Aegerion Pharmaceuticals drug candidate Lomitapide has demonstrated long-term safety and efficacy results in a 78-week Phase III clinical trial.
Lomitapide is an oral, once-daily, small molecule microsomal triglyceride transfer protein inhibitor (MTP-I), in clinical development to evaluate its ability to reduce low density lipoprotein (LDL-C) levels in patients with homozygous familial hypercholesterolaemia.
The 78-week pivotal Phase III study is a single-arm, open label trial involving 29 patients with a mean LDL-C of 337mg/dl (352 mg/dl for completers) on a variety of background lipid-lowering therapies.
The Phase III study is designed to assess the efficacy and long-term safety of lomitapide for the treatment of patients with hypercholesterolaemia, and found that the study results were consistent with data previously reported at 26 weeks and 56 weeks.
In the study, following six-week run-in period on current lipid-lowering therapy to determine baseline measurements, patients received ascending doses of Lomitapide escalated over the first 26 weeks of the trial to a maximum tolerated dose of up to 60mg/day.
The study also demonstrated that Lomitapide showed a reduction in LDL-C cholesterol from baseline was maintained at 78-weeks.
In the first 56 weeks of the Phase III trial, four patients experienced consecutive aminotransferase (ALT or AST) elevations of between five and 11 times the upper limit of normal, but between weeks 56 and 78, no additional patients experienced consecutive ALT or AST elevations of greater than five times the upper limit of normal.
"We are pleased that the 78-week data are consistent with our 56-week data," said Aegerion CEO Marc Beer.
Hypercholesterolaemia is a rare and often fatal condition characterised by severely elevated levels of LDL-C leading to life-threatening cardiovascular events.
The US Food and Drug Administration has granted orphan drug designation to Lomitapide for treating this condition.