The company conducted two phase III double-blind, placebo-controlled studies in which Huntingdon's patients were randomized to receive either placebo or 2 grams (1 gram twice daily) of miraxion daily for six months. Study data showed no statistically significant difference in either study between miraxion and placebo with regard to the primary and secondary endpoints.
The primary endpoint of the trials was a change in the Total Motor Score 4 (TMS-4) component of the Unified Huntington's Disease Rating Scale. TMS-4 has been shown to be a sensitive measure of movement disorder in patients with the disease. In addition, secondary endpoints included cognition and Total Functional Capacity outcomes. Miraxion was found to be safe and well-tolerated by patients.
Rick Stewart, CEO of Amarin, said: “We are extremely surprised and disappointed by these top-line results, and we are analyzing the data in order to better understand the full and complete data set and outcomes.”
The company added that it would continue to evaluate the potential of miraxion in treating CNS disorders and its next steps with respect to the Huntingdon's trials.