Branaplam (LMI 070) is a highly potent, selective and orally active small molecule SMN2 splicing modulator.

SMN:20 nM (IC50), ERG (human):6.3 μM (EC50)

To evaluate the efficacy of NVS-SM1, we used the SMNΔ7 mouse model, which displays a severe phenotype. In the specific colony used for this study, death typically occurs before postnatal day 15. We were pleased to observe that, in addition to a dose-dependent elevation of SMN protein in the brain, oral administration of NVS-SM1 improved body weight and extended lifespan, with 50% of the animals in the 1 mg per kg body weight group and 62% of animals in the 3 mg per kg body weight group showing increased survival.