Savara shares are crushed as PhIII trial flunks primary, key secondaries — but they can’t stop believing
Investors are in no mood to hear biotechs tout the success of a “key” secondary endpoint when the pivotal Phase III flunks the primary goal. Just ask Savara.
The Texas biotech $SVRA went looking for a silver lining as company execs bluntly conceded that Molgradex, an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF), failed to spur significantly improved treatment outcomes for patients with a rare respiratory disease called autoimmune pulmonary alveolar proteinosis, or aPAP.
The p-value was an awful 0.17.
Researchers blamed a high placebo effect, teasing out the positive aspects of an improved numerical outcome for secondaries on the 6-minute walk test and a need for whole lung lavage — a procedure used to clean the lungs of patients. Those secondaries also failed the statistical analysis.
Bruce Trapnell Cincinnati Children’s But that didn’t stop Savara from suggesting that a successful score on a patient-reported quality of life assessment/outcomes test could help set the stage for an approval.
“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,” said Bruce Trapnell, the lead investigator. “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.”
Investors weren’t buying it, though. The stock cratered on the numbers, plunging more than 70% after the bell on Wednesday.
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