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February 4, 2013
16:02 EDTGEVASynageva announces publication of sebelipase alfa study
Synageva BioPharma announced publication of the 12-week Phase I/II extension study data of sebelipase alfa in adults with lysosomal acid lipase deficiency in the online version and an upcoming print edition of Hepatology. Nine adults with LAL Deficiency were enrolled in the Phase I/II trial. After completing the initial portion of the Phase I/II trial, patients were allowed to continue treatment with sebelipase alfa as part of a long-term, open-label extension study. In the extension study, patients received four once-weekly infusions of sebelipase alfa and then transitioned to every other week infusions of sebelipase alfa. Eight of nine patients enrolled in the extension study. Data published in Hepatology were derived from the seven patients who completed the first 12 weeks of dosing in the extension study. Sebelipase alfa was well tolerated throughout the initial 12 weeks of the extension study, the company said.
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January 26, 2015
07:36 EDTGEVASynageva announecs dosing of patients concerned with SBC-103 in Phase 1/2 study
Synageva announced dosing with SBC-103 in patients with mucopolysaccharidosis IIIB has begun as part of a Phase 1/2 study. In addition, the FDA recently granted SBC-103 Fast Track designation. The trial will enroll approximately nine patients, two years of age or greater but less than 12 years of age, with definitive diagnosis of MPS IIIB and developmental delay. Patients will be treated in one of three different dosing cohorts with every other week intravenous administrations of SBC-103 for 24 weeks. Patients who meet qualifying criteria may continue therapy with SBC-103 for an extended treatment period that will last up to 128 weeks. The primary endpoint of the trial is safety and tolerability of intravenous administration of SBC-103 in patients with MPS IIIB. The study will also determine the effects of dosing with SBC-103 on the onset, magnitude, and reversibility of changes in levels of total HS in cerebral spinal fluid, serum, and urine as well as measure the effects of neurocognitive and developmental function and change in brain structures as assessed by magnetic resonance imaging. Exploratory biomarkers, SBC-103 concentration in CSF, MPS IIIB disease characteristics, symptoms, and quality of life will also be measured.

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