June 29, 2010 by MassDevice staffpSivida Corp. and Alimera Sciences file a new drug application with the Food & Drug Administration for Iluvien, a drug/device combination aimed at treating diabetic macular degeneration.
pSivida Corp. (NSDQ:PSDV) and Alimera Sciences (NSDQ:ALIM) are seeking a green light from the Food & Drug Administration for a drug/device combination to treat diabetic macular degeneration.
Watertown, Mass.-based pSivida said Alimera, which licenses the Iluvien technology from pSivida, submitted a new drug application to the FDA. The technology is designed to deliver sustained, low doses of flucocinolone acetonide to the retina at the rear of the eyeball.
Alimera asked the federal watchdog for priority review, an expedited process that could bring an FDA response during the fourth quarter, according to a press release.
There are no approved drugs to treat DME, according to pSivida president and CEO Paul Ashton, adding that Iluvien is the company's third product aimed at back-of-the-eye diseases. The first two won FDA approval and are on the market, Ashton said: Retisert, for the treatment of posterior uveitis, and Vitrasert for the treatment of AIDS-related cytomegalovirus retinitis. Both are licensed to Bausch & Lomb Inc.
The company is also working on a treatment for retinitis pigmentosa, which involves the gradual deterioration of the rods and cones that make up the retina. In April Ashton told MassDevice that its Durasert device also uses flucocinolone acetonide, a steroid, to treat the disease.
"What we've done is use a very small insertable drug delivery device to release a steroid directly into the eye that will just provide some protection and slow down the rates of vision loss," he said. "With a condition that takes 20 years to make you blind, if you slow it down by a factor of two, that's pretty good."
The partnership with Alimera has already paid dividends, namely a $15.3 million payment triggered by Alimera's April 21 initial public offering. At the time Ashton told us pSivida planned to use the cash to further develop its product pipeline. If the FDA gives the nod to Iluvien, it would trigger another, $25 million milestone payment from Alimera, plus 20 percent royalties on net profits from sales of the treatment.
For more information go to www.maculardegenerationassociation.org
Wednesday, June 30, 2010
Thursday, June 10, 2010
Researchers create retina from embryonic cells
By Adrian Galbreth
Researchers in the US have successfully created a retina from human embryonic stem cells, which offers hope to millions with degenerative eye disorders.
Experts at the University of California Irvine created an eight-layer, early stage retina from human embryonic stem cells, which is the first ever three-dimensional tissue structure to be made from stem cells.
Study leader Hans Keirstead of the Reeve-Irvine Research Center and the Sue and Bill Gross Stem Cell Research Center at the facility, said the process also marks the first step towards the development of transplant-ready retinas to treat conditions such as retinitis pigmentosa and macular degeneration, a leading cause of blindness.
"We made a complex structure consisting of many cell types. This is a major advance in our quest to treat retinal disease," he explained.
Recently, German research centre Fraunhofer-Gesellschaft claimed that a new implant made of plastic could soon offer patients the chance to see again without having to wait for cornea transplants.ADNFCR-1853-ID-19804594-ADNFCR
Researchers in the US have successfully created a retina from human embryonic stem cells, which offers hope to millions with degenerative eye disorders.
Experts at the University of California Irvine created an eight-layer, early stage retina from human embryonic stem cells, which is the first ever three-dimensional tissue structure to be made from stem cells.
Study leader Hans Keirstead of the Reeve-Irvine Research Center and the Sue and Bill Gross Stem Cell Research Center at the facility, said the process also marks the first step towards the development of transplant-ready retinas to treat conditions such as retinitis pigmentosa and macular degeneration, a leading cause of blindness.
"We made a complex structure consisting of many cell types. This is a major advance in our quest to treat retinal disease," he explained.
Recently, German research centre Fraunhofer-Gesellschaft claimed that a new implant made of plastic could soon offer patients the chance to see again without having to wait for cornea transplants.ADNFCR-1853-ID-19804594-ADNFCR
Wednesday, June 2, 2010
New Drugs for Macular Degeneration
By Emily Singer
Two genetic studies of people with age-related macular degeneration (AMD)--the most common cause of blindness in people older than 65--made a surprising discovery. Research showed that defects in a gene that is an important regulator of parts of the immune system significantly increased risk of the disease. Scientists have since identified variants in several related genes that also boost risk, and which collectively account for about 50 to 60 percent of the heritability of the disorder.
