The company announced results at the European Academy of Dermatology and Venereology Congress in Madrid, Spain.
Akari Therapeutics (NASDAQ:AKTX) has announced positive data from its ongoing bullous pemphigoid (BP) Phase 2 clinical trial at the European Academy of Dermatology and Venereology (EADV) Congress in Spain.
As quoted in the press release:
BP is a severe orphan autoimmune inflammatory blistering skin disease with no approved treatments in the U.S. and Europe. The disease is primarily treated with steroids and immunosuppressants which bring with them well known side effects and an approximately three-fold increase in mortality in the elderly BP patient population.
“The positive safety profile and rapid response to treatment with nomacopan, even with no steroid co-medication, supports a treatment strategy that could see steroid use rapidly reduced in BP patients,” commented Clive Richardson, Chief Executive Officer of Akari Therapeutics. “As a result of this encouraging data, we now plan to develop our pivotal study design in conjunction with the FDA and EMA. We intend to begin the pivotal study in H2 2020, positioning nomacopan as potentially a first-in-class treatment for this severe and debilitating disease.”
In patients with BP 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, recently published in the August 2019 edition of JCI Insight [LINK] showed a pronounced accumulation of LTB4 and C5 and its activation products in the inflamed skin of BP disease patients underlying the rationale for treatment with nomacopan which is a bifunctional inhibitor of both C5 and LTB4.
The Phase II trial, which includes up to nine mild-to-moderate bullous pemphigoid patients, is a six-week (42 days of nomacopan dosing) open-label single-arm study evaluating safety and efficacy. Patients entering the trial were typically on the topical steroid mometasone which was stopped by day 21. The main efficacy measure is the Bullous Pemphigoid Disease Area Index (BPDAI) which along with blistering is a frequently used evaluation of the extent and severity of this skin disease.