Dicerna Snags Deal With Japan’s Kyowa Hakko Kirin to Develop RNAi Cancer Treatments

Xconomy Boston — 

Dicerna Pharmaceuticals has found some deep pockets to support its approach to creating RNA interference drugs. The Watertown, MA-based company is announcing today that it has formed an alliance with Japan-based Kyowa Hakko Kirin.

Under the deal, Kyowa Hakko Kirin will get access to Dicerna’s proprietary RNAi drug technology against one undisclosed target on cancer cells. In exchange, Dicerna will get $4 million in upfront cash, plus $120 million in milestone payments for success in development and commercialization, as well as royalties on future product sales. The partnership can be broadened over time to include as many as 10 more drug targets for cancer and other diseases, each with the same financial terms. Dicerna also has an option to co-promote and equally split the profits in the U.S. on the initial cancer drug.

This is the first significant partnership for Dicerna, a company founded in 2007 on the idea that it had found a “second doorway” of RNA interference. Like Cambridge, MA-based Alnylam Pharmaceuticals and others, Dicerna is seeking to specifically silence disease-related genes. One key difference is that Dicerna’s drugs are a little longer than so-called small interfering RNA molecules, and they interact with an enzyme called dicer that’s involved at an earlier step in the RNAi process. Kyowa Hakko Kirin has a long history of manufacturing and marketing biotech drugs in Japan, and it has its own internal teams devoted to RNAi research and development.

“They looked carefully at different siRNA opportunities for some time, and they chose this one,” says Dicerna CEO Jim Jenson. “They are committed to biotech, and they are a strong player in Japan.”

Dicerna still has a lot to prove about its method, as none of its treatments are yet in clinical trials. The partnership does provide cash that will enable Dicerna to add a few new faces to its staff of 23 employees, Jenson says. It also helps provide outside validation that should enable the company to raise a Series B venture round in 2010, which he expects will be worth about $25 million, he says.

Dicerna is hopeful that its drugs will be more potent than other RNAi therapies, and that they will last longer in the body. If that can be proven, it could translate into fewer injections for patients, lower manufacturing costs, and higher profit margins.

But delivery is the major challenge in RNAi today, because most treatments given directly through injection get filtered out by the kidneys before they can have the desired effect on the target. Lots of energy is being poured into new ways to deliver the RNAi drugs in the body, and that is one important feature of the Kyowa Hakko Kirin collaboration with Dicerna. The Japanese company has its own technology for using liposomes to deliver the RNAi drugs. Dicerna is developing its own lipid nanoparticle delivery technology in house, Jenson says, although it is trying a variety of approaches, like using antibodies, peptides, small molecules, or other ways of getting an RNAi drug where it’s supposed to go.

Dicerna doesn’t envision itself becoming a research wing for Kyowa Hakko Kirin. Rather, this partnership will lead to more alliances and help the small company build up its own pipeline of wholly owned drug candidates, Jenson says. Already, he says the company has commitments from its existing investors—Oxford Bioscience Partners, Abingworth, and Skyline Ventures—to invest in a Series B venture round that just needs a new investor to join the syndicate.

That round of investment will go toward building up the Dicerna pipeline, he says. While the Japanese partnership will focus in the beginning on a “novel” target for a cancer drug, Dicerna’s own program will concentrate instead on a less-risky target that has been validated by other drugs, Jenson says. The strategy is that by silencing a validated target, there’s less chance of an unexpected safety problem emerging, which could cast a cloud over the whole RNAi field, he says.

“Clinical successes in this field are badly needed,” Jenson says.

Jenson wouldn’t say which target Dicerna is going to pursue first, but he did offer some clues. Dicerna’s ideal target will be measured by a validated biomarker, he says, so the company will be able to take a biopsy from patients and determine whether its RNAi drug is getting to where it is supposed to be in the body, and silencing the intended target. Ideally, the company won’t have to wait very long for this sort of scientific validation. Dicerna has already been working on a clinical trial design, and expects to bring that drug into human testing in 2011, Jenson says.

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