Genzyme to move Gaucher drug to Ph III

US biotechnology major Genzyme presented Phase II trial data showing that its investigational oral therapy Genz-112638, for the treatment of  type 1 Gaucher disease, met its primary endpoint, at the Lysosomal  Disease Network WORLD meeting in San Diego, California.

The study had a composite primary efficacy endpoint: a  clinically-meaningful response in at least two of three targets,  improvements in spleen size, hemoglobin and platelet levels, in  individual patients after the 52-week study period.

The data indicate that 91% of those on Genz-112638 achieved the primary  endpoint: spleen volumes had decreased by a mean of 39%, while liver  volumes were cut 17%; hemoglobin levels had risen by a mean of 1.62g/dL  of blood; platelet counts jumped 40%; and chitotriosidase levels were  51% lower among the 20 patients treated with that agent. Genzyme is  currently finalizing protocols for two Phase III trials that it expects  to initiate in mid-2009.