Savara (SVRA) investor relations material

Company overview and disease background

• Focused on building an experienced orphan-rare disease company with a single late-stage asset, MOLBREEVI, for autoimmune PAP, a severe, rare lung disease with no approved therapies in the US, EU, or UK.

• Autoimmune PAP is a chronic, lifelong disease caused by GM-CSF autoantibodies, leading to surfactant buildup and impaired oxygenation, resulting in progressive respiratory decline and risk of lung infections and fibrosis.

• Current standard of care is whole lung lavage, a complex, invasive rescue procedure available only at select specialty centers.

• Patients often face a lengthy diagnostic journey, with significant physical and emotional impact, as illustrated by a patient case requiring nine specialists and three years for diagnosis.

Product profile and clinical data

• MOLBREEVI is a once-daily inhaled biologic delivered via a proprietary eFlow nebulizer, with a BLA submitted in December and anticipated FDA review by February, aiming for US approval in August.

• Phase III IMPALA-2 trial showed statistically significant improvement in DLCO at 24 weeks, with supportive secondary endpoints in quality of life and exercise capacity; 100% of completers enrolled in open-label extension.

• Real-world data from five European patients showed no need for lung lavage after over four years on MOLBREEVI, with improved CT scans.

• Product has breakthrough designation in the US, orphan drug status in US and EU, and strong patent protection, ensuring long exclusivity periods.

Market opportunity and commercialization

• US epidemiology analysis identified approximately 5,500 autoimmune PAP patients, with a prevalence of 16 per million, and a concentrated market where 500 accounts manage 65% of patients.

• Commercial strategy targets all diagnosed patients, regardless of severity, with early intervention emphasized to potentially delay disease progression.

• Field-based commercial team and market development managers are being scaled up, with a specialty pharmacy partnership established for distribution and patient support.

• Pricing corridor set at $400,000–$500,000 per patient per year, with payers indicating minimal budget impact and expected coverage under specialty tier and Medicare Part D.