This condition can severely impact the quality of life of patients. It is clearly evident that we need a more effective treatment option for NTM, and we’re slowly but surely making progress. Savara Inc. has just released positive interim results from a Phase 2a study, bringing hope of an effective therapy for this condition.

Savara Inc. is examining the effect of Molgradex on two different forms of NTM. Molgradex is a formulation of granulocyte-macrophage colony stimulating factor, or GM-CSF. It is administered by inhalation and it basically works to stimulate the lung’s immune system. This particular study was focused on the potential efficacy of the treatment. The first type of NTM being examined is called Mycobacterium avium complex (MAC) and the second is called Mycobacterium abscessus (MABSC).

The trial included a total of 32 patients, age 18 and older who were examined for 24 weeks. All participants had experienced intolerance to the typical antibiotic treatment given for the condition. The primary endpoint was sputum culture conversion which is confirmed when 3 samples are determined to be clear of nontuberculous mycobacteria.

10 patients in the study had the MAC infection while four had the MABSC infection (which is generally more difficult to treat). Of the 10 with MAC, 4 had a negative sputum culture conversion by the 24th week. 3 had a negative culture at week 16 and week 20. The patients with MABSC did not experience any sputum culture conversions.

Researchers are encouraged by these first signs of efficacy. Additionally, the safety and tolerability of the drug look promising. In terms of safety, among all 32 patients in the study, 6 had serious adverse events and 9 patients experienced mild adverse events such as shortness of breath. 3 patients did discontinue treatment due to their experience with adverse events.

Further investigation will determine whether these positive responses will be sustained over time. But currently, the improvement of symptoms in patients is extremely promising. The trial has now been extended to 48 weeks in order to get a better read on the drug’s effects. There is a lot more to examine when it comes to Molgradex, but we can look forward to final results from this study within the first few months of 2020.