Ra Pharma Pours $8.6M Into Discovery Tech and New Rare-Disease Drug

Xconomy Boston — 

Today Ra Pharmaceuticals CEO Doug Treco will make a presentation at a conference in Las Vegas that will cap off the Cambridge, MA-based company’s weeklong unveiling. On May 16, Ra announced it had closed an $8.6 million tranche of its $27 million Series A round. And today Treco will be describing the four-year-old company’s technology platform, which is designed to generate protein-like molecules that can attack a range of diseases.

Ra is developing a class of drugs called Cyclomimetics, which are protein fragments that have been modified to improve characteristics such as stability, safety, and affinity to disease-causing targets. The company licensed the technology from Anthony Forster, a professor at Uppsala University in Sweden. Ra is funded by New Enterprise Associates, Morgenthaler Ventures, Novartis Venture Funds, and Amgen Ventures.

Ra is generating Cyclomimetics via a drug-discovery platform it dubs Extreme Diversity. It works, Treco says, by tricking ribosomes—the protein-making machinery of the cell—into assembling protein fragments that contain non-natural components. “That typically allows you to make libraries with more than 10 trillion molecules,” which can then be screened against disease targets to generate new ideas for drugs, he says.

Treco says Ra has zeroed in on four therapeutic targets and made headway on its lead program: a drug to treat hereditary angioedema (HAE), a rare disease that causes painful swelling of the face, airways, and stomach. Ra’s drug is designed to prevent acute attacks of HAE by inhibiting an enzyme that mediates the swelling. The company plans to start animal trials soon.

HAE has proven to be a hot target for drug developers of late. There are already four drugs on the market, including the recently approved icatibant (Firazyr), marketed by Irish drug giant Shire (NASDAQ: SHPGY). But Treco points out that only one product—a drug made by Exton, PA-based ViroPharma—is approved to prevent attacks, and it’s one that patients has to be given by infusion. “Our idea is to provide a daily oral formulation,” Treco says.

Cyclomimetic molecules are quite large, which can be an advantage in that they can bind to disease targets at several different contact points, Treco says. But unlike typical biotech drugs, they can be made into pills—an attribute Ra is counting on to be able to offer a convenience advantage for patients. “We want to come in with easy-to-deliver treatments,” Treco says. “We’re building all of the things that are necessary to make orally available drugs into the backbone of this.”

Other companies are trying different methods for assembling new protein-based drugs. Cambridge, MA-based Ensemble Therapeutics has been building a library of synthetic, protein-like molecules based on a technology platform developed at Harvard, and it has signed on big-named partners Pfizer and Bristol-Myers Squibb. Another Boston area firm, Adnexus, developed a protein-design system for generating oncology drugs that was so attractive to Bristol-Myers it bought the company outright in 2007.

Treco says Ra’s technology platform is so productive the company is already getting ready to line up its next four drug targets. And the company has started talking to potential drug-development partners. “We’re really going to focus on targets that Big Pharma has had difficulty addressing,” he says.