Country for PR: United Kingdom
Contributor: PR Newswire Europe
Thursday, November 19 2020 - 23:59
RedHill Announces Unanimous DSMB Recommendation to Continue Phase 2/3 COVID-19 Study with Opaganib
TEL AVIV, Israel and RALEIGH, N.C., Nov. 23, 2020, /PRNewswire-AsiaNet/--

Pre-scheduled independent Data and Safety Monitoring Board (DSMB) unanimously 
recommends continuation of the global Phase 2/3 study of orally administered 
opaganib in severe COVID-19 pneumonia 

Enrollment in the 270-patient global Phase 2/3 COVID-19 study with opaganib is 
more than 50% complete

Enrollment completed in the parallel U.S. Phase 2 study evaluating opaganib's 
safety and initial efficacy signal in 40 hospitalized patients with severe 
COVID-19 pneumonia - data expected in the coming weeks

Emergency use authorization applications planned as early as Q1/2021

Opaganib's mechanism of action potentially minimizes likelihood of resistance 
due to viral mutations

RedHill Biopharma Ltd. [ ] (Nasdaq: RDHL) 
("RedHill" or the "Company"), a specialty biopharmaceutical company, today 
announced that the global Phase 2/3 study with opaganib (Yeliva(R), 
ABC294640)[1] in patients hospitalized with severe COVID-19 pneumonia has 
received a unanimous recommendation to continue, following a pre-scheduled 
safety review by an independent Data and Safety Monitoring Board (DSMB). The 
DSMB's recommendation is based on an unblinded analysis of safety data from the 
first 70 patients treated for 14 days. 
"With each review of unblinded safety data, by independent reviewers, as part 
of our development program, our confidence in the safety profile of opaganib 
increases further," said Mark L. Levitt, M.D., Ph.D., Medical Director at 
RedHill. "We are fast compiling a robust and extensive safety data set with 
opaganib, giving us good reason to look forward to the rapid conclusion of this 
study which, if positive, is expected to provide the necessary efficacy data to 
support the next step of emergency use applications in the first quarter of 

Enrollment in the 270-patient global Phase 2/3 study with opaganib in patients 
with severe COVID-19 pneumonia (NCT04467840) 
]  is more than 50% complete. The study, approved in six countries and rapidly 
enrolling across 22 study sites, 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 prescheduled, 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 topline data expected in 
the coming weeks. This study is not powered for efficacy and is focused on 
safety evaluation and identification of efficacy signals. 

Opaganib is a novel, orally administered, sphingosine kinase-2 (SK2) selective 
inhibitor with demonstrated dual anti-inflammatory and antiviral activity that 
acts on the cause and effect of COVID-19 disease, targeting a host cell 
component involved in viral replication, potentially minimizing likelihood of 
resistance due to viral mutations.

About Opaganib (ABC294640, Yeliva(R))

Opaganib, a new chemical entity, is a proprietary, first-in-class, orally 
administered, sphingosine kinase-2 (SK2) selective inhibitor with demonstrated 
dual anti-inflammatory and antiviral activity that targets a host cell 
component, potentially minimizing the likelihood for resistance due to viral 
mutations. Opaganib has also shown anticancer activity and has the potential to 
target multiple oncology, viral, inflammatory and gastrointestinal indications. 

Opaganib is also being evaluated in a global Phase 2/3 study and a U.S. Phase 2 
study for the treatment of severe COVID-19. Opaganib 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.

Preclinical data have demonstrated both anti-inflammatory and antiviral 
activities of opaganib, with the potential to reduce inflammatory lung 
disorders, such as pneumonia, and mitigate pulmonary fibrotic damage. 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[2] 
have demonstrated that opaganib 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. 

