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Rilzabrutinib

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Also known as Prn-1008Prn1008Rilzabrutinib, (.alpha.e,3s)-(.ALPHA.E,3S)-(E)-RILZABRUTINIB(E)-PRN1008

Summary

Rilzabrutinib (CHEMBL3702854) is a phase-3 clinical-stage small molecule targeting BTK; indicated across 10 conditions including pemphigus vulgaris and autoimmune thrombocytopenic purpura.

At a glance

  • Status: Max clinical phase 3 (not approved)
  • Modality: Small molecule
  • Targets: 1 (BTK)
  • Indications: 10 conditions
  • Clinical trials: 18
  • Chemistry: 665.8 Da · C36H40FN9O3

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL3702854
NameRilzabrutinib
TypeSmall molecule
Max phase3
FDA approvedno
PubChem CID73388818
Molecular formulaC36H40FN9O3
Molecular weight665.8
InChIKeyLCFFREMLXLZNHE-GBOLQPHISA-N

SMILES: CC(C)(/C=C(\C#N)/C(=O)N1CCC[C@H](C1)N2C3=NC=NC(=C3C(=N2)C4=C(C=C(C=C4)OC5=CC=CC=C5)F)N)N6CCN(CC6)C7COC7

IUPAC name: (E)-2-[(3R)-3-[4-amino-3-(2-fluoro-4-phenoxyphenyl)pyrazolo[3,4-d]pyrimidin-1-yl]piperidine-1-carbonyl]-4-methyl-4-[4-(oxetan-3-yl)piperazin-1-yl]pent-2-enenitrile

Also known as: Prn-1008, Prn1008, PRN1008, Rilzabrutinib, Rilzabrutinib, (.alpha.e,3s)-, PRN-1008, RILZABRUTINIB, (.ALPHA.E,3S)-, (E)-RILZABRUTINIB, (E)-PRN1008

Patent coverage: 292 distinct patent families (851 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 838 (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).

GeneTargetActionpAffinityCancer dependencyUniProt
BTKBruton tyrosine kinaseInhibition8.820.7%Q06187

Broader ChEMBL bioactivity targets: 5 (assay-derived). Sample: Tyrosine-protein kinase Blk, Cytoplasmic tyrosine-protein kinase BMX, Tyrosine-protein kinase Tec, Tyrosine-protein kinase TXK, Tyrosine-protein kinase BTK.

Bioactivity

ChEMBL activities: 9 potent at pChembl ≥ 5 of 9 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
TEC9.1IC500.8nMCHEMBL_ACT_24688756
BMX9IC501nMCHEMBL_ACT_24688757
TXK8.92IC501.2nMCHEMBL_ACT_24688755
BTK8.89IC501.3nMCHEMBL_ACT_22813278
BTK8.89IC501.3nMCHEMBL_ACT_24688562
BTK8.89IC501.3nMCHEMBL_ACT_24688754
BTK8.89IC501.3nMCHEMBL_ACT_29122429
BTK8.82IC501.5nMCHEMBL_ACT_17744394
BLK8.2IC506.3nMCHEMBL_ACT_24688758

Target pathways

Aggregated over 1 target gene(s): BTK.

Top Reactome pathways

45 total, by targets touching each:

PathwayTargetsGenes
ER-Phagosome pathway1BTK
Antigen processing-Cross presentation1BTK
Adaptive Immune System1BTK
Signal Transduction1BTK
Disease1BTK
Toll Like Receptor 4 (TLR4) Cascade1BTK
MyD88:MAL(TIRAP) cascade initiated on plasma membrane1BTK
Toll Like Receptor TLR1:TLR2 Cascade1BTK
Toll Like Receptor TLR6:TLR2 Cascade1BTK
Innate Immune System1BTK
Immune System1BTK
Toll-like Receptor Cascades1BTK
Toll Like Receptor 2 (TLR2) Cascade1BTK
Signaling by Rho GTPases1BTK
RHO GTPase Effectors1BTK
Fcgamma receptor (FCGR) dependent phagocytosis1BTK
Regulation of actin dynamics for phagocytic cup formation1BTK
DAP12 interactions1BTK
DAP12 signaling1BTK
Fc epsilon receptor (FCERI) signaling1BTK
FCERI mediated Ca+2 mobilization1BTK
Signaling by GPCR1BTK
GPCR downstream signalling1BTK
G-protein beta:gamma signalling1BTK
G alpha (q) signalling events1BTK
G alpha (12/13) signalling events1BTK
Diseases of Immune System1BTK
Diseases associated with the TLR signaling cascade1BTK
MyD88 deficiency (TLR2/4)1BTK
IRAK4 deficiency (TLR2/4)1BTK

