ASCIMINIB HYDROCHLORIDE

Details

Stereochemistry	ABSOLUTE
Molecular Formula	C20H18ClF2N5O3.ClH
Molecular Weight	486.299
Optical Activity	UNSPECIFIED
Defined Stereocenters	1 / 1
E/Z Centers	0
Charge	0

SHOW SMILES / InChI

Structure Search

SMILES

Cl.O[C@@H]1CCN(C1)C2=C(C=C(C=N2)C(=O)NC3=CC=C(OC(F)(F)Cl)C=C3)C4=CC=NN4

InChI

InChIKey=HGCOOPLEWPBLOY-PFEQFJNWSA-N

InChI=1S/C20H18ClF2N5O3.ClH/c21-20(22,23)31-15-3-1-13(2-4-15)26-19(30)12-9-16(17-5-7-25-27-17)18(24-10-12)28-8-6-14(29)11-28;/h1-5,7,9-10,14,29H,6,8,11H2,(H,25,27)(H,26,30);1H/t14-;/m1./s1

HIDE SMILES / InChI

Molecular Formula	C20H18ClF2N5O3
Molecular Weight	449.838
Charge	0
Count

Stereochemistry	ABSOLUTE
Additional Stereochemistry	No
Defined Stereocenters	1 / 1
E/Z Centers	0
Optical Activity	UNSPECIFIED

Molecular Formula	ClH
Molecular Weight	36.461
Charge	0
Count

Stereochemistry	ACHIRAL
Additional Stereochemistry	No
Defined Stereocenters	0 / 0
E/Z Centers	0
Optical Activity	NONE

Description
Sources: https://www.ncbi.nlm.nih.gov/pubmed/28329763

ABL-001 (asciminib), a potent and selective allosteric tyrosine-protein kinase ABL1 inhibitor that is undergoing clinical development testing in patients with Chronic myeloid leukemia (CML) and Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia. is a tyrosine-protein kinase ABL1 inhibitor. In contrast to catalytic-site ABL1 kinase inhibitors, ABL001 binds to the myristoyl pocket of ABL1 and induces the formation of an inactive kinase conformation. ABL001 and second-generation catalytic inhibitors have similar cellular potencies but distinct patterns of resistance mutations, with genetic barcoding studies revealing pre-existing clonal populations with no shared resistance between ABL001 and the catalytic inhibitor nilotinib. ABL001 was tested on mice with a particularly aggressive type of CML. The combination of ABL001 and nilotinib led to complete disease control and eradicated CML xenograft tumors without recurrence after the cessation of treatment. ABL001 is being tested in clinical trials for treatment of CML and Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia alone and in combination with niotinib, imatinib or dasatinib.

Originator

Approval Year

Targets

Primary Target	Pharmacology	Condition	Potency
Tyrosine-protein kinase ABL1 7 Target ID: P00519 Gene ID: 25.0 Gene Symbol: ABL1 Target Organism: Homo sapiens (Human) Sources: https://www.ncbi.nlm.nih.gov/pubmed/28329763	Inhibitor 8504		1.3 nM [IC50]

Conditions

Condition	Modality	Targets	Highest Phase	Product
Chronic myelogenous leukemia 21 Sources: https://clinicaltrials.gov/ct2/show/record/NCT03106779	Primary		Phase III 665	Unknown Approved Use Unknown
Philadelphia chromosome positive acute lymphoblastic leukemia 7 Sources: https://clinicaltrials.gov/show/NCT02081378	Primary		Phase I 438	Unknown Approved Use Unknown

C_max

Value	Dose	Co-administered	Analyte	Population
679 ng/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/35616006/	40 mg single, oral dose: 40 mg route of administration: Oral experiment type: SINGLE co-administered:		ASCIMINIB plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: UNKNOWN
1781 ng/mL DRUG LABEL https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/215358s000Orig2lbl.pdf	80 mg single, oral dose: 80 mg route of administration: Oral experiment type: SINGLE co-administered:		ASCIMINIB HYDROCHLORIDE plasma	Homo sapiens population: UNKNOWN age: UNKNOWN sex: UNKNOWN food status: UNKNOWN

