Genzyme Offers Glimpse at Growth Engine, New Drugs for High Cholesterol, Gaucher’s

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had “clinically significant improvements” in bone mineral density, which can weaken over time in patients with the disease, the company said.

More detailed results on how well this drug did in the earlier trial will be presented May 15 at the Gaucher Leadership Forum in Milan, Italy. The financial types will surely be tuning in to this. Cerezyme generated $1.24 billion in sales last year, more than one-fourth of Genzyme’s total revenue. So if Genzyme can convert patients over to an oral drug with a longer patent life—and if it can offer advantages over potential generic rivals to its workhorse Cerezyme franchise—it could strengthen the company’s financial base for years. Of course, the clinical trial results will need to be confirmed in a pair of Phase III clinical trials.

—Venturing beyond the world of rare genetic diseases that it knows so well, Genzyme talked about a potential hit for lowering cholesterol, mipomersen. This drug, which the company has been co-developing with Carlsbad, CA-based Isis Pharmaceuticals since January 2008, is expected to produce a pivotal clinical trial result in the middle of this year, said John Butler, the president of Genzyme’s cardiometabolic and renal business.

Early trials of this drug, which is designed to interfere with a protein called apoB that carries “bad” cholesterol in the blood, have produced “extremely compelling” data suggesting that it can significantly reduce cholesterol for patients with a rare genetic disease that gives them high cholesterol that can’t be controlled well by existing statin drugs. This initial patient population only includes about 550 to 650 total patients, which is very “Genzyme-like,” Butler said. If that trial shows promise, Genzyme will methodically work its way down the pyramid into much bigger pools of patients, including the really big market with more than 1 million people whose cholesterol puts them at “high risk” of dangerous events like heart attacks or strokes.

Pricing is the big question mark with this drug, since Genzyme has traditionally been able to charge $200,000 or $300,000 a year per patient when it sells life-saving drugs to small groups of people—but those kind of economics obviously won’t fly with millions of people who have milder disease. Butler ducked one question on this from an investor, but he said the company expects it will be able to charge a premium over other medicines if it can generate clinical trial data to prove mipomersen offers substantial advantages over the standard of care.

—Genzyme was less thrilled to tell investors about progress with the FDA on winning approval for the larger-scale manufacturing of Myozyme. The company failed to win FDA approval on time in late February for its process of making a version of this drug for Pompe disease in a 2,000-liter bioreactor at its Allston Landing factory, which will be called Lumizyme. The Pompe treatment has suffered from supply constraints the past couple of quarters, but once Genzyme gets approval to make the drug in larger batches, it has potential to become another $1 billion hit like Cerezyme, executives say.

Genzyme is now preparing to turn in a response by mid-May to questions the FDA raised about “deficiencies” at the Allston facility. Inspectors for the FDA are expected to make an unscheduled visit “any day,” and the company believes it is ready to show them that it can meet all the requirements for mass producing the product, said Alison Lawton, senior vice president of global product access.

—The single biggest investment in Genzyme’s pipeline is with alemtuzumab (Campath) for multiple sclerosis. This drug, which is made to knock out overactive B and T cells of the immune system, is already approved for chronic lymphocytic leukemia. It showed promise in a mid-stage clinical trial for MS, with an ability to reduce flare-ups of this chronic neurodegenerative disease more than any drug on the market, according to senior vice president Terry Murdock.

The market for MS drugs is growing fast, as these products for the disease are estimated to generate $8 billion to $9 billion annually by 2011. If Genzyme can confirm its results in ongoing pivotal studies, Campath could start tapping into this market in 2012, the company said.

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