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 that it achieved the primary and
secondary endpoints for nomacopan in its fully recruited Phase II
trial in BP and is planning to discuss Phase III pivotal study
designs with the FDA and EMA.
“The role of both the leukotriene and complement pathways has
been well documented in BP and the rapid clinical response and good
safety profile seen in this phase II study was extremely positive
given nomacopan’s unique bi functional inhibition of both C5 and
LTB4,” commented Clive Richardson, Chief Executive Officer of Akari
Therapeutics. “Our next steps are to work closely with U.S. and
European regulators in order to secure a clear path forward to a
pivotal trial for this severe orphan disease which has significant
unmet clinical need and is a potential gateway to other
dermatological conditions.”
Christian Sadik, M.D., lead investigator from the Department of
Dermatology, University of Lubeck, Germany, commented, “Clinicians
continue to seek an effective treatment for BP that can be
administered in outpatient settings and minimize the use of
steroids, which often cause significant clinical complications in
this at risk population. I am therefore delighted by the promising
results of Akari’s Phase II trial in BP which shows rapid efficacy,
similar to potent steroids, but with the advantage of being well
tolerated, and possessing a good safety profile.“
Summary of Topline Results of Phase II Study
The fully recruited Phase II trial of nomacopan for treatment of
mild-to-moderate BP was a six-week open-label single-arm study
evaluating safety and efficacy in 9 patients (7 with moderate
severity, 2 mild). Patients entering the trial were at their choice
permitted to use a low dose of mometasone¹ topically to
lesions for up to Day 21 as this was the first study of nomacopan
in BP patients.
The trial achieved the main efficacy endpoints, which were
clinically significant improvements in the Bullous Pemphigoid
Disease Area Index (BPDAI) which provides an objective measure of
the area of skin affected by inflammatory skin lesions (blisters,
erythema and urticaria). Seven out of 9 patients showed a decrease
by 4 or more points (deemed a minimal clinically important
difference²) in the BPDAI activity score between baseline (Day 1)
and Day 42, while 2 patients, both with relapsing disease on
admittance to the trial, were non responders. 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.
In the graph of mean BPDAI activity for the 7 responding
patients, one patient experienced a flare in their disease after
day 28, which is common in BP where patients are typically treated
for 6 months or more with steroids to enable disease control. The
two non responders showed worsening disease with an increase in
their BPDAI score. The combined mean decline in the BPDAI index for
all 9 patients was approximately 40% at day 42.
Improvement in BPDAI
Index: https://www.globenewswire.com/NewsRoom/AttachmentNg/543b002a-330b-4ba2-adfc-38243356c5ce
Note: Nomacopan dosing initiated Day 1. The BPDAI activity score
used above is a composite objective measure of the area of skin
affected by blisters/erosions, cutaneous urticaria/erythema,
mucosal blisters/erosions.
Of the 7 responders, 3 showed an 80%+ reduction in BPDAI score
and 3 an approximately 40% reduction in BPDAI score within six
weeks of starting nomacopan. For the responders, the response on
nomacopan was rapid and is similar to what is seen with patients
treated on potent oral steroids which is standard of care in the
U.S. and parts of Europe for moderate to severe patients.
Similarly, under typical standard of care around 20% of BP patients
are considered non-responders³.
Nomacopan, dosed daily by subcutaneous injection, was well
tolerated in this frail BP patient population with multiple
comorbidities. The primary endpoint was the proportion of
participants reporting grade 3, 4 and 5 adverse events, which are
related/possibly related to nomacopan. None of the 9 patients
reported any grade 3, 4 and 5 treatment related adverse events. The
safety profile in BP mirrors over 30 cumulative patient-years of
data from patients treated with nomacopan across all conditions in
clinical development where the drug has proven well tolerated with
no drug-related serious adverse events have been observed to
date.
A summary of the topline Phase II results of nomacopan for the
treatment of BP is available under Presentations in the Investor
Relations section of the Company’s website at www.akaritx.com. The
Company expects to report full data from the Phase II study at the
European Academy of Dermatology and Venereology (EADV) congress in
October 2020.
- Experts consider that mometasone may stabilize but not improve
the symptoms of BP. Mometasone is a short acting, mid strength
topical steroid not typically given as a treatment for BP
- Wijayanti A, Zhao CY, Boettiger D et al. The Reliability,
Validity and Responsiveness of Two Disease Scores (BPDAI and ABSIS)
for Bullous Pemphigoid: Which One to Use? Acta Derm Venereol. 2017;
97:24-31
- Roujeau J-C, Morel P, Dalle E, Guillot B et al. Plasma Exchange
in Bullous Pemphigoid. Lancet 1984; 324:486-489
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
disease, most common in the elderly, 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.
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, is a
C5 complement inhibitor that also independently and specifically
inhibits leukotriene B4 (LTB4) activity. Nomacopan is being
clinically evaluated in: bullous pemphigoid (BP), atopic
keratoconjunctivitis (AKC), and thrombotic microangiopathy, or TMA.
Akari believes that the dual action of nomacopan on both C5 and
LTB4 may be beneficial in AKC and BP. Akari is also developing
other tick derived proteins, including longer acting versions.
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. 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; 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 outbreak of
coronavirus; risks related to material weaknesses in our internal
controls over financial reporting and risks relating to the
ineffectiveness of our disclosure controls and procedures; 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.
For more information
Investor Contact:Peter Vozzo Westwicke Partners (443) 213-0505
peter.vozzo@westwicke.com
Media Contact:Mary-Jane Elliott / Sukaina
Virji / Nicholas Brown Consilium Strategic Communications
+44 (0)20 3709 5700 Akari@consilium-comms.com
Akari Therapeutics (NASDAQ:AKTX)
Historical Stock Chart
From Mar 2024 to Apr 2024
Akari Therapeutics (NASDAQ:AKTX)
Historical Stock Chart
From Apr 2023 to Apr 2024