Savara's inhaled molgramostim wins crucial study for rare lung disease

Savara recently announced that their Phase III clinical trial of molgramostim, targeting adults with autoimmune pulmonary alveolar proteinosis (aPAP), successfully met its primary endpoint. This achievement was highlighted by CEO Matt Pauls, who stated that the outcomes "not only met, but exceeded, our expectations." Pauls further emphasized that the data gathered from this study could potentially make molgramostim the first and only approved therapy for aPAP in both the United States and Europe.

The pivotal IMPALA-2 trial involved 164 participants suffering from aPAP. These individuals were randomly assigned to receive either molgramostim through daily inhalation or a matching placebo. The study focused on the primary endpoint of the change in percent predicted diffusing capacity of the lungs for carbon monoxide (DLCO), measured from the baseline to week 24. Additionally, secondary endpoints included changes in percent predicted DLCO from baseline to week 48.

The results of the study were promising. The difference in treatment effects between molgramostim, an inhaled form of recombinant human GM-CSF, and placebo showed a statistically significant improvement in the mean change from baseline to week 24 in haemoglobin-adjusted percent predicted DLCO. Furthermore, this improvement was sustained through week 48.

Regarding the safety profile of molgramostim, the drug was well tolerated by participants throughout the trial. The incidence of adverse events (AEs) was comparable between the molgramostim and placebo groups. Notably, two participants discontinued molgramostim treatment due to adverse events, but these incidents were not considered related to the medication itself.

This trial's success positions molgramostim as a potential groundbreaking treatment for patients with aPAP, addressing a significant unmet medical need. The positive outcomes from the IMPALA-2 trial not only pave the way for regulatory approval but also offer hope to patients suffering from this rare lung disease.