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Therapeutic efficacy of Pomalidomide

1. Multiple Myeloma

Study 1

Study 1 was a Phase 2, multicenter, randomized, open-label study in patients with relapsed multiple myeloma who were refractory to their last myeloma therapy and had received prior treatment with lenalidomide and bortezomib. A total of 221 patients were randomized to receive pomalidomide alone or pomalidomide in combination with low-dose dexamethasone. In Study 1, the efficacy and safety of pomalidomide 4 mg once daily, administered on Days 1 through 21 of each 28-day cycle until disease progression, were evaluated alone and in combination with low-dose dexamethasone.

Baseline demographics and disease characteristics were balanced and comparable between study arms. The median patient age was approximately 61–64 years, about half were male, the majority were White, and most had an ECOG performance status of 0–1. The median number of prior lines of therapy was 5, approximately 75% had received a stem cell transplant, and approximately 60% were refractory to both bortezomib and lenalidomide.

The results of Study 1 are summarized as follows: the overall response rate (ORR) was 7.4% in the pomalidomide alone group and 29.2% in the pomalidomide plus low-dose dexamethasone group. The median duration of response (DOR) for the pomalidomide plus low-dose dexamethasone group was not reached.

Study 2

Study 2 was a Phase 3, multicenter, randomized, open-label study comparing pomalidomide plus low-dose dexamethasone to high-dose dexamethasone in adult patients with relapsed and refractory multiple myeloma who had received at least two prior regimens, including lenalidomide and bortezomib, and had demonstrated disease progression on or within 60 days of completion of the last therapy. A total of 455 patients were enrolled in the study.

Baseline patient and disease characteristics were balanced and comparable between study arms. Overall, 94% of patients were refractory to lenalidomide, 79% were refractory to bortezomib, and 74% were refractory to both lenalidomide and bortezomib.

The results of Study 2 are summarized as follows: median progression-free survival (PFS) was significantly longer in the pomalidomide plus low-dose dexamethasone group compared with the high-dose dexamethasone group. Median overall survival (OS) was also significantly longer in the pomalidomide plus low-dose dexamethasone group. The overall response rate was 23.5% in the pomalidomide plus low-dose dexamethasone group compared to 3.9% in the high-dose dexamethasone group.

2. Kaposi Sarcoma

Clinical Trial 12-C-0047 was an open-label, single-center, single-arm clinical study evaluating the safety and efficacy of pomalidomide in patients with Kaposi sarcoma. A total of 28 patients received pomalidomide 5 mg orally once daily. All HIV-positive patients continued highly active antiretroviral therapy (HAART). The study excluded patients with symptomatic pulmonary or visceral Kaposi sarcoma, a history of venous or arterial thromboembolism, or a procoagulant disorder. Patients received aspirin 81 mg once daily throughout the treatment period as thromboprophylaxis.

The median age was 52.5 years, all were male, 75% were White, and 14% were Black or African American. At enrollment, 75% of patients had advanced disease, 11% had ≥50 lesions, and 75% had received prior chemotherapy.

The primary efficacy endpoint was overall response rate (ORR), including complete response (CR), clinical complete response (cCR), and partial response (PR). The median time to first response was 1.8 months. Efficacy results are summarized as follows: the overall response rate was 71% for all patients, 67% for HIV-positive patients, and 80% for HIV-negative patients. The median duration of response for all patients was 12.1 months.