Akari Therapeutics, a late-stage biotechnology company, has provided updates on the development of its drug nomacopan for the treatment of pediatric and adult hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA). The company is making progress towards initiating a Phase 3 study of nomacopan in pediatric HSCT-TMA by the end of 2023. Furthermore, Akari plans to commence enrollment for a Phase 3 clinical trial of nomacopan in adult HSCT-TMA in 2024.
HSCT-TMA is a rare complication that can occur following stem cell transplantation. Currently, there are no approved treatments available for HSCT-TMA, and patients with severe cases face a high mortality rate. Akari's goal is to provide an approved treatment option for both children and adults dealing with this condition.
To promote consistent diagnostic criteria for HSCT-TMA, an international panel of experts has published consensus guidelines. These guidelines advocate for early screening and diagnosis of the condition, which can potentially save lives by preventing irreversible dysfunction in multiple organs.