Acucela has the ambitious goal of fundamentally changing the way doctors treat the so-called “dry” form of age-related macular degeneration, which damages the eyesight of an estimated 29 million elderly people worldwide. The company was spun out of the University of Washington in 2002 by Ryo Kubota, an ophthalmologist on the faculty at the time. His dream is to create a once-daily oral pill that can slow down the progressive vision loss that comes with the dry form of macular degeneration, and ultimately to keep these aging patients from going blind.
The company got a big lift in September 2008 when it struck a partnership with Japan-based Otsuka Pharmaceutical to co-develop its lead drug candidate, and this month it took another big step by entering the first clinical trial that will assess what kind of effect its drug will have on the eyesight of between 50 to 100 elderly patients.
“We want to build a great company, and we want to build it to last. Our investors and our partner believes in it,” Kubota says.
Since Acucela doesn’t usually make a lot of news, some background is certainly in order. Acucela is vying to develop the first effective therapy for the dry form of age-related macular degeneration. It’s caused by yellowish buildups and a loss of pigment in the retina as people age. There is no approved treatment for this disease, unlike the “wet” form of age-related macular degeneration, which can be treated with Roche’s bevacizumab (Avastin) and ranibizumab (Lucentis), which cut off blood flow to leaky vessels behind the eye. The dry form of the disease is generally milder, but the market opportunity is thought to be huge, because as populations around the world age, it is expected to double in incidence over the next two decades.
Acucela’s idea comes in the form of a once-daily pill it calls ACU-4429. The drug is designed to block an enzyme called isomerase. By blocking that enzyme, Acucela hopes to slow down the visual cycle in which Vitamin A is constantly replenished. While people need a constant source of Vitamin A to see, a fast visual cycle can also contribute to the buildup of toxic Vitamin A byproducts in the eye, particularly one called A2E.
Back in May, Acucela presented the first evidence from a clinical trial that said its drug was on track. The initial trial enrolled 36 healthy volunteers at an escalating series of doses, and basically concluded that the drug was safe and well-tolerated.
This month, Acucela is attempting to pass a tougher test. The company started enrolling elderly people with severe cases of the dry form of macular degeneration. The trial, called Envision, will randomly assign patients to get the drug, or a placebo, at a variety of once-daily doses. Patients will be followed up for three months, and researchers will look at how well they can read letters on the eye chart, and a secondary goal that will assess whether the drug is working as intended, by reducing the amount of toxic byproduct buildup in the eye. It’s possible that the trial, as long as it appears safe at escalating doses, could enroll as many as 100 patients, Kubota says. It’s expected to take about one year to complete enrollment, and results should be available by mid-2011, he says.
Although Acucela is entering uncharted territory with a new way of treating this disease, it has competition from Tampa, FL-based Sirion Therapeutics. The competing drug is an oral pill like Acucela’s, made to circulate through the bloodstream, although it is designed to hit a different target than Acucela’s, Kubota says.
While Acucela awaits results from its mid-stage clinical trial, it is working to build up a lot of organizational strength this year, Kubota says. People with expertise in clinical development, preclinical development, and regulatory affairs are being recruited this year. By the end of 2010, Acucela expects to have hired about 60 new people, bringing its staff to about 100, he says. The company hopes to have that organizational horsepower in place by the time it expects to enter the final phase of clinical trials with ACU-4429, for an even more rigorous study that will assess patients’ vision for 18 to 24 months, Kubota says.
Although it’s rare among biotech companies without marketed products, Acucela has been able to operate on a cash-flow positive basis of late, Kubota says. That’s because Otsuka is paying for the company’s R&D expenses, while Acucela has received undisclosed milestone payments for getting this far in development, Kubota says.
“We’re going to be running big clinical trials,” Kubota says. “We think we can change the disease paradigm. Our goal is to cure blindness. It’s a huge ambition.”
For more information go to www.maculardegenerationassociation.org
Friday, January 22, 2010
Sunday, January 10, 2010
Injections, supplements can help limit age-related macular degeneration
Three years ago, Catherine Leisher began noticing words were blurred when she read the newspaper. A trip to the eye doctor yielded diagnosis of a condition she had never heard of: age-related macular degeneration.
“I can see to walk, I can look out the window, and I can write,” said the 83-year-old Swatara Twp. resident who has the disease in both eyes. “But I can’t read — it’s all blurred, and I just loved to read.”
Age-related macular degeneration, which affects some 15 million Americans, leads to damage of the macula, which is the central part of the retina responsible for central vision and controls the ability to read, drive and recognize people’s faces in detail.
“It’s a major public health issue,” said Dr. Ingrid Scott, professor of ophthalmology and public health sciences at Penn State Milton S. Hershey Medical Center. “Age-related macular degeneration is the leading cause of vision impairment and blindness in people over the age of 50 in the United States and the industrialized world.”
The incidence is expected to increase as baby boomers age, said Dr. Erik Chotiner, an ophthalmologist with Memorial Eye Institute in Lower Paxton Twp. “Statistics say that 1.75 million people have advanced AMD now, but that number is expected to rise to 2.95 million by 2025,” he said.
