Biomarker & Intervention Discovery

Alzheimer's Disease and Other Dementias

This page lists 10 druggable gene targets, 39 approved or trial-tested drug candidates and 15 recent clinical trials for Alzheimer's Disease and Other Dementias, with each agent ranked by the strength of its evidence (clinical, mechanistic or correlative). Candidates include existing drugs that could be repurposed. Sources are linked so you can check every claim.

NIH funding$3.62BFY2025 · RCDC estimate
Funding levelWell-Funded
Spontaneous remissionProgressive and irreversible by definition; MMSE decline 1.07-3.4 points/year untreated; no spontaneous remission
Best-intervention remissionCholinesterase inhibitors (donepezil, rivastigmine, galantamine) slow MMSE decline to 0.2-1.37 points/year — slowing…
Gap sizeSmall for symptom slowing; biological ceiling is genuinely low with current approaches
Primary barrierbiological
Full remission profile →

Remission & chronicity

Spontaneous remission
Progressive and irreversible by definition; MMSE decline 1.07-3.4 points/year untreated; no spontaneous remission
Best-intervention remission
Cholinesterase inhibitors (donepezil, rivastigmine, galantamine) slow MMSE decline to 0.2-1.37 points/year — slowing, not stopping; lecanemab (anti-amyloid) reduced clinical decline rate by 27% at 18 months in early AD (CLARITY AD, 2022); donanemab showed 35% slowing in amyloid/tau positive group
Gap size
Small for symptom slowing; biological ceiling is genuinely low with current approaches
Primary barrier
biological
True disease reversal or cure is not achievable with any current therapy; cholinesterase inhibitors have consistent but modest effects (average 2.7-point ADAS-Cog improvement vs placebo); anti-amyloid biologics represent a genuine mechanistic step forward but are not cures and have significant side effects (ARIA — amyloid-related imaging abnormalities in 21-36%)

Biomarker Targets & Therapeutic Agents

Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC. Agents ranked: Clinical > Mechanistic > Correlative.

ACHE 243APOE 58APP 218BACE1 206BCHE 198CHRNA7 145GRIN1 183MAPT 605PSEN1 136TREM2 6
Therapeutic Agent Effect Direction Evidence Tier Potency Source
EDROPHONIUM CATION ? unclear Mechanistic
PYRIDOSTIGMINE ? unclear Mechanistic
ACOTIAMIDE HYDROCHLORIDE ↓ inhibits Mechanistic
EPTASTIGMINE ? unclear Mechanistic 290.0 nM (IC50)
ISOFLUROPHATE ↓ inhibits Mechanistic
METHADONE HYDROCHLORIDE ? unclear Mechanistic
AMBENONIUM CHLORIDE ↓ inhibits Mechanistic
AMBENONIUM ? unclear Mechanistic
PRALIDOXIME CHLORIDE ↑ activates Mechanistic
NEOSTIGMINE ↓ inhibits Mechanistic
MIMOPEZIL ? unclear Mechanistic
SKIMMIANINE ? unclear Mechanistic
ADRENOMEDULLIN ↑ activates Mechanistic
TACRINE ? unclear Mechanistic 218.78 nM (IC50)
HUMAN CALCITONIN ↑ activates Mechanistic
HUPERZINE A ↓ inhibits Mechanistic 5.0 nM (IC50)
Β-CGRP ↑ activates Mechanistic
SALMON CALCITONIN ↑ activates Mechanistic
GALACTITOL ? unclear Mechanistic
GALANTAMINE ↓ inhibits Mechanistic
ECHOTHIOPHATE IODIDE ↓ inhibits Mechanistic
AMYLIN ↑ activates Mechanistic
TURBINATINE ? unclear Mechanistic
EDROPHONIUM CHLORIDE ↓ inhibits Mechanistic
LATREPIRDINE ? unclear Mechanistic
PYRIDOSTIGMINE BROMIDE ↓ inhibits Mechanistic
DONEPEZIL ? unclear Mechanistic 2.33 nM (IC50)
8-AZAGUANINE ? unclear Mechanistic
DONEPEZIL HYDROCHLORIDE ↓ inhibits Mechanistic
PHYSOSTIGMINE SALICYLATE ↓ inhibits Mechanistic
MINAPRINE HYDROCHLORIDE ↓ inhibits Mechanistic
ADRENOMEDULLIN 2/INTERMEDIN ↑ activates Mechanistic
AC187 ↓ inhibits Mechanistic
PRAMLINTIDE ↑ activates Mechanistic
METHANESULFONYL FLUORIDE ? unclear Mechanistic
SURONACRINE MALEATE ? unclear Mechanistic
RIVASTIGMINE TARTRATE ↓ inhibits Mechanistic
KBP-088 ↑ activates Mechanistic
DESOXYCORDIFOLINE ? unclear Mechanistic
HEXAFLUORENIUM BROMIDE ↓ inhibits Mechanistic

