Disease Overview
Biomarker Targets & Therapeutic Agents
Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC. Agents ranked: Clinical > Mechanistic > Correlative.
AGTR1
| Therapeutic Agent | Effect Direction | Evidence Tier | Potency | Source |
|---|---|---|---|---|
| CANDESARTAN CILEXETIL | ↓ inhibits | Mechanistic | DGIdb | |
| CHEMBL:CHEMBL586135 | ? unclear | Mechanistic | DGIdb | |
| VALSARTAN | ↓ inhibits | Mechanistic | 4700.0 nM (Ki) | DGIdb |
| EPROSARTAN MESYLATE | ↓ inhibits | Mechanistic | DGIdb | |
| FIMASARTAN | ↓ inhibits | Mechanistic | DGIdb | |
| ASCORBIC ACID | ? unclear | Mechanistic | DGIdb | |
| OLMESARTAN MEDOXOMIL | ↓ inhibits | Mechanistic | DGIdb | |
| AZILSARTAN MEDOXOMIL | ↓ inhibits | Mechanistic | DGIdb | |
| THERAPEUTIC ANDROSTANOLONE | ? unclear | Mechanistic | DGIdb | |
| CHEMBL:CHEMBL347610 | ? unclear | Mechanistic | DGIdb | |
| CHEMBL:CHEMBL421088 | ? unclear | Mechanistic | DGIdb | |
| LOSARTAN POTASSIUM | ↓ inhibits | Mechanistic | 5.5 nM (IC50) | DGIdb |
| CHEMBL:CHEMBL1492017 | ? unclear | Mechanistic | DGIdb | |
| SPARSENTAN | ↓ inhibits | Mechanistic | DGIdb | |
| SODIUM CHLORIDE | ? unclear | Mechanistic | DGIdb | |
| CHEMBL:CHEMBL261693 | ? unclear | Mechanistic | DGIdb | |
| CHEMBL:CHEMBL1972216 | ? unclear | Mechanistic | DGIdb | |
| EPROSARTAN | ? unclear | Mechanistic | 7.3 nM (IC50) | DGIdb |
| C-FOS ANTISENSE | ? unclear | Mechanistic | DGIdb | |
| SARALASIN ACETATE | ↓ inhibits | Mechanistic | DGIdb | |
| IRBESARTAN | ↓ inhibits | Mechanistic | 1.1 nM (Ki) | DGIdb |
| DISULFIRAM | ? unclear | Mechanistic | DGIdb | |
| SAPRISARTAN | ? unclear | Mechanistic | DGIdb | |
| H2O2 | ? unclear | Mechanistic | DGIdb | |
| CHEMBL:CHEMBL591834 | ? unclear | Mechanistic | DGIdb | |
| CAPTOPRIL | ? unclear | Mechanistic | DGIdb | |
| ANGIOTENSIN II | ? unclear | Mechanistic | DGIdb | |
| CHEMBL:CHEMBL400912 | ? unclear | Mechanistic | DGIdb | |
| ATORVASTATIN CALCIUM TRIHYDRATE | ? unclear | Mechanistic | DGIdb | |
| IDOXIFENE | ? unclear | Mechanistic | DGIdb | |
| CHEMBL:CHEMBL528694 | ? unclear | Mechanistic | DGIdb | |
| IL-6 | ? unclear | Mechanistic | DGIdb | |
| ANGIOTENSIN II ACETATE | ↑ activates | Mechanistic | DGIdb | |
| LEVODOPA | ? unclear | Mechanistic | DGIdb | |
| GAMMA-INTERFERON | ? unclear | Mechanistic | DGIdb | |
| AZILSARTAN KAMEDOXOMIL | ↓ inhibits | Mechanistic | DGIdb | |
| HYDROCHLOROTHIAZIDE | ? unclear | Mechanistic | DGIdb | |
| CHEMBL:CHEMBL1445650 | ? unclear | Mechanistic | DGIdb | |
| MUSCLE RELAXANT | ? unclear | Mechanistic | DGIdb | |
| CL-329167 | ? unclear | Mechanistic | DGIdb |
Showing top 40 of 249 agents ranked by evidence tier.
LMNA
| Therapeutic Agent | Effect Direction | Evidence Tier | Potency | Source |
|---|---|---|---|---|
| HISTAMINE | ? unclear | Mechanistic | DGIdb | |
| LONAFARNIB | ? unclear | Mechanistic | DGIdb | |
| CHEMBL:CHEMBL328710 | ? unclear | Mechanistic | DGIdb | |
| CHEMBL1369905 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1604920 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1592238 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1604921 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1379757 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1458208 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1339839 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1497658 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1536795 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1458219 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1354786 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1536801 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL383200 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1594437 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1536807 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1359105 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1458225 | ~ modulates | Mechanistic | ChEMBL | |
| ETHAVERINE | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL3212696 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1379737 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1319307 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1329385 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1339811 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1604864 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1329925 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL3212874 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1394608 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1446960 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1477584 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1438389 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1475134 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1433981 | ~ modulates | Mechanistic | ChEMBL | |
| TEMAZEPAM | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1525696 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1339829 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1409613 | ~ modulates | Mechanistic | ChEMBL | |
| CHEMBL1339830 | ~ modulates | Mechanistic | ChEMBL |
Showing top 40 of 203 agents ranked by evidence tier.
