Independent Committee Recommends Phase 3 X-PECT Study to
Proceed as Planned
QUÉBEC CITY, Aug. 31, 2011
/PRNewswire/ - Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ)
(the "Company") today announced that the independent Data
Safety Monitoring Board (DSMB) for the pivotal Phase 3 X-PECT study
of perifosine in patients with refractory advanced colorectal
cancer has completed a pre-specified interim analysis for safety
and futility. The DSMB has recommended that the Phase 3 study
continue to completion, as planned.
This Phase 3 study sponsored and conducted by our North American
licensee for perifosine, Keryx Biopharmaceuticals, Inc., (Keryx),
(NASDAQ:KERX), has completed enrollment, with over 465 patients
from 65 U.S. sites. The study is being conducted pursuant to a
Special Protocol Assessment (SPA) with the Food and Drug
Administration (FDA) and with Fast-Track Designation. Furthermore,
the Company has received positive Scientific Advice from the
European Medicines Agency (EMA) for this ongoing Phase 3 trial,
which is therefore expected to be sufficient for registration in
Europe. Perifosine rights have
also been licensed to Yakult Honsha for Japan and to Handok for Korea.
The Phase 3 trial, entitled the "X-PECT" (Xeloda® +
Perifosine Evaluation in Colorectal cancer Treatment) trial, is a
randomized (1:1), double-blind trial comparing the efficacy and
safety of perifosine + capecitabine vs. placebo + capecitabine in
over 465 randomized patients with refractory advanced colorectal
cancer. Patients must have failed available therapy including
5-fluorouracil (5-FU), oxaliplatin (Eloxatin®),
irinotecan, bevacizumab (Avastin®) and, if KRAS
wild-type, failed therapy with prior cetuximab
(Erbitux®) and/or panitumumab (Vectibix®).
The primary endpoint is overall survival, with secondary endpoints
including overall response rate (complete + partial responses),
progression-free survival and safety. Approximately 360 events of
death will trigger the unblinding of the study.
Juergen Engel, Ph.D., President
and CEO of Aeterna Zentaris, commented, "We are very pleased by the
news that the DSMB has recommended that the study should proceed to
completion as planned, and thank the DSMB members for their work.
With this positive news we believe that our partner Keryx is
well positioned to complete the Phase 3 program in refractory
advanced colorectal cancer, which could bring this new therapy to
patients suffering from this disease. We continue to prepare
for the next steps and will work towards commercialization in the
rest of the world."
About Special Protocol Assessments
The Special Protocol Assessment (SPA) process is a procedure by
which the FDA provides official evaluation and written guidance on
the design and size of proposed protocols that are intended to form
the basis for a new drug application.
Final marketing approval depends on the efficacy and safety
results, including the adverse event profile, and an evaluation of
the benefit/risk of treatment demonstrated in the Phase 3 clinical
trial. The SPA agreement may only be changed through a written
agreement between the sponsor and the FDA, or if the FDA becomes
aware of a substantial scientific issue essential to product
efficacy or safety. For more information on the Special Protocol
Assessment procedure, please visit:
http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm080571.pdf.
About Perifosine
Perifosine is a novel, oral anticancer treatment that inhibits
Akt activation in the phosphoinositide 3-kinase (PI3K) pathway. The
product works by interfering with membranes of cancer cells thereby
inhibiting Akt signaling which then affects cell death, growth,
differentiation and survival. Perifosine, in combination with
chemotherapeutic agents, is currently being studied for the
treatment of multiple myeloma, colorectal cancer and other cancers,
and is the most advanced anticancer agent of its class. Perifosine,
as monotherapy, is being explored in other indications. The FDA has
granted perifosine orphan-drug designation in multiple myeloma and
neuroblastoma, and Fast Track designations in both multiple myeloma
and refractory advanced colorectal cancer. Additionally, an
agreement was reached with the FDA to conduct the Phase 3
trials in both of these indications under a Special Protocol
Assessment. Perifosine has also been granted orphan medicinal
product designation from the European Medicines Agency (EMA) in
multiple myeloma. Furthermore, perifosine has received positive
Scientific Advice from the EMA for both the multiple myeloma and
advanced colorectal cancer programs, with ongoing Phase 3 trials
for these indications expected to be sufficient for registration in
Europe. Perifosine rights have
been licensed to Keryx for North
America, to Yakult Honsha for Japan and to Handok for Korea.
About Aeterna Zentaris Inc.
Aeterna Zentaris is a late-stage oncology drug development
company currently investigating potential treatments for various
cancers including colorectal, multiple myeloma, endometrial,
ovarian, prostate and bladder cancer. The Company's innovative
approach of "personalized medicine" means tailoring treatments to a
patient's specific condition and to unmet medical needs. Aeterna
Zentaris' deep pipeline is drawn from its proprietary discovery
unit providing the Company with constant and long-term access to
state-of-the-art therapeutic options. For more information please
visit www.aezsinc.com.
Forward-Looking Statements
This press release contains forward-looking statements made
pursuant to the safe harbour provisions of the U.S. Securities
Litigation Reform Act of 1995. Forward-looking statements involve
known and unknown risks and uncertainties that could cause the
Company's actual results to differ materially from those in the
forward-looking statements. Such risks and uncertainties include,
among others, the availability of funds and resources to pursue
R&D projects, the successful and timely completion of clinical
studies, the risk that safety and efficacy data from any of our
Phase 3 trials may not coincide with the data analyses from
previously reported Phase 1 and/or Phase 2 clinical trials, the
ability of the Company to take advantage of business opportunities
in the pharmaceutical industry, uncertainties related to the
regulatory process and general changes in economic conditions.
Investors should consult the Company's quarterly and annual filings
with the Canadian and U.S. securities commissions for additional
information on risks and uncertainties relating to forward-looking
statements. Investors are cautioned not to rely on these
forward-looking statements. The Company does not undertake to
update these forward-looking statements. We disclaim any obligation
to update any such factors or to publicly announce the result of
any revisions to any of the forward-looking statements contained
herein to reflect future results, events or developments, unless
required to do so by a governmental authority or by applicable
law.
SOURCE AETERNA ZENTARIS INC.