Navigation Links
FDA Grants Cequent IND - Clears Way for First-Ever Clinical Trial of Orally Delivered RNAi Therapeutic: CEQ508, a tkRNAi Drug Candidate in Oncology
Date:12/10/2009

CAMBRIDGE, Mass., Dec. 10 /PRNewswire/ -- Cequent Pharmaceuticals, a pioneer in the development of novel products to deliver RNAi-based treatments to prevent and treat human disease, announced that the U.S. Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) approved Cequent's first IND (investigational new drug) application yesterday. This action enables Cequent to initiate the first-ever trial of an orally administered RNA interference drug in humans: CEQ508 - the company's lead drug candidate based on its proprietary tkRNAi technology. CEQ508 targets beta-catenin, a key oncogene implicated in the formation of colonic polyps and in the progression of polyps to colorectal cancer.

The IND application acceptance is the second procedural hurdle that the young company has cleared in order to begin human clinical trials of CEQ508 in the FAP (familial adenomatous polyposis) patient population. The company noted that the 14 reviewing members of the Recombinant DNA Advisory Committee (RAC) of the National Institutes of Health (NIH) voted unanimously to endorse the proposed clinical protocol after conducting an in-depth review and public discussion of the proposed CEQ508 program on September 9, 2009.*

"We submitted our IND application to the FDA on November 9, 2009. Having our first IND application accepted in the 30-day period is an extraordinary accomplishment for any company, particularly so for Cequent, given that we are working on an entirely new class of drugs," said Cequent Chief Executive Officer Peter Parker. "It speaks volumes to the quality of the submission, which comprised more than 4,000 pages. I'm so proud of our scientific team, who, in a short time, were successful in turning an intriguing RNAi research concept into a viable drug candidate ready to be tested in humans."

The FDA approved Cequent's proposed clinical protocol for the Phase I clinical trial, including the choice of the adult FAP patient population, selection of cohort size, the dosing regimen, and duration of treatment. The trial is designed to determine safety and tolerability of CEQ508, starting with a dose-escalation phase in which patients will be given one of four escalating concentrations of the drug, contingent upon safety monitoring. This initial part of the clinical trial is expected to take approximately six months. Next, following a safety review, the trial plan calls for a stable-dose phase in which additional patients will receive the highest safe dose. The FDA will permit daily dosing of 18 or more patients for 28 days. A key readout and secondary objective of the trial includes analysis of biomarker beta-catenin expression changes in the gastrointestinal tract of patients determined from biopsy samples obtained prior to taking the drug and at the end of the dosing period.

Cequent's Vice President of Drug Development, Alison Silva, commented, "I want to commend the reviewers at CBER who have been very responsive, providing critical feedback immediately following our submission and throughout the application process. They worked with us so that we could address their questions quickly and effectively to reach a successful conclusion by our action date."

With the FDA approval of Cequent's clinical-trial protocol now in hand, the company has begun working with the proposed clinical center to obtain the necessary institutional approvals. Cequent expects to begin the Phase I clinical trial 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. The Cancer Consortium maintains a registry of FAP patients and is also one of 40 National Cancer Institute-designated comprehensive cancer centers nationwide. Gideon Steinbach, M.D., Ph.D., is slated to be the principal investigator (PI) of the Cequent trial. Dr. Steinbach is associate professor of medicine at the University of Washington, and served as the PI of a previous celecoxib FAP Phase II trial that established a high standard for polyposis trials.

The upcoming Phase I FAP trial will serve as a proof of concept for Cequent's tkRNAi technology, according to Mr. Parker. "We believe our technology offers an elegant solution to the RNAi delivery problem that has stymied significant progress in the field to date. We modify live, nonpathogenic bacteria in a proprietary process to produce and deposit mediators of RNAi directly into the target cells. Follow-on drug candidates could potentially address dozens of different gene targets associated with more common and equally serious diseases. For example, we continue to make progress on our preclinical program for inflammatory bowel disease."

In preclinical testing 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.

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.

Financing update

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. In November 2009, it closed on $3.35 million in first-tranche equity financing from existing investors.

