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. 2020 Jul 16;6(1):e12050. doi: 10.1002/trc2.12050

TABLE 1.

Agents in Phase 3 of Alzheimer's disease drug development (ClinicalTrials.gov accessed February 27, 2020)

Agent CADROmechanism class Mechanism of action Therapeutic purpose Status(CT.gov ID) Sponsor Start date Estimated end date
Aducanumab Amyloid Monoclonal antibody directed at plaques and oligomers Remove amyloid (DMT)

Not yet recruiting

(NCT04241068)

 Biogen Mar 2020 Sep 2023

AGB101

(low‐dose levetiracetam)

 Synaptic plasticity/neuroprotection SV2A modulator Improve synaptic function; reduce amyloid‐induced neuronal hyperactivity (DMT)

Recruiting

(NCT03486938)

 AgeneBio, NIA Jan 2019 Nov 2022

ALZT‐OP1

(cromolyn + ibuprofen)

 Inflammation Mast cell stabilizer (cromolyn), anti‐inflammatory (ibuprofen) Microglial modulation; promote microglial clearance of amyloid (DMT)

Active, not recruiting

(NCT02547818)

 AZTherapies Sep 2015 Dec 2020

ANAVEX2‐73

(blarcamesine)

 Synaptic plasticity/neuroprotection Sigma‐1 receptor agonist, M2 autoreceptor antagonist Enhances cell signaling to ameliorate oxidative stress, protein misfolding, mitochondrial dysfunction and inflammation (DMT)

Recruiting a

(NCT03790709)

 Anavex life sciences Jul 2018 Dec 2021
AVP‐786 Neurotransmitter receptors Sigma 1 receptor agonist; NMDA receptor antagonist Improve neuropsychiatric symptoms (agitation)

Recruiting

(NCT03393520)

 Avanir Oct 2017 Jun 2021

Recruiting, extension study

(NCT02446132)

 Avanir Dec 2015 Jun 2022
AXS‐05 Neurotransmitter receptors Sigma 1 receptor agonist; NMDA receptor antagonist (dextromethorphan); dopamine‐norepinephrine reuptake inhibitor (bupropion) Improve neuropsychiatric symptoms (agitation)

Recruiting a

(NCT03226522)

 Axsome therapeutics Jul 2017 Jun 2020
Azeliragon Amyloid, inflammation RAGE antagonist Reduce amyloid transport into the brain; reduce inflammation (DMT)

Recruiting a

(NCT03980730)

 vTv Therapeutics Jun 2019 Jul 2023
BAN2401 Amyloid Monoclonal antibody directed at protofibrils Reduce protofibrillar amyloid and amyloid plaques (DMT)

Recruiting

(NCT03887455)

 Eisai, Biogen Mar 2019 Mar 2024

BHV4157

(troriluzole)

 Synaptic plasticity/neuroprotection Glutamate modulator; prodrug of riluzole Reduce synaptic levels of glutamate; improve synaptic functioning (DMT)

Active, not recruiting a

(NCT03605667)

 Biohaven pharma, ADCS Jul 2018 Dec 2020
BPDO‐1603 Undisclosed Undisclosed Undefined mechanism (cognitive enhancer)

Not yet recruiting

(NCT04229927)

 Hyundai pharmaceutical Feb 2020 Mar 2023
Brexpiprazole Neurotransmitter Receptors D2 receptor partial agonist, serotonin‐dopamine modulator Improve neuropsychiatric symptoms (agitation)

Recruiting a

(NCT03620981)

 Otsuka Aug 2018 Nov 2021

Recruiting, extension study

(NCT03594123)

 Otsuka Oct 2018 Aug 2021

Recruiting

(NCT03548584)

 Otsuka May 2018 Dec 2020

Recruiting, extension study

(NCT03724942)

 Otsuka Nov 2018 May 2021
CAD106 b Amyloid Amyloid vaccine Remove amyloid (DMT)

Active, not recruiting a

(NCT02565511)

Novartis, Amgen, NIA,

Alzheimer's Association, Banner Alzheimer's Institute

 Nov 2015 Mar 2025
COR388 Inflammation/infection Bacterial protease inhibitor targeting gingipain produced by P. gingivalis Reduce neuroinflammation and hippocampal degeneration (DMT)

Recruiting a

(NCT03823404)

 Cortexyme Mar 2019 Dec 2022
Escitalopram Neurotransmitter receptors SSRI Improve neuropsychiatric symptoms (agitation)

Recruiting

(NCT03108846)

 Johns Hopkins University, NIA Jan 2018 Aug 2022
Gantenerumab Amyloid Monoclonal antibody directed at plaques and oligomers Remove amyloid (DMT)

Active, not recruiting

(NCT02051608)

 Roche Mar 2014 Apr 2021

Active, not recruiting

(NCT01224106)

 Roche Nov 2010

Aug 2020

Recruiting

(NCT03444870)

 Roche Jun 2018 May 2023

Recruiting

(NCT03443973)

 Roche Aug 2018 May 2023
Gantenerumab and solanezumab Amyloid Monoclonal antibody directed at plaques and oligomers (gantenerumab); Monoclonal antibody directed at monomers (solanezumab) Remove amyloid; reduce amyloid production (DMT)

