RedHill Biopharma Gets BARDA Funding to Develop Opaganib for Ebola

The U.S. government's Biomedical Advanced Research and Development Authority (BARDA) has chosen opaganib for collaborative development and funding as a medical countermeasure (MCM) to treat Ebola virus disease (EBOV). This decision by BARDA supports the positive progress opaganib has made toward potential approval as an MCM for EBOV under the FDA's Animal Rule pathway. Recent studies funded by the U.S. Army demonstrated that opaganib significantly increased survival in an in vivo EBOV model. The research and development contract with BARDA includes initial funding aimed at advancing opaganib to control and contain EBOV outbreaks.

This year marks a decade since the West Africa Ebola epidemic, which resulted in 11,000 deaths. Despite the passage of time, there remains an urgent need for effective therapies, with the World Health Organization (WHO) noting that EBOV is fatal in about half of all cases. Opaganib is a novel drug that has shown broad-acting potential with mutation-resistant antiviral and anti-inflammatory properties, likely affecting vascular health—a primary target for EBOV. It is considered the first host-directed molecule to exhibit activity against EBOV in vivo, presenting a promising alternative strategy for biodefense and global health preparedness. Additional collaborations between the U.S. government and opaganib are ongoing.

The management of disease outbreaks faces significant geopolitical and logistical challenges, highlighting the need for safe, effective, oral, small-molecule therapeutics that can be easily stored, distributed, and administered during an outbreak. The selection of opaganib by BARDA, a center within the Department of Health and Human Services (HHS) Administration for Strategic Preparedness and Response (ASPR), underscores its potential as a treatment for EBOV. Under a cost-sharing contract, BARDA will partially finance the advancement of opaganib to help mitigate infection and contain EBOV outbreaks.

Opaganib has shown positive development progress toward potential FDA approval as an MCM for EBOV, particularly through studies that involve pivotal animal model efficacy. The FDA's Animal Rule allows for the use of these studies to support the approval of new drugs when human trials are not ethical or feasible. Guy Goldberg, Chief Business Officer of RedHill Biopharma, emphasized the deadly nature of EBOV, which kills about half of those infected. Despite the approval of two treatments, Inmazeb™ and Ebanga™, there remains a critical need for additional therapies that are effective and easy to distribute and administer.

Opaganib achieved a statistically significant increase in survival time when administered at 150 mg/kg twice daily in a study conducted by the United States Army Medical Research Institute of Infectious Diseases (USAMRIID). It also demonstrated a synergistic effect when combined with remdesivir, enhancing potency while maintaining cell viability in an in vitro study funded by the U.S. Army.

Currently, opaganib is also in development for various indications, including oncology, viral, inflammatory, and diabetes-related conditions, as well as COVID-19, acute respiratory distress syndrome (ARDS), and radiological and chemical protection or mitigation.

Ebola virus disease, as described by the Centers for Disease Control and Prevention (CDC), is a rare but often fatal illness caused by ebolaviruses. Transmission typically occurs through contact with an infected animal or person. The disease progresses from initial "dry" symptoms like fever and fatigue to "wet" symptoms such as diarrhea and unexplained bleeding. FDA-approved treatments for EBOV include Inmazeb™ and Ebanga™, both of which are monoclonal antibody therapies administered intravenously. There is an urgent need for small-molecule therapies that are effective against multiple strains of ebolavirus and are easier to store, distribute, and administer, particularly in regions with inadequate healthcare infrastructure.

Opaganib, a proprietary investigational drug, is an orally administered sphingosine kinase-2 (SPHK2) selective inhibitor with potential antiviral, anti-inflammatory, and anticancer properties. It targets multiple indications and is being evaluated as a potential treatment for various conditions, including Acute Radiation Syndrome (ARS) and inhalational Sulfur Mustard exposure.

RedHill Biopharma Ltd. is focused on gastrointestinal and infectious diseases, with ongoing clinical programs for multiple indications. The company promotes Talicia® for treating H. pylori infections and is developing other therapies for conditions such as COVID-19, cancer, and gastrointestinal diseases.