Dominant GO biological processes

GO termTargets
neutrophil homeostasis1
positive regulation of type III hypersensitivity1
positive regulation of type I hypersensitivity1
adaptive immune response1
B cell affinity maturation1
histamine secretion by mast cell1
positive regulation of immunoglobulin production1
regulation of B cell cytokine production1
MyD88-dependent toll-like receptor signaling pathway1
regulation of B cell apoptotic process1
mesoderm development1
peptidyl-tyrosine phosphorylation1
calcium-mediated signaling1
proteoglycan catabolic process1
negative regulation of B cell proliferation1

Indications & clinical

Indications

10 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).

IndicationTrial phaseMONDOEFO
pemphigus vulgaris3MONDO:0008219EFO:0004719
autoimmune thrombocytopenic purpura3MONDO:0008558EFO:0007160
pemphigus3MONDO:0006594EFO:1000749
atopic eczema2MONDO:0004980EFO:0000274
autoimmune hemolytic anemia2MONDO:0020108EFO:1001264
asthma2MONDO:0004979MONDO:0004979
autoimmune disease1MONDO:0007179EFO:0005809

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

Clinical trials

Total trials: 18.

Phase distribution

PhaseTrials
PHASE28
PHASE37
PHASE13

Top trials by phase / activity

NCTPhaseStatusTitle
NCT04562766PHASE3ACTIVE_NOT_RECRUITINGStudy to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
NCT06975865PHASE3RECRUITINGThe Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
NCT07007962PHASE3RECRUITINGStudy to Evaluate the Efficacy and Safety of Oral Rilzabrutinib in Adults With Immune Thrombocytopenia (ITP) Who Failed First-line Treatment
NCT07086976PHASE3RECRUITINGA Study to Investigate the Efficacy, Safety, and Pharmacokinetics of Oral Rilzabrutinib Compared With Placebo in Participants 18 Years of Age and Older With Warm Autoimmune Hemolytic Anemia
NCT07190196PHASE3RECRUITINGA 52-week Study of Rilzabrutinib Efficacy and Safety Compared to Placebo in Adults Diagnosed With IgG4-related Disease
NCT07216079PHASE3ACTIVE_NOT_RECRUITINGRilzabrutinib for the Adult Participants With Chronic ITP Who Have Completed Phase 3 Study in Japan
NCT03762265PHASE3TERMINATEDA Study of PRN1008 in Patients With Pemphigus
NCT05002777PHASE2ACTIVE_NOT_RECRUITINGEfficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia (wAIHA)
NCT06500702PHASE2RECRUITINGA Study to Evaluate the Efficacy and Safety of Frexalimab, Brivekimig, or Rilzabrutinib in Participants Aged 16 to 75 Years With Primary Focal Segmental Glomerulosclerosis or Minimal Change Disease
NCT02704429PHASE2COMPLETEDA Study of PRN1008 in Adult Patients With Pemphigus Vulgaris
NCT03395210PHASE2COMPLETEDA Study of Rilzabrutinib in Adult Patients With Immune Thrombocytopenia (ITP)
NCT04520451PHASE2COMPLETEDOpen Label Two-Arm Study to Evaluate Rilzabrutinib in IgG4-Related Disease Participants
NCT05018806PHASE2COMPLETEDProof of Concept Study of Rilzabrutinib in Adult Patients With Moderate-to-severe Atopic Dermatitis
NCT05104892PHASE2COMPLETEDProof of Concept Study of Rilzabrutinib in Adult Participants With Moderate-to-severe Asthma
NCT05107115PHASE2COMPLETEDRilzabrutinib for the Treatment of Chronic Spontaneous Urticaria in Patients Who Remain Symptomatic Despite the Use of H1 Antihistamine
NCT04748926PHASE1COMPLETEDFood Effect and Relative Bioavailability Study of Rilzabrutinib in Healthy Participants
NCT06444191PHASE1COMPLETEDPharmacokinetics (PK) of Rilzabrutinib (PRN1008) in Healthy Japanese and Caucasian Subjects
NCT06444204PHASE1COMPLETEDA Study to Evaluate the Effect of Mild and Moderate Hepatic Impairment on the Single-Dose Pharmacokinetics of Rilzabrutinib (PRN1008)

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

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

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.

