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SMT C1100 is a small molecule utrophin modulator in development to treat Duchenne muscular dystrophy. This study evaluated the safety, tolerability, and pharmacokinetics of SMT C1100 in healthy volunteers. This double-blind, placebo-controlled Phase 1 study comprised: Part 1, an escalating, single-dose with/without fasting involving 50 mg/kg, 100 mg/kg, 200 mg/kg, and 400 mg/kg doses; and Part 2, a multiple 10 day dose evaluation involving 100 mg/kg bid and 200 mg/kg bid doses. Adverse events were recorded. SMT C1100 was absorbed rapidly following single and multiple oral doses, with median tmax attained within 2-3.5 hour across all doses. Considerable variability of pharmacokinetic parameters was noted among subjects. Following single doses, systemic exposure increased in a sub-proportional manner, with the 8.0-fold dose increment resulting in 2.7- and 2.4-fold increases in AUC0-∞ and Cmax , respectively. AUC0-∞ and Cmax were estimated as 4.2- and 4.8-fold greater, respectively, following food. Systemic exposure reduced upon repeat dosing with steady-state concentrations achieved within 3-5 days of multiple bid dosing. No serious or severe adverse events were reported. SMT C1100 was safe and well tolerated with plasma concentrations achieved sufficient to cause a 50% increase in concentrations of utrophin in cells in vitro.

Original publication




Journal article


J Clin Pharmacol

Publication Date





698 - 707


Duchene muscular dystrophy, Phase 1, Utrophin, disease modifying therapy, pharmacokinetics, Administration, Oral, Adolescent, Adult, Area Under Curve, Arginine, Benzothiazoles, Benzoxazoles, Dose-Response Relationship, Drug, Double-Blind Method, Drug Administration Schedule, Half-Life, Healthy Volunteers, Humans, Male, Middle Aged, Muscular Dystrophy, Duchenne, No-Observed-Adverse-Effect Level, Utrophin, Young Adult