Cequent Pharmaceuticals, Inc. Begins Chronic Toxicology Study with CEQ508, Lead Oncology tkRNAi Drug Candidate

Published: Apr 15, 2010

CAMBRIDGE, Mass., April 15 /PRNewswire/ -- Cequent Pharmaceuticals, a pioneer in the development of novel products to deliver RNAi-based treatments to prevent and treat human disease, has initiated a long-term (26-week) toxicology study of CEQ508 - the company's lead drug candidate based on its proprietary tkRNAi technology. This study is designed to enable a Phase II clinical trial slated for 2011. CEQ508 targets beta-catenin, a key oncogene implicated in the formation of colonic polyps and in the progression of polyps to colorectal cancer. The non-human primate (NHP) study will evaluate safety and gene knock-down with once-daily oral administration of CEQ508. As an addition to this study, Cequent has begun dosing with the therapeutic candidate optioned by Novartis to enable an upcoming IND application in inflammatory bowel disease (IBD).

In previous trials with non-human primates, Cequent's tkRNAi therapeutic candidates have demonstrated potent silencing of beta-catenin, a protein known to accumulate and lead to the proliferation of polyps in affected patients, and CEQ508 exhibited an encouraging safety profile when administered as a daily oral therapeutic. The 26-week study announced today will evaluate standard toxicology parameters including clinical observations, body weight, ECGs, blood pressure, food consumption, rectal temperature, ophthalmology, urinalysis, hematology, coagulation, and serum chemistry. In addition, there will be monthly endoscopies with biopsies to examine beta-catenin levels in the gut mucosa every 28 to 30 days including at baseline (before dosing) and upon recovery (after seven days of no dosing), and fecal sample collection to analyze shedding and clearance of the bacteria through the gastrointestinal tract.

Title: IND-enabling studies for CEQ508 targeting beta-catenin of GI polyps: First oral RNAi drug

Session ID: Experimental and Molecular Therapeutics 34

Session Date and Time: Tuesday Apr 20, 2010; 2 to 5 pm (ET)

Location: Exhibit Hall A-C, Poster Section 25

Permanent Abstract Number: 4513

About Familial Adenomatous Polyposis (FAP)

FAP is a rare inherited gastrointestinal disease that causes hundreds to thousands of precancerous polyps to form in the colon of an affected individual. Approximately 35,000 people in the U.S. carry the genetic mutation inherent to the disease, and the clinical researchers studying this disease have identified virtually all FAP patients. Today, without prophylactic removal of the colon, people with FAP almost inevitably develop cancer, and there is no generally accepted pharmacological treatment available. FAP has been designated as an orphan disease under the U.S. Orphan Drug Act, which provides various incentives for sponsors to encourage development of products for rare diseases. Phase I studies of novel therapeutics for such rare, underserved diseases are often allowed to enroll patients as opposed to healthy volunteers, potentially accelerating the timeline to develop approved products.

Cequent Pharmaceuticals, Inc.

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