Amarin Corporation Announces Encouraging Preliminary Results of LAX-101 Pivotal Study in Huntington's Disease
LONDON, Oct. 28 /PRNewswire-FirstCall/ -- Amarin Corporation plc (Nasdaq: AMRN - News; Amarin) announced today the preliminary results of a Phase III study of LAX-101, an investigational, novel and proprietary product being developed for treating patients with Huntington's disease (HD).
The primary variable in the trial was the change over a one-year period in the Total Motor Score 4 (TMS-4) subscale of the Unified Huntington's Disease Rating Scale (UHDRS), the standard rating scale for trials in this disease. Preliminary results indicate that evaluable patients taking LAX-101 (the per protocol group) reached statistical significance when compared to placebo, using one of the analytical methods specified in the protocol. In addition, a majority of assessments representing the secondary endpoints, including total UHDRS score improvement, showed trends in favor of LAX-101 but did not reach statistical significance.
In the intent-to-treat group (all patients entering the study, including those who dropped out or did not comply with the protocol), preliminary results indicate that measurement of the TMS-4 primary variable did not reach significance, though trends toward improvement were observed and favored LAX- 101. LAX-101 was found to be well tolerated by patients throughout the trial. The incidence and types of adverse events reported were similar in the placebo and drug groups.
The results are from a multi-center, double-blind, randomized, placebo-controlled study of LAX-101, which enrolled 135 patients with HD at six sites, located in the United States, Canada, U.K. and Australia. These results are also consistent with findings in Phase II studies previously reported by Amarin in January 2002.
Rick Stewart, chief executive officer of Amarin, stated, "We are encouraged by the results of this trial and look forward to working closely with our partner Laxdale Ltd., who is in discussions with the U.S. Food and Drug Administration (FDA) in relation to the findings."
LAX-101 has been granted Fast Track designation by FDA as well as having received Orphan Drug designation in the U.S. and in Europe. Amarin licensed the U.S. marketing rights from U.K.-based Laxdale Ltd.
Huntington's disease is an autosomal-dominant genetic disease that has been diagnosed in approximately 30,000 patients in the U.S. The gene for HD causes the formation of abnormal proteins due to multiple repeats in a segment of the DNA of affected patients. In the U.S. it is estimated that in addition to the approximately 30,000 patients with a clinical diagnosis of HD, there are an additional number of individuals with the HD gene who are pre- symptomatic, who will eventually develop the disease. Because HD generally strikes patients during their peak earning potential years (30-50 years old) and because patients with end-stage disease require continuous nursing care, often in institutions, the annual cost to the U.S. economy for HD has been estimated to be as high as $2.5 billion. |
