Olutasidenib

Summary

Olutasidenib (CHEMBL4297610) is an approved small molecule (ATC L01XM03) targeting IDH1; indicated across 2 conditions including acute myeloid leukemia and myelodysplastic syndrome.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: L01XM03
• Targets: 1 (IDH1)
• Indications: 2 conditions
• Clinical trials: 17
• Chemistry: 354.8 Da · C18H15ClN4O2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297610
Name	Olutasidenib
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	118955396
ATC	L01XM03
Molecular formula	C18H15ClN4O2
Molecular weight	354.8
InChIKey	NEQYWYXGTJDAKR-JTQLQIEISA-N

SMILES: C[C@@H](C1=CC2=C(C=CC(=C2)Cl)NC1=O)NC3=CC=C(N(C3=O)C)C#N

IUPAC name: 5-[[(1S)-1-(6-chloro-2-oxo-1H-quinolin-3-yl)ethyl]amino]-1-methyl-6-oxopyridine-2-carbonitrile

Also known as: Ft-2102, FT-2102, Olutasidenib, Rezlidhia, OLUTASIDENIB

Patent coverage: 117 distinct patent families (235 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 170 (72%) 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
IDH1	isocitrate dehydrogenase (NADP(+)) 1	Inhibition	7	0.7%	O75874

Broader ChEMBL bioactivity targets: 2 (assay-derived). Sample: Isocitrate dehydrogenase [NADP] cytoplasmic, Isocitrate dehydrogenase [NADP], mitochondrial.

Bioactivity

ChEMBL activities: 23 potent at pChembl ≥ 5 of 25 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
IDH1	9	IC50	1	nM	CHEMBL_ACT_28943021
IDH1	8.29	IC50	5.1	nM	CHEMBL_ACT_28943024
IDH1	8.22	IC50	6	nM	CHEMBL_ACT_18931147
IDH1	8.05	IC50	9	nM	CHEMBL_ACT_18931144
IDH1	8.05	IC50	9	nM	CHEMBL_ACT_18931148
IDH1	7.89	IC50	12.9	nM	CHEMBL_ACT_28937843
IDH1	7.84	IC50	14.3	nM	CHEMBL_ACT_28937837
IDH1	7.68	IC50	21	nM	CHEMBL_ACT_18931130
IDH1	7.68	IC50	21	nM	CHEMBL_ACT_26123574
IDH1	7.67	IC50	21.2	nM	CHEMBL_ACT_18931081
IDH1	7.67	IC50	21.2	nM	CHEMBL_ACT_28678484
IDH1	7.67	IC50	21.2	nM	CHEMBL_ACT_28735304
IDH1	7.67	IC50	21.2	nM	CHEMBL_ACT_28943087
IDH1	7.41	IC50	39	nM	CHEMBL_ACT_18931145
IDH1	7.38	IC50	42	nM	CHEMBL_ACT_18931146
IDH1	7.17	IC50	67.1	nM	CHEMBL_ACT_28937846
IDH1	7.11	IC50	77	nM	CHEMBL_ACT_28937840
IDH1	7.03	IC50	94	nM	CHEMBL_ACT_18931137
IDH1	6.94	IC50	114	nM	CHEMBL_ACT_18931124
IDH1	6.94	IC50	114	nM	CHEMBL_ACT_28943090
IDH1	6.04	IC50	922	nM	CHEMBL_ACT_28943027
IDH1	5.28	IC50	5220	nM	CHEMBL_ACT_28678487
IDH1	5.28	IC50	5220	nM	CHEMBL_ACT_28735307

Target pathways

Aggregated over 1 target gene(s): IDH1.

Top Reactome pathways

5 total, by targets touching each:

Pathway	Targets	Genes
Abnormal conversion of 2-oxoglutarate to 2-hydroxyglutarate	1	IDH1
NADPH regeneration	1	IDH1
Neutrophil degranulation	1	IDH1
Peroxisomal protein import	1	IDH1
NFE2L2 regulating TCA cycle genes	1	IDH1

Dominant GO biological processes

GO term	Targets
glyoxylate cycle	1
tricarboxylic acid cycle	1
isocitrate metabolic process	1
2-oxoglutarate metabolic process	1
NADP+ metabolic process	1
NADPH regeneration	1
glutathione metabolic process	1
response to oxidative stress	1
female gonad development	1
response to steroid hormone	1
regulation of phospholipid catabolic process	1
regulation of phospholipid biosynthetic process	1

Indications & clinical

Indications

2 indications (1 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
acute myeloid leukemia	4	MONDO:0018874	EFO:0000222
myelodysplastic syndrome	1	MONDO:0018881	EFO:0000198

Clinical trials

Total trials: 17.