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.

*A video of the NIH RAC hearing is available at http://videocast.nih.gov/ram/rac090909.ram . The Cequent presentation begins at approximately 2:58 on the video.

SOURCE Cequent Pharmaceuticals, Inc.


'/>"/>
SOURCE Cequent Pharmaceuticals, Inc.
Copyright©2009 PR Newswire.
All rights reserved

Related medicine technology :

1. SRI International Receives National Institutes of Health Grants to Develop Improved Tomographic Imaging Technology
2. The Epilepsy Research Foundation and Milken Family Foundation Announce Prestigious Translational Research Grants for Two Pioneering Epilepsy Therapies
3. U.S. Food And Drug Administration (FDA) Grants Traditional Approval for INTELENCE(R) (etravirine)
4. FDA Grants GeoVax Labs, Inc. Request For Pre-IND Meeting
5. Ganeden Biotech Announces Grants for Early Investigators to Research Probiotic GanedenBC30
6. Cephalon Announces that FDA Grants Priority Review of its Supplemental New Drug Application for NUVIGIL as a Treatment for Excessive Sleepiness Associated with Jet Lag Disorder
7. Octapharma Accepting Applications for Research Grants on Immunotherapy, Coagulation Disorders and Critical Care
8. Swissmedic Grants Debiopharm Marketing Authorisation for Moapar(R), a New Therapeutic Avenue for the Treatment of Sexual Deviations
9. Swissmedic Grants Debiopharm Marketing Authorisation for Moapar(R), a New Therapeutic Avenue for the Treatment of Sexual Deviations
10. Oasmia: FDA Grants Paclical(R) Orphan Drug Designation for Ovarian Cancer in the USA
11. Rendell Administration Announces Health Research Grants From Tobacco Settlement Funds
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:2/8/2016)... Feb. 8, 2016  Unilife Corporation ("Unilife" or "Company") (NASDAQ: ... injectable drug delivery systems, today announced that it will release ... December 31, 2015 after market close on February 9, 2016. ... call to discuss these financial results.    About ... --> About Unilife Corporation ...
(Date:2/8/2016)... NORTHBROOK, Ill. , Feb. 8, 2016  Astellas Pharma ... today announced the promotion of James Robinson as ... the company,s operations in North and South America ... president, Astellas Pharma US, representing the commercial organization in ... assumed in 2013. Masao Yoshida , who ...
(Date:2/8/2016)... Palatin Technologies, Inc. (NYSE MKT: PTN), ... for the treatment of diseases with significant unmet ... the United States Patent and Trademark Office (USPTO) ... Patent Application Serial Number 14/313,258 (the ,258 application).  ... female sexual dysfunction using the formulation and dose ...
Breaking Medicine Technology:
(Date:2/8/2016)... ... ... Remember the old saying “rub some dirt on it”? Perhaps you should try ... Clay” the health benefits of integrating clay into a daily diet are numerous, as ... motivational speaker, Perry A~ has since dedicated her life to learning about the benefits ...
(Date:2/8/2016)... , ... February 08, 2016 , ... ... wide variety of organizations. DocuSyst provides a cloud hosted environment for FileHold ... installations include integration with various 3rd party applications using the FileHold web services ...
(Date:2/8/2016)... ... 2016 , ... GrassrootsHealth published data from its D*action public ... in the GrassrootsHealth cohort with substantially higher vitamin D levels than a cohort ... states Carole Baggerly, Director of GrassrootsHealth, “the safety and benefits of vitamin ...
(Date:2/8/2016)... (PRWEB) , ... February 08, 2016 , ... TopConsumerReviews.com recently ... in Mole removal products. , Moles are derived from a cluster of melanin when ... all the wrong places and create a lifetime of embarrassment. Historically, mole removal ...
(Date:2/8/2016)... ... , ... Guruji Mahendra Kumar Trivedi is offering 3 days of accelerated personal ... birthday on February 10th. During this time, people can achieve better health, greater ... people from over 40 different countries as an “ordinary man with an extraordinary gift.” ...
Breaking Medicine News(10 mins):