RedHill Biopharma Ltd (Nasdaq: RDHL), a specialty biopharmaceutical company, today announced that the U.S. Phase 2 study with opaganib (Yeliva®, ABC294640) in patients hospitalized with severe COVID-19 pneumonia has completed enrollment of the last patient in the study.

The study is not powered for statistical significance. Topline data is expected in the coming weeks.

“Completing enrollment in this U.S. Phase 2 study of orally administered opaganib in severe COVID-19 is a key milestone, indicating that we are just weeks away from important safety data and increased understanding of the potential of opaganib. In parallel, our global Phase 2/3 study is approximately 50% enrolled and is expected to undergo its first pre-planned safety analysis in the coming days and report topline data in the first quarter of 2021. We are compiling a robust data set to support the planned emergency use applications, expected as early as next quarter, subject to study success.” said Mark L. Levitt, MD, Ph.D., Medical Director at RedHill.

Opaganib has a demonstrated unique dual mode of action that is both anti-inflammatory and antiviral – acting on the cause and the effect of COVID-19. Moreover, opaganib acts on a host cell component involved in viral replication and not the virus itself, which could minimize issues of resistance due to emergence of viral mutations. In light of the encouraging data from patients with severe COVID-19 treated with opaganib under compassionate use[2] and the potent anti-SARS-CoV-2 activity that opaganib has demonstrated in vitro, we are excited to see the data from the U.S. Phase 2 study and look to rapidly complete the global Phase 2/3 development program for opaganib toward potential emergency use authorization applications.”

The randomized, double-blind, placebo-controlled Phase 2 study with opaganib enrolled 40 patients in clinical sites across the U.S. The study is not powered for statistical significance and is focused on safety evaluation and identifying a signal of efficacy.

Patients in the study were randomized at a 1:1 ratio to receive either opaganib or placebo on top of standard-of-care. The primary objective of the study is to evaluate the reduction in total oxygen requirement over the course of treatment for up to 14 days.

Secondary endpoints include time to 50% reduction in oxygen requirements, the proportion of patients without fever at Day 14, and proportion with negative nasal swabs at Day 14.

In parallel, the global Phase 2/3 study with opaganib in patients with severe COVID-19 pneumonia is approximately 50% enrolled and is expected to report topline data in the first quarter of 2021. The study is being conducted across 21 clinical sites and is on track to enroll up to 270 patients.

A first unblinded review of safety data from the first 70 patients enrolled in the study by an independent Data and Safety Monitoring Board (DSMB) is expected in the coming days. An 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. This study is focused on, and powered for, efficacy evaluation.

The study has been approved in the UK, Italy, Russia, Mexico, Brazil and Israel, with further expansion ongoing. The clinical studies with opaganib are intended to support potential emergency use applications as early as the first quarter of 2021, subject to positive results. –