Aeterna Zentaris Inc. has reached an agreement with the US Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA) for an upcoming phase III registration trial in endometrial cancer with its doxorubicin peptide conjugate, AEZS-108. The SPA agreement states that the proposed trial protocol design, clinical endpoints and planned analyses are acceptable to the FDA to support a regulatory submission.
"We are pleased with the agreement with the FDA which provides us with a clearly defined development and regulatory pathway for AEZS-108 in endometrial cancer," stated Juergen Engel, PhD, president and CEO at Aeterna Zentaris. "AEZS-108's innovative targeted approach could offer a new treatment option for women with endometrial cancer and provide the Company with a significant market opportunity."
This will be an open-label, randomized, multi-centre phase III trial conducted in North America and Europe, comparing AEZS-108 with doxorubicin as second line therapy for locally-advanced, recurrent or metastatic endometrial cancer. The trial will involve approximately 500 patients and the primary efficacy endpoint is improvement in median Overall Survival.
The SPA process is a procedure by which the FDA provides official evaluation and written guidance on the design and size of proposed protocols that are intended to form the basis for a Biologics License Application (BLA) or New Drug Application (NDA). Final marketing approval depends on the results of efficacy, the adverse event profile and an evaluation of the benefit/risk of treatment demonstrated in the phase III trial.
AEZS-108 represents a new targeting concept in oncology using a hybrid molecule composed of a synthetic peptide carrier and a well-known chemotherapy agent, doxorubicin. AEZS-108 is the first intravenous drug in advanced clinical development that directs the chemotherapy agent specifically to Luteinizing Hormone Releasing Hormone (LHRH)-receptor expressing tumours, resulting in more targeted treatment with less damage to healthy tissue. The product has successfully completed phase II studies for the treatment of ovarian and endometrial cancer and the Company is currently planning a phase III trial in endometrial cancer under a Special Protocol Assessment. AEZS-108 is also in phase II trials in prostate, breast and bladder cancer. AEZS-108 has been granted orphan drug designation by the FDA and orphan medicinal product designation from the European Medicines Agency for the treatment of ovarian cancer. Aeterna Zentaris owns the worldwide rights to AEZS-108.
Endometrial cancer is the most common gynecologic malignancy and develops when abnormal cells amass to form a tumour in the lining of the uterus. It largely affects women over the age of 50 with a higher prevalence in Caucasians and a higher mortality rate among African Americans. Approximately one in 30 women is diagnosed with endometrial cancer every year. According to the American Cancer Society, 47,130 new cases of endometrial are expected to be diagnosed in the United States in 2012, and Data Monitor expects 35,600 new cases in EU-G5 in 2013, with about 20 per cent recurrent disease.
Aeterna Zentaris is an oncology and endocrinology drug development company currently investigating treatments for various unmet medical needs.