Savara Inc (SVRA) 2025 Conference Transcript

Savara Inc (SVRA) 2025 Conference Summary Company Overview - Savara Inc is a biotech company focused on orphan rare diseases, specifically in the rare pulmonary space with a single asset, Molgrimostum inhalation solution, branded as Molbrevi [6][7] Key Points and Arguments Clinical Development - Positive phase three data for IMPALA two trial was reported, involving 164 patients with autoimmune pulmonary alveolar proteinosis (APAP) [7] - The trial showed positive results on the primary endpoint of DLCO and all key secondary endpoints at 24 and 48 weeks [7] - A Biologics License Application (BLA) resubmission is planned for Q4 2025, with a potential PDUFA date in mid-2026 [8][48] Market Opportunity - Estimated 3,600 diagnosed APAP patients in the U.S., with a potential market size of approximately 7,300 when including undiagnosed patients [10][12] - Pricing research indicates a price range of $300,000 to $500,000 per patient per year, suggesting significant pricing power [13] Treatment Insights - Molbrevi is expected to be a first-line chronic therapy for APAP, addressing the pathophysiology of the disease [14][16] - The drug is administered once daily using a proprietary nebulizer, contrasting with alternatives that require twice-daily administration [50] Regulatory Challenges - The recent refusal to file (RTF) was due to the need for additional Chemistry, Manufacturing, and Controls (CMC) data, not related to clinical trials or safety concerns [21][22] - The company is working with two drug substance partners, GEMA and Fujifilm, to ensure compliance and readiness for the BLA resubmission [31][39] Future Plans - A type A meeting with the FDA is scheduled within 30 days of the RTF, with meeting minutes expected within 90 days [30][31] - European and UK regulatory filings are on track for year-end 2025 [46] Additional Important Information - The company is preparing for an Advisory Committee (AdCom) meeting, considering it the first therapeutic for this orphan rare disease [45] - There is an ongoing Early Access Program (EAP) for patients awaiting approval of Molbrevi [57] - Approximately 10% of patients in previous trials underwent lung lavages, indicating a need for chronic therapeutic options [60] - The company is scaling up its ILD clinic pilot to identify undiagnosed APAP patients using an antibody test [61] This summary encapsulates the critical insights and developments regarding Savara Inc and its lead product, Molbrevi, as discussed in the conference.