ISIS-SMNRx: Additional Phase II data

Additional data from 24 patients ages 2-15 years with type II or III SMA in the first 3 cohorts of an open-label, dose-escalation Phase Ib/IIa trial showed that intrathecal ISIS-SMNRx led to mean increase in Hammersmith Functional Motor Scale-Expanded (HFSME) score from baseline to months 8-13 of 1.7 points in the 3 mg cohort, 3.2 points in the 6 mg cohort and 2.3 points in the 9 mg cohort. In 10 ambulatory children, ISIS-SMNRx led to mean increases from baseline to 12-16 months of 24.4 meters in the six-minute walk test (6MWT) and 22.7 meters in the upper limb mobility (ULM) test. Data were presented at the World Muscle Society meeting in Berlin. The company previously reported data from the trial (see BioCentury, Feb. 24). Isis is enrolling patients who have completed dosing in previous ISIS-SMNRx trials in an open-label extension study to receive maintenance doses of 12 mg ISIS-SMNRx every 6 months. ...