Showing top 40 of 243 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
TRIAMCINOLONE ? unclear Mechanistic
AZ7188 ↓ inhibits Mechanistic
PRAVASTATIN SODIUM ? unclear Mechanistic
LUTEIN ? unclear Mechanistic
TRETINOIN ? unclear Mechanistic
LECANEMAB ? unclear Mechanistic
PHYTOSTEROL ? unclear Mechanistic
BUTHIONINE SULFOXIMINE ? unclear Mechanistic
P2PAL18S ↓ inhibits Mechanistic
THERAPEUTIC CORTICOSTEROID ? unclear Mechanistic
VITAMIN E ? unclear Mechanistic
AZ2429 ↑ activates Mechanistic
GB88 ↓ inhibits Mechanistic
IRBESARTAN ? unclear Mechanistic
SLIGKV-NH2 ↑ activates Mechanistic
STAUROSPORINE ? unclear Mechanistic
TROGLITAZONE ? unclear Mechanistic
AEM-28 ? unclear Mechanistic
TRANS-CINNAMOYL-LIGRLO [N-[3H]PROPIONYL]-NH2 ↑ activates Mechanistic
ACE INHIBITOR ? unclear Mechanistic
I-191 ↓ inhibits Mechanistic
AZ3451 ~ modulates Mechanistic
CN-105 ? unclear Mechanistic
RECOMBINANT CYTOKINE ? unclear Mechanistic
2-FUROYL-LIGRL[N[3H]PROPIONYL]-O-NH2 ↑ activates Mechanistic
FENOFIBRATE MICRONIZED ? unclear Mechanistic
DONEPEZIL ? unclear Mechanistic
AC-98170 ↑ activates Mechanistic
HUMAN CHORIONIC GONADOTROPIN ? unclear Mechanistic
GALANTAMINE ? unclear Mechanistic
NICOTINE POLACRILEX ? unclear Mechanistic
I-287 ~ modulates Mechanistic
ANTIDEPRESSANT AGENT ? unclear Mechanistic
ISOX-CHA-CHG-ALA-ARG-DPR(4FB)-NH2 ↑ activates Mechanistic
GANCICLOVIR ? unclear Mechanistic
C391 ↓ inhibits Mechanistic
IMMUNOSUPPRESSANT ? unclear Mechanistic
AZ8838 ↓ inhibits Mechanistic
2-FUROYL-LIGRL[N-(ALEXA FLUOR 594)-O]-NH2 ↑ activates Mechanistic
ISOX-CHA-CHG-ARK(SULFO-CY5)-NH2 ↑ activates Mechanistic

