Keryx Biopharmaceuticals initiates Phase 3 registration clinical trial for perifosine

Dec 16 2009

Trial conducted pursuant to Special Protocol Assessment with the Food and Drug Administration

Æterna Zentaris Inc. (NASDAQ: AEZS; TSX: AEZ) (the "Company"), a global biopharmaceutical company focused on oncology and endocrine therapy, today announced the initiation, by its partner Keryx Biopharmaceuticals Inc. ("Keryx") (Nasdaq: KERX), of a Phase 3 registration clinical trial for perifosine (KRX-0401), the Company's PI3K/Akt pathway inhibitor, in relapsed/refractory multiple myeloma patients. Keryx is Æterna Zentaris' partner and licensee for perifosine in the United States, Canada and Mexico. Perifosine is also out-licensed to Handok in South Korea while Æterna Zentaris retains rights for the rest of the world.

The trial, entitled, "A Phase 3 Randomized Study to Assess the Efficacy and Safety of Perifosine Added to the Combination of Bortezomib (Velcade(R)) and Dexamethasone in Multiple Myeloma Patients Previously Treated with Bortezomib", is a double-blind, placebo-controlled trial comparing the efficacy and safety of perifosine vs. placebo when combined with bortezomib (Velcade(R)) and dexamethasone. The trial will enroll approximately 400 patients with relapsed or relapsed/refractory multiple myeloma. The primary endpoint is progression-free survival and secondary endpoints include overall response rate, overall survival and safety. This trial is being conducted pursuant to a Special Protocol Assessment (SPA) with the Food and Drug Administration (FDA). Additionally, the FDA has granted perifosine Orphan Drug and Fast Track designations in this indication. An estimated 40 to 50 centers throughout the United States and select centers outside of the United States will be participating in this Phase 3 trial.

The Phase 3 trial is being led by the Principal Investigator, Dr. Paul Richardson, Clinical Director of the Jerome Lipper Multiple Myeloma Center, at Dana-Farber Cancer Institute (DFCI) in Boston, MA and Dr. Kenneth C, Anderson, Chief, Division of Hematologic Neoplasia at DFCI.

Dr. Richardson's clinical research focuses on studying novel therapies for multiple myeloma. He was the lead investigator of the SUMMIT and APEX clinical trials that led to the FDA approval of bortezomib (Velcade(R)) for the treatment of multiple myeloma. More recently, he served as clinical trials core chair of the Multiple Myeloma Research Consortium, completing 5 years of service in the role this year. Dr. Anderson serves as Chair of the NCCN Multiple Myeloma Clinical Practice Guidelines Committee and chairs the scientific advisory board for the Multiple Myeloma Research Foundation. In addition to playing an integral role in the development and approval of bortezomib (Velcade(R)), Drs. Richardson and Anderson led both preclinical and early phase clinical trials for the immunomodulatory drug lenalidomide (Revlimid(R)), leading to its FDA approval in 2007.

Juergen Engel, Ph.D., President and CEO at Æterna Zentaris stated, "We are very excited with Keryx's initiation of this Phase 3 trial with perifosine, our lead oncology compound. Based on prior positive results, we believe that perifosine could become a novel oral combination treatment of great benefit to patients suffering from multiple myeloma."