At the same time that researchers identified the harmful variation linked to AMD, Gregory Hageman, now at the University of Utah, identified a protective variant found in about 20 percent of the population. "That form is so incredibly protective that people with two copies are almost guaranteed not to develop the disease," he says. Hageman founded Optherion, a startup based in New Haven, CT, and investigated how to translate the findings into new treatments. Optherion is now producing large quantities of an engineered version of the protein and doing preclinical safety and effectiveness testing--for example, examining whether the treatment can reduce ocular deposits in mice that lack the protein, says Colin Foster, Optherion's president. He declined to estimate when the company will begin clinical trials of the drug.
Scientists hope that these developments will prove to be an example of the benefits that can arise from a type of genetic study called genome-wide association. The genome-wide studies of macular degeneration were among the first and perhaps the biggest success for the approach, which employs specially designed chips dotted with markers to cheaply detect hundreds of thousands of the most common variations in the human genome. While these chips have allowed scientists to cheaply scan the genomes of many patients and healthy controls, the approach has come under increasing scrutiny in the last couple of years. Even huge studies of thousands of people have failed to identify the majority of the heritability of common diseases, such as type 2 diabetes or Alzheimer's disease.
Two genetic studies of people with age-related macular degeneration (AMD)--the most common cause of blindness in people older than 65--made a surprising discovery. Research showed that defects in a gene that is an important regulator of parts of the immune system significantly increased risk of the disease. Scientists have since identified variants in several related genes that also boost risk, and which collectively account for about 50 to 60 percent of the heritability of the disorder.
At the same time that researchers identified the harmful variation linked to AMD, Gregory Hageman, now at the University of Utah, identified a protective variant found in about 20 percent of the population. "That form is so incredibly protective that people with two copies are almost guaranteed not to develop the disease," he says. Hageman founded Optherion, a startup based in New Haven, CT, and investigated how to translate the findings into new treatments. Optherion is now producing large quantities of an engineered version of the protein and doing preclinical safety and effectiveness testing--for example, examining whether the treatment can reduce ocular deposits in mice that lack the protein, says Colin Foster, Optherion's president. He declined to estimate when the company will begin clinical trials of the drug.
Scientists hope that these developments will prove to be an example of the benefits that can arise from a type of genetic study called genome-wide association. The genome-wide studies of macular degeneration were among the first and perhaps the biggest success for the approach, which employs specially designed chips dotted with markers to cheaply detect hundreds of thousands of the most common variations in the human genome. While these chips have allowed scientists to cheaply scan the genomes of many patients and healthy controls, the approach has come under increasing scrutiny in the last couple of years. Even huge studies of thousands of people have failed to identify the majority of the heritability of common diseases, such as type 2 diabetes or Alzheimer's disease.
Thursday, May 27, 2010
Decreased antioxidant levels increase risk of age-related macular degeneration with exposure to sunlight
The journal Archives of Ophthalmology recently reported that having reduced plasma antioxidant levels and increased exposure to sunlight increases the risk of neovascular, or advanced age-related macular degeneration (AMD).
In a recent study (titled the European Eye Study), over 4,400 participants over age 65 were analyzed for the presence of macular degeneration. Their blood plasma was evaluated for vitamins C and E, the carotenoids lutein and zeaxanthin, and the mineral zinc. Each individual also completed a questionnaire regarding their typical exposure to sunlight. This was used to estimate blue light exposure from visible light, which is known to contribute to the development of macular degeneration.
Early stage macular degeneration was detected in 2,182 participants, and 101 had the advanced form of the disease. The research found no association between blue light exposure and early macular degeneration. However, among participants in the lowest quartile (25 percent) of serum vitamin C, zeaxanthin, vitamin E and zinc, exposure to blue light significantly increased the risk of advanced macular degeneration.