Showing top 40 of 58 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
VALILTRAMIPROSATE ? unclear Mechanistic
CLIOQUINOL ? unclear Mechanistic
PROPOFOL ? unclear Mechanistic
NIROGACESTAT ? unclear Mechanistic
BAPINEUZUMAB ~ modulates Mechanistic
TRIAZINATE ? unclear Mechanistic
GANTENERUMAB ~ modulates Mechanistic
NANOLUCIFERASE-LABELLED RELAXIN ↑ activates Mechanistic
ML290 ~ modulates Mechanistic
ADUCANUMAB ~ modulates Mechanistic
TAMRLX ↑ activates Mechanistic
AZD5462 ~ modulates Mechanistic
A(4-24)(B7-24)H2 ↑ activates Mechanistic
SOLANEZUMAB ~ modulates Mechanistic
GSK933776 ~ modulates Mechanistic
CAPROSPINOL ? unclear Mechanistic
CHEMBL:CHEMBL477841 ? unclear Mechanistic
(B7-33)H2 ↑ activates Mechanistic
METHYLINOSITOL ? unclear Mechanistic
3,5-DICAFFEOYLQUINIC ACID ? unclear Mechanistic
TRAMIPROSATE ~ modulates Mechanistic
RESVERATROL ? unclear Mechanistic
INSL3 ↑ activates Mechanistic
EUROPIUM-LABELLED RELAXIN ↑ activates Mechanistic
SCYLLITOL ? unclear Mechanistic
RELAXIN ↑ activates Mechanistic
AMILOMOTIDE ? unclear Mechanistic
CURCUMIN ? unclear Mechanistic 0.208 nM (Ki)
2-CHLOROADENOSINE-5-TRIPHOSPHATE ? unclear Mechanistic
DAUNORUBICIN LIPOSOMAL ? unclear Mechanistic
COMPOUND 54 ↑ activates Mechanistic
INOSITOL ? unclear Mechanistic
LECANEMAB ? unclear Mechanistic
AMPHOTERICIN B LIPOSOMAL ? unclear Mechanistic
GENTIAN VIOLET CATION ? unclear Mechanistic
[125I]RELAXIN (HUMAN) ↑ activates Mechanistic
[33P]RELAXIN (HUMAN) ↑ activates Mechanistic
B-R13/17K H2 RELAXIN ↓ inhibits Mechanistic
HYDROXYCHLOROQUINE ? unclear Mechanistic
FERULIC ACID ? unclear Mechanistic

Showing top 40 of 218 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
SOPHORAFLAVANONE G ? unclear Mechanistic
AZ-4217 ? unclear Mechanistic
LY2886721 ? unclear Mechanistic
BYAKANGELICIN ? unclear Mechanistic
CHEMBL:CHEMBL496451 ? unclear Mechanistic
AZD-3839 ? unclear Mechanistic
TAK-070 ? unclear Mechanistic
ELENBECESTAT ↓ inhibits Mechanistic
KUSHENOL A ? unclear Mechanistic
LY3202626 ? unclear Mechanistic
VERUBECESTAT ↓ inhibits Mechanistic
BUNTANETAP ? unclear Mechanistic
CTS-21166 ? unclear Mechanistic
CHEMBL:CHEMBL1412710 ? unclear Mechanistic
BERGAMOTTIN ? unclear Mechanistic
LANABECESTAT ? unclear Mechanistic
OXAZINE 89 ? unclear Mechanistic
RO5508887 ? unclear Mechanistic
LY-2811376 ? unclear Mechanistic
ATABECESTAT ↓ inhibits Mechanistic
EPIGALLOCATECHIN-3-GALLATE ? unclear Mechanistic
KURARINONE ? unclear Mechanistic
TARENFLURBIL ? unclear Mechanistic
CHEMBL:CHEMBL2047666 ? unclear Mechanistic
HGLUVALLEUPNSASPALAGLUPHEOH ↓ inhibits Mechanistic 413.0 nM (IC50)
CHEMBL302651 ↓ inhibits Mechanistic 10491.0 nM (Ki)
CHEMBL305381 ↓ inhibits Mechanistic 3134.0 nM (Ki)
CHEMBL431917 ↓ inhibits Mechanistic 22423.0 nM (Ki)
CHEMBL302833 ↓ inhibits Mechanistic 1129.0 nM (Ki)
CHEMBL78946 ↓ inhibits Mechanistic 1.6 nM (Ki)
CHEMBL65752 ↓ inhibits Mechanistic 50.1 nM (Ki)
CHEMBL65981 ↓ inhibits Mechanistic 61.4 nM (Ki)
CHEMBL66595 ↓ inhibits Mechanistic 5808.0 nM (Ki)
CHEMBL293611 ↓ inhibits Mechanistic 2.5 nM (Ki)
CHEMBL294274 ↓ inhibits Mechanistic 9.4 nM (Ki)
CHEMBL304010 ↓ inhibits Mechanistic 5.9 nM (Ki)
CHEMBL264379 ↓ inhibits Mechanistic 0.32 nM (Ki)
CHEMBL440490 ↓ inhibits Mechanistic 8.0 nM (Ki)
CHEMBL324109 ↓ inhibits Mechanistic 460.0 nM (IC50)
CHEMBL114147 ↓ inhibits Mechanistic 9000.0 nM (IC50)

