RedHill Biopharma Initiates Phase 3 Study of RHB-204 for First-Line Treatment of NTM Disease
Pubblicato il: 20/11/2020 14:01
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/ --
"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
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
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
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:
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