Paroxysmal Nocturnal Hemoglobinuria (PNH)
- PNH affects between 1 and 5 people per million.
- The annual cost of current complement-targeted therapy for PNH exceeds $400,000 per patient.
- Anti-C5 therapy is not satisfactory in all PNH patients, and the only curative treatment for PNH, which is bone marrow transplantation, has been associated with complications; allogeneic bone marrow transplantation has been associated with significant morbidity and mortality.
Age-Related Macular Degeneration (AMD)
- Around the world, there are 3 million people who have been diagnosed with AMD in at least one eye, while this group is on course to doubling by 2020.
- In the Western world, macular degeneration is the #1 cause for vision loss in those over 65 years old, and, in other parts of the world, the prevalence for this disease is going up.
- Local inhibition of complement activation is thought to hold promise for the treatment of both dry and wet AMD. Compstatin was the first complement-specific candidate entering clinical trials for this.
- Over 500,000 individuals need to undergo kidney replacement therapy for end-stage renal disease (ERSD) in the United States, but only 3.5% are able to undergo transplantation. The majority of patients rely on hemodialysis.
- 35% of those undergoing hemodialysis have a 5-year survival rate.
- Even though hemodialysis techniques and materials have been improved, the mortality rate for patients treated with hemodialysis is around 16%.
- In 2009, the cost for treating ESRD patients in the U.S. amounted to over $40 billion in public and private funds, according to National Kidney and Urologic Diseases Information Clearinghouse (NKUDIC).
Ischemia/Reperfusion (I/R) Injuries and Transplantation
- Ischemic injury affects 1.3 million people in the U.S. each year (in the form of myocardial infarction, stroke, and other thrombotic events).
- Complement activation in I/R injury as it takes place during myocardial infarction was first described more than 40 years ago.
- Experimental studies in various organ systems and clinical trials have pointed to the inhibition of the complement system as a potential means for decreasing harmful tissue injury in I/R conditions.
- Studies have indicated that complement proteins have an important role in organ damage after transplantation (via modulation of the activation of the adaptive immune response and ischemia/reperfusion).
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