Ligand Ally GSK Kick-Starts Eltrombopag Phase 3 Study In MDS

Ligand Pharmaceuticals reported that its collaborator GlaxoSmithKline has initiated a Phase III study investigating the platelet supportive care effect of eltrombopag in combination with current standard of care azacitidine in intermediate-1, intermediate-2 or high-risk patients with myelodysplastic syndromes (MDS).

The global, randomized, placebo controlled, multi-center, double blind, Phase III SUPPORT (StUdy of eltromboPag in myelodysPlastic SyndrOmes Receiving azaciTidine) study will investigate eltrombopag or placebo in combination with azacitidine in MDS patients. The trial will enroll 350 patients with a baseline platelet count of < 75Gi/L across 156 sites in 30 countries. SUPPORT’s primary efficacy endpoint will assess the effect of eltrombopag with azacitidine on platelets in patients who will are platelet transfusion free during the first 4 cycles of azacitidine. Secondary endpoints will measure overall survival, hematologic improvement, disease response, platelet and red blood cell transfusions, safety and tolerability, and adverse events among others between the two treatments.

Eltrombopag, marketed as Promacta in the U.S. and as Revolade in the EU, interacts with the TPO receptor, which leads to increased platelet production.

Myelodysplastic syndrome is a type of cancer wherein the bone marrow fails to sufficiently produce healthy blood cells and abnormal blast cells are present in the blood and/or bone marrow. MDS manifests with one or more cytopenias (reduction in number of blood cells). Patients with MDS often experience anemia-related complications, infections, or bleeding. The disease may later develop into acute myeloid leukemia (AML) in up to 45 percent of patients.

John Higgins, President and CEO of Ligand Pharmaceuticals, said, “With the start of the SUPPORT trial GSK continues to advance eltrombopag in multiple indications to boost platelets to treat thrombocytopenia, including clinical programs and regulatory approvals in oncology and infectious disease indications… The SUPPORT trial is one of more than 25 active clinical trials underway with eltrombopag.”