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. This Phase III study includes patients who are not on low-density lipoprotein (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 III 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 II 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 200mg dose of mipomersen or placebo weekly for 26 weeks, with percent change in LDL-C as the primary endpoint.
The initial indication sought for mipomersen will be for patients with homozygous FH, and enrollment in a Phase III trial in this patient population is complete. Top-line data are expected to be available in mid-2009, and the submission of a US marketing application for this indication is anticipated during the second half of 2010.
Data from the four new studies of mipomersen will continue to build the body of clinical evidence around the treatment’s value in managing high-risk, high-cholesterol patients. Data are expected to be available before the companies begin an outcome study of mipomersen, and will help inform that trial’s design. The study, anticipated to begin in mid-2010, is intended to support potential expansion of mipomersen’s label to include a broader group of at-risk, high cholesterol patients.