Azafaros Raises $146.9 Million to Advance Phase 3 Programs

Rare Daily Staff

Azafaros, a clinical-stage company focused on developing therapies for rare lysosomal storage disorders, completed a $146.9 million (€132 million) oversubscribed series B financing that will accelerate the development of its programs in Niemann-Pick disease Type C and GM1/GM2 gangliosidoses.

Jeito Capital and Forbion Growth co-led the round, with additional participation from Seroba, Pictet Group, and existing investors Forbion Ventures, Schroders Capital and BioGeneration Ventures (BGV).

Azafaros, founded in 2018 by BGV, leverages science from Leiden University and Amsterdam UMC. The company is developing a first-in-class, dual-acting drug candidate to offer new treatment options to patients with lysosomal storage disorders, a group of severe, rare genetic diseases that often cause progressive neurodegeneration and, in many cases, fatal outcomes.

GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) are lysosomal storage disorders caused by the accumulation of GM1 or GM2 gangliosides, in the central nervous system. This accumulation causes progressive and severe neurological impairment and premature death. These diseases mostly affect infants and children, and no disease-modifying treatments are currently available.

Niemann-Pick disease Type C is a progressive, life-limiting neurological lysosomal storage disorder caused by mutations in the NPC1 or NPC2 gene and aberrant endosomal-lysosomal trafficking, leading to the accumulation of various lipids, including gangliosides,   in the CNS. The onset of disease can occur throughout the lifespan of an affected individual, from prenatal life through adulthood.

Nizubaglustat is a small molecule, orally available, and brain-penetrant azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease Type C (NPC).

The U.S. Food and Drug Administration has granted the company’s experimental therapy nizubaglustat Rare Pediatric Disease designations and Orphan Drug designations, and Fast Track Designation for the treatment of Niemann-Pick disease Type C and GM1/GM2 gangliosidoses. The European Medicines Agency has granted nizubaglustat Orphan Medicinal Product Designation for the treatment of GM1 and GM2 gangliosidoses, and the UK Medicines and Healthcare Products Regulatory Agency has granted it Innovation Passport designation for the treatment of GM1 and GM2 gangliosidoses.

“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, CEO of 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. We look forward to bringing nizubaglustat to patients.”

Photo: Stefano Portolano, CEO of Azafaros