Akari Therapeutics Announces Pivotal Phase III Trial Design of Nomacopan in Pediatric Hematopoietic Stem Cell Transplant-Rela...
December 02 2019 - 8:30AM
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 the design of a pivotal Phase III
pediatric trial in HSCT-TMA following a FDA End-of-Phase II
meeting. Akari also announces that, in another hematological
condition, all six patients from the long-term Phase II PNH study
who were transfusion dependent at entry are now transfusion
independent on long-term treatment with nomacopan.
HSCT-TMA is an orphan hematological condition with no approved
treatments and 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. Following a recent
End-of-Phase II meeting with the FDA, Akari plans to initiate a
single arm responder-based study design, based on treatment with
nomacopan for up to 24 weeks. The primary endpoints are focused on
disease response defined primarily by renal improvement and reduced
transfusion dependence. The study will be in two parts, with data
from seven patients in Part A used to confirm dosing and endpoints
for Part B, with the pharmacokinetic (PK) modelling agreed with the
FDA through Akari’s participation in the Model Informed Drug
Discovery Program (MIDD). Following an interim efficacy and safety
readout from Part A and meeting with the FDA, patients would then
be recruited into Part B of the responder study.
While the role of complement inhibition is understood to play an
important role in HSCT-TMA, LTB4 may also be an important target in
reducing epithelial activation in both TMA and graft versus-host
disease2 (GVHD) which often occur simultaneously. Daily dosing with
nomacopan is also likely to be of a particular advantage in
facilitating more complete complement suppression, especially in
HSCT-TMA patients with high transfusion requirements.
HSCT-TMA is Akari’s second haematological clinical program and
follows a successful Phase II study in PNH completed in early 2018,
after which patients continued treatment with nomacopan in a
long-term safety study. New data from Akari’s ongoing long-term
study, shows that all six patients from the Phase II study who were
transfusion dependent at entry are now transfusion independent on
nomacopan, having had in all cases no transfusions for a minimum of
six months. In addition, during more than 20 cumulative
patient-years of PNH patient treatment with nomacopan, there have
been no reported drug-related serious adverse events.
“Following our meeting with the FDA, we look forward to starting
the pivotal Phase III study of nomacopan in HSCT-TMA, a potential
treatment for a high risk pediatric population which currently has
no approved therapies. If successful, we expect this will be a
gateway indication into a range of other poorly treated orphan
TMAs,” commented Clive Richardson, CEO Akari Therapeutics. “We are
pleased with the progress being made across our clinical studies
where recent positive clinical data in bullous pemphigoid (BP) and
PNH provides further support for the underlying efficacy of
nomacopan.”
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
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