Biomarker & Intervention Discovery

Cardiomyopathy

Pharmacologically actionable gene targets, therapeutic agents ranked by evidence tier, and active clinical trials.

Disease Overview

Spontaneous remission
Not established
Best-intervention remission
Gap size
Primary barrier
Biomarker Targets & Therapeutic Agents

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

AGTR1 249LMNA 203MYBPC3 1MYH7 3TNNT2 120TTN 3TTR 153
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CANDESARTAN CILEXETIL inhibits Mechanistic
CHEMBL:CHEMBL586135 ? unclear Mechanistic
VALSARTAN inhibits Mechanistic 4700.0 nM (Ki)
EPROSARTAN MESYLATE inhibits Mechanistic
FIMASARTAN inhibits Mechanistic
ASCORBIC ACID ? unclear Mechanistic
OLMESARTAN MEDOXOMIL inhibits Mechanistic
AZILSARTAN MEDOXOMIL inhibits Mechanistic
THERAPEUTIC ANDROSTANOLONE ? unclear Mechanistic
CHEMBL:CHEMBL347610 ? unclear Mechanistic
CHEMBL:CHEMBL421088 ? unclear Mechanistic
LOSARTAN POTASSIUM inhibits Mechanistic 5.5 nM (IC50)
CHEMBL:CHEMBL1492017 ? unclear Mechanistic
SPARSENTAN inhibits Mechanistic
SODIUM CHLORIDE ? unclear Mechanistic
CHEMBL:CHEMBL261693 ? unclear Mechanistic
CHEMBL:CHEMBL1972216 ? unclear Mechanistic
EPROSARTAN ? unclear Mechanistic 7.3 nM (IC50)
C-FOS ANTISENSE ? unclear Mechanistic
SARALASIN ACETATE inhibits Mechanistic
IRBESARTAN inhibits Mechanistic 1.1 nM (Ki)
DISULFIRAM ? unclear Mechanistic
SAPRISARTAN ? unclear Mechanistic
H2O2 ? unclear Mechanistic
CHEMBL:CHEMBL591834 ? unclear Mechanistic
CAPTOPRIL ? unclear Mechanistic
ANGIOTENSIN II ? unclear Mechanistic
CHEMBL:CHEMBL400912 ? unclear Mechanistic
ATORVASTATIN CALCIUM TRIHYDRATE ? unclear Mechanistic
IDOXIFENE ? unclear Mechanistic
CHEMBL:CHEMBL528694 ? unclear Mechanistic
IL-6 ? unclear Mechanistic
ANGIOTENSIN II ACETATE activates Mechanistic
LEVODOPA ? unclear Mechanistic
GAMMA-INTERFERON ? unclear Mechanistic
AZILSARTAN KAMEDOXOMIL inhibits Mechanistic
HYDROCHLOROTHIAZIDE ? unclear Mechanistic
CHEMBL:CHEMBL1445650 ? unclear Mechanistic
MUSCLE RELAXANT ? unclear Mechanistic
CL-329167 ? unclear Mechanistic

Showing top 40 of 249 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
HISTAMINE ? unclear Mechanistic
LONAFARNIB ? unclear Mechanistic
CHEMBL:CHEMBL328710 ? unclear Mechanistic
CHEMBL1369905 ~ modulates Mechanistic
CHEMBL1604920 ~ modulates Mechanistic
CHEMBL1592238 ~ modulates Mechanistic
CHEMBL1604921 ~ modulates Mechanistic
CHEMBL1379757 ~ modulates Mechanistic
CHEMBL1458208 ~ modulates Mechanistic
CHEMBL1339839 ~ modulates Mechanistic
CHEMBL1497658 ~ modulates Mechanistic
CHEMBL1536795 ~ modulates Mechanistic
CHEMBL1458219 ~ modulates Mechanistic
CHEMBL1354786 ~ modulates Mechanistic
CHEMBL1536801 ~ modulates Mechanistic
CHEMBL383200 ~ modulates Mechanistic
CHEMBL1594437 ~ modulates Mechanistic
CHEMBL1536807 ~ modulates Mechanistic
CHEMBL1359105 ~ modulates Mechanistic
CHEMBL1458225 ~ modulates Mechanistic
ETHAVERINE ~ modulates Mechanistic
CHEMBL3212696 ~ modulates Mechanistic
CHEMBL1379737 ~ modulates Mechanistic
CHEMBL1319307 ~ modulates Mechanistic
CHEMBL1329385 ~ modulates Mechanistic
CHEMBL1339811 ~ modulates Mechanistic
CHEMBL1604864 ~ modulates Mechanistic
CHEMBL1329925 ~ modulates Mechanistic
CHEMBL3212874 ~ modulates Mechanistic
CHEMBL1394608 ~ modulates Mechanistic
CHEMBL1446960 ~ modulates Mechanistic
CHEMBL1477584 ~ modulates Mechanistic
CHEMBL1438389 ~ modulates Mechanistic
CHEMBL1475134 ~ modulates Mechanistic
CHEMBL1433981 ~ modulates Mechanistic
TEMAZEPAM ~ modulates Mechanistic
CHEMBL1525696 ~ modulates Mechanistic
CHEMBL1339829 ~ modulates Mechanistic
CHEMBL1409613 ~ modulates Mechanistic
CHEMBL1339830 ~ modulates Mechanistic

