Akari Therapeutics Announces Successful End-of-Phase II 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 IIItrial 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
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.
1Feliciani et al (2015)
Background on Bullous Pemphigoid (BP)
BP is a severe orphan autoimmune inflammatory blistering skin disease with no approved treatments in the
In BP patients there is evidence that both terminal complement activation (via complement component C5) and the lipid mediator leukotriene B4 (LTB4) have a central role in driving the disease. Ex vivo data in BP patients, published in the
About Akari Therapeutics
Akari is a biopharmaceutical company focused on developing inhibitors of acute and chronic inflammation, specifically for the treatment of rare and orphan diseases, in particular those where the complement (C5) or leukotriene (LTB4) systems, or both complement and leukotrienes together, play a primary role in disease progression. Akari's lead drug candidate, nomacopan (formerly known as Coversin), is a C5 complement inhibitor that also independently and specifically inhibits leukotriene B4 (LTB4) activity.
Cautionary Note Regarding Forward-Looking Statements
Certain statements in this press release constitute “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. You should not place undue reliance upon the Company’s forward looking statements. Except as required by law, the Company undertakes no obligation to revise or update any forward-looking statements in order to reflect any event or circumstance that may arise after the date of this press release. These forward-looking statements reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control. Such risks and uncertainties for our company include, but are not limited to: needs for additional capital to fund our operations, our ability to continue as a going concern; uncertainties of cash flows and inability to meet working capital needs; an inability or delay in obtaining required regulatory approvals for nomacopan and any other product candidates, which may result in unexpected cost expenditures; our ability to obtain orphan drug designation in additional indications; risks inherent in drug development in general; uncertainties in obtaining successful clinical results for nomacopan and any other product candidates and unexpected costs that may result therefrom; difficulties enrolling patients in our clinical trials; our ability to enter into collaborative, licensing, and other commercial relationships and on terms commercially reasonable to us; failure to realize any value of nomacopan and any other product candidates developed and being developed in light of inherent risks and difficulties involved in successfully bringing product candidates to market; inability to develop new product candidates and support existing product candidates; the approval by the FDA and EMA and any other similar foreign regulatory authorities of other competing or superior products brought to market; risks resulting from unforeseen side effects; risk that the market for nomacopan may not be as large as expected; risks associated with the impact of the outbreak of coronavirus; risks associated with the
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Source: Akari Therapeutics Plc