Synageva BioPharma Corp. (“Synageva”) (NASDAQ:GEVA), a clinical stage biopharmaceutical company developing therapeutic products for rare disorders, today announced encouraging safety and tolerability data as well as effects consistent with preclinical findings and known mechanism of action. Patients from this 4-week Phase I/II trial of SBC-102 in adults with late onset LAL Deficiency continue to transition into an open-label extension study.
“Patients with LAL Deficiency have significant unmet medical need. The data from this trial, the first human study of an enzyme replacement therapy for subjects with late onset LAL Deficiency, are important, and combined with the initial safety profile of SBC-102, warrants further investigation in longer-term trials,” said Dr. Gregory Enns, MBChB, Assistant Professor of Pediatrics and the Director of the Biochemical Genetics Program at Stanford University and principal investigator in the trial.
About the Phase I/II trial of SBC-102 in late onset LAL Deficiency
The trial fully enrolled 9 patients from multiple sites in the United States and Europe. The primary objective of the study was to evaluate the safety and tolerability of SBC-102 administered weekly in adult patients with liver dysfunction due to late onset LAL Deficiency. Additional assessments included evaluating pharmacokinetics and biomarkers of SBC-102 activity including liver transaminases and serum lipids. Patients enrolled in the trial were diagnosed with LAL Deficiency and demonstrated evidence of liver involvement as assessed by the presence of hepatomegaly and/or elevated transaminases. Patients received four once-weekly infusions of SBC-102 (0.35 mg/kg, 1.0 mg/kg, or 3.0 mg/kg).SBC-102 was well-tolerated with no serious adverse events or infusion-related reactions, and all subjects completed their scheduled infusions. The most common adverse events included headache, nausea and diarrhea. The majority of adverse events were mild and unrelated to SBC-102. SBC-102 resulted in rapid and significant decreases in serum transaminases, with evidence of mobilization of lipids out of the liver and other tissues and into the blood, consistent with its mechanism of action.