Genzyme Eyes Isis Drug
Matthew Herper, 11.09.07, 6:00 AM ET


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Isis Pharmaceuticals, developer of a promising anti-cholesterol injection that produces unprecedented drops in heart-attack causing gunk in the blood, has said it needs to sign up a marketing partner before beginning a big trial next year.

The obvious choice would be any of the drug giants who dominate the $28 billion market for cholesterol-lowering drugs. Pfizer (nyse: PFE news people ), for one. Merck (nyse: MRK news people ). Maybe AstraZeneca (nyse: AZN news people ). But a surprising entrant is taking a long, hard look at Isis’ drug, previously known only as 301012 and now called mipomersen. It’s Genzyme, the biotech giant best known for selling very expensive treatments for rare and deadly genetic diseases.

Cambridge, Mass.-based Genzyme (nasdaq: GENZ news people ) has approached cardiologists with questions about the drug’s prospects, and that the biotech giant, which has sales of $3.2 billion and a market capitalization of $20 billion, seems to be taking a keen interest in mipomersen. These doctors say Genzyme has discussed mipomersen’s prospects with them at length.

Isis (nasdaq: ISIS news people ) says it is talking to lots of potential partners, but can’t comment on whether it is talking to any particular company. Genzyme said it would not comment on speculation about marketing deals.

But a Genzyme-Isis tie up to distribute mipomersen makes a great deal of sense. For one thing, mipomersen will at first be aimed squarely at the kind of market Genzyme understands best: an inherited genetic disease. And both companies are on the prowl for a partner. Isis needs one in order to start a large and expensive clinical trial testing its drug in patients who have high cholesterol that is not caused by a genetic defect.

Genzyme, for its part, has made acquisitions a key part of its growth strategy, with sales from products bought in from the outside generating 38% of total revenue in 2006, not to mention 50% of revenue growth, according to a research note by biotech analyst Geoffrey Porges at Sanford C. Bernstein. He counts 15 major deals going back to 1997, and he expects acquisition to remain a “core strategy.” But Porges doesn’t think Genzyme will want to spend on another major acquisition before the second half of 2008.

Thursday, Isis announced trials that could get mipomersen approved in the rare genetic disease familial hypercholesterolemia (FH). The worst form, caused by having two bad copies of a particular cholesterol gene, afflicts only a few hundred people. Their cholesterol levels can soar to six times the normal level and cause heart attacks in patients who are in their 20s.



Some 600,000 Americans have the more common form of FH, which is caused by a single bad copy of the cholesterol gene and causes levels of cholesterol that are just above the upper limit of normal. A study of both kinds of FH could be completed and filed with the U.S. Food and Drug Administration sometime next year.

Genzyme is used to selling drugs to small groups of patients for quite a lot of money. It has become one of the biggest biotech firms thanks to a series of rare diseases. Cerezyme, Genzyme’s first drug, treats a disease that afflicts fewer than 10,000 patients globally. Yet it generated $1 billion in total sales last year. Three other medicines sold by the company treat diseases that occur in just a few thousand people.

This model is so successful it is often imitated. Biomarin (nasdaq: BMRN news people ) and Alexion (nasdaq: ALXN news people ) have launched drugs for orphan diseases. Amicus (nasdaq: FOLD news people ) and Shire (nasdaq: SHPGY news people ) teamed today to address some of the same markets that Genzyme targets. But Genzyme has also been moving into broader markets. The drug Renagel, for kidney dialysis, brings in $500 million a year. It already sells a drug to treat FH, Cholestagel, in Europe.

But Isis would offer Genzyme a bigger opportunity: a chance to launch a drug into a far broader market. One reason Isis needs money is the company plans to launch a study next year in patients with garden-variety high cholesterol. This study will need to be far bigger and more expensive than one in patients who suffer from FH. And while existing medicines like Lipitor, Vytorin and Crestor aren’t enough for some FH patients, they work well for most patients with run-of-the-mill high cholesterol.

There’s no guarantee that just because Genzyme is looking at Isis that it will come through with a deal. Another partner could swoop in and sign up marketing rights to mipomersen. But the companies fit nicely. Food for thought as Isis prepares to hold an analyst meeting in New York on Nov. 13.

Sourced and published by Henry Sapiecha 2nd May 2009


  1. Food-Science » The Buzz: Abominable Science at HuffPo? : Page 3.14 Says:

    […] Science Articles & Inventions Online » Blog Archive » CHOLESTEROL …There’s no guarantee that just because Genzyme is looking at Isis that it will come through with a deal. Another partner could swoop in and sign up marketing rights to mipomersen. But the companies fit nicely. Food for thought as Isis … […]

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