Network Version
Branaplam Data Sheet
| Product Name: | Branaplam |
 |
|---|---|---|
| CAS No.: | 1562338-42-4 | |
| Cat. No.: | HY-19620 | |
| MWt: | 393.48 | |
| Formula: | C22H27N5O2 | |
| Purity : | >98% | |
| Solubility: | DMSO : 5 mg/mL (12.71 mM; ultrasonic and warming and heat to 80°C) | |
| Mechanisms: | Target: Cancer | |
| Biological Activity: | ||
|
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].
IC50 & Target: IC50: 20 nM (SMN)[1] EC50: 6.3 μM (hERG)[2] In Vitro: Branaplam (LMI070; NVS-SM1) 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) 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]. |
||
1 Deer Park Dr, Suite F, Monmouth Junction, NJ 08852, USA
Tel: 609-228-6898 Fax: 609-228-5909 Email: [email protected] Web:www.medchemexpress.com