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 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 patients
The phase 2 clinical trial has completed recruitment and achieved its primary endpoint of no drug-related serious adverse events and main secondary efficacy endpoints with 7 of the 9 treated patients showing a rapid and clinically significant reduction in Bullous Pemphigoid Disease Area Index (BPDAI) score. Nomacopan has the potential to replace long term steroid treatment (standard of care), which has multiple adverse effects in this elderly and frail population. Nomacopan was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of BP in September 2019.
The graph below shows the mean BPDAI activity score over the period of the trial for the 7 of 9 responders with 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.