Azafaros is a clinical-stage company developing disease-modifying therapeutics for rare lysosomal storage disorders, leveraging science from Leiden University and Amsterdam UMC. Its lead asset, nizubaglustat, is an orally available, brain-penetrant small molecule with a dual mode of action targeting CNS-involved GM1/GM2 gangliosidoses and Niemann-Pick disease Type C (NPC). Nizubaglustat has received multiple regulatory designations, including Orphan Drug Designations in the US and EU, Rare Pediatric Disease Designations, Fast Track status in the US, and an Innovation Passport from the UK MHRA. The company plans to initiate two Phase 3 pivotal studies for nizubaglustat (NPC and GM1/GM2) later this year and intends to expand its pipeline into additional indications. Azafaros was founded in 2018 and is led by an experienced team focused on rapid development and commercialization for rare genetic diseases. The recently completed oversubscribed financing strengthens its financial position to execute these clinical plans. Azafaros develops orally available small‑molecule therapeutics (azasugars) aimed at disease modification in rare genetic metabolic disorders, initially targeting lysosomal storage diseases. The company holds an exclusive license from Leiden University and Amsterdam UMC to a library of novel patented compounds discovered by researchers including Professors Hans Aerts, Hermen Overkleeft and Stan van Boeckel. Its proprietary lead compound, AZ‑3102, is an oral azasugar that interferes with glycolipid metabolism and exerts a dual mode of action on key disease pathways. Azafaros says it is advancing the lead program toward first‑in‑man studies while expanding its discovery efforts to broaden the pipeline into other rare metabolic diseases. The Series A financing provides capital to build the organization and expand the executive team to support these development plans. No operating revenues or user metrics are disclosed in the article.

Total raised

$183M

Funding rounds

2

Latest round

Series B

Latest activity

May 2025