Synageva BioPharma Corp. (“Synageva”) (NASDAQ:GEVA), a clinical stage biopharmaceutical company developing therapeutic products for rare disorders, today announced acceptance of data for oral presentation at the upcoming Society for the Study of Inborn Errors of Metabolism (SSIEM) meeting being held in Birmingham, England, September 4-7, 2012. The presentation includes a preliminary analysis of the on-going Phase I/II extension study of SBC-102 in adults with late onset LAL Deficiency.
About the Phase I/II Extension Study of SBC-102 in adults with late onset LAL Deficiency
After completing four weeks of treatment in the initial Phase I/II trial, patients were allowed to continue treatment with SBC-102 as part of a long-term, open-label, extension study. In the extension study, patients received four once-weekly infusions of SBC-102 (0.35 mg/kg, 1.0 mg/kg, or 3.0 mg/kg) and then transitioned to every other week infusions of SBC-102 (1.0 mg/kg or 3.0 mg/kg). Eight of nine patients have enrolled into the extension study. Data from seven of the nine patients completing the first 12 weeks of dosing in the extension study are included in the preliminary analysis that has been accepted for oral presentation at the upcoming SSIEM meeting. These data demonstrate further evidence of the sustained impact of SBC-102 on reducing liver transaminase levels. In addition, total cholesterol, HDL cholesterol, and triglycerides significantly improved (p<0.05) from patients’ original baseline (the beginning of the four week Phase I/II trial) to 12 weeks of the extension study. A reduction in LDL was also observed during the same time period.
SBC-102 was well tolerated through 12 weeks of the extension study. The most frequently reported adverse events of headache and diarrhea were mild in severity. Mild infusion-related reactions have been reported in some patients, none of which required modification of the infusion rate or discontinuation of SBC-102. No antidrug antibodies were detected in any of the nine subjects in the four week Phase I/II trial or in the seven subjects tested in the extension study. A single patient during the extension study experienced acute cholecystitis classified as a serious adverse event but deemed unlikely related to SBC-102 by the investigator. This event was successfully managed and the patient remains in the study. Longer-term results from this on-going extension study, including other measures of efficacy, are planned for presentation at another medical conference later this year.