Akari Therapeutics Announces First Patient to Complete Course of Treatment in the Phase 3 Part A Clinical Trial

Akari Therapeutics, Plc recently announced a patient has completed the course of investigational nomacopan treatment in the open-label, multi-center Phase 3 Part A clinical trial in pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA). Nomacopan is a bispecific recombinant inhibitor of complement C5 and leukotriene B4 (LTB4).

Three patients with severe (nephrotic range proteinuria and elevated soluble C5b-9) HSCT-TMA have been enrolled in the clinical trial. One patient completed more than 60 days of nomacopan treatment and subsequently was discharged from the hospital. Another patient died from multi-organ failure unrelated to nomacopan treatment. Dosing has begun in the third patient.

“This is promising news for children and families facing hematopoietic stem cell transplant-related TMAs who have unmet needs that are significant and urgent because there are no approved treatment options,” said Rachelle Jacques, President and CEO of Akari Therapeutics. “Recruitment into a study of treatment for a rare and emergent complication of stem cell transplants in children has inherent challenges, and it is testament to the passion and commitment of everyone involved that this important Phase 3 clinical trial is progressing on behalf of patients and their families.”

Nomacopan was granted Orphan Drug and Fast Track designations by the US FDA for pediatric HSCT-TMA. Data from the Phase 3 Part A study of nomacopan in HSCT-TMA will inform the pivotal Phase 3 Part B study that will be the basis for potential regulatory submissions in the US and Europe.

The 6-year-old patient who was discharged was treated at a clinical trial site in Manchester, England, by investigator Rob Wynn, MD. “Thrombotic microangiopathy following a stem cell transplant procedure is a rare but devastating complication made even more tragic because there are currently no approved treatments,” said Professor Rob Wynn, of Royal Manchester Children’s Hospital, part of Manchester University NHS Foundation Trust. “As we advance this important clinical trial and offer treatment to children in Manchester where formerly there was none, we are bringing new hope to families who are in desperate need, and to other clinicians who very much want to offer a treatment option.”

Thrombotic microangiopathy following a stem cell transplant procedure is a rare but serious complication of HSCT that appears to involve complement activation, inflammation, tissue hypoxia and blood clots, leading to progressive organ damage and death. The mortality rate in patients who develop severe transplant-related TMAs is 80%. Currently, there are no approved treatment options in the US or Europe.

Sites are open and recruiting in the US, UK, and Poland for the Phase 3 Part A clinical trial of investigational nomacopan in pediatric patients who have undergone allogeneic or autologous HSCT and develop HSCT-TMA within a year of transplant. Patient dosing is underway in the multi-center, open-label study that has a recruitment goal of seven pediatric patients over 6 months old.

The primary study endpoints are either independence of red blood cell transfusion or urine protein creatinine ratio of 2 mg/mg maintained over 28 days immediately prior to any scheduled clinical visit up to Week 24. According to the study protocol, patients may discontinue therapy sooner than 24 weeks, if one, or both, of the primary endpoint components has been met and the treating clinician determines there is no longer a need for continued treatment with nomacopan. Patients who have achieved the primary endpoint and are no longer receiving nomacopan will have a follow-up clinic visit 30 days after the last dose, at 24 weeks and for long-term follow-up at one and two years.

Akari Therapeutics, plc is a biotechnology company focused on developing advanced therapies for autoimmune and inflammatory diseases. Akari’s lead asset, investigational nomacopan, is a bispecific recombinant inhibitor of C5 complement activation and leukotriene B4 (LTB4) activity. The Akari pipeline includes two late-stage programs for bullous pemphigoid (BP) and thrombotic microangiopathy (TMA), as well as earlier stage research and development programs in eye and lung diseases with significant unmet need. For more information, visit akaritx.com.