Netherlands-based Azafaros, a clinical-stage company developing therapeutics for rare lysosomal storage disorders, has completed an oversubscribed €132M Series B funding round.
The investment was led by Jeito Capital, co-led by Forbion Growth, and included participation from Seroba, Pictet Group, and existing investors Forbion Ventures, Schroders Capital, and BioGeneration Ventures (BGV).
Rachel Mears, Partner at Jeito Capital, Julien Elric, Senior Principal at Jeito Capital, and, Audrey Cacaly, Principal at Forbion Growth, will join Azafaros’s Board of Directors as Board members.
Speaking on the investment, Rachel Mears, Partner at Jeito Capital, says, “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.”
Audrey Cacaly, Principal at Forbion Growth, adds, “Azafaros exemplifies the kind of science-driven, mission-focused company we seek to back. Nizubaglustat has the potential to fundamentally change the treatment landscape for rare genetic diseases, and we are proud to support their journey as they move closer to delivering real hope and this much-needed treatment option to patients and families.”
Advancing treatment paths for rare metabolic diseases
Founded in 2018, Azafaros is a clinical-stage company focused on developing treatments for rare genetic diseases.
Azafaros is based on research from professors Hans Aerts, Hermen Overkleeft, and Stan van Boeckel at Leiden University and the Academic Medical Center in the Netherlands.
The company is building a pipeline of therapeutics aimed at modifying disease progression in patients with rare lysosomal storage disorders. These disorders are genetic conditions that can lead to neurodegeneration and, in some cases, life-threatening outcomes.
Azafaros’s lead drug candidate, nizubaglustat, is a dual-acting compound currently in development for several lysosomal storage diseases. Azafaros uses its scientific knowledge and development approach to explore new paths for drug discovery and delivery.
Brief about nizubaglustat
Nizubaglustat is a small molecule and an oral drug candidate that can reach the brain and works through two biological mechanisms. It is being developed to treat lysosomal storage disorders that affect the nervous system, such as GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC).
GM1 and GM2 gangliosidosis (including Tay-Sachs and Sandhoff diseases) are lysosomal storage disorders caused by the buildup of gangliosides in the central nervous system. This leads to ongoing loss of neurological function and early death. The conditions mainly affect infants and children, and there are currently no treatments that change the course of the disease.
NPC is a progressive neurological disorder caused by mutations in the NPC1 or NPC2 gene, leading to problems with lipid processing in the body. This results in lipid buildup in the central nervous system. The disease can begin at any point in life, from before birth to adulthood.
Nizubaglustat has received several regulatory designations in the US, Europe, and the UK for the treatment of rare lysosomal storage disorders. In the US, the Food and Drug Administration (FDA) granted Rare Pediatric Disease Designations (RPDD) for GM1 gangliosidosis, GM2 gangliosidosis, and NPC.
It also received Orphan Drug Designations (ODD) for GM1 gangliosidosis, GM2 gangliosidosis (including Sandhoff and Tay-Sachs diseases), and NPC. Additionally, the FDA granted Fast Track Designation and Investigational New Drug (IND) clearance for GM1 and GM2 gangliosidoses and NPC.
In Europe, the European Medicines Agency (EMA) awarded Orphan Medicinal Product Designation (OMPD) for GM1 and GM2 gangliosidoses.
In the UK, the Medicines and Healthcare Products Regulatory Agency (MHRA) granted an Innovation Passport for GM1 and GM2 gangliosidoses. These designations are intended to support the development and regulatory review of nizubaglustat for these conditions.
Capital utilisation
The funding will allow Azafaros to move forward with the development of its main product, nizubaglustat, which is planned to begin Phase 3 trials for Niemann-Pick disease Type C and GM1/GM2 gangliosidoses later this year.
The company also plans to use the financing to work on additional treatment areas.
Stefano Portolano, CEO of Azafaros, says, “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. We look forward to bringing nizubaglustat to patients.”
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