SOURCE: Marina Biotech, Inc.

Marina Biotech, Inc.

March 14, 2011 08:30 ET

Marina Biotech Announces First Patient Enrollment in Familial Adenomatous Polyposis Clinical Trial

CEQ508 Is the First Oral RNAi-Based Therapeutic to Enter Human Clinical Studies

BOTHELL, WA--(Marketwire - March 14, 2011) - Marina Biotech, Inc. (NASDAQ: MRNA), a leading RNAi-based drug discovery and development company, today announced enrollment of the first patient in the Dose Escalating Phase of its Phase Ib/IIa clinical trial, Safety and Tolerability of An RNAi Therapeutic in FAP or START-FAP, with CEQ508 intended for the treatment of Familial Adenomatous Polyposis (FAP). CEQ508 is the first ever orally administered RNAi-based therapeutic to reach human clinical development and recently received orphan drug designation from the Food and Drug Administration (FDA). 

The Dose Escalating Phase of the START-FAP trial involves an oral regimen, administered daily for 28 days. The trial is being conducted at Massachusetts General Hospital in adult patients with FAP. Enrollment involves a thorough screening process, including an in-depth assessment and assurance that the patient meets the study inclusion criteria, and occurs between 7 and 30 days prior to drug dosing. In addition, the patient must undergo endoscopy procedures one to seven days prior to the initiation of drug dosing. Following the course of drug administration, a second round of endoscopy procedures will be performed. Biopsies taken at baseline and end-of-treatment time points will allow safety evaluations as well as analysis of biomarker changes. 

"We're pleased to announce patient enrollment -- a first step, in what we hope will be a rapid regulatory and clinical timeline to the commercialization of CEQ508 for the treatment of patients with FAP," said J. Michael French, President and CEO of Marina Biotech. "Patients participating in the START-FAP trial will make a significant commitment throughout the trial period as each patient will undergo extensive pre- and post-dosing regimen procedures as well as daily visits by a home nurse to administer the CEQ508 dose. We commend and appreciate every patient participating in this trial as their dedication lays a critical framework upon which we can quickly advance to a pivotal trial and bring a much needed therapeutic option to patients in need."

About CEQ508

CEQ508 is the first drug candidate in a novel class of therapeutic agents utilizing the transkingdom RNA interference (tkRNAi) platform. CEQ508 comprises attenuated bacteria that are engineered to enter into dysplastic tissue and release a payload of short-hairpin RNA (shRNA), a mediator in the RNAi pathway. The shRNA targets the mRNA of Beta-catenin, which is known to be dysregulated in classical FAP. CEQ508 is being developed as an orally administered treatment to reduce the levels of Beta-catenin protein in the epithelial cells of the small and large intestine. Upon enrollment, patients will be placed in one of four dose-escalating cohorts. Following completion of the dose escalation phase, the trial plan calls for a stable-dose phase in which additional patients will receive the highest safe dose. CEQ508 will be administered daily in an oral suspension for 28 consecutive days. For more information please contact clinicaltrials@marinabio.com

About FAP

CEQ508 is being developed for the treatment of Familial Adenomatous Polyposis (FAP), a hereditary condition that occurs in approximately 1:10,000 persons worldwide. FAP is caused by mutations in the Adenomatous Polyposis Coli (APC) gene. As a result of these mutations, epithelial cells lining the intestinal tract have increased levels of the protein β-catenin, which in turn, results in uncontrolled cell growth. Proliferation of the epithelial cells results in the formation of numerous (hundreds to thousands) non-cancerous growths (polyps) throughout the large intestine. By age 35, 95% of individuals with FAP have developed polyps and most will experience adverse effects including increased risk of bleeding and the potential for anemia. In more severe cases, obstruction of the intestines, abdominal pain, and severe bouts of diarrhea or constipation can occur. FAP patients are also at an increased risk of various cancers, the most concerning of which is a nearly 100% occurrence of colon cancer if measures are not taken to prevent the formation of polyps. For many patients, complete colectomy (surgical removal of the entire large intestine), usually performed in the late teenage years or early twenties, is the only viable option for treatment. However, surgical intervention is not curative as the risk of polyps forming in the remaining portions of the intestinal tract and in the small intestine continues after colectomy.

About Marina Biotech, Inc.

Marina Biotech is a biotechnology company, focused on the development and commercialization of RNA interference- (RNAi) and RNA-based therapeutics. The Marina Biotech pipeline currently includes a clinical program in Familial Adenomatous Polyposis (a precancerous syndrome) and two preclinical programs -- in hepatocellular carcinoma and bladder cancer. Marina Biotech has recently entered an exclusive agreement with Debiopharm Group for the development and commercialization of the bladder cancer program. Marina Biotech's goal is to improve human health through the development of RNAi and RNA-based compounds and drug delivery technologies that together provide superior therapeutic options for patients. Additional information about Marina Biotech is available at http://www.marinabio.com.

Forward-Looking Statements

Statements made in this news release may be forward-looking statements within the meaning of Federal Securities laws that are subject to certain risks and uncertainties and involve factors that may cause actual results to differ materially from those projected or suggested. Factors that could cause actual results to differ materially from those in forward-looking statements include, but are not limited to: (i) the ability of Marina Biotech to obtain additional funding; (ii) the ability of Marina Biotech to attract and/or maintain manufacturing, research, development and commercialization partners; (iii) the ability of Marina Biotech and/or a partner to successfully complete product research and development, including preclinical and clinical studies and commercialization; (iv) the ability of Marina Biotech and/or a partner to obtain required governmental approvals; and (v) the ability of Marina Biotech and/or a partner to develop and commercialize products that can compete favorably with those of competitors. Additional factors that could cause actual results to differ materially from those projected or suggested in any forward-looking statements are contained in Marina Biotech's most recent periodic reports on Form 10-K and Form 10-Q that are filed with the Securities and Exchange Commission. Marina Biotech assumes no obligation to update and supplement forward-looking statements because of subsequent events.

Contact Information

  • Contacts:
    Marina Biotech, Inc.
    Pete Garcia
    Chief Financial Officer
    (425) 908-3603
    Email Contact

    Alison Silva
    Vice President, Clinical Development
    (617) 995-7943
    Email Contact