Trial reportAlzheimer's & dementia : the journal of the Alzheimer's Association2025
Randomized phase 2a trial assessing a novel septin molecular glue in Alzheimer's disease.
Trial report in Alzheimer's & dementia : the journal of the Alzheimer's Association, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05478031 (A Randomized, Placebo-controlled, Double-blind, Parallel-group Phase 2a Exploratory Study With Placebo run-in to Investigate PK/PD Effects, Safety, Tolerability and Pharmacokinetics of REM0046127 Oral Suspension Compared With Placebo in Subjects With Mild to Moderate Alzheimer's Disease), which is not on this map. Cited by 2 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Randomized, Placebo-controlled, Double-blind, Parallel-group Phase 2a Exploratory Study With Placebo run-in to Investigate PK/PD Effects, Safety, Tolerability and Pharmacokinetics of REM0046127 Oral Suspension Compared With Placebo in Subjects With Mild to Moderate Alzheimer's Disease
Who cites it
2 citing papers in PubMed.
- Randomized phase 2a trial assessing a novel septin molecular glue in Alzheimer's disease.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2025Trial
- Alzheimer's disease: from molecular pathways to therapies.Molecular biomedicine · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
31 authors.
Funding
Abstract
introductionPharmacological restoration of septin filament integrity has the potential to provide symptomatic benefit and disease modification in Alzheimer's disease (AD).
methodsREM127, a septin modulator, was assessed in mild-to-moderate AD (EudraCT: 2022-000080-43) in a phase 2a trial (n = 14). PRIMARY ENDPOINTS: safety and tolerability; exploratory endpoints: pharmacokinetics, cerebrospinal fluid (CSF) biomarkers, electroencephalography (EEG), and functional outcomes.
resultsIn participants on active therapy, dose-dependent increases in serum aminotransferase were observed, leading to study discontinuation. CSF hyperphosphorylated tau (P-tau181), endpoints reflecting synaptic function and cognitive outcomes, were changed significantly (p < 0.05) to normal compared to placebo. DISCUSSION: REM127 triggers off-target liver adverse effects. Anticipated on-target outcomes suggest septin modulation has symptomatic benefit and modifies processes underlying AD. Results are considered exploratory as statistical power is constrained due to the small sample size caused by early termination. Further investigation of the therapeutic concept using an optimized septin molecular glue with an improved safety profile is warranted. HIGHLIGHTS: 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.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.