In a recent study (titled the European Eye Study), over 4,400 participants over age 65 were analyzed for the presence of macular degeneration. Their blood plasma was evaluated for vitamins C and E, the carotenoids lutein and zeaxanthin, and the mineral zinc. Each individual also completed a questionnaire regarding their typical exposure to sunlight. This was used to estimate blue light exposure from visible light, which is known to contribute to the development of macular degeneration.
Early stage macular degeneration was detected in 2,182 participants, and 101 had the advanced form of the disease. The research found no association between blue light exposure and early macular degeneration. However, among participants in the lowest quartile (25 percent) of serum vitamin C, zeaxanthin, vitamin E and zinc, exposure to blue light significantly increased the risk of advanced macular degeneration.
Friday, January 22, 2010
Promising new treatment for dry AMD in clinical trials
Acucela Inc. has announced Phase 2 clinical trials of it’s ACU-4429 for the treatment of dry age-related macular degeneration (AMD). ACU-4429 works by decreasing the levels of toxic products in the eye thus hopefully stopping the advance of dry AMD.
More than 29 million people worldwide are affected by either “wet” or “dry” AMD. The leading cause of the loss of vision in people over the age of 50 is dry AMD. 90 percent of AMD patients suffer from the dry form of AMD. Currently, there are no FDA approved therapies to treat dry AMD which will make the trials of ACU–4429 ones eagerly watched. Anti-oxidants are the only therapy available to slow or halt the progression of dry AMD
Researchers are pleased with the preclinical and early clinical data for ACU-4429. Thus far, it has demonstrated the ability to decrease toxic by-products which have had a part in the progression of dry AMD.
ACU-4429 is administered as an oral, daily pill unlike other therapies for the eyes which can involve the use of injections into the eyes.
For more information go to www.maculardegenerationassociation.org
More than 29 million people worldwide are affected by either “wet” or “dry” AMD. The leading cause of the loss of vision in people over the age of 50 is dry AMD. 90 percent of AMD patients suffer from the dry form of AMD. Currently, there are no FDA approved therapies to treat dry AMD which will make the trials of ACU–4429 ones eagerly watched. Anti-oxidants are the only therapy available to slow or halt the progression of dry AMD
Researchers are pleased with the preclinical and early clinical data for ACU-4429. Thus far, it has demonstrated the ability to decrease toxic by-products which have had a part in the progression of dry AMD.
ACU-4429 is administered as an oral, daily pill unlike other therapies for the eyes which can involve the use of injections into the eyes.
For more information go to www.maculardegenerationassociation.org
Sunday, January 10, 2010
Laureate Pharma and Iconic Therapeutics complete manufacturing hI-con1 recombinant Fc-Factor VII fusion protein
Laureate Pharma, Inc., a full-service biopharmaceutical development and protein production company specializing in the development and GMP manufacture of monoclonal antibodies, fusion proteins and other therapeutic protein products, and Iconic Therapeutics, a company focused on the development of treatments for wet adult macular degeneration (wet AMD) and other ophthalmic diseases, today announced the completion of the manufacture of the first GMP lot of hI-con1(TM) recombinant Fc-Factor VII fusion protein.
"We are very excited that Iconic Therapeutics now has material to test this new therapy for patients with macular degeneration and other diseases," said Robert J. Broeze, Ph. D., President & CEO of Laureate Pharma. "This is a testimonial to our strong partnership with Iconic and our expertise in working with fusion proteins." "Due to the unique properties of Iconic's hI-con1(TM) fusion protein product, this project involved very close collaboration between our process-development team at Laureate and Iconic's product-development team," added Michiel E. Ultee, Ph.D., Vice President of Process Sciences.
"With clinical material now in hand, we have moved a giant step forward in our plans for hI-con1(TM). We are excited about its prospects as a novel and powerful approach for the therapy of wet AMD," said Kirk Dornbush, President of Iconic Therapeutics. "We are very pleased with the strong working relationship that we established with Laureate Pharma."
hI-con1(TM) is a recombinant protein intended to specifically attack and destroy pathological blood vessels (PBVs) with no effect on normal blood vessels. Since wet AMD is characterized by the invasion of PBVs into the eye, a drug that destroys these blood vessels could be very useful in treatment of this disease. In addition, hI-con1(TM) is thought to act differently from other wet AMD treatments, which reduce the effects of pathological blood vessel invasion into the eye but do not appear to kill those blood vessels. In studies with mice and pigs, a single injection of hI-con1(TM) into the eye resulted in dramatic reduction of pathological blood vessels.
