RedHill Biopharma Ltd. has announced the issuance of a new U.S. patent (U.S. Patent No. 12,115,150) covering the identification of a novel biomarker for coronavirus pneumonia, specifically a fraction of inspired oxygen (FiO2) level of ≤60%, which is prognostic for the potential efficacy of opaganib in treating COVID-19. This patent is valid through 2041.
Clinical Data Supporting the Biomarker
The patent is based on post-hoc data from opaganib's Phase 2/3 study. The data indicated that patients with FiO2 levels of ≤60% experienced better outcomes after 14 days of opaganib treatment compared to placebo. Key findings included:
- A statistically significant increase in the number of patients no longer requiring supplemental oxygen by day 14 (76.9% vs. 63.4%; p-value = 0.033).
- A 62.6% reduction in the need for intubation or mechanical ventilation (6.84% vs. 17.91%; p-value = 0.012).
- A clinically meaningful 62% reduction in mortality by day 42 (5.98% vs. 16.7%; p-value = 0.019).
Opaganib: A Host-Directed Therapeutic
Opaganib (ABC294640) is an investigational, host-directed, orally administered small molecule drug. It functions as a selective inhibitor of sphingosine kinase-2 (SPHK2) and has demonstrated anticancer, anti-inflammatory, and antiviral activity. Its mechanism of action involves inhibiting multiple pathways, inducing autophagy and apoptosis, and disrupting viral replication by simultaneously inhibiting three sphingolipid-metabolizing enzymes in human cells (SPHK2, DES1, and GCS).
Development and Potential Applications
Opaganib is under development for various indications, including several cancers, diabetes and obesity-related disorders, gastrointestinal acute radiation syndrome (GI-ARS), Sulfur Mustard exposure, COVID-19, Ebola, and other viruses as part of pandemic preparedness. It has received several orphan-drug designations from the FDA in oncology and other diseases and has undergone studies in advanced cholangiocarcinoma (Phase 2a) and prostate cancer. Opaganib also has a Phase 1 chemoradiotherapy study protocol ready for FDA-IND submission.
Government Collaborations and Preclinical Data
Several U.S. government countermeasures and pandemic preparedness programs have selected opaganib for evaluation for multiple indications, including Acute Radiation Syndrome (ARS) and Ebola virus disease. Preclinical studies have demonstrated opaganib's antiviral activity against SARS-CoV-2, multiple variants, and other viruses like Influenza A and Ebola. In an in vivo Ebola virus study, opaganib significantly increased survival time. It also showed a synergistic effect when combined with remdesivir in an in vitro Ebola virus study.
Quote from RedHill Biopharma
"This exciting new patent is based on compelling published post-hoc data from opaganib's Phase 2/3 study, showing that patients with ≤60% FiO2 levels had better outcomes after 14 days' opaganib treatment," said Guy Goldberg, RedHill's Chief Business Officer. "This is a very interesting addition to the already strong patent portfolio protecting opaganib."
