Celgene‘s immunomodulatory agent Revlimid (lenalidomide) significantly extended the time until disease progression in patients with smoldering multiple myeloma who were not yet experiencing any symptoms of disease, according to Phase 2/3 results announced by the company.
The open-label, randomized Phase 2/3 study (NCT01169337), led by the ECOG-ACRIN Cancer Research Group and sponsored by the National Cancer Institute (NCI), was aimed at evaluating treatment safety and feasibility in smoldering multiple myeloma patients with a high risk of progressing.
Smoldering multiple myeloma is a form of multiple myeloma that typically precedes the full-blown disease. These patients are usually asymptomatic and, in most cases, don’t require treatment until the disease has progressed. But in high-risk patients, researchers believe that biological therapies that stimulate the immune system could stop the cancer cells from growing.
Revlimid is an immunomodulatory agent commonly used for treating multiple myeloma but has not yet been assessed in patients with smoldering multiple myeloma.
“In multiple myeloma, there has been an important question regarding the risks and benefits of treating patients with smoldering myeloma. We believe that the properties of Revlimid could play an important role in delaying the progression of smoldering myeloma into active myeloma,” Jay Backstrom, chief medical officer for Celgene, said in a press release.
The trial was composed of two parts. In the Phase 2 part, researchers examined Revlimid’s safety and preliminary signs of efficacy.
A total of 44 patients were enrolled in this part of the study. All patients received 25 mg of Revlimid in 28-day cycles — 21 days of Revlimid plus seven days of 325 mg of aspirin — until disease progression to symptomatic myeloma or unacceptable toxicity.
Among the 44 patients assessed for toxicity, 11 experienced serious or life-threatening adverse events, with low neutrophil levels and fatigue being the most frequent. In addition, 33% of patients responded to treatment.
Then, in the Phase 3 part, researchers tested if Revlimid extended progression-free survival, or the time until disease worsening or death, compared with observation only. The study included 180 patients.
Celgene has now reported that, according to preliminary results, patients receiving Revlimid remained alive and without signs of disease progression for longer periods than those on observation alone.
So far, the safety profile of Revlimid has also been consistent with data from previous studies. Preliminary data from the trial will now be presented at an upcoming medical meeting.