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LMI070

LMI070 Structure
LMI070
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
Hazard and Precautionary Statements (GHS)
  • 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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LMI070 Spectrum
1562338-42-4, LMI070Related Search:
  • 化学试剂
  • 抑制剂
  • 化工试剂
  • 药靶配体
  • 标准品
  • 化合物LMI 070,10 MM DMSO 溶液
  • 布拉扑兰(LMI070)
  • 5-(1H-吡唑-4-基)-2-(6-((2,2,6,6-四甲基哌啶-4-基)氧基)吡啶嗪-3-基)苯酚
  • 布拉扑兰
  • 5-(1H-吡唑-4-基)-2-(6-((2,2,6,6-四甲基哌啶-4-基)氧基)哒嗪-3-基)苯酚
  • 化合物LMI 070
  • 1562338-42-4
  • Branaplam, 10 mM in DMSO
  • SMN2,SMA,inhibit,SMN,DNA/RNA Synthesis,Inhibitor,hERG,atrophy,Potassium Channel,neuron-2,motor,KcsA,gene,orally,LMI070,splicing,LMI-070,spinal,survival,Branaplam,muscular
  • 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