The collaboration outlines plans for multiple in vivo
studies, designed to test opaganib as a potential medical
countermeasure to treat phosgene inhalation injury. The
collaboration aims to determine whether opaganib can advance into
further definitive U.S. government-sponsored development under the
FDA's Animal Rule pathway to approval
Used as a chemical weapon during World War I, phosgene, a
toxic, colorless chemical, is today widely used in industrial
processes for the manufacture of plastics and pesticides. The U.S.
Environmental Protection Agency (EPA) identified 123 sites in
the United States that could
potentially expose millions of people to phosgene due to plant
malfunction or bioterrorism[1]
Phosgene inhalation can be extremely dangerous, causing
significant, even fatal, respiratory injury. No known approved
antidote or reversal agent currently exists, and, if approved,
opaganib may provide potential for stockpiling for emergency use in
the event of a major public safety incident
With multiple U.S. government collaborations for
chemical and medical countermeasures and pandemic preparedness,
RedHill's opaganib is a novel, host-directed, potentially broad
acting, orally administered small molecule, clinical-stage drug
with demonstrated safety & efficacy profiles, being
developed for various oncology, viral infections,
inflammatory diseases and chemical and nuclear/radioprotection
indications
TEL
AVIV, Israel and RALEIGH,
N.C., Oct. 22, 2024 /PRNewswire/ -- RedHill
Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a
specialty biopharmaceutical company, today announced the signing of
a collaborative research agreement with Duke
University School of Medicine outlining plans for multiple
in vivo studies. The program is designed to test
opaganib[2] as a potential medical countermeasure to
treat phosgene inhalation injury, aimed at providing results
sufficient to advance opaganib into further definitive U.S.
government-sponsored development under the U.S. Food and Drug
Administration's (FDA) Animal Rule pathway to approval. The FDA
Animal Rule allows for the use of pivotal animal model efficacy
studies to support FDA approval of new drugs when human clinical
trials are not ethical or feasible. Under this research agreement,
RedHill will provide required drug quantities for animal studies,
analytical support to quantify drug concentrations in plasma
samples, and share dosing regimen with the Achanta Lab at
Duke University.
"Opaganib is currently being tested, by various governmental
research bodies, in multiple areas that urgently require new
chemical and medical countermeasure therapeutic options," said
Dr. Satya Achanta, D.V.M., Ph.D.,
Assistant Professor in the Department of Anesthesiology at
Duke University School of Medicine.
"Our research will help determine whether there is a potential
protective role of opaganib in limiting damage caused by phosgene
inhalation."
"Notably used as a chemical weapon during World War I, phosgene,
a toxic, colorless chemical, is today widely used in industrial
processes for the manufacture of plastics and pesticides. The U.S.
Environmental Protection Agency (EPA) identified 123 sites in
the United States that could
expose millions of people to phosgene potentially due to plant
malfunction or bioterrorism," said Gilead Raday, RedHill's Chief Operating Officer
and Head of Research and Development. "This exciting
collaboration with Duke University
School of Medicine could pave the way to a potential therapy for
the acute respiratory distress syndrome (ARDS) phosgene inhalation
can cause, which can be extremely dangerous, causing significant,
even fatal, injury. No known approved antidote or reversal agent
currently exists, and, if approved, opaganib may provide potential
for stockpiling for emergency use in the event of a major public
safety incident."
What is Phosgene inhalation injury
Phosgene inhalation injury occurs when someone breathes in
phosgene gas (COCl₂), a toxic chemical primarily used in industrial
processes. Phosgene has a distinct odor, similar to freshly mown
grass or hay. However, only 10% of the population may detect this
odor at lower concentrations. The combination of its unassuming
odor and poor human detection makes phosgene particularly
dangerous. At higher concentrations, it is extremely dangerous,
even fatal, and is a cause of acute respiratory distress syndrome
(ARDS). It is believed that no antidotes or reversal agents exist
at this time. Phosgene was notably used as a chemical weapon during
World War I.
Today, phosgene use in industry is ubiquitous - being used in
the production of polymers, plastics and pesticides. Global
estimates indicate that more than 12 million metric tons are
produced annually[3]. Despite its widespread use, some
regions poorly regulate its production, and concern exists for its
potential as a weapon of bioterrorism. Given its severe toxicity
and high mortality, research is primarily limited to an Animal Rule
pathway to approval, which allows for the use of pivotal animal
model efficacy studies to support FDA approval of new drugs when
human clinical trials are not ethical or feasible.
The EPA identified 123 sites in the
United States that could expose millions of people to
phosgene if the plant malfunctions or becomes a target of
bioterrorism.
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).
Several U.S. government countermeasures and pandemic
preparedness programs have selected opaganib for evaluation for
multiple indications, including Acute Radiation Syndrome
(ARS), Ebola virus disease and others Funding bodies include
the Radiation and Nuclear Countermeasures Program (RNCP), led by
the National Institute of Allergy and Infectious Diseases (NIAID),
part of the U.S. government Department of Health & Human
Services' National Institutes of Health and the Administration for
Strategic Preparedness and Response's (ASPR) Center for Biomedical
Advanced Research and Development Authority (BARDA).
Opaganib has demonstrated antiviral activity against SARS-CoV-2,
multiple variants, and several other viruses, such as Influenza A
and Ebola. 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 Ebola virus study, making it the
first host-directed molecule to show activity in Ebola virus
disease. 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
Ebola virus study.
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
adults[4]. 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 chemical and medical
countermeasure and pandemic preparedness use, 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 phosgene inhalation injury.
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.ncbi.nlm.nih.gov/books/NBK589660/
[2] Opaganib is an investigational new drug, not available
for commercial distribution.
[3] https://www.ncbi.nlm.nih.gov/books/NBK589660/
[4]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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SOURCE RedHill Biopharma Ltd.