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ChemGenex Launches Multi-Site Omacetaxine Clinical Trial in AML Patients

11 / 03 / 2008

ChemGenex Pharmaceuticals (ASX: CXS, NASDAQ: CXSP) announced today that it has launched a new phase 2 study evaluating the use of omacetaxine mepesuccinate (formerly homoharringtonine, HHT) in refractory or relapsed acute myeloid leukemia (AML) patients who have failed intensive chemotherapy. Initiation of the AML study represents the next step in ChemGenex's development strategy for omacetaxine, complementing the company's two ongoing clinical studies in chronic myeloid leukemia (CML).

The study will be conducted in France and is designed as a two-stage study with potential enrolment of up to 27 patients. The primary endpoint will be the proportion of patients achieving complete and partial remissions, and the secondary endpoints will include survival.

AML is a cancer of the myeloid line of white blood cells, characterized by the rapid proliferation of abnormal cells that accumulate in the bone marrow and interfere with the production of normal blood cells. AML is the most common acute leukemia affecting adults with an estimated 30,000 new cases per year in the developed world, more than twice that of CML. The incidence of AML is increasing with increased life expectancy and ageing of the general population.

Despite significant advances in cancer therapy over the past twenty years the treatment outcomes for AML patients, particularly those over 60 years old, have not improved and the overall survival rate is poor. Omacetaxine's novel mechanism of action is distinct from existing AML therapies and therefore offers a potential new approach to the treatment of the growing number of AML patients with poor clinical prognoses.
"There is a clear need for improved therapeutic treatments for AML patients who have relapsed or progressed after first line therapies, and we believe that omacetaxine may offer a new option for these patients," said Greg Collier, Ph.D., Chief Executive Officer and Managing Director of ChemGenex.

"Our understanding of the mechanism of action of omacetaxine, along with previous clinical experience in a range of leukemias, particularly in CML patients, provides a strong basis for developing omacetaxine for the treatment of AML."

Clinical Trial Details

The trial is a pilot open-label study conducted in two hospitals in France; Edouard Herriot Hospital in Lyon and Andre Mignot Hospital in Versailles.

In the induction phase, patients will receive 2.5 mg/m2 omacetaxine by subcutaneous injection twice a day for 9 consecutive days, with cycles repeated every 28 days. In the maintenance phase, patients will receive 1.25 mg/m2 omacetaxine by subcutaneous injection twice a day for 7 consecutive days, with cycles repeated every 28 days. The study will be conducted in two stages, using a Simon two-stage study design. During the first stage, 13 evaluable patients will be enrolled. If one or more responses are seen during the first stage, another 14 evaluable patients will be enrolled in the second stage. It is anticipated that a maximum of 27 patients will be enrolled into the study. The primary and secondary endpoints are response rates and survival.

About Acute Myeloid Leukemia

Acute myeloid leukemia (AML), also known as acute myelogenous leukemia, is a cancer of the myeloid line of white blood cells, characterized by the rapid proliferation of abnormal cells which accumulate in the bone marrow and interfere with the production of normal blood cells. AML is the most common acute leukemia affecting adults, and its incidence increases with age.

The effects of AML include a drop in red blood cells, platelets, and normal white blood cells. The symptoms include shortness of breath, fatigue, easy bruising and bleeding, and increased infection risk. Whilst several risk factors for AML have been identified, the specific cause of the disease is unknown. AML typically progresses rapidly without therapeutic intervention and can be fatal within months.

Acute myeloid leukemia is a potentially curable disease; but only a minority of patients are cured