Akari Therapeutics open IND with FDA for bullous pemphigoid treatment

pharmafile | April 13, 2021 | News story | Manufacturing and Production FDA 

Akari Therapeutics have opened an investigational new drug application (IND) with the FDA for its multicentre Phase III study with nomacopan, for the treatment of moderate and severe bullous pemphigoid (BP).

Akari has been granted orphan drug designation for nomacopan for the treatment of BP by both the FDA and the EMA, and sites for the Phase III study are expected to open by mid-2021, subject to the ongoing impact of COVID-19.

Akari is a biopharmaceutical company focused on developing inhibitors of acute and chronic inflammation, specifically for the treatment of rare and orphan diseases, in particular those where the complement (C5) or leukotriene (LTB4) systems, or both complement and leukotrienes together, play a primary role in disease progression.

Clive Richardson, Chief Executive Officer of Akari Therapeutics, said: “With the BP IND now open we look forward to starting the pivotal Phase III study of nomacopan in patients with this severe dermatological condition, for which there is no specific approved treatment in the US or Europe.

“Success in BP could also open up a range of other severe dermatological conditions for treatment with nomacopan where C5 and LTB4 are implicated including hidradenitis suppurativa, epidermolysis bullosa acquisita and mucous membrane pemphigoid.”

BP is a rare, autoimmune, chronic skin disorder characterised by blistering, urticarial lesions (hives) and itching. Less commonly, these blisters can involve the mucous membranes including the eyes, oral mucosa, oesophagus and genital mucosa. It typically presents in older adults as a generalised intensely itchy blistering skin condition, and can last many years in the absence of treatment.

BP is primarily treated with potent oral steroids for six months or more, which often have deleterious side effects, and a three-fold or larger increase in mortality. The prevalence of BP is estimated to be approximately 120,000 patients in US and EU, with moderate and severe patients making up around three quarters of the patient population.

Evidence from studies conducted with nomacopan indicate that nomacopan’s ability to inhibit C5 and LTB4 gives it a unique potential therapeutic advantage which is upstream of other approaches, such as cytokine inhibitors. For instance, a pre-clinical model from Sezin et al in 2019 the absolute body surface area affected by blisters was reduced by approximately 50% by inhibiting LTB4 alone but by approximately 80% by inhibiting both LTB4 and C5.

Akari’s Phase III trial design, which is a randomised placebo-controlled study with nomacopan in patients with moderate to severe BP with a primary endpoint of disease remission on minimal oral corticosteroids, was based on the Company’s successful end-of-Phase II meetings held last year with both the FDA and the EMA.

Kat Jenkins

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