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ChemGenex Announces Second Publication Demonstrating Activity of Ceflatoninr in T315I-Positive Chronic Myeloid Leukemia (CML)

24 / 09 / 2007

ChemGenex Pharmaceuticals Limited (ASX:CXS and NASDAQ:CXSP) announced today the
publication of a clinical communication in Blood (the journal of the American Society of Hematology) demonstrating clinical activity of the companys lead compound, Ceflatoninr (Homoharringtonine, HHT1) against Gleevecr (imatinib mesylate)-resistant, accelerated phase chronic myeloid leukemia (CML) associated with the T315I BCR-ABL kinase domain mutation.


The publication describes positive clinical outcomes for two CML patients with the T315I BCR-ABL kinase domain mutation, one of whom was treated with Ceflatonin, the other was treated with interferon-alpha. The T315I BCR-ABL mutation results in resistance to tyrosine kinase inhibitor (TKI) drugs, including Gleevec, Sprycelr (dasatinib), and Tasignar (nilotinib) that are currently used to treat CML patients. As a result, therapeutic options for CML patients with this mutation are very limited, and there is a significant unmet medical need for novel therapies to treat these patients.


The patient treated with Ceflatonin was resistant to Gleevec and Sprycel and had progressed to accelerated-phase CML with 100% of BCR-ABL positive cells showing the T315I mutation. The authors, Dr. Hugues de Lavallade and his associates, including Professor John M. Goldman and Dr. David Marin of the Hammersmith Hospital and Imperial College, London, UK, reported that the patient experienced a 30% reduction of T315I BCR-ABL levels within one month of the initiation of Ceflatonin treatment and a complete hematological response after five months.


The Blood publication follows a clinical report in Leukemia in May, 2007, describing clinical activity for Ceflatonin in another CML patient harboring the T315I BCR-ABL kinase domain mutation. Together, these two reports strongly support ChemGenexs clinical and regulatory approval program. ChemGenex is currently undertaking two phase 2/3 clinical trials in CML - the first trial includes Gleevec-resistant patients with the T315I BCR-ABL kinase domain mutation, and the second trial is for CML patients who are resistant to treatment with two or more of the current TKI drugs available. Positive results from the two trials may support the filing of a New Drug Application (NDA) with the US Food and Drug Administration and other regulatory authorities.