Synageva BioPharma Corp. has released a preliminary analysis of data from an on-going Phase 1/2 extension study of SBC-102 in adults with late-onset LAL Deficiency. SBC-102 is a recombinant form of the human LAL enzyme being developed as an enzyme replacement therapy for lysosomal acid lipase (LAL) deficiency, a lysosomal storage disorder (LSD).
After completing four weeks of treatment in the initial Phase 1/2 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 mg/kg, or 3 mg/kg) and then transitioned to every other week infusions of SBC-102 (1 mg/kg or 3 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. 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 1/2 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 1/2 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. The patient remains in the study.
SBC-102 has been granted orphan designations by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency. Additionally, SBC-102 received fast track designation by the FDA.
Date: October 1, 2012
Source: Synageva BioPharma Corp.