Full Press Release Details
Akari Therapeutics Provides Updates on Development of Nomacopan
in Pediatric and Adult HSCT-TMA
NEW YORK and LONDON, July 13, 2023 (GLOBE NEWSWIRE) - Akari Therapeutics,
Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, today announced
updates in the development programs investigating nomacopan in pediatric and adult hematopoietic stem cell transplant-related thrombotic
microangiopathy (HSCT-TMA). Akari is on track to begin patient enrollment in the registrational Phase 3 study of nomacopan in pediatric
HSCT-TMA that is expected to produce safety and efficacy data supportive of a potential regulatory filing and approval. Akari also is
planning to move forward into a Phase 3 double-blind placebo-controlled clinical trial of nomacopan in adult HSCT-TMA and to open enrollment
"Our goal is to bring an approved treatment
to both children and adults in desperate need, and we are deeply committed to making a positive impact on the standard of care now and
down the road for patients facing this devastating condition," said Rachelle Jacques, Akari
HSCT-TMA is a rare complication
of stem cell transplant, and it is a clinical diagnosis which is based on signs, symptoms, patient history, and physical exam. Currently,
there are no approved treatments for HSCT-TMA and among patients who have severe disease there is a mortality rate of about 80%. Of the
approximately 22,000 hematopoietic stem cell transplants that take place each year in the U.S., about 7,920 (36%) lead to TMAs. Of these
TMAs, approximately 4,250 are standard risk and 3,700 are high risk patients, with about 300 to 400 of these occurring in children and
3,200 to 3,300 occurring in adults (10 times the size of the pediatric patient population).
Consensus guidelines were recently published
by an international panel of experts that harmonize diagnosis criteria and support earlier screening and diagnosis in the care of patients.1
Historically, a lack of harmonization of diagnostic and prognostic markers has caused difficulties in the interpretation of patient outcomes.
The consensus criteria are a crucial advancement to support earlier screening and diagnosis
in the care of patients with transplant-associated TMAs that are associated with significant mortality once multiorgan dysfunction occurs,
which is difficult to reverse. The Akari HSCT-TMA Phase 3 clinical trials design has been significantly informed by these consensus criteria
for earlier diagnosis of high-risk (severe) patients.
is consensus that prospective screening and early diagnosis of TMA following transplantation can save lives by heading off the multiorgan
dysfunction that is too often irreversible and fatal," said Sonata Jodele, M.D., Cincinnati Children's Hospital Medical Center.
Akari is advancing into Phase 3 open-label
single-arm study of nomacopan in pediatric HSCT-TMA and is planning to move into a Phase 3 double-blind placebo-controlled study of nomacopan
in adult HSCT-TMA in 2023 and 2024, respectively. These advancements to Phase 3 clinical trials were supported by Akari's PK/PD
data (in adults) and U.S. Food and Drug Administration (FDA) agreement that PK/PD modelling and predictions about pediatric dosing were
sufficient to move forward.
is important that there is harmonization between adult and pediatric studies with the study design, such as inclusion criteria, because
comparability in our clinical trials data will not only help future researchers but will also help bring clarity to regulators as they
review our applications for approval," said John Neylan, M.D., Akari Chief Medical Officer.
Orphan Drug, Fast Track, and Rare Pediatric
Disease designations have been granted by the FDA for nomacopan in pediatric HSCT-TMA. With the Rare Pediatric Disease Designation, a
sponsor who receives an approval of a new drug application (NDA) or biologics license application (BLA) is eligible for a Priority Review
Voucher (PRV) to either redeem for priority review of a subsequent marketing application for a different product or sell to a third party.
Akari was recently issued a positive opinion by the European Medicines Agency (EMA) on the company's application for orphan drug
designation for nomacopan as a treatment in hematopoietic stem cell transplantation.
About Akari Therapeutics
Akari Therapeutics, plc (Nasdaq: AKTX) is
a biotechnology company developing advanced therapies for autoimmune and inflammatory diseases. Akari's lead asset, investigational
nomacopan, is a bispecific recombinant inhibitor of complement C5 activation and leukotriene B4 (LTB4) activity. Akari's pipeline
includes a Phase 3 clinical trial program investigating nomacopan for severe pediatric hematopoietic stem cell transplant-related thrombotic
microangiopathy (HSCT-TMA). Akari has been granted Orphan Drug, Fast Track and Rare Pediatric Disease designations from the FDA for nomacopan
for the treatment of pediatric HSCT-TMA. Akari's pipeline also includes a clinical program developing nomacopan for adult HSCT-TMA
and pre-clinical research of long-acting PAS-nomacopan in geographic atrophy (GA). For more information about Akari, please visit akaritx.com.
Cautionary Note Regarding Forward-Looking
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 there; 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 impact of the COVID-19 pandemic; 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.
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