Showing top 40 of 203 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
OMECAMTIV MECARBIL ? unclear Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
MAVACAMTEN inhibits Mechanistic
OMECAMTIV MECARBIL activates Mechanistic
DANICAMTIV activates Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHEMBL:CHEMBL1601846 ? unclear Mechanistic
PURPUROGALLIN ? unclear Mechanistic 3615.0 nM (IC50)
LEVOSIMENDAN ~ modulates Mechanistic
PYROGALLOL RED ? unclear Mechanistic 558.14 nM (IC50)
CHEMBL1425538 inhibits Mechanistic 676.76 nM (IC50)
CHEMBL1698288 inhibits Mechanistic 12683.0 nM (IC50)
CHEMBL1362935 inhibits Mechanistic 2724.0 nM (IC50)
CHEMBL1356785 inhibits Mechanistic 1819.0 nM (IC50)
CHEMBL1969707 inhibits Mechanistic 7597.0 nM (IC50)
CHEMBL1601846 inhibits Mechanistic 3191.0 nM (IC50)
CHEMBL1373655 inhibits Mechanistic 14960.0 nM (IC50)
CHEMBL1528814 inhibits Mechanistic 27657.0 nM (IC50)
CHEMBL246446 inhibits Mechanistic 20888.0 nM (IC50)
CHEMBL1730100 inhibits Mechanistic 16579.0 nM (IC50)
CHEMBL1730974 inhibits Mechanistic 3888.0 nM (IC50)
CHEMBL1430473 inhibits Mechanistic 10997.0 nM (IC50)
CHEMBL1563898 inhibits Mechanistic 1640.0 nM (IC50)
CHEMBL1414432 inhibits Mechanistic 19681.0 nM (IC50)
CHEMBL257359 inhibits Mechanistic 22446.0 nM (IC50)
CHEMBL1190214 inhibits Mechanistic 8598.0 nM (IC50)
CHEMBL1335321 inhibits Mechanistic 47480.0 nM (IC50)
CHEMBL69612 inhibits Mechanistic 40212.0 nM (IC50)
CHEMBL1427775 inhibits Mechanistic 7659.0 nM (IC50)
CHEMBL1424694 inhibits Mechanistic 3446.0 nM (IC50)
CHEMBL1366427 activates Mechanistic 13703.0 nM (EC50)
CHEMBL1596655 activates Mechanistic 3326.0 nM (EC50)
CHEMBL1408111 activates Mechanistic 40044.0 nM (EC50)
CHEMBL1346219 activates Mechanistic 17943.0 nM (EC50)
CHEMBL1451725 activates Mechanistic 18755.0 nM (EC50)
CHEMBL1601529 activates Mechanistic 15249.0 nM (EC50)
CHEMBL1346165 activates Mechanistic 5397.0 nM (EC50)
CHEMBL1398812 activates Mechanistic 413.02 nM (EC50)
CHEMBL1302799 activates Mechanistic 6895.0 nM (EC50)
CHEMBL1429070 activates Mechanistic 1143.0 nM (EC50)
CHEMBL1429158 activates Mechanistic 17900.0 nM (EC50)
CHEMBL1359099 activates Mechanistic 5480.0 nM (EC50)
CHEMBL1379673 activates Mechanistic 286.87 nM (EC50)
CHEMBL1468444 activates Mechanistic 278.85 nM (EC50)
CHEMBL1604510 activates Mechanistic 742.02 nM (EC50)
CHEMBL1970784 activates Mechanistic 3934.0 nM (EC50)

