SHANGHAI – Ark Biosciences Inc. successfully completed a phase I trial for AK0529, an oral drug designed to treat respiratory syncytial virus (RSV), a common virus that can be deadly in premature infants, children and the elderly by infecting the lungs and respiratory tract.

A small start-up founded just over a year ago, Ark has moved swiftly. Founder and CEO Jim Wu left his position behind at a Roche AG lab in China, deciding to go out on his own with AK0529, a project he worked on for the Swiss pharma. Roche struck a licensing deal with Ark for global and China rights for an undisclosed amount. (See BioWorld Today, April 9, 2015, and June 25, 2015.)

Currently, there is no approved drug or vaccine for RSV, but there are at least two big contenders in the space.

Last year, Johnson & Johnson, of New Brunswick, N.J., paid $1.75 billion for Alios Biopharma Inc., getting its hands on promising RSV candidate AL-8176, a phase II oral nucleoside analogue. Meanwhile, Gilead Sciences Inc., of Foster City, Calif., has been pursuing GS-5806, an oral fusion inhibitor for RSV that posted phase II results in healthy volunteers. (See BioWorld Today, Oct. 1, 2014.)

Ark's AK0529 is designed to work by inhibiting RSV replication, blocking viral entry and syncytium formation. Although coming from behind in terms of trial timeline, "we are confident we can catch up and be the leader in the field," said Wu.

Ark's phase I study, conducted in Australia, was a single and multiple dose-ascending study that looked at the safety, tolerability and pharmacokinetics of AK0529. The trial was conducted in 64 healthy subjects. The maximum single administered dose was 1,200 mg, while the multiple dose study administered 600 mg for seven days.

"The results are very good," Wu told BioWorld Today. "Clearly our compound is very safe with good pharmacokinetic properties, so we can move the compound further for testing in RSV patients."

Ark will continue to pursue its dual path – undertaking a bridging study and/or phase II trial in multiple clinical trials sites globally – as well as working with the CFDA to obtain investigational new drug application approval for studies in China, hopefully early in the new year.

The global trial will require multiple sites, Wu explained, to meet the challenge of recruiting hospitalized RSV pediatric and adult patients, and will need to be timed to coincide with the winter flu season.

He said the firm is keen to test the drug in patients as soon as possible and is exploring the use of child patients – typically much harder to recruit for an unproven drug – as well as older adults.

"It is important for industry to work on pediatric drugs," Wu said. "It is also an area with less competition, giving us a lot of space to operate."

There is significant need for the elderly to receive effective RSV treatment, too.

Results from a large-scale Asian study released in 2013 discovered RSV is tough to diagnose correctly and often remains poorly understood due to lack of diagnosis. But when properly identified and tracked, it was found the virus caused serious complications in the elderly. "Given the high morbidity and mortality of RSV in these older adults, effective antiviral treatment may have a large impact on the health care system," concluded the researcher. (See BioWorld Today, Oct. 30, 2013.)

THE NEXT GILEAD?

Fortunately for Ark, the regulatory situation in China – especially for companies developing new pediatric drugs – looks set to improve. In May 2014, key government health ministries issued a joint opinion to encourage the research and development of pediatric drugs, including provisions setting out for the first time a separate regulatory pathway for pediatric drugs, and the acceptance of clinical trial data from overseas.

Wu said he believes Ark could be the first homegrown Chinese biotech to develop a pediatric drug with global potential and he is hopeful the CFDA and the CDE will consider Ark for priority review.

Ark is also looking to diversify its pipeline with antiviral and/or pediatric drugs, and Wu said to expect several updates next month regarding the company's China program for AK0529 and successful in-licensing deals.

"Biotech is booming in China right now," Wu said. "The government is supportive and there are a lot of venture capitalists looking to invest. Unfortunately, there are not that many assets in China, so a company with good assets has lots of value. Many VCs come to talk to us and show great interest in investing," he added. "It is a good time to start a biotech in China."

Perhaps that uplift in cash and government support will help Ark realize its goals. "Our company is focused on antiviral and pediatric drugs. But our dream is to become the next Gilead," Wu said. "We try to inspire ourselves."