Molgramostim

Savara’s Partner, Trillium Health LLC, Presented Data on the Laboratory Blood Test for Diagnosing aPAP LANGHORNE, Pa.--(BUSINESS WIRE)-- Savara Inc. (Nasdaq: SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, presented a poster at the American Thoracic Society (ATS) 2024 International Conference that is taking place May 17-22, 2024, in San Diego, CA. Savara’s partner, Trillium Health LLC, presented a poster on the laboratory blood test that was recently developed to aid in the diagnosis of aPAP. Below are summaries of the posters presented: Poster Board 701: “Healthcare Burden of Pulmonary Alveolar Proteinosis” presented by E. Lee, M.D., Ph.D.; sponsored by Savara Inc. Presented data from a retrospective cohort analysis using a longitudinal claims database (IPM.ai) comparing the clinical and economic burden of a sample of 2,312 pulmonary alveolar proteinosis (PAP) patients to 9,247 non-PAP matched controls. Data demonstrated PAP patients experience higher healthcare resource utilization and higher pharmaceutical and non-pharmaceutical costs as evidenced by a 4-fold increase in hospital admissions, 6-fold increase in in-patient visits, and a 4-fold increase in cost of care, as well as higher rates of comorbidities, procedures, and therapy use (all parameters p<0.001), thereby highlighting a significant unmet need in this rare disease patient population. Click here to view the abstract. Lee E, Ataya A, McCarthy C, Cosenza J, Bielecki S, Wang TS. Healthcare burden of pulmonary alveolar proteinosis. Am J Respir Crit Care Med 2024;209:A1173. Poster Board 702: “Development of a Novel Laboratory Test for the Detection of GM-CSF Antibodies to Aid in the Diagnosis of Individuals At-risk for Autoimmune Pulmonary Alveolar Proteinosis (aPAP)” presented by A. Ali and J. Kim of Trillium Health LLC Presented data on the development and performance characteristics of a novel laboratory test for the detection of granulocyte-macrophage colony-stimulating factor (GM-CSF) antibodies in human serum to aid in the diagnosis of aPAP. Clinical accuracy of 100% was achieved in a cohort of 40 subjects (negative cohort n=20, positive cohort n=20), demonstrating Trillium’s particle-based flow cytometry immunoassay test offers a highly precise and sensitive laboratory test for the determination of GM-CSF antibody levels in human serum and, thus, can successfully be used as a tool to definitively diagnose aPAP. Click here to view the abstract. Kim J, Cornish D, Ali A. Development of a novel laboratory test for the detection of GM-CSF antibodies to aid in the diagnosis of individuals at-risk for autoimmune pulmonary alveolar proteinosis (aPAP). Am J Respir Crit Care Med 2024;209:A1174. The full content of these posters is available in the Articles and Publications page of the Savara corporate website. Additionally, the abstracts are published in a supplement of the American Journal of Respiratory and Critical Care Medicine (AJRCCM). For more details about the ATS International Conference please visit . About aPAP Autoimmune PAP is a rare lung disease characterized by the abnormal build-up of surfactant sediment in the alveoli (or air sacs) of the lungs. The surfactant consists of proteins and lipids and is an important physiological substance that lines the inside of the alveoli to prevent the lungs from collapsing. In a healthy lung, the old and inactivated 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 autoimmune PAP, GM-CSF is neutralized by antibodies against GM-CSF, rendering the macrophages unable to perform their tasks. As a result, an excess of surfactant accumulates in the alveoli, causing obstruction of gas exchange, and patients start to experience shortness of breath, often with cough and frequent fatigue. Patients may also experience chronic cough, as well as episodes of fever, chest pain, or coughing 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. About Savara Savara is a clinical stage biopharmaceutical company focused on rare respiratory diseases. Our lead program, molgramostim nebulizer solution, is an inhaled 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). 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, formerly known as Twitter: @SavaraPharma, LinkedIn: ). 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. Savara may not actually achieve any of the matters referred to in such forward-looking statements, and you should not place undue reliance on these forward-looking statements. 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 outcome of our ongoing and planned clinical trials for our product candidate, the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources for Savara’s operations and to conduct or continue planned clinical development programs, the ability to successfully develop our product candidate, the risks associated with the process of developing, obtaining regulatory approval for and commercializing drug candidates such as molgramostim that are safe and effective for use as human therapeutics, and the timing and ability of Savara to raise additional capital as needed to fund continued operations. 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. View source version on businesswire.com: Contacts Savara Inc. IR & PR Anne Erickson (anne.erickson@savarapharma.com) (512) 851-1366 Source: Savara Inc. View this news release online at:

