U.S. Phase 3 study underway to evaluate RHB-204 as a
first-line, stand-alone, oral treatment for pulmonary NTM disease -
a rare condition with no FDA-approved first-line therapy
FDA Fast Track designation, together with previously granted
QIDP designation, provides RHB-204 with eligibility for rolling NDA
review, Priority Review and Accelerated Approval
RHB-204 Orphan Drug designation extends potential market
exclusivity to 12 years post-approval
TEL AVIV, Israel and
RALEIGH, NC, Jan. 6, 2021 /PRNewswire/ -- RedHill Biopharma
Ltd. (Nasdaq: RDHL) ("RedHill" or "the Company"), a specialty
biopharmaceutical company, today announced that RHB-204 has been
granted Fast Track designation by the U.S. Food and Drug
Administration (FDA) for its development as a potential first-line,
stand-alone, oral treatment of pulmonary nontuberculous
mycobacteria (NTM) disease caused by Mycobacterium avium
Complex (MAC) – a rare disease for which there is no FDA-approved
first-line therapy.
The FDA's Fast Track designation is designed to help progress
development and speed up the review of novel therapies for serious
conditions for which there is an unmet medical need - with the aim
of getting important new therapies to patients more quickly. With
the Fast Track designation, RedHill will have access to early and
frequent communications with the FDA, to expedite the RHB-204
development program, and to a rolling review of a New Drug
Application (NDA). Having already been granted Qualified Infectious
Disease Product (QIDP) designation, RHB-204 is also eligible for
NDA Priority Review and Accelerated Approval.
RHB-204 was also recently granted Orphan Drug designation,
extending U.S. market exclusivity for RHB-204 to a potential total
of 12 years upon FDA approval.
RedHill recently initiated a Phase 3 study evaluating the safety
and efficacy of RHB-204 as a first-line treatment for pulmonary NTM
disease, to be conducted at up to 40 sites across the
U.S.
"Given the urgent need to improve therapeutic options for
patients with NTM disease, we welcome this Fast Track designation
and the regulatory support it provides in expediting the ongoing
Phase 3 development program for RHB-204 and any subsequent
potential approvals," said Patricia
Anderson, RedHill's Senior Vice President of Regulatory
Affairs. "NTM disease is thankfully rare but its prevalence is
increasing in many areas of the world. It is a notoriously
difficult to treat disease and, if not effectively treated, can
cause scarring and fibrosis in the lungs - potentially leading to
respiratory failure. Many patients fail current therapies, and more
than half will have either recurring disease or a new infection
after completing treatment [1],[2]."
RHB-204 may be eligible for use under the RedHill expanded
access policy – more details of which can be found here:
https://www.redhillbio.com/expandedaccess. The Phase 3 study
of RHB-204 is registered on www.ClinicalTrials.gov, a
web-based service by the U.S. National Institute of Health, which
provides public access to information on publicly and privately
supported clinical studies.
About Pulmonary Nontuberculous Mycobacteria (NTM)
Disease
Pulmonary nontuberculous mycobacteria (NTM) disease is a chronic
and debilitating lung disease caused by ubiquitous environmental
bacteria found in soil, as well as natural and engineered water
systems. The most common NTM symptoms include fever, weight loss,
chest pain, and blood in sputum[3]. Pulmonary NTM
disease can lead to recurring cases of bronchitis and pneumonia and
can, in some cases, lead to respiratory failure[4].
Although rare, the incidence and prevalence of pulmonary NTM
disease are increasing in many areas of the world[5].
There were an estimated 110,000 pulmonary NTM disease patients in
the U.S. in 2017, with U.S. market potential estimated at over
$500 million[6]. Pulmonary
manifestations account for 80-90% of all NTM-associated
diseases[7], and approximately 80% of pulmonary NTM
disease are caused by Mycobacterium avium Complex
(MAC)[8].
About RHB-204
RHB-204 is a proprietary, fixed-dose oral capsule containing a
combination of clarithromycin, rifabutin, and clofazimine,
developed for the treatment of pulmonary NTM disease caused by
Mycobacterium avium Complex (MAC). In addition to FDA Fast
Track designation, RHB-204 has been granted FDA Orphan Drug
designation for the treatment of NTM disease and QIDP Designation
under the Generating Antibiotic Incentives Now Act (GAIN Act),
extending U.S. market exclusivity for RHB-204 to a potential total
of 12 years to be granted at the time of FDA approval. RHB-204 is
also covered by U.S. patents which extend patent protection until
2029 and a pending U.S. patent application which, if allowed, could
extend RHB-204 patent protection until 2041.
