Akari Therapeutics announced that it achieved the primary and secondary endpoints in a phase 2 study evaluating nomacopan for the treatment of Bullous Pemphigoid. The company expects to meet with the FDA and the European Medicines Agency (EMA) to discuss a pivotal trial design in third quarter 2020.
The Phase II six-week open-label single-arm study enrolled 9 patients (7 with moderate severity, 2 mild). Patients entering the trial were permitted to use a low dose of mometasone topically to lesions for up to Day 21. The main efficacy endpoints were clinically significant improvements in the Bullous Pemphigoid Disease Area Index (BPDAI), which provides an objective measure of the area of skin affected by inflammatory skin lesions (blisters, erythema and urticaria).
Seven out of 9 patients showed a decrease by 4 or more points (deemed a minimal clinically important difference) in the BPDAI activity score between baseline (Day 1) and Day 42, while 2 patients, both with relapsing disease on admittance to the trial, were non responders. The mean BPDAI activity score over the period of the trial for the 7 of 9 responders showed more than a 20 point fall over the 42 day study and a decline in the mean BPDAI index by 29% at day 14, 70% at day 28 and 61% by day 42. One of the 7 responding patients experienced a flare in their disease after day 28, which is common in BP where patients are typically treated for 6 months or more with steroids to enable disease control. The two non- responders showed worsening disease with an increase in their BPDAI score. The combined mean decline in the BPDAI index for all 9 patients was approximately 40% at day 42. Of the 7 responders, 3 showed an 80%+ reduction in BPDAI score and 3 an approximately 40% reduction in BPDAI score within six weeks of starting nomacopan. The treatment was well tolerated.
About Bullous Pemphigoid
Bullous Pemphigoid (BP) is a severe orphan autoimmune inflammatory blistering skin disease with no approved treatments in the U.S. and Europe. This disease, most common in the elderly, is primarily treated with steroids and immunosuppressants for six months or more which bring with them deleterious side effects and an approximately three-fold increase in mortality in the BP treated population. The prevalence of BP is estimated to be over 100,000 patients in U.S. and Europe.
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.
Nomacopan is a C5 complement inhibitor that also independently and specifically inhibits leukotriene B4 (LTB4) activity. Nomacopan granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of BP in September 2019.