CAMBRIDGE, Mass., Nov. 12 /PRNewswire/ -- Cequent Pharmaceuticals, a pioneer in the development of novel products to deliver RNAi-based treatments to prevent and treat human disease, has announced that it has filed its first IND (investigational new drug) application with the U.S. Food and Drug Administration (FDA). CEQ508, an orally administered tkRNAi drug candidate, targets beta-catenin, a key oncogene implicated in the formation of colonic polyps and in the progression of polyps to colorectal cancer. Cequent expects to begin the Phase I clinical trial in the FAP (familial adenomatous polyposis) patient population during the first quarter of 2010 at the Fred Hutchinson Cancer Research Center, in Seattle, Washington, part of the Fred Hutchinson/University of Washington Cancer Consortium.
This Phase I clinical trial, as proposed to the FDA, would be conducted to determine safety and tolerability of CEQ508 at escalating doses in a total of 18 adult FAP patients. A key readout and secondary objective of the proposed trial includes analysis of biomarker (beta-catenin) expression changes in the gastrointestinal tract of patients determined from biopsy samples taken prior to taking the drug, and following a daily, 28-day dosing regimen. The principal investigator (PI) of this clinical trial will be Gideon Steinbach, M.D., Ph.D., associate professor of medicine at the University of Washington and the PI of a number of earlier FAP studies. The Cancer Consortium maintains a registry of FAP patients and is also one of 40 National Cancer Institute-designated comprehensive cancer centers nationwide.
FAP is a rare inherited gastrointestinal disease that causes hundreds to thousands of precancerous polyps to form in the colon. Approximately 35,000 people in the U.S. carry the genetic mutation inherent to the disease. 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.
Mr. Parker continued, "We have now entered a new phase in the company's lifecycle; we have transitioned away from research to focus on our clinical programs, much of which we will pursue with contract resources. As such, we have promoted Alison Silva to the new position of vice president of drug development, overseeing all clinical development and regulatory affairs for the company. Co-inventor of the tkRNAi technology, Johannes Fruehauf, M.D., will support the upcoming clinical trial through a consultancy arrangement as vice president of medical affairs."
Recent financing developments
In October 2009, Cequent announced that it had initiated a Series B round of financing, seeking to raise $15 million to take the company through Phase II clinical trials with its lead drug candidate. This week, it closed on $3.35 million in first-tranche equity financing. Participating investors included Ampersand Ventures, Gold Hill Capital, Novartis Option Fund, Pappas Ventures, and Yasuda Enterprise Development, all of whom participated in the company's Series A funding in 2007. A second tranche from this group is expected to yield $3.2 million in 2010, subject to successful advancement of Cequent's tkRNAi technology into human studies.