Ezutromid is a novel Small utrophin's translation modulator with EC50 of 0.4 uM .In vitro: 1) SMT C1100 induces increased levels of utrophin RNA in human muscle cells.2) Treatment of human DMD cells with SMT C1100 lead to a 2-fold increase in utrophin protein levels at an optimal concentration of 0.3 uM after 3 days of treatment. 3) SMT C1100 wassafe and well tolerated with plasma concentrations achieved sufcient to cause a 50% increase in concentrations of utrophin in cells. 4) SMT C1100 led to a 30% increase in Utrn mRNA level and resulted in a 2.0-fold increase in UTRN protein level .In vivo: 1)The reference for administration is 50 mg/kg. 2) it was considered that no toxicity was determined for SMT C1100 administered by oral gavage to the mouse up to dose levels of 2000 mg/kg/day for 28 days.Note: You can dissolve the products in phosphate buffered saline (PBS), 0.1% Tween-20, 5% DMSO)
References:
[1]. Tinsley JM et al. Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse. PLoS One. 2011 May 6;6(5):e19189.
[2]. Tinsley J et al. Safety, tolerability, and pharmacokinetics of SMT C1100, a 2-arylbenzoxazole utrophin modulator, following single- and multiple-dose administration to healthy male adult volunteers. J Clin Pharmacol. 2015 Jun;55(6):698-707.
[3]. Guiraud S et al. Second-generation compound for the modulation of utrophin in the therapy of DMD. Hum Mol Genet. 2015 Aug 1;24(15):4212-24.