Genzyme presented positive results from a randomized, double-blind, placebo-controlled Phase 2 trial to evaluate the effect of weekly injections of mipomersen (MIPO) on low-density lipoprotein cholesterol (LDL-C) in high-risk patients intolerant to statins.
The primary endpoint was the percent change in LDL-C in from baseline to the primary efficacy time point (PET). The 26-week trial found that weekly treatment with MIPO reduced LDL-C by 47% compared to 2% with placebo. Lp(a) and Apo B were also reduced in MIPO patients. High density lipoprotein increased in MIPO patients but the difference was not statistically significant compared to placebo.
Inhibition of ApoB synthesis with MIPO may offer an effective strategy to lower LDL-C in patients at high risk for cardiovascular disease for whom currently no treatment option is available
In the study, 33 patients were randomized to receive 26 weekly doses of 200 mg MIPO or placebo. Lipid levels were measured monthly. Safety was determined via treatment-emergent adverse events and clinical laboratory evaluations. In patients with transaminase, increased limit of normal liver fat content was assessed using magnetic resonance spectroscopy.
Mipomersen lowered LDL-particle numbers with reductions in the small LDL particles. Four MIPO and two placebo patients discontinued dosing due to adverse events. The most common adverse events in both groups were injection site reactions and flu-like symptoms. Persistent transaminase increases occurred in 33% of MIPO patients. Post-baseline MRS in 16 MIPO and one placebo patient showed elevated liver fat content. Liver needle biopsies performed in two patients showed severe hepatic steatosis with minimal inflammation and minimal to no fibrosis.
Release Date: Nov. 16, 2011 Source: Genzyme
What about them? Have you ever seen an injection site reaction from one of the injectable MS drugs? I know you're going to tell me about the new oral MS drug from NVS, do you know the side-effects that that drug encompasses? Do a book report and get back to us. t
Cripes, I informed all here four years ago that this would be the result. Remember, in the end not one dime will come down to the Isis bottom line !
Yes, Genzyme has years of work to go ! Genzyme is stunned with frustration over the problems with Mipo. Hey, I warned them with regard to the dark flaws of antisense drugs. Oh well. zzzzzzzzzzzzzz :>(