Full Press Release Details
Receives FDA Fast Track Designation for Nomacopan for Paediatric Hematopoietic Stem Cell Transplant-Related Thrombotic Microangiopathy
NEW YORK and LONDON, August 14,
2019 - 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.
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.
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.
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."
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
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
For more information
Sukaina Virji / Nicholas Brown
Consilium Strategic Communications