Enrolment Begins for Elritercept Phase 3 Trial in Lower-Risk MDS Patients

Takeda Pharmaceuticals’ TAK-226-3001 trial, taking place at King’s College Hospital in London, is a phase 3 study comparing a novel treatment, elritercept, against the current standard treatment, epoetin alfa, for anaemia in adults with lower-risk MDS. The trial is set to begin in September and aims to recruit 300 patients, some of whom may remain enrolled for up to five years.

The study’s main goal is to determine whether elritercept can reduce patients’ need for blood transfusions, and to compare its safety profile with that of current treatment. It will also track patient-reported fatigue, transfusion burden, quality of life, and immune response to therapy.

To take part, patients must be 18 or older, have a confirmed MDS diagnosis (per WHO 2016 classification), and have less than 5% blasts on an evaluable bone marrow sample collected at screening. They must also require regular red blood cell transfusions and have an ECOG performance status of 0 to 2. Patients who have previously been treated with epoetin alfa, darbepoetin alfa (Aranesp), immunomodulatory drugs, or hypomethylating agents are not eligible.

Elritercept, given as a subcutaneous injection, works by modifying the activin receptor type IIA ligand trap, inhibiting select transforming growth factor-β (TGF-β) superfamily ligands, including activin A, to promote red blood cell and platelet production at multiple stages of blood cell formation. Takeda acquired rights to the drug through a licensing agreement with Keros Therapeutics in December 2024 and is developing it for anaemia linked to blood cancers, including MDS.

The clinical study team for elritercept at King’s College London is led by Dr Austin Kulasekararaj, Consultant Haematologist and Chief Investigator. (Image: King’s College Hospital NHS Foundation Trust)