Opaganib was originally developed by U.S.-based Apogee Biotechnology Corp. and 
completed multiple successful preclinical studies in oncology, inflammation, 
GI, and radioprotection models, as well as a Phase 1 clinical study in cancer 
patients with advanced solid tumors and an additional Phase 1 study in multiple 

Under a compassionate use program, patients with severe COVID-19 (as classified 
by the WHO ordinal scale) were treated with opaganib in a leading hospital in 
Israel. Data from the treatment of these first patients with severe COVID-19 
with opaganib have been published[3]. Analysis of treatment outcomes suggested 
substantial benefit to patients treated with opaganib under compassionate use 
in both clinical outcomes and inflammatory markers as compared to a 
retrospective matched case-control group from the same hospital.

All patients in the opaganib-treated group were discharged from hospital on 
room air without requiring intubation and mechanical ventilation, whereas 33% 
of the matched case-control group required intubation and mechanical 
ventilation. Median time to weaning from high-flow nasal cannula was reduced to 
10 days in the opaganib-treated group, as compared to 15 days in the matched 
case-control group.

The development of opaganib has been supported by grants and contracts from 
U.S. federal and state government agencies awarded to Apogee Biotechnology 
Corp., including from the NCI, BARDA, the U.S. Department of Defense and the 
FDA Office of Orphan Products Development.

The ongoing studies with opaganib are registered on, a 
web-based service by the U.S. National Institute of Health, which provides 
public access to information on publicly and privately supported clinical 
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(R) for 
opioid-induced constipation in adults with non-cancer pain[4], Talicia(R) for 
the treatment of Helicobacter pylori (H. pylori) infection in adults[5], and 
Aemcolo(R) for the treatment of travelers' diarrhea in adults[6]. RedHill's key 
clinical late-stage investigational development programs include: (i) RHB-204, 
with a planned Phase 3 study for pulmonary nontuberculous mycobacteria (NTM) 
infections; (ii) opaganib (Yeliva(R)), a first-in-class SK2 selective inhibitor 
targeting multiple indications with a Phase 2/3 program for COVID-19 and Phase 
2 studies for prostate cancer and cholangiocarcinoma ongoing; (iii) RHB-104, 
with positive results from a first Phase 3 study for Crohn's disease; (iv) 
RHB-102 (Bekinda(R)), with positive results from a Phase 3 study for acute 
gastroenteritis and gastritis and positive results from a Phase 2 study for 
IBS-D; (v) RHB-107 (upamostat), a Phase 2-stage first-in-class, serine protease 
inhibitor, targeting cancer and inflammatory gastrointestinal diseases and is 
also being evaluated for COVID-19 and (vi) RHB-106, an encapsulated bowel 
preparation. More information about the Company is available at 

NOTE: This press release, provided for convenience purposes, is a translated 
version of the official press release published by the Company in the English 
language. For full press release in English language, including the 
forward-looking statements disclaimer, please visit:
Company contact:
Adi Frish
Chief Corporate & Business Development Officer
RedHill Biopharma

Media contact (U.S.):
Bryan Gibbs
Vice President
Finn Partners

[1] Opaganib is an investigational new drug, not available for commercial 
[2] Xia C. et al. Transient inhibition of sphingosine kinases confers 
protection to influenza A virus infected mice. Antiviral Res. 2018 Oct; 
158:171-177. Ebenezer DL et al. Pseudomonas aeruginosa stimulates nuclear 
sphingosine-1-phosphate generation and epigenetic regulation of lung 
inflammatory injury. Thorax. 2019 Jun;74(6):579-591.
[3] Kurd R, Ben-Chetrit E, Karameh H, Bar-Meir M, Compassionate Use of Opaganib 
For Patients with Severe COVID-19. medRxiv 2020.06.20.20099010; doi:
[4] Full prescribing information for Movantik(R)(naloxegol) is available at:   
[5] Full prescribing information for Talicia(R) (omeprazole magnesium, 
amoxicillin and rifabutin) is available at:       
[6] Full prescribing information for Aemcolo(R) (rifamycin) is available at:

Logo -

SOURCE: RedHill Biopharma Ltd.