Molbreevi (molgramostim) / Savara, Nobelpharma - LARVOL DELTA

Molgramostim for the treatment of patients with autoimmune pulmonary alveolar proteinosis (aPAP): long-term efficacy and safety results from the open-label period of the IMPALA-2 phase 3 clinical trial (ERS 2026) - "Long-term MOL treatment continuously improved pulmonary gas transfer (DLco%) and health-related quality of life (SGRQ) in aPAP patients. In addition, PBO-MOL patients showed improvements."

Clinical • P3 data • Immunology • Respiratory Diseases

Biomarker levels in patients with autoimmune pulmonary alveolar proteinosis (aPAP): results from the IMPALA-2 phase 3 clinical trial (ERS 2026) - "Levels of biomarkers associated with aPAP disease severity decreased in molgramostim-treated patients and were associated with improvements in DLco%."

Biomarker • Clinical • P3 data • Immunology • Respiratory Diseases • CEACAM5 • CK19 • KRT19

Molgramostim improves exercise capacity, distance walked, and duration of exercise in patients with autoimmune pulmonary alveolar proteinosis (aPAP): results from the IMPALA-2 Phase 3 clinical trial (ERS 2026) - "Consistent with improvements in peak METs, MOL improved distance walked and duration of exercise at Week 48 compared with PBO."

Clinical • P3 data • Immunology • Respiratory Diseases

MOLGRAMOSTIM TREATMENT EFFECTS IN AUTOIMMUNE PULMONARY ALVEOLAR PROTEINOSIS PATIENTS WITH MILD DIFFUSION IMPAIRMENT (CHEST 2026) - No abstract available

Clinical • Immunology • Respiratory Diseases

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 (CHEST 2026) - No abstract available

Clinical • Immunology • Respiratory Diseases

Savara…Provides Business Update (Businesswire) - "Marketing Authorization Applications (MAAs) Validated by the European Medicines Agency (EMA) and the U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) with Decisions Expected in Q1 2027 and Q4 2026, respectively. Announced New Data from the Phase 3 IMPALA-2 Clinical Trial Will be Presented at the American Thoracic Society (ATS) International Conference in May, Including Data from the Ongoing Open-Label Treatment Period."

EMA approval • MHRA approval • P3 data • Pulmonary Disease • Rare Diseases

Efficacy and Safety of Inhaled GM-CSF in Autoimmune Pulmonary Alveolar Proteinosis: A Systematic Review and Meta-analysis of Randomized Controlled Trials. (PubMed, Lung) - "Inhaled GM-CSF improves gas exchange, oxygenation, and dyspnea in aPAP while maintaining a favorable safety profile, indicating its potential as a noninvasive, targeted therapy."

Clinical • Journal • Retrospective data • Review • Immunology • Pulmonary Disease • Respiratory Diseases • CSF2

Savara Announces the U.S. Food & Drug Administration (FDA) Has Extended the Review Period for the Molgramostim Inhalation Solution (Molgramostim) Biologics License Application (BLA) in Autoimmune Pulmonary Alveolar Proteinosis (Autoimmune PAP) (Businesswire) - "Prescription Drug User Fee Act (PDUFA) Target Action Date Extended by Three Months to November 22, 2026....The FDA determined that the Company’s responses to recent information requests by the Agency constituted a major amendment to the BLA, resulting in a three-month extension of the PDUFA date. The Agency did not cite any safety, efficacy, or manufacturing concerns in their correspondence."

PDUFA • Pulmonary Disease • Rare Diseases

Savara Announces the U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) Has Accepted the MOLBREEVI Marketing Authorisation Application (MAA) for Autoimmune Pulmonary Alveolar Proteinosis (Autoimmune PAP) (Businesswire)

MHRA filing • Pulmonary Disease • Rare Diseases

A combined treatment with GM-CSF, fosfomycin and metronidazole for pouchitis in ulcerative colitis patients after restorative ileal pouch anal anastomosis surgery. A clinical safety and proof-of-concept study (clinicaltrialsregister.eu) - P1/2 | N=18 | Recruiting | Sponsor: Region Sjaelland, Reponex Pharmaceuticals A/S | Not yet recruiting ➔ Recruiting

Enrollment open • Gastroenterology • Gastrointestinal Disorder • Immunology • Inflammation • Inflammatory Bowel Disease • Ulcerative Colitis

Savara Announces European Medicines Agency (EMA) Validation of Marketing Authorization Application (MAA) for MOLBREEVI in Autoimmune Pulmonary Alveolar Proteinosis (Autoimmune PAP) (Businesswire) - "EMA Review of MOLBREEVI MAA Has Now Initiated, Decision Expected in Q1 2027."

EMA approval • EMA filing • Pulmonary Disease • Rare Diseases

Savara Provides Regulatory Update on the MOLBREEVI Development Program in Autoimmune Pulmonary Alveolar Proteinosis (Autoimmune PAP) (Businesswire) - "Savara...announced that it has received the Day 74 Letter from the FDA which indicated that an Advisory Committee meeting is not planned for the MOLBREEVI Biologics License Application (BLA) and the review is ongoing with a Prescription Drug User Fee Act (PDUFA) target action date of August 22, 2026. Additionally, the Company recently submitted the MAA to the EMA for MOLBREEVI for the treatment of autoimmune PAP and is on track to submit the MAA to the U.K.’s MHRA by the end of Q1 2026."