MYBPC3
| Therapeutic Agent | Effect Direction | Evidence Tier | Potency | Source |
|---|---|---|---|---|
| OMECAMTIV MECARBIL | ? unclear | Mechanistic | DGIdb |
MYH7
TNNT2
| Therapeutic Agent | Effect Direction | Evidence Tier | Potency | Source |
|---|---|---|---|---|
| CHEMBL:CHEMBL1601846 | ? unclear | Mechanistic | DGIdb | |
| PURPUROGALLIN | ? unclear | Mechanistic | 3615.0 nM (IC50) | DGIdb |
| LEVOSIMENDAN | ~ modulates | Mechanistic | DGIdb | |
| PYROGALLOL RED | ? unclear | Mechanistic | 558.14 nM (IC50) | DGIdb |
| CHEMBL1425538 | ↓ inhibits | Mechanistic | 676.76 nM (IC50) | ChEMBL |
| CHEMBL1698288 | ↓ inhibits | Mechanistic | 12683.0 nM (IC50) | ChEMBL |
| CHEMBL1362935 | ↓ inhibits | Mechanistic | 2724.0 nM (IC50) | ChEMBL |
| CHEMBL1356785 | ↓ inhibits | Mechanistic | 1819.0 nM (IC50) | ChEMBL |
| CHEMBL1969707 | ↓ inhibits | Mechanistic | 7597.0 nM (IC50) | ChEMBL |
| CHEMBL1601846 | ↓ inhibits | Mechanistic | 3191.0 nM (IC50) | ChEMBL |
| CHEMBL1373655 | ↓ inhibits | Mechanistic | 14960.0 nM (IC50) | ChEMBL |
| CHEMBL1528814 | ↓ inhibits | Mechanistic | 27657.0 nM (IC50) | ChEMBL |
| CHEMBL246446 | ↓ inhibits | Mechanistic | 20888.0 nM (IC50) | ChEMBL |
| CHEMBL1730100 | ↓ inhibits | Mechanistic | 16579.0 nM (IC50) | ChEMBL |
| CHEMBL1730974 | ↓ inhibits | Mechanistic | 3888.0 nM (IC50) | ChEMBL |
| CHEMBL1430473 | ↓ inhibits | Mechanistic | 10997.0 nM (IC50) | ChEMBL |
| CHEMBL1563898 | ↓ inhibits | Mechanistic | 1640.0 nM (IC50) | ChEMBL |
| CHEMBL1414432 | ↓ inhibits | Mechanistic | 19681.0 nM (IC50) | ChEMBL |
| CHEMBL257359 | ↓ inhibits | Mechanistic | 22446.0 nM (IC50) | ChEMBL |
| CHEMBL1190214 | ↓ inhibits | Mechanistic | 8598.0 nM (IC50) | ChEMBL |
| CHEMBL1335321 | ↓ inhibits | Mechanistic | 47480.0 nM (IC50) | ChEMBL |
| CHEMBL69612 | ↓ inhibits | Mechanistic | 40212.0 nM (IC50) | ChEMBL |
| CHEMBL1427775 | ↓ inhibits | Mechanistic | 7659.0 nM (IC50) | ChEMBL |
| CHEMBL1424694 | ↓ inhibits | Mechanistic | 3446.0 nM (IC50) | ChEMBL |
| CHEMBL1366427 | ↑ activates | Mechanistic | 13703.0 nM (EC50) | ChEMBL |
| CHEMBL1596655 | ↑ activates | Mechanistic | 3326.0 nM (EC50) | ChEMBL |
| CHEMBL1408111 | ↑ activates | Mechanistic | 40044.0 nM (EC50) | ChEMBL |
| CHEMBL1346219 | ↑ activates | Mechanistic | 17943.0 nM (EC50) | ChEMBL |
| CHEMBL1451725 | ↑ activates | Mechanistic | 18755.0 nM (EC50) | ChEMBL |
| CHEMBL1601529 | ↑ activates | Mechanistic | 15249.0 nM (EC50) | ChEMBL |
| CHEMBL1346165 | ↑ activates | Mechanistic | 5397.0 nM (EC50) | ChEMBL |
| CHEMBL1398812 | ↑ activates | Mechanistic | 413.02 nM (EC50) | ChEMBL |
| CHEMBL1302799 | ↑ activates | Mechanistic | 6895.0 nM (EC50) | ChEMBL |
| CHEMBL1429070 | ↑ activates | Mechanistic | 1143.0 nM (EC50) | ChEMBL |
| CHEMBL1429158 | ↑ activates | Mechanistic | 17900.0 nM (EC50) | ChEMBL |
| CHEMBL1359099 | ↑ activates | Mechanistic | 5480.0 nM (EC50) | ChEMBL |
| CHEMBL1379673 | ↑ activates | Mechanistic | 286.87 nM (EC50) | ChEMBL |
| CHEMBL1468444 | ↑ activates | Mechanistic | 278.85 nM (EC50) | ChEMBL |
| CHEMBL1604510 | ↑ activates | Mechanistic | 742.02 nM (EC50) | ChEMBL |
| CHEMBL1970784 | ↑ activates | Mechanistic | 3934.0 nM (EC50) | ChEMBL |
Showing top 40 of 120 agents ranked by evidence tier.
