Summary of Savara FY Conference Call - November 10, 2025 Company Overview - **Company**: Savara (NasdaqGS:SVRA) - **Focus**: Single asset rare disease company - **Product**: Mulibrev (molgramostim inhalation solution) - **Indication**: Autoimmune pulmonary alveolar proteinosis (aPAP), a rare lung disease - **Regulatory Status**: Resubmission of Biologics License Application (BLA) projected for December 2025, with Marketing Authorization Applications (MAAs) planned for Europe and the U.K. in Q1 2026 [4][6][37] Key Points and Arguments Regulatory Developments - **BLA Submission**: Initial rolling submission began in December 2024, finalized in March 2025, but received a refusal to file (RTF) from the FDA in May 2025 due to manufacturing data issues [7][8] - **Manufacturing Strategy**: Transitioning to Fujifilm in the U.K. as the primary drug substance manufacturer following the RTF, supported by a Type A meeting with the FDA [8][9] - **IMPALA-2 Trial**: First and only application for aPAP, with breakthrough designation highlighting significant unmet need. Primary endpoint (DLCO) met statistical significance at 24 weeks, with durability observed at 48 weeks [12][14][16] Clinical Trial Insights - **Trial Design**: Global study with 164 patients across 43 centers in 16 countries. Key secondary endpoints included SGRQ and exercise capacity via treadmill test, both showing significant results [15][16][18] - **Patient Retention**: 100% of patients completed the trial and transitioned to open-label extension, indicating strong engagement and treatment interest [18] Market Opportunity - **Current Management of aPAP**: Existing treatment involves whole lung lavage, a mechanical procedure that does not address the underlying disease. Mulibrev targets the root cause by addressing GM-CSF signaling [21][22] - **Market Size Update**: Recent analysis identified approximately 5,500 patients with autoimmune PAP in the U.S., a 50% increase from previous estimates of 3,600 patients [23][25] - **Physician Distribution**: Approximately 1,100 centers manage these patients, with a customer-facing group of 25-30 expected to effectively address the patient population [26][27] Commercial Strategy - **U.S. Strategy**: Plans to go it alone in the U.S. market, with a focus on building disease awareness and diagnostic testing capabilities [28][29] - **International Strategy**: Similar approach in Europe and the U.K., while Asia Pacific presents challenges due to policy issues [29][30] - **Financing**: Recent two-step financing strategy was opportunistic, aimed at ensuring sufficient runway for operations and market entry [31][33] Future Outlook - **Regulatory Timeline**: Anticipated PDUFA date around August 2026, assuming priority review is granted [40] - **Market Readiness**: Company is well-capitalized and focused on execution to meet market expectations [36][37] Additional Important Insights - **Antibody Testing Program**: Ongoing no-cost antibody testing program is being utilized, with potential for future updates on testing numbers and market expansion [35][36] - **Unmet Need**: The significant unmet need in the aPAP market underscores the potential impact of Mulibrev as a treatment option [36]