Nitric Oxide

Summary

Nitric Oxide (CHEMBL1200689) is an approved small-molecule vasodilator agent (ATC R07AX01) targeting KCNK16 and KCNK17; indicated across 57 conditions including pulmonary arterial hypertension and respiratory failure.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: R07AX01
• Targets: 2 (KCNK16, KCNK17)
• Indications: 57 conditions
• Clinical trials: 81
• Chemistry: 30.006 Da · NO

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1200689
Name	Nitric Oxide
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	145068
ChEBI	CHEBI:16480
ATC	R07AX01
Molecular formula	NO
Molecular weight	30.006
InChIKey	MWUXSHHQAYIFBG-UHFFFAOYSA-N

SMILES: [N]=O

IUPAC name: nitric oxide

ChEBI definition: A nitrogen oxide which is a free radical, each molecule of which consists of one nitrogen and one oxygen atom.

Pharmacological roles (ChEBI): neurotransmitter, signalling molecule, vasodilator agent, bronchodilator agent, radical scavenger.

Other ChEBI roles (chemical / environmental): human metabolite, Escherichia coli metabolite, mouse metabolite.

Also known as: Amidogen, oxo-, Genosyl, Inomax, Inovent, Nitric oxide, Nitric oxide trimer, Nitrogen oxide (no), Nitrosyl radical, OHM-11771, NITRIC OXIDE, nitric oxide

Patent coverage: 440,200 distinct patent families (891,773 SureChEMBL compound mentions), from 4 matched compound structure(s). One matched structure accounts for 572,580 (64%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
	Guanylyl cyclase, α1β1	Activation
KCNK16	K2P16.1			0%	Q96T55
KCNK17	K2P17.1			0.3%	Q96T54

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

Aggregated over 2 target gene(s): KCNK16, KCNK17.

Top Reactome pathways

7 total, by targets touching each:

Pathway	Targets	Genes
Neuronal System	2	KCNK16, KCNK17
Potassium Channels	2	KCNK16, KCNK17
Tandem pore domain potassium channels	2	KCNK16, KCNK17
TWIK-related alkaline pH activated K+ channel (TALK)	2	KCNK16, KCNK17
Muscle contraction	2	KCNK16, KCNK17
Phase 4 - resting membrane potential	2	KCNK16, KCNK17
Cardiac conduction	2	KCNK16, KCNK17

Dominant GO biological processes

GO term	Targets
potassium ion transport	2
potassium ion transmembrane transport	2
monoatomic ion transport	2
monoatomic ion transmembrane transport	2
endoplasmic reticulum calcium ion homeostasis	1
regulation of release of sequestered calcium ion into cytosol	1
regulation of insulin secretion involved in cellular response to glucose stimulus	1
membrane repolarization during action potential	1
regulation of action potential	1
regulation of membrane potential	1

