MethylGene Inc.

MethylGene Inc.

July 12, 2007 07:30 ET

MethylGene Initiates Single-Agent Phase II Clinical Trial With its HDAC Inhibitor, MGCD0103, in Patients With Refractory Chronic Lymphocytic Leukemia

MONTREAL, QUEBEC--(Marketwire - July 12, 2007) - MethylGene Inc. (TSX:MYG), along with its partner Pharmion Corporation (NASDAQ:PHRM), today announced the initiation of a single-agent Phase II clinical trial with its isotype-specific histone deacetylase (HDAC) inhibitor product candidate, MGCD0103, in patients with refractory chronic lymphocytic leukemia (CLL). Candidates for this study are adult patients who have refractory CLL or who are not candidates for current standard treatment options.

This open-label trial (Trial 009) will enroll up to 40 patients and be conducted at leading cancer centers in North America. MGCD0103 will be administered orally, three times per week (four weeks per cycle). MGCD0103 may also be studied in combination with rituximab, a currently marketed therapy for non-Hodgkin's lymphoma, in patients whose disease continues to progress.

Key objectives of the study will be to determine the overall response rate (complete and partial responses) of MGCD0103 as a treatment option for patients with refractory chronic lymphocytic leukemia. Secondary objectives include determining the safety profile, as well as assessing biomarkers and predictive markers for MGCD0103. The trial is expected to last up to 24 months.

Leukemias arise through several cellular events, including mutation and/or epigenetic silencing of tumor suppressor genes. The HDAC-dependent repression of transcription (which appears to occur in leukemias) is believed to be an important anticancer mechanism for HDAC inhibitors. HDAC inhibition induces cell death in CLL cells and reduces leukemic cell counts in patients with CLL.

"This trial marks the investigation of another potential indication for which MGCD0103 may be effective. We currently have four single-agent Phase II trials and two combination Phase I/II trials ongoing," said Donald F. Corcoran, President and Chief Executive Officer of MethylGene. "In June at the American Society of Clinical Oncology (ASCO) Annual Meeting, we reported encouraging preliminary results for two clinical trials in hematological indications, Hodgkin's lymphoma, myelodysplastic syndromes and acute myelogenous leukemia. Therefore, we are pleased to have this trial underway and are looking forward to seeing how MGCD0103 performs in refractory CLL patients."

"The histone deacetylase enzymes represent a novel target to inhibit in CLL and multiple preclinical studies suggest this approach should be successful. MGCD0103 has already demonstrated clinical activity in other hematologic malignancies and has several favorable properties as a therapeutic for CLL. Our hematologic malignancy group is incredibly excited about the opportunity to be a part of the preclinical and clinical development of MGCD0103 in the new CLL study in addition to the ongoing acute myelogenous leukemia trial," said Dr. John C. Byrd, D. Warren Brown Professor of Leukemia Research, Professor of Medicine and Medicinal Chemistry, Director of Hematologic Malignancies, Co-Director of the Division of Hematology-Oncology, Department of Medicine at The Ohio State University and a principal investigator for this trial.

About MGCD0103, an Oral HDAC Inhibitor

MGCD0103 is a rationally designed, oral, isotype-specific histone deacetylase (HDAC) inhibitor. MethylGene and Pharmion continue to enroll patients in a Phase I/II combination trial with the demethylating agent Vidaza® (azacitidine for injection, marketed by Pharmion) in myelodysplastic syndromes (MDS) and acute myelogenous leukemia (AML) (Trial 005); a Phase I/II trial in combination with Gemzar® in solid tumors and pancreatic cancer (Trial 006); a Phase II single-agent trial in high-risk MDS and AML (Trial 007); a Phase II single-agent trial in refractory or relapsed diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma (Trial 008); a Phase II single-agent trial in chronic lymphocytic leukemia (CLL) (Trial 009); and a Phase II single-agent trial in refractory or relapsed Hodgkin's lymphoma (Trial 010).

About Chronic Lymphocytic Leukemia (CLL)

CLL is a cancer of the blood and bone marrow. The body's bone marrow produces stem cells which eventually mature into red blood cells, white blood cells and platelets. In CLL, too many stem cells mature into malignant lymphocytes - a type of white blood cell that is less effective in fighting infections. As their numbers increase in the blood and bone marrow, there is less room for the healthy blood cells. This imbalance eventually renders the body unable to effectively cope with infections.

According to the American Cancer Society, CLL is the second most common type of leukemia in the U.S. with approximately 15,340 new cases expected to be diagnosed and an estimated 4,500 deaths in 2007. Ninety percent of CLL cases are diagnosed in people over the age of 50, with 65 being the average age of diagnosis.

About MethylGene

MethylGene Inc. (TSX:MYG) is a publicly-traded biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for cancer. The Company focuses on some of the most promising areas of oncology such as histone deacetylase (HDAC) and kinase inhibitors. The Company's lead product is MGCD0103, an oral isotype-selective HDAC inhibitor presently in multiple clinical trials for solid tumors and hematological malignancies, including Phase II monotherapy and Phase I/II combination trials with Vidaza® and Gemzar®. MGCD265 is an oral kinase inhibitor targeting the c-Met, Tie-2, Ron and VEGF receptor tyrosine kinases. In addition, MethylGene has several preclinical non-oncology programs: MGCD290, an HDAC inhibitor in combination with azoles for fungal infections; an HDAC program for Huntington's disease; and a beta-lactamase program to overcome antibiotic resistance. MethylGene's development and commercialization partners include Pharmion Corporation, Taiho Pharmaceutical, Merck and EnVivo Pharmaceuticals. Please visit our website at

Certain statements contained in this news release, other than statements of fact that are independently verifiable at the date hereof, may constitute forward-looking statements. Such statements, based as they are on the current expectations of management of MethylGene, inherently involve numerous risks and uncertainties, known and unknown, many of which are beyond MethylGene's control. These risks and uncertainties could cause future results, performance or achievements to differ significantly from the results, performance or achievements expressed or implied by such forward-looking statements. Such results, performance or achievements include, but are not limited to, the timing and effects of regulatory action; the continuation of collaborations; the results of clinical trials; the timing of enrollment or completion of clinical trials; the success, efficacy or safety of MGCD0103, MGCD265 or MGCD290; the ability to scale up, formulate and manufacture sufficient GMP, clinical or commercialization quantities of MGCD0103, MGCD265 or MGCD290, and the relative success or the lack of success in developing and gaining regulatory approval and/or market acceptance for any compound or new product including MGCD0103, MGCD265 or MGCD290. Such risks include, but are not limited to, the impact of general economic conditions, economic conditions in the pharmaceutical industry, changes in the regulatory environment in the jurisdictions in which MethylGene does business, stock market volatility, fluctuations in costs, expectations with respect to our intellectual property position and our ability to protect our intellectual property and operate our business without infringing upon the intellectual property rights of others, changes in the competitive landscape including changes in the standard of care for the various indications in which MethylGene is involved, and changes to the competitive environment due to consolidation, as well as other risks, which you are urged to read, as described in MethylGene's Annual Information Form for the fiscal year ending December 31, 2006, under the heading 'risk factors,' the final prospectus filed on February 23, 2007, and all other documents filed by the Company that can be found at Consequently, actual future results may differ materially from the anticipated results expressed in the forward-looking statements. The reader should not place undue reliance on the forward-looking statements included in this presentation. These statements speak only as an update on the date they are made and MethylGene is under no obligation to revise such statements as a result of any event, circumstance or otherwise except in accordance with law.

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