Showing top 40 of 206 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
SN-38 ? unclear Mechanistic
BMS753 ↑ activates Mechanistic
BMS-189532 ↓ inhibits Mechanistic
IRINOTECAN HYDROCHLORIDE ? unclear Mechanistic
AROTINOID ACID ↑ activates Mechanistic
DEMECARIUM BROMIDE ? unclear Mechanistic
ECHOTHIOPHATE IODIDE ? unclear Mechanistic
IMMUNOTOXIN ? unclear Mechanistic
ISOFLUROPHATE ? unclear Mechanistic
BUPIVACAINE ? unclear Mechanistic
HEXAFLUORENIUM ? unclear Mechanistic
RO 41-5253 ↓ inhibits Mechanistic
TAZAROTENE ↑ activates Mechanistic
TAMIBAROTENE ↑ activates Mechanistic
MEPTAZINOL ? unclear Mechanistic
ALITRETINOIN ↑ activates Mechanistic
TACRINE HYDROCHLORIDE ↓ inhibits Mechanistic
AGN193836 ↑ activates Mechanistic
RIVASTIGMINE ↓ inhibits Mechanistic
VOLANESORSEN ? unclear Mechanistic
BMS614 ↓ inhibits Mechanistic
PHENSERINE ? unclear Mechanistic 2500.0 nM (IC50)
MEMANTINE HYDROCHLORIDE ? unclear Mechanistic
EPTASTIGMINE ? unclear Mechanistic 0.69 nM (IC50)
BMS493 ↓ inhibits Mechanistic
ADAPALENE ↑ activates Mechanistic
RO 40-6055 ↑ activates Mechanistic
AGN193109 ↓ inhibits Mechanistic
PROPANIDID ↓ inhibits Mechanistic
RIVASTIGMINE TARTRATE ↓ inhibits Mechanistic
CD666 ↑ activates Mechanistic
DONEPEZIL ? unclear Mechanistic 8200.0 nM (IC50)
TRETINOIN ↑ activates Mechanistic
TRIAMCINOLONE ? unclear Mechanistic
ANESTHETIC AGENT ? unclear Mechanistic
GALANTAMINE ? unclear Mechanistic 18000.0 nM (IC50)
MIVACURIUM ? unclear Mechanistic
PHENOTHIAZINE ? unclear Mechanistic
TRIFAROTENE ↑ activates Mechanistic
SUCCINYLCHOLINE ? unclear Mechanistic

Showing top 40 of 198 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
FACINICLINE HYDROCHLORIDE ↑ activates Mechanistic
TALBUTAL ? unclear Mechanistic
PNU-120596 ? unclear Mechanistic
ENCENICLINE ↑ activates Mechanistic
VARENICLINE ? unclear Mechanistic
CYTISINE ? unclear Mechanistic
PRIMIDONE ? unclear Mechanistic
SSR180711 ↑ activates Mechanistic
ECULIZUMAB ? unclear Mechanistic
ZILUCOPLAN ? unclear Mechanistic
BNC375 ? unclear Mechanistic
ASM-024 ~ modulates Mechanistic
TC-6987 ↑ activates Mechanistic
ALCURONIUM ? unclear Mechanistic
BRADANICLINE ↑ activates Mechanistic
ABT-126 ↑ activates Mechanistic
AZD0328 ↑ activates Mechanistic
DONEPEZIL ? unclear Mechanistic
THIOPENTAL ? unclear Mechanistic
PHA-543613 ↑ activates Mechanistic
GTS-21 ↑ activates Mechanistic
SECOBARBITAL ? unclear Mechanistic
BUTOBARBITAL ? unclear Mechanistic
METHARBITAL ? unclear Mechanistic
AQW051 ↑ activates Mechanistic
ROZANOLIXIZUMAB ? unclear Mechanistic
HEXOBARBITAL ? unclear Mechanistic
PHENOBARBITAL ? unclear Mechanistic
TREBANANIB ? unclear Mechanistic
APN1125 ↑ activates Mechanistic
GALANTAMINE ? unclear Mechanistic
ND0801 ↑ activates Mechanistic
NICOTINE POLACRILEX ? unclear Mechanistic
EFGARTIGIMOD ALFA ? unclear Mechanistic
ZIPRASIDONE ? unclear Mechanistic
BUTABARBITAL ? unclear Mechanistic
BUTALBITAL ? unclear Mechanistic
RIVASTIGMINE ? unclear Mechanistic
MEPHOBARBITAL ? unclear Mechanistic
AVL-3288 ~ modulates Mechanistic

