Company profile: Azafaros
1.1 - Company Overview
Company description
- Provider of oral azasugar-based therapeutic agents targeting rare lysosomal storage disorders, including the investigational small molecule nizubaglustat for GM1 and GM2 gangliosidoses and Niemann-Pick disease type C. Offers the PRONTO natural history study and the RAINBOW clinical study evaluating nizubaglustat, plus an expanded access policy for AZ-3102 limited to clinical trial participation.
Products and services
- Nizubaglustat: An orally available small molecule therapeutic engineered to treat rare lysosomal storage diseases, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C, by reducing harmful lipid accumulation
- RAINBOW study: A clinical study evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of nizubaglustat in patients with GM2 gangliosidosis or Niemann-Pick disease type C
- PRONTO study: A prospective longitudinal study aimed at understanding the natural progression of GM1 and GM2 gangliosidoses in children to support future clinical trials
Key contacts
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Financial details
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1.2 - Competitors and similar companies to Azafaros
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ProFactor Pharma
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- Rationale: This competitor operates in a highly similar market segment with overlapping features.
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Brabant Pharma
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Engage Therapeutics
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Website
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Imara
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2.M&A buyers
2.1 Potential strategic acquirers in the sector
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2.2 - Strategic buyer groups for Azafaros
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3. Investors and private equity firms
3.1 - Buyout funds investing in similar companies to Azafaros
2.2 - Growth funds investing in similar companies to Azafaros
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4 - Top valuation comps for Azafaros
4.2 - Public trading comparable groups for Azafaros
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