Positive Results from Neurotech's NT-501 Phase 2 Dry AMD (Geographic Atrophy) Study Demonstrate Proof of Concept
Virtual Clinical Trials
Global Clinical Development Collaboration Solutions
www.trialinteractive.com
Validate Company's ECT Technology and Support Initiation of Pivotal Studies
LINCOLN, R.I.--(BUSINESS WIRE)--Mar 26, 2009 - 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.
About NT-501
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.
Monday, March 30, 2009
Saturday, March 21, 2009
Macular degeneration may have same biomarker as kidney function
Macular degeneration may have same biomarker as kidney function
Publish date: Mar 5, 2009
Source:Optamology Times
Madison, WI—Cystatin C, a biomarker of kidney function, also may be associated with the incidence of age-related macular degeneration (AMD), said researchers.
Adjusted analyses found that serum levels of the protein were associated with the incidence of both early and exudative AMD, according to Ronald Klein, MD, MPH, and colleagues.
There was an association between chronic kidney disease and macular degeneration, but the association between cystatin C and the degenerative eye disease was intensified in patients without chronic kidney disease.
"This suggests that this relationship might not be due to kidney-related processes," the researchers said.
Studies on the relationship of kidney disease and AMD have been few and inconsistent, so researchers conducted a prospective cohort study of 4,926 patients aged 43 to 86 years, in Beaver Dam, WI.
Publish date: Mar 5, 2009
Source:Optamology Times
Madison, WI—Cystatin C, a biomarker of kidney function, also may be associated with the incidence of age-related macular degeneration (AMD), said researchers.
Adjusted analyses found that serum levels of the protein were associated with the incidence of both early and exudative AMD, according to Ronald Klein, MD, MPH, and colleagues.
There was an association between chronic kidney disease and macular degeneration, but the association between cystatin C and the degenerative eye disease was intensified in patients without chronic kidney disease.
"This suggests that this relationship might not be due to kidney-related processes," the researchers said.
Studies on the relationship of kidney disease and AMD have been few and inconsistent, so researchers conducted a prospective cohort study of 4,926 patients aged 43 to 86 years, in Beaver Dam, WI.
Saturday, March 14, 2009
Pill Shows Promise for Treatment of Macular Degeneration
By JEAN ENERSEN / KING 5 News
Beatrice Dean has had the dry form of macular degeneration for 20 years.
"You get to the point where you don't read any longer, you don't write, you just don't see things straight on,” she said.
A National Institute of Health study found that certain eye vitamins can help, to a point. Dr Richard Bensinger of Swedish Medical Center says this was the first breakthrough.
"It showed a very significant slowing of the condition, not a cure, or stoppage,” he said.
Now the Acucela biotech firm in Bothell is working on a pill that may finally stop the disease altogether.
Founder Ryo Kubota says the goal is to prevent dry form from progressing into the more severe wet form, which can cause blindness overnight.
"Primarily it will be preventative and slow down the disease, but in animal test studies that we've done, we've show in can reduce the already accumulated toxic byproducts,” said Dr. Kubota.
In theory, the drug works by blocking the damage before it starts.
Who will get macular degeneration?
"There really is no risk factor that's known except for everybody's favorite, smoking,” said Dr. Kubota.
Dr. Kobota hopes this research will make all the difference since macular degeneration cases are expected to rise as baby boomers get older.
“We're hoping this drug to be on the market in five to 10 years,” he said.
Human trials are just beginning. We'll keep you up to date on the research and when the study will begin recruiting locally.
Beatrice Dean has had the dry form of macular degeneration for 20 years.
"You get to the point where you don't read any longer, you don't write, you just don't see things straight on,” she said.
A National Institute of Health study found that certain eye vitamins can help, to a point. Dr Richard Bensinger of Swedish Medical Center says this was the first breakthrough.
"It showed a very significant slowing of the condition, not a cure, or stoppage,” he said.
Now the Acucela biotech firm in Bothell is working on a pill that may finally stop the disease altogether.
Founder Ryo Kubota says the goal is to prevent dry form from progressing into the more severe wet form, which can cause blindness overnight.
