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

QUÉBEC CITY, June 21, 2012 /CNW Telbec/ - 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 ( 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).


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 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

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.


For further information:

Investor Relations
Ginette Beaudet Vallières
Investor Relations Coordinator
(418) 652-8525 ext. 265

Media Relations
Paul Burroughs
Director of Communications
(418) 652-8525 ext. 406

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