Fasudil

Summary

Fasudil (CHEMBL38380) is a phase-3 clinical-stage small-molecule EC 2.7.11.1 (non-specific serine/threonine protein kinase) inhibitor (ATC C04AX32) targeting ROCK1 and ROCK2; indicated across 5 conditions including cardiovascular disorder and retinal vein occlusion; with CIViC clinical evidence for 1 variant-indication association (e.g. GATA2 EXPRESSION in lung adenocarcinoma).

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• ATC class: C04AX32
• Targets: 2 (ROCK1, ROCK2)
• Indications: 5 conditions
• Clinical trials: 11
• Precision-oncology evidence (CIViC): 1 variant–indication association
• Chemistry: 291.37 Da · C14H17N3O2S

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL38380
Name	Fasudil
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	3547
ChEBI	CHEBI:43871
ATC	C04AX32
Molecular formula	C14H17N3O2S
Molecular weight	291.37
InChIKey	NGOGFTYYXHNFQH-UHFFFAOYSA-N

SMILES: C1CNCCN(C1)S(=O)(=O)C2=CC=CC3=C2C=CN=C3

IUPAC name: 5-(1,4-diazepan-1-ylsulfonyl)isoquinoline

ChEBI definition: An isoquinoline substituted by a (1,4-diazepan-1-yl)sulfonyl group at position 5. It is a Rho-kinase inhibitor and its hydrochloride hydrate form is approved for the treatment of cerebral vasospasm and cerebral ischemia.

Pharmacological roles (ChEBI): geroprotector, EC 2.7.11.1 (non-specific serine/threonine protein kinase) inhibitor, vasodilator agent, nootropic agent, neuroprotective agent, antihypertensive agent, calcium channel blocker.

Also known as: AT 877, AT-877, Fasudil, HA 1077, HA-1077, NSC-759827, ZK-258594, fasudil, SID11113364, SID26751605, SID92708292, SID104171358

Parent form; salt/anhydrous children: CHEMBL541388, CHEMBL4459126

Patent coverage: 3,507 distinct patent families (11,953 SureChEMBL compound mentions), from 3 matched compound structure(s). One matched structure accounts for 11,425 (96%) 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
ROCK1	Rho associated coiled-coil containing protein kinase 1	Inhibition	5.57	1.4%	Q13464
ROCK2	Rho associated coiled-coil containing protein kinase 2	Inhibition	5.89	1.1%	O75116

Broader ChEMBL bioactivity targets: 60 (assay-derived). Sample: Leucine-rich repeat serine/threonine-protein kinase 2, Survival motor neuron protein, C-C motif chemokine 2, Cyclin-dependent kinase 13, 5-hydroxytryptamine receptor 2B, Alpha-2A adrenergic receptor, Alpha-2B adrenergic receptor, Progesterone receptor, Menin/Histone-lysine N-methyltransferase MLL, Protein kinase C (PKC).

