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TEL AVIV, Israel and RALEIGH, NC, Jan. 6, 2021 /PRNewswire=KYODO JBN / --

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. [https://www.redhillbio.com/RedHill/] (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: https://mma.prnewswire.com/media/1334141/RedHill_Biopharma_Logo.jpg  

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

SOURCE: RedHill Biopharma