AsiaNet  86781

TEL AVIV, Israel and RALEIGH, N.C., November 20, 2020, /PRNewswire=KYODO JBN/--

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. [https://www.redhillbio.com/RedHill/  ] (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 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 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

2041.  

About RedHill Biopharma    

RedHill Biopharma Ltd. (Nasdaq: RDHL)

[https://finance.yahoo.com/quote/RDHL?p=RDHL&.tsrc=fin-srch ] 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(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

+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

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,

https://doi.org/10.1093/cid/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;

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(R) (naloxegol) is available at:

www.Movantik.com.  

10. Full prescribing information for Talicia(R) (omeprazole magnesium,

amoxicillin and rifabutin) is available at: www.Talicia.com.        

11. Full prescribing information for Aemcolo(R) (rifamycin) is available at:

www.Aemcolo.com.

SOURCE: RedHill Biopharma Ltd.