Full Press Release Details
Akari Therapeutics Announces Acceleration of the Phase 3 Clinical
Trial of Nomacopan for Treatment of Pediatric HSCT-TMA into Pivotal Part B and Addition of a New Pipeline Program Developing Nomacopan
for HSCT-TMA in Adults
NEW YORK and LONDON, Feb. 13, 2023 (GLOBE NEWSWIRE) -- Akari Therapeutics,
Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, today announced
the Phase 3 clinical trial of nomacopan in pediatric patients with hematopoietic stem cell transplant-related thrombotic microangiopathy
(HSCT-TMA) is moving into Part B sooner than expected and the company is beginning the planning and design for the pivotal study in pediatric
patients over 2 years of age. Akari also announces it has added a new pipeline program that will develop nomacopan as a potential treatment
for adult HSCT-TMA, which will include a study that is supportive of the pediatric program. Study enrollment for the adult program is
expected in 2024. Nomacopan is a novel bispecific inhibitor of complement C5 and leukotriene B4 (LTB4) in clinical development for pediatric
and adult HSCT-TMA. Long-acting PAS-nomacopan is in pre-clinical development for geographic atrophy secondary to dry age-related macular
Part A of the Phase 3 clinical trial of nomacopan in pediatric HSCT-TMA
was designed to include three cohorts: ages 0.5 to <2 years, 2 to <9 years and 9 to <18 years. Akari enrolled patients
in the two older age groups and, based on feedback from the FDA, the PK/PD data from these patients from the Part A study are consistent
with the predictions derived from Akari's latest PK/PD model. Since enrollment for the youngest age cohort (ages 0.5 to <2 years)
in the Part A pediatric study is not completed Akari is keeping the study open for this youngest group of pediatric patients while advancing
the pivotal Part B study of nomacopan in the older pediatric patients.
Akari participated in the FDA Model-Informed Drug Development (MIDD)
program to refine the Akari PK/PD model suitability and doses for the Phase 3 clinical trial of nomacopan in pediatric HSCT-TMA. Clinical
data from 38 subjects (in previous clinical studies and healthy volunteers) were included in the Akari PK/PD model, which was used to
predict dosing for pediatric HSCT-TMA patients through 10,000 virtual patient simulations. These informed FDA MIDD interactions that confirmed
PK/PD model suitability and doses selected for the nomacopan Phase 3 Part A clinical trial in pediatric HSCT-TMA. An expanded PK/PD model
using data from 55 patients treated with nomacopan was reviewed in the recent Type C interaction with the FDA along with PK/PD data from
the Part A study. According to FDA feedback, the model is predictive and supports a simple, fixed dosing regimen of nomacopan in the upcoming
Phase 3 pivotal Part B clinical trial in pediatric HSCT-TMA patients who are ages 2 years and older.
"We made the important decision to not wait for Part A to be
completed before we reached out to the FDA for Type C feedback on our next steps toward the pivotal study, an interaction which we had
originally estimated would be mid-year 2023. This earlier feedback enabled the acceleration into the Part B clinical trial of nomacopan
in pediatric HSCT-TMA," said Rachelle Jacques, President and CEO of Akari. "Our decision to initiate a new pipeline program
to develop nomacopan as a potential treatment for adults with HSCT-TMA was also an important one and will support our pediatric program.
The significant unmet need is shared among both children and adults with dismal outcomes and no approved treatment options."
Thrombotic microangiopathy following a stem cell transplant procedure
is a rare but serious complication of HSCT that appears to involve complement activation, inflammation, tissue hypoxia and blood clots,
leading to progressive organ damage and death. The mortality rate across both adult and pediatric patients who develop severe transplant-related
TMAs is 80%.1 Currently, there are no approved treatment options for adult or pediatric patients in the U.S. or Europe.
Of the approximate 22,000 HSCTs that occur annually in the U.S., it is estimated that 8,400 of these lead to TMAs.2-4 Approximately
3,400 are moderate to severe, complement-mediated TMAs and about 200 to 300 of these patients are children. The adult HSCT-TMA population
is >10 times the size of the pediatric population.2-4
"Because the youngest age cohort is six months to two years,
it is a very small group of patients and quite challenging to recruit into the study. With no approved treatment options and high mortality
rates in this condition, it is important that we urgently move forward into the pivotal Part B clinical trial of nomacopan in the older
age cohorts of pediatric HSCT-TMA patients," said John Neylan, M.D., Executive Vice President and Chief Medical Officer of Akari.
"We don't want to leave any family behind, and we intend to keep Part A open so we may continue our efforts to enroll the
youngest HSCT-TMA patients in this study."
Because Part A is continuing, Akari is reporting that the Part A PK/PD
data to date is sufficient to move into Part B with the older pediatric age cohorts and a full readout will be provided upon completion
of the Part A study of nomacopan in pediatric HSCT-TMA.
In 2022, Akari was granted the Rare Pediatric Disease Designation for
nomacopan in the treatment of pediatric HSCT-TMA. The Rare Pediatric Disease Designation is an important addition to the Orphan Drug and
Fast Track designations previously granted by the FDA for nomacopan in pediatric HSCT-TMA.
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 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 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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