European rare disease biotech Azafaros grabs $28M early funding round

Azafaros' cash haul will be used to help bring its leading drug into the clinic. (Pixabay)

Azafaros has grabbed €25 million ($27.5 million) in a series A investment boost as it looks to push on with its pipeline focused on rare metabolic disorders.

The financing round for the two-year-old biotech was led by Forbion, with extra cash coming from BioMedPartners and founding investor BioGeneration Ventures.

Leiden, Netherlands-based Azafaros was spun out to work on the science coming out of Leiden University and Amsterdam University Medical Center and translate these early ideas into disease-modifying treatments for rare genetic metabolic disorders including lysosomal storage diseases (LSDs).

LSDs are actually a group of about 50 rare inherited metabolic disorders that result from defects in lysosomal function, with Gaucher, Fabry, Sanfilippo and Pompe diseases being some of the more common and well known. Older drugs for the disorders work as enzyme replacements, but newer approaches are looking to gene therapies to help.

Azafaros’ leading compound is AZ-3102, which will “initially address inherited life-threatening lysosomal storage diseases for which there are no effective therapies today,” although it did not give specifics on which LSD it would focus on.

The med is an orally available azasugar compound that the biotech says interferes with the metabolism of glycolipids and “uniquely affects several key disease pathways through a dual mode of action.” It’s still in the preclinical stage and details are thin, but the biotech is hustling toward clinical trials.

“The Series A financing round is a very significant achievement and endorsement for Azafaros. It allows us to build our organization and expand our executive team with passionate and experienced people and to advance our rare metabolic disorders pipeline,” said Olivier Morand, CEO at Azafaros.

Carlo Incerti, chairman of Azafaros, added: “I dedicated most of the last three decades of my professional life to the discovery and development of drugs for rare disorders. Azafaros represents the ideal continuation of this journey as it is developing new therapies which have the potential to meaningfully impact patients suffering from LSDs through a unique mode of action. These are major unmet medical needs, particularly in such diseases affecting the central nervous system. Azafaros’ lead oral small molecule holds the promise of becoming an innovative approach to treating these conditions.”