New therapy for anaemic myelodysplastic cancer patients

Bristol Myers Squibb (BMS) has announced Reblozyl^® (luspatercept-aamt), the first erythroid maturation agent, showed improvement in red blood cell transfusion independence (RBC-TI) and haemoglobin (Hb) increase in severely anaemic adults with very low, low or intermediate-risk myelodysplastic syndromes (MDS).

Reblozyl is a first-line therapy that promotes late-stage red blood cell maturation in animal models. The drug is approved in the US for treating:

• Anaemia in adults with beta thalassemia who require regular red blood cell transfusions
• Anaemia failing an erythropoiesis stimulating agent and requiring two or more red blood cell units over eight weeks in adults with very low to intermediate-risk myelodysplastic syndrome with ring sideroblasts (MDS-RS) or myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).

The Phase III open-label, randomised trial (NCT03682536) evaluated the efficacy and safety of Reblozyl versus epoetin alfa. The primary endpoint assessed in this study was RBC-TI for 12 weeks, with a mean haemoglobin increase ≥1.5g/dL. The key secondary endpoints were RBC-TI for 24 weeks, RBC-TI ≥12 weeks and erythroid response of at least eight weeks during weeks 1-24 of the study.

The safety results for the trial were consistent with previous studies. No new safety issues were reported.

“While advancements have been made in the treatment of anaemia for patients with myelodysplastic syndromes, there remains a significant need for new and better first-line treatment options for patients with transfusion-dependent MDS,” explained Dr Noah Berkowitz, Senior Vice President of Haematology Development at BMS.

BMS will do a full evaluation of the clinical trial data and work with investigators to present results at an upcoming medical meeting. The company will also discuss results with health authorities.

The drug is being developed and commercialised alongside Merck following Merck’s acquisition of Acceleron Pharma, Inc. in November 2021.