Brain teaser time: Name the Bothell, WA-based biotech company that has never raised a nickel from local VCs, has scored a partnership with a Big Pharma company, operates on a cash-flow positive basis, and plans to more than double in size from 40 employees to about 100 this year.
Think it’s Alder Biopharmaceuticals? Guess again. It’s Acucela.
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.”
