- Branaplam
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- $42.00 / 1mg
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2025-09-30
- CAS:1562338-42-4
- Min. Order:
- Purity: 98.93%
- Supply Ability: 10g
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| Product Name: | LMI070 | | Synonyms: | NVS-SM1, Branaplam;LMI070;NVS-SM1;NVS-SM1; LMI-070;;LMI070 (Branaplam);Branaplam;LMI070 (NVS-SM1);5-(1H-pyrazol-4-yl)-2-(6-((2,2,6,6-tetramethylpiperidin-4-yl)oxy)pyridazin-3-yl)phenol | | CAS: | 1562338-42-4 | | MF: | C22H27N5O2 | | MW: | 393.48 | | EINECS: | | | Product Categories: | | | Mol File: | 1562338-42-4.mol |  |
| | LMI070 Chemical Properties |
| 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 | | InChI | InChI=1S/C22H27N5O2/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) | | InChIKey | STWTUEAWRAIWJG-UHFFFAOYSA-N | | SMILES | C1(O)=CC(C2=CNN=C2)=CC=C1C1=NN=C(OC2CC(C)(C)NC(C)(C)C2)C=C1 |
| | LMI070 Usage And Synthesis |
| 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].
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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].
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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].
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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.
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| 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.
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| | 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 | 1-Boc-pyrazole-4-boronic acid pinacol ester-->5-chloro-2-(4,4,5,5-tetramethyl-1,3,2-dioxaborolan-2-yl)phenol-->2-Bromo-5-chlorophenol-->3-chloro-6-((2,2,6,6-tetramethylpiperidin-4-yl)oxy)pyridazine-->2,2,6,6-Tetramethyl-4-piperidinol |
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