Country for PR: United Kingdom
Contributor: PR Newswire Europe
Saturday, November 21 2020 - 00:20
RedHill Biopharma Initiates Phase 3 Study of RHB-204 for First-Line Treatment of NTM Disease
TEL AVIV, Israel and RALEIGH, N.C., November 20, 2020, /PRNewswire-AsiaNet/--

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

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 resistance[1]," 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, a 
web-based service by the U.S. National Institute of Health, which provides 
public access to information on publicly and privately supported clinical 

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 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 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). 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 
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 

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(R); (v) the Company's ability to 
successfully commercialize and promote Talicia(R), and Aemcolo(R) and 
Movantik(R); (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

Media contact (U.S.):
Bryan Gibbs
Vice President
Finn Partners
+1 212 529 2236 

1. 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, 
2. 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. 
3. Kim RD, et al. Pulmonary Nontuberculous Mycobacterial Disease. Prospective 
Study of a Distinct Preexisting Syndrome Am J Respir Crit Care Med. 2008; 
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(R) (naloxegol) is available at:   
10. Full prescribing information for Talicia(R) (omeprazole magnesium, 
amoxicillin and rifabutin) is available at:        
11. Full prescribing information for Aemcolo(R) (rifamycin) is available at:

SOURCE: RedHill Biopharma Ltd.