Thursday, August 7, 2008

Genzyme’s cholesterol-lowering drug in Phase 3 trial

By Mass High Tech Staff

Cambridge-based Genzyme Corp. and Isis Pharmaceuticals Inc., of Carlsbad, Calif., have announced the start of Phase 3 clinical trials of the companies’ lipid-lowering drug, mipomersen, used for the treatment of heterozygous familial hypercholesterolemia (heFH), a genetic disease that inhibits the body’s ability to metabolize low-density lipoprotein cholesterol.

According to Genzyme CEO and chairman Henri Termeer, mipomersen is a “potential blockbuster” that could improve the standard of care for patients who don’t respond to conventional treatments to reduce cholesterol.

The clinical trial is expected to address the impact and safety of the mipomersen drug on about 100 patients diagnosed with heFH and coronary heart disease. The companies said they plan three additional trials in the second half of 2008.

Under terms of the mipomersen licensing and collaboration agreement, Genzyme (Nasdaq: GENZ) will adopt regulatory responsibilities for the U.S. Food and Drug Administration, as well as with European regulatory authorities.

In June, Genzyme reported it had paid its final license fee of $175 million to Isis for mipomersen. In February, Isis (Nasdaq: ISIS) received a $150 million payment from Genzyme to purchase five million shares of Isis common stock at $30 per share.

The pair of companies have updated the deal terms so that Isis will contribute up to $50 million in additional development funding for mipomersen, bringing Isis’ development funding commitment up to $125 million. Isis and Genzyme will share development costs equally after that, officials said. The new deal also means Isis could receive $75 million in milestone payments early.

With 10,000 employees, Genzyme reported a 2007 profit of $480.2 million on revenue of $3.8 billion.