AUC

Value	Dose	Co-administered	Analyte	Population
7000 ng × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/35616006/	40 mg single, oral dose: 40 mg route of administration: Oral experiment type: SINGLE co-administered:		ASCIMINIB plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: UNKNOWN
15112 ng × h/mL DRUG LABEL https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/215358s000Orig2lbl.pdf	80 mg single, oral dose: 80 mg route of administration: Oral experiment type: SINGLE co-administered:		ASCIMINIB HYDROCHLORIDE plasma	Homo sapiens population: UNKNOWN age: UNKNOWN sex: UNKNOWN food status: UNKNOWN

T_1/2

Value	Dose	Co-administered	Analyte	Population
12.6 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/35616006/	40 mg single, oral dose: 40 mg route of administration: Oral experiment type: SINGLE co-administered:		ASCIMINIB plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: UNKNOWN

F_unbound

Value	Dose	Co-administered	Analyte	Population
3% DRUG LABEL https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/215358s000Orig2lbl.pdf	80 mg single, oral dose: 80 mg route of administration: Oral experiment type: SINGLE co-administered:		ASCIMINIB HYDROCHLORIDE plasma	Homo sapiens population: UNKNOWN age: UNKNOWN sex: UNKNOWN food status: UNKNOWN

Sourcing

Vendor/Aggregator	ID	URL
1PlusChem LLC	1P001LZU	https://www.1pchem.com/search?query=1P001LZU
BLD Pharm	BD764501	http://www.bldpharm.com/search/Search.html?keyword=BD764501
Chem Scene	CS-7655	http://www.chemscene.com/goproductList/searchProductList?productObj=CS-7655
eMolecules	194971991	https://orderbb.emolecules.com/search/#?query=194971991
eMolecules	295311369	https://orderbb.emolecules.com/search/#?query=295311369
eMolecules	303899010	https://orderbb.emolecules.com/search/#?query=303899010
eNovation Chemicals	D621061	https://www.enovationchem.com/Products.asp?SEARCHTEXT=D621061&searchbtn.x=0&searchbtn.y=0
mcule	MCULE-6613972351	https://mcule.com/MCULE-6613972351/
MedChem Express	HY-104010	https://www.medchemexpress.com/search.html?q=HY-104010&ft=&fa=&fp=
Molnova	M12066	https://www.molnova.com/en/serch-list.html?keywords=M12066
MolPort	MolPort-046-416-854	https://www.molport.com/shop/molecule-link/MolPort-046-416-854
MolPort	MolPort-046-423-263	https://www.molport.com/shop/molecule-link/MolPort-046-423-263
MuseChem	I002918	https://www.musechem.com/product/I002918.html
Selleck Chemicals	S8555	http://www.selleckchem.com/search.html?searchDTO.searchParam=S8555

PubMed

Title	Date	PubMed
Identification and characterization of activating ABL1 1b kinase mutations: impact on sensitivity to ATP-competitive and allosteric ABL1 inhibitors.	2017-05	27890928
The allosteric inhibitor ABL001 enables dual targeting of BCR-ABL1.	2017-03-30	28329763
Second line small molecule therapy options for treating chronic myeloid leukemia.	2017-01	27901368

Patents

Sample Use Guides

In Vivo Use Guide

40 mg ABL001 twice a day administered in adult patients with Chronic Myelogenous Leukemia

Route of Administration: Oral

In Vitro Use Guide

BL001 potently inhibited the proliferation of Ba/F3 cells expressing native BCR-ABL1 or a variety of BCR-ABL1 point mutations (IC50 range: 1.3-113.5 nM), with no observed toxicity to parental Ba/F3 cells up to 10 uM. Interestingly, however, ABL001 demonstrated little to no activity against a small panel of BCR-ABL1 compound mutations tested (G250E/T315I, E255K/T315I, E255V/T315I).

Substance Class	Chemical Created by `admin` on `Wed Apr 02 00:59:51 GMT 2025` Edited by `admin` on `Wed Apr 02 00:59:51 GMT 2025`
Record UNII	C5U34S9XFV
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

ASCIMINIB HYDROCHLORIDE

Official Name

English

SCEMBLIX

Preferred Name

English

ASCIMINIB HYDROCHLORIDE [USAN]

Common Name

English

ASCIMINIB HYDROCHLORIDE [JAN]

Common Name

English

ABL001-AAA

Code

English

Asciminib hydrochloride [WHO-DD]

Common Name

English

ASCIMINIB HYDROCHLORIDE [ORANGE BOOK]

Common Name

English

Code System Code Type Description

SMS_ID

300000011169