Summary of Savara Management Conference Call Company Overview - Savara is a single-asset company focused on orphan rare diseases, specifically in the rare lung disease space with an inhaled biologic called Molbrivi [2][3] - The company has positive Phase III data for autoimmune pulmonary alveolar proteinosis (autoimmune PAP) and plans to submit a Biologics License Application (BLA) this month, with Marketing Authorization Applications (MAAs) in Europe and the U.K. by the end of Q1 [2][3] Indication and Treatment - Autoimmune PAP is characterized by symptoms such as shortness of breath, cough, and fatigue, with patients typically undergoing a procedure called whole lung lavage, which is a complex and rarely performed procedure [4][7] - Current treatment options are limited, with steroids being ineffective and Rituximab used off-label without proven efficacy [9][10] Patient Population and Prevalence - The estimated prevalence of autoimmune PAP ranges from 6 to 26 per million, with a more robust U.S. claims database analysis suggesting approximately 5,500 patients diagnosed with PAP [12][13][15] - There is a small patient registry with about 150 patients, primarily adults [11] Clinical Data and Efficacy - The primary endpoint in clinical trials is the Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO), which is a surrogate marker for gas transfer [34] - The trial showed a significant difference in DLCO change from baseline, with a 9.8 percentage point improvement, which resonates with physicians despite not having a defined minimal clinically important difference (MCID) for autoimmune PAP [39][42] - Secondary endpoints, such as the St. George's Respiratory Questionnaire (SGRQ), also showed significant benefits [42] Pricing Strategy - Preliminary pricing analysis suggests a range of $400,000 to $500,000 per patient per year, with potential for pricing power based on the clinical benefits demonstrated [33] Safety and Tolerability - The trial reported no significant safety signals, and patient feedback indicated a positive response to the treatment, with all trial completers opting for an open-label extension [43][44] Manufacturing and Quality Control - The company is aligned with Fujifilm as its primary drug substance partner and is resubmitting documentation this month [50][51] Key Takeaways - Savara is positioned to address a significant unmet need in the treatment of autoimmune PAP with its inhaled biologic, Molbrivi - The company is actively working on regulatory submissions and has demonstrated promising clinical data that may support its market entry - The pricing strategy reflects the rarity of the condition and the potential for substantial clinical benefits, which could facilitate payer acceptance and coverage [32][33]