Savara Presented Long-Term Efficacy and Safety Data from the Ongoing IMPALA-2 Phase 3 Clinical Trial Open-Label (OL) Extension of Molgramostim Inhalation Solution (Molgramostim) in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
-- Data Were Presented at the American Thoracic Society (ATS) International Conference 2026 --
-- All Patients Receive Molgramostim During the OL Treatment Period; 100% of Patients Who Completed the Double-Blind (DB) Period of the Trial Entered into the OL Extension --
-- OL Data Showed Continued Improvement in Patients Who Had Received Molgramostim During DB Period --
-- Efficacy Improved in Patients Who Had Received Placebo During DB Period and Switched to Molgramostim in OL Treatment Period --
Savara Inc. (NASDAQ:SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, presented a poster at the ATS 2026 International Conference that is taking place May 15-20, 2026, in Orlando, Florida. The poster reports long-term efficacy and safety data from the first 48 weeks of the ongoing OL treatment period of the IMPALA-2 Phase 3 clinical trial evaluating molgramostim for the treatment of aPAP. IMPALA-2 is the longest and largest clinical trial conducted in aPAP and enrolled 164 patients.
Long-term efficacy and safety data from IMPALA-2 were presented. Below is a summary of the poster presentation.
Poster Board 403: "Long-Term Efficacy and Safety of Molgramostim in Patients with Autoimmune Pulmonary Alveolar Proteinosis (aPAP): Results from the IMPALA-2 Trial Open-Label Treatment Period," presented by B.C. Trapnell, M.D.; sponsored by Savara Inc.
During the DB period, aPAP patients received nebulized molgramostim 300 µg (n=81) or placebo (n=83) once daily; all patients received molgramostim once daily during the OL period. Results from the first 48 weeks of the 96-week OL period were reported. Efficacy was assessed by change from baseline in percent predicted diffusing capacity of the lungs for carbon monoxide (DLco%) and by St. George's Respiratory Questionnaire Total (SGRQ-T) and Activity (SGRQ-A) scores assessing respiratory health-related quality of life (HRQoL). Exercise capacity is not evaluated in the OL treatment period.
One hundred and sixty-four patients with aPAP enrolled in the DB period, 160 (98%) completed the DB period, and all 160 of those patients opted to continue into the OL period. As of the data cut-off, 9 out of 160 patients discontinued the OL period—a 94% retention rate.
Long-term treatment with molgramostim continuously improved pulmonary gas transfer and respiratory HRQoL in patients with aPAP. In addition, placebo crossover patients demonstrated improved pulmonary gas transfer and respiratory HRQoL following initiation of molgramostim treatment.
Durability of Effect During First 48 Weeks of OL Treatment Period
Data from the first 48 weeks of the OL treatment period showed continuous and sustained improvements in pulmonary gas transfer, as measured by DLco% and respiratory HRQoL, as measured by SGRQ-T and SGRQ-A scores.
Pulmonary Gas Transfer Improvements
- At Week 48 of the DB period, mean (standard error [SE]) changes from baseline in DLco% were 11.6 (1.4) for the molgramostim group and 3.9 (1.5) for the placebo group.
- During the OL period, patients who received molgramostim in both the DB and OL periods (MOL-MOL) continued to show improvement in DLco% through Week 96, with a mean increase of 2.8 (1.2) during Weeks 48–96 and an overall mean increase from baseline (Weeks 0–96) of 14.7 (1.6). In patients who received placebo during the DB period and crossed over to molgramostim in the OL period (PBO-MOL), DLco% increased through Week 96, with a mean increase of 8.8 (1.6) during Weeks 48–96.
Respiratory HRQoL Improvements
Both SGRQ-T and SGRQ-A scores range from 0-100, with higher values indicating worse quality of life. Results showed continued improvement in MOL-MOL patients and notable improvements in PBO-MOL crossover patients in SGRQ-T and SGRQ-A scores.
- At Week 48 of the DB period, mean (standard error [SE]) changes from baseline in SGRQ-T and SGRQ-A scores were -10.8 (2.3) and -12.8 (3.1), respectively, for the molgramostim group and -6.1 (2.1) and -7.8 (2.7), respectively for the placebo group.
- For SGRQ-T and SGRQ-A scores during the OL period, MOL-MOL patients showed continued improvement during Weeks 48–96 with mean changes of −3.8 (1.6) and −4.2 (2.5) (SGRQ-T and SGRQ-A), respectively, and overall changes from baseline (Weeks 0–96) of −15.0 (2.6) and −18.3 (3.4), respectively. In PBO-MOL patients, corresponding mean changes were −6.5 (1.6) and −7.9 (2.0), respectively, during Weeks 48–96.
Safety and Tolerability During First 48-Weeks of OL Treatment Period
Data from the OL treatment period indicate that molgramostim was generally well tolerated, with safety and tolerability results consistent with those previously reported for the DB treatment period. There were no study discontinuations due to treatment-related adverse events.
"We are pleased to see that after longer-term drug exposure, the MOL-MOL patients showed continuous improvements in efficacy and quality of life, and the PBO-MOL patients showed improvements in the same outcome measures while receiving molgramostim during the OL treatment period—similar to molgramostim patients during the double-blind period," said Bruce Trapnell, M.D., Professor of Medicine and Pediatrics at the University of Cincinnati College of Medicine and the Lead Clinical Investigator of the IMPALA-2 trial. "Furthermore, we continued to see a very high patient retention rate, attesting to the tolerability of molgramostim over time. These results are similar to results from the open-label period of the IMPALA trial in that they indicate that longer-term treatment with molgramostim results in durability of effect. I believe these data strongly support the potential of molgramostim to be an effective pharmacologic treatment option for aPAP."
The full content of this poster will be available on the Congresses and Publications page of the Savara corporate website. The abstract is 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 https://conference.thoracic.org/index.php.
Autoimmune PAP is a rare lung disease characterized by the abnormal build-up of surfactant in the alveoli. 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 inhalation solution (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 a proprietary investigational eFlow® Nebulizer System (PARI Pharma GmbH) specifically developed for inhalation of molgramostim. 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 www.savarapharma.com and 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 risks associated with our ability to successfully develop, obtain regulatory approval for, and commercialize molgramostim for aPAP; the actions and decisions of regulatory authorities; 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; 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.
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