Azixa™

Description

EP90745 is a series of apoptosis inducer compounds for the treatment of cancer.  In August 2006, positive clinical results were reported for Azixa™, the lead compound in the series, in a total of 66 patients – the first confirmed clinical proof of concept for EpiCept’s proprietary live cell high-throughput caspase-3 screening technology. In a Phase I study of Azixa™ in cancer that has metastasized to the brain, the drug achieved its maximum tolerated dose in patients. A measured reduction in tumor size was also noted in certain patients, suggesting evidence of activity.

In November 2009, interim results were reported for a clinical trial of Azixa™ in melanoma metastases in which ten of the 22 patients treated achieved stable disease and two patients achieved confirmed partial responses. The median progression-free survival was 2.8 months.

In June 2010, an update was provided at the meeting of the American Society of Clinical Oncology (ASCO).  In the glioblastoma trial, six subjects achieved stable disease and two subjects had achieved partial responses.  One subject’s partial response duration was 7.8 months; the additional patient’s response was, at the time of the ASCO report, 16 months in duration and has been classified as almost a complete response.

In November 2010, a poster was presented with updated results from an ongoing open–label Phase II monotherapy study of Azixa™ in treatment-experienced patients with glioblastoma multiforme (GBM) at the Society for Neuro-Oncology Scientific Meeting and Education Day in Montreal Canada. The poster focused on the subset of patients with the poorest prognosis, whose disease progressed following both first and second line treatments, including Avastin®. Azixa™ monotherapy was well-tolerated and demonstrated anti-tumor activity with a mean overall survival of 105 days in patients who had failed prior Avastin® (bevacizumab) treatment. In this cohort, one patient, in his twelfth month of Azixa™ treatment and whose treatment is continuing, has seen tumor regression of more than 80%, and four additional patients experienced stable disease.

In December 2010, a controlled two-arm Phase IIb study of Azixa™ was initiated as a front-line treatment for GBM.  The study was to enroll up to 120 newly diagnosed GBM patients in the U.S. and India in order to evaluate Azixa™ combination therapy as a first line GBM treatment.  The trial was to compare standard of care in this disease with standard of care in combination with Azixa™.

Azixa™ has received orphan drug status in the United States for the treatment of glioblastoma.

Stage of Development

In February 2012, development activities were suspended.  The Company currently has no plans to pursue further development of Azixa™ on its own.