Akari Therapeutics Announces Receipt of Orphan Drug Designation for Coversin from the U.S. FDA for Treatment of Paroxysmal No...
September 12 2016 - 7:15AM
Akari Therapeutics (NASDAQ:AKTX), an emerging growth,
development-stage biopharmaceutical company, announced today that
the U.S. Food and Drug Administration (FDA) has granted to Akari an
Orphan Drug Designation for recombinant protein inhibitor of
complement factor 5 for the treatment of Paroxysmal Nocturnal
Hemoglobinuria (PNH). Coversin, Akari’s lead clinical product,
which falls within the Orphan Drug Designation, is a
second-generation complement inhibitor that acts on complement
component-C5, preventing release of C5a and formation of C5b-9
(also known as the membrane attack complex or MAC).
PNH is an ultra-rare, life-threatening and debilitating disease
of the blood with an estimated 8,000-10,000 patients across North
America and Europe. Due to an acquired genetic deficiency,
uncontrolled complement activation in PNH patients allows their own
complement system to attack and destroy blood cells, leading to
life-threatening complications.
"We are pleased to receive Orphan Drug Designation in the United
States for PNH, and now have this designation for PNH and Guillain
Barre Syndrome, or GBS, in the US and EU," said Dr. Gur Roshwalb,
CEO of Akari. "We have continued to see complete complement
inhibition and symptom control in a PNH patient with resistance to
eculizumab, who has been self-administering subcutaneous Coversin
for over 7 months. We believe that Coversin, when approved, could
provide important benefits for all patients with PNH.”
The FDA grants Orphan Drug Designation status to products that
treat rare diseases, providing incentives to sponsors developing
drugs or biologics. The FDA defines rare diseases as those
affecting fewer than 200,000 people in the United States at the
time of designation. Orphan Drug Designation provides the sponsor
certain benefits and incentives, including a period of marketing
exclusivity if regulatory approval of the drug is ultimately
received for the designated indication, potential tax credits for
certain activities, eligibility for orphan drug grants, and the
waiver of certain administrative fees. The receipt of Orphan Drug
Designation status does not change the regulatory requirements or
process for obtaining marketing approval and designation does not
mean that marketing approval will be received.
About Akari Therapeutics Plc
Akari is a clinical-stage biopharmaceutical company focused on
the development and commercialization of innovative therapeutics to
treat orphan autoimmune and inflammatory diseases. Akari’s lead
drug, Coversin is a second-generation complement inhibitor that
acts on complement component-C5, preventing release of C5a and
formation of C5b-9 (also known as the membrane attack complex or
MAC). C5 inhibition is growing in importance in a range of rare
autoimmune diseases related to dysregulation of the complement
component of the immune system, including Paroxysmal Nocturnal
Hemoglobinuria (PNH), atypical Hemolytic Uremic Syndrome (aHUS),
and Guillain Barré syndrome (GBS).
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: an inability or delay in obtaining
required regulatory approvals for Coversin and any other product
candidates, which may result in unexpected cost expenditures; risks
inherent in drug development in general; uncertainties in obtaining
successful clinical results for Coversin and any other product
candidates and unexpected costs that may result therefrom; failure
to realize any value of Coversin 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 Coversin may not
be as large as expected; 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; our inability to obtain
additional capital on acceptable terms, or at all; unexpected cost
increases and pricing pressures; uncertainties of cash flows and
inability to meet working capital needs; and risks and other risk
factors detailed in our public filings with the U.S. Securities and
Exchange Commission, including our Annual Report on Form 10-K filed
on March 23, 2016. 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.
Contact:
Investor & Media Contact:
The Trout Group
Tricia Truehart
ttruehart@troutgroup.com
646-378-2953
Media Contact:
Susan Forman / Laura Radocaj
Dian Griesel Int'l.
(212) 825-3210
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