Indications & clinical

Indications

57 indications (5 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
pulmonary arterial hypertension	4	MONDO:0015924	EFO:0001361
respiratory failure	4	MONDO:0021113	HP:0002093
respiratory system disorder	4	MONDO:0005087	EFO:0000684
pulmonary hypertension	4	MONDO:0005149	MONDO:0005149
cutaneous leishmaniasis	3	MONDO:0005446	EFO:0005046
bronchiolitis	3	MONDO:0002465	HP:0011950
lung disorder	3	MONDO:0005275	EFO:0003818
ischemia reperfusion injury	3	MONDO:0005203	EFO:0002687
congenital heart disease	3	MONDO:0005453	EFO:0005207
acute respiratory distress syndrome	3	MONDO:0006502	EFO:1000637
acute kidney injury	3	MONDO:0002492	HP:0001919
pneumonia	3	MONDO:0005249	EFO:0003106
newborn respiratory distress syndrome	3	MONDO:0700081	EFO:1000644
persistent fetal circulation syndrome	3	MONDO:0022430	EFO:1001103
aspiration pneumonitis	3	MONDO:0002572	EFO:1001399
brain injury	3	MONDO:0043510	MONDO:0043510
severe acute respiratory syndrome	3	MONDO:0005091	EFO:0000694
bronchopulmonary dysplasia	3	MONDO:0019091	MONDO:0019091
congestive heart failure	2	MONDO:0005009	EFO:0000373
pulmonary embolism	2	MONDO:0005279	EFO:0003827
neuralgia	2	MONDO:0021667	EFO:0005762
malaria	2	MONDO:0005136	EFO:0001068
opiate dependence	2	MONDO:0005530	EFO:0005611
tinea pedis	2	MONDO:0005984	EFO:0007512
stroke disorder	2	MONDO:0005098	EFO:0000712
myocardial ischemia	2	MONDO:0024644	EFO:1001375
ST-elevation myocardial infarction	2	MONDO:0041656	EFO:0008585
cardiac arrest	2	MONDO:0000745	EFO:0009492
sinusitis	2	MONDO:0005961	EFO:0007486
viral pneumonia	2	MONDO:0006012	EFO:0007541
acute myocardial infarction	2	MONDO:0004781	EFO:0008583
cystic fibrosis	2	MONDO:0009061	MONDO:0009061
sickle cell disease	2	MONDO:0011382	MONDO:0011382
coronary artery disorder	2	MONDO:0005010	EFO:0001645
drug dependence	2	MONDO:0005303	EFO:0003890
anemia	1	MONDO:0002280	EFO:0004272
decubitus ulcer	1	MONDO:0004646	EFO:0007067
chronic obstructive pulmonary disease	1	MONDO:0005002	EFO:0000341
brain ischemia	1	MONDO:0005299	MONDO:0005299
neoplasm	1	MONDO:0005070	MONDO:0004992
oral candidiasis	1	MONDO:0005886	EFO:0007406
colorectal neoplasm	1	MONDO:0005335	MONDO:0005575
idiopathic pulmonary fibrosis	0	MONDO:0800504	EFO:0000768

14 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 81.

Phase distribution

Phase	Trials
PHASE2	31
Not specified	13
PHASE1/PHASE2	10
PHASE3	9
PHASE1	8
EARLY_PHASE1	4
PHASE4	3
PHASE2/PHASE3	3

