Singular Focus
- Orphan and ultra-orphan diseases with limited therapeutic options
- Medicines with high therapeutic value
- Emphasis on extremely small patient populations and pediatric indications
Commercial & Mid-to-Late Stage Development
- Orphan medicine product repositioning – Leverage underutilized medicines
- Fund only development projects with established proof of concept, no basic research or “discovery”
- Strong partnerships with academic researchers, government agencies (NIH) and early phase development companies
Business Model Based on Enabling Others
- Unique business model pairs a core management team of orphan disease experts with an extensive network of academic researchers, specialized consultants, 3rd party service providers and contractors
- We enable the development of new medicines using a non-conventional infrastructure
Patient Focus
- “Patient First – Profit First” paradigm
- Strong patient assistance – No patient goes without our medicines
- Global Scope, Global Commercialization – in all countries with Orphan Legislation and “named patient” access programs