QUÉBEC CITY, June 21, 2012
/PRNewswire/ - Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ)
(the "Company") today announced that Phase 1 trial results for
its oral PI3K/Akt inhibitor, perifosine, showed the drug's activity
against chemo-resistant and radio-resistant neuroblastoma, while
allowing good quality of life and sparing vital organs.
Neuroblastoma is a type of childhood cancer which usually begins in
nerve tissues. Data were presented yesterday by Brian H. Kushner, MD, of the Memorial
Sloan-Kettering Cancer Center in New
York, during a poster session at the Advances in
Neuroblastoma Research Conference which is being held in
Toronto, Canada.
The Study
This was an open-label dose-escalating Phase 1 trial to assess
toxicity and efficacy of perifosine, given in monotherapy to
patients with neuroblastoma (clinicaltrials.gov
NCT00776867). Patients were dosed using 50mg tablets and
received a loading dose (100-200mg/m2) of perifosine on
day 1, followed by daily maintenance doses
(50-75mg/m2) until progressive disease or dose-limiting
toxicity. Disease evaluation was every 8 weeks.
The poster reported on the outcome of 24 patients treated to
date; patients had a median age of 8.7 years (range 4.7 to
33.5) and a median disease duration of 4.6 years (range 2.5 to
8.0). Three patients were treated for neuroblastoma refractory to
primary therapy, and 21 for neuroblastoma resistant to salvage
therapy after 1 to 5 (median 2) prior relapses. Prior therapy
included high-dose conventional induction and 2nd line
chemotherapy (all patients); myeloablative chemotherapy and stem
cell transplantation (10 patients) and/or targeted radiotherapy
with I-131-MIBG (9 patients).
Results
Anti-neuroblastoma activity was evident by a 50%
progression-free survival rate at 12 months (Standard Error ±11%)
and included 1 complete remission (CR) based on a normalized MIBG
scan and 3 patients with improved MIBG scan and normalized bone
marrow histology over prolonged follow-up (up to 37+months). No
significant toxicity was seen, in particular no grade 3 problems,
and no safety issues were encountered in 6 patients who started
treatment with pre-existing thrombocytopenia and/or grade 3
elevations in liver enzymes.
Conclusions
- Perifosine was well tolerated, without major toxicity - hence,
compatible with good quality of life;
- Perifosine monotherapy may help in progression-free survival of
patients with persistent/stable MIBG-positivity in skeletal
sites;
- Perifosine may have a possible role with chemotherapy,
radiation therapy, and/or other agents active in PI3K/Akt
pathway.
Juergen Engel, PhD, President and
CEO at Aeterna Zentaris stated, "These data again emphasize
perifosine's anticancer activity, as was the case earlier this week
with the article published in Cancer, outlining perifosine's
anticancer activity in renal cell carcinoma. On both occasions, the
authors alluded to perifosine's potential as a combination therapy
which further supports our current Phase 3 trial in multiple
myeloma in which perifosine is used in combination with bortezomib
and dexamethasone."
The poster titled, "Targeting PI3K/Akt Pathway: Perifosine
Monotherapy for Resistant Neuroblastoma in a Phase1/1B Study",
B. Kushner, N-K.V. Cheung, S. Modak, O. Becher,
E.M. Basu, S.S. Roberts, K.
Kramer, I.J. Dunkel, is available at
the following link.
About Perifosine
Perifosine is a novel, oral PI3K/Akt inhibitor. It is currently
in a Phase 3 trial in multiple myeloma for which it has been
granted orphan drug and Fast Track designations by the Food and
Drug Administration (FDA), as well as positive Scientific Advice
and orphan medicinal product designation from the European
Medicines Agency. It has also been granted orphan drug designation
by the FDA for neuroblastoma. Furthermore, perifosine is in
earlier-stage development for other cancer indications. Rights for
perifosine have been out licensed to Yakult Honsha Co. Ltd. for
Japan, to Handok Pharmaceuticals
Co. Ltd. for Korea and to Hikma Pharmaceuticals PLC for the
Middle East and certain countries
in North Africa. Aeterna Zentaris
holds rights for the rest of the world.
About Aeterna Zentaris
Aeterna Zentaris is an oncology and endocrinology drug
development company currently investigating treatments for various
unmet medical needs. The Company's pipeline encompasses compounds
at all stages of development, from drug discovery through to
marketed products. 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.