The U.S. government's Biomedical Advanced
Research and Development Authority (BARDA) selected opaganib for
joint development & funding as a medical countermeasure (MCM)
to treat Ebola virus disease (EBOV)
--
The
funding advances opaganib's positive development progress to date
on the expected FDA Animal Rule pathway toward potential approval
as an MCM for EBOV
--
Recent U.S. Army-funded
studies showed that opaganib delivered a statistically significant
increase in survival in an in vivo EBOV model. The BARDA research
and development contract provides initial funding for the
collaboration, in pursuit of advancing opaganib to mitigate
infection and contain EBOV outbreaks
--
This
year marks the 10th anniversary of the West Africa Ebola
epidemic in which 11,000 people died, and there is still an urgent
need for effective and useable therapies, with EBOV proving fatal
in around half of all cases according to the World Health
Organization (WHO)1
--
Opaganib, a novel potentially broad-acting drug,
has shown mutation-resistant antiviral and anti-inflammatory
activity, likely to directly impact vascular health - one of the
main targets for EBOV dysfunction. It is believed to be the first
host-directed molecule to show activity in EBOV in vivo and
represents an alternative host-directed therapeutic strategy for
biodefense and global health preparedness. Additional U.S.
government collaborations with opaganib are
ongoing
--
Significant geopolitical and
logistical challenges exist in managing outbreaks of disease and
there is an urgent need for safe and effective, oral, small
molecule therapeutics that can be stored and easily distributed and
administered in an outbreak
TEL-AVIV, Israel and RALEIGH, N.C., Oct. 14,
2024 /PRNewswire/ -- RedHill Biopharma Ltd. (Nasdaq:
RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical
company, today announced that the U.S. government's Biomedical
Advanced Research and Development Authority (BARDA), a center of
the Department of Health and Human Services (HHS)' Administration
for Strategic Preparedness and Response (ASPR), has selected
opaganib2 for development to treat exposure to Ebola
virus disease (EBOV).
Under this cost-sharing contract, BARDA will provide partial
funding for the company to further advance opaganib to mitigate
infection and contain EBOV outbreaks. To date, opaganib has made
positive development progress on the expected Animal Rule pathway
towards potential approval as a treatment for EBOV. The Animal Rule
allows for the use of pivotal animal model efficacy studies to
support U.S. Food and Drug Administration (FDA) approval of new
drugs when human clinical trials are not ethical or feasible.
"EBOV is deadly, killing, on average, half of all those who
contract it. This year marks 10 years
since the West Africa Ebola epidemic in which 11,000 people died,
and yet there are still no host-directed, small molecule therapies
approved to provide effective and useable treatment strategies,"
said Guy Goldberg, RedHill's
Chief Business Officer. "Currently only Inmazeb™, a combination
of three monoclonal antibodies, and Ebanga™, a single monoclonal
antibody, are FDA-approved to treat EBOV, as such there is an
urgent medical need for additional effective and easy to distribute
and administer EBOV therapies. There are also enormous geopolitical
and logistical challenges to overcome in managing outbreaks such as
EBOV, and others like Mpox, and so new host-directed, small
molecule therapeutic options for biodefense and global health
preparedness could prove to be major life-saving advances – this is
especially true if they are capable of viral mutation-resistance,
have extended shelf-lives for long-term storage, are relatively
straightforward to transport to hard-to-reach territories, and are
easy to administer without the need for cold-storage or
injections."
Opaganib delivered a statistically significant increase in
survival time when given at 150 mg/kg twice a day (BID) in a United
States Army Medical Research Institute of Infectious Diseases
(USAMRIID) in vivo EBOV study, making it the first
host-directed molecule to show activity in EBOV. Opaganib also
recently demonstrated a distinct synergistic effect when combined
individually with remdesivir (Veklury®, Gilead Sciences Inc.),
significantly improving potency while maintaining cell viability,
in a U.S. Army-funded and conducted in vitro EBOV study.
Opaganib is currently also in development for multiple oncology,
viral, inflammatory and diabetes and obesity-related indications,
including COVID-19, acute respiratory distress syndrome (ARDS) and
radiological and chemical protection or mitigation.
