Shares of the thinly traded small-cap biotech Savara Inc SVRA were plunging to a record low Thursday in the wake of a clinical trial readout from the company.
Austin, Texas-based Savara, which specializes in therapies for orphan lung diseases, said Wednesday that the Phase 3 IMPALA study evaluating Molgradex as a treatment for autoimmune alveolar pulmonary proteinosis, or aPAP, did not meet the primary endpoint.
The goal was to demonstrate a statistically significant difference in the alveolar-arterial oxygen gradient, or A-aDO2, versus a placebo.
An average improvement of 12.1 mmHg was observed in the continuous dosing arm compared to an 8.8-mmHg improvement in the placebo arm, with the estimated 4.6-mmHg difference not considered statistically significant.
Molgradex is an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor. The study tested 138 patients, who were randomized to receive either 300 micrograms of Molgradex once daily continuously over 24 weeks; 300 micrograms of Molgradex 300 microgram and matching placebo once daily in seven-day intermittent cycles of each; or an inhaled placebo once daily continuously over 24 weeks.
The investigational therapy met one of the three secondary endpoints by showing a statistically significant difference in the St. George Respiratory Questionnaire, according to Savara.
The number of treatment emergent adverse events, including serious adverse events, occurred with a similar frequency and severity in the Molgradex treatment arm as with the placebo.
Savara said it plans to meet with the U.S. FDA and European Medicines Agency to discuss the data and the path forward.
The company also said it plans to submit the full data set from the IMPALA study to a peer-reviewed scientific journal or to an upcoming medical meeting for consideration.
Savara shares were down 75.59% at $2.58 at the time of publication Thursday.
Photo courtesy of Savara.
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