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FDA Grants Fast Track Designation to CEQ508 for Familial Adenomatous Polyposis
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The FDA has granted fast track designation to CEQ508, an investigational RNAi therapeutic for familial adenomatous polyposis, the manufacturer announced.
According to a press release, the manufacturer has also received a U.S. patent allowance covering the delivery technology for CEQ508 (transkingdom RNA interference [tkRNAi], Marina Biotech), which covers a range of vectors for bacterial mediated gene silencing and fundamental invasive bacterium for delivering RNA therapeutics. CEQ508 is the first drug candidate in a novel class using this delivery platform, and the drug contains attenuated bacteria designed to enter dysplastic tissue and release short-hairpin RNA, which targets the mRNA of beta-catenin, known to be dysregulated in classical familial adenomatous polyposis. The drug is being developed as an oral treatment that will reduce beta-catenin protein levels in small and large intestinal epithelial cells.
“We believe that fast track designation, together with orphan drug status previously granted by the FDA for CEQ508, provides Marina a unique opportunity to address an unmet medical need and bring CEQ508 to patients with familial adenomatous polyposis as quickly as possible,” Michael French, president and CEO of Marina, said in the release. “The fast track designation for CEQ508 can significantly reduce the review time of a new drug application and therefore reduce the time to market. In addition, the recent U.S. patent allowance for the tkRNAi delivery technology expands the broad international coverage of this technology, which now includes related patents granted in Europe, Japan, Korea, Australia and Canada. Fast track designation for CEQ508 and the further expansion of the intellectual property estate surrounding the tkRNAi platform continues to build the value of our pipeline and, in particular, our lead clinical program.”
The company’s Safety and Tolerability of an RNAi Therapeutic in Familial Adenomatous Polyposis (START-FAP) study aims to evaluate the safety and tolerability of CEQ508, as well as the inhibition of the drug’s gene target, beta-catenin messenger RNA. According to the release, the dose escalation phase of the clinical trial has already been completed, in which two cohorts (three patients in each) received different doses of oral CEQ508 once a day for 28 days. The company expects a third cohort in the study (stable dose phase) to be initiated in 2016 upon receipt of funding or a development or marketing partner.
“The oral delivery of CEQ508 is unique in the RNAi and nucleic acid space,” Alan W. Dunton, MD, chief medical officer of Marina, said in the release. “CEQ508 has been well tolerated and no serious adverse events have been reported. The data collected for cohorts one and two indicates some impact on mRNA, an encouraging finding at this stage of development.”
Familial adenomatous polyposis affects around one in 10,000 people globally, is linked to a nearly 100% risk for colon cancer if untreated, and there is no medication available to treat the condition currently, according to the release.
Disclosures: French and Dunton are employees of Marina.