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Table 3 Repurposed agents currently in Phase II in the Alzheimer’s disease development pipeline (as of February 27, 2020)

From: Repurposed agents in the Alzheimer’s disease drug development pipeline

Agent name Phase Agent mechanism class Therapeutic purpose under study Drug class FDA-approved indication(s) Therapeutic field ClinicalTrials.gov ID(s) (EudraCT ID)
Benfotiamine II Metabolic Improve multiple cellular processes; minimize decline in glucose utilization (DMT) Synthetic thiamine Diabetic nephropathy; type 2 diabetes Antidiabetic NCT02292238
Candesartan II Anti-amyloid, neuroprotective, metabolic Improve vascular function and reduce amyloid (DMT) Angiotensin II Receptor Blocker Hypertension; heart failure Cardiovascular NCT02646982
Cilostazol II Neuroprotective Reduce accumulation of amyloid and reduce tau phosphorylation; improve cerebral circulation (DMT) Antiplatelet Intermittent claudication Hematologic-oncologic NCT02491268
Dapagliflozin I/II Metabolic Improve insulin sensitivity (DMT) Sodium-glucose co-transporter 2 inhibitor Adjunct therapy for type 2 diabetes Antidiabetic NCT03801642
Daratumumab II Anti-inflammatory Broad range immunomodulatory effects; regulate microglia (DMT) Human antibody targeting CD38 Multiple myeloma Hematologic-oncologic NCT04070378
Dasatinib + Quercetin (Combo) I/II Anti-inflammatory Senolytic therapy approach to reduce senescent cells and tau aggregation (DMT) Tyrosine kinase inhibitor + antioxidant/anti-inflammatory (Flavonoid) Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase Hematologic-oncologic NCT04063124
Deferiprone II Anti-Amyloid, neuroprotective Reduce reactive oxygen species that damage neurons; effect on amyloid and BACE pathology (DMT) Iron chelator Iron overload (thalassemia syndromes) Hematologic-oncologic NCT03234686
Dronabinol II Neurotransmitter-based Improve neuropsychiatric symptoms (agitation) Cannabinoid Anorexia (associated with AIDS); nausea/vomiting (associated with chemotherapy) Cannabinoid NCT02792257
Insulin glulisine II Metabolic Enhance cell signaling and growth; promote neuronal metabolism (DMT) Insulin Diabetes Antidiabetic NCT02503501
Lenolidomide II Anti-inflammatory Reduce inflammatory cytokines (TNF-a, IL-6, IL-8); reduce inflammatory and AD-associated biomarkers; improve cognition (DMT) Anti-neoplastic Multiple myeloma Hematologic-oncologic NCT04032626
Levetiracetam II Neuroprotective Reduce amyloid induced neuronal hyperactivity (DMT) Anticonvulsant Adjunct therapy for partial-onset seizures; juvenile myoclonic epilepsy; primary generalized tonic-clonic seizures Neurologic NCT03489044
(2016–003109-32)
Levetiracetam II Neuroprotective Reduce amyloid induced neuronal hyperactivity (DMT) Anticonvulsant Adjunct therapy for partial-onset seizures; juvenile myoclonic epilepsy; primary generalized tonic-clonic seizures Neurologic NCT02002819
Levetiracetam II Neuroprotective Reduce amyloid induced neuronal hyperactivity (DMT) Anticonvulsant Adjunct therapy for partial-onset seizures; juvenile myoclonic epilepsy; primary generalized tonic-clonic seizures Neurologic NCT03875638
Levetiracetam (AGB101) II Neuroprotective Reduce amyloid induced neuronal hyperactivity (DMT) Anticonvulsant Adjunct therapy for partial-onset seizures; juvenile myoclonic epilepsy; primary generalized tonic-clonic seizures Neurologic NCT03461861
Liraglutide II Metabolic Enhance cell signaling; improve CNS glucose metabolism (DMT) Glucagon-like peptide-1 agonist Adjunct therapy for type 2 diabetes; risk reduction of major cardiovascular events Antidiabetic NCT01843075 (2013-000962-13)
Lithium II Neurotransmitter-based Improve neuropsychiatric symptoms (agitation/aggression with or without psychosis) Antimanic Bipolar disorder Psychiatric NCT02129348