Bioactivity

ChEMBL activities: 111 potent at pChembl ≥ 5 of 133 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
ROCK1	7.5	Ki	31.62	nM	CHEMBL_ACT_9656597
ROCK1	7.5	Ki	31.62	nM	CHEMBL_ACT_9660835
ROCK2	7.35	Kd	45	nM	CHEMBL_ACT_26618873
ROCK1	7.3	Kd	50	nM	CHEMBL_ACT_26618870
PRKX	7.3	Ki	50.12	nM	CHEMBL_ACT_9581368
PRKX	7.3	Ki	50.12	nM	CHEMBL_ACT_9585119
ROCK2	7.3	Ki	50.12	nM	CHEMBL_ACT_9631141
ROCK2	7.3	Ki	50.12	nM	CHEMBL_ACT_9631341
ROCK2	7.11	Kd	78	nM	CHEMBL_ACT_10863424
PRKACA	7.1	Ki	79.43	nM	CHEMBL_ACT_9649222
PRKACA	7.1	Ki	79.43	nM	CHEMBL_ACT_9657626
PKN3	7	Kd	99	nM	CHEMBL_ACT_17927716
ROCK1	7	Ki	100	nM	CHEMBL_ACT_5123977
CLK1	6.82	Kd	152	nM	CHEMBL_ACT_17892370
ROCK2	6.8	IC50	158	nM	CHEMBL_ACT_13836550
ROCK2	6.8	IC50	158	nM	CHEMBL_ACT_24662732
ROCK2	6.8	IC50	158	nM	CHEMBL_ACT_24973406
ROCK2	6.8	IC50	158	nM	CHEMBL_ACT_29095221
ROCK2	6.75	IC50	180	nM	CHEMBL_ACT_1854724
CYP2D6	6.7	Potency	199.5	nM	CHEMBL_ACT_4982145
CYP2D6	6.7	AC50	199.5	nM	CHEMBL_ACT_6050196
ROCK2	6.66	IC50	220	nM	CHEMBL_ACT_22401859
ROCK1	6.58	IC50	260	nM	CHEMBL_ACT_14543540
ROCK1	6.58	IC50	260	nM	CHEMBL_ACT_16493392
ROCK1	6.58	IC50	260	nM	CHEMBL_ACT_22396440
ROCK1	6.52	IC50	300	nM	CHEMBL_ACT_8047297
ROCK2	6.5	IC50	320	nM	CHEMBL_ACT_14543544
ROCK2	6.5	IC50	320	nM	CHEMBL_ACT_16493393
ROCK2	6.5	IC50	320	nM	CHEMBL_ACT_22396439
CLK4	6.5	Ki	316.2	nM	CHEMBL_ACT_9682868

Target pathways

Aggregated over 2 target gene(s): ROCK1, ROCK2.

Top Reactome pathways

40 total, by targets touching each:

Pathway	Targets	Genes
Developmental Biology	2	ROCK1, ROCK2
Signal Transduction	2	ROCK1, ROCK2
Disease	2	ROCK1, ROCK2
Signaling by VEGF	2	ROCK1, ROCK2
Signaling by Rho GTPases	2	ROCK1, ROCK2
RHO GTPase Effectors	2	ROCK1, ROCK2
EPH-Ephrin signaling	2	ROCK1, ROCK2
Signaling by GPCR	2	ROCK1, ROCK2
Semaphorin interactions	2	ROCK1, ROCK2
GPCR downstream signalling	2	ROCK1, ROCK2
EPHB-mediated forward signaling	2	ROCK1, ROCK2
EPHA-mediated growth cone collapse	2	ROCK1, ROCK2
Sema4D in semaphorin signaling	2	ROCK1, ROCK2
G alpha (12/13) signalling events	2	ROCK1, ROCK2
Sema4D induced cell migration and growth-cone collapse	2	ROCK1, ROCK2
Axon guidance	2	ROCK1, ROCK2
VEGFA-VEGFR2 Pathway	2	ROCK1, ROCK2
RHO GTPases Activate ROCKs	2	ROCK1, ROCK2
Infectious disease	2	ROCK1, ROCK2
RHOA GTPase cycle	2	ROCK1, ROCK2
Signaling by Receptor Tyrosine Kinases	2	ROCK1, ROCK2
RHO GTPase cycle	2	ROCK1, ROCK2
RHOB GTPase cycle	2	ROCK1, ROCK2
RHOC GTPase cycle	2	ROCK1, ROCK2
RHOH GTPase cycle	2	ROCK1, ROCK2
RHOBTB1 GTPase cycle	2	ROCK1, ROCK2
Nervous system development	2	ROCK1, ROCK2
Potential therapeutics for SARS	2	ROCK1, ROCK2
SARS-CoV Infections	2	ROCK1, ROCK2
RHOBTB GTPase Cycle	2	ROCK1, ROCK2

