PhIII: Liver enzymes spike in 7% of mipomersen group

Isis and Genzyme touted a full set of late-stage data on their cholesterol drug mipomersen, underscoring the therapy's success in hitting its primary endpoint and detailing the issue with elevated liver enzymes seen in a slice of the drug group.

Building on their release in early August, researchers found a 28 percent reduction in LDL levels in the drug group compared to a five percent increase among the placebo arm. "Having these data presented is a great milestone for the mipomersen program," said Paula Soteropoulos, vice president and general manager of Genzyme's cardiovascular business. "The data underscore our belief that mipomersen has the potential to help those familial hypercholesterolemia patients who are 'left behind' by current therapies, and are in need of new treatment options."

Genzyme wants to use the drug to treat familial hypercholesterolemia, a genetic disorder that triggers high levels of bad cholesterol. But analysts have been buzzing that a spike in liver enzymes--a classic red flag for researchers--could significantly curtail the drug's overall market reach.

According to their release from the European Society of Cardiology's Congress in Stockholm, Sweden, liver enzymes spiked in seven percent of the drug group, forcing three of the 83 patients in the drug arm to drop out of the study. Another seven were forced out citing other side effects such as injection site reactions.

"In all four of the Phase III studies we have completed, we have seen consistent and robust reductions in LDL cholesterol and other atherogenic lipids that support our plan to initially target homozygous and severe heterozygous familial hypercholesterolemia patients," Isis Pharmaceuticals Chairman and CEO Stanley Crooke, said in a statement.

- check out the Genzyme/Isis release
- read the Reuters story
- here's the report from the Boston Globe

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