Azafaros bags €132m for rare neuro-metabolic disorder drugs

Netherlands biotech Azafaros is poised to start phase 3 trials of its lead drug for a trio of rare lysosomal storage disorders (LSDs), helped by €132 million ($147 million) in second-round financing from European venture capital firms.

The Series B – led by Jeito Capital and Forbion Growth – will be used to fund two late-stage trials of Azafaros' nizubaglustat in Niemann-Pick disease Type C (NPC) disease and GM1/GM2 gangliosidoses, which lead to the accumulation of lipids in organs, including the brain, and cause neurological problems and developmental delays.

The drug, a small-molecule inhibitor of ceramide glucosyltransferase and non-lysosomal neutral glucosylceramidase (NLGase), was discovered in the lab of Professor Hans Aerts of the University of Leiden and – according to the biotech – has the potential to modify the course of all three diseases with oral daily dosing.

Azafaros has completed a first-in-human clinical study in healthy subjects, showing a favourable safety and tolerability profile and positive pharmacokinetics and pharmacodynamics data, and plans to start the phase 3 trials before the end of this year.

LSDs are a group of over 70 diseases that are characterised by dysfunction of lysosomes – organelles that clear toxic materials and defective cellular components from cells – and most are inherited as autosomal recessive traits. These disorders are individually rare but collectively affect 1 in 5,000 live births.

Part of what is behind Azafaros' enthusiasm for nizubaglustat is the drug's potential to provide treatment for multiple LSDs, regardless of their underlying disease mechanism. Most current therapies for LSDs are specific for a particular condition and, for many of them, no treatment exists at all other than palliative care.

The drug has been awarded orphan drug designations in both the US and Europe, as well as fast-track status in the US.

"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," said Stefano Portolano, Azafaros' chief executive.

"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," he added. "We look forward to bringing nizubaglustat to patients."

The financing was supported by Seroba, Pictet Group and existing investors Forbion Ventures, Schroders Capital, and BioGeneration Ventures.