BioCentury
ARTICLE | Clinical News

SMT C1100: Preliminary Phase I data

October 15, 2012 7:00 AM UTC

Top-line data from a double-blind, placebo-controlled, dose-escalation Phase I trial in 48 healthy volunteers showed that all doses of a nanoparticle aqueous suspension reformulation of oral SMT C1100 were well tolerated and met the primary safety endpoint. All subjects were within the normal limits for blood pressure, pulse rate, body temperature and 12-lead ECG data. Summit also said that the aqueous formulation improved absorption levels of the compound that were "above those anticipated to be needed to achieve clinical efficacy." An increase in a single dose of 50 mg/kg to 400 mg/kg SMT C1100 increased blood plasma levels of the product for several hours to above the estimated 0.2 µM level required to achieve a 50% increase in utrophin levels as measured by in vitro cell assays from cells of DMD patients.

In Part 1 of the trial, patients received single doses of 50, 100, 200 or 400 mg/kg SMCT C1100. In Part 2, patients received multiple doses of 100 or 200 mg/kg SMCT C1100. Data from Part 2 of the trial are data expected this year. The trial is being funded in part by not-for-profits including the Muscular Dystrophy Association. Data were presented at the World Muscle Society meeting in Perth. ...