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,
Movantik® for opioid-induced constipation in
adults[9], Talicia® for the
treatment of Helicobacter pylori (H. pylori) infection in
adults[10], and Aemcolo®
for the treatment of travelers' diarrhea in
adults[11]. RedHill's key clinical late-stage
development programs include: (i) RHB-204, with an
ongoing Phase 3 study for pulmonary nontuberculous mycobacteria
(NTM) disease; (ii) opaganib (Yeliva®), a
first-in-class SK2 selective inhibitor targeting
multiple indications with a Phase 2/3 program for COVID-19 and
Phase 2 studies for prostate cancer and cholangiocarcinoma ongoing;
(iii) RHB-104, with positive results from a first Phase 3
study for Crohn's disease; (iv) RHB-102
(Bekinda®), with positive results from a Phase 3
study for acute gastroenteritis and gastritis and positive results
from a Phase 2 study for IBS-D; (v) RHB-107
(upamostat), a Phase 2-stage serine protease inhibitor with
a planned Phase 2/3 study in symptomatic COVID-19 and targeting
multiple other cancer and inflammatory gastrointestinal diseases;
and (vi) RHB-106, an encapsulated bowel preparation.
More information about the Company is available at
www.redhillbio.com / https://twitter.com/RedHillBio.
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. 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 Company will not succeed to
complete the patient recruitment; the risk that the Company will
not receive the relevant data required for benefiting from the Fast
Track designation; the risk that the U.S. Phase 3 clinical study
evaluating RHB-204 will not be successful or, if successful, will
not suffice for regulatory marketing approval without the need for
additional clinical and/or other studies; 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® and
Movantik®; (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 March 4, 2020.
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.
References:
[1] Henkle E, et al. Patient-Centered Research
Priorities for Pulmonary Nontuberculous Mycobacteria (NTM)
Infection. An NTM Research Consortium Workshop Report Annals of
the American Thoracic Society 2016; S379-84.
[2] Daley CL, et al. Treatment of Nontuberculous
Mycobacterial Pulmonary Disease: An Official ATS/ERS/ESCMID/IDSA
Clinical Practice Guideline: Executive Summary. Clinical
Infectious Diseases.
Ciaa241, https://doi.org/10.1093/cid/ciaa241.
[3] Kim RD, et al. Pulmonary Nontuberculous
Mycobacterial Disease. Prospective Study of a Distinct Preexisting
Syndrome Am J Respir Crit Care Med. 2008;
178(10):1066–74.
[4] The American Lung Association, 2020.
[5] Henkle E, et al. Population-based Incidence of
Pulmonary Nontuberculous Mycobacterial Disease in Oregon 2007 to 2012 Annals of the American
Thoracic Society. 2015; 12(5):642-7.
[6] Foster|Rosenblatt, 2017.
[7] Griffith DE, et al. An official ATS/IDSA
statement: diagnosis, treatment, and prevention of nontuberculous
mycobacterial diseases Am J Respir Crit Care Med.
2007;175(4):367-416.
[8] Prevots DR et al. Nontuberculous mycobacterial
lung disease prevalence at four integrated health care delivery
systems. Am J Respir Crit Care Med 2010;
182:970-76; Winthrop KL, et al. Pulmonary
nontuberculous mycobacterial disease prevalence and clinical
features: an emerging public health disease. Am J Respir Crit
Care Med 2010; 182: 977-82
[9] Full prescribing information for Movantik®
(naloxegol) is available at: www.Movantik.com.
[10] Full prescribing information for Talicia®
(omeprazole magnesium, amoxicillin and rifabutin) is available at:
www.Talicia.com.
[11] Full prescribing information for Aemcolo®
(rifamycin) is available at: www.Aemcolo.com.
Logo:
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Company
contact:
Adi Frish
Chief Corporate &
Business Development Officer
RedHill
Biopharma
+972-54-6543-112
adi@redhillbio.com
|
Media contact
(U.S.):
Bryan
Gibbs
Vice
President
Finn
Partners
+1 212 529
2236
bryan.gibbs@finnpartners.com
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