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).
"This study is another important milestone in the development of tkRNAi-based therapeutics – an entirely new class of drugs – and it accomplishes two significant objectives at once," said Cequent Chief Executive Officer Peter Parker. "First, it serves as a Phase II-enabling toxicity study for our FAP drug, CEQ508. The FDA accepted our IND application for CEQ508 in December, and we expect to initiate our first-in-man Phase I trial in FAP (familial adenomatous polyposis) patients for CEQ508 this quarter. Second, it will provide important data on the Novartis IBD target in support of an FDA IND application, which we expect to file early next year. We believe the IBD target could be useful for patients suffering from ulcerative colitis. We designed the study to evaluate the safety profile of both drug candidates, as well as to demonstrate efficacy of our tkRNAi technology to silence the intended gene target.
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.
Cequent Vice President of Drug Development, Alison Silva, will present additional details about Cequent's clinical development program on Tuesday, April 20, 2010, at the American Association for Cancer Research (AACR) annual meeting in Washington, D.C., which will be held at the Walter E. Washington Convention Center. Details can be found below:
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.
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 entering clinical development, are orally administered drug candidates targeting colon-cancer prevention and inflammatory bowel disease. The company designed its powerful tkRNAi technology as a therapeutic 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, Inc.