Synageva BioPharma Announces Interim Data at 8th Annual Lysosomal Disease Network World Symposium

Synageva BioPharma Corp. GEVA 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.