Dutch Biotech Azafaros Secures €132M Series B Funding for Late-Stage Trials of Brain-Penetrant Drug

Azafaros, a Dutch biotech company, has successfully raised €132 million ($147 million) in a Series B funding round to advance its rare neurological disease drug, nizubaglustat, through two late-stage clinical trials. The substantial investment underscores growing interest in treatments for lysosomal storage disorders and highlights the potential of brain-penetrant therapies in addressing unmet medical needs.

Funding Details and Investor Support

The Series B round was led by Jeito Capital and Forbion Growth, with participation from existing investors BioGeneration Ventures, Forbion Ventures, and Schroders Capital. This latest funding follows a €25 million ($27.5 million) Series A investment in 2020, bringing Azafaros' total raised capital to over €157 million since its founding in 2018.

Stefano Portolano, CEO of Azafaros, expressed enthusiasm about the fundraise, stating, "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."

Nizubaglustat: A Promising Candidate for Rare Brain Disorders

Nizubaglustat, the company's lead candidate, is a brain-penetrant azasugar developed to treat rare lysosomal storage disorders affecting the brain. The drug is specifically targeting GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC). These conditions are part of a group of about 50 rare inherited metabolic disorders resulting from defects in lysosomal function.

The technology behind nizubaglustat originated from research conducted at Leiden University and Amsterdam UMC in the Netherlands. Its development represents a significant step forward in addressing the challenges of treating neurological lysosomal storage disorders, which often lack effective therapies due to the difficulty of penetrating the blood-brain barrier.

Clinical Development and Future Plans

With the new funding, Azafaros plans to initiate Phase 3 trials of nizubaglustat in both GM1 and GM2 gangliosidoses and NPC. These late-stage studies will be crucial in determining the drug's efficacy and safety in larger patient populations.

In addition to advancing nizubaglustat, the company intends to use part of the funds to expand its pipeline to other indications. While specific details about these additional programs have not been disclosed, this expansion suggests Azafaros' commitment to addressing multiple areas of unmet need in rare neurological disorders.