RARE Daily

Azafaros Raises $28 Million to Advance Rare Metabolic Disorders Pipeline

February 6, 2020

Dutch biotech Azafaros has raised $28 million in a series A financing, which will be used to expand the company’s executive team and advance its preclinical rare inherited metabolic disorders pipeline into human trials.

Photo: Carlo Incerti, chairman of Azafaros

Forbion led the round with participation from BioMedPartners and founding investor BioGeneration Ventures.

Azafaros was established in 2018 to translate innovative science from Leiden University and Amsterdam University Medical Center into a pipeline of small molecules with disease-modifying potential for rare genetic metabolic disorders such as lysosomal storage diseases.

The company’s lead compound AZ-3102 is an orally available azasugar compound that interferes with the metabolism of glycolipids and uniquely affects several key disease pathways through a dual mode of action. Azafaros’ initial target is inherited lysosomal storage diseases such as Gaucher, Tay-Sachs, Fabry, Sanfilippo, and Pompe diseases. 

Lysosomes are cellular compartments filled with a variety of enzymes that are involved in the turnover and degradation of proteins, polysaccharides, nucleic acids, or lipids. For example, dysfunctions in these enzymes in the brain can result in the cytotoxic accumulation of degradation products, resulting in severe symptoms including developmental delays, seizures, respiratory infections, loss of vision and hearing, and cognitive functions.

“These are major unmet medical needs, particularly in such diseases affecting the central nervous system,” said Carlo Incerti, chairman of Azafaros and operating partner at Forbion. “Azafaros’ lead oral small molecule holds the promise of becoming an innovative approach to treating these conditions.”

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