80 molecules share ≥1 primary target. Top 60 by shared-target count:

MoleculeSourceStatusShared targets
CRIZOTINIBChEMBL + PubChemPhase 4 (approved)BTK
RITLECITINIBChEMBL + PubChemPhase 4 (approved)BTK
ACALABRUTINIBChEMBLPhase 4 (approved)BTK
BOSUTINIBChEMBLPhase 4 (approved)BTK
BRIGATINIBChEMBLPhase 4 (approved)BTK
CERITINIBChEMBLPhase 4 (approved)BTK
DASATINIBChEMBLPhase 4 (approved)BTK
ENTRECTINIBChEMBLPhase 4 (approved)BTK
FEDRATINIBChEMBLPhase 4 (approved)BTK
FUTIBATINIBChEMBLPhase 4 (approved)BTK
IBRUTINIBChEMBLPhase 4 (approved)BTK
MITOXANTRONEChEMBLPhase 4 (approved)BTK
NERATINIBChEMBLPhase 4 (approved)BTK
NINTEDANIBChEMBLPhase 4 (approved)BTK
OLMUTINIBChEMBLPhase 4 (approved)BTK
OSIMERTINIBChEMBLPhase 4 (approved)BTK
PIRTOBRUTINIBChEMBLPhase 4 (approved)BTK
PONATINIBChEMBLPhase 4 (approved)BTK
SUNITINIBChEMBLPhase 4 (approved)BTK
TIRABRUTINIBChEMBLPhase 4 (approved)BTK
VANDETANIBChEMBLPhase 4 (approved)BTK
ZANUBRUTINIBChEMBLPhase 4 (approved)BTK
ABIVERTINIBChEMBLPhase 3BTK
ALISERTIBChEMBLPhase 3BTK
CANERTINIBChEMBLPhase 3BTK
CEDIRANIBChEMBLPhase 3BTK
DOVITINIBChEMBLPhase 3BTK
ENTOSPLETINIBChEMBLPhase 3BTK
EVOBRUTINIBChEMBLPhase 3BTK
FENEBRUTINIBChEMBLPhase 3BTK
LESTAURTINIBChEMBLPhase 3BTK
NEMTABRUTINIBChEMBLPhase 3BTK
ORELABRUTINIBChEMBLPhase 3BTK
POZIOTINIBChEMBLPhase 3BTK
PYROTINIBChEMBLPhase 3BTK
REMIBRUTINIBChEMBLPhase 3BTK
ROCILETINIBChEMBLPhase 3BTK
SARACATINIBChEMBLPhase 3BTK
TESEVATINIBChEMBLPhase 3BTK
TOLEBRUTINIBChEMBLPhase 3BTK
APITOLISIBChEMBLPhase 2BTK
AT-9283ChEMBLPhase 2BTK
ATUZABRUTINIBChEMBLPhase 2BTK
BIIB-091ChEMBLPhase 2BTK
BMS-754807ChEMBLPhase 2BTK
BMS-919373ChEMBLPhase 2BTK
BMS-986142ChEMBLPhase 2BTK
BRANEBRUTINIBChEMBLPhase 2BTK
CENISERTIBChEMBLPhase 2BTK
CEP-11981ChEMBLPhase 2BTK
DANUSERTIBChEMBLPhase 2BTK
DEFOSBARASERTIBChEMBLPhase 2BTK
EDRALBRUTINIBChEMBLPhase 2BTK
ELSUBRUTINIBChEMBLPhase 2BTK
FORETINIBChEMBLPhase 2BTK
ILORASERTIBChEMBLPhase 2BTK
MILREBRUTINIBChEMBLPhase 2BTK
PELITINIBChEMBLPhase 2BTK
POSELTINIBChEMBLPhase 2BTK
R-406ChEMBLPhase 2BTK

About this page

Generated deterministically by Sugi Atlas from primary biomedical databases via BioBTree. Every record on this page traces to a primary source.

Generated
Atlas version
v1.1.2
BioBTree
v2.0.2

Sources 30

This page pulls from 30 datasets indexed by BioBTree:

chebichembl_activitychembl_moleculechembl_targetcivic_evidenceclingen_dosageclinical_trialsdepmapefoensemblentrezgogoparentgtopdbgtopdb_interactiongtopdb_ligandhgncmeshmondomsigdbpatent_compoundpharmgkbpharmgkb_guidelinepubchempubchem_activityreactomereactomeparentrefseqtranscriptuniprot