Country for PR: United Kingdom
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Friday, January 01 2021 - 09:39
RedHill Biopharma Announces Positive Top-Line Safety and Efficacy Data from Phase 2 COVID-19 Study of Opaganib
TEL AVIV, Israel and RALEIGH, N.C., Dec. 31, 2020 /PRNewswire-AsiaNet/ --

- Preliminary data from the non-powered U.S. Phase 2 study of 40 hospitalized 
patients shows that orally-administered opaganib was safe, with no material 
safety differences between opaganib and control arms

- Consistent trends demonstrate greater improvement in reducing oxygen 
requirement by end of treatment at Day 14 in the opaganib-treated arm across 
key primary and secondary efficacy outcomes, correlating with clinical 
improvement as defined by the World Health Organization (WHO) ordinal scale

- The opaganib-treated arm demonstrated a greater improvement in reaching room 
air within 14 days (52.6% vs. 22.2%); greater improvement in reduction to 50% 
supplemental oxygen by Day 14 (89.5% vs. 66.7%); a higher proportion of 
patients discharged by Day 14 (73.7% vs. 55.6%) and a greater reduction in the 
median total oxygen requirement (AUC) over 14 days (68.0% vs. 46.7%)

- Top-line data from the global Phase 2/3 COVID-19 study in 270 hospitalized 
patients expected Q1/2021 and an interim DSMB futility analysis is expected in 
the coming weeks

- Opaganib targets a human cell component involved in viral replication, 
potentially minimizing the likelihood for resistance due to viral mutations

RedHill Biopharma Ltd. [] (Nasdaq: RDHL) 
("RedHill" or the "Company"), a specialty biopharmaceutical company, today 
announced that preliminary top-line data from its U.S. Phase 2 study with 
orally-administered opaganib (Yeliva(R), ABC294640)[1] in patients hospitalized 
with COVID-19 pneumonia demonstrated positive safety and efficacy signals.

The randomized, double-blind, placebo-controlled U.S. Phase 2 proof-of-concept 
study with opaganib (NCT04414618 
) enrolled 40 patients requiring oxygen support. The study was not powered for 
statistical significance and aimed to evaluate safety and identify preliminary 
signs of activity. Patients in the study were randomized at a 1:1 ratio to 
receive either opaganib or placebo on top of standard-of-care (SoC) and were 
followed up for up to 42 days post treatment initiation.

- Top-line results from the study found opaganib to be safe, with no material 
safety differences between the opaganib and placebo treatment arms. Overall, 
fewer patients suffered from serious adverse events (SAEs) in the opaganib 
treatment arm than in the placebo arm. In this small sample size, there were 
few events of intubation or fatality and these were balanced between the two 

- The opaganib-treated arm demonstrated a consistent trend of greater 
improvement in reducing oxygen requirement by end of treatment on Day 14 across 
key primary and secondary efficacy outcomes, correlating with clinical 
improvement as defined by the World Health Organization (WHO) ordinal scale: 

- A greater improvement in the proportion of patients reaching room air and no 
longer requiring oxygen support by Day 14 vs. the control arm (52.6% vs. 22.2%).
- A greater improvement in the proportion of patients with 50% reduction in 
supplemental oxygen by day 14 vs. the control arm (89.5% vs. 66.7%).
- A higher proportion of patients discharged by Day 14 vs. the control arm 
(73.7% vs. 55.6%).
- A greater reduction from baseline of the median total oxygen requirement 
(AUC) over 14 days vs. the control arm (68.0% vs. 46.7%).

Full analysis of the data, including viral and inflammatory biomarker analyses, 
baseline risk factors and SoC background therapy stratifications, is expected 
in the coming weeks. The Company will provide the data for peer review when 

"We are pleased with these encouraging top-line results from our exploratory 
Phase 2 study which confirm opaganib's safety and demonstrate promising signals 
of activity when treating patients with COVID-19 and who require oxygen 
support. These preliminary results support our ongoing global Phase 2/3 study 
in severe COVID-19 pneumonia, which is expected to read out in Q1/2021. We 
continue to work diligently to compile a robust data set to support potential 
filing of global emergency use applications," said Mark L. Levitt, MD, Ph.D., 
Medical Director at RedHill. 

Gilead Raday, RedHill's Chief Operating Officer, added: "Opaganib has a unique 
dual mode of action that is both anti-inflammatory and antiviral – acting on 
both the cause and the effects of COVID-19. Opaganib targets sphingosine 
kinase-2, a human cell component involved in viral replication and not the 
virus itself. The mounting evidence of new SARS-CoV-2 mutations emerging 
globally underscores the importance of this unique mechanism, which potentially 
minimizes the risk of viral resistance to therapy. The trends of patient 
improvement shown by the preliminary top-line data support the ongoing Phase 
2/3 study with opaganib, which will provide a more in-depth understanding of 
opaganib's activity."

The efficacy of opaganib in severe COVID-19 pneumonia is being further explored 
in an ongoing global Phase 2/3 study and is expected to report top-line data in 
the first quarter of 2021. This study (NCT04467840 
) is being conducted across approximately 30 clinical sites in several 
countries and is on track to enroll up to 270 patients. The study has undergone 
two unblinded reviews of safety data by an independent Data and Safety 
Monitoring Board (DSMB), with unanimous recommendations to continue the study. 
An interim DSMB futility analysis will be conducted in the coming weeks, 
evaluating data from the first 135 subjects that have reached the primary 

The top-line results from the U.S. Phase 2 study of opaganib in patients 
hospitalized with COVID-19 pneumonia are preliminary and were provided to the 
Company by an independent third-party following an initial independent analysis 
and remain subject to additional review and analysis. Such review and analysis 
may result in findings inconsistent with the results disclosed in this release 
and may not be replicated in future studies.

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 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 both anti-inflammatory and antiviral 
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. 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 

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[3], Talicia(R) for 
the treatment of Helicobacter pylori (H. pylori) infection in adults[4], and 
Aemcolo(R) for the treatment of travelers' diarrhea in adults[5]. RedHill's key 
clinical late-stage investigational development programs include: (i) RHB-204, 
with an ongoing 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 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, 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 
top-line data from the Phase 2/2 study and in receiving data to support 
emergency use applications or in making such emergency use applications, if at 
all; 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, reporting of 
top-line 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®; (v) 
the Company's ability to successfully commercialize and promote Movantik(R), 
Talicia(R) 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.

Logo -

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. Full prescribing information for Movantik(R)(naloxegol) is available at:
4. Full prescribing information for Talicia(R)(omeprazole magnesium, 
amoxicillin and rifabutin) is available at:
5. Full prescribing information for Aemcolo(R)(rifamycin) is available at:

Source: RedHill Biopharma Ltd.