Indication: Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS), Generalized Myasthenia Gravis (gMG), NMOSD
Fit Score: 9.5 | Ada Surface Ability: 9 | Company Motivation: 10
Market Opportunity
USA Addressable Undiagnosed: 4,693 patients
DACH Addressable: 998 patients
ROW Addressable: 5,400 patients
Patient Finder Fee (USA): $40,000/patient/year
Ada Revenue Opportunity (USA):
- 1% penetration: $1.88M/year
- 5% penetration: $9.4M/year
Clinical Profile
Underdiagnosis Rate: 70% (only 30% of diagnosed PNH patients receive complement inhibitors)
Key Symptoms: Dark urine (PNH), acute kidney injury triad (aHUS), muscle weakness patterns (gMG)
Diagnostic Delay: PNH - years; aHUS - acute but delayed complement inhibitor initiation; gMG - 3-5 years to identify refractory cases
Treatment Duration: Lifelong complement inhibition
Commercial Context
Annual Revenue: ~$10B combined rare disease portfolio (Ultomiris Q4 2024: $1.1B, +33% YoY)
Competitive Pressure: High - Biosimilar eculizumab emerging 2026+, oral alternatives (iptacopan)
Patent Expiry: Soliris - LOE 2025-2026; Ultomiris - protected through 2030+
Pitch Hook: "5,000+ undiagnosed PNH, aHUS, and refractory gMG patients in the US are experiencing life-threatening complications - 70% never reach diagnosis. Ada can surface these ultra-rare patients when they search for fatigue, dark urine, or muscle weakness, generating $3-18M annually while protecting Ultomiris market share ahead of biosimilar competition."