Biomarker & Intervention Discovery

Cardiomyopathy

This page lists 7 druggable gene targets and 15 recent clinical trials for Cardiomyopathy, with each agent ranked by the strength of its evidence (clinical, mechanistic or correlative). Sources are linked so you can check every claim.

Disease Overview

Spontaneous remission
Not established
Best-intervention remission
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Gap size
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Primary barrier
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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

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

NCT07867288 Phase: 1 Started: 2026-10

The purpose of this study is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses of HTX-001 in human adult participants.

NCT07023614 Phase: 2 Started: 2025-10-06

FORTITUDE-HCM is a global, multicenter, double-blind, parallel-group, placebo-controlled Phase 2b study that will assess the efficacy and safety of ninerafaxstat compared to placebo on top of Standard of Care in patients with symptomatic nHCM

NCT06922994 Phase: 2 Started: 2025-03-31

The goal of this clinical trial is to learn if Atorvastatin 80 mg is effective to avoid functional right ventricular deterioration in patients affected by Arrhythmogenic Cardiomyopathy. It will also learn about the safety of Atorvastatin 80 mg in this type of patients. The main questions it aims to ...

NCT07023341 Phase: 3 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. Thi...

NCT06183931 Phase: 3 Started: 2024-01-11

The primary objective of this study is to access the efficacy of ALXN2220 in the treatment of adult participants with ATTR-CM by evaluating the difference between the ALXN2220 and placebo groups as assessed by the total occurrences of all-cause mortality (ACM) and cardiovascular (CV) clinical events...

NCT07207811 Phase: 3 Started: 2025-10-02

This study will find out if a new medicine called NNC6019-0001 can help reduce the risk of heart-related death and illness in participants with a condition called transthyretin amyloid cardiomyopathy (ATTR-CM), which affects the heart. Participants will either receive NNC6019-0001 or a placebo (a tr...

NCT07052903 Phase: 3 Started: 2025-07-02

The purpose of this study is to: * Evaluate the efficacy of nucresiran compared to placebo on reducing all-cause mortality and cardiovascular (CV) events * Evaluate the efficacy of nucresiran compared to placebo on additional assessments of CV events and/or death * Evaluate the efficacy of nucresira...

NCT07298044 Phase: 4 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 ...

NCT06260709 Phase: 2 Started: 2024-02-20

This study will test a medicine, NNC6019-0001, for people who have a heart disease due to TTR amyloidosis. It will look at how safe this medicine is in the long term and if it can reduce symptoms of a heart disease due to TTR amyloidosis, such as heart failure. It is an extension to a study called "...

NCT07213583 Phase: 2 Started: 2025-08-28

This is a Phase 2, single-arm, multicenter study to evaluate the pharmacodynamics and safety of re-treatment with ALXN2220 in previous participants of Study NI006-101.

NCT07861035 Phase: 1 Started: 2026-06-23

Ischemic cardiomyopathy occurs when the heart muscle is permanently weakened due to blocked coronary arteries that limit blood flow. Current treatments, including medications and lifestyle changes, can manage symptoms but cannot restore the damaged heart muscle. Stem cell therapy offers a promising ...

NCT07210723 Phase: 2/3 Started: 2026-02-13

The Sponsor is studying an investigational medication called danicamtiv to determine if it can help people with genetic and familial dilated cardiomyopathy (DCM). Investigational means that the safety and effectiveness of danicamtiv have not been established. Currently, there are no approved drugs t...

NCT07771465 Phase: 3 Started: 2026-08-26

Danicamtiv is an investigational medication, which means it is still being studied and has not been approved. The purpose of this study is to learn how well danicamtiv works, how safe it is, how well people can take it when used in patients with genetic or familial dilated cardiomyopathy (DCM). Ther...

NCT07755904 Phase: 3 Started: 2026-09-02

The main purpose of this trial is to find out whether BHB-1893 works better than metoprolol in adults with symptoms of obstructive hypertrophic cardiomyopathy (oHCM). The trial includes adults who are currently being treated for oHCM or who have not yet been treated.

NCT07851779 Phase: 4 Started: 2026-01-14

Multicenter, prospective, non-inferiority, randomized, single-blind (primary endpoint evaluators blinded, patients not blinded) clinical trial. We aim to evaluate if the withdrawal of heart failure treatment is safe in patients with non-ischemic cardiomyopathy, negative genetic test, no evidence of ...


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 "..."