Sirion gets FDA Fast Track designation for sight-preserving drug
Tampa Bay Business Journal
Sirion Therapeutics Inc. announced positive results from a clinical trial evaluating a treatment for certain persons with macular degeneration.
The Phase II trial evaluated fenretinide as a treatment for geographic atrophy associated with age-related macular degeneration.
Sirion describes fenretinide as an oral vitamin A binding protein antagonist. Geographic atrophy is the most advanced form of dry age-related macular degeneration.
An analysis of the Phase II data showed slower growth of lesions in patients treated with oral fenretinide than in patients who received a placebo, a release from Sirion said.
Sirion will continue the Phase II study and will meet with scientific advisers and the Food and Drug Administration to design a Phase III study, the release said.
The effort by Sirion to develop fenretinide as a treatment got an additional boost when the FDA granted it Fast Track designation, the release said. Fast Track programs of the FDA are designated to facilitate the development of drugs that are intended to treat serious or life threatening conditions and that demonstrate the potential to address an unmet medical need for such a condition.
Sirion is a privately held biopharmaceutical company based in Tampa focused on discovery, development and commercialization of products to protect and preserve eyesight.
Sunday, April 26, 2009
Sunday, April 19, 2009
New Drug May Help Treat, Reverse Advanced Macular Degeneration
Apr 13, 2009
Reporter: CBS News
Email Address: news@kbtx.com
As the baby boomers age and live longer, a severe sight problem called Macular Degeneration is becoming more common.
But, there are new treatments to help the condition that can lead to blindness.
Sylvia Moore was diagnosed with macular degeneration three years ago.
"If I look straight ahead at somebody's face, I don't see them at all, you'd see a blank spot, a black spot," says Moore of the disease.
Macular Degeneration is most common in seniors.
It happens when blood vessels in the back of the eye start leaking and is the leading cause of blindness in people over 65.
The good news is doctors now have more weapons than ever to fight the condition, and in some cases even reverse it.
When it's caught early, vitamin therapy can slow down the progression of macular degeneration, and in some cases laser surgery can halt vision loss.
But the latest treatment is a drug that's injected directly into the eye.
The medication targets the abnormal blood vessels leading to its shutdown and hopefully to the improvement in vision.
Moore has been getting the injections for three months and is already seeing a difference.
"Its scary when you see a needle coming at you but it doesn't hurt.... I'd rather have that than blindness," Moore says.
A new study predicts some 9 million americans will be diagnosed with macular degeneration next year.
That number is expected to double in the next 40 years.
Apr 13, 2009
Reporter: CBS News
Email Address: news@kbtx.com
As the baby boomers age and live longer, a severe sight problem called Macular Degeneration is becoming more common.
But, there are new treatments to help the condition that can lead to blindness.
Sylvia Moore was diagnosed with macular degeneration three years ago.
"If I look straight ahead at somebody's face, I don't see them at all, you'd see a blank spot, a black spot," says Moore of the disease.
Macular Degeneration is most common in seniors.
It happens when blood vessels in the back of the eye start leaking and is the leading cause of blindness in people over 65.
The good news is doctors now have more weapons than ever to fight the condition, and in some cases even reverse it.
When it's caught early, vitamin therapy can slow down the progression of macular degeneration, and in some cases laser surgery can halt vision loss.
But the latest treatment is a drug that's injected directly into the eye.
The medication targets the abnormal blood vessels leading to its shutdown and hopefully to the improvement in vision.
Moore has been getting the injections for three months and is already seeing a difference.
"Its scary when you see a needle coming at you but it doesn't hurt.... I'd rather have that than blindness," Moore says.
A new study predicts some 9 million americans will be diagnosed with macular degeneration next year.
That number is expected to double in the next 40 years.
Saturday, April 11, 2009
Eye 'compensates for blind spot'
Eye 'compensates for blind spot'
Eye Peripheral vision is retained
Partially sighted and registered blind people can be taught to read and see faces again using the undamaged parts of their eyes, say experts.
