Radiation exposure from a "dirty" bomb, a contaminated municipal water supply or a sabotaged nuclear reactor is a potential threat to national security. Exposure to radiation can lead to acute radiation syndrome (ARS), also called radiation sickness, a life-threatening illness that can damage or destroy multiple organs in the body. Currently, no treatment for the condition has been approved by the Food and Drug Administration (FDA). The investigators are working to develop RLIP76-LyoPL, a drug that can be given 24 hours or longer after radiation exposure. RLIP76-LyoPL prevents damage and death of cells injured by radiation. The drug could be given to civilians, military personnel and first responders as either a protectant against potential radiation exposure or as a treatment for ARS.
Radiation exposure can lead to ARS, a serious illness affecting multiple organs. Currently, radiation countermeasures (RCMs) are limited to supportive care only. The FDA has not approved any drugs to treat ARS, making RCMs a critical unmet need.
Terapio Corp. is developing an RCM that could be used as either a pre-exposure prophylactic or a post-exposure mitigator for civilians, military personnel and first responders. Terapio has demonstrated efficacy of the drug RLIP76- LyoPL when administered 24 hours or longer after radiation exposure, a vital criterion for the Department of Health and Human Services (HHS) when considering an acquisition for the Strategic National Stockpile.
Terapio’s therapeutic pipeline is based on RLIP76, a naturally occurring, membrane-associated transport protein encapsulated in a commercially available liposome that is lyophilized (RLIP76-LyoPL) using scalable methods. RLIP76 has a unique and known mechanism of action that allows it to prevent cell damage or death in the radiation cellular injury pathway. The long-term goal of the project is to provide HHS with a licensed RCM product for potential acquisition by the Strategic National Stockpile. NIH’s Radiation Nuclear Countermeasures program at the National Institute of Allergy and Infectious Diseases is co-funding the pre-clinical studies for this project.
Terapio Corp., Austin, Texas
Casey Cunningham, M.D.
Public Health Impact
Completion of scale-up and regulatory-compliant manufacturing of RLIP76-LyoPLmay enable Terapio to file a Biologics License Application with the FDA and provide HHS with a licensed RCM product for the Strategic National Stockpile that can be administered at least 24 hours after radiation exposure for the treatment of ARS.
Preclinical data did not warrant filing the Investigational New Drug application because efficacy data could not be reproduced with the non-GMP (Good Manufacturing Practice) drug product. Work on this project is complete.
- Process development
- Synthesis of non-GMP drug substance
- Manufacture of non-GMP drug product