RedHill Biopharma Ltd., a specialty biopharmaceutical company, today announced that it has completed enrollment and randomized the last patient in the global Phase 2/3 study with opaganib (Yeliva?, ABC294640)[1] in patients hospitalized with severe COVID-19 pneumonia (NCT04467840). A total of 475 patients were randomized, more than the planned 464.

The primary endpoint of the study is the proportion of patients breathing room air without oxygen support by Day 14. The study captures additional important outcome measures in the follow up period of up to 6 weeks, such as the time to hospital discharge, improvement according to the World Health Organization Ordinal Scale for Clinical Improvement and incidence of intubation and mortality.

An evaluation of the blinded blended intubation and mortality rates to date is encouraging as compared to reported rates of mortality from large platform studies such as RECOVERY, and other studies in similar patient populations[2]. Furthermore, four independent DSMB recommendations to continue the study were already provided following unblinded safety and futility reviews.

"Enrollment completion of this 475-patient global study of oral opaganib for COVID-19 is a truly exciting milestone in the urgent search for an effective pill to treat COVID-19, positioning opaganib as a leading novel, dual-action, investigational COVID-19 oral treatment," said Mark L. Levitt, MD, Ph.D., Medical Director at RedHill. "Acting on the cause and effect of COVID-19 through a dual antiviral and anti-inflammatory effect, opaganib is host-targeted and is therefore expected to be effective against emerging viral variants. With waves of COVID-19 continuing to wash over many countries, coupled with the specter of new variants, it is more critical than ever that the world has access to an oral pill to treat COVID-19."

In addition to ongoing discussions with the FDA and other regulators, the Company has also met with the EMA to talk about a European pathway. As with all discussions with regulatory bodies, next steps will be guided by study results. Discussions are also ongoing with potential partners who are interested in the rights to opaganib in various countries. 

About Opaganib (Yeliva?, ABC294640)

Opaganib, a new chemical entity, is a proprietary, first-in-class, orally-administered, sphingosine kinase-2 (SK2) selective inhibitor, with dual anti-inflammatory and antiviral activity, that is host-targeted and is therefore expected to be effective against emerging viral variants. Opaganib has also shown anticancer activity and has the potential to target multiple oncology, viral, inflammatory, and gastrointestinal indications.

Opaganib is being evaluated as a treatment for COVID-19 pneumonia in a global Phase 2/3 study and has demonstrated positive safety and efficacy signals in preliminary top-line data from a 40-patient U.S. Phase 2 study.

Opaganib has also received Orphan Drug designation from the U.S. FDA for the treatment of cholangiocarcinoma and is being evaluated in a Phase 2a study in advanced cholangiocarcinoma and in a Phase 2 study in prostate cancer.

Opaganib demonstrated potent antiviral activity against SARS-CoV-2, the virus that causes COVID-19, completely inhibiting viral replication in an in vitro model of human lung bronchial tissue. Additionally, preclinical in vivo studies have demonstrated opaganib's potential to ameliorate inflammatory lung disorders, such as pneumonia, and has shown decreased fatality rates from influenza virus infection and ameliorated Pseudomonas aeruginosa-induced lung injury by reducing the levels of IL-6 and TNF-alpha in bronchoalveolar lavage fluids[3].

The ongoing 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.   

About RedHill Biopharma    

RedHill Biopharma Ltd. (Nasdaq: RDHL) is a specialty biopharmaceutical company primarily focused on gastrointestinal and infectious diseases. RedHill promotes the gastrointestinal drugs, Movantik? for opioid-induced constipation in adults[4], Talicia? for the treatment of Helicobacter pylori (H. pylori) infection in adults[5], and Aemcolo? for the treatment of travelers' diarrhea in adults[6]. RedHill's key clinical late-stage development programs include: (i) RHB-204, with an ongoing Phase 3 study for pulmonary nontuberculous mycobacteria (NTM) disease; (ii) opaganib (Yeliva?, ABC294640), a first-in-class SK2 selective inhibitor targeting multiple indications with positive Phase 2 COVID-19 data and an ongoing Phase 2/3 program for COVID-19 and Phase 2 studies for prostate cancer and cholangiocarcinoma ongoing; (iii) RHB-107 (upamostat), a serine protease inhibitor in a U.S. Phase 2/3 study as treatment for symptomatic COVID-19, and targeting multiple other cancer and inflammatory gastrointestinal diseases; (iv) RHB-104, with positive results from a first Phase 3 study for Crohn's disease; (v) RHB-102 (Bekinda?), with positive results from a Phase 3 study for acute gastroenteritis and gastritis and positive results from a Phase 2 study for IBS-D; and (vi) RHB-106, an encapsulated bowel preparation.