Randomized phase 2a trial assessing a novel septin molecular glue in Alzheimer's disease
Septin 6/7 molecular glue REM127 was assessed in symptomatic participants with Alzheimer's disease (AD). REM127 triggers off-target effects suggesting liver adverse effects. REM127 brain exposure was consistent with saturated target engagement. Biomarker and cognitive outcomes were changed consistent with therapeutic benefit. Septin modulation may restore synaptic function and mitigate pathology in AD.
Authors
- P. Anderer (ORCID: https://orcid.org/0000-0002-3869-6678)
- Emilio Franco‐Macías (ORCID: https://orcid.org/0000-0003-4186-1037)
- Katrien Princen (ORCID: https://orcid.org/0000-0002-7209-6422)
- Suzanne Hendrix (ORCID: https://orcid.org/0000-0002-2374-7727)
- Marc Fivaz (ORCID: https://orcid.org/0000-0003-1003-7934)
- Steven Ramael (ORCID: https://orcid.org/0000-0002-5920-7191)
- Vincent S. Staggs (ORCID: https://orcid.org/0000-0002-6232-9149)
- Clive Ballard (ORCID: https://orcid.org/0000-0003-0022-5632)
- Vincent Hayman
- Craig Mallinckrodt
- Merçé Boada (ORCID: https://orcid.org/0000-0003-2617-3009)
- John Harrison (ORCID: https://orcid.org/0000-0002-0225-4923)
- Xavier Morató (ORCID: https://orcid.org/0000-0002-3012-2864)
- Jordi A. Matías‐Guiu (ORCID: https://orcid.org/0000-0001-5520-2708)
- Marieke Voets (ORCID: https://orcid.org/0000-0003-1329-3407)
- Manfred Windisch (ORCID: https://orcid.org/0000-0003-1490-7933)
- Koen De Witte (ORCID: https://orcid.org/0000-0002-1246-6217)
- Jeffrey Cummings
- Gerard Griffioen (ORCID: https://orcid.org/0000-0003-2492-6046)
- Charlotte Elisabeth Teunissen (ORCID: https://orcid.org/0000-0002-4061-0837)
- Henrik Zetterberg (ORCID: https://orcid.org/0000-0003-3930-4354)
- Juan Pablo Tartari (ORCID: https://orcid.org/0000-0002-7548-2687)
- Lentel Pringels
- Everard G.B. Vijverberg (ORCID: https://orcid.org/0000-0003-3879-884X)
- Anne Corbett (ORCID: https://orcid.org/0000-0003-2015-0316)
- Eveline Debroux (ORCID: https://orcid.org/0009-0005-2718-5580)
- Ann Brinkmalm Westman
- Mieke Nuytten
- Asunción Lafuente
- Sam Dickson
- Eline Byl
Institutions
- Institute of Physics (GB)
- University of Nevada, Las Vegas (US)
- University of Wisconsin–Madison (US)
- University of Exeter (GB)
- Wiltshire College (GB)
- Sahlgrenska University Hospital (SE)
- Instituto de Salud Carlos III (ES)
- Statistics Belgium (BE)
- Universitat Internacional de Catalunya (ES)
- Biomedical Research Networking Center on Neurodegenerative Diseases (ES)
- INC Research (United States) (US)
- Instituto de Investigación Sanitaria del Hospital Clínico San Carlos (ES)
- Amsterdam Neuroscience (NL)
- Cambridge Cognition (United Kingdom) (GB)
- Siesta Group (Austria) (AT)
- Gesundheit Österreich (AT)
- UK Dementia Research Institute (GB)
- Amsterdam University Medical Centers (NL)
- Hospital Universitario Virgen del Rocío (ES)
- TetraLogic Pharmaceuticals (United States) (US)
- University College London (GB)
- Vrije Universiteit Amsterdam (NL)
- KU Leuven (BE)
Publication Details
- Journal
- Alzheimer s & Dementia
- Published
- 2025-09-01
- DOI
- https://doi.org/10.1002/alz.70537
- Citations
- 3
- Primary Topic
- Microbial Metabolic Engineering and Bioproduction
- Type
- article
- Field-Weighted Citation Impact
- 2.42
Funders
- Agentschap Innoveren en Ondernemen
- European Commission
- Vetenskapsrådet
- Vlaamse regering
- HORIZON EUROPE Framework Programme