Aeterna Zentaris Announces Phase 1 Data of Single Agent Perifosine in the Treatment of Recurrent Pediatric Solid Tumors, Including Patients with Advanced Brain Tumors and Neuroblastoma
Perifosine demonstrated to be safe and tolerable, with early signs of clinical benefit in advanced refractory pediatric neuroblastoma patients
QUEBEC CITY, June 7 /PRNewswire-FirstCall/ -- Aeterna Zentaris
Inc. (TSX: AEZ; Nasdaq: AEZS), (the "Company") a late-stage drug
development company specialized in oncology and endocrine therapy,
today announced that Phase 1 data of perifosine (KRX-0401) in
recurrent pediatric solid tumors was presented yesterday in the
pediatric solid tumor poster discussion session held at the 46th
Annual Meeting of the American Society of Clinical Oncology (ASCO)
taking place in Chicago. Perifosine is the Company's novel,
potentially first-in-class, oral anti-cancer agent that inhibits
Akt activation in the phosphoinositide 3-kinase (PI3K) pathway.
This study, conducted by the Memorial Sloan-Kettering Cancer Center
pediatric group, marks the first time that perifosine has been
administered in a pediatric patient setting. Perifosine is also
currently in Phase 3 trials in multiple myeloma and colorectal
cancer, as well as in Phase 2 trials for multiple types of
cancer.
Study Design
This Phase I Study of perifosine for Recurrent Pediatric Solid
Tumors is a single center, open-label, dose-escalating study to
assess safety, tolerability, pharmacokinetics (PK), and to identify
any dose limiting toxicity (DLT) of single agent perifosine in
pediatric patients with any solid tumor that has failed standard
therapy. Eleven patients (4 males, 7 females), at a median age of
13 years (5-18) were treated in this study to date. The following
tumor types were treated thus far: high-grade glioma (5),
medulloblastoma (2), neuroblastoma (3), and ependymoma (1). Most
patients were heavily pretreated, with a median of three prior
lines of therapy. Cohorts of three patients were treated at three
dose levels: 25mg/m(2)/day, 50mg/m(2)/day and 75mg/m(2)/day using
50mg tablets of perifosine after a loading dose on day 1, and
taking into account the drug's long half-life ((greater
than)100hrs). No DLTs were observed at any of the three dose
levels; dose level 4 is currently open for accrual. PK data thus
far suggests similar drug absorption by pediatric patients relative
to adult patients treated with single agent perifosine.
Of particular interest are the early signs of clinical activity
observed in two of the three patients with Stage 4 refractory
neuroblastoma. Both patients were refractory to prior treatments
upon entering the study and achieved stable disease for 48 weeks
and 55+ weeks (ongoing). The investigators concluded that
perifosine is well-tolerated in children with recurrent solid
tumors and that these early signals of activity warrant further
investigation in patients with advanced neuroblastoma and select
brain tumors. Previously, perifosine has been shown to target
activation of Akt in neuroblastoma cells and xenografts and to
significantly inhibit tumor growth in vivo and improve the survival
of mice bearing neuroblastoma tumors.
Juergen Engel, Ph.D., President and CEO of Aeterna Zentaris
commented, "We are very pleased with the data presented for
perifosine in pediatric tumors at ASCO which are encouraging for
its future clinical development in this indication, and further
confirm its potential as a novel therapeutic approach for multiple
forms of cancer."
A copy of the abstract #9540 entitled, "Phase 1 Study of Single
Agent Perifosine for Recurrent Pediatric Solid Tumors", lead author
Oren J. Becher, M.D., can be accessed through the ASCO website,
www.asco.org
About Perifosine
Perifosine, a novel, potentially first-in-class, oral Akt
inhibitor, is currently in Phase 3 trials for multiple myeloma and
advanced colorectal cancer, under Special Protocol Assessment and
Fast Track designation granted by the Food and Drug Administration
(FDA) for both indications. FDA has also granted perifosine
orphan-drug status for multiple myeloma. Furthermore, the European
Medicines Agency (EMA) has issued a positive Scientific Advice as
well as a positive opinion for Orphan Medicinal Product designation
for perifosine for multiple myeloma.
Perifosine is also in a Phase 1 trial in pediatric patients, as
well as in other Phase 1 and Phase 2 trials for several other tumor
types.
Perifosine is licensed to Keryx Biopharmaceuticals Inc. (Keryx)
(NASDAQ: KERX) , in the United States, Canada
and Mexico. Aeterna Zentaris has also out-licensed perifosine to
Handok in South Korea, while retaining rights for the rest of the
world.
About Aeterna Zentaris Inc.
Aeterna Zentaris Inc. is a late-stage drug development company
specialized in oncology and endocrine therapy. News releases and
additional information are available at 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, which 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 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
the 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
except if we are required by a governmental authority or applicable
law.
Source: AETERNA ZENTARIS INC.
CONTACT: Investor Relations: Ginette Vallieres, Investor
Relations
Coordinator, (418) 652-8525, ext. 265, gvallieres@aezsinc.com;
Media
Relations: Paul Burroughs, Director of Communications, (418)
652-8525 ext.
406, pburroughs@aezsinc.com
Posted: June 2010