For more information go to WWW.MACULARDEGENERATIONASSOCIATION.ORG
"We are very excited that Iconic Therapeutics now has material to test this new therapy for patients with macular degeneration and other diseases," said Robert J. Broeze, Ph. D., President & CEO of Laureate Pharma. "This is a testimonial to our strong partnership with Iconic and our expertise in working with fusion proteins." "Due to the unique properties of Iconic's hI-con1(TM) fusion protein product, this project involved very close collaboration between our process-development team at Laureate and Iconic's product-development team," added Michiel E. Ultee, Ph.D., Vice President of Process Sciences.
"With clinical material now in hand, we have moved a giant step forward in our plans for hI-con1(TM). We are excited about its prospects as a novel and powerful approach for the therapy of wet AMD," said Kirk Dornbush, President of Iconic Therapeutics. "We are very pleased with the strong working relationship that we established with Laureate Pharma."
hI-con1(TM) is a recombinant protein intended to specifically attack and destroy pathological blood vessels (PBVs) with no effect on normal blood vessels. Since wet AMD is characterized by the invasion of PBVs into the eye, a drug that destroys these blood vessels could be very useful in treatment of this disease. In addition, hI-con1(TM) is thought to act differently from other wet AMD treatments, which reduce the effects of pathological blood vessel invasion into the eye but do not appear to kill those blood vessels. In studies with mice and pigs, a single injection of hI-con1(TM) into the eye resulted in dramatic reduction of pathological blood vessels.
For more information go to WWW.MACULARDEGENERATIONASSOCIATION.ORG
Monday, January 4, 2010
New research findings may help stop age-related macular degeneration at the molecular level
Scientists discover the relationship between 2 blood proteins plays a pivotal role in staving off the condition
Researchers at University College London say they have gleaned a key insight into the molecular beginnings of age-related macular degeneration, the No. 1 cause of vision loss in the elderly, by determining how two key proteins interact to naturally prevent the onset of the condition.
In a paper to be published in a forthcoming issue of the Journal of Biological Chemistry, the team reports for the first time how a common blood protein linked to the eye condition reins in another protein that, when produced in vastly increased amounts in the presence of inflammation or infection, can damage the eye.
"By starting to understand these interactions in greater detail, we can begin to devise methods that will ultimately prevent the development of blindness in the elderly," said Zuby Okemefuna, the lead author of the paper to be published Jan. 8.
Age-related macular degeneration, or AMD, is painless but affects the macula, the part of the retina that allows one to see fine detail. One form of the debilitating condition, known as "wet" AMD, occurs when abnormal and fragile blood vessels grow under the macula, leaking blood and fluid and displacing and damaging the macula itself. The second form, "dry" AMD, occurs when light-sensitive cells in the macula slowly break down.
It is believed that both forms start on a common molecular route and then deviate into dry or wet AMD, explained the research leader, Steve Perkins.
"The earliest hallmark of AMD is the appearance of protein, lipid and zinc deposits under the retinal pigment epithelial cells," he said, adding that the yellowish deposits, usually discovered by an ophthalmologist, are commonly known as "drusen."
The researchers studied two proteins involved in drusen formation -- blood protein Factor H and a second blood protein known as C-reactive protein -- and showed that Factor H binds to C-reactive protein when C-reactive protein is present in large amounts, as in the case of infection, to reduce the potentially damaging effects of an overactive immune system.
"In the eye, during the normal processes of aging, cells will die naturally for all sorts of reasons," Okemefuna said. "The blood supply to the eye will bring C-reactive protein with it, and a low level of C-reactive protein activity will enable the normal processes of clearance of dead cells at the retina through mild inflammation. In conditions of high inflammation, the levels of C-reactive protein in the retina will increase dramatically."
Uncontrolled C-reactive protein activity causes damage to the retina, which is followed by more inflammation and then even more damage to the retina, and so forth.
