AsiaNet 88206

TEL AVIV, Israel and RALEIGH, NC, Feb. 23, 2021 /PRNewswire=KYODO JBN/ --

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

quarter

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

protein

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

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

myeloma.

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 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[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 www.redhillbio.com / https://twitter.com/RedHillBio.          

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.

References:

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

distribution.

[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:

www.Movantik.com.  

[4] Full prescribing information for Talicia® (omeprazole magnesium,

amoxicillin and rifabutin) is available at: www.Talicia.com.      

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

www.Aemcolo.com.

Logo: https://mma.prnewswire.com/media/1334141/RedHill_Biopharma_Logo.jpg  

Company contact:

Adi Frish

Chief Corporate & Business Development Officer

RedHill Biopharma

+972-54-6543-112

adi@redhillbio.com

Media contacts:

U.S.: Bryan Gibbs, Finn Partners

+1 212 529 2236

bryan.gibbs@finnpartners.com

UK: Amber Fennell, Consilium

+44 (0) 7739 658 783

fennell@consilium-comms.com

SOURCE: RedHill Biopharma Ltd.