CAMBRIDGE, Mass.—With the intent to make Genzyme Corp. the lead sponsor of the cholesterol-lowering drug mipomersen, the company announced yesterday that it struck a license and collaboration deal with Isis Pharmaceuticals for the drug that will provide Isis with an upfront license fee of $175 million. In February, Genzyme also received a $150 million payment from Genzyme to purchase 5 million Isis shares at $30 per share.
The latest announcement amends a deal the two companies struck early this year. It also follows regulatory delays for the drug which occurred in late April, when the FDA requested carcinogenicitydata from two ongoing pre-clinical studies of the drug, which aims to treat homozygous familial hypercholesterolemia (hoFH), a severe form of high cholesterol that h can lead to early onset of cardiovascular disease. News of this saw Isis shares tumble $4.81 in a single day from $16.80 to $11.99. Isis shares closed trading on Tuesday at $14.18.
Under the terms of the amended deal, in addition to the license fee, Isis has agreed to provide up to an additional $50 for the development of mipomersen, which could bring its total development funding to $125 million. In exchange for the additional funding, Isis could earn milestone payments of $75 million.
"Mipomersen is an innovative treatment that has the potential to change the standard of care for severely ill patients whose needs cannot be addressed by current cholesterol-lowering therapies," said Henri A. Termeer, Genzyme's chairman and CEO, in a prepared statement. "This treatment is an important addition to Genzyme's robust late-stage pipeline."
In response to guidance received from the FDA, the companies have modified the initial development plan for mipomersen, subject to further discussions with the agency.
These changes include the addition of clinical studies of mipomersen in apheresis-eligible patients; consolidation of the planned filings for heterozygous FH patients and other high-risk, high cholesterol patients into a single registration in the United States; and acceleration of the planned outcome study so that it can be used to support the consolidated U.S. filing.
The filing is now expected in 2010.