Phase 3 RHB-204 Trial Begins for NTM Lung Disease

On November 20, 2020, biopharmaceutical company RedHill Biopharma Ltd. announced the initiation of a Phase 3 clinical trial to study the orally administered RHB-204. The drug is designed to treat patients with nontuberculous mycobacteria (NTM) lung disease caused by Mycobacterium avium Complex (MAC). Currently, there are no first-line therapies for this condition.


125 patients will enroll in the Phase 3 trial to evaluate the safety, efficacy, and tolerability of RHB-204. Altogether, this double-blind trial will take place at up to 40 sites around America. Participants will receive either RHB-204 or a placebo. Although some patients with NTM lung disease are asymptomatic, this trial will only utilize symptomatic patients. Endpoints include:

  • The sustainability of clinical benefit
  • The durability of microbiological response
  • Sputum culture conversion (after 6 months)
  • Patient-reported data into their fatigue, respiratory function, and physical abilities

Developing an effective first-line treatment for NTM lung disease is crucial. Typically, treatment centers around long-term antibiotics. However, since NTM lung disease is also treatment-averse, and fails to respond to many treatments, patients are in severe need of a new option. Could that be RHB-204?

This drug, which received QIDP and Orphan Drug designations, is a proprietary, orally-administered therapy which combines clofazimine, rifabutin, and clarithromycin to treat NTM lung disease. Currently, if the FDA approves this treatment, RedHill BioPharma is looking at up to 12 years of market exclusivity. The drug is also patent-protected until 2029 and potentially until 2041.

Nontuberculous Mycobacteria (NTM) Lung Disease

Nature is beautiful – but sometimes it can hold bacteria or viruses that make us sick. In the case of nontuberculous mycobacteria (NTM) lung disease, the mycobacteria which cause the illness are naturally found in water and soil. When mycobacteria get inside of the lungs, they infect the airways and tissue. Ultimately, this causes a chronic lung infection characterized by pulmonary inflammation. However, not everyone exposed to mycobacteria get sick. Risk factors include cystic fibrosis, a prior tuberculosis diagnosis, lowered immune systems, and COPD (among others). Generally, NTM lung disease impacts older individuals more than younger individuals.

While some patients are asymptomatic, others will show symptoms. These include:

  • Unintended weight loss
  • Coughing up blood or bloody mucus
  • Excess mucus
  • A severe and chronic cough
  • Shortness of breath / difficulty breathing
  • Night sweats
  • Intense fatigue
  • Fever
  • Chest pain
  • Recurrent respiratory infections
  • Respiratory failure

Learn more about NTM lung disease.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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