Showing top 40 of 120 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
PROTEASE INHIBITOR ? unclear Mechanistic
CHEMBL5653589 inhibits Mechanistic 11.93 nM (Kd)
CHEMBL3752910 inhibits Mechanistic 49.56 nM (Kd)
Therapeutic Agent Effect Direction Evidence Tier Potency Source
APIGENIN ? unclear Mechanistic 18000.0 nM (IC50)
ACORAMIDIS ~ modulates Mechanistic 314.0 nM (Kd)
INOTERSEN ? unclear Mechanistic
ACORAMIDIS HYDROCHLORIDE ~ modulates Mechanistic
TAFAMIDIS ~ modulates Mechanistic 274.0 nM (Kd)
MASOPROCOL ? unclear Mechanistic
PATISIRAN ? unclear Mechanistic
ETHYL CAFFEATE ? unclear Mechanistic 23000.0 nM (EC50)
REVUSIRAN ? unclear Mechanistic
EPLONTERSEN ? unclear Mechanistic
CAFFEIC ACID PHENETHYL ESTER ? unclear Mechanistic 8600.0 nM (EC50)
DIHYDROGUAIARETIC ACID ? unclear Mechanistic 6300.0 nM (EC50)
VUTRISIRAN ? unclear Mechanistic
TAFAMIDIS MEGLUMINE ~ modulates Mechanistic
BENZYL CAFFEATE ? unclear Mechanistic 16000.0 nM (EC50)
PATISIRAN SODIUM ? unclear Mechanistic
INOTERSEN SODIUM ? unclear Mechanistic
VUTRISIRAN SODIUM ? unclear Mechanistic
EPLONTERSEN SODIUM ? unclear Mechanistic
TRICLOSAN inhibits Mechanistic 53500.0 nM (IC50)
CHEMBL394034 inhibits Mechanistic 120.0 nM (Kd)
CHEMBL240165 inhibits Mechanistic 42.0 nM (Kd)
CHEMBL241454 inhibits Mechanistic 27.0 nM (Kd)
CHEMBL241453 inhibits Mechanistic 88.0 nM (Kd)
CHEMBL240808 inhibits Mechanistic 27.0 nM (Kd)
CHEMBL438498 inhibits Mechanistic 0.2 nM (Kd)
CHEMBL240806 inhibits Mechanistic 0.3 nM (Kd)
CHEMBL240805 inhibits Mechanistic 5.7 nM (Kd)
CHEMBL240804 inhibits Mechanistic 13.9 nM (Kd)
DEXTROTHYROXINE inhibits Mechanistic 7170.0 nM (IC50)
CHEMBL241025 inhibits Mechanistic 1940.0 nM (IC50)
CHEMBL240807 inhibits Mechanistic 3160.0 nM (IC50)
CHEMBL392872 inhibits Mechanistic 41000.0 nM (IC50)
CHEMBL238590 inhibits Mechanistic 45000.0 nM (IC50)
CHEMBL392873 inhibits Mechanistic 55000.0 nM (IC50)
CHEMBL241575 inhibits Mechanistic 20000.0 nM (IC50)
CHEMBL241573 inhibits Mechanistic 20000.0 nM (IC50)
FLUFENAMIC ACID inhibits Mechanistic 2900.0 nM (IC50)
CHEMBL60569 inhibits Mechanistic 6300.0 nM (IC50)
CHEMBL571374 inhibits Mechanistic 11000.0 nM (IC50)

Showing top 40 of 153 agents ranked by evidence tier.


Clinical Trials

Recruiting and recently completed trials from ClinicalTrials.gov. Data retrieved 2026-07-03.

NCT07505199 Phase: PHASE1 Started: 2026-05-21

This study aims to evaluate the safety and efficacy of fibroblast activation protein (FAP)-targeted autologous immunosuppressive chimeric antigen receptor dendritic cell (iCDC) therapy in patients with ischemic cardiomyopathy, and to explore its potential as a novel therapeutic strategy for this dis...

