Savara has discontinued development of Molgradex inhaled recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) for the treatment of nontuberculous mycobacterial (NTM) lung infection and will not restart the Phase 2a ENCORE study of Molgradex for NTM in cystic fibrosis patients, the company said. Development of Molgradex for the treatment of autoimmune pulmonary alveolar proteinosis (aPAP) will continue.
In March 2020, Savara announced that it had paused the ENCORE study and the Phase 3 AVAIL studies of AeroVanc vancomycin DPI for the treatment of MRSA lung infections in CF patients due to the COVID-19 pandemic. A Phase 2a study of Molgradex for the treatment of NTM in non-CF patients (OPTIMA) was completed in March 2020. The ENCORE study had been initiated in April 2019 and had enrolled 14 out of an expected 30 patients at the time it was stopped in March 2020.
Savara Chief Medical Officer Badrul Chowdhury commented, “Discontinuing the exploratory ENCORE study is very disappointing and I extend our sincere gratitude to the patients who participated, especially during the trying times of this pandemic. Many factors contributed to this decision, most notably the effects of COVID-19, which resulted in small patient numbers and operational disruptions at clinical sites. The approval of the triple-combination CFTR modulator, which transformed the standard-of-care for this patient population, further confounded our ability to evaluate the potential effect of Molgradex in this study as we are unable to differentiate which drug was primarily responsible for the observed culture conversions. While the results of ENCORE are interesting, additional controlled studies would be required to accurately understand the therapeutic potential of Molgradex in combination with the triple-combination modulator treatment.”
In June 2019, Savara announced that the Phase 3 IMPALA study of Molgradex for PAP failed to meet its primary endpoint and several months later, the company said that the FDA had discouraged submission of a BLA for Molgradex for the treatment of PAP at that time. However, the FDA granted Molgradex orphan drug designation for automimmune PAP in December 2019, and the PAP Foundation, a patient advocacy group, supports approval of the drug for aPAP.
Read the Savara press release.
