Savara Inc. (NASDAQ: SVRA) has announced positive results from the pivotal phase 3 IMPALA-2 clinical trial for its molgramostim nebulizer solution in treating autoimmune pulmonary alveolar proteinosis (APAP). The trial showed statistically significant improvements in various key endpoints compared to the placebo.
The primary endpoint, the percent predicted diffusing capacity of the lungs for carbon monoxide (DLCO) at week 24, showed a statistically significant improvement versus placebo. This improvement was sustained at week 48, a secondary endpoint, demonstrating the durability of the treatment's effect.
Moreover, the trial also revealed statistically significant improvements in the St. George's Respiratory Questionnaire (SGRQ) total score at week 24, with additional secondary endpoints reaching nominal significance. Notably, 97% of patients completed the double-blind treatment through week 48 with no trial drug related adverse events leading to discontinuation, and 100% of patients completing the 48-week double-blind period elected to participate in the 96-week open-label period.
The company plans to complete the Biologics License Application (BLA) submission in the first half of 2025, reflecting the confidence in the potential of molgramostim as a safe and efficacious treatment option for APAP patients. Molgramostim has also been granted orphan drug, fast track, and breakthrough therapy designation from the U.S. Food and Drug Administration, orphan drug designation from the European Medicines Agency, and innovative passport and promising innovative medicine designation from the UK’s Medicines and Healthcare products Regulatory Agency for the treatment of APAP.
The trial results were well received by the company's leadership, with Matt Pauls, the Chair and CEO of Savara, expressing that the results not only met but exceeded their expectations. He also highlighted the potential positioning of molgramostim to be the first and only approved therapeutic for APAP in the U.S. and Europe.
The IMPALA-2 trial, a global, pivotal, phase 3, 48-week, randomized, double-blind, placebo-controlled clinical trial, was conducted at 43 clinical trial sites across 16 countries.
This positive outcome in the pivotal phase 3 trial marks a significant milestone for Savara Inc. and the APAP community, potentially offering a new treatment option for a rare respiratory disease with high unmet medical need. Today the company's shares have moved 0.9% to a price of $3.83. If you want to know more, read the company's complete 8-K report here.
