Nitto Denko and Quark Pharmaceuticals to jointly develop siRNA anti-fibrotic drug

Quark also signs licensing option agreement with Novartis for p53 suppressor

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FREMONT, Calif.—Nitto Denko will use its specialty delivery vehicle and targeting technology for fibrosis together with Quark Pharmaceuticals' proprietary RNAi technology to file for an IND for a new anti-fibrotic drug, under a collaboration announced last month.

The collaboration is designed to develop siRNA drugs using Quark's RNAi technologies and novel structures that provide freedom to operate in the siRNA intellectual property space coupled with Nitto Denko's drug delivery technologies for novel therapeutics.

The Nitto Denko technology is based on the research of Prof. Yoshiro Niitsu of Sapporo Medical University's School of Medicine and his group, as published in Nature Biotechnology Vol. 26 Issue 4 Pg. 431-442 (Apr 2008), "Resolution of liver cirrhosis using vitamin A–coupled liposomes to deliver siRNA against a collagen-specific chaperone." In the study, Niitsu and his team used vitamin A-coupled liposomes to deliver small interfering RNA (siRNA) against gp46, the rat homolog of human heat shock protein 47, to hepatic stellate cells. The approach almost completely resolved liver fibrosis and prolonged survival in rats with otherwise lethal dimethylnitrosamine-induced liver cirrhosis in a dose- and duration-dependent manner.

The collaboration will have an initial budget of double-digit million dollars to achieve the first IND from the U.S. Food and Drug Administration (FDA) by early 2012.

Quark claims to have the largest clinical-stage siRNA pipeline in the industry, based on its success at modifying the siRNA sequence based on the target.

"The key is adapting each siRNA with tailor-made modifications to reduce the electrostatic attraction and achieve immediate separation," says Dr. Daniel Zurr, Quark's CEO. "This collaboration is a perfect marriage between the core competencies of the two companies. We'll be using our technologies, intellectual property and capabilities to quickly bring drug candidates to clinical stage, and Nitto will provide its delivery technologies and therapeutic strategy as well as their world-class capabilities in oligonucleotide production."

Nitto Denko, he points out, is a major world supplier of polymeric formulations for drug delivery and has a leading market share of the amidite block, the major raw material for siRNA manufacture. In addition, Zurr notes that Quark's new partner may serve as its calling card with other companies in Japan that are interested in siRNA technology. In fact, he says he plans to orchestrate a road show in the immediate future to tour the country along with Quark's chief science officer and its medical director.

"I am very confident that siRNA drugs directed simultaneously to one or more specific target genes are the appropriate approach for therapies for a number of fibrotic diseases that are currently a totally unmet medical need," comments Niitsu. "Our research has demonstrated that the adequate siRNA, appropriately delivered to the liver, caused regression of liver fibrosis and significantly prolonged survival time in siRNA-treated animals. It is very likely that this approach is suitable to fibrotic diseases in other organs as well."

In its other recent agreement, Quark granted Novartis AG an option to obtain an exclusive worldwide license to develop and commercialize its p53 temporary inhibitor siRNA drug QPI-1002, currently in Phase II clinical trials. Quark will initially receive a non-refundable fee of  $10 million. In the event that Novartis exercises the option, Quark would receive option exercise fees and milestone payments that could potentially total $670 million. In addition, Quark would be entitled to potential royalties on sales of licensed products.

Expressing his enthusiasm over the agreement, Zurr states that, "with its world-leading expertise in transplantation and acute care, Novartis will provide invaluable support to the global development of QPI-1002, in development for the prevention of acute kidney injury (AKI) in patients undergoing cardiac surgery and for delayed graft function (DGF) in kidney transplant patients. The gene target of QPI-1002, p53, is a major player in apoptotic cell death; its temporary suppression rescues cells and prevents them from dying in conditions of severe stress such as ischemia, potentially opening opportunities for Novartis to novel treatments in additional indications."

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