Azafaros develops therapeutic agents that target rare metabolic disorders through metabolic pathway modulation and advanced drug discovery techniques. The company provides effective treatment options for patients with unmet medical needs in this specialized healthcare segment.
Funding
$146.7M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
FCJCFounders
Product
Problem
Lysosomal storage disorders (LSDs) are a group of over 70 rare genetic diseases characterized by lysosomal dysfunction, leading to the accumulation of specific substances within cells. These disorders often result in progressive neurodegenerative conditions and affect multiple organ systems, with limited or no disease-modifying treatments available for many of them.
Solution
Azafaros is a clinical-stage biopharmaceutical company developing disease-modifying therapeutics for rare lysosomal storage disorders. Their lead asset, nizubaglustat, is an orally available azasugar designed to treat the central nervous system and interfere with the metabolism of glycosphingolipids. Nizubaglustat features a unique dual mode of action to address the underlying causes of diseases like GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC). The company's approach combines a deep understanding of rare genetic disease mechanisms with advanced drug discovery techniques to provide effective treatment options for patients with unmet medical needs.
Target Audience
The primary target audience includes patients suffering from rare lysosomal storage disorders, particularly GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC), as well as their families and the clinicians who treat them.
Features
- Orally available, brain-penetrant azasugar compound
- Dual mode of action targeting glycosphingolipid metabolism
- Potential treatment for GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC)
- Demonstrated a positive safety profile in Phase 2 clinical trials
- Observed preliminary improvements or stabilization of clinical endpoints in Phase 2 study
- Granted Orphan Drug Designation by the FDA and EMA for GM1 gangliosidosis
- Clinical Trial Application approved in multiple European countries for Phase 3 studies