Clinical data of pomalidomide for treating patients with multiple myeloma presented at ASH 2010

Celgene International Sàrl (NASDAQ: CELG) today announced that its next IMiD^® compound, pomalidomide, was evaluated in three separate investigational trials of patients with multiple myeloma who were relapsed or refractory after prior treatments including lenalidomide, thalidomide and bortezomib. The clinical data were presented at the 52^nd Annual Meeting of the American Society of Hematology in Orlando, Florida.

Abstract #859 - Phase 2 Study of 2 Modalities of Pomalidomide (CC4047) Plus Low-Dose Dexamethasone as Therapy for Relapsed Multiple Myeloma, IFM 2009-02

In this first study, presented by Xavier Leleu, M.D., of the Intergroupe Francophone du Myelome, patients with disease that was refractory to lenalidomide and bortezomib were enrolled and received 4mg of pomalidomide on days 1-21 of each 28-day cycle (arm A) or continuously on days 1-28 of each 28-day cycle (arm B). Dexamethasone was given orally at 40 mg daily on days 1, 8, 15 and 22 of each cycle to all patients.

In arm A, the overall response rate (ORR) was 42% (18/43), including 7% (3/43) of patients achieving a very good partial response (VGPR) and 2% (1/43) achieving a complete response (CR). In arm B, the ORR was 39% (16/41) with 5% (2/41) of patients achieving a VGPR and no patients achieving a CR. The median durations of response were 119 (28-280) days and 126 (28-280) days in arms A and B, respectively. Median event-free survival times were 219 and 152 days for arms A and B, respectively.

Toxicity consisted primarily of myelosuppression in both arms. The occurrence of neuropathy, worsening of pre-existing neuropathy or thromboembolic events was not observed.

Abstract #863 - Pomalidomide Plus Low-Dose Dexamethasone in Myeloma Refractory to Both Bortezomib and Lenalidomide: Comparison of Two Dosing Strategies in Dual-Refractory Disease

In a Phase 2 study presented by Martha Lacy, M.D., of the Mayo Clinic, two different doses of pomalidomide (2 mg daily and 4 mg daily in 28-day cycles) with low dose dexamethasone 40 mg (days 1, 8, 15 and 22 of each 28-day cycle) were studied. Patients with disease refractory to both lenalidomide and bortezomib were enrolled in each cohort of this study. The median number of previous multiple myeloma treatments received was six in both arms. In addition more than 40% of patients in both arms had poor prognostic molecular markers.

Objective responses (minor response or better) were seen in 49% (17) and 40% (14) of patients in the 2 mg and 4 mg groups, respectively, with PR or better being observed in 26% (9) of patients in each group. The majority of patients remain in disease remission at time of reporting. Overall survival at six months was 78% (95% CI: 65-95) and 69% (95% CI: 53-89) for the 2 mg and 4 mg groups, respectively.

Overall, grade 3 or 4 haematological adverse events were seen in 49% and 66% of patients in the 2 mg and 4 mg groups, respectively, with the most common grade 3 and 4 adverse events being neutropenia, thrombocytopenia and anaemia. Cases of neuropathy possibly attributed to pomalidomide treatment were seen in 20% (7) and 29% (10) of patients in the 2 mg and 4 mg groups, respectively.

Abstract #864 Study to determine the maximum tolerated dose (MTD) of pomalidomide alone or in combination with low-dose dexamethasone in patients with relapsed and refractory myeloma

In the third study, presented by Paul G. Richardson, M.D., of the Dana-Farber Cancer Institute, patients with disease refractory to lenalidomide and bortezomib were first evaluated to determine the maximum tolerated dose (MTD) of pomalidomide in the phase 1 portion of the trial which was followed by an ongoing phase 2 open-label portion comparing patients receiving pomalidomide plus dexamethasone versus pomalidomide alone. The median number of previous multiple myeloma treatments received was six.

Thirty-eight patients were enrolled in the phase 1 phase of the study, and the MTD was determined to be 4 mg per day on days 1-21 of a 28-day cycle.

Of the 28 evaluable patients who were treated single-agent pomalidomide, objective responses (minor response or better) were observed in 50% (14/28) of patients. PR or better was seen in 25% (7/28), with one patient achieving a complete response. Median overall survival was 79.6 weeks (95% CI: 61.9 - NE).

During the study, 50% (19) of patients had at least one severe adverse event. The most common grade 3 or 4 adverse events were neutropenia (52%; 20), anaemia (21%; 8), fatigue (18%; 7) and thrombocytopenia (16%; 6).

Enrolment to the phase 2 portion of the study has followed and is now fully accrued>

These data are from investigational studies. Pomalidomide is not an approved product for any indication.