to the degeneration of photoreceptors and retinal pigment epithelial cells, a chronic condition called geographic atrophy (GA). There are currently no approved GA therapies for the nearly 1 million individuals affected in the United States.
Neurotech's lead product, NT-501, consists of encapsulated human cells genetically modified to secrete ciliary neurotrophic factor (CNTF). CNTF is a growth factor capable of rescuing dying photoreceptors and protecting them from degeneration. NT-501 is designed to continually deliver a therapeutic dose of CNTF into the back of the eye.
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 thereby 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 advanced dry age-related macular degeneration (dry AMD) and retinitis pigmentosa (RP). The Company's portfolio of product candidates also includes treatments for wet AMD. 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, thereby overcoming a major obstacle in the treatment of retinal disease. To learn more, please visit our web site at www.neurotechusa.com.
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