Akari Data Back Trial Expansion in Orphan Autoinflammatory Diseases

• Coversin, a first-in-class combined complement C5 and leukotriene B4 (LTB4) inhibitor, is currently being developed to treat four orphan diseases:
  • Thrombotic microangiopathies (TMAs)
  • Bullous pemphigoid (BP)
  • Atypical keratoconjunctivits (AKC)
  • Paroxysmal nocturnal hemoglobinuriua (PNH)
• All four clinical programs are currently in Phase I/II, Phase II or Phase III, with initial data in BP and AKC expected in the first quarter of 2019
• New data in two post-transplant TMA pediatric patients treated with Coversin on a named patient basis, presented at the Inborn Errors Working Party (IEWP) meeting, Leiden, The Netherlands, on September 30, 2018, showed in both patients that signs of TMA (red blood cell fragments, thrombocytopaenia, elevated LDH, and hypertension) resolved following treatment with Coversin

Akari Therapeutics, Plc (NASDAQ:AKTX), a biopharmaceutical company focused on the development and commercialization of innovative therapeutics to treat orphan autoimmune and inflammatory diseases where complement and or leukotriene systems are implicated, today announced new clinical data in post-transplant thrombotic microangiopathies.

“This new data represents an exciting development and helps underpin our strategy of using Coversin to target a range of orphan inflammatory diseases with unmet need. These early results from patients with TMA reinforce our belief that Coversin may prove to be effective in diseases where terminal complement activation is critical and dysfunction of LTB4 may also play a role,” said Clive Richardson interim CEO of Akari Therapeutics. “TMAs are challenging conditions in which localized microvascular injury plays a crucial role. We believe that in several diseases, both complement and LTB4 may be involved in the localized pathology. If so, Coversin could provide a novel treatment opportunity for such indications.”

At the annual meeting of the IEWP of the European Society for Blood and Marrow Transplantation (EBMT) in Leiden, The Netherlands, the results from two pediatric patients with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) treated with Coversin were reported by Dr. Robert Chiesa, the treating clinician at Great Ormond Street Hospital (GOSH), London, UK. These patients were treated on a named patient basis at GOSH and Birmingham Children’s Hospital.

HSCT-TMA is a complication in up to 30% of patients following bone marrow transplantation. In severe cases, pediatric TMA carries a mortality of more than 80%¹. Currently, HSCT-TMA has no approved treatment. In both of the pediatric patients treated with Coversin, there was rapid reduction of the markers of complement activation and normalization of markers that are elevated in TMA: platelet count, red blood cell fragments, thrombocytopaenia, elevated LDH, and hypertension. The child treated at GOSH made a complete recovery and Coversin was discontinued after seven weeks. In the second patient, despite resolution of the TMA markers, the patient subsequently died of lung damage which was considered unrelated to treatment with Coversin.

https://www.nasdaq.com/press-release/akari-therapeutics-announces-new-clinical-data-in-posttransplant-thrombotic-microangiopathies-data-20180930-00005