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Details

Stereochemistry ACHIRAL
Molecular Formula C22H27N5O2.ClH
Molecular Weight 429.943
Optical Activity NONE
Defined Stereocenters 0 / 0
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of BRANAPLAM HYDROCHLORIDE

SMILES

Cl.CC1(C)CC(CC(C)(C)N1)OC2=CC=C(N=N2)C3=CC=C(C=C3O)C4=CNN=C4

InChI

InChIKey=XJIMIVJABPKGIY-UHFFFAOYSA-N
InChI=1S/C22H27N5O2.ClH/c1-21(2)10-16(11-22(3,4)27-21)29-20-8-7-18(25-26-20)17-6-5-14(9-19(17)28)15-12-23-24-13-15;/h5-9,12-13,16,27-28H,10-11H2,1-4H3,(H,23,24);1H

HIDE SMILES / InChI

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

Molecular Formula C22H27N5O2
Molecular Weight 393.4821
Charge 0
Count
Stereochemistry ACHIRAL
Additional Stereochemistry No
Defined Stereocenters 0 / 0
E/Z Centers 0
Optical Activity NONE

LMI-070 is an experimental compound being developed by Novartis Pharma as a treatment for spinal muscular atrophy (SMA). It is a small-molecule drug which modifies alternative splicing of the SMN2 gene, bringing about increased levels of SMN protein. LMI-070 originated from a high-throughput phenotypic screening hit, pyridazine 2, and evolved via multiparameter lead optimization. In a severe mouse SMA model, LMI-070 treatment increased full-length SMN RNA and protein levels, and extended survival. LMI-070 is taken orally, usually in a liquid form once a week. It is in phase II clinical trial.

Approval Year

PubMed

PubMed

TitleDatePubMed
Small Molecules in Development for the Treatment of Spinal Muscular Atrophy.
2016 Nov 23
Substance Class Chemical
Created
by admin
on Sat Dec 16 11:21:41 UTC 2023
Edited
by admin
on Sat Dec 16 11:21:41 UTC 2023
Record UNII
317C6VX21O
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
BRANAPLAM HYDROCHLORIDE
USAN  
Official Name English
BRANAPLAM HYDROCHLORIDE [USAN]
Common Name English
LMI-070 HYDROCHLORIDE
Code English
LMI070 HYDROCHLORIDE
Code English
5-(1H-PYRAZOL-4-YL)-2-(6-((2,2,6,6-TETRAMETHYLPIPERIDIN-4-YL)OXY)PYRIDAZIN-3-YL)PHENOL HYDROCHLORIDE
Systematic Name English
NVP-LMI070-AAA
Code English
PHENOL, 5-(1H-PYRAZOL-4-YL)-2-(6-((2,2,6,6-TETRAMETHYL-4-PIPERIDINYL)OXY)-3-PYRIDAZINYL)-, HYDROCHLORIDE (1:1)
Systematic Name English
Code System Code Type Description
CAS
1562334-51-3
Created by admin on Sat Dec 16 11:21:41 UTC 2023 , Edited by admin on Sat Dec 16 11:21:41 UTC 2023
NON-SPECIFIC STOICHIOMETRY
CAS
1562338-39-9
Created by admin on Sat Dec 16 11:21:41 UTC 2023 , Edited by admin on Sat Dec 16 11:21:41 UTC 2023
PRIMARY
PUBCHEM
135565041
Created by admin on Sat Dec 16 11:21:41 UTC 2023 , Edited by admin on Sat Dec 16 11:21:41 UTC 2023
PRIMARY
SMS_ID
300000044778
Created by admin on Sat Dec 16 11:21:41 UTC 2023 , Edited by admin on Sat Dec 16 11:21:41 UTC 2023
PRIMARY
NCI_THESAURUS
C188504
Created by admin on Sat Dec 16 11:21:41 UTC 2023 , Edited by admin on Sat Dec 16 11:21:41 UTC 2023
PRIMARY
FDA UNII
317C6VX21O
Created by admin on Sat Dec 16 11:21:41 UTC 2023 , Edited by admin on Sat Dec 16 11:21:41 UTC 2023
PRIMARY
USAN
JK-245
Created by admin on Sat Dec 16 11:21:41 UTC 2023 , Edited by admin on Sat Dec 16 11:21:41 UTC 2023
PRIMARY
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ACTIVE MOIETY