The MEDALIST study assessed the efficacy and safety of luspatercept versus placebo in patients with very low, low or intermediate risk myelodysplastic syndromes (MDS) with chronic anaemia and refractory to, intolerant of, or ineligible for treatment with an erythropoietin-stimulating agent (ESA), ring sideroblast-positive and require frequent RBC transfusions.

The drug achieved its key goal of inducing significant improvement in red blood cell transfusion independence of at least eight consecutive weeks compared to placebo, as well as the key secondary endpoint of showing a "highly statistically significant improvement" in RBC transfusion independence of at least 12 consecutive weeks during the first 24 weeks.

The results "demonstrate the potential clinical benefit of luspatercept as an erythroid maturation agent for the treatment of chronic anemia in patients with low-to-intermediate risk MDS," said Celgene’s chief medical officer Jay Backstrom.

"Based on these results, we look forward to preparing the dossier for global regulatory submissions and also investigating the clinical potential of luspatercept in ESA-naïve, low-to-intermediate risk MDS patients through the initiation of our Phase III COMMANDS study."

The companies said they plan to submit regulatory applications in the US and Europe in the first half of 2019.

MDS are a group of cancers in which immature blood cells in the bone marrow fail to mature and therefore do not become healthy blood cells. Symptoms can include tiredness, shortness of breath, easy bleeding, or frequent infections.