Akari Therapeutics’ Nomacopan Granted U.S. Orphan Drug Designation for Hematopoietic Stem Cell Transplantation-Associated T...
August 30 2019 - 7:00AM
Akari Therapeutics, Plc (Nasdaq: AKTX), a biopharmaceutical company
focused on innovative therapeutics to treat orphan autoimmune and
inflammatory diseases where the complement (C5) and/or leukotriene
(LTB4) systems are implicated, today announced that the U.S. Food
and Drug Administration (FDA) has granted orphan drug designation
for nomacopan for the treatment of hematopoietic stem cell
transplantation-associated thrombotic microangiopathy (HSCT-TMA).
Orphan drug designation for nomacopan follows Fast
Track designation that the Company received from the FDA earlier in
August 2019 for the same indication in pediatric patients. The
Company continues to progress towards a pivotal trial for HSCT-TMA
with nomacopan, which is expected to start in the fourth quarter of
2019.
“We are pleased to obtain orphan drug designation
for nomacopan in HSCT-TMA, a devastating rare disease for which
there are currently no approved treatments,” said Clive Richardson,
Chief Executive Officer of Akari Therapeutics. “The granting of
orphan drug designation and Fast Track designation by the FDA for
nomacopan underscores the significant unmet medical need in this
disease. We look forward to taking advantage of the opportunities
that FDA orphan drug designation and Fast Track designation provide
across all stages of drug development in order to bring this
potential new treatment option to patients as rapidly as
possible.”
Orphan drug designation by the FDA is granted to
promote the development of drugs that target conditions affecting
200,000 or fewer U.S. patients annually and that are expected to
provide significant therapeutic advantage over existing treatments.
Orphan designation qualifies Akari for various benefits, including
seven years of market exclusivity following marketing approval, tax
credits on U.S. clinical trials, eligibility for orphan drug
grants, and a waiver of certain administrative fees.
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 both AKC and BP.
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; 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
Akari Therapeutics (NASDAQ:AKTX)
Historical Stock Chart
From Aug 2024 to Sep 2024
Akari Therapeutics (NASDAQ:AKTX)
Historical Stock Chart
From Sep 2023 to Sep 2024