Savara announces new data from Pivotal Phase 3 IMPALA-2 trial

Savara announced that new data from the Phase 3 IMPALA-2 clinical trial of molgramostim in patients with autoimmune Pulmonary Alveolar Proteinosis were presented at the European Respiratory Society Congress 2024 in Vienna, Austria. As previously announced, the IMPALA-2 trial met its primary endpoint, achieving statistical significance in change from baseline in hemoglobin-adjusted percent predicted diffusing capacity of the lungs for carbon monoxide at Week 24. This statistically significant treatment difference was sustained through Week 48, a secondary endpoint, which demonstrated durability of effect. The treatment difference between molgramostim and placebo for mean change from baseline to Week 24 in SGRQ Total Score, a secondary endpoint, achieved statistical significance. Two additional secondary endpoints reached nominal significance: SGRQ Activity Score at Week 24 and exercise capacity using a treadmill test at Week 48. Molgramostim was well tolerated and demonstrated a favorable benefit/risk profile in the IMPALA-2 trial. In addition, 97% of patients completed the 48-week double-blind treatment period, with only 2 patients discontinuing molgramostim due to adverse events which were assessed by the investigator as not related to study treatment. One hundred percent of the patients who completed the double-blind period elected to participate in the 96-week open-label period. The mean change from baseline in disease severity score, which reflects symptoms and arterial partial pressure of oxygen, was significantly more improved at Weeks 24 and 48 with molgramostim compared with placebo. Results from a DLCO% responder analysis demonstrated significantly higher proportions of responders with molgramostim compared with placebo at Weeks 24 and 48. Odds ratios were determined for patients achieving greater than or equal to 5, 7, and 10 percentage point improvements in DLCO% from baseline. Results from an SGRQ Total Score responder analysis showed that numerically and significantly higher proportions of patients achieved each responder threshold of greater than or equal to 4-, 8-, and 12-point improvements with molgramostim compared to placebo. GGO score, a radiological measure of surfactant burden, was significantly improved with molgramostim compared with placebo at Week 24. Savara plans to complete submission of the Biologics License Application for molgramostim in aPAP in the first half of 2025. Molgramostim has been granted Orphan Drug, Fast Track, and Breakthrough Therapy designations 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.