RHB-204 (Nontuberculous Mycobacteria (NTM) Infections)

RHB-204 is a proprietary and potentially groundbreaking 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. NTM infections have been increasing worldwide over the past two decades1. Pulmonary manifestations account for 80–90% of all NTM-associated diseases2, with approximately 80% of pulmonary NTM infections in the U.S. associated with mycobacterium avium complex (MAC)1. Pulmonary NTM disease symptoms can include fever, weight loss, chronic or recurring cough, chest pain, blood in sputum and fatigue1. Treatment of NTM infection is difficult, requiring multiple antibiotics and an extended treatment course due to the risk of development of resistance5. As a result, new antimicrobial agents for NTM are urgently needed3.

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 the 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) infection.

 Griffith, David E., et al. "An official ATS/IDSA statement: diagnosis, treatment, and prevention of nontuberculous mycobacterial diseases." American journal of respiratory and critical care medicine 175.4 (2007): 367-416.

2 Wassilew, Nasstasja, et al. "Pulmonary disease caused by non-tuberculous mycobacteria." Respiration 91.5 (2016): 386-402.

3 Martiniano, Stacey L., et al. "Safety and Effectiveness of Clofazimine for Primary and Refractory Nontuberculous Mycobacterial Infection." Chest (2017).