Verdiperstat

Summary

Verdiperstat (CHEMBL4297594) is a phase-3 clinical-stage small molecule targeting MPO; indicated across 4 conditions including multiple system atrophy and amyotrophic lateral sclerosis.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 1 (MPO)
• Indications: 4 conditions
• Clinical trials: 10
• Chemistry: 253.32 Da · C11H15N3O2S

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297594
Name	Verdiperstat
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	11528958
Molecular formula	C11H15N3O2S
Molecular weight	253.32
InChIKey	FVJCUZCRPIMVLB-UHFFFAOYSA-N

SMILES: CC(C)OCCN1C2=C(C(=O)NC1=S)NC=C2

IUPAC name: 1-(2-propan-2-yloxyethyl)-2-sulfanylidene-5H-pyrrolo[3,2-d]pyrimidin-4-one

Also known as: Azd 3241, AZD-3241, Azd3241, AZD3241, BHV-3241, BHV-3421, Verdiperstat, VERDIPERSTAT

Parent form; salt/anhydrous children: CHEMBL5191541

Patent coverage: 33 distinct patent families (140 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 137 (98%) 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
MPO	myeloperoxidase	Inhibition	6.2	0.2%	P05164

Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Myeloperoxidase.

Bioactivity

ChEMBL activities: 2 potent at pChembl ≥ 5 of 2 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
MPO	6.2	IC50	630	nM	CHEMBL_ACT_18877013
MPO	6.2	IC50	630	nM	CHEMBL_ACT_20685714

Target pathways

Aggregated over 1 target gene(s): MPO.

Top Reactome pathways

2 total, by targets touching each:

Pathway	Targets	Genes
Neutrophil degranulation	1	MPO
Events associated with phagocytolytic activity of PMN cells	1	MPO

Dominant GO biological processes

GO term	Targets
response to yeast	1
hypochlorous acid biosynthetic process	1
respiratory burst involved in defense response	1
defense response	1
response to oxidative stress	1
response to mechanical stimulus	1
removal of superoxide radicals	1
response to food	1
response to lipopolysaccharide	1
low-density lipoprotein particle remodeling	1
defense response to bacterium	1
hydrogen peroxide catabolic process	1
negative regulation of apoptotic process	1
defense response to fungus	1
response to gold nanoparticle	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
multiple system atrophy	3	MONDO:0007803	EFO:1001050
amyotrophic lateral sclerosis	2	MONDO:0004976	MONDO:0004976
Parkinson disease	2	MONDO:0005180	MONDO:0005180
frontotemporal dementia	1	MONDO:0017276	MONDO:0010857

Clinical trials

Total trials: 10.

Phase distribution

Phase	Trials
PHASE1	3
PHASE2/PHASE3	2
PHASE2	2
PHASE3	1
EARLY_PHASE1	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04297683	PHASE2/PHASE3	RECRUITING	HEALEY ALS Platform Trial - Master Protocol
NCT03952806	PHASE3	COMPLETED	Study of BHV-3241 in Participants With Multiple System Atrophy
NCT04436510	PHASE2/PHASE3	COMPLETED	HEALEY ALS Platform Trial - Regimen B Verdiperstat
NCT01527695	PHASE2	COMPLETED	PET Study in Parkinson’s Disease Patients
NCT02388295	PHASE2	COMPLETED	AZD3241 PET MSA Trial, Phase 2, Randomized,12 Week Safety and Tolerability Trial With PET in MSA Patients
NCT05184569	PHASE1	RECRUITING	Veri-T: A Trial of Verdiperstat in Patients With svPPA Due to TDP-43 Pathology
NCT00729443	PHASE1	COMPLETED	Investigating Safety, Tolerability and Pharmakinetics When Giving Single and Fractionated Doses of AZD3241 to Healthy Volunteers
NCT00914303	PHASE1	COMPLETED	Study to Evaluate Different Extended Release (ER)-Formulations and Multiple Ascending Dosing of AZD3241
NCT04616456	EARLY_PHASE1	COMPLETED	Effect of Verdiperstat on Microglial Activation in Well-characterized MSA Patients
NCT05086094	Not specified	NO_LONGER_AVAILABLE	Expanded Access Protocol of Verdiperstat in Patients With Multiple System Atrophy (MSA)

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.

25 molecules share ≥1 primary target. Top 25 by shared-target count:

Molecule	Source	Status	Shared targets
DAPSONE	ChEMBL + PubChem	Phase 4 (approved)	MPO
HYDRALAZINE	ChEMBL + PubChem	Phase 4 (approved)	MPO
ISONIAZID	ChEMBL + PubChem	Phase 4 (approved)	MPO
MEFENAMIC ACID	ChEMBL + PubChem	Phase 4 (approved)	MPO
MELATONIN	ChEMBL + PubChem	Phase 4 (approved)	MPO
METOCLOPRAMIDE	ChEMBL + PubChem	Phase 4 (approved)	MPO
PAROXETINE	ChEMBL + PubChem	Phase 4 (approved)	MPO
PROPYLTHIOURACIL	ChEMBL + PubChem	Phase 4 (approved)	MPO
TRYPTOPHAN	ChEMBL + PubChem	Phase 4 (approved)	MPO
NIMESULIDE	ChEMBL	Phase 4 (approved)	MPO
QUERCETIN	ChEMBL + PubChem	Phase 3 (approved)	MPO
FLUFENAMIC ACID	ChEMBL	Phase 2	MPO
FTIVAZIDE	ChEMBL	Phase 2	MPO
LEVOSULPIRIDE	ChEMBL	Phase 2	MPO
MITIPERSTAT	ChEMBL	Phase 2	MPO
MOLIBRESIB	ChEMBL	Phase 2	MPO
Acetaldehyde	PubChem	Approved	MPO
Aspirin	PubChem	Approved	MPO
Celecoxib	PubChem	Approved	MPO
Chloral Hydrate	PubChem	Approved	MPO
Formaldehyde	PubChem	Approved	MPO
glutaral	PubChem	Approved	MPO
protocatechualdehyde	PubChem	Approved	MPO
Serotonin	PubChem	Approved	MPO
tyrosine	PubChem	Approved	MPO