Azafaros has secured 132 million euros ($147 million) in series B funds that the Dutch biotech will use to push its rare neurological disease drug through two late-stage trials.
The candidate in question is nizubaglustat, a brain-penetrant azasugar that Azafaros is aiming at rare lysosomal storage disorders affecting the brain, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC).
The tech behind nizubaglustat comes from Leiden University and Amsterdam UMC in the Netherlands and led to BioGeneration Ventures founding the biotech in 2018.
BioGeneration Ventures joined in the latest fundraise alongside fellow existing investors Forbion Ventures and Schroders Capital. The series B round was led by Jeito Capital and Forbion Growth and follows a 25 million euro ($27.5 million) series A investment in 2020.
“This successful series B round marks a significant milestone for Azafaros, allowing us to accelerate the development of nizubaglustat and leverage our scientific understanding and competencies to bring additional candidates into development,” CEO Stefano Portolano, M.D., said in the May 13 release.
“The fact that we have been able to attract leading life sciences investors to join our existing, strong group of specialist investors is a testament to the impressive accomplishments of the team and the large unmet medical need that currently exists for patients with these hugely debilitating neurological diseases,” Portolano added. “We look forward to bringing nizubaglustat to patients.”
As well as using the series B funds to support phase 3 trials of nizubaglustat in both GM1 and GM2 gangliosidoses and NPC, the biotech will also employ some of the cash to expand its pipeline to other indications—although the company has yet to unveil more details about its portfolio beyond nizubaglustat.
Lysosomal storage disorders are 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.
NPC in particular prevents the body from moving and using cholesterol and other lipids in cells, in turn causing a buildup of fats in the brain, liver, spleen or lungs. Last year saw the FDA approve Zevra Therapeutics’ Miplyffa, also known as arimoclomol, to treat NPC in combination with the enzyme inhibitor miglustat, which has long been used as the primary, albeit off-label, treatment for NPC patients.