Savara has announced the initiation of the Phase 2a ENCORE study of its Molgradex inhaled recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) for the treatment of nontuberculous mycobacterial (NTM) lung infection in cystic fibrosis patients. The company is also developing Molgradex for the treatment of NTM lung infections in non-CF patients and for autoimmune pulmonary alveolar proteinosis (aPAP).
In May 2018, the company provided an update on 2 CF patients who had received Molgradex treatment for NTM lung infections at the Mayo Clinic, and whose cases had been reported in the European Respiratory Journal, noting that both had achieved culture conversion, and the second patient had not received antibiotic treatment.
The ENCORE trial is expected to enroll approximately 30 adult CF patients who have chronic Mycobacterium avium complex (MAC) or Mycobacterium abscessus lung infections that cannot be treated with antibiotics. Patients in the open-label, non-controlled study will receive 300 µg of Molgradex once a day for 48 weeks, followed by 24 weeks of follow up.
Savara CEO Rob Neville said, “People living with CF who have chronic NTM lung infection are in need of improved treatment options to complement current antibiotic regimens. By stimulating the lung’s immune system, we expect Molgradex to enhance the body’s natural ability to fight infection. Somewhat analogous to oncology immunotherapies, we believe anti-infective immunotherapy has the potential to transform the way many refractory lung infections are treated.”
Read the Savara press release.
