Genzyme and Isis Begin New Clinical Trials of Mipomersen
Genzyme Corp. and Isis Pharmaceuticals, Inc. announced that they have begun two new studies of mipomersen, a novel lipid-lowering drug in late-stage development, and a third is currently screening patients. These three trials will provide additional data on mipomersen in high-risk patient populations. The new studies, together with ongoing trials, will also substantially increase the size of the database of patients treated with mipomersen over the next 18 months.
The trial currently screening patients will evaluate the safety and efficacy of mipomersen in patients with severe hypercholesterolemia who are on a maximally tolerated lipid-lowering regimen (previously referred to as apheresis-eligible patients). This phase 3 study includes patients who are not on LDL cholesterol apheresis but who have such severely elevated LDL-C levels that they are eligible for the procedure, and will enroll up to 75 patients.
In addition, the companies recently initiated a phase 3 trial in hypercholesterolemic patients on maximally tolerated dosages of statins who are at high risk for coronary heart disease. This study will enroll up to 180 patients. The companies have also begun a new phase 2 study in high-risk, high-cholesterol patients who are intolerant to statins, which will enroll up to 30 patients.
All three new trials are double-blind, placebo-controlled studies in which patients will be randomized 2:1 to receive a 200 mg dose of mipomersen or placebo weekly for 26 weeks, with percent change in LDL-C as the primary endpoint.
The companies in August began a phase 3 trial of mipomersen in patients with heterozygous familial hypercholesterolemia (heFH), a genetic disorder that causes severely elevated LDL-C levels.
The initial indication sought for mipomersen will be for patients with homozygous FH, and enrollment in a phase 3 trial in this patient population is complete. Top-line data are expected to be available in mid-2009, and the submission of a U.S. marketing application for this indication is anticipated during the second half of 2010. Genzyme continues to engage in discussions with regulatory authorities outside the U.S. regarding the development path for mipomersen.
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