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Synageva Reports Closing of Targeted Enrollment in Phase 1/2 Trial with SBC-103 for Mucopholysaccharidosis IIIB


Synageva BioPharma Corp. (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, today announced the completion of enrollment in a Phase 1/2 trial with SBC-103 in patients with mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome).

About the trial

The trial reached its targeted enrollment of nine patients, who are two years of age or greater but less than 12 years of age, with a definitive diagnosis of MPS IIIB and developmental delay. Patients are being treated in one of three different dosing cohorts (0.3 mg/kg, 1.0 mg/kg or 3.0 mg/kg) with every other week intravenous administrations of SBC-103 for 24 weeks. Patients who meet qualifying criteria may continue dosing with SBC-103 for an extended period.

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 evaluate the effects of dosing with SBC-103 on the change from baseline levels of total heparan sulfate (HS) in urine, serum, and cerebral spinal fluid (CSF), as well as measure the effects on neurocognitive and developmental function and change in brain structures as assessed by magnetic resonance imaging.

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