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Marina Says Initial Tox Data Shows FAP Drug to be Safe

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Marina Biotech this week announced that a six-month non-human primate toxicology study of its oral familial adenomatous polyposis treatment CEQ508 has shown the drug to be safe.

FAP is an inherited, colorectal cancer syndrome characterized by the growth of colorectal polyps. Though the polyps are initially benign, they become malignant in nearly all cases in the absence of colectomy, according to the company.

CEQ508 is based on Cequent Pharmaceuticals so-called transkingdom RNAi technology, and is designed to inhibit the oncogene beta-catenin, which is expected to prevent new polyp formation and possibly slow the progression to malignancy of existing ones.

Marina picked up the drug candidate when it acquired Cequent earlier this year (GSN 4/1/2010).

In the primate study, CEQ508 was administered in an oral suspension once daily to the animals for 180 consecutive days, according to Marina. "During this period of time, no toxicity or test article related adverse events were observed."

The company noted that the toxicology study was extended by three months to support the potential requirements for a phase II trial. CEQ508 is expected to enter phase Ia/IIb testing this year.

"These interim results and the completion of the full nine-month safety study in non-human primates demonstrates the potential for the long-term treatment of patients with our novel RNAi-based therapeutic," Marina President and CEO Michael French said in a statement.