For more information on our current clinical studies, please click here.
A single dose, open label, cohort dose escalation Phase 1 study of pegsitacase (formerly Uricase-PEG 20) was carried out in gout patients. This study enrolled 12 subjects who were administered single intramuscular doses of pegsitacase at 0.5, 1 or 2 IU/kg. Safety and tolerability of pegsitacase, as well as pharmacokinetics, pharmacodynamics and immunogenicity were assessed.
Safety and Tolerability of Pegsitacase
The safety profile of pegsitacase was excellent in this study. No adverse events beyond grade 2 were observed. No allergic reactions were reported.
Single-Dose Pharmacokinetics of Intramuscular Pegsitacase
The data indicated that pegsitacase is quickly available after IM injection, with a Tmax approximately 3 days. The drug is eliminated slowly, with the circulating half-life estimated at approximately 8 days. Pegsitacase exhibited linear pharmacokinetics over the dose range tested in this study. The time-concentration curve obtained for the three doses studies is shown in the figure.
Single-Dose Pharmacodynamics of Intramuscular Pegsitacase
Treatment resulted in a dose dependent decrease in peripheral blood uric acid, with doses of 1-2 IU/kg sufficient to eliminate most, if not all, detectable plasma uric acid for 7 days and to maintain levels under 6 mg/dL for 15 days (see figure).
Immunogenicity of a Single Dose of Pegsitacase
All subjects were tested for the presence of antibodies to pegsitacase. None of the individuals had antibodies to pegsitacase that exceeded extremely low titers, nor did any subject show evidence of neutralizing antibodies.
