Last updated on April 23, 2014 at 16:13 EDT

Aeterna Zentaris: Phase 1 Results Show Perifosine’s Activity Against Neuroblastoma

June 21, 2012

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/m(2)) of perifosine on day 1, followed by daily maintenance doses (50-75mg/m(2)) 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 2(nd) line chemotherapy (all patients); myeloablative chemotherapy and stem
cell transplantation (10 patients) and/or targeted radiotherapy with
I-131-MIBG (9 patients).


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.


        --  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
        --  Perifosine may have a possible role with chemotherapy,
            radiation therapy, and/or other agents active in PI3K/Akt

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: PR Newswire