Savient Pharmaceuticals reported positive results from two phase III trials, GOUT 1 [Gout Outcomes and Uric acid Treatment] and GOUT 2, of Puricase, a pegylated recombinant urate oxidase for the treatment of gout. The disease is caused by the deposit of urate crystals (via uric acid) in the body's tissues such as joints, tendons and organs. Most traditional treatments have centered on blocking the production of uric acid in the body, whereas Puricase attempts to reduce the levels of uric acid in the blood stream.
Puricase (8 mg) 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.
The primary endpoint was normalization of plasma uric acid during months 3 and 6 of the clinical trials. In the ITT analysis the mean responder rate for the every two-week dose group pooled across both studies was 42% (p is less than 0.001) and the mean responder rate for the every four-week dose group was 35% (p is less than 0.001).
In the per protocol analysis the responder rate for the every two weeks dose group was 61% (p is less than 0.001), and every four weeks was 50% (p is less than 0.001). The placebo responder rate for placebo was zero in both the ITT and Per Protocol analyses. A key secondary endpoint was reduction of gout tophi (grouped crystal deposits).
The every two week dose arm attained statistical significance in the pre-specified pooled analysis (p equal to 0.005) for the elimination of gout tophi. The every four week dose group did not attain statistical significance. Treatment was generally well tolerated.
Based on these results, Savient plans to file a (Biologic Li cease Application) BLA with the U.S. Food and Drug Administration (FDA) in 2008. Puricase was licensed from Duke University in 1998. According to the Centers for Disease Control (CDC), Gout may affect as much as 2.7% of the U.S. population alone.