Savara (SVRA) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Company overview and disease focus

• Focused on developing molgramostim, an inhaled biologic for rare respiratory disease APAP.

• APAP is a rare autoimmune lung disease with no approved drug treatments; current standard is lung lavage, a temporary and invasive procedure.

• Molgramostim aims to replace deficient GM-CSF in the lungs, addressing the root cause of APAP.

• The drug is delivered via a customized nebulizer sourced through an exclusive partnership.

Clinical trial results and efficacy

• IMPALA-2 phase III trial showed statistically significant improvement in lung function (DLCO) at weeks 24 and 48.

• Secondary endpoints, including quality of life (SGRQ) and exercise capacity, also showed significant or positive trends.

• 100% of eligible patients continued into the open-label extension, indicating strong interest and perceived benefit.

• Additional data presented at ERS showed significant improvement in disease severity and surfactant burden.

• Safety profile was comparable to placebo, with no drug-related discontinuations.

Market potential and commercial strategy

• Estimated 5,000 APAP patients in the U.S., with potential for higher prevalence as awareness and diagnostics improve.

• Pricing expected in the $300,000-$500,000 per patient per year range, consistent with rare disease analogs.

• Disease awareness campaigns and free antibody testing are underway to increase diagnosis rates.

• Commercial launch could be supported by a small, targeted field team.