Ion channel drug candidates in preclinical development and clinical trials

Nowadays more than 80 ion channel drug candidates are in preclinical development or clinical trials.
Updated September 2020

NameOther namesMechanism of actionIndicationTypeStatusCompany
ACP-044CT-044
Reactive species decomposition accelerant preventing hyper-activation of TRPA1
Acute post-operative, chronic neuropathic pain
Phase I
ACADIA Pharmaceuticals
AM-6120, AM-8145, AM-0422Nav1.7 inhibitorPeptideDiscoveryAmgen
AP30663KCa inhibitorAtrial fibrillationPhase I/IIAcesion Pharma
AP-325
Positive allosteric modulator of GABA-A
Neuropathic pain
Small Molecule
Phase IIa
Algiax Pharmaceuticals
ASN008Na+ channel blockerPruritus, atopic dermatitis, painPhase IIaAsana BioSciences
ASN009P2X3 antagonistCoughPreclinicalAsana BioSciences
ASP-0819KCa3.1 activatorFibromyalgiaPhase IIAstellas Pharma
AUT00201Modulator of Kv3 channelsSchizophrenia, hearing disordersPhase IAutifony Therapeutics
AuxoraCM4620-IECRAC inhibitorSevere COVID-19 pneumonia, Viral pneumonia, Acute pancreatitisSmall MoleculePhase IICalciMedica
AV-1014-Cl-KYN
NMDAR antagonist
CNS disorders
Preclinical
VistaGen Therapeutics
BAY-1817080P2X3 inhibitorEndometriosis and persistent
chronic cough
Phase IBayer
BAY-1902607P2X3 inhibitorPersistent chronic coughPhase IIBayer
BAY-2253651TASK inhibitorObstructive sleep apnoeaPhase IIBayer
BIIB093SUR1-TRPM4 blockerBrain ContusionSmall MoleculePhase IIBiogen
BIIB095Nav1.7 inhibitorNeuropathic painSmall MoleculePhase IBiogen
BIIB104PF-04958242AMPAR activatorCognitive impairment associated
with schizophrenia
Phase IIaBiogen
BIL010tnfP2X7Skin cancerAntibodyPhase IBiosceptre
BIL06vnfP2X7Skin cancerPeptide vaccinePhase IBiosceptre
BLU-5937P2X3 antagonistRefractory chronic coughPhase IIbBELLUS Health
BNC-210alpha7 nAChRs antagonistAgitation, post-traumatic stress disorder, generalised anxiety disorder Phase IIBionomics
CAD-1883KCa activatorAtaxia or essential tremorsPhase I/IICadent Therapeutics
CC 8464ASP 1807Nav1.7 antagonistNeuropathic and inflammatory painSmall MoleculePhase IChromocell/Astellas Pharma
CVL-865Positive allosteric modulator of alpha-2/3/5 subunits of the GABA-ADrug-resistant focal onset epilepsyPhase I/IICerevel Therapeutics
CVL-865Positive allosteric modulator of alpha-2/3/5 subunits of the GABA-AAcute anxietyPhase ICerevel Therapeutics
CX-8998 T-type calcium channel modulatorAbsence epilepsy, essential tremorPhase IICavion 
DalazatideShK-186Kv1.3 inhibitorMyositis, Psoriasis Synthetic peptidePhase IIKv1.3 Therapeutics
DSP-2230NaV1.7 and NaV1.8 inhibitorNeuropathic painSmall MoleculePhase IAlphaNavi Pharma
DSP-3905NaV1.7 inhibitorNeuropathic painSmall MoleculePhase ISunovion Pharmaceuticals
ENX-101sub-type selective GABA-A modulatorDiseases of the nervous systemPreclinicalEngrail Therapeutics
ETD001ENaC blockerRespiratory diseasesPreclinicalEnterprise Therapeutics
ETD002TMEM16A potentiatorCystic fibrosisPhase IEnterprise Therapeutics
EvenamideNav blockerSchizophreniaPhase IINewron Pharmaceuticals
Evt201GABAA receptor PAMInsomniaPhase IIEvotec
FX301FunapideNav1.7 inhibitorAcute post-operative painPreclinicalFlexion
GalicaftorABBV-2222CFTRCystic fibrosisPhase IIAbbVie
GFB-887TRPC5 inhibitorFocal segmental glomerulosclerosis, diabetic nephropathyPhase I/IIGoldfinch Bio
Granexin gelalphaCT1, a peptide mimetic of the Cx43 carboxyl terminusCutaneous
radiation injury, Diabetic foot and venous leg ulcers
Synthetic peptidePhase IIIFirstString Research
GRC 17536TRPA1 antagonistPainful diabetic neuropathyPhase IIGlenmark 
GSK-2798745TRPV4 inhibitorHeart failurePhase IIGlaxoSmithKline
HalneuronTetrodotoxinNav blockerSevere neuropathic pain, cancer related painSmall MoleculePhase IIIWex Pharmaceuticals
