Good and Bad News on New Cholesterol Medications

We have a good news bad news situation.  The bad news is that Pfizer once a powerhouse in the development of new cardiovascular compounds (remember Lipitor?) has all but removed itself from the field.  The loss of close to one billion dollars on torcetrapib in clinical trials has given it a new course in research, one that apparently does not include cardiovascular diseases.  Torcetrapib is a compound that raises HDL by up to 100% and had all of us itching to get our hands on.  As I have blogged about before, the FDA now requires new drugs to show a clinical benefit and not just a chemical benefit.  Torcetrapib did indeed raise HDL levels by 100% but in the process caused an excess of death in the treatment group, so it was pulled from development.  Another drug in this new group known as cholesteryl-ester transfer protein inhibitors remains in study.  It is known as anacetrapib and is presently completing its phase three study known as DEFINE.

What’s the good news?  Pfizer had another drug in its research arm which is also full of promise.  This compound is known as ApoA-1 Milano and has been known since the results of a small study were published in 2003.  This compound has now been sold to The Medicines Company, which is the company that brought us Angiomax, the drug which is currently the preferred means of anticoagulation during angioplasty.  The price will amount to $410 million dollars if milestones are met.

Why do we care about ApoA-1 Milano?  It’s an interesting story that starts in the small northern Italian village of Limone sul Garda, a beautiful spot on Lake Garda, which is Italy’s largest lake.  This town had no road until the mid 1930’s and before that was only accessible from the lake or by walking.  In a town of 1,000 persons, over a dozen are over 100 years old.  45 of the townspeople were found to have a variant of apolipoprteinA-1 which is the main protein component of HDL.  Their HDL levels are surprisingly low which also fed the mystery.  It is from these types of discoveries that science advances.  The work was started at the University of Milan and that is how the compound was named.  The compound was then sold to a company called Esperion which was subsequently acquired by Pfizer and that is how we got to this point.

Next…why all the interest?

Tags: Anacetrapib, ApoA-1 Milano, Cholesteryl-ester, HDL, Torcetrapib
Posted in Cholesterol | No Comments »