Seelos Therapeutics Inc (NASDAQ:SEEL) is moving forward with a Phase 2b/3 study of its drug Trehalose (SLS-005) in patients with Sanfilippo syndrome types A and B after receiving written feedback from the European Medicines Agency (EMA).
Based on the agency’s guidance, Seelos will design an open-label, non-placebo controlled study for the treatment of Sanfilippo patients in the European Union.
"The EMA's recommendation to use appropriate natural history data of Sanfilippo syndrome patients allows us to offer active therapy to all children enrolled in the study," CEO Raj Mehra said in a statement. "This devastating disease currently lacks any approved treatment and we look forward to taking this next step in advancing our SLS-005 program."
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Sanfilippo syndrome is a neurodegenerative disease that affects the central nervous system, typically in children. Over time, patients suffer a loss of speech and other cognitive skills, heart problems and often death prior to adulthood.
Trehalose has been shown to reduce the aggregation of misfolded proteins and also help to reduce the accumulation of pathologic material. The triggers autophagy — the body’s means of purging itself of damaged cells — via the activation of transcription factor EB (TFEB), a critical factor in lysosomal and autophagy gene expression.
Seelos also produces SLS-002, an intranasal racemic ketamine depression drug. The company previously said it plans to seek guidance from the US Food and Drug Administration (FDA) this month for an adaptive Phase III trial of SLS-002 for Acute Suicidal Ideation and Behavior (ASIB) in patients with Major Depressive Disorder (MDD).
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