Country for PR: United Kingdom
Contributor: PR Newswire Europe
Wednesday, February 24 2021 - 00:33
RedHill Biopharma Announces Planned Expansion of Opaganib Global Phase 2/3 COVID-19 Study to the U.S.
TEL AVIV, Israel and RALEIGH, NC, Feb. 23, 2021 /PRNewswire-AsiaNet / --

Following review of data from the U.S. Phase 2 study by the FDA, RedHill plans 
to expand the global Phase 2/3 study of orally-administered opaganib for severe 
COVID-19 to the U.S. 

The study has enrolled over 50% of the targeted 464 patients globally; U.S. 
study activities expected to expand the study to a total of 8 countries and 
approximately 40 recruiting sites; Top-line results expected in the second 

Opaganib demonstrated dual anti-inflammatory and antiviral activity and targets 
a human cell component involved in viral replication and is therefore expected 
to be effective against emerging viral variants with mutations in the spike 

RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty 
biopharmaceutical company, today announced its plans to expand the Company's 
global Phase 2/3 study of opaganib[1] in patients with severe COVID-19 to the 
U.S., following U.S. Food and Drug Administration (FDA) review of the data from 
the U.S. Phase 2 study of opaganib and receipt of its recommendations.   

Expansion of the global Phase 2/3 study to the U.S. will entail adjustments to 
the protocol based on the FDA's recommendations and ongoing discussions. The 
expansion of the study to the U.S. will help further speed-up enrollment, 
expanding the study to a total of 8 countries and approximately 40 recruiting 
sites, with additional sites and countries being added. The 464-patient study 
is over 50% enrolled and is expected to deliver top-line data in the second 
quarter of 2021. 

The global Phase 2/3 study recently underwent a positive DSMB futility review, 
which is suggestive that the study has the potential for a positive outcome. 
RedHill recently announced positive top-line safety and efficacy data from the 
non-powered U.S. Phase 2 study with opaganib in patients with COVID-19 
pneumonia, in which opaganib demonstrated greater improvement in reducing 
oxygen requirement by end of treatment on Day 14 across key primary and 
secondary efficacy outcomes. The Phase 2 data also showed no material safety 
differences between the opaganib and placebo treatment arms - further adding to 
the growing safety database for opaganib. 

Opaganib is a novel, orally-administered sphingosine kinase-2 (SK2) inhibitor 
with demonstrated antiviral, anti-inflammatory, and anti-thrombotic activity, 
that targets a human cell component involved in viral replication and is 
therefore expected to be effective against emerging viral variants with 
mutations in the spike protein. 

About Opaganib (Yeliva®, ABC294640)

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 indications. 

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 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 U.S. Phase 2 study.

Preclinical data have demonstrated anti-inflammatory, antiviral and 
anti-thrombotic activities of opaganib, with the potential to ameliorate 
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. Opaganib also demonstrated reduced 
blood clot length, weight and total thrombus score in a preclinical model of 
Acquired Respiratory Distress Syndrome. 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 

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 

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® for opioid-induced constipation in 
adults[3], Talicia® for the treatment of Helicobacter pylori (H. pylori) 
infection in adults[4], and Aemcolo® for the treatment of travelers' diarrhea 
in adults[5]. 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. 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 a full analysis of the data from the U.S. Phase 2 
trial evaluating opaganib, the timing of potential emergency use applications 
for opaganib and the timing of reporting of top-line data 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 
COVID-19 Phase 2/3 study evaluating opaganib will not be successful; the risk 
of a delay in receiving top-line data from the Phase 2/3 study and in receiving 
data to support emergency use applications or in making such emergency use 
applications, if at all; the risk that data received from the Phase 2/3 study, 
even if successful, will not be sufficient to support filing or approval of 
emergency use applications or other marketing applications in certain or all 
geographical regions; the risk that the full analysis of data from the U.S. 
Phase 2 clinical study evaluating opaganib will be delayed or will differ from 
the preliminary data; 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 
will be delayed; the risk that the Phase 2/3 study will not provide a clear 
picture of opaganib's potential in treating severe COVID-19; 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 severe 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, 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®; (v) the Company's ability to successfully 
commercialize and promote Movantik®, Talicia® and Aemcolo®; (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] 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] Full prescribing information for Movantik® (naloxegol) is available at:   

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

[5] Full prescribing information for Aemcolo® (rifamycin) is available at:


Company contact:
Adi Frish
Chief Corporate & Business Development Officer
RedHill Biopharma

Media contacts:
U.S.: Bryan Gibbs, Finn Partners
+1 212 529 2236
UK: Amber Fennell, Consilium
+44 (0) 7739 658 783

SOURCE: RedHill Biopharma Ltd.