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Wednesday, January 06 2021 - 23:44
RedHill Biopharma's RHB-204 Granted FDA Fast Track Designation for NTM Disease
TEL AVIV, Israel and RALEIGH, NC, Jan. 6, 2021 /PRNewswire-AsiaNet / --

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   

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 

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 

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: 
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 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 / 

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.


[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,

[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® (naloxegol) is available at:   

[10] Full prescribing information for Talicia® (omeprazole magnesium, 
amoxicillin and rifabutin) is available at:       

[11] Full prescribing information for Aemcolo® (rifamycin) is available at:


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

SOURCE: RedHill Biopharma