"It's the debris of broken up retinal cells, some of which is caused by this cycle, that is deposited as drusen," Okemefuna said.
The team also found that a genetically different form of Factor H does not bind to the C-reactive protein quite as well as the normal one, making people who carry the modified protein more vulnerable to an immune system attack in the eye and, thus, drusen buildup.
"In normal individuals, further damage to the retina by prolonged exposure to high levels of C-reactive protein is prevented by Factor H. C-reactive protein also prevents Factor H from clumping together and initiating the processes that lead to drusen formation," Perkins said. "Both these 'good' activities of Factor H are much reduced in the genetically different form of Factor H."
While there is no known cure for AMD, existing therapies aim to treat the symptoms and delay progression.
"It is interesting how the interaction of these two blood proteins protects the eye during crisis," Perkins said. "The two proteins also can be involved in a rare and often fatal cause of kidney failure in children. We now are better positioned to begin to work out preventative strategies for these diseases."
For more information go to www.maculardegenerationassociation.org
Researchers at University College London say they have gleaned a key insight into the molecular beginnings of age-related macular degeneration, the No. 1 cause of vision loss in the elderly, by determining how two key proteins interact to naturally prevent the onset of the condition.
In a paper to be published in a forthcoming issue of the Journal of Biological Chemistry, the team reports for the first time how a common blood protein linked to the eye condition reins in another protein that, when produced in vastly increased amounts in the presence of inflammation or infection, can damage the eye.
"By starting to understand these interactions in greater detail, we can begin to devise methods that will ultimately prevent the development of blindness in the elderly," said Zuby Okemefuna, the lead author of the paper to be published Jan. 8.
Age-related macular degeneration, or AMD, is painless but affects the macula, the part of the retina that allows one to see fine detail. One form of the debilitating condition, known as "wet" AMD, occurs when abnormal and fragile blood vessels grow under the macula, leaking blood and fluid and displacing and damaging the macula itself. The second form, "dry" AMD, occurs when light-sensitive cells in the macula slowly break down.
It is believed that both forms start on a common molecular route and then deviate into dry or wet AMD, explained the research leader, Steve Perkins.
"The earliest hallmark of AMD is the appearance of protein, lipid and zinc deposits under the retinal pigment epithelial cells," he said, adding that the yellowish deposits, usually discovered by an ophthalmologist, are commonly known as "drusen."
The researchers studied two proteins involved in drusen formation -- blood protein Factor H and a second blood protein known as C-reactive protein -- and showed that Factor H binds to C-reactive protein when C-reactive protein is present in large amounts, as in the case of infection, to reduce the potentially damaging effects of an overactive immune system.
"In the eye, during the normal processes of aging, cells will die naturally for all sorts of reasons," Okemefuna said. "The blood supply to the eye will bring C-reactive protein with it, and a low level of C-reactive protein activity will enable the normal processes of clearance of dead cells at the retina through mild inflammation. In conditions of high inflammation, the levels of C-reactive protein in the retina will increase dramatically."
Uncontrolled C-reactive protein activity causes damage to the retina, which is followed by more inflammation and then even more damage to the retina, and so forth.
"It's the debris of broken up retinal cells, some of which is caused by this cycle, that is deposited as drusen," Okemefuna said.
The team also found that a genetically different form of Factor H does not bind to the C-reactive protein quite as well as the normal one, making people who carry the modified protein more vulnerable to an immune system attack in the eye and, thus, drusen buildup.
"In normal individuals, further damage to the retina by prolonged exposure to high levels of C-reactive protein is prevented by Factor H. C-reactive protein also prevents Factor H from clumping together and initiating the processes that lead to drusen formation," Perkins said. "Both these 'good' activities of Factor H are much reduced in the genetically different form of Factor H."
While there is no known cure for AMD, existing therapies aim to treat the symptoms and delay progression.
"It is interesting how the interaction of these two blood proteins protects the eye during crisis," Perkins said. "The two proteins also can be involved in a rare and often fatal cause of kidney failure in children. We now are better positioned to begin to work out preventative strategies for these diseases."
For more information go to www.maculardegenerationassociation.org
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