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news.Synageva BioPharma has started dosing in its Phase I/II trial with SBC-103 to treat patients with a rare disease called mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome).
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MPS IIIB is caused by a decrease in alpha-N-acetyl-glucosaminidase (NAGLU) enzyme activity which leads to the buildup of abnormal amounts of heparan sulfate (HS) in the brain and other organs.
The company said that accumulation of abnormal HS in multiple cells and tissues leads to severe cognitive decline, behavioral problems, speech loss, increasing loss of mobility and premature death.
The US Food and Drug Administration (FDA) had granted fast track designation for SBC-103, which is a recombinant form of natural human NAGLU designed to replace the missing (or deficient) NAGLU enzyme.
In the trial, the company will enroll around nine patients, two years of age or greater but less than 12 years of age, with definitive diagnosis of MPS IIIB and developmental delay.
During the trial, patients will be treated in one of three different dosing cohorts (0.3mg/kg, 1.0mg/kg or 3.0mg/kg) with every other week intravenous administrations of SBC-103 for 24 weeks.
The company said that patients who meet qualifying criteria may continue therapy with SBC-103 for an extended treatment period that will last up to 128 weeks.
The trial’s primary endpoint is safety and tolerability of intravenous administration of SBC-103 in patients with MPS IIIB.
Synageva president and chief executive officer Sanj Patel said: "Lysosomal disease enzyme replacement therapies with the recombinant form of the natural human enzymes are the gold standard of treatment for patients with these devastating diseases, and the results from this clinical trial will help further our understanding of the role of SBC-103 in MPS IIIB."