Akari Therapeutics announces acceleration of Phase 3 trial of nomacopan

Akari Therapeutics announced the Phase 3 clinical trial of nomacopan in pediatric patients with hematopoietic stem cell transplant-related thrombotic microangiopathy, HSCT-TMA, is moving into Part B sooner than expected and the company is beginning the planning and design for the pivotal study in pediatric patients over 2 years of age. 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. Part A of the Phase 3 clinical trial of nomacopan in pediatric HSCT-TMA was designed to include three cohorts. 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 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. Akari participated in the FDA Model-Informed Drug Development program to refine the Akari PK/PD model suitability and doses for the Phase 3 clinical trial of nomacopan in pediatric HSCT-TMA. Clinical data from 38 subjects were included in the Akari PK/PD model, which was used to predict dosing for pediatric HSCT-TMA patients through 10,000 virtual patient simulations. These informed FDA MIDD interactions that confirmed PK/PD model suitability and doses selected for the nomacopan Phase 3 Part A clinical trial in pediatric HSCT-TMA. An expanded PK/PD model using data from 55 patients treated with nomacopan was reviewed in the recent Type C interaction with the FDA along with PK/PD data from the Part A study. "We made the important decision to not wait for Part A to be completed before we reached out to the FDA for Type C feedback on our next steps toward the pivotal study, an interaction which we had originally estimated would be mid-year 2023. This earlier feedback enabled the acceleration into the Part B clinical trial of nomacopan in pediatric HSCT-TMA," said Rachelle Jacques, President and CEO of Akari. "Our decision to initiate a new pipeline program to develop nomacopan as a potential treatment for adults with HSCT-TMA was also an important one and will support our pediatric program. The significant unmet need is shared among both children and adults with dismal outcomes and no approved treatment options."