Dutch clinical-stage company Azafaros has raised €132m in a Series B funding round, with the primary aim of advancing its Phase 3 clinical trials for innovative treatments targeting lysosomal storage disorders.
The financing round was led by Jeito Capital and co-led by Forbion Growth, while receiving additional investments from Seroba, Pictet Group. Existing backers including Forbion Ventures, BioGeneration Ventures (BGV), and Schroders Capital also took part in the round.
This funding will facilitate the expedited development of Azafaros’ leading compound, nizubaglustat, which is projected to commence Phase 3 trials for Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses by the end of the year.
Additionally, the financing will support the expansion of Azafaros’ pipeline to address other medical conditions.
Azafaros CEO Stefano Portolano said: “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.
“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.”
Established in 2018 by BioGeneration Ventures and utilising scientific research from Leiden University and Amsterdam UMC, Azafaros develops therapeutics for rare lysosomal storage disorders.
These disorders often result in progressive neurodegeneration and can be fatal.
Nizubaglustat is a small molecule that can be administered orally. It is designed to penetrate the brain and operates through a unique dual mode of action.
This drug candidate aims to provide treatment options for lysosomal storage disorders with neurological implications.
Nizubaglustat has been granted several regulatory designations from international health authorities.
These include rare paediatric disease designations for GM1 and GM2 gangliosidoses and NPC.
The drug candidate has also received orphan drug designations for GM1 and GM2 gangliosidosis, including Sandhoff and Tay-Sachs Diseases, as well as NPC. Additionally, it has been granted fast track designation along with investigational new drug (IND) clearance from the US Food and Drug Administration (FDA).
In Europe, it has received orphan medicinal product designation from the European Medicines Agency (EMA) and an Innovation Passport from the UK Medicines and Healthcare products Regulatory Agency (UK MHRA).
Jeito Capital partner Rachel Mears said: “Azafaros has been impressive in its execution with nizubaglustat poised to begin Phase 3 clinical development and the potential to significantly improve the lives of NPC and GM1/GM2 patients.
“We are excited to support and accelerate the Azafaros team in this important next step in the Company’s clinical development journey. Leading this round further demonstrates Jeito’s commitment to making a meaningful difference in patients’ lives by pursuing much needed benefits for those suffering from rare diseases.”
