LANGHORNE, Pa.--(BUSINESS WIRE)--
(Nasdaq: SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, today announced a manuscript titled, was published online in the journal ERJ Open Research. The outcomes data presented suggest that treatment with molgramostim addresses the underlying pathophysiology of aPAP, resulting in improved lung function, decreased disease burden, restored patient functionality, and reduction of clinical symptoms, and may enable resumption of daily life activities.
This case series retrospectively evaluated five aPAP patients who received molgramostim through European single-patient access, supplied by Savara. Following treatment with molgramostim (mean duration of 4.2 years), improvements in disease severity were shown across pulmonary gas transfer, measured by percent predicted diffusing capacity of the lung for carbon monoxide (DLco) and alveolar-arterial oxygen gradient (A-aDO2), as well as activities of daily living. Additionally, surfactant burden was reduced as indicated by high-resolution computed tomography scans taken before and after molgramostim treatment. Furthermore, while four of the five patients had at least one whole lung lavage (WLL) prior to treatment with molgramostim, none of the five patients required WLL after more than one year on treatment, suggesting molgramostim may reduce the need for WLL. No reported serious adverse events occurred, and treatment was well tolerated.
“Long-term outcomes data from these case studies are encouraging and support our belief that treatment with molgramostim may address the root cause of aPAP, resulting in improved and sustained patient benefit,” said Matt Pauls, Chair and Chief Executive Officer, Savara. “We are committed to our goal of providing the aPAP community with the first and only pharmaceutical treatment option in the U.S. and Europe and look forward to completing the rolling Biologics License Application (BLA) submission in the U.S. by the end of 1Q25 and the Marketing Authorization Application (MAA) in Europe by the end of 2025.”
Autoimmune PAP is a rare lung disease characterized by the abnormal build-up of surfactant in the alveoli (or air sacs) of the lungs. Surfactant consists of proteins and lipids and is an important physiological substance that lines the alveoli to prevent them from collapsing. In a healthy lung, excess surfactant is cleared and digested by immune cells called alveolar macrophages. Alveolar macrophages need to be stimulated by granulocyte-macrophage colony-stimulating factor (GM-CSF) to function properly in clearing surfactant, but in aPAP, GM-CSF is neutralized by antibodies against GM-CSF, rendering macrophages unable to adequately clear surfactant. As a result, an excess of surfactant accumulates in the alveoli, causing impaired gas exchange, resulting in clinical symptoms of shortness of breath, often with cough and frequent fatigue. Patients may also experience episodes of fever, chest pain, or coughing up blood, especially if secondary lung infection develops. In the long-term, the disease can lead to serious complications, including lung fibrosis and the need for a lung transplant.
Savara is a clinical stage biopharmaceutical company focused on rare respiratory diseases. Our lead program, molgramostim, is a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar proteinosis (aPAP). Molgramostim is delivered via an investigational eFlow® Nebulizer System (PARI Pharma GmbH) specifically developed for inhalation of a large molecule. Our management team has significant experience in rare respiratory diseases and pulmonary medicine, identifying unmet needs, and effectively advancing product candidates to approval and commercialization. More information can be found at , X: and .
Forward-Looking Statements
Savara cautions you that statements in this press release that are not a description of historical fact are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words referencing future events or circumstances such as “expect,” “intend,” “plan,” “anticipate,” “believe,” and “will,” among others. Such statements include, but are not limited to, statements regarding the suggestion that treatment with molgramostim addresses the underlying pathophysiology of aPAP and our belief that it may address the root cause of aPAP, resulting in improved and sustained patient benefit, and statements related to the anticipated timing of our BLA and MAA submissions. These forward-looking statements are based upon Savara’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, the risks associated with our ability to successfully develop, complete the requisite regulatory submissions and obtain regulatory approval for, and commercialize molgramostim for aPAP; the risks and uncertainties related to the impact of widespread health concerns or changing economic or geopolitical conditions; the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations; and the availability of sufficient financial and other resources for Savara’s operations and to conduct or continue planned clinical development programs. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. For a detailed description of our risks and uncertainties, you are encouraged to review our documents filed with the SEC including our recent filings on Form 8-K, Form 10-K and Form 10-Q. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Savara undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as may be required by law.
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