Leuprolide depot II Metabolic Alleviates negative effects of elevated GnRH and gonadtropins on the brain (DMT) Gonadotropin-releasing hormone agonist Endometriosis; uterine leiomyomata Hormonal NCT03649724
Montelukast (buccal film) II Anti-inflammatory Reduce inflammatory pathways (neuronal injury, blood-brain barrier integrity, amyloid-ß accumulation); effect on cognition and AD biomarkers (DMT) Leukotriene receptor antagonist Asthma; exercise-induced bronchoconstriction; allergic rhinitis Anti-inflammatory NCT03402503
Montelukast (tablet) II Anti-inflammatory Reduce inflammatory pathways that mediate neuronal injury, blood-brain barrier integrity, amyloid-ß accumulation (DMT) Leukotriene receptor antagonist Asthma; exercise-induced bronchoconstriction; allergic rhinitis Anti-inflammatory NCT03991988
Nicotinamide II Anti-tau, neuroprotective Reduce tau-induced microtubule depolymerization; reduce phosphorylation of tau (DMT) Vitamin B3 Pellagra Vitamin NCT03061474
Nicotine (transdermal patch) II Neurotransmitter-based Enhance acetylcholine signaling (cognitive enhancer) Alkaloid Smoking cessation withdrawal symptoms Alkaloid NCT02720445
Nilotinib II Anti-amyloid, anti-tau Autophagy enhancer; promotes clearance of amyloid and tau proteins (DMT) Tyrosine kinase inhibitor Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase Hematologic-oncologic NCT02947893
Perindopril II Neuroprotective, anti-inflammatory Improves vascular functioning (DMT) Angiotensin-converting-enzyme inhibitor Essential hypertension; stable coronary artery disease Cardiovascular NCT02085265
Prazosin II Neurotransmitter-based Improve neuropsychiatric symptoms (agitation) Alpha-receptor antagonist Hypertension Cardiovascular NCT03710642
Rifaximin II Anti-inflammatory Improve cognition and function by lowering blood ammonia; lower circulatory pro-inflammatory cytokines secreted by harmful gut bacteria (DMT) Non-systemic antibiotic Traveler’s diarrhea; overt hepatic encephalopathy; irritable bowel syndrome (diarrhea subtype) Infectious disease NCT03856359
Riluzole II Neuroprotective Reduce glutamate-mediated excitotoxicity (DMT) Benzothiazole Amyotrophic lateral sclerosis Neurologic NCT01703117
Sagramostim II Anti-amyloid, neuroprotective Stimulates innate immune system to remove amyloid pathology; increase neuronal connectivity (DMT) Human granulocyte-macrophage colony-stimulating factor Bone marrow stimulation Hematologic-oncologic NCT01409915
Sodium phenylbutyrate II Neuroprotective Blocks nerve cell death and modulates neuroinflammation (DMT) Anti-hyperammonemia Adjunct for chronic urea cycle disorders Genetics NCT03533257
Tacrolimus II Neuroprotective Prevents amyloid-ß dendritic spine loss; normalizes cranial nerve activity in the brain (DMT) Calcineurin inhibitor Prevent rejection of organ transplants Immunologic NCT04263519
Telmisartan II Neuroprotective, anti-inflammatory Improves vascular functioning (DMT) Angiotensin II Receptor Blocker Hypertension; diabetic neuropathy; risk reduction of stroke Cardiovascular NCT02085265
Thiethylperazine II Anti-amyloid Activates transport protein ABCC1 and enhances transport amyloid-ß peptides from the brain into the blood (DMT) Phenothiazine Nausea/vomiting Neurologic NCT03417986 (2014-000870-20)
Valacyclovir II Neuroprotective, anti-inflammatory Reduce amyloid-ß aggregation by preventing overproduction of amyloid in response to infection (DMT) Antiviral (nucleoside analog) Herpes labialis; genital herpes Infectious disease NCT03282916
Valacyclovir II Neuroprotective, anti-inflammatory Reduce amyloid-ß aggregation by preventing overproduction of amyloid in response to infection (DMT) Antiviral (nucleoside analog) Herpes labialis; genital herpes Infectious disease NCT02997982
  1. Abbreviation: DMT disease-modifying therapy