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news.The US Food and Drug Administration (FDA) has granted fast track designation for Marina Biotech's investigational RNAi therapeutic, CEQ508, to treat familial adenomatous polyposis (FAP), for which currently there are no pharmaceutical approach available.
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FAP affects an estimated one in 10,000 people across the world and is associated with a near 100% risk of colon cancer if untreated.
Additionally, the company announced the allowance of US 13/196,436 covering the tkRNAi delivery technology, the delivery technology for CEQ508, in the US.
The patent covers a wide range of vectors for bacterial mediated gene silencing, as well as the fundamental invasive bacterium for delivering RNA therapeutics.
Marina Biotech president and chief executive officer Michael French said: "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.
"In addition, the recent US 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."
The company is currently carrying out a single-center, US-based trial (START-FAP), which is evaluating safety and tolerability of CEQ508 in patients with FAP.
The START-FAP trial is also evaluating the inhibition of ß-catenin messenger RNA (mRNA), the gene target for CEQ508.
The first two groups in the dose escalation phase of the START-FAP clinical trial have been completed.
The company noted that six patients with FAP completed the trial, three in group one at a dose of 1×108 colony forming units (cfu)/day and three in second group at 1×109 cfu/day.