Savara Inc. (NASDAQ: SVRA) shares were absolutely crushed on Thursday after the company released its late-stage results for its treatment of autoimmune pulmonary alveolar proteinosis (aPAP). Unfortunately, the study did not meet its primary endpoint.
This was a Phase 3 clinical study evaluating Molgradex, an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor, for the treatment of aPAP.
Overall, 138 patients were randomized and received treatment for 24 weeks in one of three arms:
- Molgradex 300 µg administered once daily continuously over 24 weeks
- Molgradex 300 µg, and matching placebo, administered once daily in seven-day intermittent cycles of each
- Inhaled placebo administered once daily continuously over 24 weeks
Ultimately, an average alveolar-arterial oxygen gradient (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, the study did not meet its primary endpoint.
Bruce Trapnell, M.D., lead investigator of the Impala study in the United States and professor of Medicine and Pediatrics, University of Cincinnati College of Medicine, commented:
Taken together, the trends in reduction of A-aDO2, as well as the improvement in the diffusion capacity, are very consistent with reduction of the surfactant burden that causes the clinical symptoms of aPAP. Most importantly, the impressive improvement in quality of life, as measured by the SGRQ, suggest that Molgradex not only improved objective measures of oxygenation, but also had a clinically meaningful therapeutic effect. I believe these data demonstrate that Molgradex can become an important pharmacological treatment option for patients with aPAP.
Shares of Savara were last seen down about 76% at $2.56, in a 52-week range of $2.55 to $14.06. The consensus analyst price target of $23.33.