QUEBEC CITY, July 14 /PRNewswire-FirstCall/ - 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. AEterna 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.
Perifosine, a novel, potentially first-in-class, oral Akt inhibitor, is currently in Phase 3 trials in the United States for advanced colorectal cancer and multiple myeloma, under Special Protocol Assessment and Fast Track designation granted by the FDA for both indications. FDA has also granted perifosine orphan-drug designation for multiple myeloma and neuroblastoma. In Europe, the European Medicines Agency ("EMA") has issued positive Scientific Advice for perifosine in multiple myeloma and colorectal cancer, as well as positive opinion for Orphan Medicinal Product designation for perifosine in 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.
About Orphan-Drug Designation
Orphan-drug designation is granted by the FDA Office of Orphan Drug Products to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity if the drug is the first of its type approved for the specified indication or if it demonstrates superior safety, efficacy, or a major contribution to patient care versus another drug of its type previously granted the designation for the same indication, as well as with tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and waiver of Prescription Drug User Fee Act (PDUFA) filing fees.
According to the American Cancer Society, neuroblastoma is the most common cancer in infants (less than 1 year old) and accounts for about 7% of all pediatric cancers. There are about 650 new cases of neuroblastoma each year in the United States, and, while in rare cases neuroblastoma is detected by ultrasound in utero, the average age at the time of diagnosis is approximately 1 to 2 years, with 90% of cases diagnosed before age 5. In about 2 of 3 cases, the disease has already spread (metastasized) to other parts of the body at the time of diagnosis and as a result, treatment options can be limited.
Neuroblastoma exhibits a wide range of behavior. Some infant neuroblastomas may just go away without treatment (spontaneously regress), while other neuroblastomas may be resistant to very intensive multimodal treatment, and, in these cases, neuroblastoma is considered to be one of the most aggressive and difficult to cure childhood cancers.
To date, no FDA approved therapies exist for the treatment of neuroblastoma. Chemotherapy is the mainstay of neuroblastoma treatment. The type of chemotherapy and its intensity are determined by the age of the patient and the extent of the disease (risk-factors). However, because these cancers can be hard to treat, additional therapies are needed in order to delay progression and extend survival.
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 www.aezsinc.com.
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 AETERNA ZENTARIS INC.