Benzinga | Oct 1, 2020 09:38AM EDT

Akari Therapeutics Reports Successful Scientific Advice Meeting with EMA For Nomacopan For Treatment Of Bullous Pemphigoid

Akari Therapeutics, Plc (NASDAQ:AKTX), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where the complement and/or leukotriene systems are implicated, announces completion of a scientific advice meeting with the European Medicines Agency (EMA) in advance of initiating a Phase III pivotal study of nomacopan for the treatment of bullous pemphigoid (BP). Following prior similar agreement from the U.S. Food and Drug Administration (FDA) at an End of Phase II meeting, the EMA has agreed to Akari's proposal for a Phase III randomized placebo-controlled study with nomacopan in patients with moderate to severe BP with a primary endpoint of disease remission on minimal oral corticosteroids (OCS). Akari believes the overlapping advice now received from both agencies allows the pivotal Phase III study design to satisfy both the FDA and EMA recommendations.

BP is a severe orphan autoimmune inflammatory blistering skin disease with no approved treatments in the U.S. and Europe with an approximately three-fold increase in mortality in the BP population treated with current standard of care. The prevalence of BP is estimated to be over 100,000 patients in the U.S. and Europe.

In the Phase III study, patients will be randomized to receive either nomacopan plus oral corticosteroids (OCS) or placebo plus OCS for six months. Following an initial stabilization phase, the steroids will be tapered according to disease response to a minimal level of OCS (< 0.1mg/kg/d prednisone or equivalent) which is considered safe. If disease response is rapid, as was seen in the nomacopan Phase II study in BP patients, OCS could be tapered to the minimal level within six weeks. Once patients are on minimal OCS plus either nomacopan or placebo, the primary endpoint will be achieved by those patients with complete disease remission on minimal OCS for eight weeks or longer during the 24-week treatment period. Patients will then enter an extension study to provide at least six months of additional data. An important secondary endpoint is reduction in cumulative OCS use on nomacopan as high dose OCS are regarded as unsafe in this vulnerable patient population.

Clive Richardson, Chief Executive Officer of Akari Therapeutics, said, "Following our successful FDA End of Phase II meeting, we are very pleased that the EMA has also agreed that we can proceed with our proposed pivotal Phase III study. The advice received from both agencies provides a clear pathway to a potential approval in Europe and the U.S. for nomacopan in patients with BP. We plan to initiate the study in both the U.S. and Europe during the first half of 2021."