Neuroblastoma is rare but is the most common infancy
malignancy with a median age of diagnosis of 17 months. In the
U.S., it accounts for up to around 10% of all childhood cancer
cases and 15% of pediatric cancer-related
deaths[1],[2]
Orphan Drug designation provides for seven-years' marketing
exclusivity should opaganib be approved in neuroblastoma and may
confer additional benefits such as accelerated development and
review times, potential grant funding and possible tax
credits
The neuroblastoma market is expected to reach almost
$1.5 billion before
mid-2030s[3]
This is the second orphan drug designation by the FDA for
opaganib in oncology, after cholangiocarcinoma (bile duct cancer,
CCA)
With multiple U.S. government collaborations ongoing,
opaganib is a novel, host-directed, potentially broad acting,
orally administered small molecule drug with demonstrated safety
& efficacy profiles
TEL
AVIV, Israel and RALEIGH, N.C., Aug. 26,
2024 /PRNewswire/ -- RedHill Biopharma Ltd.
(Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty
biopharmaceutical company, today announced that the U.S. Food and
Drug Administration (FDA) has granted orphan-drug designation to
opaganib for treatment of neuroblastoma, a type of childhood cancer
that develops from immature nerve cells and accounts for 15% of all
pediatric cancer-related deaths.
Orphan Drug designation provides for a seven-year marketing
exclusivity period should opaganib be approved in neuroblastoma and
may confer additional benefits such as accelerated development and
review times, potential grant funding and possible tax credits.
"RedHill is proud to have received a second orphan-drug
designation for opaganib in oncology, following its previous
designation for cholangiocarcinoma (CCA, also known as bile duct
cancer). This designation for neuroblastoma – the most common
infancy malignancy and for which new options are urgently needed –
adds to opaganib's potential as a novel oncological agent," said
Dr. Mark Levitt, Chief Scientific
Officer at RedHill. "Opaganib has broad oncology potential with
promising preliminary clinical data in solid tumor cancers such as
prostate cancer and CCA, and data from a range of U.S. government
supported and Apogee conducted preclinical studies in various
indications, including radioprotection, and also in combination
with RedHill's RHB-107. We also see such utility extending to the
potential for opaganib to have a sensitizing effect in hormone
receptor pathway inhibition therapy, which the Company expects to
test in a planned externally funded Phase 2 study."
Neuroblastoma usually affects children aged five or younger but
does also occur in older children. In the
United States, approximately 650 new cases are diagnosed
each year, accounting for around 10% of all childhood cancer cases.
Treatment typically involves a combination of surgery,
chemotherapy, radiation therapy, and sometimes targeted therapy or
immunotherapy. New options are urgently needed.
The neuroblastoma market is expected to reach almost
$1.5 billion before the middle of the
next decade.
Opaganib is in development for multiple oncology, viral,
inflammatory and diabetes and obesity-related indications,
including COVID-19, Ebola, acute respiratory distress syndrome
(ARDS) and radio/chemical protection.
About Neuroblastoma
Neuroblastoma is a type of cancer most commonly affecting babies
and young children. It is rare but is the most common infancy
malignancy with a median age of diagnosis of 17 months. In the U.S.
it accounts for up to around 10% of all childhood cancer cases. It
affects around 11 to 13 kids per million children aged younger than
15 years old – with incidence rising to 65 per million in babies
under one year old, equating to approximately 650 new cases each
year.
Neuroblastoma originates from nerve cells called neuroblasts,
which are found in the adrenal glands (located above the kidneys)
and in nerve tissue along the spine, chest, abdomen, or pelvis. The
exact cause of neuroblastoma is not well understood, but genetic
mutations and abnormalities are known to play a role. Some cases
may be linked to genetic syndromes or family history, although most
occur sporadically without a clear inherited pattern.
The outlook for children with neuroblastoma can vary widely.
Factors affecting prognosis include the stage of the disease at
diagnosis, the age of the child, and specific biological
characteristics of the tumor. Neuroblastoma is known for its
ability to sometimes regress spontaneously in very young children,
but it can also be aggressive and challenging to treat, accounting
for 15% of pediatric cancer-related deaths.
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 diseases, including obesity-related syndromes,
prostate cancer and cholangiocarcinoma (bile duct cancer),
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 two U.S. government
countermeasures programs for Acute Radiation Syndrome (ARS) and
Sulfur Mustard exposure, both funded by the NIH: 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); and the Chemical Medical Countermeasures
(Chem MCM) Program and the Chemical Countermeasures Research
Program (CCRP), managed respectively by the Administration for
Strategic Preparedness and Response (ASPR) / Biomedical Advanced
Research and Development Authority (BARDA) and NIH/NIAID selected
opaganib for evaluation as a potential medical countermeasure (MCM)
against Sulfur Mustard exposure.
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 medRxiv.
Opaganib has received Orphan Drug designation from the FDA for
the treatment of neuroblastoma and cholangiocarcinoma 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 drugs
Talicia®, for the treatment of Helicobacter
pylori (H. pylori) infection in adults[4], and
Aemcolo®, for the treatment of travelers'
diarrhea in adults[5]. 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 statements regarding 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: market and other conditions; the Company's ability to
regain compliance with the Nasdaq Capital Market's minimum bid
price 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.
1.
https://www.ncbi.nlm.nih.gov/books/NBK448111/#:~:text=Neuroblastoma%20is%20the%20most%20common,of%20pediatric%20cancer%2Drelated%20deaths.
2. Yan P, Qi F, Bian L, et al. Comparison of Incidence
and Outcomes of Neuroblastoma in Children, Adolescents, and Adults
in the United States: A
Surveillance, Epidemiology, and End Results (SEER) Program
Population Study. Med Sci Monit.
2020;26:e927218. Published 2020 Nov 29.
doi:10.12659/MSM.927218.
3. Shinde S., Pawar P. and Deshmukh R., Neuroblastoma
Drugs Market Size, Share, Competitive Landscape and Trend Analysis
Report, by Type, by Route of Administration, by Distribution
Channel: Global Opportunity Analysis and Industry Forecast,
2023-2032, Allied Market Research, Jan.
2024,
https://www.alliedmarketresearch.com/neuroblastoma-drugs-market-A172697.
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.
5. 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.
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
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SOURCE RedHill Biopharma Ltd.
