Results of Phase 2 Study Investigating Luspatercept in MDS

The results of a phase 2 study that explored the effects of luspatercept in patients with lower-risk myelodysplastic syndromes (MDS) were presented at the 22nd Congress of the European Hematology Association by Acceleron Pharma Inc. The company is developing the drug with Celgene.

The phase 2 study included lower-risk MDS patients who received therapeutic doses of luspatercept. Of the 88 patients in the study, 50% reached a clinically meaningful erythroid response, according to the International Working Group’s Hematologic Improvement Erythroid criteria. This response was reflected through an increase in hemoglobin or a decrease in red blood cell (RBC) transfusion burden.

“This phase 2 update further supports our confidence that luspatercept could become a potential first-in-class treatment for lower-risk MDS patients. With some patients continuing on study for more than 26 months, we are very encouraged by both the durability of response and safety profile of luspatercept,” said Habib Dable, president and CEO of Acceleron. “With phase 3 trials across 2 indications ongoing and new studies planned, we and Celgene remain committed to exploring the full opportunity for luspatercept to transform patients’ lives.”

Furthermore, 38% of 60 patients that received 2 or more units of RBC per 8 weeks transfusion burden had achieved RBC transfusion independence at 8 weeks or more while those with low transfusion burden increased in hemoglobin for up to 26 months as many continued to be treated.

“The longer term results of these phase 2 studies reinforce the potential of inhibiting ligands in the TGF-beta superfamily for patients with lower-risk MDS,” said Michael Pehl, president of hematology/oncology for Celgene. “With the phase 3 study now fully enrolled, we look forward to advancing luspatercept as part of our ongoing commitment to individuals with MDS around the world.”