Capture Pharmaceuticals, Inc. is developing a drug called C2E2 for treatment of individuals who have been contaminated by radioactive transuranic elements, either by accident or following a nuclear terrorism event. C2E2 is a radionuclide decorporation agent that works by binding these elements in the bloodstream and enhancing their elimination before they can be trapped inside target organs such as the liver and skeleton. Because it can be taken orally, C2E2 has significant advantages for use in a mass casualty scenario over currently available products which must be administered by intravenous injection, and is superior to other competing technologies. The ultimate goal of this project is to advance C2E2 through FDA product registration and manufacture it for sale to local, national and international parties that need to prepare for the potential of mass contamination of the population with radioactive elements like plutonium and americium. This includes state and federal government agencies responsible for maintaining stockpiles of critical medicines (e.g., the U.S. Strategic National Stockpile), as well as foreign entities where the threat of nuclear terrorism is constant and imminent. Capture had a pre-IND meeting with the FDA at which time the regulatory pathway for C2E2 was established. A number of IND-enabling tasks have been completed, including detailed pre-formulation characterization of the drug molecule, extensive safety-toxicology assessments and efficacy studies in animal models. These studies demonstrated the feasibility of C2E2 as an oral radionuclide decorporation agent and justify submission of this Direct-to-Phase II SBIR proposal. The goal of this SBIR proposal, which will be conducted by Capture and its academic partner (UNC), is to complete well-defined IND-enabling activities that will bring the C2E2 product to a sufficient Technology Readiness Level so that subsequent funding from the Biomedical Advanced Research and Development Authority (BARDA) can be secured for manufacturing/scale- up activities, safety assessment in humans, efficacy measurements in animals as per the 'Animal Rule', and submission of a New Drug Application. This will be achieved by completing metabolic and bioavailability studies with C2E2, developing the Chemistry, Manufacturing & Controls section of the IND, completing safety- pharmacology studies and preparing an IND application. The continuing need for a radionuclide decorporation agent for accidental or deliberate contamination with transuranic elements has been highlighted by a 2014 INTERPOL report which warned of the increasing threat of nuclear terrorism and called for urgent preparedness. In order to expand the commercial utility of C2E2, its ability to serve as a Gd detoxifying agent following administration of MRI contrast agents will also be assessed. The goals of this SBIR application fit the BARDA Strategic Plan for 2011-2016, which emphasizes development of medical countermeasures not being developed by private industry to address unmet needs resulting from intentional threats to public health like nuclear terrorism, as well as the goals of the Public Health Emergency Medical Countermeasures Enterprise.
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