Savient’s Puricase meets Phase III endpoint

Puricase 8mg administered by a two-hour intravenous infusion every two weeks or every four weeks met the primary efficacy endpoint in the intent to treat (ITT) and per protocol analyses in each of two replicate, six-month Phase III clinical trials. The primary efficacy endpoint specified under the special protocol assessment was normalization of plasma uric acid during months three and six of the clinical trials.

Moreover, the every two week dose group also attained statistical significance for the a priori definition of reduction of gout tophi in the pre-specified pooled analysis, while the every four week dose group revealed a favorable numerical trend for this secondary endpoint. Analysis of other secondary efficacy endpoints also showed favorable numerical trends in one or both pegloticase dose groups.

Zeb Horowitz, senior vice president and chief medical officer, said: “We believe that the Savient gout studies are very important in two regards. We believe that the Phase III data show that pegloticase has the potential to be the first effective therapeutic option to control uric acid in treatment-failure gout patients. Second, we believe that our assessment of tophi in gout 1 and gout 2 by digital photography and image analysis uniquely demonstrates the attainment of a statistically significant and medically relevant clinical outcome, in this most difficult to treat gout patient population.”