Journal Articles and Reviews
Lizama BN, Keeling E, Cho E, et al. Sigma-2 receptor modulator CT1812 alters key pathways and rescues retinal pigment epithelium (RPE) functional deficits associated with dry age-related macular degeneration (AMD). Sci Rep 15, 4256 (2025). https://doi.org/10.1038/s41598-025-87921-9
Weissman SA, Kassl CJ, Zegar S, et al. Commercial Route Development of Sigma-2 Receptor Modulator, CT1812 Leveraging Photoflow, and HTS Technologies. Organic Process Research & Development. 29 (2), 373-388 DOI: 10.1021/acs.oprd.4c00412
Croney R, Fristed E, Masterman D, et al. ADNI Private Partners Scientific Board (PPSB) Diversity, Equity, and Inclusion (DE&I) Working Group: A new collaboration that crosses boundaries for industry, academia, and under-represented patients. Alzheimer’s Dement. 2025; e14248. https://doi.org/10.1002/alz.14248
Donkor N, Kiehlbauch CC, Pappenhagen N, et al. Neuroprotective effect of Sigma-2 modulator CT2074 in a mouse model of ocular hypertension. Experimental Eye Research 249 (2024) 110143.
Lizama BN, Willaims C, North HA, et al. CT1812 biomarker signature from a meta-analysis of CSF proteomic findings from two Phase 2 clinical trials in Alzheimer’s disease. Alzheimer’s Dement. 2024; 1-21.
Vijverberg E, de Haan W, Scheijbeler E. et al. A Pilot Electroencephalography Study of the Effect of CT1812 Treatment on Synaptic Activity in Patients with Mild to Moderate Alzheimer’s Disease. J Prev Alzheimers Dis (2024).
Lizama BN, North HA, Pandey K, et al. An interim exploratory proteomics biomarker analysis of a phase 2 clinical trial to assess the impact of CT1812 in Alzheimer’s disease. Neurobiology of Disease 199 (2024).
Colom-Cadena M, Toombs J, Simzer E, Holt K, McGeachan R, et al. Transmembrane protein 97 is a potential synaptic amyloid beta receptor in human Alzheimer’s disease. Acta Neuropathol 147, 32 (2024).
van Dyck, C.H., Mecca, A.P., O’Dell, R.S. et al. A pilot study to evaluate the effect of CT1812 treatment on synaptic density and other biomarkers in Alzheimer’s disease. Alz Res Therapy 16, 20 (2024).
LaBarbera, K.M., Sheline, Y.I., Izzo, N.J. et al. A phase 1b randomized clinical trial of CT1812 to measure Aβ oligomer displacement in Alzheimer’s disease using an indwelling CSF catheter. Transl Neurodegener 2023, 12(24).
Lizama BN, Kahle J, Catalano SM, et al. Sigma-2 Receptors—From Basic Biology to Therapeutic Target: A Focus on Age-Related Degenerative Diseases. Int. J. Mol. Sci. 2023, 24(7), 6251.
Rishton GM, Look GC, Ni Z-J, et al. Discovery of Investigational Drug CT1812, an Antagonist of the Sigma-2 Receptor Complex for Alzheimer’s Disease. ACS Med Chem Lett. 2021 Aug 9;12(9):1389-1395.
LaBarbera KM, Limegrover C, Rehak C, et al. Modeling the mature CNS: A predictive screening platform for neurodegenerative disease drug discovery. J Neurosci Methods. 2021 Jul 1;358:109180.
Izzo NJ, Yuede CM, LaBarbera KM, et al. Preclinical and clinical biomarker studies of CT1812: A novel approach to Alzheimer’s disease modification. Alzheimer’s Dement. 2021 Aug; 17(8):1365-1382
Limegrover CS, Yurko R, Izzo NJ, et al. Sigma‐2 receptor antagonists rescue neuronal dysfunction induced by Parkinson’s patient brain‐derived α‐synuclein. J Neurosci Res. 2021; 00: 1– 16.
