Orphan Drug Designation has been granted to ABC294640 by the FDA for the treatment of cholangiocarcinoma. ABC294640 is a phase II-stage, first-in-class, orally-administered sphingosine kinase-2 (SK2) selective inhibitor with anticancer and anti-inflammatory activities, targeting multiple oncology, inflammatory, and gastrointestinal indications. By inhibiting the SK2 enzyme, ABC294640 blocks the synthesis of sphingosine 1-phosphate (S1P), a lipid signaling molecule that promotes cancer growth and pathological inflammation.
The phase I study of ABC294640 in patients with advanced solid tumors, conducted at the Medical University of South Carolina Hollings Cancer Center, Charleston, successfully met its primary and secondary endpoints, demonstrating the drug is well-tolerated and can be safely administered to cancer patients at doses that provide circulating drug levels predicted to have therapeutic activity.
Of the three patients with cholangiocarcinoma treated in the phase I study, all of whom had prior therapy, one subject achieved a sustained partial response (OS=20.3 months) and the other two subjects had prolonged stable disease (OS=17.6 and 16.3 months)