Opaganib (ABC294640), a proprietary investigational drug, is a first-in-class, orally administered sphingosine kinase-2 (SPHK2)
selective inhibitor with anticancer, anti-inflammatory and antiviral activity, targeting multiple potential diseases, including gastrointestinal acute radiation syndrome (GI-ARS), COVID-19 and cholangiocarcinoma (bile duct cancer).
Opaganib's suggested mechanism of action, which was published in the journal Drug Design,
Development and Therapy, is host-directed and potentially broad-acting and is expected to maintain effect against
emerging viral variants.
Opaganib is thought to work through the inhibition of multiple pathways, the induction of autophagy and
apoptosis, and disruption of viral replication, through simultaneous inhibition of three
sphingolipid-metabolizing enzymes in human cells (SPHK2, DES1 and GCS).
RedHill announced that
opaganib was selected by the U.S. Government's Radiation and Nuclear Countermeasures Program (RNCP), led
by the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health,
for the nuclear medical countermeasures product development pipeline as a potential treatment for
ARS.
Opaganib has completed a 463-patient global Phase 2/3 study in hospitalized patients with severe COVID-19 pneumonia (NCT04467840). Despite not meeting the study’s primary endpoint, several efficacy trends in key patient sub-groups were achieved.
RedHill announced data from the prespecified analyses of all Phase 2/3 study patients, which showed improvements compared to the control arm with respect to median time to viral RNA clearance. In addition, RedHill announced positive data, which was also published in medRxiv, from a post-hoc analysis in a subset of moderately severe COVID-19 patients evaluating clinical parameters including mortality, time to discharge and time to recovery. Overall adverse events were balanced between the opaganib and placebo groups.
Opaganib
has undergone a U.S. Phase 2a clinical study in patients with advanced, unresectable cholangiocarcinoma
and was
granted FDA Orphan Drug designation for the treatment of cholangiocarcinoma.
An investigator-sponsored Phase 2 study with opaganib in prostate cancer has completed enrollment and patient
follow up is ongoing at MUSC Hollings Cancer Center and Emory University (NCT04207255).
Opaganib
has completed a Phase 1 clinical study in cancer patients with advanced solid tumors and an
investigator-sponsored Phase 1b clinical study in the treatment of multiple myeloma undertaken at Duke
University Medical Center.
The
clinical studies with opaganib are registered on www.clinicaltrials.gov,
a web-based service by the U.S. National Institute of Health, which provides public access to
information on publicly and privately supported clinical studies.
Virtual
Patent Marking
*
Yeliva® is a proposed tradename for the drug product containing opaganib, which is
subject to review by the FDA at the time of NDA filing