Showing top 40 of 145 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
COMPOUND 53 [PMID: 33038794] ↑ activates Mechanistic
PAREGORIC ? unclear Mechanistic
STING AGONIST 2 ↑ activates Mechanistic
LATREPIRDINE ? unclear Mechanistic
CNS-5161 ↓ inhibits Mechanistic
MEMANTINE HYDROCHLORIDE ~ modulates Mechanistic
LANICEMINE ↓ inhibits Mechanistic
ULEVOSTINAG ↑ activates Mechanistic
ELIPRODIL ? unclear Mechanistic
NBQX ? unclear Mechanistic
RAPASTINEL ? unclear Mechanistic
DEXANABINOL ? unclear Mechanistic
NEBOGLAMINE ~ modulates Mechanistic
HALOPERIDOL DECANOATE ? unclear Mechanistic
ORPHENADRINE ? unclear Mechanistic
GW468816 ↓ inhibits Mechanistic
ACAMPROSATE CALCIUM ↓ inhibits Mechanistic
AMANTADINE HYDROCHLORIDE ↓ inhibits Mechanistic
H-151 ↓ inhibits Mechanistic
D-SERINE ? unclear Mechanistic
AZD8108 ↓ inhibits Mechanistic
KETAMINE HYDROCHLORIDE ~ modulates Mechanistic
DELUCEMINE ↓ inhibits Mechanistic
EVT-101 ↓ inhibits Mechanistic
MSA-2 ↑ activates Mechanistic
ESKETAMINE HYDROCHLORIDE ? unclear Mechanistic
2'3'-CGAMP ↑ activates Mechanistic
COMPOUND 40 [PMID: 33470814] ↑ activates Mechanistic
PHILANTHOTOXIN 343 ? unclear Mechanistic
FELBAMATE ↓ inhibits Mechanistic
INDANTADOL ↓ inhibits Mechanistic
STING AGONIST 3 ↑ activates Mechanistic
ORPHENADRINE HYDROCHLORIDE ↓ inhibits Mechanistic
CYCLOSERINE ? unclear Mechanistic
RADIPRODIL ↓ inhibits Mechanistic
PERZINFOTEL ↓ inhibits Mechanistic
ESKETAMINE ~ modulates Mechanistic
ADU-S100 ↑ activates Mechanistic
RALFINAMIDE ↓ inhibits Mechanistic
SNX281 ↑ activates Mechanistic