EMA filing • FDA event • MHRA filing • Pulmonary Disease • Rare Diseases

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 (ATS 2026) - No abstract available

Clinical • Immunology • Respiratory Diseases

Efficacy of recombinant human GM-CSF compared to placebo for autoimmune pulmonary alveolar proteinosis: a systematic review and meta-analysis. (PubMed, BMC Pulm Med) - No abstract available

Journal • Retrospective data • Review • Immunology • Respiratory Diseases • CSF2

Molgramostim Improves Exercise Distance and Duration in Patients With Autoimmune Pulmonary Alveolar Proteinosis (aPAP): Results From the IMPALA-2 Phase 3 Clinical Trial (ATS 2026) - No abstract available

Clinical • P3 data • Immunology • Respiratory Diseases

Savara Announces the U.S. Food and Drug Administration (FDA) Filed the MOLBREEVI Biologics License Application (BLA) in Autoimmune Pulmonary Alveolar Proteinosis (Autoimmune PAP) (Businesswire) - "The FDA granted Priority Review with a PDUFA action date of August 22, 2026."

FDA filing • PDUFA • Priority review • Pulmonary Disease • Rare Diseases

Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP). (clinicaltrials.gov) - P3 | N=5 | Recruiting | Sponsor: Savara Inc. | Not yet recruiting ➔ Recruiting | Trial completion date: Aug 2027 ➔ Dec 2027 | Trial primary completion date: Jan 2027 ➔ Jun 2027

Enrollment open • Trial completion date • Trial primary completion date • Immunology • Pediatrics • Respiratory Diseases

Savara Announces Amendment to Hercules Capital Debt Facility Providing up to $75M of Additional Debt Funding Upon U.S. Food and Drug Administration (FDA) Approval of MOLBREEVI (Businesswire) - "When Combined with Recently Announced $75M Royalty Financing, the Company Will Have Access to ~$150M of Non-Dilutive Capital for Launch of MOLBREEVI...The Company resubmitted the MOLBREEVI Biologics License Application (BLA) to the FDA in December 2025. If Priority Review is granted, MOLBREEVI could potentially be approved in 3Q 2026. The Company remains on track to file the Marketing Authorization Applications (MAA) for MOLBREEVI in Europe and the U.K. by the end of 1Q 2026."

FDA approval • Filing • Financing • Immunology • Respiratory Diseases

Pseudovax - A Cancer Vaccine for Patients With Pseudomyxoma Peritonei (clinicaltrials.gov) - P1 | N=10 | Recruiting | Sponsor: Oslo University Hospital

Checkpoint inhibition • IO biomarker • New P1 trial • Tumor mutational burden • Oncology • CSF2 • GNAS

ANTHERx Rare Pharmacy…and Savara Inc…announced an exclusive distribution agreement for MOLBREEVI. (Nasdaq) - "MOLBREEVI, a non-glycosylated form of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF), is an inhaled investigational medicine being developed for autoimmune PAP, a rare and chronic lung disease caused by GM-CSF autoantibodies which block GM-CSF signaling and reduce surfactant clearance—resulting in difficulty breathing and, ultimately, hypoxemic respiratory failure."

Licensing / partnership • Pulmonary Disease

Savara Resubmits the Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for MOLBREEVI for the Potential Treatment of Autoimmune Pulmonary Alveolar Proteinosis (Autoimmune PAP) (Businesswire) - "The Company has requested Priority Review of the application."

FDA filing • Pulmonary Disease • Rare Diseases

Savara Announces European Patent Office (EPO) Intends to Grant a Patent for the Liquid Formulation of MOLBREEVI (Businesswire) - "Once Granted, the Patent Will Provide Protection for MOLBREEVI in Europe Until March 2041....'We expect to resubmit the MOLBREEVI BLA to the FDA this month and are preparing to submit the MAA submissions in the EU and the U.K. by the end of 1Q 2026'."

EMA filing • FDA filing • MHRA filing • Patent • Pulmonary Disease • Respiratory Diseases

RELATIONSHIP BETWEEN PULMONARY GAS TRANSFER, RESPIRATORY HEALTH-RELATED QUALITY OF LIFE, EXERCISE CAPACITY, AND SURFACTANT BURDEN IN AUTOIMMUNE PULMONARY ALVEOLAR PROTEINOSIS (CHEST 2025) - "PURPOSE: Molgramostim inhalation solution (MOL) is an investigational recombinant granulocyte-macrophage colony stimulating factor being evaluated for the treatment of autoimmune pulmonary alveolar proteinosis (aPAP) in a Phase 3 clinical trial (IMPALA-2)... Changes in DLco% were correlated with changes in SGRQ-T (a measure of respiratory health-related quality of life), exercise capacity, and GGO (a measure of surfactant burden) in patients with aPAP. CLINICAL IMPLICATIONS: Results support the clinical relevance of changes in DLco% in aPAP."

Clinical • HEOR • Surfactant • Immunology • Respiratory Diseases

EFFICACY OF INHALED MOLGRAMOSTIM ACCORDING TO SEVERITY OF AUTOIMMUNE PULMONARY ALVEOLAR PROTEINOSIS (CHEST 2025) - "Molgramostim was effective in patients with more or less severe disease as measured by DLco% at trial randomization. CLINICAL IMPLICATIONS: Molgramostim was effective in aPAP patients regardless of disease severity based on DLco%."

Clinical • Immunology • Respiratory Diseases

Recombinant GM-CSF drug evaluation review. (PubMed, Immunotherapy) - "Molgramostim and sargramostim are rGM-CSF which can be self-administered at home by patients with aPAP either subcutaneously or using a handheld nebulizer. In all clinical studies to date, rGM-CSF has shown a favorable safety profile with no dose limiting toxicity. This supports the application of inhaled rGM-CSF for the treatment of aPAP and the possibility of WLL as a rescue therapy."

Journal • Review • Immunology • Interstitial Lung Disease • Pulmonary Disease • Rare Diseases • Respiratory Diseases • CSF2