Amarin Announces Successful Data From Huntington's Disease Program
Amarin Corporation has met with FDA following the completion of a comprehensive data review from its large-scale Phase III studies of Miraxion to treat Huntington's disease (HD). The FDA indicated that one additional Phase III trial demonstrating robust results, in conjunction with the confirmatory evidence from the existing clinical data, may be sufficient clinical data to support a New Drug Application.
Rick Stewart, Chief Executive Officer of Amarin, commented "We are pleased with the results of the comprehensive clinical data review and are encouraged by the outcome of our recent dialogue with the FDA. We are now in discussions with the Huntington Study Group to determine the optimal design of such a single Phase III trial. We are also considering whether Amarin will conduct this study itself or seek a collaborative partner with which to advance Miraxion in Huntington's disease."
Since the most recent update on Miraxion to treat HD on August 1, 2007, the Company has continued a comprehensive analysis of all clinical data with its advisors. This additional analysis supports the preliminary findings, suggesting a clinical benefit from a longer treatment period for Miraxion.
Positive 12-month results from U.S. Phase III Trial (TREND-HD)
The TREND-HD trial, conducted by the Huntington Study Group in the U.S. and Canada in 316 patients, was a 6-month, double-blind placebo controlled study followed by a further 6-month extension period where all patients received 2 grams per day of Miraxion. The primary endpoint was the 6-month change in TMS-4, a scale that measures motor symptoms. As previously announced on April 24, 2007, at the 6-month time point, there was no statistically significant difference in efficacy between the Miraxion group and the placebo group.
Analysis of the 12-month data showed a statistically significant difference in TMS-4 between the long term Miraxion group (12-months treatment) and those patients who had switched to Miraxion at 6-months. Treatment codes were not broken at the switching point so investigators and patients remained blinded to receipt of Miraxion or placebo in the initial 6- month period. These results suggest that there is benefit from a longer treatment period with Miraxion and are consistent with the 24-month open-label data from the earlier 135-patient trial completed in 2003.
Positive Additional Analysis
A longitudinal analysis, which is a widely accepted and validated alternative statistical methodology, was applied to all available clinical data as part of the comprehensive review.
TREND-HD Trial Outcome
The longitudinal methodology was applied to both the 3 and 6-month combined results as well as the 3, 6, 9 and 12-month combined results. A statistically significant difference between the Miraxion and placebo groups was identified in each of the 3-6 month and the 3, 6, 9 and 12-month analyses.
Earlier 135 patient Trial Outcome
In the earlier 135-patient study, the longitudinal analysis showed a statistically significant difference between Miraxion and placebo in the per protocol patient groups and, additionally, a greater degree of statistical significance in the intent to treat genetic sub-group of patients with a CAG(less than or equal to)44 than in the previous analysis.
Analysis was also conducted on the 24-month open label data from the 135-patient trial, in which patients initially randomized to treatment with placebo had been switched to Miraxion and treated for 12 months. After switching to Miraxion, these patients demonstrated an effect of similar magnitude to patients randomized to treatment with Miraxion at baseline.