Savara (NASDAQ:SVRA) executives highlighted recent regulatory and commercial milestones for its lead program, molgramostim inhalation solution (Molbreevi), during a discussion at Oppenheimer’s 36th Annual Healthcare Life Science Conference. Management said the U.S. Food and Drug Administration (FDA) has accepted and filed the company’s biologics license application (BLA) for Molbreevi in autoimmune pulmonary alveolar proteinosis (aPAP), granted priority review, and assigned a projected Prescription Drug User Fee Act (PDUFA) action date of Aug. 22.
FDA review underway with priority designation
Chief Executive Officer Matt Pauls said the FDA’s acceptance of the BLA and priority review designation marks what he described as the “home stretch” following the company’s positive Phase III program. He characterized Savara as a single-asset company “fully valued around Molbreevi” and said the organization is driving the program forward with confidence in its path to U.S. approval.
Company emphasized Phase III outcomes across multiple measures
Executive Vice President Brian Robinson, MD, discussed the clinical rationale for Molbreevi and the data package supporting the BLA. Robinson said Molbreevi is intended to address key manifestations of aPAP, which he described as including impaired pulmonary gas transfer, poor respiratory quality of life, reduced patient function, and surfactant burden.
According to Robinson, data from Savara’s IMPALA program showed improvement from baseline across several measures, including:
- DLCO (diffusing capacity of the lung for carbon monoxide)
- SGRQ (a respiratory quality-of-life measure)
- Exercise capacity
- Surfactant burden
Robinson said these results, taken together, support the company’s confidence in the overall application.
Management described unmet need and current care as procedure-based
Pauls and Robinson emphasized that there are currently no approved therapeutics for aPAP in the U.S., Europe, or the U.K. Pauls described the disease as a serious, long-term, chronic, rare pulmonary condition with significant unmet need.
Chief Commercial Officer Braden Parker and Pauls pointed to whole lung lavage as the primary existing intervention, which Pauls characterized as a rescue procedure used in severe cases. Parker described whole lung lavage as a hospital-based procedure where patients are intubated and one lung is ventilated while the other lung is washed. He added that the procedure is offered only in select centers, requires hospitalization and operating room and intensive care unit resources, and is not standardized, while also not addressing the underlying cause of disease.
Robinson said Molbreevi is designed to address the pathophysiology of aPAP by activating macrophages, which in turn supports surfactant homeostasis and improves gas exchange.
U.S. patient estimates, launch targeting, and specialty pharmacy selection
On market sizing, Parker said published epidemiology estimates vary widely, ranging from six to seven patients per million up to approximately 26 per million. He said Savara conducted claims database analysis and identified roughly 5,500 known and diagnosed patients in the U.S., which he said equates to about 16 patients per million and represents a “floor” for the U.S. market. Parker said the company believes all 5,500 patients could benefit from Molbreevi upon approval, citing feedback from physicians indicating they would offer the therapy regardless of disease severity.
Regarding commercialization, Parker said the U.S. aPAP market appears concentrated, with the top 500 accounts managing roughly two-thirds of patients based on the company’s claims analysis. He said Savara is planning a field presence of roughly 30 customer-facing personnel, including leadership, and expects to add a small number of field reimbursement specialists. Parker said the company is aiming to hire the field team in the second quarter to support disease awareness and testing education ahead of a potential approval.
Parker also discussed the company’s selection of PANTHERx Rare as the exclusive specialty pharmacy partner in the U.S. He cited PANTHERx’s focus on rare and orphan diseases, experience with exclusive specialty pharmacy models, existing pulmonary relationships, and familiarity with device-based therapies as reasons for the choice. Parker said Savara is working with PANTHERx on pharmacy programming and patient services intended to help patients and providers navigate reimbursement and other U.S. healthcare system logistics.
In addition, Parker noted an interstitial lung disease (ILD) clinic pilot program at the University of Florida focused on testing, and said Savara would look to expand that program to other ILD clinics prior to approval.
Cash position and potential non-dilutive financing tied to approval
Pauls said Savara last reported $264 million in cash. He also outlined two sources of potential non-dilutive funding contingent on FDA approval: a royalty agreement with RTW that would provide $75 million at approval in exchange for a tiered royalty structure, and a separate debt facility with Hercules that could provide up to $75 million at the company’s discretion upon approval. Pauls said these arrangements would provide access to up to $150 million in additional non-dilutive financing around the time of the PDUFA date, if approval occurs, and stated that the company is “funded for a very long time” and in “great shape.”
About Savara (NASDAQ:SVRA)
Savara Inc (NASDAQ:SVRA) is a clinical-stage biopharmaceutical company focused on developing and commercializing therapies for rare respiratory diseases. The company specializes in in-licensing, advancing and potentially bringing to market novel treatments that address severe pulmonary conditions with high unmet medical need.
The company’s lead candidate, Molgradex (recombinant human granulocyte-macrophage colony-stimulating factor, or GM-CSF), is under regulatory review for the treatment of autoimmune pulmonary alveolar proteinosis (PAP), a rare lung disease characterized by the accumulation of surfactant.
