Savara (SVRA) Leerink’s Global Healthcare Conference 2025 summary

Recent progress and regulatory milestones

• Positive top-line results from the global phase 3 IMPALA-2 trial for MOLBREEVI were announced in mid-2024, supporting a rolling BLA submission to the FDA by the end of June 2024.

• Anticipates a 60-day FDA review with priority status, targeting a PDUFA date in late November or early December 2024.

• Plans to file an MAA in Europe by the end of 2024, with a strategy to potentially launch independently in key European markets.

• Strong engagement with regulators due to breakthrough designation and established partnership with PARI for the proprietary nebulizer device.

• Confident in probability of regulatory success and maintaining full value of global rights due to a strong balance sheet.

Disease overview and unmet need

• Autoimmune PAP (aPAP) is a rare, chronic lung disease caused by autoantibodies against GM-CSF, leading to surfactant buildup and impaired gas exchange.

• Current standard of care is whole lung lavage, a complex, non-curative, and traumatic procedure requiring repeat interventions.

• Diagnosis is often delayed by 18–24 months due to nonspecific symptoms; a new dry blood spot test was recently launched to accelerate diagnosis.

• Many patients remain undiagnosed, and there is a significant need for a chronic therapeutic that addresses the underlying disease mechanism.

Patient identification and market development

• Epidemiology estimates 3,600 diagnosed aPAP patients in the U.S., with machine learning analyses suggesting a total prevalence above 7,000.

• Direct outreach to 1,000 accounts has identified 450 patients, with a goal of 1,000 at launch to support early adoption.

• The dry blood spot test is being promoted to physicians and integrated into diagnostic workflows to shorten time to diagnosis.

• European prevalence is estimated at around 5,000 patients in core markets, with similar disease distribution as the U.S.