Savara Announces Manuscript on Long-Term Outcomes With Molgramostim Inhalation Solution (Molgramostim) in Autoimmune Pulmonary Alveolar Proteinosis (aPAP) Published in ERJ Open Research
Savara Inc. (SVRA) announced the publication of a manuscript in ERJ Open Research detailing long-term outcomes of molgramostim inhalation solution in treating autoimmune pulmonary alveolar proteinosis (aPAP). The retrospective study evaluated five aPAP patients who received molgramostim through European single-patient access over a mean duration of 4.2 years.
The study demonstrated significant improvements including enhanced lung function, decreased disease burden, and reduced clinical symptoms. Key findings showed improvements in pulmonary gas transfer and activities of daily living. Notably, while four patients required whole lung lavage (WLL) before treatment, none needed it after one year of molgramostim use. The treatment was well-tolerated with no serious adverse events reported.
Savara plans to complete its rolling Biologics License Application (BLA) submission in the U.S. by the end of Q1 2025 and submit a Marketing Authorization Application (MAA) in Europe by the end of 2025.
Savara Inc. (SVRA) ha annunciato la pubblicazione di un manoscritto in ERJ Open Research che dettaglia i risultati a lungo termine della soluzione per inalazione di molgramostim nel trattamento della proteinosi alveolare autoimmune (aPAP). Lo studio retrospettivo ha valutato cinque pazienti con aPAP che hanno ricevuto molgramostim tramite accesso individuale in Europa per una durata media di 4,2 anni.
Lo studio ha dimostrato miglioramenti significativi, tra cui un aumento della funzionalità polmonare, una riduzione del carico della malattia e una diminuzione dei sintomi clinici. I risultati chiave hanno mostrato miglioramenti nel trasferimento di gas polmonare e nelle attività della vita quotidiana. È importante notare che, mentre quattro pazienti richiedevano lisi polmonare totale (WLL) prima del trattamento, nessuno di essi ne ha avuto bisogno dopo un anno di utilizzo di molgramostim. Il trattamento è stato ben tollerato, senza segnalazioni di eventi avversi gravi.
Savara prevede di completare la sua domanda di Licenza Biologica (BLA) negli Stati Uniti entro la fine del primo trimestre del 2025 e di presentare una domanda di Autorizzazione al Mercato (MAA) in Europa entro la fine del 2025.
Savara Inc. (SVRA) anunció la publicación de un manuscrito en ERJ Open Research que detalla los resultados a largo plazo de la solución de inhalación de molgramostim en el tratamiento de la proteinosis alveolar autoinmune (aPAP). El estudio retrospectivo evaluó a cinco pacientes con aPAP que recibieron molgramostim a través de acceso individual en Europa durante una duración media de 4,2 años.
El estudio demostró mejoras significativas, incluyendo una mejor función pulmonar, reducción de la carga de la enfermedad y disminución de los síntomas clínicos. Los hallazgos clave mostraron mejoras en la transferencia de gases pulmonares y en las actividades de la vida diaria. Notablemente, mientras que cuatro pacientes requerían lavado pulmonar total (WLL) antes del tratamiento, ninguno lo necesitó después de un año de uso de molgramostim. El tratamiento fue bien tolerado sin reportar eventos adversos graves.
Savara planea completar su solicitud de Licencia Biológica (BLA) en EE. UU. para finales del primer trimestre de 2025 y presentar una Solicitud de Autorización de Comercialización (MAA) en Europa para finales de 2025.
사바라 Inc. (SVRA)는 자가면역 폐 알베올리 단백질 침착증 (aPAP) 치료에서 몰그램스팀 흡입 용액의 장기적인 결과를 자세히 설명하는 원고를 ERJ Open Research에 발표했다고 발표했습니다. 이 회고적 연구는 평균 4.2년 동안 유럽의 단일 환자 접근을 통해 몰그램스팀을 받은 5명의 aPAP 환자를 평가했습니다.
이 연구는 폐 기능 향상, 질병 부담 감소 및 임상 증상 감소를 포함한 중요한 개선 사항을 입증했습니다. 주요 발견은 폐 가스 전이 및 일상 생활 활동에서의 개선을 보여주었습니다. 특히, 네 명의 환자가 치료 전에는 전폐 세척 (WLL)을 필요로 했지만, 몰그램스팀 사용 1년 후에는 아무도 그것을 필요로 하지 않았습니다. 치료는 잘 견딜 수 있었으며 심각한 부작용 보고는 없었습니다.
사바라는 2025년 1분기 말까지 미국에서 생물학적 제제 허가 신청 (BLA)을 완료하고, 2025년 말까지 유럽에서 마케팅 승인 신청 (MAA)을 제출할 계획입니다.