Recruiting a , c

(NCT01760005)

 Washington University, Eli Lilly, Roche, NIA, Alzheimer's Association Dec 2012 Mar 2021
Ginkgo biloba Metabolism and bioenergetics Plant extract with antioxidant properties Improve brain blood flow and mitochondrial function (cognitive enhancer)

Recruiting a

(NCT03090516)

 Nanjing Medical University Aug 2016 Mar 2020
Guanfacine Neurotransmitter receptors Alpha‐2 adrenergic agonist Modulation of noradrenergic deficit (cognitive enhancer)

Recruiting

(NCT03116126)

 Imperial College London, UK National Institute of Health Research Jan 2019 Mar 2021
Icosapent ethyl (IPE) Synaptic plasticity/neuroprotection Purified form of the omega‐3 fatty acid EPA Improve synaptic function; reduce inflammation (DMT)

Recruiting a

(NCT02719327)

 VA Office of Research and Development, University of Wisconsin, Madison Jun 2017 Nov 2021
Losartan and amlodipine and atorvastatin + exercise Vasculature Angiotensin II receptor blocker (losartan), calcium channel blocker (amlodipine), cholesterol agent (atorvastatin) Vascular risk reduction; preservation of cognitive function (DMT)

Active, not recruiting a

(NCT02913664)

 University of Texas Southwestern Sep 2016 Mar 2022
Masitinib Inflammation/immunity Tyrosine kinase inhibitor Modulation of mast cell‐related inflammatory processes; reduce amyloid protein and tau phosphorylation (DMT)

Active, not recruiting

(NCT01872598)

 AB Science Jan 2012 Dec 2019
Metformin Metabolism and bioenergetics Insulin sensitizer Improve CNS glucose metabolism (DMT)

Not yet recruiting a

(NCT04098666)

 Columbia University, NIA, EMD Serono Apr 2020 Apr 2024
Methylphenidate Neurotransmitter receptors Dopamine reuptake inhibitor Improve neuropsychiatric symptoms (apathy)

Active, not recruiting

(NCT02346201)

 Johns Hopkins University, NIA Jan 2016 Jun 2020
Mirtazapine Neurotransmitter Receptors Alpha‐1 antagonist Improve neuropsychiatric symptoms (agitation)

Recruiting

(NCT03031184)

 University of Sussex Jan 2017 Jul 2020
Octohydro‐aminoacridine Succinate Neurotransmitter receptors Acetylcholinesterase inhibitor Improve acetylcholine signaling (cognitive enhancer)

Recruiting

(NCT03283059)

 Shanghai Mental Health Center, Changchun‐Huayang High‐tech, Jiangsu Sheneryang High‐tech Aug 2017 Feb 2021
Solanezumab Amyloid Monoclonal antibody directed at monomers Remove amyloid and prevent aggregation (DMT)

Active, not recruiting

(NCT02008357)

 Eli Lilly, ATRI Feb 2014 Jul 2022

Tricaprilin

 Metabolism and bioenergetics Ketone body stimulant; caprylic triglyceride Induce ketosis to improve mitochondrial function and neuronal metabolism (DMT)

Not yet recruiting

(NCT04187547)

 Cerecin Jul 2020 Dec 2022

TRx0237

(LMTX)

Tau

 Tau protein aggregation inhibitor Reduce tau mediated neuronal damage (DMT)

Recruiting

(NCT03446001)

 TauRx Therapeutics Jan 2018 Dec 2022
Zolpidem and zoplicone Neurotransmitter receptors Positive allosteric modulator of GABA‐A receptors Improve neuropsychiatric symptoms (sleep disorders)

Recruiting

(NCT03075241)

 Brasilia University Hospital Oct 2016 Dec 2020

Abbreviations: ADCS, Alzheimer's disease cooperative study; ATRI, Alzheimer's Therapeutic Research Institute; BACE, beta‐site amyloid precursor protein cleaving enzyme; CADRO, Common Alzheimer's Disease and Related Disorders Research Ontology; DMT, disease‐modifying therapy; EPA, eicosapentaenoic acid; GABA, gamma‐aminobutyric acid; NIA, National Institute on Aging; SSRI, selective serotonin reuptake inhibitor; SV2A, synaptic vesicle protein 2A

Note: Twenty‐nine agents in 36 Phase 3 clinical trials currently ongoing as of February 27, 2020 according to ClinicalTrials.gov.

Note: Bolded terms represent new agents into the 2020 Phase 3 pipeline since 2019.

Note: The following agents have been identified as terminated per company press releases and have been removed from the current pipeline although they are still listed as ongoing on ClinicalTrials.gov: CNP520/umibecestat (NCT03131453), E2609/elenbecestat (NCT02956486, NCT03036280).

a

Phase 2/3 trials.

b

CNP520 (umibecestat) has been removed from the GENERATION 1 trial.

c

DIAN‐TU trial has been completed and failed to meet its clinical outcomes for both gantenerumab and solanezumab. Secondary analyses are pending.