- Thinly traded nano cap Akari Therapeutics (NASDAQ:AKTX +15.4%) is up out of the gate this morning following its announcement that the FDA has signed off on the endpoints for a pivotal study evaluating second-generation complement inhibitor Coversin for the treatment of pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA), a disorder with a mortality rate of more than 80%.
- The endpoint will be clinically meaningful treatment variables, including markers of complement activation and markers that are elevated in thrombotic microangiopathy like platelet count, red blood cell fragments, thrombocytopenia, elevated LDH (enzyme) and hypertension.
- The study should launch in Q4.