Phase 2/3 COVID-19 study evaluates safety, potential identification of preliminary efficacy signals

Top-line data from the 270-patient global Phase 2/3 COVID-19 study expected Q1/2021. Top-line data from the 40-patient U.S. Phase 2 study of opaganib in severe COVID-19 expected in the coming days; this non-powered study was designed to evaluate the safety and potential identification of preliminary efficacy signals in support of the global Phase 2/3 study of opaganib.

Opaganib potentially minimizes the likelihood of resistance due to viral mutations by targeting a human cell component involved in viral replication

RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, today announced that the global Phase 2/3 study with orally-administered opaganib (Yeliva®, ABC294640) in patients hospitalized with severe COVID-19 pneumonia has received a second unanimous recommendation to continue, following a second independent Data Safety Monitoring Board (DSMB) safety review.

The DSMB's recommendation is based on an analysis of unblinded safety data from the first 155 patients treated for 14 days.

This second unanimous independent DSMB recommendation to continue the global Phase 2/3 study of opaganib in patients with severe COVID-19 confirms the safety of opaganib and means we can continue to focus on completing enrollment for this study as rapidly as possible, with a view to having top-line data in the first quarter of 2021. If successful, we expect to make subsequent global emergency use applications the same quarter."

Mark L. Levitt, MD, PhD, Medical Director, RedHill Biopharma Ltd

Enrollment in the 270-patient global Phase 2/3 study of orally-administered opaganib in hospitalized patients with severe COVID-19 pneumonia (NCT04467840) is more than 60% complete and is on track to deliver top-line data in the first quarter of 2021.

This study is focused on and powered for efficacy evaluation. A pre-scheduled, unblinded futility interim analysis will be conducted by the DSMB in the coming weeks, evaluating data from the first 135 subjects that have reached the primary endpoint.

The parallel U.S. Phase 2 study with opaganib (NCT04414618) has completed enrollment of all 40 subjects, with top-line data expected by year's end. This study is not powered for efficacy and is focused on safety evaluation and potential identification of preliminary efficacy signals.

Opaganib is a novel, orally-administered, sphingosine kinase-2 (SK2) selective inhibitor with a preclinically demonstrated triple-action mechanism that inhibits viral replication, reduces the hyper-immune inflammatory response, and diminishes ARDS-related thrombosis (blood clots) - a dangerous complication of COVID-19 disease - in effect acting on the cause and effect of COVID-19 disease.

Opaganib's target is a human host cell component involved in viral replication, potentially minimizing the likelihood of resistance due to viral mutations.