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

Monday, June 7, 2010 General News
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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,

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

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.


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