In Hindsight, Savara Wishes It Led With Patient-Reported Secondary Endpoint For Molgradex

It’s fairly standard for a biotech to frame a study that missed its primary endpoint as offering promise on other measures, but Savara Inc.’s attempt to depict its unsuccessful Phase III IMPALA study of Molgradex in autoimmune alveolar pulmonary proteinosis (aPAP) as a “successful failed study” is not resonating with investors.

The Austin, TX-based biotech reported after market closing on 12 June that Molgradex – an inhaled recombinant formulation of human granulocyte colony-stimulating factor (GM-CSF) – missed the primary endpoint in a 138-patient, placebo-controlled pivotal study. The trial did not hit a primary endpoint of improvement from baseline in alveolar-arterial oxygen gradient (A-aDO2), an outcome it attributed mainly to a strong placebo effect