Two randomised controlled phase 2 studies of the oral neutrophil elastase inhibitor alvelestat in alpha-1 antitrypsin deficiency
Alvelestat effectively suppressed NE and its activity at both doses, but only the 240 mg twice-daily dose demonstrated relevant efficacy compared to placebo on disease activity biomarkers with a favourable safety profile. These findings support progression of the 240 mg twice-daily dose into a clinical end-point study.
Authors
- Vivian Lin (ORCID: https://orcid.org/0000-0003-2300-826X)
- Elizabeth Westfall
- Inmaculada Aban (ORCID: https://orcid.org/0000-0002-0563-1691)
- Wissam Chatila (ORCID: https://orcid.org/0000-0002-5276-5166)
- Igor Barjaktarević (ORCID: https://orcid.org/0000-0002-8096-0858)
- Hanan A. Tanash (ORCID: https://orcid.org/0000-0002-0913-2347)
- Hui‐Chien Kuo (ORCID: https://orcid.org/0000-0003-2159-7216)
- C.S. Pirozzi (ORCID: https://orcid.org/0000-0002-1533-1916)
- Kenneth R. Chapman (ORCID: https://orcid.org/0000-0002-2498-9859)
- Jeanine D’Armiento (ORCID: https://orcid.org/0000-0002-6219-6083)
- Mark T. Dransfield (ORCID: https://orcid.org/0000-0002-9207-1820)
- Robin J. Parks (ORCID: https://orcid.org/0000-0002-4959-2233)
- Jamie Inshaw
- Kelly Moffitt
- Umur Hatipoğlu (ORCID: https://orcid.org/0000-0002-0863-6471)
- Monica Goldklang (ORCID: https://orcid.org/0000-0002-7019-3130)
- Robert A. Sandhaus (ORCID: https://orcid.org/0000-0002-7937-2485)
- Alice Margaret Turner (ORCID: https://orcid.org/0000-0002-5947-3254)
- Jacqueline Parkin
- Adzoa Ekue
- Ingrid Louise Titlestad (ORCID: https://orcid.org/0000-0001-6418-0088)
- Michael Drummond (ORCID: https://orcid.org/0000-0002-6968-4610)
- J. Michael Wells (ORCID: https://orcid.org/0000-0001-9612-5454)
- Charlie Strange
- Megan Devine (ORCID: https://orcid.org/0000-0003-3279-1110)
- R A Stockley
Institutions
- University of North Carolina at Chapel Hill (US)
- Cleveland Clinic (US)
- Medical University of South Carolina (US)
- University of California, Los Angeles (US)
- University of Southern Denmark (DK)
- University of Toronto (CA)
- University of Utah (US)
- Queen Elizabeth Hospital Birmingham (GB)
- Odense University Hospital (DK)
- National Jewish Health (US)
- University of Alabama at Birmingham (US)
- CTI BioPharma (United Kingdom) (GB)
- Birmingham VA Medical Center (US)
- Romax Technology (United Kingdom) (GB)
- Skåne University Hospital (SE)
- Exploristics (United Kingdom) (GB)
- Columbia University (US)
- University of Birmingham (GB)
- Temple University (US)
- The University of Texas Health Science Center at Tyler (US)
- University of Colorado Denver (US)
Publication Details
- Journal
- European Respiratory Journal
- Published
- 2025-09-18
- DOI
- https://doi.org/10.1183/13993003.01019-2025
- Citations
- 6
- Primary Topic
- Protease and Inhibitor Mechanisms
- Type
- article
- Field-Weighted Citation Impact
- 5.62