This project is supported in whole or in part with federal funds
from the Department of Health and Human Services; Administration
for Strategic Preparedness and Response; Biomedical Advanced
Research and Development Authority (BARDA), under contract number
75A50124C00059.
About Ebola virus disease:
According to the Centers
for Disease Control and Prevention (CDC), Ebola disease is a rare
and often deadly illness, caused by infection by one of a group of
four viruses, known as ebolaviruses. Transmission of the disease is
mostly through contact with an infected animal (bat or nonhuman
primate) or a sick or dead person infected with an ebolavirus. The
course of the illness typically progresses from "dry" symptoms
initially (such as fever, aches and pains, and fatigue), and then
progresses to "wet" symptoms (such as diarrhea, vomiting and
unexplained hemorrhaging, bleeding or bruising) as the person
becomes sicker. Currently only Inmazeb™ (atoltivimab, maftivimab,
and odesivimab-ebgn, Regeneron Pharmaceuticals, Inc.), a
combination of three monoclonal antibodies and Ebanga™
(ansuvimab-zykl, Ridgeback Biotherapeutics, LP), a single
monoclonal antibody, are FDA-approved to treat EBOV. Both are
intravenously administered direct acting monoclonal antibody
antivirals that bind to glycoproteins on the Ebola virus's surface
to prevent the virus from entering a person's cells. There is an
urgent need for host-directed small molecule therapies that may be
effective against multiple strains of ebolavirus, less likely to be
impacted by viral mutation, and that are easy to store, distribute
and administer, especially in areas where healthcare services and
infrastructures may be sub-optimal.
About Opaganib (ABC294640)
Opaganib, a proprietary
investigational host-directed and potentially broad-acting drug, is
a first-in-class, orally administered sphingosine kinase-2 (SPHK2)
selective inhibitor with anticancer, anti-inflammatory and
antiviral activity, targeting multiple potential indications,
including several cancers, diabetes and obesity-related disorders,
gastrointestinal acute radiation syndrome (GI-ARS), Sulfur Mustard
exposure, COVID-19, Ebola and other viruses as part of pandemic
preparedness.
Opaganib's host-directed action is thought to work through the
inhibition of multiple pathways, the induction of autophagy and
apoptosis, and disruption of viral replication, through
simultaneous inhibition of three sphingolipid-metabolizing enzymes
in human cells (SPHK2, DES1 and GCS).
Opaganib has been selected for evaluation by multiple NIH-funded
U.S. government countermeasures programs: The Radiation and Nuclear
Countermeasures Program (RNCP), led by the National Institute of
Allergy and Infectious Diseases (NIAID), part of the HHS National
Institutes of Health, for the nuclear medical countermeasures (MCM)
product development pipeline selected opaganib for development as a
potential treatment for Acute Radiation Syndrome (ARS). Opaganib
will also be evaluated as a potential countermeasure for
inhalational Sulfur Mustard exposure under a joint screening core
operated by the BARDA Chemical Medical Countermeasures (Chem MCM)
Program and the NIH/NIAID Chemical Countermeasures Research Program
(CCRP).
Opaganib has demonstrated antiviral activity against SARS-CoV-2,
multiple variants, and several other viruses, such as Influenza A
and Ebola.
Being host-targeted, and based on data accumulated to date,
opaganib is expected to maintain effect against emerging viral
variants. In prespecified analyses of Phase 2/3 clinical data in
hospitalized patients with moderate to severe COVID-19, oral
opaganib demonstrated improved viral RNA clearance, faster time to
recovery and significant mortality reduction in key patient
subpopulations versus placebo on top of standard of care. Opaganib
has demonstrated its safety and tolerability profile in more than
470 people in multiple clinical studies and expanded access use.
Data from the opaganib global Phase 2/3 study was published
in Microorganisms.
Opaganib has received several orphan-drug designations from the
FDA in oncology and other diseases and has undergone studies in
advanced cholangiocarcinoma (Phase 2a) and prostate cancer.
Opaganib also has a Phase 1 chemoradiotherapy study protocol ready
for FDA-IND submission.
Opaganib has also shown positive preclinical results in renal
fibrosis, and has the potential to target multiple oncology,
radioprotection, viral, inflammatory, and gastrointestinal
indications.