When only the central vision is lost, as with the leading cause of blindness, age-related macular degeneration, peripheral vision remains intact.
And patients can be taught to exploit this, the Macular Disease Society says.
It has developed a training scheme and is calling for professionals to adopt the system across the UK.
The macula is a small area of the retina at the back of the eye made up of specialist cells which process central vision as well as the fine detail of what we see.
Our scheme has transformed lives - helping people to relearn basic skills they thought to have lost for good
Tom Bremridge
Macular Disease Society
People with macular degeneration rarely go totally blind but even those with a relatively mild version of the disease cannot drive and have difficulty reading, recognising faces and watching television.
But studies show people can be taught to use their peripheral vision to fill in the gaps, using "eccentric viewing" and "steady eye techniques".
When someone with central vision loss looks directly at an object it may disappear, go faint, blur or distort. But when they look above, below or to one side of it, they see it more clearly.
Blind spots
Eccentric viewing helps people find exactly where to focus their gaze to make their vision better.
Once this position is identified, they can be taught how to read again using the steady eye technique.
Instead of moving the eyes from left to right to read a sentence, the person should keep their eyes completely still and move the text to the left so that each word in turn moves into the area of best vision.
All UK patients with central vision loss should have the opportunity to try eccentric viewing
Mr Winfried Amoaku
Royal College of Ophthalmologists
Macular Disease Society chief executive Tom Bremridge said: "Eccentric viewing works by making the most of vision that remains.
"Our scheme has transformed lives - helping people to relearn basic skills they thought to have lost for good.
"We have 86 volunteer trainers, all with central vision loss themselves, who have trained more than 310 people in their own communities, and our waiting list of nearly 1,200 people grows every day.
"We are keen that other service providers - social services, private practitioners and primary care trusts - now take up the baton."
Mr Winfried Amoaku, of the Royal College of Ophthalmologists, said eccentric viewing could help some patients with central vision loss "cope with everyday tasks such as identifying coins while out shopping, watching television and reading".
"The trouble is, we don't know who will benefit until they have tried the training.
"All UK patients with central vision loss should have the opportunity to try eccentric viewing techniques to see if they can benefit," he said.
Marek Karas, of the Royal National Institute of Blind People, also supported the research advances.
"Although there is still ongoing discussion among experts over the best form of training for this type of therapy, we welcome with interest these latest developments."
Between 25 and 30 million people worldwide have macular degeneration. But as the population ages, this figure will rise.
It is estimated that the number of people affected will triple by 2025.
Eye Peripheral vision is retained
Partially sighted and registered blind people can be taught to read and see faces again using the undamaged parts of their eyes, say experts.
When only the central vision is lost, as with the leading cause of blindness, age-related macular degeneration, peripheral vision remains intact.
And patients can be taught to exploit this, the Macular Disease Society says.
It has developed a training scheme and is calling for professionals to adopt the system across the UK.
The macula is a small area of the retina at the back of the eye made up of specialist cells which process central vision as well as the fine detail of what we see.
Our scheme has transformed lives - helping people to relearn basic skills they thought to have lost for good
Tom Bremridge
Macular Disease Society
People with macular degeneration rarely go totally blind but even those with a relatively mild version of the disease cannot drive and have difficulty reading, recognising faces and watching television.
But studies show people can be taught to use their peripheral vision to fill in the gaps, using "eccentric viewing" and "steady eye techniques".
When someone with central vision loss looks directly at an object it may disappear, go faint, blur or distort. But when they look above, below or to one side of it, they see it more clearly.
Blind spots
Eccentric viewing helps people find exactly where to focus their gaze to make their vision better.
Once this position is identified, they can be taught how to read again using the steady eye technique.
Instead of moving the eyes from left to right to read a sentence, the person should keep their eyes completely still and move the text to the left so that each word in turn moves into the area of best vision.
All UK patients with central vision loss should have the opportunity to try eccentric viewing
Mr Winfried Amoaku
Royal College of Ophthalmologists
Macular Disease Society chief executive Tom Bremridge said: "Eccentric viewing works by making the most of vision that remains.
