The phase 2 HORIZON study is a pivotal, single-arm, multicenter, phase 2 study evaluating the safety and efficacy of melflufen in combination with dexamethasone in patients with relapsed refractory multiple myeloma. The study included 157 heavily pretreated patients, who had received >2 earlier lines of therapy with immunomodulatory agents (IMiDs) and proteasome inhibitors (PIs) and were refractory to pomalidomide and/or daratumumab.
Patients who have failed lenalidomide and proteasome inhibitor treatment and are refractory to pomalidomide and/or daratumumab have limited or no treatment options left. The introduction of a new treatment class with a novel compound, may represent a potentially important therapeutic alternative.
The complete top-line results from the pivotal phase 2 HORIZON study were presented at the European Hematology Association meeting, in June 2020. The HORIZON study started in Q1 2017.