Leiden-based Azafaros has successfully secured €132 million in an oversubscribed Series B financing round to accelerate development of its lead candidate nizubaglustat for rare lysosomal storage disorders. The funding will support two pivotal Phase 3 clinical programs scheduled to begin later this year.
The financing round was led by Jeito Capital and co-led by Forbion Growth, with additional participation from Seroba, Pictet Group, and existing investors Forbion Ventures, Schroders Capital, and BioGeneration Ventures (BGV).
Advancing Treatment for Devastating Rare Diseases
Nizubaglustat is being developed to treat Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses, rare genetic disorders that primarily affect children and cause progressive neurodegeneration. These lysosomal storage disorders currently have limited or no disease-modifying treatment options available.
"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, Chief Executive Officer at Azafaros. "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."
A Novel Dual-Acting Mechanism
Nizubaglustat is a small molecule, orally available and brain-penetrant azasugar with a unique dual mode of action. The compound has received significant regulatory support, including:
- Rare Pediatric Disease Designations for GM1/GM2 gangliosidoses and NPC
- Orphan Drug Designations in both the US and Europe
- Fast Track Designation from the FDA
- Innovation Passport from the UK Medicines and Healthcare Products Regulatory Agency
These designations highlight the significant unmet medical need and the potential impact of nizubaglustat on patient outcomes.
Addressing Critical Unmet Needs
GM1 and GM2 gangliosidoses (including Tay-Sachs and Sandhoff diseases) are characterized by the accumulation of gangliosides in the central nervous system, resulting in severe neurological impairment and premature death. Similarly, Niemann-Pick disease Type C is caused by mutations in the NPC1 or NPC2 gene, leading to abnormal lipid accumulation and progressive neurological deterioration.
Rachel Mears, Partner at Jeito Capital who will join Azafaros's Board of Directors, emphasized the potential impact: "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."
Building on Strong Scientific Foundations
Founded in 2018, Azafaros was built on scientific discoveries from Leiden University and Amsterdam University Medical Center. The company aims to develop a pipeline of disease-modifying therapeutics for rare genetic diseases.
"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," noted Audrey Cacaly, Principal at Forbion Growth, who will also join the company's Board of Directors.
Expanding Beyond Initial Indications
Beyond the two lead indications, the financing will enable Azafaros to expand its pipeline to address additional rare diseases. The company's approach challenges traditional development pathways to accelerate bringing new drugs to patients with rare diseases who urgently need treatment options.
Dr. Rafaèle Tordjman, Founder and CEO of Jeito Capital, highlighted the investment's alignment with their mission: "This investment reflects Jeito's commitment to accelerating the development of impactful therapies for patients with high unmet needs. Azafaros has the potential to develop new efficient, safe and tolerable therapeutic options for young patients suffering from progressive debilitating and even fatal rare metabolic disorders that generates very high expectations."
With the new funding secured, Azafaros is positioned to advance its clinical programs and potentially deliver transformative therapies to patients suffering from these devastating rare diseases.
