Branaplam (hydrochloride)

Branaplam (LMI070; NVS-SM1) hydrochloride is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC50 of 20 nM for SMN. Branaplam hydrochloride inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam hydrochloride elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model[1][2].

Product Specifications

CAS Number

[1562338-39-9]

Product Name Alternative

LMI070 (hydrochloride) ; NVS-SM1 (hydrochloride)

UNSPSC

12352005

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/branaplam-hydrochloride.html

Concentration

10mM

Purity

99.43

Solubility

DMSO : 250 mg/mL (ultrasonic) |H2O : 200 mg/mL (ultrasonic)

Smiles

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

Molecular Formula

C22H28ClN5O2

Molecular Weight

429.94

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.