The looming threat of nuclear attacks and recent non-terrorist related accidents (Fukushima nuclear disaster) has increased national and global attention to the need for medical countermeasures. RxBio has identified a first-in class proprietary small molecule-Rx100 which significantly protects the gut and the hematopoietic system from exposure to lethal doses of ionizing radiation. We have demonstrated the radiomitigant efficacy of Rx100 in murine and non-human primate (NHP) models for GI-ARS (Gastrointestinal Acute Radiation Syndrome). RxBio has completed a spectrum of drug development activities in consultation with FDA. The overall goal of this proposal is to complete manufacturing chemistry studies to support regulatory activities in advancing Rx100.lysine for eventual FDA licensure. The Phase I program specific aims are: 1) to evaluate the raw material standards for acceptance to manufacture highly pure Rx100.lysine by examining the stability of oleyl alcohol and investigating potential degraded or source impurities of oleyl alcohol via identification and structural characterization and 2) to investigate process improvement studies and stressing of current chemistry process in each manufacturing step and demonstrate the robustness of modified process via lab scale synthesis of highly pure API. Proposed studies will be completed in 12 months with two major deliverables: a) Establishing the acceptance criteria for oleyl alcohol for GMP scale-up (effort: 1 - 5th month); b) Improved manufacturing process to generate highly pure Rx100.lysine and batch record for lab scale synthesis (effort: 6 - 12th month).
Inevitably, nuclear accidents occur; and nuclear attacks and radiation terrorism have become a real threat to our nation since September 11, 2001. Currently, there are no FDA approved medical countermeasures available today for treating radiation injury. We are proposing to develop a novel class of radiation medical countermeasures to protect the American people and mankind from radiation injury.