Photos by Jared Soares | The Roanoke Times

Roanoke ophthalmologist Timothy Byrnes checks Freda King's eyes late last month after giving her an injection to treat her age-related macular degeneration, an eye condition. King said the injections sound worse than they are, but she would still prefer eyedrops.

Freda King, 82, gets her eyes checked last month in Roanoke before receiving an injection to treat age-related macular degeneration. A Roanoke company is trying to devise a new treatment.

The tiny bottle holds topical eyedrops that someday could radically transform the treatment of age-related macular degeneration -- an increasingly prevalent eye disease which, as it progresses, robs people of their sharp, central vision.

The key caveat here is "could" transform.

The drug, under development by Roanoke-based startup OcuCure Therapeutics, remains years away from market and must travel a formidable, expensive path to get there.

OcuCure, born and bred at the Carilion Biomedical Institute in Roanoke but now housed in a small office downtown, has cleared a few early hurdles.

It seems the company, one of CBI's star startups, recently moved ahead one giant step.

According to OcuCure, animal testing confirms that the eyedrop solution moves from the surface of the eye to the back -- penetrating to the treatment site for AMD "in a significant amount," according to Sunder Malkani, the company's president and chief executive officer.

"It's an important milestone for us," said Sam English, OcuCure's vice president of pharmaceuticals.

Previous testing and research in rats and rabbits at Eastern Virginia Medical School have suggested the drops are safe, effective and can be viably commercially produced, English said.

Payoff years away

Today, the most effective treatment for "wet" AMD, a more advanced form of the disease, requires regular injections into the eye.

Last Thursday, Freda King received her shot from Dr. Timothy Byrnes, a Roanoke ophthalmologist who is a retinal specialist.

The injections sound worse than they are, King said. But the eyedrops would be a whole lot better, she said.

But before the eyedrops reach King and millions of others with AMD, many more years of research and development, the securing of patents, regulatory approval and other potential barricades loom. Malkani estimates the process might require an additional four to five years.

"We've been working three or four years already," he said.

OcuCure has "three patents percolating through the system," English said. The startup hopes to "get into the Phase 1 human clinical trials in about 15 months," he said.

According to the U.S. Food and Drug Administration, Phase 1 trials try to determine dosing, document how a drug is metabolized and excreted, and identify acute side effects. Usually, a small number of healthy volunteers are used in Phase 1 trials.

There's another "could."

Patient investors could get rich.

That might happen if OcuCure, a privately held Virginia corporation, actually takes the topical eyedrops to market. Or, better yet, sells the concoction to some big, deep-pocket pharmaceutical company before the really costly final phase of human testing begins.

Pharmaceutical Research and Manufacturers of America estimates in a 2008 profile of the industry that it takes 10 to 15 years on average to develop a drug and that development costs about $802 million.

Of course, many consumer advocacy groups, including Public Citizen, argue that the pharmaceutical industry dramatically inflates those numbers to justify the high cost of prescription medicines.

OcuCure's bank account is a world away from $802 million.

The company launched in 2005 at CBI -- where English once served as coordinator of product development, research manager and director of university relations. He also worked as director of research for Carilion Clinic before becoming a consultant to inventors and entrepreneurs and an executive for OcuCure.

"The Carilion Biomedical Institute's help and its support have been instrumental to getting us where we are," Malkani said.

In 2006, OcuCure announced it had received about $1.5 million in seed money. Investors included CBI and individual "angel investors" who were willing to risk funding a startup because of the prospect of huge profits if all goes well.

English said the company has raised about $3 million to date. He did not disclose how much OcuCure has spent.

Byrnes is an OcuCure investor.

"There certainly is a lot of promise in this medication, but there are still many things that must happen before it reaches a pharmacy shelf," he said.

The promise

On June 30, 2006, the FDA announced it had approved a new drug, Lucentis, for treatment of the wet form of AMD.

In wet AMD, the more severe form, the growth of abnormal, leaky blood vessels eventually damages the macula, or central retina, the area of the eye responsible for central vision.

Dry AMD is more common, affecting about 85 percent of AMD patients. In the dry form, the delicate tissues of the macula become thinned and slowly lose function.

Today, nothing is available to treat dry AMD, which can become the more damaging wet AMD.

According to the FDA, wet AMD accounts for only 10 percent of all cases of macular degeneration, but this more severe form is responsible for 80 percent of the associated vision loss.

Lucentis, manufactured by Genentech, a publicly traded biotechnology company, is injected into the eye to transport the medicine to the back of it. It is designed to treat wet AMD by blocking new blood vessel growth and leakiness, which ultimately cause vision loss.

"Lucentis is a huge step forward in treating wet AMD from what was available before," English said.

Though rare, injections into the eye can lead to infection, bleeding in the eyes and retinal detachment, Byrnes said.

OcuCure hopes its topical eyedrops and a key, small molecule it delivers to the back of the eye would best Lucentis, which is reaping huge revenues for Genentech, in the treatment of AMD.

Why?

OcuCure believes its small molecule, discovered several years ago by a chemist at the University of Virginia, could stop development of new abnormal blood vessels and cause immature vessels to regress without affecting mature vessels, Malkani said.

In other words, if OcuCure is right, the drops could treat both dry and wet AMD and prevent the transition from dry to wet.

"If it does work, it will truly revolutionize the treatment of AMD," Byrnes said. "If you can keep the ones with dry AMD from going to the wet form, then you've hit a home run."

In addition, OcuCure's eyedrops might have applications for the treatment of diabetic retinopathy and other back-of-the-eye diseases.

Age-related macular degeneration affects more than 1.75 million people in the United States, according to the National Eye Institute. The institute anticipates the nation's aging baby boomers will push that number to nearly 3 million by 2020.

OcuCure estimates its eyedrops could reap annual revenues of $1 billion.

Early research suggests the drops might be an effective treatment also for diabetic retinopathy, Malkani said. The NEI estimates that 4.1 million adults 40 and older in the U.S. have diabetic retinopathy. An aging and increasingly obese population will cause this number to increase.

Meanwhile, Byrnes sounds like an investor without illusions. Many startup pharmaceutical companies fizzle and die long before Phase III clinical trails in humans, he said.