Iclepertin

Summary

Iclepertin (CHEMBL5314576) is a phase-3 clinical-stage small molecule targeting SLC6A9; indicated across 4 conditions including alzheimer disease and kidney failure.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 1 (SLC6A9)
• Indications: 4 conditions
• Clinical trials: 22
• Chemistry: 512.4 Da · C20H18F6N2O5S

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL5314576
Name	Iclepertin
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	155259577
Molecular formula	C20H18F6N2O5S
Molecular weight	512.4
InChIKey	MYHDQTVHHMSLEF-DDBGAENHSA-N

SMILES: C[C@H](C(F)(F)F)OC1=C(C=C(C=C1)S(=O)(=O)C)C(=O)N2C[C@@H]3C[C@@]3(C2)C4=NOC(=C4)C(F)(F)F

IUPAC name: [5-methylsulfonyl-2-[(2R)-1,1,1-trifluoropropan-2-yl]oxyphenyl]-[(1R,5R)-1-[5-(trifluoromethyl)-1,2-oxazol-3-yl]-3-azabicyclo[3.1.0]hexan-3-yl]methanone

Also known as: BI 425809, Iclepertin, ICLEPERTIN

Patent coverage: 5 distinct patent families (14 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 12 (86%) 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
SLC6A9	GlyT1	Inhibition	8.3	0.7%	P48067

Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Sodium- and chloride-dependent glycine transporter 1.

Bioactivity

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

Target	pChembl	Type	Value	Unit	Activity ID
SLC6A9	8.3	IC50	5	nM	CHEMBL_ACT_26335998

Target pathways

Aggregated over 1 target gene(s): SLC6A9.

Top Reactome pathways

4 total, by targets touching each:

Pathway	Targets	Genes
Transport of small molecules	1	SLC6A9
R-HSA-425366	1	SLC6A9
SLC-mediated transmembrane transport	1	SLC6A9
SLC-mediated transport of neurotransmitters	1	SLC6A9

Dominant GO biological processes

GO term	Targets
neurotransmitter uptake	1
glycine transport	1
sodium ion transmembrane transport	1
positive regulation of hemoglobin biosynthetic process	1
regulation of synaptic transmission, glycinergic	1
glycine secretion, neurotransmission	1
positive regulation of heme biosynthetic process	1
transport across blood-brain barrier	1
glycine import across plasma membrane	1
amino acid transmembrane transport	1
neurotransmitter transport	1
amino acid transport	1
transmembrane transport	1

Indications & clinical

Indications

4 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
Alzheimer disease	2	MONDO:0004975	MONDO:0004975
kidney failure	1	MONDO:0001106	HP:0000083

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

Clinical trials

Total trials: 22.

Phase distribution

Phase	Trials
PHASE1	17
PHASE3	4
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04846868	PHASE3	COMPLETED	Clinical Trial of Iclepertin Effect on Cognition and Functional Capacity in Schizophrenia (CONNEX-1)
NCT04846881	PHASE3	COMPLETED	Clinical Trial of Iclepertin Effect on Cognition and Functional Capacity in Schizophrenia (CONNEX-2)
NCT04860830	PHASE3	COMPLETED	CONNEX-3: A Study to Test Whether Iclepertin Improves Learning and Memory in People With Schizophrenia
NCT05211947	PHASE3	TERMINATED	A Study to Test Long-term Safety of Iclepertin in People With Schizophrenia Who Took Part in a Previous CONNEX Study
NCT03859973	PHASE2	COMPLETED	This Study Tests Whether BI 425809 Together With Brain Training Using a Computer Improves Mental Functioning in Patients With Schizophrenia
NCT02337283	PHASE1	COMPLETED	Safety, Tolerability, and Pharmacokinetics of Multiple Rising Doses of BI 425809 Tablets for 12 Days to Young and Elderly Healthy Male and Female Volunteers and Comparison of Pharmacokinetics of a Single Oral Dose of BI 425809 (Morning Versus Evening)
NCT02342717	PHASE1	COMPLETED	Relative Bioavailability of a Single Oral Dose of BI 425809 When Administered Alone or in Combination With Multiple Oral Doses of Itraconazole in Healthy Male Subjects
NCT02362516	PHASE1	COMPLETED	Pharmacokinetics and Pharmacodynamic Effect of Different Multiple Oral Doses of BI 425809
NCT02383888	PHASE1	COMPLETED	Single Rising Dose Trial of BI 425809 for Healthy Japanese and Chinese Male Subjects
NCT02783040	PHASE1	COMPLETED	Interaction of BI 425809 With Midazolam, Warfarin, Omeprazole and Digoxin
NCT03082183	PHASE1	COMPLETED	A Study in Healthy Men to Test Whether Rifampicin Influences the Amount of BI 425809 in the Blood
NCT03783000	PHASE1	COMPLETED	A Study in Healthy Men to Measure the Amount of BI 425809 in the Blood When Taken as a Tablet
NCT03905096	PHASE1	COMPLETED	A Study in Healthy Men and Women to Test Which Effects Donepezil and BI 425809 Have on Each Other
NCT03988803	PHASE1	COMPLETED	A Study in Healthy Men and Women to Test Which Effects Memantine and BI 425809 Have on Each Other
NCT04602221	PHASE1	COMPLETED	A Study in Healthy Men to Test Whether BI 409306, BI 425809 or Lamotrigine Can Reverse the Memory Problems Caused by Ketamine
NCT05076409	PHASE1	COMPLETED	A Study in Healthy Men to Test How Fluconazole Influences the Amount of BI 425809 in the Blood
NCT05347004	PHASE1	COMPLETED	A Study in Healthy People to Test How BI 425809 is Taken up in the Body When Taken With or Without Food
NCT05613777	PHASE1	COMPLETED	A Study in Healthy Women to Test Whether BI 425809 Influences the Amount of a Contraceptive in the Blood
NCT05718843	PHASE1	COMPLETED	A Study to Test How Iclepertin is Taken up in the Blood of People With and Without Kidney Problems
NCT05723874	PHASE1	COMPLETED	A Study in Healthy Men to Test Whether Bosentan Influences the Amount of BI 425809 in the Blood
NCT05731895	PHASE1	COMPLETED	A Study to Test How Iclepertin is Taken up in the Blood of People With and Without Liver Problems
NCT06070597	PHASE1	COMPLETED	A Study in Healthy People to Test Whether Iclepertin Has an Effect on Cardiac Safety

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

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.

5 molecules share ≥1 primary target. Top 5 by shared-target count:

Molecule	Source	Status	Shared targets
GLYCINE	ChEMBL	Phase 4 (approved)	SLC6A9
BITOPERTIN	ChEMBL	Phase 3	SLC6A9
ORG-25935	ChEMBL	Phase 2	SLC6A9
PF-03463275	ChEMBL	Phase 2	SLC6A9
SARCOSINE	ChEMBL	Phase 2	SLC6A9