Branaplam hydrochloride

Modify Date: 2024-01-02 18:28:59

Branaplam hydrochloride structure	Common Name	Branaplam hydrochloride
	CAS Number	1562338-39-9	Molecular Weight	429.94
	Density	N/A	Boiling Point	N/A
	Molecular Formula	C₂₂H₂₈ClN₅O₂	Melting Point	N/A
	MSDS	N/A	Flash Point	N/A

Use of 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].

Name	Branaplam hydrochloride

Description	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].
Related Catalog	Research Areas >> Cancer Signaling Pathways >> Cell Cycle/DNA Damage >> DNA/RNA Synthesis Signaling Pathways >> Membrane Transporter/Ion Channel >> Potassium Channel
In Vitro	Branaplam (LMI070; NVS-SM1) hydrochloride treatment induces changes in the levels of 175 genes in human fibroblasts[1].
In Vivo	Branaplam (LMI070; NVS-SM1; 3, 10, 30 mg/kg; oral) hydrochloride 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) hydrochloride has a CL of 25 mL/min/kg and an AUC of 3.03 μM•h[2]. A single Branaplam (oral; 30 mg/kg) hydrochloride 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) hydrochloride improves body weight and extendes lifespan in n SMNΔ7 mice[1].
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. [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.

Molecular Formula	C₂₂H₂₈ClN₅O₂
Molecular Weight	429.94

Branaplam hydrochloride

Use of Branaplam hydrochloride

Names

Branaplam hydrochloride Biological Activity

Chemical & Physical Properties