Akari, which focuses on developing therapies for treating orphan autoimmune and inflammatory diseases, said it had a positive Type B, pre-IND meeting with the FDA regarding its lead product candidate Coversin for pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy, or HSCT-TMA.
Type B meetings are routine meetings a sponsor of a drug has with the FDA at predefined endpoints. A pre-IND meeting is held between the company and the FDA ahead of the filing of an investigational new drug application, a means for securing legal exemption from the federal law that mandates an approved marketing application for the drug to be transported or distributed across state lines.
HSCT, commonly known as bone marrow transplantation, refers to the intravenous infusion of hematopoietic stem and progenitor cells in a bid to establish marrow and immune functions. TMA is a type of damage that can occur in blood capillaries in many vital organs, most commonly in the kidney and brain, that can impede blood flow.
Akari said it has finalized a framework for the trial design with the FDA, with the response of selected, clinically meaningful treatment variables to Coversin being taken as the primary endpoint in the proposed pivotal trial.
Why It’s Important
HSCT-TMA is an orphan condition that has an estimated fatality rate of over 80 percent in children, according to Akari.
Akari is evaluating Coversin in four indications: bullous pemphigoid, atopic keratoconjunctivitis, atypical hemolytic uremic syndrome and paroxysmal nocturnal hemoglobinuria.
Akari said it plans to commence a trial in Europe and U.S. in the fourth quarter.
Akari shares were ripping higher by 154.93 percent percent to $4.67 on over 1,200 times their average volume at the time of publication Wednesday.
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