Savara Inc. (SVRA) a annoncé la publication d'un manuscrit dans ERJ Open Research détaillant les résultats à long terme de la solution d'inhalation de molgramostim dans le traitement de la protéinoses alvéolaire auto-immune (aPAP). L'étude rétrospective a évalué cinq patients aPAP ayant reçu du molgramostim par le biais d'un accès individuel en Europe pendant une durée moyenne de 4,2 ans.
L'étude a montré des améliorations significatives, notamment une fonction pulmonaire accrue, une réduction de la charge de la maladie et des symptômes cliniques diminués. Les résultats clés ont montré des améliorations dans le transfert gazeux pulmonaire et les activités de la vie quotidienne. Notamment, alors que quatre patients nécessitaient un lavage pulmonaire total (WLL) avant le traitement, aucun ne l'a nécessité après un an d'utilisation de molgramostim. Le traitement a été bien toléré, sans rapports d'événements indésirables graves.
Savara prévoit de finaliser sa demande de Licence biopharmaceutique (BLA) aux États-Unis d'ici la fin du premier trimestre 2025 et de soumettre une Demande d'Autorisation de Mise sur le marché (MAA) en Europe d'ici la fin de 2025.
Savara Inc. (SVRA) gab die Veröffentlichung eines Manuskripts in ERJ Open Research bekannt, das die langfristigen Ergebnisse der Inhalationslösung Molgramostim bei der Behandlung der autoimmunen alveolären Proteinosis (aPAP) detailliert beschreibt. Die retrospektive Studie bewertete fünf aPAP-Patienten, die Molgramostim über den europäischen Einzelpatientenzugang über einen Durchschnittszeitraum von 4,2 Jahren erhalten hatten.
Die Studie zeigte signifikante Verbesserungen, einschließlich einer verbesserten Lungenfunktion, einem verringerten Krankheitsausmaß und reduzierten klinischen Symptomen. Zu den wichtigsten Ergebnissen gehörten Verbesserungen im pulmonalen Gastransfer und den Aktivitäten des täglichen Lebens. Bemerkenswerterweise benötigten vier Patienten vor der Behandlung eine vollständige Lungenwaschung (WLL), während dies nach einem Jahr Molgramostim-Anwendung bei keinem mehr erforderlich war. Die Behandlung wurde gut vertragen, ohne dass schwere Nebenwirkungen berichtet wurden.
Savara plant, die Einreichung ihrer fortlaufenden Biologics License Application (BLA) in den USA bis Ende des ersten Quartals 2025 abzuschließen und eine Marketinggenehmigungsanfrage (MAA) in Europa bis Ende 2025 einzureichen.
- Treatment showed effectiveness in improving lung function and reducing disease burden
- Eliminated need for whole lung lavage procedures after one year of treatment
- No serious adverse events reported, demonstrating good safety profile
- Clear regulatory pathway with BLA and MAA submissions planned for 2025
- Small sample size of only five patients in the study
- Long regulatory timeline with submissions not until 2025
Insights
The publication in ERJ Open Research marks a pivotal development for Savara's molgramostim program, presenting compelling evidence for its potential as a first-in-class treatment for aPAP. The data reveals several critical clinical benefits:
- Sustained improvement in lung function metrics (DLco and A-aDO2)
- Elimination of whole lung lavage requirements after one year of treatment
- Reduction in surfactant burden confirmed through imaging
- Strong safety profile with no serious adverse events
While the sample size is , the extended treatment duration of 4.2 years provides robust evidence for long-term efficacy and safety. The elimination of whole lung lavage procedures is particularly significant, as this represents a major burden in current standard of care.
The timing of this publication strategically aligns with Savara's regulatory submissions, with the BLA submission planned for Q1 2025 and MAA submission by end of 2025. Given the rare disease status of aPAP and the absence of approved pharmaceutical treatments, molgramostim could potentially secure a significant first-mover advantage in both U.S. and European markets.
The data's publication in a peer-reviewed journal strengthens Savara's regulatory position and could facilitate faster review processes, given the clear unmet medical need in aPAP treatment. The demonstrated ability to address the underlying pathophysiology, rather than just managing symptoms, positions molgramostim as a potential paradigm-shifting therapy in this rare disease space.
-- Real World, Retrospective Outcomes Data Suggest Molgramostim Addresses the Underlying Pathophysiology of aPAP Resulting in Improved Lung Function, Decreased Disease Burden, Restored Patient Functionality, and Reduction of Clinical Symptoms--
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
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 www.savarapharma.com, X: @SavaraPharma 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. 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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Media and Investor Contact:
Savara Inc.
Temre Johnson, Executive Director, Corporate Affairs
ir@savarapharma.com
Source: Savara Inc.
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