Rationale and study design

O-12-M1 is an international, multicenter, open-label phase 1/2 study, run in Europe and USA, to establish the dose of melflufen in combination with dexamethasone and investigate the treatment response in patients with relapsed refractory multiple myeloma, who were refractory to the last line of therapy.

The Phase 1 part established 40 mg melflufen+dexamethasone as the recommended Phase 2 dose. The phase 2 part included 45 relapsed refractory multiple myeloma patients who were heavily pre-treated. They had received a median of 4 prior lines of therapy and were refractory to lenalidomide (an immunomodulatory drug, IMiD) and bortezomib (a proteasome inhibitor, PI).

The study started mid 2013, completed the enrollment of patients in late 2016 and presented final results in 2017.