Dominant GO biological processes

GO term	Targets
mitotic cytokinesis	2
epithelial to mesenchymal transition	2
aortic valve morphogenesis	2
smooth muscle contraction	2
signal transduction	2
Rho protein signal transduction	2
positive regulation of cardiac muscle hypertrophy	2
positive regulation of gene expression	2
negative regulation of angiogenesis	2
actin cytoskeleton organization	2
regulation of cell adhesion	2
cortical actin cytoskeleton organization	2
actomyosin structure organization	2
regulation of actin cytoskeleton organization	2
positive regulation of MAPK cascade	2

Indications & clinical

Indications

5 indications (0 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
cardiovascular disorder	3	MONDO:0004995	EFO:0000319
retinal vein occlusion	2	MONDO:0006951	EFO:1001157
retinopathy of prematurity	2	MONDO:0006952	EFO:1001158
dementia	2	MONDO:0001627	HP:0000726
amyotrophic lateral sclerosis	2	MONDO:0004976	MONDO:0004976

Clinical trials

Total trials: 11.

Phase distribution

Phase	Trials
PHASE2	8
PHASE2/PHASE3	2
PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06861192	PHASE2/PHASE3	RECRUITING	Clinical Study of Fasudil Hydrochloride and PD1 Inhibitor Combined With Androgen Deprivation in Neoadjuvant Therapy of Prostate Cancer
NCT00498615	PHASE3	COMPLETED	A Rho-kinase Inhibitor (Fasudil) in the Treatment of Raynaud’s Phenomenon
NCT03391219	PHASE2/PHASE3	UNKNOWN	Combined Intravitreal Injection of Bevacizumab and Fasudil Versus Bevacizumab Alone for Macular Edema Secondary to Retinal Vein Occlusion in Previously Treated Patients
NCT05218668	PHASE2	ACTIVE_NOT_RECRUITING	Rho Kinase Inhibitor in Amyotrophic Lateral Sclerosis (REAL)
NCT06362707	PHASE2	RECRUITING	Fasudil Trial for Treatment of Early Alzheimer’s Disease (FEAD)
NCT07250542	PHASE2	RECRUITING	A Clinical Study on the Efficacy and Safety of Fasudil Hydrochloride Combined With Immunotherapy in the Treatment of Metastatic Castration-resistant Prostate Cancer
NCT00120718	PHASE2	COMPLETED	The Effect of Fasudil on Vascular Function in Humans
NCT01935518	PHASE2	UNKNOWN	A Clinical Trial of Safety and Efficacy of Fasudil in Subjects With Amyotrophic Lateral Sclerosis (ALS)
NCT03792490	PHASE2	COMPLETED	Inhibition of Rho Kinase (ROCK) With Fasudil as Disease-modifying Treatment for ALS
NCT04734379	PHASE2	UNKNOWN	Rho Kinase (ROCK) Inhibitor in Tauopathies - 1
NCT04793659	PHASE2	COMPLETED	Fasudil fOr redUcing elopemeNt and Spatial Disorientation

Clinical evidence (CIViC)

Variant × indication × effect (1 predictive associations from 1 curated evidence items):

Variant	Indication	Effect	Therapy	Level	CIViC
GATA2 EXPRESSION	Lung Adenocarcinoma	Sensitivity/Response	Bortezomib + Fasudil	CIViC D	EID301

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

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.

