Company Has Also Requested Scientific Advice on its Phase 3 Program with Perifosine in Refractory Advanced Colorectal Cancer.

{{image}}Quebec City, QC (Press Release) – Æterna Zentaris Inc. (NASDAQ: AEZS,TSX: AEZ) (the "Company"), a late-stage drug devel­op­ment com­pany specialized in on­col­ogy and en­do­crine ther­apy, today announced that it has requested Scientific Advice from the European Medicines Agency (EMA) to assure the acceptability of the recently ini­ti­ated Phase 3 pro­grams for the devel­op­ment of its lead anticancer com­pound, perifosine, in its two lead indi­ca­tions, multiple myeloma and refractory ad­vanced colorectal cancer. Previously, agree­ment was reached with the U.S. Food and Drug Admin­istra­tion (FDA) on a Special Protocol Assessment (SPA) for the pivotal studies in each of the lead indi­ca­tions, which will be sponsored by Keryx Bio­pharma­ceuticals Inc., (Keryx), (NASDAQ: KERX), Æterna Zentaris's licensee for perifosine in North America.

In multiple myeloma, the SPA-agreed upon Phase 3 study is a double-blind, placebo-controlled study of perifosine com­bined with bor­tez­o­mib (Velcade(R)) in bor­tez­o­mib pre­treated patients. Progression-free survival will be the pri­mary efficacy end­point in this trial, which will in­clude follow-up for over­all survival. The advice from the EMA indicates that, in principle, the proposed study is con­sidered suf­fi­cient to provide all data nec­es­sary to sup­port a mar­ket­ing authori­za­tion of perifosine in com­bi­na­tion with bor­tez­o­mib (Velcade(R)). At the relevant time, the actual approved indi­ca­tion in Europe will have to take into account the avail­a­bil­ity of lipo­somal doxorubicin (Caelyx(R)), which in the European Union is approved in bor­tez­o­mib pre­treated patients with multiple myeloma. Further, the EMA has con­firmed that the planned electro­cardio­gram (ECG) evaluations in­cluded in the Phase 3 study will suffice to assess the cardiac safety of perifosine. Therefore, the Company does not in­tend to ini­ti­ate any addi­tional study with perifosine in Europe for the multiple myeloma indi­ca­tion.

Æterna Zentaris has also requested Scientific Advice for the devel­op­ment of perifosine in refractory ad­vanced colorectal cancer, where the SPA-agreed upon Phase 3 study is a double-blind placebo-controlled study com­par­ing the com­bi­na­tion of perifosine and capecitabine (Xeloda(R)) with single-agent capecitabine (Xeloda(R)). Patients in this study will be intensively pre­treated and will have failed all avail­able treat­ment options except capecitabine (Xeloda(R)). Overall survival will be analyzed as the pri­mary efficacy end­point. For both indi­ca­tions, safety data from studies of perifosine in other indi­ca­tions and drug com­bi­na­tions will be used as sup­port­ive in­for­ma­tion to define the clin­i­cal safety profile of perifosine.

Dr. Juergen Engel, Pres­i­dent and CEO of Æterna Zentaris stated, "We are very pleased with the out­come of our discussions with the European Medicines Agency so far. For the devel­op­ment of perifosine in multiple myeloma, we believe that the planned North American clin­i­cal pro­gram, which is sponsored by our partner Keryx, is suf­fi­cient to sup­port an appli­ca­tion for mar­ket­ing authori­za­tion in Europe and the rest of the world. We are looking for­ward to the out­come of our discussions with the EMA which is ex­pec­ted during the third quarter of this year, re­gard­ing our devel­op­ment pro­gram in colorectal cancer."

About Scientific Advice

Scientific Advice is a pro­ce­dure offered by the European Medicines Agency to stakeholders for clarification of questions arising during devel­op­ment of medicinal prod­ucts. The scope of Scientific Advice is limited to scientific issues, i.e. to quality, non-clinical and clin­i­cal aspects of the con­cerned medicinal prod­uct not yet unequivocally covered by published scientific guidelines. Scientific Advice focuses on devel­op­ment strategies rather than pre-evaluation of data to sup­port a Marketing Authori­za­tion Application. Scientific Advice is legally non-binding and is based on the current scientific knowledge which may be subject to future changes.

About Perifosine

Perifosine is a novel, poten­tially first-in-class, oral anticancer agent that modulates Akt, and a number of other key signal transduction path­ways, in­clud­ing the JNK and MAPK path­ways, all of which are path­ways asso­ci­ated with pro­grammed cell death, cell growth, cell dif­fer­en­tiation and cell survival. The effects of perifosine on Akt are of particular interest because of the importance of this path­way in the devel­op­ment of most cancers, with evi­dence that it is often activated in tumors that are resistant to other forms of anticancer ther­apy, and the dif­fi­culty encountered thus far in the discovery of drugs that will inhibit this path­way without causing excessive toxicity. High levels of activated Akt (pAkt) are seen frequently in many types of cancer and have been correlated with poor prognosis.

About Æterna Zentaris Inc.

Æterna Zentaris Inc. is a late-stage drug devel­op­ment com­pany specialized in on­col­ogy and en­do­crine ther­apy. News releases and addi­tional in­for­ma­tion are avail­able at www.aezsinc.com.

Forward-Looking Statements

This press release con­tains for­ward-looking state­ments made pursuant to the safe harbor 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.

Source: Æterna Zentaris.