Akari Therapeutics, plc (Nasdaq: AKTX) is a biotechnology company developing advanced therapies for autoimmune and inflammatory diseases. Akari’s lead asset, investigational nomacopan, is a bispecific recombinant inhibitor of complement C5 activation and leukotriene B4 (LTB4) activity.
Pediatric & Adult HSCT-TMA
Akari’s pipeline includes a Phase 3 clinical trial investigating nomacopan for pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA). Akari has been granted Orphan Drug, Fast Track and Rare Pediatric Disease designations from the FDA for nomacopan for the treatment of pediatric HSCT-TMA. With the Rare Pediatric Disease Designation, Akari is eligible to receive a Priority Review Voucher (PRV) at approval. Orphan drug designation also has been granted by the European Commission for nomacopan for the treatment of hematopoietic stem cell transplantation. In addition to pediatric HSCT-TMA, Akari is developing nomacopan in a Phase 3 clinical trial program for adult HSCT-TMA, a patient population that is 10 times the size of the pediatric population.
Geographic Atrophy (GA)
Akari’s pipeline also includes pre-clinical research of long-acting PAS-nomacopan in geographic atrophy (GA), a mass market. The company expects PAS-nomacopan pre-clinical research will be completed shortly and development will advance into clinical trials in 2024.