BRANAPLAM HYDROCHLORIDE

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 Search

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

Description
Sources: https://www.treatsma.uk/treatments/branaplam/ | https://adisinsight.springer.com/drugs/800041467 | https://www.ncbi.nlm.nih.gov/pubmed/30407821

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.

Originator

Approval Year

Targets

Primary Target	Pharmacology	Condition	Potency
Survival motor neuron protein 2 Target ID: CHEMBL1293232 Sources: https://www.ncbi.nlm.nih.gov/pubmed/26030728 \| https://smanewstoday.com/lmi070-novartis \| http://www.sma-europe.eu/clinical-trials/modulation-of-the-smn2-back-up-gene-small-molecules/novartis-small-molecule-branaplam-lmi070/ \| http://www.ema.europa.eu/docs/en_GB/document_library/Orphan_designation/2018/05/WC500249083.pdf	Modulator 828

PubMed

Title	Date	PubMed
Small Molecules in Development for the Treatment of Spinal Muscular Atrophy.	2016 Nov 23	27490705
Discovery of Small Molecule Splicing Modulators of Survival Motor Neuron-2 (SMN2) for the Treatment of Spinal Muscular Atrophy (SMA).	2018 Dec 27	30407821
Protocol for a phase II, monocentre, double-blind, placebo-controlled, cross-over trial to assess efficacy of pyridostigmine in patients with spinal muscular atrophy types 2-4 (SPACE trial).	2018 Jul 30	30061431

Substance Class	Chemical Created by `admin` on `Sat Dec 16 11:21:41 GMT 2023` Edited by `admin` on `Sat Dec 16 11:21:41 GMT 2023`
Record UNII	317C6VX21O
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

BRANAPLAM HYDROCHLORIDE

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