Showing top 40 of 183 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
METHYSERGIDE MALEATE ? unclear Mechanistic
CHEMBL:CHEMBL582473 ? unclear Mechanistic
CHELERYTHRINE CHLORIDE ? unclear Mechanistic
CHEMBL:CHEMBL606167 ? unclear Mechanistic
GRINIMIBINE ? unclear Mechanistic
GOSURANEMAB ↓ inhibits Mechanistic
PLUMBAGIN ? unclear Mechanistic
WORTMANNIN ? unclear Mechanistic
HEXACHLOROPHENE ? unclear Mechanistic
ACID BLUE 129 ? unclear Mechanistic
CHLORANIL ? unclear Mechanistic
INDIRUBIN-3'-MONOXIME ? unclear Mechanistic
NSC-663284 ? unclear Mechanistic
CARBIDOPA ANHYDROUS ? unclear Mechanistic
CHEMBL:CHEMBL601618 ? unclear Mechanistic
BROMOENOL LACTONE ? unclear Mechanistic
METHYLENE BLUE ANHYDROUS ? unclear Mechanistic
DIHYDROERGOCRISTINE MESYLATE ? unclear Mechanistic
ANGUSTIBALIN ? unclear Mechanistic
CHEMBL:CHEMBL310934 ? unclear Mechanistic
CHEMBL:CHEMBL528181 ? unclear Mechanistic
CHEMBL:CHEMBL609606 ? unclear Mechanistic
CALCIMYCIN ? unclear Mechanistic
CHEMBL:CHEMBL1989853 ? unclear Mechanistic
DOCEBENONE ? unclear Mechanistic
NPPB ? unclear Mechanistic
AG 9 ? unclear Mechanistic
CHEMBL:CHEMBL601353 ? unclear Mechanistic
3,4-DICHLOROISOCOUMARIN ? unclear Mechanistic
CEAROIN ? unclear Mechanistic
SULFURETIN ? unclear Mechanistic
2,3-DIHYDROXY-4-METHOXY-4'-ETHOXYBENZOPHENONE ? unclear Mechanistic
CHEMBL:CHEMBL211670 ? unclear Mechanistic
DIHYDROERGOCRISTINE ? unclear Mechanistic
NIFEDIPINE ? unclear Mechanistic
CHEMBL:CHEMBL339587 ? unclear Mechanistic
EPINEPHRINE BITARTRATE ? unclear Mechanistic
RABEPRAZOLE ? unclear Mechanistic
CHEMBL:CHEMBL578905 ? unclear Mechanistic
PICEATANNOL ? unclear Mechanistic

Showing top 40 of 605 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
AVAGACESTAT ↓ inhibits Mechanistic
RG-4733 ↓ inhibits Mechanistic
TETRADECANOYLPHORBOL ACETATE ? unclear Mechanistic
NIROGACESTAT ↓ inhibits Mechanistic
CHEMBL:CHEMBL247471 ? unclear Mechanistic
CHEMBL:CHEMBL392068 ? unclear Mechanistic
SEMAGACESTAT ↓ inhibits Mechanistic
BEGACESTAT ↓ inhibits Mechanistic
TARENFLURBIL ~ modulates Mechanistic
IC261 ? unclear Mechanistic
NOTCH SIGNALING PATHWAY INHIBITOR MK0752 ? unclear Mechanistic
E2012 ? unclear Mechanistic
GSI-136 ? unclear Mechanistic
NIROGACESTAT HYDROBROMIDE ? unclear Mechanistic
CHEMBL392068 ↑ activates Mechanistic 0.114 nM (EC50)
CHEMBL2159511 ↓ inhibits Mechanistic 46.0 nM (IC50)
CHEMBL2159510 ↓ inhibits Mechanistic 5.0 nM (IC50)
CHEMBL2159509 ↓ inhibits Mechanistic 8.0 nM (IC50)
CHEMBL2159691 ↓ inhibits Mechanistic 37.0 nM (IC50)
CHEMBL2159690 ↓ inhibits Mechanistic 48.0 nM (IC50)
CHEMBL2159689 ↓ inhibits Mechanistic 8.0 nM (IC50)
CHEMBL2159687 ↓ inhibits Mechanistic 18.0 nM (IC50)
CHEMBL2159686 ↓ inhibits Mechanistic 377.0 nM (IC50)
CHEMBL2159688 ↓ inhibits Mechanistic 320.0 nM (IC50)
CHEMBL2398571 ↓ inhibits Mechanistic 4000.0 nM (IC50)
CHEMBL2398570 ↓ inhibits Mechanistic 39810.72 nM (IC50)
CHEMBL2398569 ↓ inhibits Mechanistic 39810.72 nM (IC50)
CHEMBL2398568 ↓ inhibits Mechanistic 39810.72 nM (IC50)
CHEMBL2398567 ↓ inhibits Mechanistic 31622.78 nM (IC50)
CHEMBL2398579 ↓ inhibits Mechanistic 31622.78 nM (IC50)
CHEMBL2398578 ↓ inhibits Mechanistic 31622.78 nM (IC50)
CHEMBL2396675 ↓ inhibits Mechanistic 39810.72 nM (IC50)
CHEMBL2398577 ↓ inhibits Mechanistic 31622.78 nM (IC50)
CHEMBL2398576 ↓ inhibits Mechanistic 31622.78 nM (IC50)
CHEMBL2398575 ↓ inhibits Mechanistic 25118.86 nM (IC50)
CHEMBL2398574 ↓ inhibits Mechanistic 31622.78 nM (IC50)
CHEMBL2398573 ↓ inhibits Mechanistic 31622.78 nM (IC50)
CHEMBL2398572 ↓ inhibits Mechanistic 39810.72 nM (IC50)
CHEMBL3397716 ↑ activates Mechanistic 3200.0 nM (EC50)
CHEMBL3397715 ↑ activates Mechanistic 13800.0 nM (EC50)

