Savara Inc (SVRA) 2025 Conference Transcript

Savara Inc (SVRA) 2025 Conference Summary Company Overview - Savara Inc is a rare orphan respiratory pulmonology-focused company with a single asset, a novel inhaled biologic called molgermostim inhalation solution [4][5] - The company is actively pursuing FDA approval for autoimmune pulmonary alveolar proteinosis (aPAP) and plans to file for MAA in Europe and the UK by the end of the year [5][11] Disease Background - Autoimmune PAP is a chronic rare autoimmune disease characterized by the development of autoantibodies to GM-CSF, leading to impaired gas exchange in the lungs [8][10] - The disease manifests with symptoms such as cough, shortness of breath, and fatigue [10] Patient Population - Estimated prevalence of aPAP ranges from 6-26 per million, with approximately 3,600 diagnosed patients in the U.S. [11][12] - The company believes there are many undiagnosed patients, indicating a robust addressable market for commercialization [12][13] Clinical Program and Results - The IMPALA 2 trial is the largest and longest randomized clinical trial for aPAP, enrolling 64 patients across 43 centers in 16 countries [22] - Primary endpoint was the change in DLCO (diffusion capacity of the lung for carbon monoxide) at 24 weeks, which was statistically significant [23][24] - The trial also showed significant clinical benefits, including improvements in respiratory quality of life and exercise capacity [26][28] Regulatory Process - Savara completed a rolling BLA submission in March, with a 60-day review period expected to conclude in May [32] - The company anticipates priority review due to breakthrough designation, with a potential PDUFA date in November [32][33] Market Preparation and Payer Research - Current treatments for aPAP include lung lavage, which is a rescue procedure, and off-label drugs [40] - Savara is guiding a price range of $300,000 to$500,000 per patient per year, with 87% of payers indicating they would cover it within this range [42] - The company expects 50-70% commercial coverage for patients affected by aPAP [43] Launch Strategy - Savara is implementing a dry blood spot antibody testing program and has initiated a pilot clinic at the University of Florida to identify undiagnosed patients [46][48] - The company aims to educate pulmonologists about aPAP and the availability of testing to facilitate earlier diagnosis [49] Financial Position - As of the last report, Savara has $196 million in cash and has structured a non-dilutive financing option of up to$170 million [50] - The current cash position is expected to last through the second quarter of 2027, with various options available for additional funding [51] Key Takeaways - Savara is on track for potential FDA approval of molgermostim for aPAP, with significant clinical trial success and a robust market strategy [53] - The company is preparing for a launch that could significantly impact the treatment landscape for aPAP, a rare and underserved patient population [53]