On Jul. 22, 2020, RedHill Biopharma announced that it had submitted a Clinical Trial Application (CTA) with the Mexican Federal Committee for the Protection against Sanitary Risks and initiated CTA submission with the Brazilian Health Regulatory Agency for the Phase 2/3 clinical study evaluating opaganib (Yeliva, ABC294640)1 in patients hospitalized with severe SARS-CoV-2 infection (the cause of COVID-19) and pneumonia.

RedHill’s CTA applications for this study had already been approved in the UK and Russia and a similar application is under review in Italy, with plans to expand the study to additional countries.

The multi-center, randomized, double-blind, parallel-arm, placebo-controlled Phase 2/3 study was set to enroll up to 270 patients with severe COVID-19 pneumonia requiring hospitalization and treatment with supplemental oxygen. Subjects will be randomized at a 1:1 ratio to receive either opaganib or placebo, along with standard-of-care therapy. The primary endpoint of the study was to evaluate the proportion of patients requiring intubation and mechanical ventilation by Day 14. An unblinded futility interim analysis will be conducted by an independent data safety monitoring board (DSMB), when approximately 100 subjects have been evaluated for the primary endpoint.

Enrollment was also ongoing for a randomized, double-blind, placebo-controlled Phase 2 clinical study with opaganib in the U.S. This study was set to enroll up to 40 patients with severe COVID-19 pneumonia requiring hospitalization and supplemental oxygen, with enrollment expected to be completed in August. This clinical trial is not powered for statistical significance. 

Opaganib, a new chemical entity, is a proprietary, first-in-class, orally-administered, sphingosine kinase-2 (SK2) selective inhibitor with anticancer, anti-inflammatory and anti-viral activities, targeting multiple oncology, viral, inflammatory and gastrointestinal indications. By inhibiting SK2, opaganib impacts multiple cellular pathways which are associated with cancer growth, viral replication and pathological inflammation.

Opaganib was originally developed by U.S.-based Apogee Biotechnology Corp. and completed multiple successful pre-clinical studies in oncology, inflammation, GI and radioprotection models, as well as a Phase 1 clinical study in cancer patients with advanced solid tumors.

Opaganib received Orphan Drug designation from the U.S. FDA for the treatment of cholangiocarcinoma and is being evaluated in a Phase 2a study in advanced cholangiocarcinoma and in a Phase 2 study in prostate cancer. Opaganib is also being evaluated for the treatment of coronavirus (COVID-19).