Akari Therapeutics Announces New Clinical Data that Show Long-Term Self-Administered Nomacopan is Well-Tolerated and Substant...
December 11 2020 - 8:55AM
Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage
biopharmaceutical company focused on innovative therapeutics to
treat orphan autoimmune and inflammatory diseases where the
complement and/or leukotriene systems are implicated, announces new
data on the efficacy and safety profile of long-term
self-administration of nomacopan for treatment of patients with
PNH.
“The data we have accrued on the use of self-administered
nomacopan in PNH demonstrate that the drug is well tolerated with a
positive clinical response, supporting nomacopan’s potential for
both short and long-term use across our other clinical programs,”
said Clive Richardson, Chief Executive Officer of of Akari
Therapeutics. “We are now leveraging this positive PNH data to
explore regional partnering opportunities.”
The data from 19 PNH patients2 treated for a median of 18.5
months are derived from the Phase II COBALT trial (n=8 patients),
the Phase II trial which specifically recruited
eculizumab-resistant patients (n=2 patients), the Phase III
CAPSTONE trial (n = 9 patients), and from the long-term safety
study CONSERVE (n = 15), which accepted patients from the Phase II
and Phase III studies. Sixteen of the 19 PNH patients were
transfusion dependent prior to treatment with nomacopan, of whom 14
were treated with nomacopan for six months or more.
The data show that long term self-administration of nomacopan by
PNH patients:
- Reduced transfusion dependence (the most clinically relevant
efficacy endpoint) by 79% in the 14 formerly transfusion dependent
patients treated with nomacopan for at least six months, with a
median time since last transfusion of 13.8 months.
- In the Phase III CAPSTONE trial, all patients dosed with
nomacopan (n=4) were transfusion independent for the six-month
treatment period while all patients on placebo (n=5) remained
transfusion dependent, which was the primary end point of the
study. The difference is statistically significant at the p=0.034
level. The active and placebo groups were very similar in terms of
transfusion requirements prior to treatment.
- The number of units of packed red blood cells (U PRBC) received
per month declined in all 16 of the transfusion dependent PNH
patients by 77% from a mean value of 0.91 U PRBC per month prior to
treatment with nomacopan to 0.21 U PRBC per month on
nomacopan3.
- The three PNH patients who were transfusion independent prior
to treatment with nomacopan remained transfusion independent on
nomacopan.
- None of the 19 PNH patients treated with nomacopan for over 30
cumulative patient-years had a reported major adverse vascular
event (MAVE).
- There has only been a single reported serious adverse event
(urinary tract infection) in one patient that was considered
possibly related to nomacopan. None of the patients has had a
meningococcal infection reported.
- Terminal complement activity was below the lower limit of assay
quantification (10 CH50 U Equivalent/ml) in all patients at all
time points measured during long-term treatment on nomacopan.
Transfusion independence as used above is defined as a period of
at least six months without transfusion. This duration is
considered clinically important as it is the same duration of
transfusion independence as used for the primary endpoint in
CAPSTONE and also the pivotal studies of eculizumab and ravulizumab
that led to approval of these drugs for the treatment of PNH.
The 79% transfusion independence reported for the 14 PNH
patients all formerly transfusion dependent who were treated with
nomacopan for at least six months, compares favorably to the
treatment of PNH patients on long-term eculizumab therapy where
between approximately 50%-60% of transfusion dependent patients
become transfusion independent in a 12-month period (Brodsky et
al., 2008, SHEPHERD study on eculizumab, Hillmen et al 2013).
Similar data has been reported in the more recent head to head
Phase III comparative study of eculizumab and ravulizumab (Wook Lee
et al., 2019) where transfusion independence in the two arms
respectively increased from 21% to 66% of and 23% to 74% of
patients over the first 6 months of treatment.
The transfusion data from the CONSERVE long term study is
considered particularly important because it provides “real world”
data from a total of 15 patients on nomacopan treatment as a
long-term self-administered therapy. In CONSERVE, clinicians were
free to follow their normal transfusion practices and patients were
only scheduled to visit the clinic at three to six monthly
intervals, mimicking “real world” clinical practice.
Dr. Austin Kulasekararaj, Lead PNH clinician at King’s College
Hospital, London, UK, the principal investigator on the long-term
CONSERVE safety and efficacy study of nomacopan in patients with
PNH, commented, “The CAPSTONE study was originally designed to
achieve approximately 50% transfusion independence in line with
Phase III PNH pivotal trials using eculizumab, so this mature data
demonstrate the positive clinical benefits of nomacopan as a
terminal complement inhibitor in PNH. Equally important,
self-administered nomacopan appears to be well tolerated with an
excellent safety profile.”
The Company’s lead programs for nomacopan are focused on
diseases with no approved treatments, including BP and pediatric
HSCT-TMA. Data from the Company’s PNH programs (CAPSTONE and
CONSERVE), while discontinued, support the Company’s main clinical
programs, all of which have complement dysregulation playing an
important role.
Full clinical details on all 19 PNH patients treated with
nomacopan will be provided in a manuscript that is currently in
preparation.
1Hematopoietic stem cell
transplant-related thrombotic microangiopathy
219 PNH patients: 13 male and 6
female; median age 39; age range 22 – 69. Three of the 19 patients
had a history of thrombosis and 3 other patients a history of
aplastic anaemia.
3The number of U PRBC received per
month was simply derived by dividing the total number of U PRBC
received by all transfusion dependent patients in the 12 months
before nomacopan treatment (n = 16; total U PRBC = 174; total
months 192), and by dividing the total number of U PRBC received by
the same patients while on nomacopan (n = 16; total U PRBC = 65;
total months 313). In the Wook Lee et al., 2019 study on eculizumab
and ravulizumab efficacy in PNH patients the authors reported that
the mean total number of U PRBC transfused during the first 6-month
treatment period was 4.8 (SD + 5.1) on ravulizumab and 5.6 (SD +
5.9) on eculizumab.
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.
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. You should not place
undue reliance upon the Company’s forward looking statements.
Except as required by law, the Company undertakes no obligation to
revise or update any forward-looking statements in order to reflect
any event or circumstance that may arise after the date of this
press release. 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; our ability to enter into collaborative,
licensing, and other commercial relationships and on terms
commercially reasonable to us; 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 COVID-19 pandemic; 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.
Investor Contact:
Peter VozzoWestwicke+1 (443)
213-0505peter.vozzo@westwicke.com
Media Contact:
Sukaina Virji / Lizzie SeeleyConsilium Strategic
Communications+44 (0)20 3709 5700Akari@consilium-comms.com
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