Savara announces results of IMPALA, a phase III study of Molgradex for the treatment of autoimmune alveolar pulmonary proteinosis

Savara Inc.an orphan lung disease company, announced top line data from IMPALA, a pivotal Phase III clinical study evaluating Molgradex, an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF), for the treatment of aPAP (Autoimmune Alveolar Pulmonary Proteinosis ).

138 patients were randomized and received treatment for 24 weeks in one of three arms: 1) Molgradex 300 µg administered once daily continuously over 24 weeks, 2) Molgradex 300 µg, and matching placebo, administered once daily in 7-day intermittent cycles of each, or 3) inhaled placebo administered once daily continuously over 24 weeks. Primary Endpoint in the Intention-to-Treat (ITT) Population: An average A-aDO2 improvement of 12.1 mmHg was observed in the continuous dosing arm, compared to an average A-aDO2 improvement of 8.8 mmHg in the placebo arm. With an estimated 4.6 mmHg treatment difference (p=0.17), the study did not meet its primary endpoint .