Savara (SVRA) H.C. Wainwright 4th Annual BioConnect Investor Conference summary

Company and product overview

• Currently developing MOLBREEVI, an inhaled GM-CSF therapy for autoimmune PAP, with a PDUFA date set for November 22nd this year.

• MOLBREEVI targets a rare disease with 5,500 identified U.S. patients and a pricing corridor of $400,000-$500,000 per patient per year.

• 85% of payers have indicated willingness to cover MOLBREEVI with simple prior authorization.

• If approved, MOLBREEVI would be the first therapy for aPAP, addressing the root cause rather than providing temporary relief.

• The company has about 25 field staff focused on disease awareness and physician engagement.

Disease background and diagnosis

• aPAP is caused by antibodies to GM-CSF, leading to surfactant accumulation and progressive respiratory symptoms.

• Symptoms are nonspecific, often resulting in delayed or missed diagnosis, with patients seen in both community and academic settings.

• The company has implemented a dried blood spot test and the ClearPath program to facilitate diagnosis, offered at no cost to providers and patients.

• The ClearPath program is being utilized, with pilot programs at ILD clinics and plans to expand to more centers.

Clinical data and physician perspectives

• IMPALA-2 phase III trial showed statistically significant improvements in DLco and quality of life measures at weeks 24 and 48.

• Additional exploratory endpoints demonstrated functional improvements in exercise capacity and duration.

• Open-label extension and biomarker data further support efficacy and are being presented at major conferences.

• Physicians express interest in treating all symptomatic aPAP patients, regardless of severity, to prevent disease progression.

• Even mildly affected patients show motivation to seek treatment, as seen in trial screening data.