Acute Radiation Syndrome

Acute Radiation Syndrome

Opaganib (ABC294640) is a proprietary, investigational, first-in-class orally administered sphingosine kinase-2 (SPHK2) selective inhibitor.

Opaganib is being evaluated as a new therapeutic approach to mitigate gastrointestinal acute radiation syndrome (GI-ARS) and was selected by the U.S. Government's Radiation and Nuclear Countermeasures Program (RNCP) for development as a potential treatment for ARS as part of the nuclear medical countermeasures product development pipeline (announced February 2023). The RNCP is led by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH).

The U.S. Food and Drug Administration (FDA) have provided guidance on the applicability of the Animal Rule regulatory pathway for opaganib for ARS, utilizing pivotal animal model efficacy studies instead of human efficacy trials.

In an ARS setting, opaganib is thought to exert its protective effects via an anti-inflammatory mechanism of action involving ceramide elevation and reduction of sphingosine 1-phosphate (S1P) in human cells, supported by data from eight U.S. Government-funded in vivo studies published in the International Journal of Molecular Sciences.

Opaganib, an oral, small molecule pill with five-year stability data is easy to administer and distribute, supporting, if approved, potential central government stockpiling.