Opaganib awarded a further $1.7 million in U.S. Government funding for
development as a medical countermeasure for gastrointestinal acute
radiation syndrome (ARS)
The Small Business Innovation Research (SBIR)
grant, given to RedHill's development partner, Apogee, is in
addition and complementary to the multimillion dollar-valued U.S.
Government Radiation and Nuclear
Countermeasures Program (RNCP) product pipeline
development contract awarded to opaganib following its selection by
the RNCP for ARS development
This SBIR grant, and the earlier
selection by the RNCP, follows FDA confirmation of Animal Rule
regulatory pathway applicability for opaganib for ARS, utilizing
pivotal animal model efficacy studies as the basis for FDA approval
instead of human efficacy trials
Opaganib, a novel oral, small molecule pill
with a five-year shelf-life, is easy to administer and distribute,
supporting potential central government stockpiling for use in mass
casualty radiological or nuclear incidents, if approved by the
FDA
Opaganib is being developed for multiple
indications, including COVID-19, acute respiratory distress
syndrome (ARDS), oncology and additional indications
TEL
AVIV, Israel and RALEIGH,
N.C., July 21, 2023 /PRNewswire/ -- RedHill
Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a
specialty biopharmaceutical company, today announced that
opaganib[1] has been awarded a further $1.7 million in U.S. Government funding, via a
Small Business Innovation Research (SBIR) grant to the Company's
development partner, Apogee Biotechnology Corporation ("Apogee").
This SBIR grant will support research to further the development of
opaganib as a medical countermeasure (MCM) for gastrointestinal
acute radiation syndrome (GI-ARS). This grant is in addition and
complementary to the multimillion dollar-valued U.S. Government
Radiation and Nuclear Countermeasures Program (RNCP) product
pipeline development contract awarded to opaganib following its
selection by the RNCP for ARS development.
![RedHill_Biopharma_Logo RedHill_Biopharma_Logo](https://mma.prnewswire.com/media/1334141/RedHill_Biopharma_Logo.jpg)
The mission of the SBIR programs is to support scientific
excellence and technological innovation through the investment of
Federal research funds in critical American priorities to build a
strong national economy.
Dror Ben-Asher, CEO of RedHill
Biopharma, said: "In light of ongoing regional geo-political
instabilities, it is important for us to bring together all the
pieces of the development jigsaw for opaganib as a potential
medical countermeasure for acute radiation syndrome. Opaganib is
now being supported by several multimillion dollar-valued
government grants. This latest additional U.S. Government funding
from the SBIR, in addition to the RNCP development collaboration,
is expected to help ensure that we and our partner Apogee can
rapidly progress opaganib's development for ARS. In parallel, we
plan to continue our existing collaborations and discussions with
other U.S. Government agencies and other governments regarding
opaganib for ARDS, COVID-19 and other indications, as well as for
our other new chemical entity (NCE) under clinical development,
RHB-107 (upamostat)[2]."
In an ARS setting, opaganib is thought to exert its protective
effects via an anti-inflammatory mechanism of action involving
ceramide elevation and reduction of sphingosine 1-phosphate. This
is believed to reduce inflammatory damage to normal tissue and thus
suppress toxicity from unintended ionizing radiation exposure. A
recent publication in the International Journal of Molecular
Sciences, entitled "Opaganib Protects against Radiation Toxicity:
Implications for Homeland Security and Antitumor
Radiotherapy" describes the collective results of eight U.S.
Government-funded in vivo studies by Apogee, as well as
additional experiments, establishing opaganib's potential radiation
protection capabilities[3]. In the relevant
study models, opaganib was associated with protection of normal
tissue, including the GI tract, from damage due to ionizing
radiation exposure from total-body exposure. Additional independent
studies demonstrate the role of inhibition of sphingosine kinase-2
(SPHK2), the primary target of opaganib, in radioprotection in bone
marrow, with knockout of SPHK2 showing enhanced survival in mice
irradiated with lethal doses of whole-body
radiation[4].
Opaganib, a novel, oral, small molecule pill with a five-year
shelf-life, is easy to administer and distribute, supporting
potential central government stockpiling for use in mass casualty
nuclear radiation incidents, if approved.
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 new chemical entity, is an orally administered,
first-in-class proprietary selective inhibitor of SPHK2 with
suggested anti-inflammatory, anticancer, radioprotective and
antiviral activity. The SPHK2 inhibitor 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).
In addition to development for gastrointestinal ARS,
opaganib is also being developed as a host-directed antiviral
against SARS-CoV-2 and other viruses and has received Orphan Drug
designation from the FDA for the treatment of cholangiocarcinoma. A
Phase 2a study in advanced cholangiocarcinoma and a prostate cancer
study is ongoing. Opaganib also has a Phase 1 chemoradiotherapy
study protocol ready for FDA-IND submission.
Opaganib has demonstrated broad-acting, host-directed, antiviral
activity against SARS-CoV-2, multiple variants, and several other
viruses, such as Influenza A. 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.
Data from the opaganib global Phase 2/3 study has been submitted
for peer review and recently published in medRxiv.
Opaganib has also shown positive preclinical results in renal
fibrosis, and has the potential to target multiple oncology,
radioprotection, viral, inflammatory, and gastrointestinal
indications. Opaganib has demonstrated its safety and tolerability
profile in more than 470 people in multiple clinical studies and
expanded access use.
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
Talicia®, for the treatment of Helicobacter
pylori (H. pylori) infection in
adults[5], and Aemcolo®,
for the treatment of travelers' diarrhea in
adults[6]. 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, 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/ twitter.com/RedHillBio.
Forward Looking Statements
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 include statements regarding anticipated the
addition of new revenue generating products, out-licensing of the
Company's development pipeline assets, timing of opaganib's
development for Acute Radiation Syndrome, non-dilutive development
funding from RHB-107 and its inclusion in a key platform study.
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 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 HB-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, 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, as well as risks and uncertainties
associated with 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 28, 2023. 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: Financials
[1] Opaganib is an investigational new drug, not
available for commercial distribution.
[2] RHB-107 is an investigational new drug, not
available for commercial distribution.
[3] Maines LW, Schrecengost RS, Zhuang Y, Keller SN,
Smith RA, Green CL, Smith CD. Opaganib Protects against Radiation
Toxicity: Implications for Homeland Security and Antitumor
Radiotherapy. International Journal of Molecular Sciences. 2022;
23(21):13191. https://doi.org/10.3390/ijms232113191.
[4] Li C. et al., Loss of Sphingosine Kinase 2
Promotes the Expansion of Hematopoietic Stem Cells by Improving
Their Metabolic Fitness. Blood. October
2022;140(15):1686-1701.
[5] 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.
[6] Aemcolo® (rifamycin) is indicated for
the treatment of travelers' diarrhea caused by noninvasive strains
of Escherichia coli in adults. For full prescribing
information see: www.aemcolo.com.
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SOURCE RedHill Biopharma Ltd.