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Friday, November 20 2020 - 09:00
RedHill Biopharma Completes Enrollment for COVID-19 U.S. Phase 2 Study with Opaganib – Data Expected in the Coming Weeks
TEL AVIV, Israel and RALEIGH, NC, Nov. 20, 2020 /PRNewswire-AsiaNet/ --

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

A parallel global Phase 2/3 study with orally administered opaganib for severe 
COVID-19 is approximately 50% enrolled - on track to enroll all 270 patients 
and report topline data in Q1/2021

Potential emergency use applications expected as early as Q1/2021

Opaganib's unique dual anti-inflammatory and antiviral activity acts on the 
cause and effect of COVID-19 disease with host cell targeting, minimizing 
potential for resistance due to viral mutations   

RedHill Biopharma Ltd. (Nasdaq: RDHL) []  
("RedHill" or the "Company"), a specialty biopharmaceutical company, today 
announced that the U.S. Phase 2 study with opaganib (Yeliva(R), ABC294640)[1] 
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 
raw=2&rank=1]  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 

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 of viral replication, potentially minimizing the likelihood of viral 
resistance. Opaganib has also shown anticancer activity and has the potential 
to target multiple oncology, viral, inflammatory, and gastrointestinal 

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

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[3] 
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, COVID-19 patients (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[2]. 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 

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, 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 

This press release contains "forward-looking statements" within the meaning of 
the Private Securities Litigation Reform Act of 1995. Such statements may be 
preceded by the words "intends," "may," "will," "plans," "expects," 
"anticipates," "projects," "predicts," "estimates," "aims," "believes," 
"hopes," "potential" or similar words and includes statements regarding the 
timing of the reporting of data from the U.S. Phase 2 trial evaluating 
opaganib, the timing of potential emergency use applications of opaganib. 
Reporting of topline data, safety analysis and of unblinded futility interim 
analysis for the global Phase 2/3 study with opaganib. Forward-looking 
statements are based on certain assumptions and are subject to various known 
and unknown risks and uncertainties, many of which are beyond the Company's 
control and cannot be predicted or quantified, and consequently, actual results 
may differ materially from those expressed or implied by such forward-looking 
statements. Such risks and uncertainties include, without limitation, the risk 
that the Company's Phase 2/3 study evaluating opaganib will not be successful; 
the risk of a delay in receiving data to support emergency use applications or 
in making such emergency use applications, if at all; the risk that the U.S. 
Phase 2 clinical study evaluating opaganib will not be successful and the risk 
that the reporting of data from this clinical study will be delayed if at all; 
the risk that the Company will not initiate the Phase 2/3 study for opaganib in 
certain geographies, will not expand this study to additional countries and 
that it will not be successful and that enrollment, reporting of topline data, 
safety analysis and/or unblinded futility interim analysis will be delayed; the 
risk that other COVID-19 patients treated with opaganib will not show any 
clinical improvement; the development risks of early-stage discovery efforts 
for a disease that is still little understood, including difficulty in 
assessing the efficacy of opaganib for the treatment of COVID-19, if at all; 
intense competition from other companies developing potential treatments and 
vaccines for COVID-19; the effect of a potential occurrence of patients 
suffering serious adverse events using opaganib under compassionate use 
programs, as well as risks and uncertainties associated with (i) the 
initiation, timing, progress and results of the Company's research, 
manufacturing, preclinical studies, clinical trials, and other therapeutic 
candidate development efforts, and the timing of the commercial launch of its 
commercial products and ones it may acquire or develop in the future; (ii) the 
Company's ability to advance its therapeutic candidates into clinical trials or 
to successfully complete its preclinical studies or clinical trials (iii) the 
extent and number and type of additional studies that the Company may be 
required to conduct and the Company's receipt of regulatory approvals for its 
therapeutic candidates, and the timing of other regulatory filings, approvals 
and feedback; (iv) the manufacturing, clinical development, commercialization, 
and market acceptance of the Company's therapeutic candidates and Talicia(R); 
(v) the Company's ability to successfully commercialize and promote 
Movantik(R), Talicia® and Aemcolo(R); (vi) the Company's ability to establish 
and maintain corporate collaborations; (vii) the Company's ability to acquire 
products approved for marketing in the U.S. that achieve commercial success and 
build and sustain its own marketing and commercialization capabilities; (viii) 
the interpretation of the properties and characteristics of the Company's 
therapeutic candidates and the results obtained with its therapeutic candidates 
in research, preclinical studies or clinical trials; (ix) the implementation of 
the Company's business model, strategic plans for its business and therapeutic 
candidates; (x) the scope of protection the Company is able to establish and 
maintain for intellectual property rights covering its therapeutic candidates 
and commercial products and its ability to operate its business without 
infringing the intellectual property rights of others; (xi) parties from whom 
the Company licenses its intellectual property defaulting in their obligations 
to the Company; (xii) estimates of the Company's expenses, future revenues, 
capital requirements and needs for additional financing; (xiii) the effect of 
patients suffering adverse events using investigative drugs under the Company's 
Expanded Access Program; and (xiv) competition from other companies and 
technologies within the Company's industry. More detailed information about the 
Company and the risk factors that may affect the realization of forward-looking 
statements is set forth in the Company's filings with the Securities and 
Exchange Commission (SEC), including the Company's Annual Report on Form 20-F 
filed with the SEC on March 4, 2020. All forward-looking statements included in 
this press release are made only as of the date of this press release. The 
Company assumes no obligation to update any written or oral forward-looking 
statement, whether as a result of new information, future events or otherwise 
unless required by law.  


[1] Opaganib is an investigational new drug, not available for commercial 

[2] 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:

[3] 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.

[4] Full prescribing information for Movantik(R) (naloxegol) is available at:   

[5] Full prescribing information for Talicia® (omeprazole magnesium, 
amoxicillin and rifabutin) is available at:       

[6] Full prescribing information for Aemcolo(R) (rifamycin) is available at:

Logo - 



Company contact:

Adi Frish
Chief Corporate & Business Development Officer
RedHill Biopharma

Media contact (U.S.):
Bryan Gibbs
Vice President
Finn Partners
+1 212 529 2236

Source: RedHill Biopharma Ltd.