"Primarily it will be preventative and slow down the disease, but in animal test studies that we've done, we've show in can reduce the already accumulated toxic byproducts,” said Dr. Kubota.
In theory, the drug works by blocking the damage before it starts.
Who will get macular degeneration?
"There really is no risk factor that's known except for everybody's favorite, smoking,” said Dr. Kubota.
Dr. Kobota hopes this research will make all the difference since macular degeneration cases are expected to rise as baby boomers get older.
“We're hoping this drug to be on the market in five to 10 years,” he said.
Human trials are just beginning. We'll keep you up to date on the research and when the study will begin recruiting locally.
Saturday, March 7, 2009
Brain Adapts to Age-Related Eye Disease
Brain Adapts to Age-Related Eye Disease
Neurons seek input from undamaged areas to compensate, study finds
Posted March 3, 2009
TUESDAY, March 3 (HealthDay News) -- When macular degeneration causes one to start losing his or her sight, the affected neurons simply start seeking visual input from other, non-affected parts of the eye, Massachusetts Institute of Technology researchers report.
People Who Read This Also Read
Learning to Relax by Paying Attention
'Fasting Signal' Offers Clues to Insulin Resistance in the Obese
Good Parents, Bad Results
Do Teenagers Need Vitamins of Their Own?
Retinal Gene Is Linked to Childhood Blindness
"This study shows us one way that the brain changes when its inputs change. Neurons seem to want to receive input: When their usual input disappears, they start responding to the next best thing," senior author Nancy Kanwisher, of MIT's McGovern Institute for Brain Research, said in an university news release.
The researchers found when the cells in the fovea, the part of the retina responsible for the central field of vision, were damaged by macular degeneration (MD) -- the neuron attached to them begin responding to stimuli in an undamaged section -- a type of internal reorganization of the eye's visual map as opposed to the cortex's work being shifting to other neurons.
"Our study shows that the changes we see in neural response in people with MD are probably driven by the lack of input to a population of neurons, not by a change in visual information-processing strategy," Kanwisher said.
The findings are published in the March 4 issue of the Journal of Neuroscience.
Macular degeneration, the most common form of adult blindness, affects almost 2 million people in the United States. Patients often compensate for lack of central vision by rolling their eyes upward so they can utilize the preferred retinal locus (PRL), an undamaged area under and adjacent to the affected part of the retina.
"Macular degeneration is a great opportunity to learn more about plasticity in the adult cortex," Kanwisher said.
Neurons seek input from undamaged areas to compensate, study finds
Posted March 3, 2009
TUESDAY, March 3 (HealthDay News) -- When macular degeneration causes one to start losing his or her sight, the affected neurons simply start seeking visual input from other, non-affected parts of the eye, Massachusetts Institute of Technology researchers report.
People Who Read This Also Read
Learning to Relax by Paying Attention
'Fasting Signal' Offers Clues to Insulin Resistance in the Obese
Good Parents, Bad Results
Do Teenagers Need Vitamins of Their Own?
Retinal Gene Is Linked to Childhood Blindness
"This study shows us one way that the brain changes when its inputs change. Neurons seem to want to receive input: When their usual input disappears, they start responding to the next best thing," senior author Nancy Kanwisher, of MIT's McGovern Institute for Brain Research, said in an university news release.
The researchers found when the cells in the fovea, the part of the retina responsible for the central field of vision, were damaged by macular degeneration (MD) -- the neuron attached to them begin responding to stimuli in an undamaged section -- a type of internal reorganization of the eye's visual map as opposed to the cortex's work being shifting to other neurons.
"Our study shows that the changes we see in neural response in people with MD are probably driven by the lack of input to a population of neurons, not by a change in visual information-processing strategy," Kanwisher said.
The findings are published in the March 4 issue of the Journal of Neuroscience.
Macular degeneration, the most common form of adult blindness, affects almost 2 million people in the United States. Patients often compensate for lack of central vision by rolling their eyes upward so they can utilize the preferred retinal locus (PRL), an undamaged area under and adjacent to the affected part of the retina.
"Macular degeneration is a great opportunity to learn more about plasticity in the adult cortex," Kanwisher said.
Subscribe to:
Posts (Atom)
