Alexion Provides Update on Phase 2 Clinical Trial with Eculizumab in Antibody Mediated Rejection (AMR) in Living-Donor Kidney...
January 07 2015 - 4:15PM
Business Wire
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today reported
results from its randomized, open-label, multicenter Phase 2
clinical trial to determine the safety and efficacy of eculizumab
in the prevention of antibody mediated rejection (AMR) in
living-donor kidney transplant recipients requiring
desensitization. The primary composite endpoint, defined as the
occurrence of biopsy-proven AMR, graft loss, patient death, or loss
to follow-up at Week 9 post-transplant, did not reach statistical
significance. While the primary composite endpoint rate in the
eculizumab arm was as expected from earlier studies with
eculizumab, the rate in the control arm was lower than was
expected, based on natural history studies reported in the
literature.
“Although eculizumab performed in line with the results reported
from prior eculizumab studies, which showed similarly low AMR rates
in high-risk patient populations, we are disappointed that this
trial did not meet its primary composite endpoint,” said Martin
Mackay, Ph.D., Executive Vice President and Global Head of R&D
at Alexion. “We expect to complete the data analyses and discuss
these results with regulators, and are currently developing plans
to commence a clinical trial with eculizumab as a treatment for
patients diagnosed with AMR.”
About the Clinical Trial
This Phase 2 trial enrolled 102 patients receiving kidney
transplants from living donors, all of whom were at risk of AMR,
based on elevated levels of donor-specific antibodies. After
screening, patients were randomized into two groups of 51 patients
each, with one group receiving eculizumab, and a control group
receiving the anti-rejection standard of care specified by the
institution in which each patient’s transplant took place. Patients
in the standard of care control arm were eligible to receive
eculizumab treatment for presumed AMR. Data were collected during
the nine-week treatment period for the primary composite endpoint,
with additional safety and efficacy endpoints measured through 12
months post-transplant. In the analysis of the 9-week data, for the
primary composite endpoint, the rate was 9.8% in the eculizumab arm
and 15.7% in the control arm (P=0.554). No safety signal has been
reported to the Company by the independent data monitoring
committee. Alexion expects that the data from the study will be
presented at a future medical meeting.
About AMR
Acute antibody mediated rejection (AMR) is a severe and
potentially life-threatening condition that can lead to severe
allograft damage resulting in rapid loss of function and possible
loss of the transplanted organ.1 Patients who are sensitized (have
high levels of donor-specific-antibodies [DSAs]) are at high risk
for developing acute AMR.1,2 The historical rate of AMR in
high-risk living-donor kidney transplant recipients has been
reported as high as 41%.3 Acute AMR is believed to be primarily a
result of uncontrolled complement activation caused by DSAs.1,2
Currently, there are no approved therapies for the prevention or
treatment of acute AMR.
About Alexion
Alexion is a biopharmaceutical company focused on serving
patients with severe and rare disorders through the innovation,
development and commercialization of life-transforming therapeutic
products. Alexion is the global leader in complement inhibition and
has developed and markets Soliris® (eculizumab) as a treatment for
patients with paroxysmal nocturnal hemoglobinuria (PNH) and
atypical hemolytic uremic syndrome (aHUS), two debilitating,
ultra-rare and life-threatening disorders caused by chronic
uncontrolled complement activation. Soliris is currently approved
in nearly 50 countries for the treatment of PNH and in nearly 40
countries for the treatment of aHUS. Alexion is evaluating other
potential indications for Soliris in additional severe and
ultra-rare disorders beyond PNH and aHUS, and is developing other
highly innovative biotechnology product candidates, including
asfotase alfa, across multiple therapeutic areas. This press
release and further information about Alexion can be found at:
www.alexionpharma.com.
[ALXN-G]
Safe Harbor Statement
This news release contains forward-looking statements, including
statements related to potential medical benefits of eculizumab in
AMR, our future plans for eculizumab in AMR, and the results of
discussions with regulators regarding eculizumab in AMR.
Forward-looking statements are subject to factors that may cause
Alexion's results and plans to differ from those expected,
including, for example, that results from the Phase 2 study may not
support a regulatory submission, the possibility that studies of
eculizumab in AMR may be delayed, prevented or suspended, the
possibility that results of clinical trials are not predictive of
safety and efficacy results of eculizumab in AMR, and a variety of
other risks set forth from time to time in Alexion's filings with
the Securities and Exchange Commission, including but not limited
to the risks discussed in Alexion's Quarterly Report on Form 10-Q
for the period ended September 30, 2014. Alexion does not intend to
update any of these forward-looking statements to reflect events or
circumstances after the date hereof, except when a duty arises
under law.
References
1. Takemoto SK, Zeevi A, Feng S, et al. National conference to
assess antibody-mediated rejection in solid organ transplantation.
Am J Transplant. 2004; 4(7):1033-41.
2. Collins AB, Schneeberger EE, Pascual MA, et al. Complement
activation in acute humoral renal allograft rejection: diagnostic
significance of C4d deposits in peritubular capillaries. J Am Soc
Nephrol. 1999;10(10):2208-14.
3. Stegall MD1, Diwan T, Raghavaiah S, et al. Terminal
complement inhibition decreases antibody-mediated rejection in
sensitized renal transplant recipients. Am J Transplant. 2011
Nov;11(11):2405-13.
Alexion:MediaIrving Adler, 203-271-8210Executive Director,
Corporate CommunicationsorKim Diamond, 203-439-9600Senior Director,
Corporate CommunicationsorInvestorsElena Ridloff, CFA,
203-699-7722Executive Director, Investor Relations
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