SUMMIT, N.J.--(BUSINESS WIRE)--Aug. 5, 2009-- Celgene Corporation (NASDAQ: CELG) today announced the presentation of final results from two company-sponsored studies of Amrubicin in relapsed small cell lung cancer (SCLC) at the 13^th World Conference on Lung Cancer in San Francisco, Calif. The presentations included safety and efficacy data of two phase II studies of the next-generation therapy in second-line patients with SCLC both sensitive and refractory to first-line platinum based chemotherapy, respectively. A third presentation detailed the results of a pooled cardiac safety analysis from the phase II studies.

Amrubicin is an anthracyline-like molecule with potent topoisomerase II inhibition and represents the first solid tumor-focused compound to complete phase II studies for Celgene.

The first study compared Amrubicin to topotecan, a common and approved treatment for SCLC, in patients with extensive-disease SCLC who were sensitive to first-line platinum-based chemotherapy. Dr. Robert Jotte, of the Rocky Mountain Cancer Centers in Denver, Colo., presented the results from the trial where patients were randomized to either 40 mg/m² IV of Amrubicin on days 1-3 of a 21-day cycle (n=50) or topotecan 1.5 mg/m² IV daily on days 1-5 of a 21-day cycle (n=26). Of the 76 patients enrolled in the study, 72 were treated.

The study demonstrated that the overall response rate (ORR) was significantly higher in patients treated with Amrubicin (44.0%) compared to patients treated with topotecan (11.5%) (p=0.005), meeting the primary endpoint of the study (to demonstrate an Amrubicin ORR of ≥ 25%). In addition, the complete response rate was 12% for the Amrubicin arm compared to 4% for the topotecan arm. Of note, for patients aged 65 years or older, the ORR for patients receiving Amrubicin was 46% compared to 7% for patients receiving topotecan.

The median overall survival for patients receiving Amrubicin was 9.3 months (95% CI, range 5.8-12.2) compared to 7.7 months for patients receiving topotecan (95% CI, range 4.5-14.0). Median progression-free survival was 4.6 months for the Amrubicin arm (95% CI:2.1, 6.1) compared to 3.3 months for the topotecan arm (95% CI: 2.2., 5.4) and the median time to progression was 5.6 months for the Amrubicin arm (95% CI: 2.8, 6.9) compared to 3.0 months for the topotecan arm(95% CI: 1.4, 4.4).

In the study, the most common grade 3 or higher adverse events for patients receiving Amrubicin compared to topotecan were neutropenia (61.2% vs. 78.3%, respectively), thrombocytopenia (38.8% vs. 60.9%) and leukopenia (38.8% vs. 39.1%). Dose reductions, primarily due to reversible myelosuppression, took place in 43% of Amrubicin patients and 44% of topotecan patients. Additionally, 4 patients in the Amrubicin arm and 1 patient in the topotecan arm died due to neutropenic complications.

In the second presentation, Dr. David S. Ettinger of the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University presented results from a study of Amrubicin monotherapy in patients with extensive disease SCLC refractory to first-line platinum-based chemotherapy.

In the study, 75 patients who had no response to first-line chemotherapy or progression within 3 months of treatment completion were enrolled. Of these, 69 received 40 mg/m2 IV of Amrubicin on days 1-3 of a 21-day cycle and treatment continued until disease progression, unacceptable toxicity or withdrawal.

The ORR was 21.3%, with 1 patient (1.3%) achieving a complete response. In patients who did not respond to platinum-based first line therapy (e.g. progressive or stable disease as best response), the ORR was 16%, and the response in elderly patients (≥ 65 years) was 20% which was similar to the overall population (21.3%). The median duration of response was 4.3 months (95% CI: 3.1, 5.8) and the median time to progression was 3.8 months (95% CI: 2.7, 4.2).

Additionally, median progression free survival for patients in the study was 3.3 months (95% CI: 2.5, 4.0) and median overall survival was 6.1 months (95% CI: 4.9, 7.2).