51 molecules share ≥1 primary target. Top 51 by shared-target count:

Molecule	Source	Status	Shared targets
BELUMOSUDIL	ChEMBL + PubChem	Phase 4 (approved)	ROCK1, ROCK2
CRIZOTINIB	ChEMBL + PubChem	Phase 4 (approved)	ROCK1, ROCK2
BARICITINIB	ChEMBL	Phase 4 (approved)	ROCK1, ROCK2
BOSUTINIB	ChEMBL	Phase 4 (approved)	ROCK1, ROCK2
CAPIVASERTIB	ChEMBL	Phase 4 (approved)	ROCK1, ROCK2
FEDRATINIB	ChEMBL	Phase 4 (approved)	ROCK1, ROCK2
MIDOSTAURIN	ChEMBL	Phase 4 (approved)	ROCK1, ROCK2
MOMELOTINIB	ChEMBL	Phase 4 (approved)	ROCK1, ROCK2
NETARSUDIL	ChEMBL	Phase 4 (approved)	ROCK1, ROCK2
RUXOLITINIB	ChEMBL	Phase 4 (approved)	ROCK1, ROCK2
SUNITINIB	ChEMBL	Phase 4 (approved)	ROCK1, ROCK2
TOFACITINIB	ChEMBL	Phase 4 (approved)	ROCK1, ROCK2
UPADACITINIB	ChEMBL	Phase 4 (approved)	ROCK1, ROCK2
AFURESERTIB	ChEMBL	Phase 3	ROCK1, ROCK2
ALVOCIDIB	ChEMBL	Phase 3	ROCK1, ROCK2
DOVITINIB	ChEMBL	Phase 3	ROCK1, ROCK2
LESTAURTINIB	ChEMBL	Phase 3	ROCK1, ROCK2
LINIFANIB	ChEMBL	Phase 3	ROCK1, ROCK2
RIPASUDIL	ChEMBL	Phase 3	ROCK1, ROCK2
CENISERTIB	ChEMBL	Phase 2	ROCK1, ROCK2
DECERNOTINIB	ChEMBL	Phase 2	ROCK1, ROCK2
ILORASERTIB	ChEMBL	Phase 2	ROCK1, ROCK2
LAUROGUADINE	ChEMBL	Phase 2	ROCK1, ROCK2
R-406	ChEMBL	Phase 2	ROCK1, ROCK2
RG-547	ChEMBL	Phase 2	ROCK1, ROCK2
RIPASUDIL HYDROCHLORIDE DIHYDRATE	ChEMBL	Phase 2	ROCK1, ROCK2
SAR-407899 FREE BASE	ChEMBL	Phase 2	ROCK1, ROCK2
SU-014813	ChEMBL	Phase 2	ROCK1, ROCK2
UCN-01	ChEMBL	Phase 2	ROCK1, ROCK2
UPROSERTIB	ChEMBL	Phase 2	ROCK1, ROCK2
VEROSUDIL	ChEMBL	Phase 2	ROCK1, ROCK2
ZOTIRACICLIB	ChEMBL	Phase 2	ROCK1, ROCK2
Afatinib	PubChem	Approved	ROCK1, ROCK2
Binimetinib	PubChem	Approved	ROCK1, ROCK2
dacomitinib	PubChem	Approved	ROCK1, ROCK2
Fostamatinib	PubChem	Approved	ROCK1, ROCK2
Gefitinib	PubChem	Approved	ROCK1, ROCK2
Idelalisib	PubChem	Approved	ROCK1, ROCK2
Pazopanib	PubChem	Approved	ROCK1, ROCK2
regorafenib	PubChem	Approved	ROCK1, ROCK2
Selumetinib	PubChem	Approved	ROCK1, ROCK2
Trametinib	PubChem	Approved	ROCK1, ROCK2
CERITINIB	ChEMBL	Phase 4 (approved)	ROCK2
PALBOCICLIB	ChEMBL	Phase 4 (approved)	ROCK2
VANDETANIB	ChEMBL	Phase 4 (approved)	ROCK2
BMS-690514	ChEMBL	Phase 2	ROCK1
FORETINIB	ChEMBL	Phase 2	ROCK2
PH-797804	ChEMBL	Phase 2	ROCK2
SIMUROSERTIB	ChEMBL	Phase 2	ROCK1
VX-702	ChEMBL	Phase 2	ROCK1
Belzutifan	PubChem	Approved	ROCK2