Acceleron Pharma Inc. said luspatercept continued to provide clinically meaningful benefits to patients with a certain type of bone marrow cancer.
The Cambridge, Mass.-based biopharmaceutical company is evaluating the investigational drug in patients with lower-risk myelodysplastic syndromes in two phase 2 studies. Patients with MDS, a type of cancer, do not produce enough red blood cells, which causes chronic anemia that can lead to fatigue and increased death rates.
MDS-related chronic anemia often fails to respond to unapproved therapies, requiring many patients to undergo frequent red blood cell transfusions.
Results from the phase 2 trials showed 55% of 101 patients had a clinically meaningful improvement in erythroid, or immature red blood cells. Meanwhile, 30 of 68 patients who were previously dependent on red blood cell transfusions did not have to undergo the procedure for at least eight weeks.
Further, multiple patients remain on treatment through 40 months, showing significant increases in hemoglobin and reduced need for red blood cell transfusions.
Side effects included ascites, or abnormal fluid accumulation in the abdomen; increase in bilirubin, a yellow pigment in the blood; bone pain; hypertension; inflammation of the mucus membranes; platelet count increase; and transformation to acute myeloid leukemia, a cancer of the blood and bone marrow.
Serious side effects were general physical health deterioration, muscular weakness, musculoskeletal pain and muscle pain.
Acceleron and Celgene Corp. are jointly developing luspatercept as part of a global collaboration. Luspatercept is not yet approved for any use in any country.
Top-line results from the phase 3 Medalist trial are on track for the middle of 2018, Acceleron said. The company will launch the phase 3 Commands study in first-line, lower-risk MDS in the third quarter, while the phase 2 Beyond study in non-transfusion-dependent beta-thalassemia — a blood disorder that affects hemoglobin production — and a phase 2 trial in myelofibrosis — a serious bone marrow disorder that affects blood cell production — are ongoing.
The 2018 American Society of Clinical Oncology meeting is expected to bring together more than 32,000 professionals from all over the world, with more than 2,500 study abstracts to be presented on site and an additional 3,350 abstracts to be published online.