There are two types of the disease, dry and wet, which both can distort vision. Dry macular degeneration, the most common form, usually progresses gradually. The wet form, which occurs when abnormal or leaking blood vessels grow underneath the retina in the area of the macula, can lead to more rapid loss of central vision.
At first, Leisher, who suffers from the wet form, was frightened that she would eventually become blind. However, new drugs injected directly into the eye offer hope to people who suffer from the wet form of AMD.
The drugs, available since 2004, are called anti-VEGF, which stands for “vascular endothelial growth factor” — a family of proteins shown to be important in causing the growth of abnormal blood vessels that characterizes wet macular degeneration.
“Before these drugs, treatment was more primitive and really only helped stabilize, not improve, vision,” Chotiner said.
The anti-VEGF medications, which are injected into the back of the eye with a small needle, work by decreasing the growth of abnormal, leaky blood vessels.
“It can be uncomfortable, but I’ve never had a patient who wasn’t able to tolerate it,” said Scott, who administers a numbing medication to the eye before giving the shot. “With anti-VEGF drugs, 40 percent of patients have visual improvement, and 95 percent have stabilization of vision.”
“My vision is brighter,” said Leisher, who receives her injections at Penn State Milton S. Hershey Medical Center. “A lot of people I know won’t take the shots because they are afraid of the needle. I hesitated at first, but I think I’d be much worse off if I didn’t do it.”
Another option, called photodynamic therapy, involves injecting a photosensitive drug into a vein in the arm. The dye travels to the blood vessels in the eye and localizes preferentially to the abnormal blood vessels. A cold laser beam is shown into the eye and temporarily reduces blood flow to the abnormal blood vessels. This therapy can slow the rate of vision loss but is much less likely to improve vision like the anti-VEGF medications can, Scott said.
Randee Erb, a 62-year-old New Holland resident diagnosed with wet macular degeneration last spring, has had four anti-VEGF injections and is noticing that her blurred vision is better.
“I dread getting the shots,” said Erb, who has to take the day off work when she gets the injections because they make her eye burn. “But they give me hope that I will be able to see clearly again.”
Every day Erb tests her eyesight by looking at an Amsler grid, a chart of vertical and horizontal lines, which she has taped to her refrigerator. The grid helps patients gauge whether their vision is worsening and they should see their doctor, based on how clear and straight the lines appear. As in Erb’s case the daily exercise can also bring good news. “At first it was all gray; now I’m slowly seeing lines,” she said.
Some patients with AMD might benefit from taking vitamin supplements that can reduce the risk of severe vision loss due to the disease by 25 percent and/or reduce the risk of developing wet AMD by 25 percent, Scott said. Called the AREDS formulation, it includes vitamins A, C, E, zinc and copper.
There are preventative measures that people can take to decrease their chances of developing AMD, doctors said. These include wearing sunglasses, not smoking, cutting down on saturated fats and eating dark green, leafy and orange vegetables such as broccoli, spinach, carrots and sweet potatoes.
“It’s extremely important to have regular eye exams,” Scott said. “People have often just accepted a decline in vision as part of aging when, in fact, many causes of declining vision can be treated. For example, it’s been shown in clinical trial after clinical trial among patients with wet AMD that the leading predictive factor for a patient’s visual outcome is how early they are diagnosed and treated.”
For more information go to WWW.MACULARDEGENERATIONASSOCIATION.ORG
“I can see to walk, I can look out the window, and I can write,” said the 83-year-old Swatara Twp. resident who has the disease in both eyes. “But I can’t read — it’s all blurred, and I just loved to read.”
Age-related macular degeneration, which affects some 15 million Americans, leads to damage of the macula, which is the central part of the retina responsible for central vision and controls the ability to read, drive and recognize people’s faces in detail.
“It’s a major public health issue,” said Dr. Ingrid Scott, professor of ophthalmology and public health sciences at Penn State Milton S. Hershey Medical Center. “Age-related macular degeneration is the leading cause of vision impairment and blindness in people over the age of 50 in the United States and the industrialized world.”
The incidence is expected to increase as baby boomers age, said Dr. Erik Chotiner, an ophthalmologist with Memorial Eye Institute in Lower Paxton Twp. “Statistics say that 1.75 million people have advanced AMD now, but that number is expected to rise to 2.95 million by 2025,” he said.
There are two types of the disease, dry and wet, which both can distort vision. Dry macular degeneration, the most common form, usually progresses gradually. The wet form, which occurs when abnormal or leaking blood vessels grow underneath the retina in the area of the macula, can lead to more rapid loss of central vision.
At first, Leisher, who suffers from the wet form, was frightened that she would eventually become blind. However, new drugs injected directly into the eye offer hope to people who suffer from the wet form of AMD.
The drugs, available since 2004, are called anti-VEGF, which stands for “vascular endothelial growth factor” — a family of proteins shown to be important in causing the growth of abnormal blood vessels that characterizes wet macular degeneration.
“Before these drugs, treatment was more primitive and really only helped stabilize, not improve, vision,” Chotiner said.