Showing top 40 of 136 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHEMBL6174051 ↑ activates Mechanistic 3.0 nM (EC50)
CHEMBL6165036 ↑ activates Mechanistic 2.0 nM (EC50)
CHEMBL6172797 ↑ activates Mechanistic 1.0 nM (EC50)
CHEMBL6166220 ↑ activates Mechanistic 5.0 nM (EC50)
CHEMBL6165261 ↑ activates Mechanistic 4.0 nM (EC50)
CHEMBL6166127 ↑ activates Mechanistic 2.0 nM (EC50)

Clinical Trials

Drug, biologic and supplement trials (treatment or prevention) from ClinicalTrials.gov whose main condition is Alzheimer's Disease and Other Dementias: recruiting, active or completed, most recently updated first. Data retrieved 2026-10-28.

NCT05891496 Phase: 3 Started: 2023-06-20

The study is being conducted to understand how the medicine, semaglutide, affects the immune system and other biological processes in people with Alzheimer's disease. Semaglutide is a medicine that doctors can prescribe in some countries for the treatment of type 2 diabetes and excess body weight. T...

NCT07602582 Phase: 3 Started: 2026-05-22

The main purpose of this study is to determine if participants who previously took donanemab get clinical benefit when they receive annual doses. For each participant, the study will last up to 2.5 years and will include 6 visits.

NCT06976216 Phase: 3 Started: 2025-07-14

The purpose of this study is to evaluate the efficacy and safety of KarXT + KarX-EC for cognitive impairment in Alzheimer's Disease

NCT06957418 Phase: 2 Started: 2026-07-06

The goal of the Alzheimer's Tau Platform (ATP) is to evaluate the safety and effectiveness of tau-directed therapies, alone or in combination with the anti-amyloid monoclonal antibody, donanemab, in adults aged 50-80 with late preclinical or early prodromal Alzheimer's disease. This platform trial a...

NCT07688213 Phase: 2 Started: 2026-07-28

This is a randomized, placebo-controlled Phase 2 study to evaluate the efficacy and safety of SAR448851 in early Alzheimer's disease (AD) participants. The purpose of this study is to measure efficacy and safety with once daily oral SAR448851 compared to placebo in participants with mild cognitive i...

NCT06874621 Phase: 1/2 Started: 2025-03-03

This study is to be conducted in participants with early Alzheimer's Disease to test VY7523, a new drug being researched for treatment of Alzheimer's Disease. This study will look at how safe the drug is and how it works in the brain. It was first tested in normal, healthy participants who volunteer...

NCT06881836 Phase: 2 Started: 2025-04-24

This is a Phase 2, double-blind, parallel-group, placebo-controlled study to assess safety, tolerability, pharmacokinetics, and efficacy of ONO-2020 in participants with mild to moderate Alzheimer's disease (AD). This study aims to determine whether administering ONO-2020, an epigenetic regulator, m...

NCT06125951 Phase: 2/3 Started: 2024-04-12

Xanamem® is being developed as a potential treatment for symptomatic, early stages of Alzheimer's Disease (AD) and Major Depressive Disorder (MDD). This XanaMIA Phase 2b/3 study is to investigate the safety, tolerability, and efficacy of Xanamem in in mild or moderate dementia due to AD. Trial parti...

