Seelos Therapeutics Inc (NASDAQ:SEEL), the biotech company, has won a crucial advance in its development plans in Europe and the US for trehalose (SLS-OO5), its investigational molecule, which takes aim at a number of neurodegenerative diseases.
The New York company has succeeded in setting up a meeting in March this year with the European Medicines Agency to seek scientific advice and assistance for a clinical study in Europe of trehalose, to assess its effectiveness in treating patients with Type A and B versions of Sanfilippo syndrome, a rare childhood disorder that triggers fatal brain damage.
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“We are truly excited to be working with the EMA to formulate the Sanfilippo study in the EU,” said Dr. Raj Mehra, the chairman and CEO of Seelos, in a statement. “There are currently no approved therapies in the EU for this devastating neurological condition.”
A meeting with the EMA is critical as it offers guidance and incentives for the research and advance of treatments for rare diseases across the European Union.
Alongside its push into Europe, Seelos has also provided the Food and Drug Administration with a protocol for its planned expanded access study to treat patients with Type C and Type D versions of Sanfilippo as well as patients with Type A and Type B versions of the disease who don’t meet the criteria to enter a Phase 2b/III clinical trial.
Trehalose is promising as it has been shown to reduce the aggregation of misfolded proteins and also help to reduce the accumulation of pathologic material. The way this molecule works is that it 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.
Diseases which are connected by the common factor of protein aggregations include Sanfilippo syndrome, Oculopharyngeal Muscular Dystrophy (OPMD), Spinocrebellar Ataxia (SCA3), Parkinson’s Disease, Amyotrophic Lateral Sclerosis (ALS), Alzheimer’s disease, Huntington’s disease and Friedrich Ataxia (FA).