Aeterna Zentaris Inc., a specialty biopharmaceutical company engaged in developing and commercialising novel treatments in oncology, endocrinology and women's health, announced that it has reached its goal of recruiting 500 patients for its pivotal phase 3 ZoptEC (Zoptarelin Doxorubicin in Endometrial Cancer) clinical study with zoptarelin doxorubicin in women with advanced, recurrent or metastatic endometrial cancer.

The trial is being conducted in over 120 sites in North America, Europe and Israel. The primary efficacy endpoint is improvement in overall survival.

Following its first pre specified interim analysis last April, a Data and Safety Monitoring Board recommended that the ZoptEC phase 3 study continue as planned. A second interim analysis is expected during Q4, 2015 at approximately 192 events, with the final analysis planned at an anticipated 384 events. The trial is expected to be completed by the end of 2016.

David A. Dodd, chairman and chief executive officer of Aeterna Zentaris, commented “We are very excited to have completed patient recruitment for our ZoptEC phase 3 trial in endometrial cancer earlier than expected and I would like to thank everyone involved in this project for their steadfast commitment. We believe zoptarelin doxorubicin has the potential to become the first FDA approved medical therapy for advanced, recurrent endometrial cancer. This could result in its rapid adoption as a novel core therapy for patient treatment and management, and therefore, could represent a significant market opportunity for the company.”

“Moving forward, we are continuing to develop our commercialisation plans regarding zoptarelin doxorubicin in this indication, including establishing additional partnerships in territories that we do not intend to pursue ourselves. Furthermore, contingent on the success of the ZoptEC programme, we have additional areas of interest for further therapeutic development, including ovarian, prostate and triple negative breast cancer. Our commitment is to provide therapies to patients and their physicians that can potentially improve and extend the quality of lives.”

The ZoptEC phase 3 trial is an open-label, randomized-controlled study, comparing the efficacy and safety of zoptarelin doxorubicin, a hybrid molecule composed of a synthetic peptide carrier and a well known chemotherapy agent, doxorubicin, to doxorubicin alone. It is being conducted under a Special Protocol Assessment with the US Food and Drug Administration (FDA). Patients are centrally randomized in a 1:1 ratio and receive either zoptarelin doxorubicin (267 mg/m2) or doxorubicin (60 mg/m2) intravenously, every 3 weeks and for up to 9 cycles. Response will be evaluated every 3 cycles during treatment, thereafter, every 12 weeks until progression. All patients will be followed for survival as the primary efficacy endpoint (EP). Secondary EPs include progression free survival, objective response-rate, and clinical benefit rate.

Zoptarelin doxorubicin represents a new targeting concept in oncology using a hybrid molecule composed of a synthetic peptide carrier and a well-known chemotherapy agent, doxorubicin. Zoptarelin doxorubicin is the first intravenous drug in advanced clinical development that directs the chemotherapy agent specifically to LHRH-receptor expressing tumours, which could result in a more targeted treatment with less damage to healthy tissue. The company is currently conducting a ZoptEC phase 3 trial in women with advanced, recurrent or metastatic endometrial cancer, while zoptarelin doxorubicin is also in an investigator initiated phase 2 trial in prostate cancer. Aeterna Zentaris owns the worldwide rights to this compound except in China (including Hong Kong and Macau) where rights have been out-licensed to Sinopharm A-Think Pharmaceuticals, a subsidiary of Sinopharm, the largest medical and healthcare group in China and on Fortune's Global 500 list. On April 16, 2015, thecCompany announced the filing of a patent application intended to strengthen the exclusivity of zoptarelin doxorubicin through a unique, significantly lower cost in the manufacturing process.

Endometrial cancer is the most common gynecologic malignancy in developed countries 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. According to the American Cancer Society, there will be approximately 55,000 new cases of endometrial cancer in the US alone in 2015, with about 20 per cent of recurring disease.