Novacea, Inc. announced the initiation of a phase II trial of AQ4N (banoxantrone), an investigational anti-cancer prodrug, in patients with relapsed or refractory acute lymphoblastic leukaemia (ALL).
This phase II multi centre, single arm, open-label study will enrol approximately 56 evaluable patients. The primary efficacy objective is the response rate (confirmed complete response or confirmed complete response without platelet recovery) achieved over 3 cycles of AQ4N. The secondary efficacy objectives are duration of response and overall survival.
Additionally, the study will evaluate the safety and tolerability of AQ4N as a monotherapy for patients with ALL, whose disease had either relapsed or were refractory to one or more prior chemotherapy regimens, the company said in a recent press release.
"As part of our expanded development plans for AQ4N, we believe the product candidate has the potential to help patients with acute lymphoblastic leukaemia, a devastating disease with limited treatment options. AQ4N is a proprietary anti-cancer prodrug with a novel mechanism of action. In pre-clinical models, we have observed that the activated AQ4 metabolite demonstrated potent cytotoxicity against tumour cells derived from haematological malignancies, including those from ALL and other forms of leukaemia," said John G. Curd, M.D., president and chief medical officer, Novacea.
Acute lymphoblastic leukaemia (ALL) is a type of cancer that starts from white blood cells in the bone marrow. According to the American Cancer Society, in 2007 there will be about 44,000 new cases of all types of leukaemia in the United States. Of these, about 5,200 will be ALL. Although this disease primarily affects children, about 1,100 cases will be in adults. Approximately 1,400 people will die of ALL in the United States in 2007; about two-thirds of them will be adults.
Novacea is developing AQ4N as a novel, tumour-selective prodrug with applicability to multiple tumour types, both in combination with a number of chemotherapeutic agents and as a monotherapy for haematological malignancies. AQ4N is an investigational prodrug designed to address the challenges of treating certain cancers with significant hypoxic (oxygen-starved) regions. AQ4N is selectively bio reduced by cytochrome P450 to AQ4, a potent DNA intercalator and topoisomerase II inhibitor. Preclinical studies demonstrate that AQ4N selectively targets lymphoblastoid cell lines as well as the hypoxic portions of solid tumours.
AQ4N was originally discovered by Professor Laurence Patterson, Ph.D., currently director of the Institute of Cancer Research at the University of Bradford in England, working in collaboration with the intellectual property and a life sciences company, BTG International Limited.
Novacea, Inc. is a biopharmaceutical company focused on in-licensing, developing and commercializing novel cancer therapies. Novacea has two product candidates in clinical trials, including Asentar, which currently is in a phase III clinical trial for androgen-independent prostate cancer, or AIPC, and a phase II study for advanced pancreatic cancer. Asentar is the subject of the development and commercialization agreement with Schering-Plough. Novacea's second product candidate, AQ4N, is a hypoxia-activated prodrug that is currently in a phase 1b/2a clinical trial in glioblastoma multiforme.