Akari Therapeutics Announces Successful End-of-Phase II Meeting With FDA to Initiate Pivotal Phase III Study for Treatment of...
August 12 2020 - 8:23AM
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, announces a successful End of Phase 2
(EOP2) meeting with the U.S. Food and Drug Administration (FDA)
regarding Akari’s proposed pivotal Phase III program for the
treatment of BP.
The FDA has agreed to a two-part pivotal trial with Part A and
Part B having the same structure, duration, endpoints and target
population of moderate and severe BP patients.
In the Phase III study, patients will be randomized to receive
either nomacopan plus oral corticosteroids (OCS) or placebo plus
OCS. Following an initial stabilization phase, the steroids will be
tapered according to disease response to a minimal level of OCS
(< 0.1mg/kg/d prednisone or equivalent) which is considered
safe. If disease response is rapid, as was seen in the nomacopan
Phase II study in patients with BP, OCS could be tapered to the
minimal level within six weeks. The goal of conventional OCS
tapering is to achieve minimal therapy (prednisone
<0.1mg/kg/day) within four to six months after initiation of
treatment1. Patients will only have their OCS tapered if their
disease continues to respond to treatment as the OCS dose is
decreased.
Once patients are on minimal OCS plus either nomacopan or
placebo, the primary endpoint will be achieved by those patients
with complete disease remission for eight weeks or longer. The
duration of the study is six months after which patients may be
eligible to enter a separate one-year long-term safety study to
provide at least six months of additional safety data.
Part A of the study is the same design as Part B but smaller and
with the objective of comparing the Company’s target dose
(comparable to dosing used in the Company’s hematopoietic stem cell
transplant-related thrombotic microangiopathy (HSCT-TMA) and
paroxysmal nocturnal hemoglobinuria (PNH) Phase III programs) with
a lower dose of nomacopan and with placebo. Following Part A and
discussion with the FDA, Part B will be conducted at the same trial
sites using the optimal dose from Part A.
Clive Richardson, Chief Executive Officer of Akari Therapeutics,
said, “Following our positive Phase II study, we are very pleased
that the FDA has agreed with the pivotal study design and provided
a clear pathway to a potential approval for nomacopan in patients
with BP. Success in this study would also open up a range of other
dermatological conditions with related pathology.”
Russell P. Hall, M.D., Professor of Dermatology, Duke University
School of Medicine, who attended Akari’s EOP2 meeting, said, “These
proposed studies are expected to provide the critical data needed
to assess the efficacy of nomacopan in providing rapid control of
the inflammation in the skin of patients with bullous pemphigoid
and minimize the need for high dose systemic corticosteroids in
this very vulnerable patient population.”
1Feliciani et al (2015)
Background on Bullous Pemphigoid (BP)
BP is a severe orphan autoimmune inflammatory blistering skin
disease with no approved treatments in the U.S. and Europe. This
chronic disease may last several years in the absence of treatment
and has a tendency to relapse. BP is most common in the elderly and
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.
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.
This underlies the rationale for treatment with nomacopan which is
a unique bifunctional inhibitor of both C5 and LTB4 and a range of
downstream cytokines. In addition to BP, the Company believes this
unique mode of action underpins the activity of nomacopan across
the Company’s other target conditions – TMA-HSCT, COVID pneumonia
and ophthalmology.
About Akari Therapeutics 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. Akari's lead
drug candidate, nomacopan (formerly known as Coversin), is a C5
complement inhibitor that also independently and specifically
inhibits leukotriene B4 (LTB4) activity.
Cautionary Note Regarding Forward-Looking
Statements Certain statements in this press release
constitute “forward-looking statements” within the meaning of the
Private Securities Litigation Reform Act of 1995. You should not
place undue reliance upon the Company’s forward looking statements.
Except as required by law, the Company undertakes no obligation to
revise or update any forward-looking statements in order to reflect
any event or circumstance that may arise after the date of this
press release. These forward-looking statements reflect our current
views about our plans, intentions, expectations, strategies and
prospects, which are based on the information currently available
to us and on assumptions we have made. Although we believe that our
plans, intentions, expectations, strategies and prospects as
reflected in or suggested by those forward-looking statements are
reasonable, we can give no assurance that the plans, intentions,
expectations or strategies will be attained or achieved.
Furthermore, actual results may differ materially from those
described in the forward-looking statements and will be affected by
a variety of risks and factors that are beyond our control. Such
risks and uncertainties for our company include, but are not
limited to: needs for additional capital to fund our operations,
our ability to continue as a going concern; uncertainties of cash
flows and inability to meet working capital needs; an inability or
delay in obtaining required regulatory approvals for nomacopan and
any other product candidates, which may result in unexpected cost
expenditures; our ability to obtain orphan drug designation in
additional indications; risks inherent in drug development in
general; uncertainties in obtaining successful clinical results for
nomacopan and any other product candidates and unexpected costs
that may result therefrom; difficulties enrolling patients in our
clinical trials; our ability to enter into collaborative,
licensing, and other commercial relationships and on terms
commercially reasonable to us; failure to realize any value of
nomacopan and any other product candidates developed and being
developed in light of inherent risks and difficulties involved in
successfully bringing product candidates to market; inability to
develop new product candidates and support existing product
candidates; the approval by the FDA and EMA and any other similar
foreign regulatory authorities of other competing or superior
products brought to market; risks resulting from unforeseen side
effects; risk that the market for nomacopan may not be as large as
expected; risks associated with the impact of the outbreak of
coronavirus; risks associated with the SEC investigation; inability
to obtain, maintain and enforce patents and other intellectual
property rights or the unexpected costs associated with such
enforcement or litigation; inability to obtain and maintain
commercial manufacturing arrangements with third party
manufacturers or establish commercial scale manufacturing
capabilities; the inability to timely source adequate supply of our
active pharmaceutical ingredients from third party manufacturers on
whom the company depends; unexpected cost increases and pricing
pressures and risks and other risk factors detailed in our public
filings with the U.S. Securities and Exchange Commission, including
our most recently filed Annual Report on Form 20-F filed with the
SEC. Except as otherwise noted, these forward-looking statements
speak only as of the date of this press release and we undertake no
obligation to update or revise any of these statements to reflect
events or circumstances occurring after this press release. We
caution investors not to place considerable reliance on the
forward-looking statements contained in this press release.
Investor Contact:
Peter Vozzo Westwicke +1 (443) 213-0505
peter.vozzo@westwicke.com
Media Contact:
Sukaina Virji / Lizzie Seeley Consilium Strategic
Communications +44 (0)20 3709 5700Akari@consilium-comms.com
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