RedHill Biopharma Announces Approval of Compassionate Use of Opaganib for COVID-19 in Italy

Authorization of compassionate use granted by the Italian authorities, aided by RedHill’s strategic partner, Cosmo Pharmaceuticals N.V. (SIX: COPN)

Approximately 160 patients with life-threatening clinical manifestations planned to be treated in three major hospitals in northern Italy

A compassionate use program is ongoing in Israel where opaganib has been administered to the first patient at a leading hospital

Discussions ongoing in the U.S. and other countries

RedHill preparing for potential ramp-up of manufacturing of opaganib

TEL-AVIV, Israel and RALEIGH, N.C., April 06, 2020 (GLOBE NEWSWIRE) -- RedHill Biopharma Ltd. (Nasdaq: RDHL) (“RedHill” or the “Company”), 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.

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 studies¹ 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.

To find out more about RedHill Biopharma's Expanded Access policy, please look here for additional information: www.redhillbio.com/expandedaccess.

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.

About Opaganib (ABC294640, Yeliva^®)
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. By inhibiting SK2, opaganib blocks the synthesis of sphingosine 1-phosphate (S1P), a lipid-signaling molecule that promotes cancer growth and pathological inflammation. By inhibiting SK2, opaganib potentially blocks viral replication complex 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. The development of opaganib was supported by grants and contracts from U.S. federal and state government agencies awarded to Apogee Biotechnology Corp., including NCI, BARDA, the U.S. Department of Defense and the FDA Office of Orphan Products Development.

About RedHill Biopharma  
RedHill Biopharma Ltd. (Nasdaq: RDHL) is a specialty biopharmaceutical company focused on gastrointestinal diseases. RedHill promotes the gastrointestinal drugs Movantik^® for opioid-induced constipation in adults², Talicia^® for the treatment of Helicobacter pylori (H. pylori) infection in adults³ and Aemcolo^® for the treatment of travelers’ diarrhea in adults⁴. RedHill’s key clinical late-stage development programs include: (i) RHB-104, with positive results from a first Phase 3 study for Crohn's disease; (ii) RHB-204, with a planned pivotal Phase 3 study for pulmonary nontuberculous mycobacteria (NTM) infections; (iii) RHB-102 (Bekinda^®), with positive results from a Phase 3 study for acute gastroenteritis and gastritis and positive results from a Phase 2 study for IBS-D; (iv) Opaganib (Yeliva^®), a first-in-class SK2 selective inhibitor, targeting multiple oncology, inflammatory and gastrointestinal indications, with an ongoing Phase 1/2a study for cholangiocarcinoma; (v) RHB-106, an encapsulated bowel preparation, and (vi) RHB-107, a Phase 2-stage first-in-class, serine protease inhibitor, targeting cancer and inflammatory gastrointestinal diseases. More information about the Company is available at www.redhillbio.com.