FACTBOX-Genzyme, Shire, Protalix Gaucher drug trial data

Feb. 11 | Thu Feb 11, 2010 4:52pm EST

Feb. 11 (Reuters) - Genzyme Corp GENZ.O, Shire Plc (SHP.L) and Protalix Biotherapeutics Inc (PLX.A) are each developing rival drugs to treat Gaucher disease, a rare genetic disorder in which patients are deficient in an enzyme that breaks down certain fats in the body, potentially leading to organ damage and death.

Genzyme currently holds a monopoly in the $1.2 billion market with its best-selling drug, Cerezyme. Shire and Protalix hope to have competing drugs approved this year. Genzyme is also in mid-stage trials of an experimental oral Gaucher treatment. The other drugs are given intravenously.

All three reported data on Thursday at the Lysosomal Disease Network World Symposium in Miami, Florida, and although the drugs were not pitted directly against each other in a head to head trial, a rough sense of how they stack up can be gleaned from individual trials. [ID:nN11231275]

The figures below show 12-month data from the pivotal Phase III trial of Shire's velaglucerase alfa; data from Protalix's Phase III taliglucerase alfa trial at nine months and historical data for Cerezyme at nine months.

They also show two-year results from a Phase II trial of Genzyme's experimental Gaucher pill, eliglustat tartrate, formerly known as GENZ-112638. [ID:nN11227266]

Companies typically benchmark the success of a Gaucher disease drug based on four measures: Hemoglobin, which carries oxygen through the blood; platelets, which help blood to clot; and decreases in spleen and liver volume.

Hemoglobin increase:

*Shire 23.3 percent

*Protalix 22.2 percent

*Cerezyme up 24 percent

Platelet count:

*Shire 65.9 percent

*Protalix 72.1 percent

*Cerezyme 43.5 percent

*eliglustat tartrate 81 percent

Spleen volume decrease:

*Shire 50 percent

*Protalix 38 percent

*Cerezyme 47.1 percent

*eliglustat tartrate 52 percent

Liver volume decrease:

*Shire 17 percent

*Protalix 11.1 percent

*Cerezyme 21.4 percent

*eliglustat tartrate 24 percent

(Reporting by Toni Clarke; editing by Andre Grenon)

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