Opaganib (ABC294640) is a first-in-class, proprietary sphingosine kinase-2 (SK2) selective inhibitor, administered orally, with anticancer, anti-inflammatory and anti-viral activities.

Opaganib is an investigational drug targeting several potential oncology, inflammatory and gastrointestinal indications. Opaganib is also under development as a potential therapy for COVID-19.

Opaganib inhibits SK2, a lipid kinase that catalyzes formation of the lipid signaling molecule sphingosine 1-phosphate (S1P). S1P promotes cancer growth, and proliferation and pathological inflammation, including TNFα signaling and other inflammatory cytokine production. Specifically, by inhibiting the SK2 enzyme, opaganib blocks the synthesis of S1P which regulates fundamental biological processes such as cell proliferation, migration, immune cell trafficking and angiogenesis, and are also involved in immune-modulation and suppression of innate immune responses from T cells. Preliminary evidence suggests that because of its specificity for targeting SK2, rather than SK1, opaganib may have a better therapeutic ratio than nonspecific sphingosine kinase inhibitors or those targeting only SK1.

Opaganib completed numerous successful pre-clinical studies in oncology, GI-inflammation and radioprotection models, as well as a successful Phase 1 clinical study in cancer patients with advanced solid tumors.

A Phase 2a clinical study with opaganib in patients with advanced, unresectable, intrahepatic and extrahepatic cholangiocarcinoma is ongoing at renowned U.S. medical sites.

Opaganib was granted FDA Orphan Drug designation for the treatment of cholangiocarcinoma.

An investigator-sponsored Phase 2 study with opaganib in prostate cancer is ongoing at MUSC Hollings Cancer Center and Emory University.

RedHill announced top-line data and further analysis from its global Phase 2/3 study with opaganib in severe COVID-19 pneumonia. Results from prespecified analyses on endpoints of mortality and time to clinical recovery of Phase 2/3 opaganib data were recently released, in addition to previously announced data evaluating median time to viral RNA clearance vs. placebo.

RedHill also previously announced in December 2020 top-line safety and efficacy data from a Phase 2 study of opaganib in the U.S.

An investigator sponsored Phase 1b clinical study of opaganib for the treatment of multiple myeloma has been completed at Duke University Medical Center.

The clinical studies with opaganib are registered on www.clinicaltrials.gov, a web-based service by the U.S. National Institute of Health, which provides public access to information on publicly and privately supported clinical studies.

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* Yeliva® is a proposed tradename for the drug product containing opaganib, which is subject to review by the FDA at the time of NDA filing