HSCT-TMA

Akari is currently conducting a pivotal Phase 3 clinical trial in pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA) expected to support a regulatory filing. 

• Akari has received the Orphan Drug, Fast Track and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for nomacopan in pediatric HSCT-TMA 
• The European Commission has granted orphan drug designation to nomacopan as a treatment in hematopoietic stem cell transplantation 
• The pediatric HSCT-TMA development program is advancing to the registrational portion of the Phase 3 clinical trial in children older than two years of age and is on track to begin enrollment by the end of 2023 

In addition to the pediatric indication, Akari is developing nomacopan for adult HSCT-TMA. 

• The adult HSCT-TMA development program has FDA Orphan Drug designation 
• Enrollment in the double-blind placebo-controlled clinical trial of nomacopan in adult HSCT-TMA is expected to begin enrollment in 2024 

Geographic Atrophy (GA)

Akari’s pipeline includes pre-clinical research of long-acting PAS-nomacopan in geographic atrophy (GA), a potentially blockbuster mass market indication. 

• The company has completed evaluation of long-acting PAS-nomacopan candidates and selected a single drug candidate to move forward into clinical trials for treatment of GA 
• Development remains on track for submission of an Investigational New Drug application to the FDA in the first half of 2024 and the start of clinical trials in the second half of 2024