Phase distribution

Phase	Trials
PHASE2	11
PHASE1/PHASE2	3
PHASE1	2
PHASE4	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07486713	PHASE4	RECRUITING	Olutasidenib DDI Study in Patients With IDH1 Mutation Positive Malignancies
NCT05564390	PHASE2	RECRUITING	MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)
NCT06161974	PHASE2	RECRUITING	Study of Olutasidenib and Temozolomide in HGG
NCT06445959	PHASE1/PHASE2	RECRUITING	Phase 1b/2 Study of Decitabine and Venetoclax in Combination With the Targeted Mutant IDH1 Inhibitor Olutasidenib
NCT06566742	PHASE2	RECRUITING	A Phase 2 Study Evaluating Olutasidenib in Patients With IDH1-mutated Clonal Cytopenia of Undetermined Significance and Lower-risk Myelodysplastic/Syndromes/Chronic Myelomonocytic Leukemia.
NCT06668584	PHASE2	RECRUITING	A Phase II Open-label Study of Olutasidenib Post-transplant Maintenance Therapy for Patients With IDH1-mutated Myeloid Malignancies
NCT06782542	PHASE2	RECRUITING	Olutasidenib, Venetoclax, and Azacitidine in IDH1 Mutated Newly Diagnosed Acute Myeloid Leukemia Patients Eligible for Intensive Induction Chemotherapy
NCT07032727	PHASE2	RECRUITING	Olutasidenib Combined With Co-targeted Therapy in Relapsed or Refractory IDH1-mutated Myeloid Malignancies Harboring Activated Signaling Pathway Mutations
NCT07153497	PHASE2	NOT_YET_RECRUITING	Testing the Use of an IDH1 Inhibitor, Olutasidenib, in Acute Myeloid Leukemia Added to ASTX727 and Venetoclax; in High-Risk MDS Added to ASTX727; and Alone in Low Risk MDS (A MyeloMATCH Treatment Substudy)
NCT07304011	PHASE2	RECRUITING	Olutasidenib With Azacitidine Followed by Olutasidenib Maintenance for the Treatment of IDH1-mutated Acute Myeloid Leukemia in Patients With Prior Treatment With Venetoclax Plus a Hypomethylating Agent
NCT07471841	PHASE2	NOT_YET_RECRUITING	Olutasidenib in Relapsed IDH1 Mutated AML Patients Who Have Previously Received Venetoclax
NCT07604064	PHASE2	RECRUITING	A Clinical Trial of Olutasidenib in Patients With Acute Myeloid Leukemia
NCT02719574	PHASE1/PHASE2	COMPLETED	Open-label Study of FT-2102 With or Without Azacitidine or Cytarabine in Patients With AML or MDS With an IDH1 Mutation
NCT03684811	PHASE1/PHASE2	COMPLETED	A Study of FT-2102 in Patients With Advanced Solid Tumors and Gliomas With an IDH1 Mutation
NCT06597734	PHASE2	WITHDRAWN	A Phase 2 Study Evaluating Olutasidenib in Combination With Hypomethylating Agents in Patients With IDH1-mutated Higher-risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Advanced Myeloproliferative Neoplasm
NCT06543381	PHASE1	RECRUITING	Olutasidenib for the Treatment of Patients With IDH1 Mutated AML, MDS or CMML After Donor Hematopoietic Cell Transplant
NCT07411586	PHASE1	NOT_YET_RECRUITING	Phase 1/1b Trial Of Olutasidenib And Ziftomenib For NPM1 And IDH1 Co-Mutated Acute Myeloid Leukemia

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.

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.

8 molecules share ≥1 primary target. Top 8 by shared-target count:

Molecule	Source	Status	Shared targets
VORASIDENIB	ChEMBL + PubChem	Phase 4 (approved)	IDH1
ENASIDENIB	ChEMBL	Phase 4 (approved)	IDH1
IVOSIDENIB	ChEMBL	Phase 4 (approved)	IDH1
CRELOSIDENIB	ChEMBL	Phase 2	IDH1
DS-1001B	ChEMBL	Phase 2	IDH1
IDH305	ChEMBL	Phase 2	IDH1
SAFUSIDENIB	ChEMBL	Phase 2	IDH1
ZUCLOMIPHENE	ChEMBL	Phase 2	IDH1