The company has filed an Investigational New Drug application with the US Food and Drug Administration for a clinical study of its drug SLS-005, also known as Trehalose.
Seelos is collaborating with Team Sanfilippo Foundation, a non-profit foundation formed by parents of children with the condition. The foundation provided a grant that is going towards funding the study.
The parties are also expanding the study from its original parameters. In addition to the study for patients with Sanfillipo syndrome types A and B, which are more common, Seelos is now able to offer a separate expanded access study for patients with types C and D, as well as those with A or B who don’t meet the initial entry criteria.
“We are very excited to be working with Team Sanfilippo to offer this innovative therapy to these patients for whom there is no effective treatment," Seelos' Chief Medical Officer Warren Wasiewski said. “Trehalose, with its multiple mechanisms of action, has the potential to be effective in a number of neurologic diseases for which we intend to explore.”
Sanfilippo syndrome is a degenerative disease that affects the central nervous system, typically in children. It is caused by a deficiency in certain enzymes involved in the degradation of sulfate. Over time, patients suffer a loss of speech and other cognitive skills, heart problems and often death prior to adulthood.
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