In the study, the most commonly occurring grade 3 or higher adverse events were neutropenia (66.7%), thrombocytopenia (40.6%), leukopenia (34.8%) and anemia (30.4%). Dose reductions occurred in 38% of patients and 3 patients experienced fatal serious adverse events that were not related to disease progression with 1 case considered possibly related to study treatment.

“Advances in small cell lung cancer are particularly relevant as there have been few new options for patients in recent years,” said Dr. Ettinger. “The data presented today show that Amrubicin is an active compound with a manageable safety profile in an area of significant unmet need. We look forward to the results of future studies in this disease with this drug.”

In the final presentation, Dr. David Spigel of the Sarah Cannon Research Institute in Nashville, Tenn. described data from a pooled study of the two phase II studies, attempting to confirm the cardiac safety profile of Amrubicin through an analysis of decreases in left-ventricular ejection fraction (LVEF). LVEF is a measure of cardiac contractility, measuring the fraction of blood pumped out of the left ventricle with each heart beat. One of the main disadvantages of the use of anthracyclines is the increased risk of heart damage and reduced LVEF with cumulative doses exceeding 450 mg/m².

The pooled analysis evaluated 117 patients treated with Amrubicin across the two trials. In the study, 115 patients had a baseline LVEF percentage recorded (median 60%: range 50-85) and were evaluated based on changes in LVEF level from this mark, as well as according to cumulative Amrubicin dosing range.

The study demonstrated minimal changes in LVEF percentage from the baseline, and the median LVEF figures were similar across all cumulative dosing ranges, including patients who received a cumulative dose >1000 mg/m² of Amrubicin (n=16), where the median change in LVEF from the baseline was 2%.

In the pooled study, cardio specific grade 3 or higher adverse events were acute myocardial infarction (n=2), atrial fibrillation (n=2) and atrial flutter (n=1). No treatment-related cardiac mortalities occurred on the study.

“The data from these studies are encouraging as they demonstrate that Amrubicin compares favorably to both historical data and currently utilized therapies across multiple patient groups, including elderly patients and those with poor prognostic factors,” said Dr. Jotte. “Additionally, these data suggest that Amrubicin has an improved cardiotoxicity profile compared to traditional anthracyclines. We believe these results warrant further study of Amrubicin in this hard to treat area of disease.”

About Amrubicin

Amrubicin is a third-generation, synthetic anthracycline analogue that has demonstrated substantial clinical efficacy in the treatment of small cell lung cancer. Amrubicin is a potent topoisomerase II inhibitor and is being studied as a single agent and in combination with anti-cancer therapies for a variety of solid tumors, including lung and breast cancers.

Amrubicin is currently approved and marketed in Japan for the treatment of both small cell lung cancer and non-small cell lung cancer by Nippon Kayaku, a Japanese pharmaceutical firm focused on oncology, which licensed Japanese marketing rights from Dainippon Sumitomo Pharma Co., Ltd. (DSP), the original developer of the therapy. DSP also licensed the North American and European Union rights of Amrubicin to Pharmion Corporation, which was acquired by Celgene Corporation in 2008.

About Small Cell Lung Cancer

Small cell lung cancer is a disease in which malignant cells form in the tissues of the lung, and which occurs almost exclusively in people who smoke. While small cell lung cancer constitutes approximately 15 percent of all lung cancers, SCLC tends to be more aggressive and fast growing than the more common non-small cell lung cancer. Of the estimated 65,000 patients diagnosed with SCLC each year in the US and EU, approximately 60 percent of patients have extensive disease at diagnosis, and the remaining 40 percent present with localized, or limited stage, disease.

About Celgene

Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global pharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation. For more information, please visit the Company's website at www.celgene.com.

This release contains forward-looking statements which are subject to known and unknown risks, delays, uncertainties and other factors not under the Company's control, which may cause actual results, performance or achievements of the Company to be materially different from the results, performance or other expectations expressed or implied by these forward-looking statements. These factors include results of current or pending research and development activities, actions by the FDA and other regulatory authorities, and other factors described in the Company's filings with the Securities and Exchange Commission such as our 10K, 10Q and 8K reports.

Source: Celgene Corporation

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