On April 23, 2019, Akari Therapeutics, Plc (Nasdaq: AKTX), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where the complement and/or leukotriene systems are implicated, announced positive initial Phase II clinical data from the first three of bullous pemphigoid (BP) patients in an ongoing clinical trial.
Bullous pemphigoid is a severe orphan inflammatory skin disease currently treated primarily with steroids and immunosuppressants which bring with them well known side effects. Treatment response and steroid potency varies significantly based on the severity of the disease, although flares and relapses frequently occur.
In patients with bullous pemphigoid there is evidence that both terminal complement activation (C5) and the lipid mediator leukotriene B4 (LTB4) have a central role in driving the disease. Ex vivo data, from a recent study at Lubeck University, in BP patients showed a pronounced accumulation of LTB4 and C5 and its activation products in the inflamed skin of bullous pemphigoid disease patients.
The Phase II trial for up to nine mild-to-moderate bullous pemphigoid patients is a six-week open-label single-arm study evaluating safety and with the main efficacy measure the Bullous Pemphigoid Disease Area Index (BPDAI) a frequently used evaluation of the extent and severity of the disease.
Initial results from the first three patients showed that Nomacopan (Coversin), dosed daily subcutaneously, was well tolerated in three elderly patients (>65 years), and that there were no drug-related adverse events.