Cited by 1Alzheimer's disease research and treatmentsDementia and Cognitive Impairment ResearchPharmacological Receptor Mechanisms and Effects

The DOI matches its Crossref record; 44 of the 44 references it cites were found in a registry or an index; 20 of the 65 sources its claims cite were checked for support.

Impact of zervimesine on the neuroinflammatory biomarker GFAP and related proteomic molecular correlates in plasma of participants from a phase 2 clinical trial in Alzheimer’s disease

Open access

Abstract

Background: Zervimesine (CT1812) is an investigational brain-penetrant small molecule modulator of the sigma-2 receptor (S2R/ TMEM97 ), currently in clinical development for the treatment of Alzheimer’s disease (AD) and dementia with Lewy bodies (DLB) that selectively prevents and displaces the binding of amyloid beta (Aβ) and α-synuclein oligomers from neuronal synapses. Given the mechanism of action, it was hypothesized that zervimesine might be more effective in patients with lower levels of AD pathology. Indeed, in the SHINE trial, a completed Phase 2, randomized, double-blind, clinical trial conducted in participants with AD, a robust, 95%, slowing of cognitive decline, as assessed via ADAS-Cog11, was observed in a pre-specified subgroup of participants with lesser AD pathology (i.e., low p-tau217 subgroup) compared to a 38% slowing in the overall modified intent-to-treat (mITT)) population. Methods: In SHINE, exploratory plasma biomarkers were assessed using both a targeted and an unbiased, proteomics discovery approach in plasma from participants at baseline and end of study. Treatment effects of zervimesine relative to placebo were assessed in both the mITT population and in a low p-tau217 subgroup, who entered the study with lower (< 1pg/ml) plasma p-tau217 concentrations. Plasma biomarkers Aβ40, Aβ42, GFAP, NfL and BD-tau were assessed using clinically validated targeted assays, along with untargeted TMT-mass spectrometry (MS)-based discovery proteomics followed by bioinformatic, pathway and correlation analyses. Results: Collectively, plasma biomarker findings in the low p-tau217 subgroup were more robust than in the mITT population. Levels of GFAP were significantly decreased and NfL, Aβ42 and Aβ40 levels trended towards a decrease with zervimesine compared to placebo. Proteomics analyses identified candidate pharmacodynamic biomarkers of zervimesine, and gene ontology and pathway analyses pointed to an impact on amyloid biology, trafficking, lipid metabolism, and immune response. Bioinformatics and correlation analyses with GFAP identified biomarkers that may reflect pathway engagement of S2R and/or decreased neuroinflammation. Conclusions: Exploratory plasma biomarker findings align with the degree of clinical benefit in SHINE mITT and low p-tau217 populations, and support future trial enrichment with patients with lower levels of pathology as defined by lower baseline plasma levels of p-tau217. Trial registration: July 20th, 2018 ClinicalTrials.gov Identifier NCT03507790 https://clinicaltrials.gov/study/NCT03507790 .

Overview

AI snapshot

Written from the paper’s full text.

TL;DR

In the SHINE Phase 2 trial, exploratory plasma biomarker and proteomic analyses showed that zervimesine treatment effects were more robust in participants with low baseline plasma p-tau217 (<1 pg/ml) than in the overall mITT population. In that low p-tau217 subgroup, plasma GFAP significantly decreased with zervimesine versus placebo, with trends toward decreases in Aβ42, Aβ40, and NfL.

Key findings

  • In the low p-tau217 subgroup, zervimesine significantly decreased plasma GFAP versus placebo (LS mean −28.35; SE 11.687; p = 0.0186), with trends (p ≤ 0.10) toward reductions in Aβ42, Aβ40, and NfL; no treatment effects or trends were observed in the high p-tau217 subgroup (N = 60).
  • In the mITT population, only plasma BD-tau showed a trend toward reduction versus placebo (p = 0.09); no treatment-related changes were observed in GFAP, NfL, Aβ42, or Aβ40, and no change was seen in Aβ42/40 ratio (LS mean 0.0019; SE 0.00148; p = 0.2048).
  • TMT-MS proteomics detected more than 2000 proteins in mITT plasma; 58 proteins were significantly differentially abundant with zervimesine (53 increased, 5 decreased; p ≤ 0.05), while 103 were differentially abundant in the low p-tau217 subgroup (75 increased, 28 decreased; p ≤ 0.05). Pathway analyses implicated amyloid biology, trafficking, lipid metabolism, and immune response.
  • In the low p-tau217 subgroup, 286 TMT-MS proteins correlated with plasma GFAP in zervimesine-treated participants; after removing 3 shared with placebo, 283 “Drug Selective” correlates remained, and 15 of these were also differentially abundant (zervimesine vs placebo, p ≤ 0.10), including GPX3 and CTSG.

