Summary of Savara's Conference Call Company Overview - **Company**: Savara - **Focus**: Orphan-rare disease company with a single late-stage product, Mobrevi, a novel inhaled biologic for autoimmune pulmonary alveolar proteinosis (autoimmune PAP) [2][3] Industry Context - **Disease**: Autoimmune PAP is a severe, rare lung disease with no approved therapeutic options in the U.S., Europe, or the U.K. [3] - **Current Treatment**: Patients undergo a lengthy diagnostic journey, often requiring a lung lavage procedure, which is not a sustainable long-term solution [5][6][7] Product Details - **Product**: Mobrevi (molgramostim inhalation solution) - **Administration**: Delivered via nebulization, once daily at 300 micrograms using a proprietary eFlow nebulizer system [10] - **Clinical Trials**: - BLA submitted in December based on positive results from the IMPALA-2 trial, which enrolled 164 subjects [11][15] - Primary endpoint (DLCO) showed statistically significant improvement at 24 weeks compared to placebo [12] - Secondary endpoints included SGRQ and exercise capacity, also showing significant improvement [12][13] Market Opportunity - **Patient Population**: Estimated 5,500 diagnosed autoimmune PAP patients in the U.S., equating to approximately 16 patients per million [17][31] - **Market Dynamics**: The U.S. PAP market is concentrated, with the top 500 accounts managing about 65% of identified patients [17] - **Pricing Strategy**: Anticipated pricing corridor of $400,000-$500,000 per patient per year, with expected coverage under typical prior authorization criteria [22][35] Regulatory and Commercial Strategy - **Regulatory Designations**: Mobrevi has received breakthrough designation in the U.S. and orphan drug designation in Europe, providing 12 and 10 years of exclusivity, respectively [16] - **Commercial Preparation**: Active recruitment of a market development team to prepare for potential approval and launch later this year [18][20] - **Diagnostic Test**: Development of a free blood test for diagnosing autoimmune PAP, with 100% sensitivity and specificity [19] Financial Position - **Capitalization**: Company is well-capitalized with approximately $264 million in cash [24] - **Investment Thesis**: Significant unmet need in the market, with potential for blockbuster revenue given the pricing and patient population [24] Additional Insights - **Patient Experience**: The journey to diagnosis can take years, with significant physical and emotional impacts on patients [9] - **Commercial Strategy**: Focus on both moderate and severe patients, emphasizing the importance of early intervention [32] Conclusion - Savara is positioned to address a significant unmet need in the treatment of autoimmune PAP with Mobrevi, supported by strong clinical data and a robust commercial strategy aimed at improving patient outcomes and accelerating diagnosis [24][36]