RHB-204 (Nontuberculous Mycobacteria (NTM) Infections)

RHB-204 is a proprietary and innovative investigational drug in oral capsule formulation, with potent intracellular, antimycobacterial and anti-inflammatory properties.

Nontuberculous mycobacteria (NTM) are ubiquitous environmental bacteria, found in the soil as well as natural and engineered water systems. Studies from North America, Europe, and Asia have all shown increasing NTM disease incidence over the last two decades1. Pulmonary manifestations account for 80–90% of all NTM-associated diseases, with up to 91% of pulmonary NTM infections in the South and Northeast regions of the U.S. associated with Mycobacterium avium complex (MAC)2. The most common pulmonary NTM disease symptoms are chronic or recurring cough and fatigue. Other symptoms may include fever, weight loss, chest pain and blood in sputum3. Treatment of NTM infection can be difficult, requiring multiple antibiotics and an extended treatment course due to the risk of development of resistance4. There is also no FDA approved therapy for first-line treatment of NTM. As a result, new and effective antimicrobial agents for NTM are urgently needed, particularly for first-line therapy.

RHB-204 was granted Qualified Infectious Disease Product (QIDP) designation by the FDA for the treatment of NTM Infections.

The QIDP designation was granted under the FDA's Generating Antibiotic Incentives Now (GAIN) Act, which is intended to encourage development of new antibiotic drugs for the treatment of serious or life-threatening infections that have the potential to pose a serious threat to public health.

RedHill is planning, subject to regulatory approvals, to initiate a pivotal Phase 3 study with RHB-204 for a first-line, stand-alone treatment of pulmonary NTM infections caused by Mycobacterium avium Complex (MAC).

The study is intended to assess the efficacy and safety of RHB-204 as a first-line treatment of pulmonary NTM disease caused by Mycobacterium avium complex (MAC).




1 Ratnatunga CN, Lutzky VP, Kupz A, Doolan DL, Reid DW, Field M, Bell SC, Thomson RM and Miles JJ (2020) The Rise of Non-Tuberculosis Mycobacterial Lung Disease. Front. Immunol. 11:303. doi: 10.3389/fimmu.2020.00303.

2 Spaulding AB, Lai YL, Zelazny AM, et al.: Geographic Distribution of Nontuberculous Mycobacterial Species Identified among Clinical Isolates in the United States, 2009-2013. Ann Am Thorac Soc. 2017; 14(11): 1655–61.

3 Kim RD et al Pulmonary Nontuberculous Mycobacterial Disease. Prospective Study of a Distinct Preexisting Syndrome Am J Respir Crit Care Med Vol 178. pp 1066–1074, 2008.

4 Daley CL, et al Treatment of Nontuberculous Mycobacterial Pulmonary Disease: An Official ATS/ERS/ESCMID/IDSA Clinical Practice Guideline: Executive Summary. Infectious Diseases Society of America. July 6, 2020.