Study 102 was a double-blind, randomized, placebo-controlled Phase 1 clinical study to assess the safety, tolerability, and pharmacokinetic profile of three doses of AEOL 10150 administered by subcutaneous ("SC") injection or infusion in patients with ALS. Three groups of six subjects (four receiving drug, two placebo, and total 18 subjects) were studied. Each subject in the first two cohorts received bid SC injections of AEOL 10150 or placebo for six days followed by a single SC injection on the seventh day for a total of 13 injections. In the first cohort, each injection was 40 mg and in the second cohort each injection was 60 mg.
There were two dosing modifications in the third cohort. First, the dosage was changed from a total fixed daily dose to a weight dependent dose (i.e. mg/kg) to ensure that each subject received the same dose irrespective of weight. Second, the method of subcutaneous compound delivery was changed from subcutaneous administration via needle and syringe to continuous subcutaneous delivery via infusion cannula. Subjects in the third cohort received a daily dose of 2 mg/kg delivered by osmotic infusion pump over 24 hours for 6.5 days.
The safety results can be summarized as follows: All subjects
completed the study. There were no serious or clinically
significant adverse events. Mild decreases in sitting systolic and
diastolic blood pressure were more fr