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00390065	PHASE4	COMPLETED	Follow-Up at 6 Years of a Cohort of Children Born Very Prematurely
NCT01717209	PHASE4	COMPLETED	Inhaled Nitric Oxide and Inhaled Prostacyclin After Cardiac Surgery for Heart Transplant or LVAD Placement
NCT06622291	PHASE4	COMPLETED	Perioperative Nitric Oxide Prevents Acute Kidney Injury in Acute Type A Aortic Dissection Patients
NCT04216927	PHASE3	RECRUITING	NO During CPB in Neonates to Reduce Risk of AKI
NCT00240487	PHASE3	COMPLETED	Nitric Oxide Administration for Acute Respiratory Distress Syndrome
NCT00551642	PHASE3	COMPLETED	Safety and Efficacy Study of Nitric Oxide for Inhalation on Chronic Lung Disease in Premature Babies
NCT00748423	PHASE2/PHASE3	COMPLETED	Effect of Inhaled Nitric Oxide in Acute Chest Syndrome (INOSTA Study)
NCT01503801	PHASE2/PHASE3	COMPLETED	Inhaled Nitric Oxide to Prevent and Treat Bronchopulmonary Dysplasia
NCT02836899	PHASE3	COMPLETED	Effect of Nitric Oxide in Cardiac Surgery Patients With Endothelial Dysfunction.
NCT03053388	PHASE3	COMPLETED	Evaluation of the Efficacy Safety and Tolerability of Nitric Oxide Given Intermittently Via Inhalation to Subjects With Bronchiolitis
NCT03455218	PHASE2/PHASE3	COMPLETED	Nitric Oxide Administration During Pediatric Cardiopulmonary Bypass Surgery to Prevent Platelet Activation
NCT03661385	PHASE3	COMPLETED	Nitric Oxide During Bypass for Arterial Switch Operation
NCT04807413	PHASE3	UNKNOWN	Nitric Oxide During Cardiopulmonary Bypass in Adult Surgery
NCT05109611	PHASE3	TERMINATED	Nitric Oxide Nasal Spray (NONS) as Prevention for Treatment of Individuals at Risk of Exposure to COVID-19 Infection
NCT05599919	PHASE3	COMPLETED	Nitric Oxide Nasal Spray (NONS) To Treat and Prevent the Exacerbation of Infection in Individuals With Mild COVID-19
NCT03023449	PHASE2	ACTIVE_NOT_RECRUITING	Diffuse Optical Monitoring With Inhaled Nitric Oxide
NCT00001716	PHASE2	COMPLETED	Effects of Nitric Oxide and Nitroglycerin in Patients With Sickle Cell Anemia
NCT00014040	PHASE2	COMPLETED	Nitric Oxide Inhalation Therapy to Relieve Chest Pain in Patients With Coronary Artery Disease
NCT00060840	PHASE2	COMPLETED	The Effects of Nitric Oxide for Inhalation During Left Ventricular Assists Device (LVAD) Implantation
NCT00094887	PHASE2	COMPLETED	Nitric Oxide Inhalation to Treat Sickle Cell Pain Crises
NCT00142051	PHASE2	TERMINATED	Inhaled Nitric Oxide for Pediatric Painful Sickle Crisis
NCT00568061	PHASE2	TERMINATED	Nitric Oxide in Myocardial Infarction Size
NCT00570245	PHASE2	COMPLETED	Safety and Efficacy Study of Nitric Oxide in Patients Going Through Lung Transplantation
NCT00585013	PHASE1/PHASE2	COMPLETED	Inhaled NO as an Anti-inflammatory and Anti-reperfusion Agent in Infants and Children Undergoing Cardiopulmonary Bypass
NCT00613808	PHASE2	TERMINATED	Safety and Efficacy of Gaseous Nitric Oxide on Venous Stasis Leg Ulcers
NCT00771342	PHASE2	COMPLETED	Efficacy Study to Evaluate the Effect of Nitric Oxide on the Treatment of Tinea Pedis (Athlete’s Foot)
NCT00771368	PHASE1/PHASE2	TERMINATED	Reduction of Bacteria in MRSA Positive Ulcers
NCT00790608	PHASE2	TERMINATED	Clinical Trial on the Reduction of Methicillin Resistant Staphylococcus Aureus (MRSA)
NCT00823095	PHASE2	TERMINATED	Topical Nitric Oxide Trial in Chronic Non-Healing Wounds
NCT01165047	PHASE2	COMPLETED	Nitric Oxide, GeNO Nitrosyl Delivery System
NCT01351493	PHASE1/PHASE2	UNKNOWN	Study on the Safety and Efficacy of Nitric Oxide Gel in Subjects With Pressure Sore
NCT01398384	PHASE2	COMPLETED	Effects of Nitric Oxide for Inhalation in Myocardial Infarction Size
NCT01768884	PHASE1/PHASE2	COMPLETED	Evaluation of the Safety and Tolerability of Nitric Oxide (NO) Via Inhalation to Subjects With Bronchiolitis
NCT01958944	PHASE1/PHASE2	COMPLETED	Evaluation of the Safety and Tolerability of Inhaled Nitric Oxide to Subjects With Cystic Fibrosis
NCT02217683	PHASE2	WITHDRAWN	Use of Inhaled Nitric Oxide to Prevent Pulmonary Hypertension Associated to Stored Blood Transfusion
NCT02295566	PHASE2	COMPLETED	RATNO, Reducing Antibiotic Tolerance Using Nitric Oxide in CF - a Phase 2 Pilot Study
NCT02345616	PHASE2	UNKNOWN	Role of the Nitric Oxide (NO) in Pre-oxygenation Before Anesthetic Induction in Patients With a Pulmonary Hypertension in Cardiac Surgery. Pilot Study of Feasibility
NCT02498535	PHASE2	TERMINATED	Efficacy and Safety of Inhaled Nitric Oxide (NO) in Cystic Fibrosis (CF) Patients
NCT02734953	PHASE2	COMPLETED	Effect of iNO on Invasively Derived Pulmonary Pressures in Patients With PAH
NCT02871011	PHASE2	COMPLETED	Nitric Oxide Releasing Solution (NORS) Footbath to Treat Athlete’s Foot

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 0 clinical and 1 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

Related molecules

Related molecules

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

3 molecules share ≥1 primary target. Top 3 by shared-target count:

Molecule	Source	Status	Shared targets
Chloroform	PubChem	Approved	KCNK16, KCNK17
Isoflurane	PubChem	Approved	KCNK16
Quinidine	PubChem	Approved	KCNK16