Genzyme's Gaucher Disease Drug Shows Promising Results

Biotech company Genzyme Corp. announced Wednesday that its experimental drug to treat genetic disorder Gaucher disease has shown encouraging results in a Phase II clinical trial. The drug, Genz-112638, is an orally-delivered medication used to treat Gaucher, an enzyme deficiency that can cause liver and spleen enlargement, anemia and brittle bones. Initial data indicate that Genz-112638 "may produce a rapid and meaningful impact on important clinical endpoints," and the company is in contact with regulatory agencies to expedite development. Gaucher is usually treated with Genzyme's drug Cerezyme, which is delivered by infusion rather than by mouth. Cerezyme generates annual revenues for the company of over $1 billion. A successful oral version would be critical for Genzyme, since Cerezyme is about to face competition from Amicus Therapeutics, a biotech that is developing its own oral Gaucher medication. Amicus, which filed for an IPO in March, is working on oral versions of other Genzyme drugs as well, including Fabrazyme, which treats the genetic disorder Fabry disease, and Myozyme, which treats the muscle disorder Pompe disease. Full results of the trial of Genz-112638 will be available in 2008.

Sources: Press release, Reuters, MoneyCentral, MarketWatch
Commentary: Millenium Ends Its Bid For AnorMED, Leaving Genzyme On Top • Searching for Value Investments in the Drug Stock Universe
Stocks/ETFs to watch: Genzyme Corp. (GENZ). Competitors: Abbott Laboratories (ABT), Amgen Inc. (AMGN), Johnson & Johnson (JNJ). ETFs: Biotech HOLDRs (BBH), iShares Nasdaq Biotechnology (IBB)
Conference call transcripts: Q1 2007

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