Rare Disease Biotech, Azafaros Finds Big Money in Small Molecules, Raises US$ 28M in Series A

By Ruchi Jhonsa, Ph.D.

Established in 2018, the Dutch biotech has raised $28 million in Series A funding for developing small molecules as a treatment for rare genetic metabolic disorders such as lysosomal storage diseases. This funding round was led by Forbion, with participation from BioMedPartners and founding investor BioGeneration Ventures. Expressing his joy at the successful funding round, Dr. Carlo Incerti, the Chairman said, “Azafaros represents the ideal continuation of his journey in the discovery of drugs for rare disorders like lysosomal storage disorders. The lead molecule developed by Azafaros holds the potential of becoming an innovative approach in treating these conditions”.

Lysosomal storage disorders (LSDs) are a group of 50 rare inherited conditions that are characterized by the accumulation of waste products in the lysosomes. Gaucher, Fabry, Sanfilippo, Pompe, and Niemann-Pick disease are some of the common and well-known LSDs. Clinical phenotypes may vary according to the waste accumulated in the cells and may include central nervous manifestations, kidney failure, and heart conditions. Treatments are very limited in their scope and are often given to delay disease progression after the damage has occurred. However, trials are underway for developing enzyme replacement and gene therapies for these disorders.

Azafaros was founded with an aim to fight these rare metabolic diseases by a team of experienced industry professionals and scientists who have dedicatedly worked in the field of rare genetic metabolic diseases like LSDs. Spun out of Leiden University, the company holds an exclusive license from LU and Amsterdam UMC to novel patented small molecule compounds which were developed by Prof. Hans Aerts, Prof. Hermen Overkleeft, Prof. Stan Van Boeckel and their coworkers at the Leiden Institute of Chemistry. The company’s lead molecule 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. Through this molecule, the company initially plans to target LSDs such as Gaucher, Tay-Sachs, Fabry, Sanfilippo, and Pompe diseases.

“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 experiences people and to advance our rare metabolic disorders pipeline” said Olivier Morand, the CEO.

Professor Hans Aerts, one of the top experts in the field stated: “Azafaros has secured the capital to develop a pipeline of first-in-class small molecules that have great potential for treating patients with rare diseases. Based on their mode of action, these compounds provide the prospect for a disease-modifying treatment combined with a convenient oral administration route that allows a life-long treatment.”

References

1. https://www.azafaros.com/wp-content/uploads/2020/02/20200206_Press-Release-Azafaros-Series-A_Final.pdf

Author

Ruchi Jhonsa