Methods

  • SHINE (NCT03507790) was a completed international, multicenter, randomized, double-blind, placebo-controlled 36-week Phase 2 trial in adults aged 50–85 years with mild to moderate AD (MMSE 18–26); participants were randomized 1:1:1 to placebo, 100 mg, or 300 mg zervimesine orally once daily for 6 months. Plasma biomarker analyses included participants with baseline and 6-month plasma (mITT N = 117; low p-tau217 subgroup N = 57; high p-tau217 subgroup N = 60), with p-tau217 subgroups defined by baseline plasma p-tau217 below versus at/above the median of 1.0 pg/ml.
  • Targeted plasma biomarkers Aβ40, Aβ42, GFAP, and NfL were measured with the SIMOA Neuro4-Plex E Kit, and brain-derived tau with a SIMOA-based assay; change from baseline was calculated.
  • Untargeted discovery proteomics used TMT-MS on the first 57 treatment-compliant participants (n = 20 placebo, n = 37 pooled zervimesine) with baseline and 6-month plasma; low p-tau217 subgroup N = 24 (n = 8 placebo, n = 16 zervimesine). Differential abundance was assessed by ANOVA with unadjusted p ≤ 0.05, followed by GO, STRING, MetaCore pathway analyses, and Pearson correlation of protein change from baseline with plasma GFAP change from baseline in the low p-tau217 subgroup.

Limitations

  • The paper states limited sample size, with even smaller subgroups, which may affect differential abundance analysis and lead to false positives or negatives.
  • The paper states that proteomics differential abundance and correlation analyses used unadjusted p-values without controlling for multiple comparisons, increasing type 1 error risk; candidate biomarkers should be viewed with caution until replicated.
  • The paper states that the p-tau217 subgroup analysis was prespecified but not the primary analysis, and that plasma biomarkers may reflect peripheral changes rather than brain-relevant disease processes; replication in larger trials is warranted.

Model-written summary. Check it against the paper before relying on it.

Integrity signals

Registry

Crossref · 10.1186/s13195-026-02025-4

Cites

44 of 44 references verified0 citation errors · 0 not checkable

Statistics

Nothing to recompute.

Verification

Claims the paper makes

20 of 65 cited sources checked · 5 supported · 0 flagged · accuracy not yet measured

Show all 69 claims

  • Probably not a substantial claim. 19
  • It may state two separate claims as one. 28
  • The quote only partly states this claim. 3
38 of 38
  • The paper relies on this without support.

    Zervimesine is a modulator of the sigma-2 receptor (S2R/TMEM97).

    “Zervimesine (CT1812) is an orally administered small molecule modulator of the sigma-2 receptor (S2R/TMEM97) in clinical development for treatment of Alzheimer’s disease (AD; NCT03507790; NCT05531656) and dementia with Lewy bodies (DLB; NCT05225415).”
  • The paper relies on this without support.

    GFAP is a neuroinflammatory biomarker.

    “the neuroinflammatory marker GFAP”
  • The paper relies on this without support.

    NfL is a recognized biomarker of neurodegeneration for multiple neurodegenerative disorders, including AD, in which CSF levels are elevated relative to healthy non-demented control.

    “NfL is a recognized biomarker of neurodegeneration for multiple neurodegenerative disorders, including AD, in which CSF levels are elevated relative to healthy non-demented control.”
  • The paper relies on this without support.

    HSPA5 and HSP90AB1 are members of the heat shock protein family and as chaperone proteins are involved in controlling protein folding and ER homeostasis.

    “HSPA5, HSP90AB1are both members of the heat shock protein family and as chaperone proteins are involved in controlling protein folding and ER homeostasis.”
  • The paper relies on this without support.

    Elevated secreted GFAP levels is a well-established marker of astrogliosis, detectable in plasma due to blood-brain-barrier disruption, and elevated in plasma of patients with AD.

    “Elevated secreted GFAP levels is a well-established marker of astrogliosis, detectable in plasma due to blood-brain-barrier disruption, and elevated in plasma of patients with AD.”
5 of 5

Self-check

Percentages, sums and reported differences re-computed from the paper’s own numbers.

Each contradiction between two passages listed here was confirmed by an independent check.

  • Conflicting differential-abundance threshold for the 15 GFAP correlates

    Minor

    The results say the 15 proteins met a differential-abundance threshold of p ≤ 0.10, while the discussion describes the same 15 as meeting p < 0.05. The stricter threshold is not supported by the reported comparison.

