Savara, which just announced the discontinuation of its Aironite development program after the failure of a Phase 2 trial to meet its endpoint, says that it has initiated a Phase 2a clinical study of Molgradex inhaled recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) for the treatment of nontuberculous mycobacterial (NTM) lung infection.
The company announced in October 2017 that it intended to develop Molgradex for NTM lung infections in addition to developing Molgradex for autoimmune pulmonary alveolar proteinosis (PAP). The OPTIMA study is now getting underway while the Phase 3 IMPALA study of Molgradex for the treatment of PAP continues.
The primary endpoint for the open-label, non-controlled OPTIMA study will be sputum culture conversion, when the patient has at least 3 sputum samples in a row with no NTM growth. The study is expected to enroll 30 patients with chronic M. abscessus or Mycobacterium avium complex lung infections.
Savara CEO Rob Neville said, “The initiation of the OPTIMA study is a major milestone for Savara as we begin to explore the potential of Molgradex as a novel therapeutic approach for NTM infection. We believe that Molgradex may significantly improve patient outcomes by stimulating the innate immune system in the lungs, as compared with targeting bacteria directly, thereby avoiding problems of antibiotic resistance and antibiotic intolerance. We believe Molgradex will be eligible for Orphan Status as well as Qualified Infectious Disease Product Status, and if the results of the OPTIMA study meet our expectations, the product may also qualify for Breakthrough Therapy Designation.”
Read the Savara press release.
