Akari Therapeutics announced that positive data from an ongoing phase 2 trial evaluating nomacopan for treating the rare skin disease bullous pemphigoid (BP) will be presented at the 28th European Academy of Dermatology and Venereology (EADV) Congress in Madrid, Spain. Treatment with nomacopan in the trial was well tolerated and resulted in rapid onset of clinical improvement in five of six patients.
The ongoing Phase II trial, which includes up to nine mild-to-moderate bullous pemphigoid patients (>55 years), 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.
- Four of the six patients were classified as at the upper limit of moderate BP. These patients, who showed only a minor improvement on mometasone prior to initiation on nomacopan, saw a mean 41% and 63% decline in BPDAI score at day 21 and 42, respectively, and a 59% and 68% decline in blister score at day 21 and 42, respectively.
- Of the two mild patients, one patient showed a 100% decline in blisters by Day 21, sustained to day 42, and a decline in BPDAI score of 55% and 45% by day 21 and day 42, respectively. The other patient showed no response either on nomacopan or when switched to rescue steroids at day 28. This patient entered the trial after disease relapse while on steroid treatment.
- Three of the patients who responded received mometasone steroids tapered to zero by day 21. Two other patients were on nomacopan only, with no steroids, and showed a similar beneficial response with a mean 39% and 79% drop in BPDAI activity at day 21 and day 42, respectively, and a 57% and 93% drop in blisters at day 21 and 42, respectively.
Nomacopan dosed daily subcutaneously was well tolerated and there were no reported drug-related serious adverse events.
Akari Therapeutics plans to meet with the FDA and EMA to discuss the design of a pivotal study, anticipated to begin in H2 2020.
Nomacopan was recently granted orphan drug designation by the FDA for the treatment of BP.
About bullous pemphigoid
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.
Nomacopan is a recombinant small protein (16,740 Da) that acts on complement component-C5, preventing release of C5a and formation of C5b–9 (also known as the membrane attack complex, or MAC), and independently also inhibits leukotriene B4, or LTB4, activity, both elements that are co-located as part of the immune/inflammatory response.