Savara announces interim results from Phase 2a OPTIMA study
Savara announced interim results from OPTIMA, a Phase 2a clinical study evaluating its lead product candidate Molgradex, an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor, or GM-CSF, for the treatment of nontuberculous mycobacterial, or NTM, lung infection. The ongoing study is evaluating treatment of both Mycobacterium avium complex, or MAC, infection and the more difficult-to-treat Mycobacterium abscessus, or MABSC, infection. Savara believes microbiological data from this early analysis demonstrate an encouraging efficacy signal, with a favorable safety profile. The interim analysis focused on efficacy, as assessed by microbiological results, in 14 patients who completed the 24-week treatment period and had culture results available up to at least the 16-week timepoint. Ten of the evaluable patients have MAC infection and four have MABSC infection. Of the patients with MAC infection, eight are in treatment Group 1 and two are in treatment Group 2. The four evaluable MABSC patients are evenly split between both treatment groups. Safety and tolerability was assessed for all 32 patients enrolled in the study. The data show that among the 10 patients with MAC infection, four experienced a consistent sputum smear conversion to negative by week 24, and three experienced a negative sputum culture at weeks 16 and 20, with culture results pending for the week 24 timepoint. Sputum smear or sputum culture conversions were not observed in the four patients with MABSC infection. Due to the generally slow growth of nontuberculous mycobacteria on culture media, a sputum sample can be determined negative only after eight weeks of observation, causing a corresponding lag time in the assessment of the culture data. Safety was assessed in the total study population. Among the 32 patients, six experienced serious adverse events, or SAEs, including one patient who died by suicide. The death was considered unrelated to treatment. The majority of SAEs consisted of hospitalizations due to pulmonary exacerbations or worsening of NTM infection, of which one was considered possibly treatment-related. GM-CSF was generally well tolerated, with nine patients reporting mostly mild, potentially treatment-related respiratory adverse events. Respiratory adverse events were defined as shortness of breath, chest tightness or wheeze. A total of three patients discontinued treatment due to adverse events. Based on the microbiological data and safety profile, which provides the basis to continue treating patients for a longer period of time, the duration of the OPTIMA study is extended from 24 to 48 weeks. This increases the ability to observe a more robust anti-infective effect, including culture conversions. Final results from OPTIMA are now expected by Q1 of 2020.