RHB-204 is being evaluated as a first-line, stand-alone, oral
treatment for pulmonary nontuberculous mycobacteria (NTM) disease -
a rare condition with no FDA-approved first-line therapy
The Phase 3 study is expected to recruit up to 125 patients
across approximately 40 U.S. clinical sites
RHB-204 Orphan Drug designation and QIDP designation extend
potential market exclusivity up to 12 years post-approval and
provide eligibility for Fast-Track development and NDA Priority
Review
TEL AVIV, Israel and
RALEIGH, N.C., Nov. 20, 2020 /PRNewswire/ -- RedHill
Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or "the Company"), a
specialty biopharmaceutical company, today announced that it has
initiated its Phase 3 study to evaluate the safety and efficacy of
RHB-204 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.
"NTM is a debilitating disease that can cause scarring, fibrosis
and the formation of cavities or pits in the lungs, which can lead
to potentially fatal respiratory failure. People with existing lung
conditions, such as bronchiectasis and those with COPD, are
particularly susceptible," said Prof. Kevin Winthrop, MD, MPH, Professor of Infectious
Diseases, Oregon Health & Science University, and study
Principal Investigator. "NTM is notoriously resistant to most
antibiotics and challenging to treat, and there is no FDA-approved
first-line therapy for the approximately 110,000 cases of NTM
infection in the U.S. This study of orally-administered RHB-204, if
successful, represents an opportunity to make a breakthrough in
managing NTM infections."
"Treatment of NTM disease requires multiple antibiotics and an
extended treatment course due to the risk of development of
resistance1," said Aida
Bibliowicz, RedHill's Vice President of Clinical
Affairs. "Many patients fail these types of therapies and more
than half will have either recurring disease or a new infection
after completing treatment2, making new treatment
options for NTM an urgent need."
The multi-center, randomized, double-blind, two-part,
placebo-controlled, parallel-group Phase 3 study will be conducted
at up to 40 sites across the U.S. and aims to enroll 125 patients,
randomized at a 3:2 ratio to receive either RHB-204 or placebo. The
study is designed to evaluate the safety and efficacy of RHB-204 in
patients with symptomatic Mycobacterium avium Complex (MAC)
lung disease. Study endpoints include sputum culture conversion at
month six of treatment with RHB-204, compared to placebo and
patient-reported outcomes, including improvements in physical
functioning, respiratory symptoms and fatigue. Following this
assessment (part one of the study), patients may be eligible to
continue double-blinded treatment for up to 16 months (part two).
Sustainability of clinical benefit and durability of
microbiological response will be assessed at month 16 and again
three months after treatment completion.
RHB-204 was recently granted Orphan Drug designation, extending
U.S. market exclusivity for RHB-204 by an additional seven years,
for a potential total of 12 years upon FDA approval. RHB-204 had
also previously been granted a Qualified Infectious Disease Product
(QIDP) designation by the FDA, providing eligibility for Fast-Track
development, NDA Priority Review and a five-year extension of U.S.
market exclusivity, if approved.
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 sputum3. Pulmonary NTM disease
can lead to recurring cases of bronchitis and pneumonia and can, in
some cases, lead to respiratory failure4. Although rare,
the incidence and prevalence of pulmonary NTM disease are
increasing in many areas of the world5. There were an
estimated 110,000 pulmonary NTM disease patients in the U.S. in
20176. Pulmonary manifestations account for 80-90% of
all NTM-associated diseases7, 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). RHB-204 was granted both
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
adults9, Talicia® for the
treatment of Helicobacter pylori (H. pylori) infection in
adults10, and Aemcolo® for
the treatment of travelers' diarrhea in
adults11. 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.
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 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.
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
|
- 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.
- 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.
- 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.
- The American Lung Association, 2020.
- 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.
- Foster|Rosenblatt, 2017.
- 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.
- 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
- Full prescribing information for Movantik®
(naloxegol) is available at: www.Movantik.com.
- Full prescribing information for Talicia®
(omeprazole magnesium, amoxicillin and rifabutin) is available at:
www.Talicia.com.
- Full prescribing information for Aemcolo®
(rifamycin) is available at: www.Aemcolo.com.