Izzo NJ, Colom-Cadena M, Riad AA, et al. Proceedings from the Fourth International Symposium on σ-2 Receptors: Role in Health and Disease. 7(6) ENEURO .0317-20.2020 1–7
Limegrover, CS, LeVine, H, III, Izzo, NJ, et al. Alzheimer’s Protection Effect of A673T Mutation May Be Driven by Lower Aβ Oligomer Binding Affinity. J Neurochem. 2020; 00: 1–15
Colom-Cadena M, Spires-Jones T, Zetterberg H, et al. The clinical promise of biomarkers of synapse damage or loss in Alzheimer’s disease. Alz Res Therapy 12, 21 (2020)
Grundman M, Morgan R, Lickliterd JD, et al. A phase 1 clinical trial of the sigma-2 receptor complex allosteric antagonist CT1812, a novel therapeutic candidate for Alzheimer’s disease. Alzheimers Dement (N Y). 2019 Jan 23; 5:20-26
Izzo NJ, Xu J, Zeng C, et al. Alzheimer’s therapeutics targeting amyloid beta 1-42 oligomers II: Sigma-2/PGRMC1 receptors mediate Abeta 42 oligomer binding and synaptotoxicity PLoS One. 2014 Nov 12; 9(11):e111899
Izzo NJ, Staniszewski A, To L, et al. Alzheimer’s therapeutics targeting amyloid beta 1-42 oligomers I: Abeta 42 oligomer binding to specific neuronal receptors is displaced by drug candidates that improve cognitive deficits. PLoS One. 2014 Nov 12; 9(11):e111898
Medical and Scientific Meeting Presentations
Hamby ME, Kavanagh S, Di Caro V, et al. Positive Impact of CT1812 Treatment on Plasma Biomarkers in Lower p-tau217 Subgroup Aligns with Clinical Benefits in Mild-to-Moderate AD Patients. Poster presented at AD/PD 2025; Vienna, Austria
Di Caro V, Cho E, Lizama B, et al. Identification of Molecular Correlates with CT1812 Treatment-related decrease in NfL CSF Levels Connected to Sigma-2 Receptor. Poster presented at AD/PD 2025; Vienna, Austria
Lizama BN, Pandey K, Duong D, et al. CSF Proteomic Biomarker Analysis from Phase 2 Study SHINE Identified Effects of S2R Modulator CT1812 in Alzheimer’s Disease. Poster presented at AD/PD 2025; Vienna, Austria
Lizama BN, Pandey K, Duong D, et al. Identification of CSF Proteins that Correlate with Cognitive Outcomes in Participants of Phase 2 Study SHINE evaluating Effects of CT1812 in Patients with Alzheimer’s Disease. Poster presented at AD/PD 2025; Vienna, Austria
Galvin JE, Tolea MI, Fargo KN, et al. Results from COG1201: A Proof-of-Concept Study of CT1812 in Participants with Mild-to-Moderate Dementia with Lewy Bodies. Slides presented at International Lewy Body Dementia Conference (ILBDC) 2025; Amsterdam, The Netherlands
Galvin JE, Tolea MI, Iaci JF, et al. SHIMMER: Baseline Data and Early Lessons from the Ongoing Phase 2 Signal-finding Study of CT1812 in Mild-to-Moderate Dementia with Lewy Bodies (DLB). Poster presented at Clinical Trials on Alzheimer’s Disease (CTAD); Madrid, Spain.
Woodward M, Vijverberg EGB, Di Caro V, et al. Results from COG0201: a Randomized, Placebo-controlled, Double-blind, International, Phase 2 Study to Evaluate the Safety and Efficacy of CT1812 in Adults with Mild-to-Moderate Alzheimer’s Disease: Focus on Pre-specified Lower p-tau217 Subgroup. Slides presented at Clinical Trials on Alzheimer’s Disease (CTAD) 2024; Madrid, Spain
Lizama BN, Reaver A, Cho E, et al. Sigma-2 Receptor Modulators Alter Low-density Lipoprotein Receptor-mediated Lipid Trafficking in Retinal Pigmented Epithelial Cells. Poster presented at 2024 Keystone Conference on Dry Age-related Macular Degeneration; Pacific Grove, CA.
