QUEBEC CITY, July 14 /PRNewswire-FirstCall/ - Aeterna Zentaris
Inc. (NASDAQ: AEZS, TSX: AEZ) (the "Company"), a late-stage drug
development company specialized in oncology and endocrine therapy,
today announced that its partner, Keryx Biopharmaceuticals, Inc.
("Keryx") (Nasdaq: KERX), has been granted orphan-drug designation
by the U.S. Food and Drug Administration ("FDA") for perifosine,
Aeterna Zentaris' novel, potentially first-in-class, oral Akt
inhibitor, for the treatment of neuroblastoma. Neuroblastoma is a
cancer of the nervous system affecting mostly children and infants
for which there are no FDA approved therapies. Keryx is Aeterna
Zentaris' partner and licensee for perifosine in the United States, Canada and Mexico. Æterna Zentaris has also out-licensed
perifosine to Handok in South
Korea, while retaining rights for the rest of the world.
Juergen Engel, Ph.D., President
and CEO of Aeterna Zentaris stated, "The orphan-drug designation in
neuroblastoma is another important milestone in the development of
perifosine as a novel approach to treating cancer patients,
particularly in this area of unmet medical need. We are looking
forward to perifosine's future development in neuroblastoma and in
other indications with our partner Keryx."
Phase 1 data of perifosine in recurrent pediatric solid tumors,
including neuroblastoma, were presented last month in the pediatric
solid tumor poster discussion session held at the 46th Annual
Meeting of the American Society of Clinical Oncology ("ASCO").
Investigators from the Memorial Sloan-Kettering Cancer Center
concluded that perifosine was demonstrated to be safe and well
tolerated in children with advanced solid tumors and that
perifosine may have antitumor clinical activity as a single agent
in neuroblastoma. Additionally, in a preclinical study recently
published in the Journal of the National Cancer Institute,
perifosine showed a statistically significant reduction in
neuroblastoma cell survival, slowed or regressed tumor growth, and
increased survival in mice bearing neuroblastoma tumors.
A decreased level of activated Akt was also observed in
perifosine-treated neuroblastoma cells and xenograft tumors.
About Perifosine
Perifosine, a novel, potentially first-in-class, oral Akt
inhibitor, is currently in Phase 3 trials in the United States for advanced colorectal
cancer and multiple myeloma, under Special Protocol Assessment and
Fast Track designation granted by the FDA for both indications. FDA
has also granted perifosine orphan-drug designation for multiple
myeloma and neuroblastoma. In Europe, the European Medicines Agency ("EMA")
has issued positive Scientific Advice for perifosine in multiple
myeloma and colorectal cancer, as well as positive opinion for
Orphan Medicinal Product designation for perifosine in 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.
About Orphan-Drug Designation
Orphan-drug designation is granted by the FDA Office of Orphan
Drug Products to novel drugs or biologics that treat a rare disease
or condition affecting fewer than 200,000 patients in the U.S. The
designation provides the drug developer with a seven-year period of
U.S. marketing exclusivity if the drug is the first of its type
approved for the specified indication or if it demonstrates
superior safety, efficacy, or a major contribution to patient care
versus another drug of its type previously granted the designation
for the same indication, as well as with tax credits for clinical
research costs, the ability to apply for annual grant funding,
clinical research trial design assistance and waiver of
Prescription Drug User Fee Act (PDUFA) filing fees.
About Neuroblastoma
According to the American Cancer Society, neuroblastoma is the
most common cancer in infants (less than 1 year old) and accounts
for about 7% of all pediatric cancers. There are about 650 new
cases of neuroblastoma each year in the
United States, and, while in rare cases neuroblastoma is
detected by ultrasound in utero, the average age at the time of
diagnosis is approximately 1 to 2 years, with 90% of cases
diagnosed before age 5. In about 2 of 3 cases, the disease has
already spread (metastasized) to other parts of the body at the
time of diagnosis and as a result, treatment options can be
limited.
Neuroblastoma exhibits a wide range of behavior. Some infant
neuroblastomas may just go away without treatment (spontaneously
regress), while other neuroblastomas may be resistant to very
intensive multimodal treatment, and, in these cases, neuroblastoma
is considered to be one of the most aggressive and difficult to
cure childhood cancers.
To date, no FDA approved therapies exist for the treatment of
neuroblastoma. Chemotherapy is the mainstay of neuroblastoma
treatment. The type of chemotherapy and its intensity are
determined by the age of the patient and the extent of the disease
(risk-factors). However, because these cancers can be hard to
treat, additional therapies are needed in order to delay
progression and extend survival.
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 harbor 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.