NCT07426419 Phase: PHASE1, PHASE2 Started: 2026-06

This is a Phase 1/2, open-label, dose-exploration and dose-expansion, clinical trial evaluating the safety, tolerability, pharmacodynamics, and preliminary efficacy of a single intravenous infusion of AFTX-201 in adults with dilated cardiomyopathy caused by a BAG3 gene mutation

NCT07516288 Phase: PHASE1 Started: 2026-05-11

This study aims to evaluate the safety and preliminary efficacy of a second administration of fibroblast activation protein (FAP)-targeted immunosuppressive chimeric antigen receptor dendritic cells (CAR-DC) in patients with end-stage dilated cardiomyopathy. Previous clinical research has shown tha...

NCT07172971 Phase: PHASE1 Started: 2026-07-01

This is a pharmacokinetic study (PK Study) to better understand empagliflozin dosing in pediatric Duchenne muscular dystrophy patients. Empagliflozin is currently used off-label in this population due to the mortality benefits seen in adult cardiomyopathy and heart failure. Investigators will perfor...

NCT01143454 Phase: N/A Started: 2010-07-21

Background: \- Researchers are interested in studying individuals who have known or suspected metabolic, inflammatory or genetic diseases that may put them at a high risk for heart diseases or diseases of their blood vessels. Depending on the condition being studied, both affected and nonaffected i...

NCT06965621 Phase: PHASE2 Started: 2025-11-05

This is a Phase 2 study evaluating the positron-emitting radiopharmaceutical 18F-mFBG as an imaging agent for quantification of myocardial sympathetic innervation. The study will examine a group of stable patients with heart failure (HF) from ischemic cardiomyopathy. All subjects will have left vent...

NCT07137338 Phase: PHASE1 Started: 2026-06

This is a Phase 1, open-label, dose-escalation trial to characterize the safety, tolerability, and preliminary efficacy of RP-A701 following a single IV administration in high-risk adult patients with BAG3-DCM.

NCT07674758 Phase: N/A Started: 2025-01-06

Dystrophin associated heart dysfunction is a leading cause of death in patients with Duchenne and Becker Muscular dystrophy (DMD/BMD) and Duchenne and Becker muscular dystrophy carriers (MDC); however, the evolution of heart dysfunction is not well-understood. The central objectives of this proposal...

NCT07479641 Phase: PHASE1 Started: 2026-04-14

This study will investigate the bioequivalence between two formulations of HRS-1893 tablets. Safety and tolerability will also be assessed.

NCT07298044 Phase: PHASE4 Started: 2026-02-13

Transthyretin (TTR) is a protein made by the liver that helps transport thyroid hormone and vitamin A in the blood. In some people, this protein breaks down and forms harmful clumps called amyloid. TTR amyloid gets deposited in the heart wall and stops it from pumping blood properly, which may lead ...

NCT06563895 Phase: PHASE3 Started: 2025-05-12

Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque-like substance that accumulates in different organs in the body and can cause damage to the organ. There are two ways that the TTR protein can fall ap...

NCT04489355 Phase: N/A Started: 2013-01-28

The study focuses on the development of a new personalized approach to diagnostics and surgical treatment of patients with ischemic cardiomyopathy. The algorithm for selection of patients for certain type of cardiac surgery will be developed. The models for prediction of the risks and outcomes of ca...

NCT07541833 Phase: N/A Started: 2026-02-05

The purpose of this study is to assess the real-world effectiveness and safety of mavacamten in adults diagnosed with symptomatic obstructive hypertrophic cardiomyopathy (HOCM) receiving cibenzoline in Japan

NCT07023341 Phase: PHASE3 Started: 2025-06-30

Researchers are looking for a better way to treat Japanese people who have symptomatic obstructive hypertrophic cardiomyopathy (symptomatic oHCM). Obstructive hypertrophic cardiomyopathy (oHCM) is a type of heart disease where the heart muscles become thicker than normal due to over contraction. Th...

NCT07245420 Phase: NA Started: 2026-09

Childhood cancer survivors who received certain treatments are at a higher risk of developing heart problems in the future. This study is looking at ways to educate childhood cancer survivors about that risk and encourage them to receive a recommended heart screening test.


Agents Found By Disease-Level Search

Searched directly from “Cardiomyopathy” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 7-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).

Not yet run. python -m biomarker_pipeline.run_disease_agent_discovery --disease "..."

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