MethylGene Inc.

MethylGene Inc.

December 11, 2006 07:30 ET

Positive Data Reported at ASH Annual Meeting for MethylGene's MGCD0103, an Oral, Isotype-Selective HDAC Inhibitor

Interim Results Report Seven Responders in Phase I/II Clinical Trial Evaluating MGCD0103 and Vidaza'R' Combination Phase II Portion to Commence in First Quarter 2007

MONTREAL, QUEBEC--(CCNMatthews - Dec. 11, 2006) - MethylGene Inc. (TSX:MYG), along with its partner Pharmion Corporation (NASDAQ:PHRM), today announced clinical data from its Phase I/II combination trial with Vidaza® (Trial 005) and from a Phase I single-agent trial (Trial 004) for its isotype-selective, histone deacetylase (HDAC) inhibitor, MGCD0103, as well as a brief update for its other MGCD0103 clinical trials. The clinical data was presented at two poster sessions at the 48th Annual Meeting of the American Society of Hematology (ASH) in Orlando on Sunday, December 10th, 2006.

Phase I/II HDAC poster (Trial 005): "Phase I/II study of the oral isotype-selective histone deacetylase (HDAC) inhibitor MGCD0103 in combination with azacitidine in patients with high-risk Myelodysplastic Syndromes (MDS) or Acute Myelogenous Leukemia (AML)" described interim results for 23 patients with MDS or AML at dose levels of MGCD0103 ranging from 35 to 135 mg. Dosing is currently ongoing at the approximate maximum tolerated dose of single agent MGCD0103 (110 mg) administered orally three times a week without interruption. Azacitidine (Vidaza®) is administered at its approved dose and schedule of 75mg/m2 SC daily x 7 every 28 days.

Seven out of 23 evaluable patients, in the dose escalation Phase I portion of the trial, achieved responses (7 out of 20 at previously demonstrated active doses). Three patients achieved complete response (CR) consisting of normalization of bone marrow blasts and peripheral blood counts, three patients experienced complete bone marrow response without peripheral platelet recovery (CR-p) or neutrophil recovery and one patient achieved a partial response (PR) which involved partial recovery of the bone marrow along with full recovery of platelets and neutrophils in the peripheral blood. A total of six evaluable patients, including four of the responding patients, remain on study and continue to receive the combination therapy. A majority of patients exhibited substantial reduction in HDAC activity during treatment with the combination. Analysis of DNA methylation is ongoing. The combination of azacitidine (Vidaza®) with MGCD0103 has encouraging safety, PK and clinical activity profiles and appears to be well-tolerated in patients with advanced AML/MDS. Three dose limiting toxicity events which were gastrointestinal in nature were reported. Two of the three events occurred at the highest dose level of 135 mg of MGCD0103. Once the maximum tolerated dose (MTD) of MGCD0103 in this combination is documented, the study will expand and continue as a Phase II study in this patient population. This is expected to occur in the first quarter of 2007. Preliminary PK data indicate the absence of drug-drug interactions as the half lives for both azacitidine (Vidaza®) and MGCD0103 appear to be unaffected by the combination.

Phase I HDAC poster (Trial 004): "A Phase I Study of MGCD0103 Given as a Twice Weekly Oral Dose in Patients with Leukemia (AML, ALL or CML) or Myelodysplastic Syndromes (MDS)" described results for MGCD0103 in 19 patients treated at doses levels of 40, 53, 66 and 83 mg/m2/day. Treatment has been well-tolerated to date and the maximum tolerated dose has not yet been reached. The twice weekly schedule is supported by preclinical and clinical data suggesting that MGCD0103 promotes H3/H4 acetylation in peripheral blood mononuclear cells (PBMC) that continues beyond 24 hours, thus providing the opportunity for intermittent administration. Significant inhibition of total HDAC enzyme activity in PBMCs was observed in the majority of patients. Four patients experienced stable disease. The most frequent documented side effects are fatigue and gastro-intestinal in nature. Dose escalation continues in this patient population.

"We remain encouraged by the accumulating data from these and other trials regarding the potential utility of MGCD0103 in cancer, both as a single agent and in combination with other cancer agents," commented Donald F. Corcoran, President and Chief Executive Officer of MethylGene. "Based on these data, we will continue to increase our investment in MGCD0103 clinical trials, with the goal of identifying a potential registrational pathway."

Update on the Advancement of Ongoing MGCD0103 Clinical Trials

Trial 008: This Phase II single-agent trial in patients with relapsed or refractory B-cell lymphomas, specifically diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma, is ongoing. Preliminary and interim data from this trial are expected to be disclosed during ASCO or at other scientific venues during the first half of 2007.

Trial 010: This ongoing Phase II single-agent trial in patients with relapsed on refractory Hodgkin's lymphoma has completed Stage One and rapidly progressed to Stage Two of the protocol based on preliminary evidence of safety and activity in this patient population. Preliminary and interim data from this trial are also expected to be disclosed during ASCO or at other scientific venues during the first half of 2007.

Other Ongoing MGCD0103 Clinical Trials

Trial 006: Phase I/II clinical trial of MGCD0103 in combination with Gemzar® in patients with solid tumors and pancreatic cancer continues to enroll patients in the dose-escalation phase.

Trial 007: Phase II single-agent clinical trial of MGCD0103 in patients with high-risk myelodysplastic syndromes (MDS) or acute myelogenous leukemia (AML) was initiated in December 2006.

About MethylGene

MethylGene is a publicly-traded biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics in cancer. Two cancer product candidates are currently in clinical development: MGCD0103, partnered with Pharmion Corporation and Taiho Pharmaceutical Co., Ltd., and MG98, partnered with MGI Pharma, Inc. MethylGene has an exclusive license agreement with Merck & Co. for the development and commercialization of small molecule beta-lactamase inhibitors to overcome antibiotic resistance. MethylGene has partnered its non-oncology HDAC program for neurodegenerative diseases with EnVivo Pharmaceuticals. MethylGene has a portfolio of preclinical programs for its multi-targeted kinase and histone deacetylase (HDAC) inhibitors for both oncology and non-oncology indications, and continues to seek partnering opportunities in these areas. 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 MG98; 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 and MG98. 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 2005, under the heading "risk factors," 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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