Pivotal Phase 3 IMPALA-2 Trial Remains On-track, Top Line Results Expected by End of 2Q 2024 48-week placebo-controlled trial is evaluating molgramostim nebulizer solution (molgramostim), a novel inhaled biologic, for the treatment of autoimmune Pulmonary Alveolar Proteinosis (aPAP), a rare lung disease Pending Results from the IMPALA-2 Trial, a Biologics License Application (BLA) Filing is Anticipated in 1H 2025 With ~$143M in Cash and Short-term Investments, the Company Continues to Believe it is Sufficiently Capitalized into 2026 LANGHORNE, Pa.--(BUSINESS WIRE)-- Savara Inc. (Nasdaq: SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, reported financial results for the first quarter ending March 31, 2024 and provided a business update. “The IMPALA-2 trial remains on-track and we look forward to reporting top line results by the end of the second quarter,” said Matt Pauls, Chair and CEO, Savara. “Following that, and assuming positive data, we expect to BLA in the first half of 2025. Importantly, with $143 million in cash and investments, we believe we are capitalized into 2026 which is well beyond the data read-out and includes investments in programs such as the extension of the IMPALA-2 open-label period from 48 weeks to 96 weeks, anticipated launch of a global Expanded Access program for molgramostim, build out of the U.S. Commercial infrastructure, and pre-Commercial preparations in Europe.” First Quarter Financial Results (Unaudited) Savara's net loss for the first quarter of 2024 was $20.3 million, or $(0.11) per share, compared with a net loss of $10.6 million, or $(0.07) per share, for the first quarter of 2023. Research and development expenses increased by $8.1 million, or 92.3%, to $16.8 million for the three months ended March 31, 2024 from $8.7 million for the three months ended March 31, 2023. The increase was largely due to the performance of tasks related to the molgramostim program, which included approximately $4.3 million associated with chemistry, manufacturing, and controls activities and primarily driven by initiatives at second source drug substance manufacturers, $1.0 million related to the IMPALA-2 clinical trial, including clinical research organization (CRO) related activities, $0.6 million associated with regulatory affairs and quality assurance work, and $2.2 million due to an increase in personnel and related costs. General and administrative expenses increased by $2.3 million, or 67.4%, to $5.6 million for the three months ended March 31, 2024 from $3.4 million for the three months ended March 31, 2023. The increase was due to personnel and related costs of $0.6 million, certain commercial activities of $1.1 million, and other overhead of $0.6 million primarily driven by patient advocacy activities. As of March 31, 2024, the Company had cash, cash equivalents and short-term investments of approximately $143.0 million and debt of approximately $26.4 million. About Savara Savara is a clinical stage biopharmaceutical company focused on rare respiratory diseases. Our lead program, molgramostim nebulizer solution, is an inhaled 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). 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, formerly known as Twitter: @SavaraPharma, LinkedIn: ). 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 related to the expected timing of reporting top line data from the IMPALA-2 trial, the anticipated timing of the BLA filing, our belief the Company is capitalized into 2026, and the planned investments in the extension of the IMPALA-2 open label period, the anticipated launch of a global Expanded Access program, the build out of the U.S. Commercial infrastructure, and pre-Commercial preparations in Europe. Savara may not actually achieve any of the matters referred to in such forward-looking statements, and you should not place undue reliance on these forward-looking statements. 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 and uncertainties relating to the impact of widespread health concerns impacting healthcare providers or patients, disruptions or inefficiencies in the supply chain and geopolitical conditions, the outcome of our ongoing and planned clinical trials for our product candidate, the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources for Savara’s operations and to conduct or continue planned clinical development programs, the ability to successfully develop our product candidate, the risks associated with the process of developing, obtaining regulatory approval for and commercializing drug candidates such as molgramostim that are safe and effective for use as human therapeutics, and the timing and ability of Savara to raise additional capital as needed to fund continued operations. 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. Financial Information to Follow Savara Inc. and Subsidiaries Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except for share and per share amounts) Unaudited Three months ended March 31, 2024 2023 Operating expenses: Research and development $ 16,807 $ 8,738 General and administrative 5,636 3,366 Depreciation and amortization 32 8 Total operating expenses 22,475 12,112 Loss from operations (22,475 ) (12,112 ) Other income (expense), net: 2,129 1,555 Net loss attributable to common stockholders $ (20,346 ) $ (10,557 ) Net loss per share - basic and diluted $ (0.11 ) $ (0.07 ) Weighted average shares - basic and diluted 182,550,109 152,781,580 Other comprehensive (loss) gain (471 ) 144 Total comprehensive loss $ (20,817 ) $ (10,413 ) Savara Inc. and Subsidiaries Condensed Consolidated Balance Sheet Data (in thousands) (Unaudited) March 31, December 31, 2024 2023 Cash, cash equivalents, and short-term investments $ 143,043 $ 162,319 Working capital 136,377 155,350 Total assets 158,295 177,564 Total liabilities 36,434 37,192 Stockholders’ equity: 121,861 140,372