Savara (SVRA) Guggenheim’s Inaugural Healthcare Innovation Conference summary

Key regulatory and clinical updates

• Rolling BLA submission for Molbreevi to begin by year-end, with full submission by end of Q1, ahead of previous 1H 2025 guidance.

• MAA filing in Europe targeted for end of 2025, marking the first formal timeline for this region.

• IMPALA-2 phase 3 trial met primary and key secondary endpoints, showing durable efficacy at 24 and 48 weeks.

• High probability of regulatory approval anticipated in the U.S. and globally based on robust data.

• Cash position of $219 million extends runway through Q2 2027.

Commercial strategy and market opportunity

• U.S. market profiling targets 1,000 aPAP patients at launch, with 3,600 currently diagnosed and identified through claims analysis.

• Multi-tiered approach focuses on centers of excellence, ILD clinics, and broader pulmonology centers.

• Additional 3,700 likely or highly likely patients identified via machine learning, potentially doubling the addressable market.

• Disease education and diagnostic testing (ClearPath) are key to expanding patient identification.

• Dried blood spot test launch planned for 1H next year to simplify and broaden patient screening.

International expansion and competitive landscape

• European market seen as comparable in size and structure to the U.S., with plans to launch independently but open to partnerships.

• MAA filing in Europe by end of 2025, with further details to be shared in future updates.

• No direct competitors in U.S. or Europe; whole lung lavage remains a rescue therapy, while Japan has a recent approval of Sargramostim.

• Focus remains on capturing the confirmed U.S. patient base and growing the market through ongoing diagnostic efforts.