Savara (SVRA) Oppenheimer 35th Annual Healthcare Life Sciences Conference 2025 summary

Company overview and strategic focus

• Focused exclusively on rare respiratory diseases, with Molbreevi as the lead asset targeting APAP.

• Well-funded with $219 million in cash and projected runway through 2027.

• Preparing for BLA filing in the US by end of Q1 and MAA filing in Europe by year-end.

• APAP is an ultra-rare, underdiagnosed lung disease with no approved therapies in the US or Europe.

Disease background and current treatment landscape

• APAP is a rare autoimmune lung disease, representing 90% of PAP cases, typically affecting adults aged 30–50.

• Symptoms include shortness of breath, cough, and fatigue, with diagnosis often delayed by 18 months.

• Current standard of care is whole lung lavage, a complex, non-standardized, and temporary rescue procedure.

• Whole lung lavage is only available at specialized centers and often requires repeat procedures.

Molbreevi mechanism, clinical data, and regulatory path

• Molbreevi is an inhaled recombinant GM-CSF designed to restore surfactant metabolism in APAP.

• IMPALA-2, the largest APAP trial, met its primary endpoint (DLCO improvement at 24 weeks) and showed durable effects at 48 weeks.

• Secondary endpoints, including quality of life and exercise capacity, showed positive trends.

• IMPALA-1 provides supportive data for regulatory submissions.

• BLA submission expected by end of Q1, with potential PDUFA date at end of November or early December if priority review is granted.