Akari Therapeutics Gets Regulatory Approval to Conduct Phase 2 Study in Paroxysmal Nocturnal Hemoglobinuria

Akari Therapeutics

disclosed Wednesday that the UK Medicines & Healthcare products Regulatory Agency (MHRA) has given its approval to conduct a second stage study in patients with paroxysmal nocturnal hemoglobinuria (PNH). According to the company, Coversin is a second-generation complement inhibitor, which acts on complement component-C5 and curbs the release of C5a apart from the formation of C5b-9, which is also called as the membrane attack complex or MAC. Akari said that C5 inhibition was getting more importance in a range of rare autoimmune diseases in connection with the dysregulation of the complement component of the immune system. That included paroxysmal nocturnal hemoglobinuria (PNH), atypical Hemolytic Uremic Syndrome (aHUS), and Guillain Barré syndrome (GBS). The company's CEO, Gur Roshwalb, commented, "We are very excited to initiate this study and recruit patients. We believe the data from this Phase 2 study in PNH will further the positive results previously reported from our Phase 2 eculizumab resistance study, and our Phase Ia and ongoing Phase Ib studies, and demonstrate the clinical benefit and potential competitive advantages of Coversin." Akari said that the second stage open label trial would study Coversin for 90 days in maximum of 10 patients with PNH who are not getting any other complement therapy. The company indicated that the primary endpoint of the study is the reduction in Lactate Dehydrogenase (LDH), an important blood marker of hemolysis, at day 28. The stock lost 1.23 percent on Tuesday.