ChemicalBook > CAS DataBase List > LMI070
LMI070
LMI070
- CAS No.1562338-42-4
- Chemical Name:LMI070
- CBNumber:CB93053903
- Molecular Formula:C22H27N5O2
- Formula Weight:393.48
- MOL File:1562338-42-4.mol
LMI070 Property
- Boiling point 660.0±55.0 °C(Predicted)
- Density 1.171±0.06 g/cm3(Predicted)
- storage temp. Store at -20°C
- solubility DMSO:3.0(Max Conc. mg/mL);7.62(Max Conc. mM)
- form A crystalline solid
- pka 7.84±0.40(Predicted)
- color White to yellow
- FDA UNII P12R69543A
- Symbol(GHS)
- Signal word
- Hazard statements
- Precautionary statements
LMI070 Chemical Properties,Usage,Production
- Uses Branaplam (LMI070; NVS-SM1) is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC50 of 20 nM for SMN. Branaplam inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model[1][2].
-
in vivo
Branaplam (LMI070; NVS-SM1; 3, 10, 30 mg/kg; oral) produces dose-dependent elevations of SMN2-FL transcript and SMN protein in brain and spinal cord[1].
? Branaplam (1 mg/kg of IV; 3 mg/kg of PO) has a CL of 25 mL/min/kg and an AUC of 3.03 μM?h[2].
? A single Branaplam (oral; 30 mg/kg) results in significant and durable SMN protein elevation in brain for up to 160 hours in C/+ mice[1].
? Branaplam (oral; 0.03, 0.1, 0.3, 1, 3 mg/kg) improves body weight and extendes lifespan in n SMNΔ7 mice[1].Animal Model: C/+ SMA mouse model[1] Dosage: 3, 10, 30 mg/kg Administration: Oral Result: Produced dose-dependent elevations of SMN2-FL transcript and SMN protein in brain and spinal cord. Animal Model: Male Sprague-Dawley rat[2] Dosage: 1 mg/kg (IV);3 mg/kg (PO) (Pharmacokinetic Analysis) Administration: IV or PO Result: Had a CL of 25 mL/min/kg and an AUC of 3.03 μM?h. -
References
[1] Palacino J, et al. SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice. Nat Chem Biol. 2015 Jul;11(7):511-517. DOI:10.1038/nchembio.1837
[2] Cheung AK, et al. Discovery of Small Molecule Splicing Modulators of Survival Motor Neuron-2 (SMN2) for the Treatment of Spinal Muscular Atrophy (SMA). J Med Chem. 2018 Dec 27;61(24):11021-11036. DOI:10.1021/acs.jmedchem.8b01291
LMI070 Preparation Products And Raw materials
Raw materials
Preparation Products
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1562338-42-4, LMI070Related Search:
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- 化合物LMI 070
- 1562338-42-4
- Branaplam, 10 mM in DMSO
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- Branaplam
- Branaplam (LMI070)
- Phenol, 5-(1H-pyrazol-4-yl)-2-[6-[(2,2,6,6-tetramethyl-4-piperidinyl)oxy]-3-pyridazinyl]-
- LMI 070;LMI-070;LMI070
- 5-(1H-pyrazol-4-yl)-2-(6-((2,2,6,6-tetramethylpiperidin-4-yl)oxy)pyridazin-3-yl)phenol
- LMI070 (NVS-SM1)
- LMI070 (Branaplam)
- NVS-SM1; LMI-070;
- NVS-SM1, Branaplam
- NVS-SM1
- LMI070