Acceleration of phase III clinial trial of nomacopan to treat pediatric HSCT-TMA into pivotal part B and addition of new pipeline programme

Akari also announces it has added a new pipeline program that will develop nomacopan as a potential treatment for adult HSCT-TMA, which will include a study that is supportive of the pediatric program. Study enrollment for the adult program is expected in 2024. Nomacopan is a novel bispecific inhibitor of complement C5 and leukotriene B4 (LTB4) in clinical development for pediatric and adult HSCT-TMA. Long-acting PAS-nomacopan is in pre-clinical development for geographic atrophy secondary to dry age-related macular degeneration.

Part A of the Phase III clinical trial of nomacopan in pediatric HSCT-TMA was designed to include three cohorts: ages 0.5 to <2 years, between 2 to 9 years and more than 9 to less than 18 years. akari enrolled patients in the two older age groups and, based on feedback from the fda, the pk pd data from these patients from the part a study are consistent with the predictions derived from akari’s latest pk pd model. since enrollment for the youngest age cohort (ages 0.5 to><2 years) in the part a pediatric study is not completed akari is keeping the study open for this youngest group of pediatric patients while advancing the pivotal part b study of nomacopan in the older pediatric patients.