Branaplam

• Description :

  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].

• CAS Number :

  [1562338-42-4]

• Product Name Alternative :

  LMI070; NVS-SM1

• UNSPSC :

  12352005

• Hazard Statement :

  H302, H312, H332

• Target :

  DNA/RNA Synthesis; Potassium Channel

• Type :

  Reference compound

• Related Pathways :

  Cell Cycle/DNA Damage; Membrane Transporter/Ion Channel

• Applications :

  Cancer-programmed cell death

• Field of Research :

  Cancer

• Assay Protocol :

  https://www.medchemexpress.com/LMI070.html

• Purity :

  99.78

• Solubility :

  DMSO : 3.33 mg/mL (ultrasonic; warming; heat to 60°C)

• Smiles :

  OC1=CC(C2=CNN=C2)=CC=C1C3=CC=C(OC4CC(C)(C)NC(C)(C)C4)N=N3

• Molecular Formula :

  C22H27N5O2

• Molecular Weight :

  393.48

• Precautions :

  H302, H312, H332

• References & Citations :

  [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.|[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.

• Shipping Conditions :

  Room Temperature

• Storage Conditions :

  4°C (Powder, sealed storage, away from moisture)

• Scientific Category :

  Reference compound1

• Clinical Information :

  Phase 3