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Cequent to Present First Proof of Activity of an Oral RNAi Drug in Non-Human Primates at February Keystone Conference

Tuesday, January 6, 2009 General News J E 4
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CAMBRIDGE, Mass., Jan. 6 Cequent Pharmaceuticals, a pioneer in the development of novel products to deliver RNAi-based treatments to prevent and treat human disease, announced that the company has recently completed a successful toxicology study of its candidate CEQ501, an orally administered tkRNAi therapeutic targeting the primary oncogene (beta-catenin, CTNNB1) in FAP (familial adenomatous polyposis). FAP is an inherited gastrointestinal disease that causes hundreds of polyps to form in the colon. Today, without prophylactic removal of the colon, people with FAP almost inevitably develop colon cancer, and there is no medical treatment available. Cequent will present its findings, the first-ever proof of activity of an oral RNAi drug in non-human primates, in a poster at the Keystone Symposia Conference, Therapeutic Modulation of RNA Using Oligonucleotides. The conference will be held February 8 - 13 in Lake Louise, Alberta Canada.



"Delivery of RNAi into the targeted cells and tissues remains one of the biggest challenges in the development of RNAi-based therapeutics - a new class of drugs designed to work by effectively deactivating the specific gene or genes implicated in the progression of a disease," said Cequent President and CEO, Peter Parker. "We have shown previously in cell-culture assays and in a mouse model for human colon cancer that our tkRNAi technology effectively suppresses beta-catenin, a key oncogene in FAP. These new results are very encouraging, and represent another significant milestone in our work to turn the promise of RNAi into safe and effective therapies to treat devastating diseases, like FAP, and improve the lives of those affected."



The study showed that tkRNAi targeting beta-catenin was well tolerated at high dosing levels, with no product-related adverse events, no increase in serum cytokines, and no gross or histopathologic abnormalities noted. Orally administered bacteria expressing short hairpin RNA (shRNA) against beta-catenin significantly reduced messenger RNA (mRNA) levels of beta-catenin in the mucosa of the small and large intestine, an effect that was reversible after termination of dosing. Samples obtained from the gastrointestinal mucosa indicate that oral tkRNAi treatment was effective in up to 84 percent of animals, and target gene levels were suppressed between 58 to 64 percent compared to control animals. Pharmacokinetic studies verified the presence of the engineered shRNA in the intestinal mucosa, proving successful delivery of the active hairpin RNA component by the bacterial system. Cequent developed the current tkRNAi candidate on the basis of an E. coli delivery platform obtained from the laboratory of Patrice Courvalin at the Institut Pasteur. Cequent is planning to start a Phase I clinical trial for the indication of FAP in 2009.



About Cequent Pharmaceuticals, Inc. (www.cequentpharma.com)



An early-stage biopharmaceutical company, Cequent is pioneering the development of novel therapeutics to prevent and treat a wide range of human disorders - from inflammatory disease to cancer - based on the company's proprietary technology, TransKingdom RNA interference (tkRNAi). Cequent's first products, now in pre-clinical development, are drug candidates targeting colon-cancer prevention and inflammatory bowel disease. The company designed its powerful tkRNAi technology to deactivate specific disease-causing genes safely and effectively, using non-pathogenic bacteria as an engine to produce and deliver RNAi directly into cells. It is based on ground-breaking scientific research originating at the Institut Pasteur (Paris, France) and at the Beth Israel Deaconess Medical Center/Harvard Medical School. A privately held company based in Cambridge, Massachusetts, Cequent was established in 2006.



SOURCE Cequent Pharmaceuticals
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