Genzyme competition may get clearer at conference:What: Lysosomal Disease Network World Congress

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PREVIEW-Genzyme competition may get clearer at conference

What: Lysosomal Disease Network World Congress

* When: Feb. 10-12

* Genzyme, Shire, Protalix to present Gaucher data

By Toni Clarke

BOSTON, Feb 8 (Reuters) - Genzyme Corp (GENZ.O), Shire Plc (SHP.L) and Protalix Biotherapeutics Inc (PLX.A) will each be presenting data this week that could give hints as to how the competitive landscape for Gaucher disease treatments could look a year or two from now.

Cambridge, Massachusetts-based Genzyme, which currently owns the $1.2 billion Gaucher market with Cerezyme, its biggest-selling drug, faces competition from Protalix and Shire, whose rival products are expected to win approval by the end of the year.

Gaucher is a rare genetic disorder in which patients lack an enzyme that breaks down certain fats in the body, leading to organ damage and even death. It affects roughly 5,000 people worldwide.

Manufacturing problems at Genzyme last year led to a shortage of Cerezyme and gave an unexpected boost to British drugmaker Shire and Israel-based Protalix, as U.S. regulators smoothed their path towards approval.

All three companies will be presenting data at the Lysosomal Disease Network World Congress in Miami, beginning Feb. 10, that they hope will show their drugs to advantage.

"To a far greater extent than in past years, we believe this meeting may have a high profile for Genzyme, with the first full presentation of competitive data of Gaucher programs from both Shire and Protalix," said John Sonnier, an analyst at William Blair & Co, in a research note."

Protalix, which recently signed a marketing agreement with Pfizer Inc (PFE.N), the world's biggest drugmaker, is expected to present detailed data from a late-stage trial of its experimental drug Uplyso, also known as taliglucerase.

In October, the company said initial data from the trial showed the drug reduced the volume of spleens enlarged by fat build-up by a statistically significant amount after nine months. It also showed that only 6 percent of patients developed antibodies to the drug compared with a rate of 15 percent for Cerezyme.

Most biotech drugs are made using mammalian cells. Uplyso is made using genetically engineered carrot cells.

Protalix completed the filing of its marketing application with U.S. regulators in December, and analysts had expected the drug to win approval by the middle of this year. But Protalix said recently that the U.S. Food and Drug Administration wants additional information, a request analysts said could push back approval by six months.

Even so, some analysts are optimistic that Pfizer's involvement will help maximize Uplyso's market share, and Ritu Baral, an analyst at Canaccord Adams, predicts the drug will capture 15 percent or more of the market.

"We think Cerezyme will lose market share due in part to a strong Pfizer-led marketing effort," she said in a research report.

Shire will be presenting full data from the first of three late-stage trials of its drug, velaglucerase alfa. Last August, Shire said that initial results showed the trial met its main goal and that patients taking 60 units per kilogram of the drug had a statistically significant increase in hemoglobin levels from baseline after 12 months.

Significant improvements were also seen in platelet count and spleen size. The changes were also seen in patients taking a 45-unit per kilogram dose.

Genzyme will be presenting data from its new Gaucher disease drug, GENZ-112638. Unlike Cerezyme, or Shire and Protalix's drugs, which are given intravenously, Genzyme's new drug is taken in capsule form -- a much more convenient delivery method.

Genzyme is likely to showcase the impact of the drug on bone pain. Last year the company presented data from a mid-stage trial showing it was similar to Cerezyme in its impact on platelet and hemoglobin levels and spleen size after one year.

"We believe that the emergent profile of GENZ-112638 is attractive, and feel an oral therapeutic option in the Gaucher disease arena could play a prominent role," said Sonnier. (Reporting by Toni Clarke, editing by Gerald E. McCormick)

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