Acucela, Turning 10, Gears Up for Pivotal Test With Eye Drug

Any one of a thousand little decisions can trip up an experimental drug on its long journey to the marketplace. Now, after 10 years in business, Seattle-based Acucela will be making a lot of important decisions that will determine whether it someday sells a drug that could help many people from going blind.

Acucela, along with its partner Otsuka Pharmaceutical, is designing the key clinical trial this year for a first-of-its-kind daily pill for the so-called “dry” form of age-related macular degeneration. Acucela has been emboldened to take that step, investing years of effort and millions of dollars, after it passed a mid-stage clinical trial of this drug, ACU-4429.

“Some important decisions are in front of the company now,” says founder and CEO Ryo Kubota. “It’s a really exciting moment for us.”

The 80-person company, founded when Kubota left his job as an ophthalmology researcher at the University of Washington, has spent the last decade maneuvering into position to find out if it can come up with a new drug to help stop elderly people from losing their sight. Acucela, which has raised $40 million in three rounds of venture financing, is betting that it has discovered the first treatment that can slow down the accumulation of toxic metabolites in the retina, which causes progressive vision loss in elderly folks.

Ryo Kubota, Acucela CEO

While there have been some notable advancements in treatment of the “wet” form of macular degeneration, in which blood vessels leak behind the eye to obscure vision, there hasn’t been much progress in treating the “dry” form of disease Acucela is going after. If Acucela can prove its drug can make a difference over the next couple of years, it could put itself in position to sell the first FDA-approved drug for the “dry” form of macular degeneration—a condition that damages the eyesight of more than 20 million people worldwide. That number is expected to double in the next 20 years, as populations age around the world.

Acucela still has a lot of work to do before it can even think seriously about marketing its drug, dubbed ACU-4429.

Here’s what is known so far: Acucela’s compound was recently tested in a mid-stage study of 56 patients, who were between 75 and 80 years old and got the new once-daily pill or a placebo. These patients had their pupils dilated, and were exposed to three minutes of bright light. Researchers then looked at electroretinograms to see how their rod cells recovered over time.

What the test showed was that the Acucela drug could slow down the visual cycle of rod cells in the eye. That’s thought to be an indicator that a drug might slow down the disease, because as people live longer, they essentially experience many cell divisions that yield toxic byproducts—particularly one called A2E, which ends up accumulating in the retina and obscuring vision over time.

Researchers said the Acucela drug reached a steady state after about 14 days, and the effect continued … Next Page »

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