Savara (SVRA) Guggenheim Securities Emerging Outlook: Biotech Summit 2026 summary

Program and regulatory update

• MOLBREEVI, a late-stage asset for autoimmune PAP, has a resubmitted BLA with the FDA, expecting acceptance and priority review, targeting a PDUFA date in August.

• Manufacturing was shifted to Fujifilm after a prior refusal to file, with successful tech transfer and alignment on analytical comparability.

• MAAs for Europe and UK are on track for filing by the end of Q1, with plans to commercialize independently in those regions.

• Japan remains a wait-and-see market, with strategy to be determined.

• Company is well-capitalized, with $264 million on the balance sheet and access to additional non-dilutive capital upon FDA approval.

Clinical data and endpoints

• Phase III IMPALA-2 study showed statistically significant improvement in DLco at 24 and 48 weeks, the primary and key secondary endpoints.

• SGRQ total and activity scores, as well as exercise tolerance, demonstrated robust secondary endpoint results.

• DLco is a clinically relevant, reproducible marker for autoimmune PAP, supporting its use as a primary endpoint.

• SGRQ improvements met the minimal clinically important difference of four points, consistent with related pulmonary diseases.

Commercial strategy and market opportunity

• Claims analysis identified about 5,500 diagnosed aPAP patients in the US, with line of sight to 1,000 patients, representing 20% of the market.

• Top 500 accounts cover two-thirds of the market, supporting efficient patient activation post-approval.

• Specialty pharmacy partner PANTHERx will support product distribution and patient access.

• Disease awareness campaigns and diagnostic testing (including a dry blood spot test) are expanding identification of new patients.

• There is a bolus of patients expected to be ready for therapy at launch, with additional uptake as awareness grows.