Akari Therapeutics Announces Initiation of Pivotal Phase III Trial of Nomacopan in Pediatric Hematopoietic Stem Cell Transpla...
December 23 2019 - 8:45AM
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 a U.S. Food and Drug
Administration (FDA) investigational new drug application (IND) is
open for its multicenter Phase III study for the treatment of
pediatric HSCT-TMA with nomacopan, allowing clinical sites to open
in the first quarter of 2020.
“With the pediatric HSCT-TMA IND now open we look forward to
starting the pivotal Phase III study of nomacopan in HSCT-TMA, a
potential treatment for a high risk pediatric population that
suffer very high death rates and for which there are currently no
approved therapies. If successful, we expect HSCT-TMA to be a
gateway into a range of other poorly treated orphan TMAs,”
commented Clive Richardson, CEO of Akari Therapeutics. “In
addition, following the recent successful completion of our Phase
II bullous pemphigoid study, we expect data from our Phase I/II
atopic keratoconjunctivitis trial in early 2020 and interim data
from our Phase III paroxysmal nocturnal hemoglobinuria trial in the
first half of 2020.”
HSCT-TMA is an orphan hematological condition that occurs in up
to 30% of patients who have received a hematopoietic stem cell
transplant (HSCT). There are no approved treatments for pediatric
HSCT-TMA, and it has an estimated mortality rate of more than 80%
in children with the severe form of the disease1. It is this severe
form that is being targeted with nomacopan which is a bifunctional
inhibitor of complement C5 and leukotriene B4 (LTB4). Following the
recent end-of-Phase II meeting with the FDA, Akari has now opened
an IND to initiate its pivotal pediatric HSCT-TMA study based on a
single arm responder-based design. Recruitment will be focused on
specialist pediatric sites in the U.S. and Europe where treatment
tends to be concentrated in specialist centres.
Whilst the role of complement inhibition is understood to play
an important role in pediatric HSCT-TMA, the Company believes LTB4
may also be an important target in reducing epithelial activation
in both TMA and graft versus-host disease2 (GVHD) which often occur
simultaneously. The Company believes daily dosing with nomacopan
may also be of particular advantage in facilitating more complete
complement suppression, especially in HSCT-TMA patients with high
transfusion requirements.
As previously announced, this two-part pivotal Phase III study
of nomacopan in pediatric patients with HSCT-TMA is based on
guidance from the Company’s end-of-Phase II meeting with the FDA.
Part A of the trial is a dose confirmation study. Part B of the
trial is a single arm responder-based efficacy study that will
follow an interim analysis of Part A and a meeting with the FDA.
Akari has both FDA fast track and orphan status for this
program.
1 Sonata Jodele, et al. New approaches in the
diagnosis, pathophysiology, and treatment of pediatric
hematopoietic stem cell transplantation associated thrombotic
microangiopathy. Transfus Apher Sci . 2016 April; 54(2):
181–190
2 Takatsuka, et al. Predicting the severity of
intestinal graft-versus-host disease from leukotriene B4 levels
after bone marrow transplantation. Transplantation 2000, 26:
1313-1316
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).
Nomacopan is currently being clinically evaluated in four
indications: bullous pemphigoid (BP), atopic keratoconjunctivitis
(AKC), thrombotic microangiopathy (TMA), and paroxysmal nocturnal
hemoglobinuria (PNH). 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 regarding, among other
things, statements related to the offering, the expected gross
proceeds and the expected closing of the offering. 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; our ability to enter into collaborative,
licensing, and other commercial relationships and on terms
commercially reasonable to us; 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 departure of our former Chief
Executive Officers and other executive officers; 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 VozzoWestwicke(443)
213-0505peter.vozzo@westwicke.com
Media Contact:
Sukaina Virji / Nicholas Brown / Lizzie
SeeleyConsilium Strategic Communications+44 (0)20 3709
5700Akari@consilium-comms.com
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