DiCaro V, Lizama BN, Cho E, et al. Exploratory CSF Proteomics Biomarker Outcomes of The Phase 2 Clinical Trial SHINE to Assess The Effects of CT1812 in Alzheimer’s Patients. Poster presented at 2024 Alzheimer’s Association International Conference (AAIC); Philadelphia, PA.
Cho E, Caldwell J, Pandey K, et al. CSF Phosphoproteomics Biomarker Analysis from the Phase 2 Clinical Trial SHINE to Elucidate the Role of CT1812 in Alzheimer’s Disease. Poster presented at 2024 Alzheimer’s Association International Conference (AAIC); Philadelphia, PA.
Di Caro V, Cho E, Knezovich N, et al. Top-Line CSF Biomarker Outcomes From The Phase 2 Clinical Trial SHINE In Alzheimer’s Patients. Poster presented at 2024 Alzheimer’s Association International Conference (AAIC); Philadelphia, PA.
Vijverberg EGB, Catalano S, Hamby ME, et al. Results from COG0201: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 2 Study to Evaluate the Safety and Efficacy of CT1812 in Subjects with Mild-to-Moderate Alzheimer’s Disease. Poster presented at 2024 Alzheimer’s Association International Conference (AAIC); Philadelphia, PA.
Caldwell J, Cho E, Caggiano AO, DiCaro V, Hamby ME.Characterization of Human iPSC-derived Neurons as an In Vitro Model to Investigate Sigma-2 Receptor Functions. Poster presented at 2024 Keystone Conference on Neurodegenerative Diseases.
Reaver A, Lizama BN, Caggiano AO, Hamby ME. Differentiated retinal pigment epithelial cells as a model for uncovering sigma-2 receptor functions and novel therapeutics for dry AMD. Poster presented at 2024 Association for Research in Vision & Ophthalmology.
Di Caro V, Pandey K, Cho E, et al. Identification of New Pharmacodynamic Biomarkers of CT1812 that Correlate with Favorable Functional Connectivity of the Brain. e-Poster presented at: AD/PD™ 2024 Alzheimer’s & Parkinson’s Diseases Conference; Lisbon, Portugal.
Lizama B, Duong D, Pandey K, et al. Plasma Proteomic Analysis from Alzheimer’s Patients in SPARC Clinical Trial to Identify Pharmacodynamic Biomarkers of the S2R Modulator CT1812. Poster presented at: AD/PD™ 2024 Alzheimer’s & Parkinson’s Diseases Conference; Lisbon, Portugal.
Di Caro V, Pandey K, Duong D, et al. Analysis of CSF Samples from a Phase 2 Clinical Trial in Alzheimer’s Patients Show that CT1812 can Modulate α-synuclein. Poster presented at: AD/PD™ 2024 Alzheimer’s & Parkinson’s Diseases Conference; Lisbon, Portugal.
Caldwell J, Cho E, Knezovich N, et al. A transcriptomic analysis to investigate the role of sigma-2 receptor modulators CT1812 and CT2168 in a mouse model of Alzheimer’s disease. Poster presented Nov 2023 at: Society for Neuroscience (SfN) annual meeting; Washington DC.
Cho E, Caldwell J, Pandey K, et al. A proteomic analysis to elucidate the role of sigma-2 receptor modulators CT1812 and CT2168 in a mouse model of Alzheimer’s disease. Poster presented Nov 2023 at: Society for Neuroscience (SfN) annual meeting; Washington DC.