HBI-3000Multi-channel inhibitorAtrial fibrillationPhase IHUYA Bioscience International
JNJ-55308942P2X7 inhibitorNeuroinflammation and anhedoniaPhase IJohnson & Johnson
KB-3061Kv7 modulatorKCNQ2 epileptic encephalopathyPreclinicalKnopp Biosciences
KCP-506alpha9alpha10 nAChR antagonistChronic painPhase IKineta 
Kv7 modulatorKv7 modulatorNeuropathic pain, hearing disorders, ALSDiscoveryKnopp Biosciences
MK-7264GefapixantP2X3 antagonistChronic coughSmall MoleculePhase IIIMerck, Roche
NBI-1065845TAK-653AMPAR activatorDepressionPhase IINeurocrine Biosciences, Takeda
NBI-827104ACT-709478T-type calcium channel blockerPediatric epilepsyPhase I/IINeurocrine Biosciences, Idorsia
NBI-921352XEN901Nav1.6 inhibitorEpilepsySmall MoleculePhase I/IINeurocrine Biosciences, Xenon
NMD670Skeletal muscle specific inhibitor of ClC-1Myasthenia gravisSmall MoleculePhase I/IIaNMD Pharma
NOC-100
NTX-1175
Charged sodium channel blocker
Chronic and acute cough
Small Molecule
Phase I
Nocion Therapeutics
OV101GaboxadolGABA-A agonistFragile X syndrome, Angelman syndromeSmall MoleculePhase II-IIIOvid Therapeutics
P-1037VX-371ENaC inhibitorPulmonary diseasesPhase IIParion Sciences, Vertex Pharmaceuticals 
P-1055VX-551ENaC inhibitorPulmonary diseasesPreclinicalParion Sciences, Vertex Pharmaceuticals 
P-321ENaC inhibitorDry eye diseasePhase IIParion Sciences, Takeda
P2X4 inhibitorP2X4 inhibitorEndometriosisPhase IBayer
P2X7 MABP2X7 monoclonal antibodyAutoimmune disordersAntibodyPreclinicalIntegral Molecular
PRAX-114GABA-A positive allosteric modulatorMajor depressive disorder, perimenopausal depressionSmall MoleculePhase IIPraxis Precision Medicines
PRAX-944T-type calcium channel blockerEssential tremorSmall MoleculePhase IIPraxis Precision Medicines
PRAX-562Pediatric epilepsy, adult cephalgiaSmall MoleculePhase IPraxis Precision Medicines
PRAX-020/021Genetic epilepsiesPreclinicalPraxis Precision Medicines
PRAX-222Genetic epilepsiesPreclinicalPraxis Precision Medicines
RalfinamideNav blocker, Cav2.2 blockerNeuropathic painPhase IINewron Pharmaceuticals
ResiniferatoxinTRPV1 agonistCancer painSmall MoleculePhase ISorrento Therapeutics
RG-7816GABA-A receptor alpha5
subunit-targeting NAM
Autism spectrum disorderPhase IRoche
S-600918P2X3 antagonistRefractory chronic coughPhase IIbShionogi
SAN711GABAA alpha3 modulatorNeuropathic pain disordersPreclinicalSaniona
SAN903KCa3.1 inhibitorRare inflammatory/fibrotic diseases, hereditary xerocytosis PreclinicalSaniona
SAGE-217Zuranolon, S-812217GABA-A positive allosteric modulatorMajor depressionSmall MoleculePhase IISage Therapeutics, Shionogi
SAGE-324GABA-A receptor PAMEssential tremor and Parkinson
disease
Phase ISage Therapeutics
SAGE-689GABA-A modulatorGABA hypofunctionPreclinicalSage Therapeutics
SAGE-718NMDAR activatorNMDAR hypofunctionPhase ISage Therapeutics
SAGE-904NMDAR modulatorNMDAR hypofunctionPreclinicalSage Therapeutics
SenicapocKCa3.1 inhibitorHereditary xerocytosis and
Alzheimer disease
Phase ISpringWorks Therapeutics/
University of California, Davis
SOR-C13TRPV6 inhibitorSolid tumor cancerSynthetic peptidePhase IbSoricimed Biopharma
ST-2427Nav1.7 inhibitorPost-operative painSmall MoleculeINDSiteOne Therapeutics
STK-001Upregulates Nav1.1 protein expressionDravet syndromeAntisense oligonucleotidePhase I/IIaStoke Therapeutics
TRPC4 and TRPC5 inhibitorTRPC4 and TRPC5 inhibitorAnxiety disorder and depressionPhase IHydra/Boehringer Ingelheim
VX-150Nav1.8 inhibitorAcute and neuropathic painSmall MoleculePhase IIVertex Pharmaceuticals 
XEN007FlunarizineCav2.1 modulatorMigraine and other neurological disordersSmall MoleculePhase IXenon
XEN1101Kv7 modulatorEpilepsyPhase IIXenon
XEN496Retigabine Kv7 modulatorKCNQ2 and pediatric epilepsyPhase IIXenon
XEN-D0501TRPV1 antagonist Type 2 diabetesPhase IIPila Pharma