Benzinga | Aug 12, 2020 08:27AM EDT

Akari Therapeutics Announces End Of Meeting With FDA To Initiate Pivotal Phase III Study For Treatment Of Bullous Pemphigoid With Nomacopan

* Phase III randomized placebo-controlled study in moderate to severe bullous pemphigoid (BP) patients with a primary endpoint of complete disease remission on minimal oral corticosteroids (OCS) agreed to with the FDA.

* Treatment arms will be tapered to minimal OCS and an important secondary endpoint is reduction in cumulative OCS on nomacopan as high dose OCS are regarded as unsafe in this vulnerable patient population.

* BP Phase III trial expected to start H1 2021

* Nomacopan has been granted orphan drug designation by the FDA and the EMA for the treatment of BP

* Nomacopan has potential to replace long term steroid treatment (standard of care) in BP, which has multiple adverse effects in this elderly and frail population

NEW YORK and LONDON, Aug. 12, 2020 (GLOBE NEWSWIRE) -- 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 a successful End of Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA) regarding Akari's proposed pivotal Phase III program for the treatment of BP.

The FDA has agreed to a two-part pivotal trial with Part A and Part B having the same structure, duration, endpoints and target population of moderate and severe BP patients.

In the Phase III study, patients will be randomized to receive either nomacopan plus oral corticosteroids (OCS) or placebo plus OCS. 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 patients with BP, OCS could be tapered to the minimal level within six weeks. The goal of conventional OCS tapering is to achieve minimal therapy (prednisone <0.1mg/kg/day) within four to six months after initiation of treatment1. Patients will only have their OCS tapered if their disease continues to respond to treatment as the OCS dose is decreased.

Once patients are on minimal OCS plus either nomacopan or placebo, the primary endpoint will be achieved by those patients with complete disease remission for eight weeks or longer. The duration of the study is six months after which patients may be eligible to enter a separate one-year long-term safety study to provide at least six months of additional safety data.

Part A of the study is the same design as Part B but smaller and with the objective of comparing the Company's target dose (comparable to dosing used in the Company's hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA) and paroxysmal nocturnal hemoglobinuria (PNH) Phase III programs) with a lower dose of nomacopan and with placebo. Following Part A and discussion with the FDA, Part B will be conducted at the same trial sites using the optimal dose from Part A.

Clive Richardson, Chief Executive Officer of Akari Therapeutics, said, "Following our positive Phase II study, we are very pleased that the FDA has agreed with the pivotal study design and provided a clear pathway to a potential approval for nomacopan in patients with BP. Success in this study would also open up a range of other dermatological conditions with related pathology."

Russell P. Hall, M.D., Professor of Dermatology, Duke University School of Medicine, who attended Akari's EOP2 meeting, said, "These proposed studies are expected to provide the critical data needed to assess the efficacy of nomacopan in providing rapid control of the inflammation in the skin of patients with bullous pemphigoid and minimize the need for high dose systemic corticosteroids in this very vulnerable patient population."

1Feliciani et al (2015)