| Therapeutic Agent | Effect Direction | Evidence Tier | Potency | Source |
|---|---|---|---|---|
| APIGENIN | ? unclear | Mechanistic | 18000.0 nM (IC50) | DGIdb |
| ACORAMIDIS | ~ modulates | Mechanistic | 314.0 nM (Kd) | DGIdb |
| INOTERSEN | ? unclear | Mechanistic | DGIdb | |
| ACORAMIDIS HYDROCHLORIDE | ~ modulates | Mechanistic | DGIdb | |
| TAFAMIDIS | ~ modulates | Mechanistic | 274.0 nM (Kd) | DGIdb |
| MASOPROCOL | ? unclear | Mechanistic | DGIdb | |
| PATISIRAN | ? unclear | Mechanistic | DGIdb | |
| ETHYL CAFFEATE | ? unclear | Mechanistic | 23000.0 nM (EC50) | DGIdb |
| REVUSIRAN | ? unclear | Mechanistic | DGIdb | |
| EPLONTERSEN | ? unclear | Mechanistic | DGIdb | |
| CAFFEIC ACID PHENETHYL ESTER | ? unclear | Mechanistic | 8600.0 nM (EC50) | DGIdb |
| DIHYDROGUAIARETIC ACID | ? unclear | Mechanistic | 6300.0 nM (EC50) | DGIdb |
| VUTRISIRAN | ? unclear | Mechanistic | DGIdb | |
| TAFAMIDIS MEGLUMINE | ~ modulates | Mechanistic | DGIdb | |
| BENZYL CAFFEATE | ? unclear | Mechanistic | 16000.0 nM (EC50) | DGIdb |
| PATISIRAN SODIUM | ? unclear | Mechanistic | Open Targets | |
| INOTERSEN SODIUM | ? unclear | Mechanistic | Open Targets | |
| VUTRISIRAN SODIUM | ? unclear | Mechanistic | Open Targets | |
| EPLONTERSEN SODIUM | ? unclear | Mechanistic | Open Targets | |
| TRICLOSAN | ↓ inhibits | Mechanistic | 53500.0 nM (IC50) | ChEMBL |
| CHEMBL394034 | ↓ inhibits | Mechanistic | 120.0 nM (Kd) | ChEMBL |
| CHEMBL240165 | ↓ inhibits | Mechanistic | 42.0 nM (Kd) | ChEMBL |
| CHEMBL241454 | ↓ inhibits | Mechanistic | 27.0 nM (Kd) | ChEMBL |
| CHEMBL241453 | ↓ inhibits | Mechanistic | 88.0 nM (Kd) | ChEMBL |
| CHEMBL240808 | ↓ inhibits | Mechanistic | 27.0 nM (Kd) | ChEMBL |
| CHEMBL438498 | ↓ inhibits | Mechanistic | 0.2 nM (Kd) | ChEMBL |
| CHEMBL240806 | ↓ inhibits | Mechanistic | 0.3 nM (Kd) | ChEMBL |
| CHEMBL240805 | ↓ inhibits | Mechanistic | 5.7 nM (Kd) | ChEMBL |
| CHEMBL240804 | ↓ inhibits | Mechanistic | 13.9 nM (Kd) | ChEMBL |
| DEXTROTHYROXINE | ↓ inhibits | Mechanistic | 7170.0 nM (IC50) | ChEMBL |
| CHEMBL241025 | ↓ inhibits | Mechanistic | 1940.0 nM (IC50) | ChEMBL |
| CHEMBL240807 | ↓ inhibits | Mechanistic | 3160.0 nM (IC50) | ChEMBL |
| CHEMBL392872 | ↓ inhibits | Mechanistic | 41000.0 nM (IC50) | ChEMBL |
| CHEMBL238590 | ↓ inhibits | Mechanistic | 45000.0 nM (IC50) | ChEMBL |
| CHEMBL392873 | ↓ inhibits | Mechanistic | 55000.0 nM (IC50) | ChEMBL |
| CHEMBL241575 | ↓ inhibits | Mechanistic | 20000.0 nM (IC50) | ChEMBL |
| CHEMBL241573 | ↓ inhibits | Mechanistic | 20000.0 nM (IC50) | ChEMBL |
| FLUFENAMIC ACID | ↓ inhibits | Mechanistic | 2900.0 nM (IC50) | ChEMBL |
| CHEMBL60569 | ↓ inhibits | Mechanistic | 6300.0 nM (IC50) | ChEMBL |
| CHEMBL571374 | ↓ inhibits | Mechanistic | 11000.0 nM (IC50) | ChEMBL |
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.
The purpose of this study is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses of HTX-001 in human adult participants.
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
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 ...
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...
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...
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...
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...
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 ...
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 "...
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.
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 ...
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...
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...
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.
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 "..."