Azafaros

Associate Sponsor

Founded in 2018, Azafaros builds upon decades of groundbreaking research conducted by Professors Hans Aerts, Hermen Overkleeft, and Stan van Boeckel at Leiden University and the Academic Medical Center in the Netherlands. With a focus on addressing unmet needs in the field of rare diseases, Azafaros is at the forefront of innovation in small molecule therapies.

Our lead clinical-stage program, nizubaglustat, is a cutting-edge, orally available, brain-penetrant small molecule. Its unique dual mode of action offers the potential to treat a range of devastating lysosomal storage diseases, including GM1 and GM2 gangliosidoses, Niemann-Pick disease type C (NPC), and other metabolic disorders. In early studies, nizubaglustat demonstrated a favorable safety profile and robust target engagement in both plasma and cerebrospinal fluid (CSF) in healthy volunteers. It has since shown additional promise in a Phase 2 trial involving GM2 and NPC patients, confirming its good safety profile and offering early indications of clinical benefit.

Now poised for the next stage, Azafaros is preparing to initiate Phase 3 studies in 2025. This significant milestone underscores our commitment to bringing life-changing treatments to patients affected by these debilitating conditions.

Backed by a strong syndicate of leading investors, including Forbion, BioGeneration Ventures, BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital, Azafaros is uniquely positioned to bring life-changing therapies to patients who need them most.

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