Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the "Company") today announced that a manuscript entitled "Perifosine Plus Bortezomib and Dexamethasone in patients with Relapsed/Refractory Multiple Myeloma Previously Treated with Bortezomib: Results of a Multicenter Phase 1/2 Trial" reporting Phase 1/2 combination activity of perifosine in the treatment of advanced multiple myeloma (MM) patients, was selected for publication in the October 10, 2011 online edition of the Journal of Clinical Oncology (JCO).
Perifosine, is the Company's novel, potentially first-in-class, oral anti-cancer drug that inhibits Akt activation in the phosphoinositide 3-kinase (PI3K) pathway, which is currently being investigated in metastatic colorectal cancer, as well as other cancer indications.
The ongoing Phase 3 study in MM is being conducted under a Special Protocol Assessment (SPA), and Fast Track designation, as agreed with the Food & Drug Administration (FDA). Perifosine is currently also in Phase 3 clinical development for refractory advanced colorectal cancer, also under an SPA and Fast Track designation.Perifosine has been granted Orphan Drug Status by FDA and orphan medicinal product designation from the European Medicines Agency (EMA) in MM, and has received positive Scientific Advice from the EMA for both the MM and advanced colorectal cancer programs, with the ongoing Phase 3 trials for these indications expected to be sufficient for registration in Europe.
In this Phase 1/2 study, perifosine in combination with bortezomib +/- dexamethasone was evaluated in 84 heavily pre-treated patients with relapsed or relapsed/refractory MM. All patients were required to receive prior bortezomib and most were bortezomib refractory (73%). The combination demonstrated an overall response rate (ORR) including MR or > of 41% in all evaluable patients, with stable disease observed in an additional 41% of evaluable patients. The ORR was 65% for bortezomib-relapsed patients and 32% for patients with bortezomib-refractory disease. Median progression-free survival (PFS) was 6.4 months, with a median PFS of 8.8 months in the bortezomib-relapsed population. Median overall survival (OS).Therapy was generally well-tolerated; toxicities, including gastrointestinal side-effects and fatigue, proved manageable. No treatment-related mortality was seen.
The investigators concluded the data reported for both safety and efficacy in this patient population were encouraging for the continued study of perifosine.
Data from this study were previously presented at the 2009 American Society of Hematology (ASH) conference.
A copy of the article featured in the current online edition of the Journal of Clinical Oncology can be obtained at: http://jco.ascopubs.org/content/early/2011/10/04/JCO.2010.33.9788.abstract?sid=d0e30e69-718c-46ae-81bc-367fdc9d9cb7
Perifosine is a novel, oral anticancer treatment that inhibits Akt activation in the phosphoinositide 3-kinase (PI3K) pathway. The product works by interfering with membranes of cancer cells thereby inhibiting Akt signaling which then affects cell death, growth, differentiation and survival. Perifosine, in combination with chemotherapeutic agents, is currently being studied for the treatment of multiple myeloma, colorectal cancer and other cancers, and is the most advanced anticancer agent of its class. Perifosine, as monotherapy, is being explored in other indications. The FDA has granted perifosine orphan-drug designation in multiple myeloma and neuroblastoma, and Fast Track designations in both multiple myeloma and refractory advanced colorectal cancer. Additionally, an agreement was reached with the FDA to conduct the Phase 3 trials in both of these indications under a Special Protocol Assessment. Perifosine has also been granted orphan medicinal product designation from the European Medicines Agency (EMA) in multiple myeloma. Furthermore, perifosine has received positive Scientific Advice from the EMA for both the multiple myeloma and advanced colorectal cancer programs, with ongoing Phase 3 trials for these indications expected to be sufficient for registration in Europe. Perifosine rights have been licensed to Keryx for North America, to Yakult Honsha for Japan and to Handok for Korea.
About Aeterna Zentaris Inc.
Aeterna Zentaris is a late-stage oncology drug development company currently investigating potential treatments for various cancers including colorectal, multiple myeloma, endometrial, ovarian, prostate and bladder cancer. The Company's innovative approach of "personalized medicine" means tailoring treatments to a patient's specific condition and to unmet medical needs. Aeterna Zentaris' deep pipeline is drawn from its proprietary discovery unit providing the Company with constant and long-term access to state-of-the-art therapeutic options. For more information please visit www.aezsinc.com.
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 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.
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