A Triangle-based biotech company may have the answer to both vastly reducing the cost of new drug discovery while also significantly speeding up the process of finding successful drug candidates to fight a wide range of indications, including cancer, central nervous system disorders and heart disease.

The last week in June, RTP-based Cloud Pharmaceuticals, disclosed its first design of an active pro-drug for Crohn’s Disease and Inflammatory Bowel Disease in collaboration with the University of Florida (UF), and Ben-Gurion University of the Negev (BGU).

A pro-drug only becomes a drug when it is activated in the body by a specific, targeted protein. In this case, the pro-drug targets a membrane protein called PLA2. Studies have shown that PLA2 activity in severely inflamed mucosa of IBD patients is significantly higher than in normal colonic mucosa, so PLA2 may be exploited as a drug-activating enzyme, allowing drug targeting to the site(s) of gut inflammation.

But that’s only the latest of the company’s projects.

In 2015, Cloud Pharmaceuticals completed 17 drug designs using its unique Quantum Molecular Design (QMD) platform that it says can dramatically lower the cost of drug discovery–which a Tufts University study currently pegs at $2.5 billion. Part of that cost is because traditional methods of drug discovery result in only one drug compound out of 10,000 surviving clinical trials to achieve FDA approval.

At the leading edge

Using the QMD platform, rather than older methods such as high throughput screening, could slash discovery costs by 90 percent, according to the company. Cloud computing enables more sophisticated computational chemistry coupled with rapid synthesis and optimization to help companies identify compounds that are better suited for development from the outset.

QMD uses the super computing power of the cloud to search a “virtual universe” and identify new molecules that are designed directly from the characteristics of the binding pocket of the biological target. It filters molecules for chemical properties desirable for drugs and other properties that may be required. This “in silico” research, using computers to model and test potential drug molecules, is going to make drug discovery more efficient and less expensive.

Don Van Dyke, chief operating officer at Cloud, explains that “All the good molecules are in that virtual universe somewhere.” But that universe is so large it can be daunting to explore even for super computers. The number representing the size of that virtual universe of molecules is many times greater than the one representing the number of seconds since the beginning of the physical universe. “The way we can go out and interrogate virtual space using artificial intelligence is what sets up apart,” he says.

“A slam dunk, home run and world cup”

The difference between now and a year ago when the company didn’t have data is that “Every time we’ve tested, it resulted in a molecule active at the level we predicted. It’s a slam dunk, world cup, and home run for us,” in terms of the data it has now, says Van Dyke.

Following the work completed in the cloud, the carefully screened hit list is ready for further preclinical development steps. Applied to a much more qualified list of candidates, they result in a much higher success rate, and more rapid progress at lower cost on novel and hard-to-drug targets. Cloud Pharmaceuticals partners with contract research organizations (CROs) and risk-sharing partners to perform many of these steps. That includes the pro-drug for Crohn’s and IBD.

Some cash and a financial interest in a drug are part of its partnership arrangements, Van Dyke says. “We are at the leading edge of a wave beginning to achieve results. We’ve achieved more for less investment than anyone out there.”

The QMD process deploys on multiple cloud services: Amazon Web Services, ProfitBricks, Cloud & Heat, Google, and Microsoft Azure. “We designed it to run on any cloud platform,” Van Dyke says.

The company has raised about $3 million from investors and grants and is preparing to raise an institutional round of from $5 million to $10 million. Serial entrepreneur Ed Addison, CEO and chair, and Lawrence Husick, in-house counsel and chief innovation officer, founded the company in 2009.

Two of Addison’s previous ventures, BioFortis in Baltimore and Inclinix, a Wilmington CRO, merged with public companies. The third, TeraDisc, was a precursor to Cloud Pharmaceuticals. The company has 28 employees counting advisors.

It will be hiring computational and medicinal chemists and business development people with new funding, Van Dyke says.

The QMD technology is based on work by researchers at Duke University who developed novel algorithms to search chemical space. Cloud Pharmaceuticals improved and revised Duke’s original approach.

Cloud retains an ownership position in all of its partnership arrangements, which, in addition to universities, will likely soon include large pharmas. “We’re close to one with a top ten company and are in talks with eight others,” Van Dyke says.

http://www.cloudpharmaceuticals.com