"Our scheme has transformed lives - helping people to relearn basic skills they thought to have lost for good.
"We have 86 volunteer trainers, all with central vision loss themselves, who have trained more than 310 people in their own communities, and our waiting list of nearly 1,200 people grows every day.
"We are keen that other service providers - social services, private practitioners and primary care trusts - now take up the baton."
Mr Winfried Amoaku, of the Royal College of Ophthalmologists, said eccentric viewing could help some patients with central vision loss "cope with everyday tasks such as identifying coins while out shopping, watching television and reading".
"The trouble is, we don't know who will benefit until they have tried the training.
"All UK patients with central vision loss should have the opportunity to try eccentric viewing techniques to see if they can benefit," he said.
Marek Karas, of the Royal National Institute of Blind People, also supported the research advances.
"Although there is still ongoing discussion among experts over the best form of training for this type of therapy, we welcome with interest these latest developments."
Between 25 and 30 million people worldwide have macular degeneration. But as the population ages, this figure will rise.
It is estimated that the number of people affected will triple by 2025.
Sunday, April 5, 2009
FDA Approval Recommended for Implantable Macular Degeneration Device
FDA Approval Recommended for Implantable Macular Degeneration Device
By Emily P. Walker, Washington Correspondent, MedPage Today
Published: March 30, 2009
GAITHERSBURG, Md., March 30 -- An FDA advisory panel agreed that the agency should approve a miniature implantable telescope to improve central vision in elderly people with end-stage age-related macular degeneration.
The Ophthalmic Devices Panel voted unanimously Friday to recommend approval for the Implantable Miniature Telescope, made by Vision Care Ophthalmic Technologies, for use in patients who are 65 or older.
The thumbs-up came despite the device's association with loss of corneal cells over time.
Many elderly people would likely choose the telescope -- which could help maintain their ability to carry out daily tasks such as recognizing faces and reading books -- even if it meant developing corneal edema four or five years down the road, the panel decided.
Four years after the implant, about 15% of patients will have a significant depletion in endothelial cells, which can lead to corneal edema, according to an estimate by the panel's biostatistician, Karen Bandeen-Roche, Ph.D., of Johns Hopkins University in Baltimore.
The telescope works by directing light from the lens onto still-functioning portions of the macula to maintain central sight. It increases vision up to about three meters, helping patients perform close-up tasks, though doing nothing for their ability to drive.
The device would be implanted in one eye, after patients wear an external version for a few days to get a feel for it. Because it only improves central vision, implant recipients would rely on the opposite eye for peripheral sight.
The outcome of the Friday meeting was a major victory for the Vision Care, especially because the FDA turned down its original application in 2006 because of safety concerns.
New, longer-term data convinced the panel that the telescope can deliver improved vision for certain patients and that the associated cell loss is an acceptable risk.
Vision Care applied for a much narrower indication this time around. So narrow, in fact, that only about one-quarter of the participants included in the company's original trial would meet the new criteria, according to Henry Hudson, M.D., an vitreoretinal specialist in Tucson, Ariz., and lead investigator of the main clinical study submitted to the FDA.
The device was originally intended for patients 55 and older, but the sponsors bumped the age requirement up to 65. The new application also excludes patients with corneal guttata, and those who have an anterior chamber depth of less than 3 mm. Patients must also have retinal cell counts that are normal for their age group before receiving the implant.
The application calls for the device to be implanted by cornea specialists who are trained by Vision Care.
The panel's approval was based largely on a trial involving 206 patients with incurable central vision disorders and who were over 55 and had a need for cataract surgery.
The primary endpoint for efficacy was improvement of two lines or greater in either near or distance vision, which was achieved in 86% of patients at follow-up.
In a four-year follow-up that the company completed at the request of the FDA, 68% of the patients achieved a two-line or greater gain in either long distance or near vision four years after the surgery.
The panel heard from three octogenarians who said that if it weren't for their implantable telescopes, they would not be able to read books, make crafts, recognize their grandchildren, or carry out daily chores.