The anti-VEGF medications, which are injected into the back of the eye with a small needle, work by decreasing the growth of abnormal, leaky blood vessels.
“It can be uncomfortable, but I’ve never had a patient who wasn’t able to tolerate it,” said Scott, who administers a numbing medication to the eye before giving the shot. “With anti-VEGF drugs, 40 percent of patients have visual improvement, and 95 percent have stabilization of vision.”
“My vision is brighter,” said Leisher, who receives her injections at Penn State Milton S. Hershey Medical Center. “A lot of people I know won’t take the shots because they are afraid of the needle. I hesitated at first, but I think I’d be much worse off if I didn’t do it.”
Another option, called photodynamic therapy, involves injecting a photosensitive drug into a vein in the arm. The dye travels to the blood vessels in the eye and localizes preferentially to the abnormal blood vessels. A cold laser beam is shown into the eye and temporarily reduces blood flow to the abnormal blood vessels. This therapy can slow the rate of vision loss but is much less likely to improve vision like the anti-VEGF medications can, Scott said.
Randee Erb, a 62-year-old New Holland resident diagnosed with wet macular degeneration last spring, has had four anti-VEGF injections and is noticing that her blurred vision is better.
“I dread getting the shots,” said Erb, who has to take the day off work when she gets the injections because they make her eye burn. “But they give me hope that I will be able to see clearly again.”
Every day Erb tests her eyesight by looking at an Amsler grid, a chart of vertical and horizontal lines, which she has taped to her refrigerator. The grid helps patients gauge whether their vision is worsening and they should see their doctor, based on how clear and straight the lines appear. As in Erb’s case the daily exercise can also bring good news. “At first it was all gray; now I’m slowly seeing lines,” she said.
Some patients with AMD might benefit from taking vitamin supplements that can reduce the risk of severe vision loss due to the disease by 25 percent and/or reduce the risk of developing wet AMD by 25 percent, Scott said. Called the AREDS formulation, it includes vitamins A, C, E, zinc and copper.
There are preventative measures that people can take to decrease their chances of developing AMD, doctors said. These include wearing sunglasses, not smoking, cutting down on saturated fats and eating dark green, leafy and orange vegetables such as broccoli, spinach, carrots and sweet potatoes.
“It’s extremely important to have regular eye exams,” Scott said. “People have often just accepted a decline in vision as part of aging when, in fact, many causes of declining vision can be treated. For example, it’s been shown in clinical trial after clinical trial among patients with wet AMD that the leading predictive factor for a patient’s visual outcome is how early they are diagnosed and treated.”
For more information go to WWW.MACULARDEGENERATIONASSOCIATION.ORG
Monday, January 4, 2010
Smoking Increases Risk Of Age-Related Macular Degeneration
According to a new study, it is not a good idea for older people to continue to smoke, as smoking is the second most common risk factor for developing age-related macular degeneration (AMD).
AMD is a disease that progressively damages the macula i. e. the centre of the retina that makes it possible for us see fine details. Degeneration of the macula prevents people from being able to read, recognise faces or drive, due to experiencing darkness or blurring of their central vision.
What this means is that it is never too late to quit smoking, as even the eyes of the elderly benefit from kicking the habit.
The new study trying to determine whether age influences the effects of smoking on the risk of developing AMD, followed a group of 1,958-women of whom 75 smoked. These women 78-years of age at the start of the baseline exam underwent retinal photographs at five year intervals.
Their retinal images were compared at ages 78 and 83 to see whether AMD had made an appearance, including evaluating whether smoking played a role in their likelihood of developing the disease.
Researchers found that the women who smoked had 11% higher rates of AMD overall, than other women of the same age. As well, they found that 80-plus women who smoked were 5.5-times more likely than the same age women who did not smoke to develop AMD.
The bottom line is that aging increases the risk of AMD, but smoking raises the risk even more.
The study’s findings have been published in the January edition of the American Journal of Ophthalmology.
For more information go to www.maculardegenerationassociation.org
AMD is a disease that progressively damages the macula i. e. the centre of the retina that makes it possible for us see fine details. Degeneration of the macula prevents people from being able to read, recognise faces or drive, due to experiencing darkness or blurring of their central vision.
What this means is that it is never too late to quit smoking, as even the eyes of the elderly benefit from kicking the habit.
The new study trying to determine whether age influences the effects of smoking on the risk of developing AMD, followed a group of 1,958-women of whom 75 smoked. These women 78-years of age at the start of the baseline exam underwent retinal photographs at five year intervals.
Their retinal images were compared at ages 78 and 83 to see whether AMD had made an appearance, including evaluating whether smoking played a role in their likelihood of developing the disease.
Researchers found that the women who smoked had 11% higher rates of AMD overall, than other women of the same age. As well, they found that 80-plus women who smoked were 5.5-times more likely than the same age women who did not smoke to develop AMD.
The bottom line is that aging increases the risk of AMD, but smoking raises the risk even more.
The study’s findings have been published in the January edition of the American Journal of Ophthalmology.
For more information go to www.maculardegenerationassociation.org
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