Aeterna Zentaris' partner Keryx receives FDA orphan-drug designation for perifosine in neuroblastoma

Jul 15 2010

Aeterna Zentaris Inc. (NASDAQ: AEZS, TSX: AEZ) (the "Company"), a late-stage drug development company specialized in oncology and endocrine therapy, today announced that its partner, Keryx Biopharmaceuticals, Inc. ("Keryx") (Nasdaq: KERX), has been granted orphan-drug designation by the U.S. Food and Drug Administration ("FDA") for perifosine, Aeterna Zentaris' novel, potentially first-in-class, oral Akt inhibitor, for the treatment of neuroblastoma. Neuroblastoma is a cancer of the nervous system affecting mostly children and infants for which there are no FDA approved therapies. Keryx is Aeterna Zentaris' partner and licensee for perifosine in the United States, Canada and Mexico. Æterna Zentaris has also out-licensed perifosine to Handok in South Korea, while retaining rights for the rest of the world.

Juergen Engel, Ph.D., President and CEO of Aeterna Zentaris stated, "The orphan-drug designation in neuroblastoma is another important milestone in the development of perifosine as a novel approach to treating cancer patients, particularly in this area of unmet medical need. We are looking forward to perifosine's future development in neuroblastoma and in other indications with our partner Keryx."

Phase 1 data of perifosine in recurrent pediatric solid tumors, including neuroblastoma, were presented last month in the pediatric solid tumor poster discussion session held at the 46th Annual Meeting of the American Society of Clinical Oncology ("ASCO"). Investigators from the Memorial Sloan-Kettering Cancer Center concluded that perifosine was demonstrated to be safe and well tolerated in children with advanced solid tumors and that perifosine may have antitumor clinical activity as a single agent in neuroblastoma. Additionally, in a preclinical study recently published in the Journal of the National Cancer Institute, perifosine showed a statistically significant reduction in neuroblastoma cell survival, slowed or regressed tumor growth, and increased survival in mice bearing neuroblastoma tumors.

A decreased level of activated Akt was also observed in perifosine-treated neuroblastoma cells and xenograft tumors.