The committee suggested that the original trial cohort -- of which 130 are alive and still have the implant -- be followed for additional five years to monitor for complications such as rates of corneal transplant, retinal detachments and corneal edema.
The committee, chaired by Jayne Weiss, M.D., an ophthalmologist from the Kresge Eye Institute in Detroit, also said patients should be informed of the risk of cell loss and the possible benefits of improved central vision associated with the device.
The lead researcher agreed.
"The key consideration is proper patient expectation to get the maximum effect of this technology," Dr. Hudson said.
By Emily P. Walker, Washington Correspondent, MedPage Today
Published: March 30, 2009
GAITHERSBURG, Md., March 30 -- An FDA advisory panel agreed that the agency should approve a miniature implantable telescope to improve central vision in elderly people with end-stage age-related macular degeneration.
The Ophthalmic Devices Panel voted unanimously Friday to recommend approval for the Implantable Miniature Telescope, made by Vision Care Ophthalmic Technologies, for use in patients who are 65 or older.
The thumbs-up came despite the device's association with loss of corneal cells over time.
Many elderly people would likely choose the telescope -- which could help maintain their ability to carry out daily tasks such as recognizing faces and reading books -- even if it meant developing corneal edema four or five years down the road, the panel decided.
Four years after the implant, about 15% of patients will have a significant depletion in endothelial cells, which can lead to corneal edema, according to an estimate by the panel's biostatistician, Karen Bandeen-Roche, Ph.D., of Johns Hopkins University in Baltimore.
The telescope works by directing light from the lens onto still-functioning portions of the macula to maintain central sight. It increases vision up to about three meters, helping patients perform close-up tasks, though doing nothing for their ability to drive.
The device would be implanted in one eye, after patients wear an external version for a few days to get a feel for it. Because it only improves central vision, implant recipients would rely on the opposite eye for peripheral sight.
The outcome of the Friday meeting was a major victory for the Vision Care, especially because the FDA turned down its original application in 2006 because of safety concerns.
New, longer-term data convinced the panel that the telescope can deliver improved vision for certain patients and that the associated cell loss is an acceptable risk.
Vision Care applied for a much narrower indication this time around. So narrow, in fact, that only about one-quarter of the participants included in the company's original trial would meet the new criteria, according to Henry Hudson, M.D., an vitreoretinal specialist in Tucson, Ariz., and lead investigator of the main clinical study submitted to the FDA.
The device was originally intended for patients 55 and older, but the sponsors bumped the age requirement up to 65. The new application also excludes patients with corneal guttata, and those who have an anterior chamber depth of less than 3 mm. Patients must also have retinal cell counts that are normal for their age group before receiving the implant.
The application calls for the device to be implanted by cornea specialists who are trained by Vision Care.
The panel's approval was based largely on a trial involving 206 patients with incurable central vision disorders and who were over 55 and had a need for cataract surgery.
The primary endpoint for efficacy was improvement of two lines or greater in either near or distance vision, which was achieved in 86% of patients at follow-up.
In a four-year follow-up that the company completed at the request of the FDA, 68% of the patients achieved a two-line or greater gain in either long distance or near vision four years after the surgery.
The panel heard from three octogenarians who said that if it weren't for their implantable telescopes, they would not be able to read books, make crafts, recognize their grandchildren, or carry out daily chores.
The committee suggested that the original trial cohort -- of which 130 are alive and still have the implant -- be followed for additional five years to monitor for complications such as rates of corneal transplant, retinal detachments and corneal edema.
The committee, chaired by Jayne Weiss, M.D., an ophthalmologist from the Kresge Eye Institute in Detroit, also said patients should be informed of the risk of cell loss and the possible benefits of improved central vision associated with the device.
The lead researcher agreed.
"The key consideration is proper patient expectation to get the maximum effect of this technology," Dr. Hudson said.
Monday, March 30, 2009
Positive Results from Neurotech's NT-501 Phase 2 Dry AMD
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.
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.
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.
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