About RedHill Biopharma
RedHill Biopharma Ltd.
(Nasdaq: RDHL) is a specialty biopharmaceutical company primarily
focused on gastrointestinal and infectious diseases. RedHill
promotes the gastrointestinal drug Talicia®, for
the treatment of Helicobacter pylori (H. pylori) infection
in adults3. RedHill's key clinical late-stage
development programs include: (i) opaganib (ABC294640),
a first-in-class oral broad-acting, host-directed
SPHK2 selective inhibitor with potential for pandemic preparedness,
targeting multiple indications with a U.S. government collaboration
for development for Acute Radiation Syndrome (ARS), a Phase 2/3
program for hospitalized COVID-19, and a Phase 2 program in
oncology; (ii) RHB-107 (upamostat), an oral
broad-acting, host-directed, serine protease inhibitor with
potential for pandemic preparedness is in late-stage development as
a treatment for non-hospitalized symptomatic COVID-19, with
non-dilutive external funding covering the entirety of the RHB-107
arm of the 300-patient Phase 2 adaptive platform trial, and is also
targeting multiple other cancer and inflammatory gastrointestinal
diseases; (iii) RHB-102, with potential UK submission
for chemotherapy and radiotherapy induced nausea and vomiting,
positive results from a Phase 3 study for acute gastroenteritis and
gastritis and positive results from a Phase 2 study for IBS-D;
(iv) RHB-104, with positive results from a first Phase
3 study for Crohn's disease; and (v) RHB-204, a
Phase 3-stage program for pulmonary nontuberculous mycobacteria
(NTM) disease.
More information about the Company is available at
www.redhillbio.com / X.com/RedHillBio.
Forward-Looking Statements
This press release
contains "forward-looking statements" within the meaning of the
Private Securities Litigation Reform Act of 1995 and may discuss
investment opportunities, stock analysis, financial performance,
investor relations, and market trends. 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 include among
others, statements regarding the potential effects of opaganib in
the treatment of Ebola and other indications. 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: market
and other conditions; the Company's ability to maintain compliance
with the Nasdaq Capital Market's listing requirements; the risk
that the addition of new revenue generating products or
out-licensing transactions will not occur; the risk that acceptance
onto the RNCP Product Development Pipeline will not guarantee
ongoing development or that any such development will not be
completed or successful; the risk that the FDA does not agree with
the Company's proposed development plans for opaganib for any
indication; the risk that observations from preclinical studies are
not indicative or predictive of results in clinical trials; the
risk that the FDA pre-study requirements will not be met and/or
that the Phase 3 study of RHB-107 in COVID-19 outpatients will not
be approved to commence or if approved, will not be completed or,
should that be the case, that we will not be successful in
obtaining alternative non-dilutive development funding for RHB-107;
the risk that RHB-107's late-stage development for non-hospitalized
COVID-19 will not benefit from the resources redirected from the
terminated RHB-204 Phase 3 study, and that the Phase 2/3 COVID-19
study for RHB-107 may not be successful and, even if successful,
such studies and results may not be sufficient for regulatory
applications, including emergency use or marketing applications,
and that additional COVID-19 studies for opaganib and RHB-107 are
likely to be required; the risk that the Company will not
successfully commercialize its products; as well as risks and
uncertainties associated with (i) the initiation, timing, progress
and results of the Company's research, manufacturing, pre-clinical
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
pre-clinical studies or clinical trials or the development of a
commercial companion diagnostic for the detection of MAP; (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
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 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, pre-clinical 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 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 experiences using investigative drugs under the Company's
Expanded Access Program; (xiv) competition from other companies and
technologies within the Company's industry; and (xv) the hiring and
employment commencement date of executive managers. 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 April 8, 2024. 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.
Company contact:
Adi
Frish
Chief Corporate & Business Development Officer
RedHill Biopharma
+972-54-6543-112
adi@redhillbio.com
Category: R&D
1
https://www.who.int/news-room/fact-sheets/detail/ebola-virus-disease
2 Opaganib is an investigational new drug, not available for
commercial distribution.
3 Talicia® (omeprazole magnesium, amoxicillin and
rifabutin) is indicated for the treatment of H. pylori infection in
adults. For full prescribing information see: www.Talicia.com.
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