Acucela, the Bothell, WA-based biotech company, is on a mission to develop the first effective treatment for the leading cause of blindness in elderly people. Today, it is presenting some preliminary data that shows its drug has passed an early safety test and is ready to advance to the next phase, in which it begins to make a case for whether the treatment really works.
The company is presenting data today on its lead internal drug candidate, ACU-4429, at the Association for Research in Vision and Ophthalmology’s annual meeting in Fort Lauderdale, FL. I got an overview of the results from Acucela CEO Ryo Kubota, an ophthalmologist who was the lead author on the scientific presentation at the meeting.
The company’s drug is designed to be an oral pill for what is known as the “dry” form of age-related macular degeneration. This disease is caused by yellowish buildups and a loss of pigment in the retina as people age, which causes a gradual loss of vision. There is no approved treatment for condition, unlike the “wet” form of disease, which can be treated with Genentech’s bevacizumab (Avastin) and ranibizumab (Lucentis), which cut off blood flow to leaky vessels behind the eye. The dry form of the disease, which is generally milder, nonetheless damages the eyesight of more than 20 million people worldwide, and with aging populations, it is expected to double in incidence over the next two decades.
Acucela still has a long way to go before it can tap into that market. The company tested its drug in 36 healthy volunteers between the ages of 55 and 80 who took a series of escalating doses, Kubota says. Researchers found the drug to be safe and well-tolerated, and lasted four to six hours in the bloodstream. Some secondary measurements—which will need confirmation in larger studies—suggested that the drug may slow down the cell division that causes the toxic buildup of deposits that damage eyesight, Kubota says.
Acucela was happy to see that the drug didn’t appear to cause any increase in liver enzymes that might suggest liver damage, which has been associated with earlier-generation compounds from a class of retinoid drugs, Kubota says. The newer drug candidate is a non-retinoid compound, he says.
“We could slow down the accumulation of toxic byproducts in the retina,” Kubota says. “This is a groundbreaking result.”
There are other companies that hope to make similar claims. Tampa, FL-based Sirion Therapeutics announced last month that its experimental drug, fenretinide, was able to reduce the growth rate of lesions in a Phase II clinical trial of patients after six months, and the effect increased over time. Othera Pharmaceuticals of Exton, PA also has generated some buzz in the ophthalmology world, with positive results from a trial that suggested its eye drop for the dry form of macular degeneration was able to help slow down the rate of vision loss.
Acucela is planning its next steps to amass the kind of convincing evidence ACU-4429 will need to become a competitive treatment, Kubota says. Acucela, along with its partner, Japan-based Otsuka Pharmaceuticals, plan to start a preliminary Phase II clinical trial this summer of 30 to 40 patients that will examine whether the treatment can slow down the visual cycle, a measurement thought to be an early indicator of whether a drug can successfully help preserve vision. Then, the company will advance to a larger Phase II trial that will run 18 to 24 months to get a more compelling read on the drug’s effectiveness, Kubota says.
Past years at this meeting have focused mainly on innovation in the wet form of age-related macular degeneration (AMD), although Kubota says he thinks that’s starting to change. “It’s blooming now in the world of dry AMD as well,” he says. “Wet AMD is being targeted now, and the next big thing is dry AMD.”
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