Heightened geopolitical tensions and Homeland Security
concerns call for the development of potential therapies that can
be quickly and easily mobilized in the event of a mass casualty
nuclear or radiological incident
There are currently no known approved therapies for
gastrointestinal acute radiation syndrome (GI-ARS)
Positive results from new in vivo studies of opaganib as a
treatment for GI-ARS, undertaken as part of the U.S. government's
Radiation and Nuclear Countermeasures Program
(RNCP) product pipeline development contract, further
confirm opaganib's protective activity
Discussions now ongoing with the National Institutes of
Health's (NIH) National Institute of Allergy and Infectious
Diseases (NIAID), which leads the RNCP, regarding plans for the
next phase of development along the U.S. Food and Drug
Administration's (FDA) Animal Rule pathway to approval
TEL
AVIV, Israel and RALEIGH,
N.C., Dec. 10, 2024 /PRNewswire/ -- RedHill
Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a
specialty biopharmaceutical company, today announced positive
results from new in vivo studies of
opaganib[1] as a treatment for gastrointestinal acute
radiation syndrome (GI-ARS), undertaken as part of the U.S.
government's Radiation and Nuclear Countermeasures Program (RNCP)
product pipeline development contract awarded to opaganib, that
further confirm opaganib's protective activity in models of
GI-ARS.
The Company, together with the National Institutes of Allergy
and Infectious Diseases (NIAID), which leads the U.S. government's
RNCP, now plan to discuss the next phase of opaganib's U.S.
government-funded development, along the U.S. Food and Drug
Administration's (FDA) Animal Rule pathway, as a medical
countermeasure against GI-ARS. The FDA's Animal Rule pathway to
approval allows for pivotal animal model efficacy studies to
support FDA approval of new drugs when human clinical trials are
not ethical or feasible.
"There are currently no known approved therapies for GI-ARS.
Growing geopolitical tensions and Homeland Security concerns call
for development of new potential therapies that can be quickly and
easily deployed in the event of a mass casualty nuclear or
radiological incident," said Gilead
Raday, RedHill's Chief Operating Officer and Head of
Research and Development. "Opaganib, as an oral, small molecule
pill that is highly stable with a more than five-year shelf-life,
is easy to administer and distribute, supporting, if approved,
potential central stockpiling by governments for use when
needed."
With multiple U.S. government collaborations for chemical and
medical countermeasures and pandemic preparedness, 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.
About Acute Radiation Syndrome (ARS)
ARS, sometimes known as radiation toxicity or radiation
sickness, is generally rare; however, public health emergencies,
such as a nuclear power plant accident or detonation of a nuclear
device, could affect large numbers of people. ARS is an acute
illness caused by irradiation of the body by a high dose of
penetrating radiation in a short period of time. Much of the damage
caused by ARS is caused by inflammation secondary to the direct
effects of ionizing radiation itself.
Current treatments for ARS are supportive care, including blood
transfusions, antibiotics, etc., as well as the availability of
four approved products to mitigate hematologic-ARS (three growth
factors to address neutropenia and one to mitigate
thrombocytopenia). However, other radiation-induced clinical
manifestations that have been observed in natural history studies,
and remain unaddressed with the current treatments, include GI-ARS,
cutaneous injury, and late effects in the lung, heart, and kidneys.
Opaganib, an SPHK2 inhibitor, may offer a new therapeutic approach
to mitigate GI-ARS. It has also been reported in the literature
that inhibition of SPHK2 promotes the viability and robustness of
hematopoietic stem cells, even in the face of radiation damage,
supporting increased survival.
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), chemical exposure indications, 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 U.S
commercialization and development of drugs for gastrointestinal
diseases, infectious diseases and oncology. RedHill promotes the
gastrointestinal drug Talicia®, for the treatment
of Helicobacter pylori (H. pylori) infection in
adults[2]. RedHill's key clinical late-stage
development programs include: (i) opaganib (ABC294640),
a first-in-class, orally administered sphingosine kinase-2 (SPHK2)
selective inhibitor with anticancer, anti-inflammatory and
antiviral activity, targeting multiple indications with U.S.
government and academic collaborations for development for
radiation and chemical exposure indications such as 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 GI-ARS. 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.
Disclaimer: The views and opinions of authors expressed
herein do not necessarily state or reflect those of the U.S.
government and shall not be used for advertising or product
endorsement purposes. Reference herein to any specific commercial
products, process, or service by trade name, trademark,
manufacturer, or otherwise, does not constitute or imply its
endorsement, recommendation, or favoring by the U.S. government and
shall not be used for advertising or product endorsement
purposes.
1. Opaganib is an investigational new drug, not
available for commercial distribution.
2. 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.
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
Category: R&D
View original
content:https://www.prnewswire.com/news-releases/radioprotective-activity-of-redhills-opaganib-for-gi-ars-confirmed-in-new-rncpniaid-study---discussions-ongoing-with-us-government-on-advanced-development-302327260.html
SOURCE RedHill Biopharma Ltd.