1 of 1

Citations

Cites

Flagged first, then by citations

Show all 44 references

Research works printed but not linked: 7

  1. Ashton NJ, Brum WS, Molfetta GD, Benedet AL, Arslan B, Jonaitis E, et al. Diagnostic accuracy of a plasma phosphorylated tau 217 immunoassay for Alzheimer disease pathology. JAMA Neurol. 2024;81:255–63. https://doi.org/10.1001/jamaneurol.2023.5319. · DOI · Paper page
  2. Sperling RA, Selkoe DJ, Reyderman L, Cao Y, van Dyck CH. Does the Current Evidence Base Support Lecanemab Continued Dosing for Early Alzheimer’s Disease? [Perspectives Session]. Alzheimer’s Assoc Int Conf. Philadelphia, PA; 2024.
  3. Cognition Therapeutics. Cognition Therapeutics Study of Zervimesine (CT1812) in Early Alzheimer’s Disease Surpasses 50% Enrollment Target [Press Release]. 2025 Jul 1; https://ir.cogrx.com/press_releases/cognition-therapeutics-study-of-zervimesine-ct1812-in-early-alzheimers-disease-surpasses-50-enrollment-target/.
  4. Cognition Therapeutics. Cognition Therapeutics’ Analysis Correlates Biomarker Changes with Cognitive Benefit in Alzheimer’s Population [Press Release]. 2024 Nov 25; https://ir.cogrx.com/press_releases/cognition-therapeutics-analysis-correlates-biomarker-changes-with-cognitive-benefit-in-alzheimers-population/.
  5. Cognition Therapeutics. Cognition Therapeutics presents phase 3 plan for zervimesine (CT1812) in Alzheimer’s disease at Clinical Trials on Alzheimer’s Disease (CTAD) conference. [Press Release]. 2025 Dec 1; https://ir.cogrx.com/press_releases/cognition-therapeutics-presents-phase-3-plan-for-zervimesine-ct1812-in-alzheimers-disease-at-clinical-trials-on-alzheimers-disease-ctad-conference/.
  6. Lilly. Results from Lilly’s Landmark Phase 3 Trial of Donanemab Presented at Alzheimer’s Association Conference and Published in JAMA [Press Release]. 2023 Jul 17; https://investor.lilly.com/news-releases/news-release-details/results-lillys-landmark-phase-3-trial-donanemab-presented.
  7. Eisai. New Clinical Data Demonstrates Three Years of Continuous Treatment with Dual-Acting LEQEMBI® (lecanemab-irmb) Continues to Significantly Benefit Early Alzheimer’s Disease Patients Presented at AAIC 2024. [Press Release]. 2024. https://www.eisai.com/news/2024/news202456.html.

Cited by

Sorted by citations

See all 1

Metadata

Authors
Di Caro, V., Cho, E., Thiel, J., Lizama, B. N., Koel-Simmelink, M. J. A., Pandey, K., Duong, D., Seyfried, N. T., Grundman, M., Teunissen, C. E., Zetterberg, H., Blennow, K., Caggiano, A. O., & Hamby, M. E.
Affiliations
  • Cognition Therapeutics (United States) (US)
  • Amsterdam UMC Location Vrije Universiteit Amsterdam (NL)
  • Emory University (US)
  • University of California San Diego (US)
  • University of Wisconsin–Madison (US)
  • Sahlgrenska University Hospital (SE)
Corresponding author
Hamby, Mary E.
Venue
Alzheimer's Research & Therapy
Published
2026-04-06
Volume
18
Issue
1
ISSN
1758-9193 (ISSN-L)
OpenAlex
W7151006868
Cited by
1
References
44
Citations by year
2026: 1
FWCI
1.66
Type
journal article
Language
en
Open access
Locations
  • Alzheimer s Research & Therapy (publishedVersion, cc-by)
  • PubMed (publishedVersion)
  • PubMed Central (publishedVersion, cc-by)
  • Pure Amsterdam UMC (publishedVersion, cc-by)
  • DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
Funders
  • National Institute on Aging (1R01AG058660)
  • Alzheimer's Drug Discovery Foundation
Topics
  • Life Sciences › Neuroscience › Cellular and Molecular Neuroscience › Alzheimer's disease research and treatments
  • Health Sciences › Medicine › Psychiatry and Mental health › Dementia and Cognitive Impairment Research
  • Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmacology › Pharmacological Receptor Mechanisms and Effects
Keywords
Alzheimer's disease, sigma-2 receptor, TMEM97, neuroinflammation, glial fibrillary acidic protein, plasma biomarkers, phosphorylated tau, amyloid-beta, neurofilament light chain, pharmacodynamic biomarkers, dementia with Lewy bodies, amyloid-beta binding, lipid metabolism, immune response, SHINE trial
MeSH terms
Neuroinflammatory Diseases, Aged, Alzheimer Disease, Double-Blind Method, Female, Glial Fibrillary Acidic Protein, Humans, Male, Biomarkers, Amyloid beta-Peptides, Proteomics

About these checks

Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.

A language model drew the claims from the paper on 2026-11-10, under version 3 of the claim definition (model deepseek-v4.1-flash, prompt extract-v3).

A second model labelled each claim on 2026-11-10 (model jev-1.13-20260917, definition revision gold-guideline-v0.2).

Cited sources are checked with a language model’s help (gpt-6-sol).

Statistics and arithmetic are recomputed, not judged.

Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.

What counts as a claim ›How we measure confidence ›How we check this ›

Are you an author, or do you see a problem with this page? Request a review