Akari Therapeutics Receives FDA Fast Track Designation for Nomacopan for Pediatric Hematopoietic Stem Cell Transplant-Related...
August 14 2019 - 8:00AM
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, today announced that the U.S. Food and Drug
Administration (FDA) has granted Fast Track designation for
nomacopan for the treatment of hematopoietic stem cell transplant
associated thrombotic microangiopathy (HSCT-TMA) in pediatric
patients. The Fast Track designation supports Akari’s plans for
expedited approval of nomacopan for HSCT-TMA, with the pivotal
trial expected to commence in the fourth quarter of 2019.
Fast Track designation is intended to facilitate the development
and expedite the review of new drugs which show promise in treating
serious or life-threatening conditions and address unmet medical
needs. Drugs that receive this designation benefit from more
frequent communications and meetings with FDA to review the drug's
development plan including the design of the proposed clinical
trials, use of biomarkers and the extent of data needed for
approval. Drugs with Fast Track designation may qualify for
priority review to expedite the FDA review process, if relevant
criteria are met.
HSCT-TMA is an orphan condition with an
estimated fatality rate of more than 80% in children with the
severe disease. There are currently no approved treatments for
HSCT-TMA but there is strong evidence for the role of complement
and potentially LTB4 in the etiology of the disease.
In September 2018, Akari announced that in the
first two HSCT-TMA patients treated with nomacopan as part of a UK
named patient program, it had observed disease resolution by way of
a rapid reduction of the markers of complement activation as well
as normalization of markers that are elevated in HSCT-TMA patients
(platelet count, red blood cell fragments, thrombocytopenia,
elevated LDH and hypertension). On the basis of existing treatment
data from these HSCT-TMA patients and a further 20 cumulative
patient-years of data from other patients treated with nomacopan,
Akari is working with the FDA Model Informed Drug Development
(MIDD) Program to optimize the pediatric dosing with nomacopan for
the planned pivotal HSCT-TMA trial.
Clive Richardson, interim CEO of Akari
Therapeutics, said, “Fast Track designation provides our HSCT-TMA
clinical program a pathway for an expedited approval for nomacopan,
and we plan to commence a pivotal trial in the fourth quarter of
2019. We see HSCT-TMA as a gateway into a range of other poorly
treated orphan TMAs in both pediatric and adult patients, and are
optimistic about the potential for nomacopan to offer an improved
standard of care for these orphan conditions with high mortality
rates.”
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. 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, BP and
other orphan inflammatory conditions. 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 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 informationInvestor 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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