RedHill Biopharma Ltd., a specialty biopharmaceutical company focused on gastrointestinal diseases, today announced that it has received authorization from the Italian National Institute for Infectious Diseases and Central Italian Ethics Committee (EC) for an expanded access program (EAP) allowing immediate compassionate use of its investigational drug, opaganib (Yeliva®, ABC294640), in Italy for patients with confirmed coronavirus (COVID-19) infection with life-threatening clinical manifestations.

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Approximately 160 patients are planned to be treated in three major hospitals in northern Italy, one of the current major epicenters of the pandemic.

“The approved opaganib expanded access program allows physicians in the three major hospitals in Italy to treat patients at high risk of developing pneumonia and those with pneumonia, including acute respiratory distress syndrome, secondary to SARS-CoV-2 infection,” said Mark L. Levitt, MD, Ph.D., medical director at RedHill.“RedHill is working diligently to evaluate the potential of opaganib as a treatment for COVID-19 to help patients worldwide in urgent need of a treatment option. I would like to thank our partners at Cosmo Pharmaceuticals for their immense assistance supporting the process in Italy.”

A total of 131 subjects have been dosed with opaganib to date in ongoing and completed Phase 1 and Phase 2 clinical studies in oncology indications in the U.S., in pharmacokinetic studies in healthy volunteers in the U.S., and under the existing FDA-approved expanded access requests from physicians for individual oncology patients, establishing safety and tolerability in humans both in the U.S. and ex-U.S.

Opaganib, a new chemical entity, is a proprietary, first-in-class, orally-administered, sphingosine kinase-2 (SK2) selective inhibitor with anticancer, anti-viral and anti-inflammatory activities, targeting multiple oncology, inflammatory and gastrointestinal indications. Pre-clinical data have demonstrated both anti-viral and anti-inflammatory activities of opaganib, with the potential to reduce lung inflammatory disorders, such as pneumonia, and mitigate pulmonary fibrotic damage. Several prior pre-clinical studies support the potential role of SK2 in the replication-transcription complex of positive-strand single-stranded RNA viruses, similar to coronavirus, and its inhibition may potentially inhibit viral replication. Pre-clinical in vivo studies have demonstrated that opaganib decreased fatality rates from influenza-virus infection and ameliorated Pseudomonas aeruginosa-induced lung injury.

RedHill and its strategic partner, Cosmo Pharmaceuticals, have agreed that Cosmo will become the exclusive or main commercial supplier in the future, subject to opaganib demonstrating positive clinical results, negotiation of final terms and the necessary regulatory approvals. Accordingly, the parties have initiated the manufacturing tech transfer process.

Discussions are ongoing in the U.S. and other countries.

Opaganib has undergone a Phase 1 clinical study in patients with advanced solid tumors and is currently being investigated, individually and in combination with hydroxychloroquine, in a Phase 1/2a study in advanced cholangiocarcinoma (bile duct cancer), and in a Phase 2 study in prostate cancer.

The development of opaganib has been supported by grants and contracts from U.S. federal and state government agencies awarded to U.S.-based Apogee Biotechnology Corp., including the U.S. National Cancer Institute (NCI), the U.S. Department of Health and Human Services’ Biomedical Advanced Research and Development Authority (BARDA), the U.S. Department of Defense and the FDA Office of Orphan Products Development.