Azafaros' secures €132 million for rare disease treatment
Azafaros secures €132M to advance trials for rare neurological therapies, targeting lysosomal storage disorders with innovative treatments.
Published on May 14, 2025
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Azafaros, a clinical-stage biotech company, has successfully closed a €132 million funding round to advance its innovative therapies for rare lysosomal storage disorders. Led by Jeito Capital and co-led by Forbion Growth, this oversubscribed Series B financing will propel the Leiden-based company's lead compound, nizubaglustat, into late-stage trials later this year. Nizubaglustat has received Orphan Drug Designations and Fast Track status in the US and Europe.
The company is targeting lysosomal storage diseases (LSDs), a group of more than 50 rare genetic disorders characterized by enzyme deficiencies that lead to the accumulation of toxic materials in body cells and organs. These buildups arise due to the body's inability to break down certain substances, such as lipids and carbohydrates, causing severe cellular damage that affects various body parts. Treatments for LSDs, like those Azafaros is developing, focus on symptom management and can significantly influence the quality of life for those affected. While not curative, such therapies are crucial for reducing the progression of these debilitating conditions.
Advancing therapies
Azafaros' lead drug candidate, nizubaglustat, is poised to enter Phase 3 clinical trials targeting Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses. Nizubaglustat, a brain-penetrant azasugar, has already been awarded Orphan Drug Designations and Fast-track approval in the U.S. and Europe, reflecting its potential to meet significant unmet medical needs. Through these trials, Azafaros aims to verify the efficacy of nizubaglustat in modifying disease trajectory and improving patients' quality of life by mitigating the neurological impact of these rare disorders.
In tackling LSDs, Azafaros employs a strategic approach centered around its Natural History Study known as PRONTO (PROspective Neurological Disease Trajectory Study), aimed at understanding the disease progression among patients with GM1 and GM2 gangliosidoses. This study, now in its final stages after initiating in 2021, involves 31 participants across multiple countries, collecting crucial data to aid in the development of future treatment options and enhancing the clinical pathway for nizubaglustat.
Stefano Portolano, CEO at Azafaros, concluded: “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.”