Reaver AJ, Lizama BN, Pandey K, et al. Exploratory omics study reveals potential roles of sigma-2 receptor modulators in AAV1/2A53T-aSyn rat model of Parkinson’s disease. Poster presented Nov 2023 at: Society for Neuroscience (SfN) annual meeting; Washington DC.
Lizama BN, Reaver AJ, Pandey K, et al. Unbiased proteomic and transcriptomic analysis of Sigma-2 receptor modulation in an in vivo model of synucleinopathy. Poster presented Nov 2023 at: Society for Neuroscience (SfN) annual meeting; Washington DC.
Di Caro V, Pandey K, Lizama B, et al. Proteomic Analysis in a Phase 2 Clinical Trial Studying CT1812 to Identify CSF and Plasma Pharmacodynamic Biomarkers and Molecular Correlates of EEG in Alzheimer’s Patients. Poster presented Oct 2023 at: Clinical Trials on Alzheimer’s Disease (CTAD); Boston, Massachusetts.
Lizama B, Cho E, Duong D, et al. Proteomic Analysis of Plasma in a Phase 2 Clinical Trial in Alzheimer’s Patients to Identify Pharmacodynamic Biomarkers of the S2R Modulator CT1812. Poster presented Oct 2023 at: Clinical Trials on Alzheimer’s Disease (CTAD); Boston, Massachusetts.
Di Caro V, Cho E, Caldwell J, et al. Brain transcriptomic and proteomic analyses in an in vivo AD model further elucidate the role of the sigma-2 receptor modulator CT1812 in Alzheimer’s disease. Poster presented at: 2023 Alzheimer’s Association’s International Conference (AAIC); Amsterdam, The Netherlands.
Pandey K, Duong D, Waybright L, et al. Pharmacodynamic Effects of the S2R Modulator CT1812 in Alzheimer’s Disease (AD) Patients Observed in a Meta-analysis of CSF Proteomes from SPARC and SHINE Part A. Poster presented at: AD/PD™ 2023 International Conference on Alzheimer’s and Parkinson’s Diseases
Lizama B, Waybright L, Malagise E, et al. Unbiased Transcriptomic Analysis of the sigma-2 Modulator, CT1812 in Cell-based Models of Dry Age-related Macular Degeneration. Poster presented at: 2023 Association for Research in Vision & Ophthalmology
Galvin JE, Tolea MI, Hamby ME, et al. A Randomized, Double-blind, Placebo-controlled, Phase 2, Six-month Study to Evaluate the Safety, Tolerability and Exploratory Efficacy of CT1812 in Participants with Mild-to-Moderate Dementia with Lewy Bodies. Poster presented at: 2022 Clinical Trials on Alzheimer’s Disease Congress (CTAD); San Francisco, CA.
Pandey K, Waybright L, Duong DM, et al. CSF Proteomics Biomarker Analysis from the SPARC Clinical Trial: to Assess the Effect of the Sigma-2 Receptor (S2R) Modulator CT1812 in Alzheimer’s Disease Patients. Oral presentation at: 2022 Alzheimer’s Association’s International Conference (AAIC); San Diego, CA.
Seyfried N, Waybright L, Duong D, et al. Proteomic Analysis of CSF in a Phase 2 Clinical Trial for AD to Identify Pharmacodynamic Biomarkers of the S2R Modulator CT1812. Poster presented at: AD/PD™ 2022 International Conference on Alzheimer’s and Parkinson’s Diseases; Barcelona, Spain.
Malagise E, Keeling E, Knezovich N, et al. Sigma-2 receptor modulators rescue POS trafficking deficits in RPE cell-based models of dry AMD. Poster presented at: 2022 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO); Denver, CO.
Izzo NJ, LaBarbera KM, Yuede CM, et al. Experimental Therapeutic CT1812 Demonstrates Target Engagement in a Phase 1b Clinical Trial to Measure Displacement of Aβ Oligomers Into CSF. Poster presented at: AD/PD™ 2022 International Conference on Alzheimer’s and Parkinson’s Diseases; Barcelona, Spain.
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