NCT07094516 Phase: 2 Started: 2025-08-07

This is a multicentre, randomized, double-blind, placebo-controlled, parallel group Phase II study to evaluate the efficacy and safety of VHB937 in participants with early AD followed by an Extension. The double-blind part is 72 weeks long, followed by an extension.

NCT07033494 Phase: 2 Started: 2025-07-16

Researchers want to know if the study treatment called MK-2214 works to slow certain changes in the brains of people with Alzheimer's disease (AD). AD is a type of dementia that can cause loss of memory, communication (such as speech), and decision-making skills. It can limit a person's ability to d...

NCT05531526 Phase: 3 Started: 2022-12-23

This AR1001-ADP3-US01 protocol is a double-blind, randomized, placebo-controlled, multi- center, parallel-group comparison pivotal Phase 3 study to evaluate the efficacy and safety of AR1001 for the treatment of participants with early AD.

NCT07717411 Phase: 3 Started: 2026-11-30

This study will evaluate the efficacy and safety of trontinemab in participants with biomarker evidence of Alzheimer's Disease (AD) pathology but with no cognitive or functional impairment, who are at risk for progression to mild cognitive impairment (MCI) due to AD or dementia due to AD.

NCT07636811 Phase: 2 Started: 2026-08-17

The purpose of the study is to evaluate the effect of mivelsiran in adult participants with early-stage DS-AD and to characterize the safety, tolerability, and pharmacodynamics (PD) of mivelsiran. The study will be conducted over 2 periods: a 24-month double-blind period and an optional 12-month ope...

NCT06976203 Phase: 3 Started: 2025-07-21

The purpose of this study is to evaluate the efficacy and safety of KarXT + KarX-EC for cognitive impairment in Alzheimer's Disease

NCT07214727 Phase: 1 Started: 2025-10-15

The purpose of this study is to: * Evaluate the safety and tolerability of intrathecal (IT) ALN-5288 in patients with Alzheimer's Disease (AD) * Evaluate the pharmacodynamic (PD) and pharmacokinetic (PK) effects of ALN-5288 after dose administration


Agents Found By Disease-Level Search

Searched directly from “Alzheimer's Disease and Other Dementias” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 10-gene target panel above. This surfaces agents whose mechanism doesn't route through a curated gene (combination therapies, standard-of-care drugs, targets outside the panel).

39 agents shown (approved or with trial evidence) — 39 not found by the gene-target search above.

risperidoneNOT in gene-target list

· 6 trials · source: ClinicalTrials.gov
NCT00417482 NCT00208819 NCT00034762

OlanzapineNOT in gene-target list

· 3 trials · source: ClinicalTrials.gov
NCT00015548 NCT00245206 NCT00208819

ML-007C-MANOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT07459660 NCT06887192

KarXTNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT06585787 NCT06947941

QuetiapineNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00015548 NCT00208819

NSA-789NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00795730

Photo elicitationNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04103580

LithiumNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02129348

Building Better Caregivers Online WorkshopNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00799786

TRAIN-ADNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03244917

hMSCNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06781333

SemaglutideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05891496

ValproateNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00071721

BI 409306 25 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02392468

BI 409306 50 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02392468

Haloperidol-HaloperidolNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00009217

Social DancingNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03475316

Treadmill WalkingNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03475316

CitalopramNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00015548

Speeko for ElderspeakNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04064164

GSK1034702NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00937846

MIM-DASHNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05734638

MIM OnlyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05734638

DASH OnlyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05734638

divalproexNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00208819

EscitalopramNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01119638

AlgorithmNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05779839

Random MatchNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05779839

Connect-Home PlusNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05887388

Changing Talk Online (CHATO)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04610944

KarX-ECNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06947941

Pimavanserin 34 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03325556

Pimavanserin 20 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03325556

F-18NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03625128

AripiprazoleNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00245206

GENUSNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07496918

Tactile GENUSNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07496918

Empatica EmbracePlusNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06326424

TRAIN AD 2.0NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05950607

Generated by the Open Source Medicine Foundation Biomarker Pipeline — 2026-10-08. Data sources: DGIdb, Open Targets, ChEMBL, PubMed, ClinicalTrials.gov. Not medical advice.