Savara Reports Publication of Case Reports of Inhaled Granulocyte-Macrophage Colony Stimulating Factor for the Treatment of Nontuberculous Mycobacteria
"NTM and in particular M.abscessus are very difficult to eradicate in patients with chronic lung disease, such as CF, and seriously affect patient morbidity," stated
"We believe the
NTM lung infection is a considerable therapeutic challenge due to the unique ability of these bacteria to evade the normal killing mechanisms of alveolar macrophages, a type of immune cell responsible for killing bacteria in the lungs. Scientific research in various animal models, including GM-CSF knock out mice, have demonstrated that GM-CSF plays an important role as an effector molecule activating macrophages to kill mycobacteria, with or without the simultaneous use of antibiotics. Among the various NTM species, M. abscessus is a particularly challenging clinical problem, being one of the most resistant organisms to antibiotics. Resistance to antibiotics in general is an increasing problem globally, but importantly, GM-CSF is not an antibiotic. Instead, it stimulates the human immune response without targeting the bacteria directly, thus avoiding the problem of inducing antibiotic resistance. The two published clinical case reports suggest that the promising animal data on the antimycobacterial efficacy of GM-CSF may be translatable to humans, and that GM-CSF may enable eradication or reduction of the bacterial burden in patients with refractory M. abscessus or other NTM lung infection.
Savara is currently preparing to initiate a 30-patient, multi-center, open-label Phase 2a clinical trial to investigate the efficacy and safety of Molgradex for the treatment of chronic NTM lung infection. Subjects with either M. abscessus or Mycobacterium avium complex (MAC) infection will be enrolled, with all subjects having either antibiotic refractory infection or intolerance to standard NTM antibiotics. Subjects with CF will not be enrolled. The trial will consist of 24 weeks of Molgradex treatment, followed by a 12-week observational period. The primary endpoint will be sputum culture conversion defined as at least three consecutive negative sputum cultures. Secondary endpoints include other microbiological indicators, exercise capacity, and patient reported outcomes.
About NTM Lung Infection
NTM lung infection is a rare and serious lung disorder associated with increased rates of morbidity and mortality. Nontuberculous mycobacteria are naturally-occurring organisms and NTM lung infection can occur when an individual inhales the organism from their environment and develops a slowly progressive and destructive lung disease. NTM lung infection is typically characterized by cough, fatigue, and weight loss. NTM infection often becomes chronic and requires long courses of multiple antibiotics, and despite the aggressive treatment regimens, treatment failure rates are high, and recurrence of infection common. Chronic NTM lung infection can have a significant impact on quality of life. There are approximately 50,000 to 80,000 individuals affected by NTM lung infection in the U.S, the most common types involving MAC, and M. abscessus. There have been few advancements in new systemic treatments for NTM lung infection. However, in a recent Phase 3 clinical trial by
Molgradex is an inhaled formulation of recombinant human GM-CSF, in Phase 3 development for PAP and in preparation for Phase 2a development for nontuberculous mycobacteria, or NTM, lung infection. Molgradex is delivered via an investigational eFlow® Nebulizer System (
Forward Looking Statements
Savara cautions you that statements in this press release that are not a description of historical fact are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words referencing future events or circumstances such as "expect," "intend," "plan," "anticipate," "believe," and "will," among others. Such statements include, but are not limited to, statements relating to the increasing scientific evidence of GM-CSF's important role in activating macrophages to kill mycobacteria, our belief that the
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