Elritercept Fast Tracked for Lower-Risk Myelodysplastic Syndromes

The Food and Drug Administration (FDA) has granted Fast Track designation to elritercept (KER-050) for the treatment of anemia in adults with very low-, low-, or intermediate-risk myelodysplastic syndromes (MDS).

Elritercept is an activin receptor type IIA-Fc fusion protein designed to increase red blood cell (RBC) and platelet production by inhibiting the signaling of a subset of the transforming growth factor beta (TGF-ß) family of proteins to promote hematopoiesis. The designation is supported by data from an ongoing, open-label, phase 2 trial (ClinicalTrials.gov Identifier: NCT04419649) that is assessing the effects of elritercept in patients with very low-, low-, or intermediate-risk MDS. 

Of the 79 patients who received at least 1 dose of elritercept, 60 had completed at least 24 weeks of treatment or discontinued as of the data cut-off date. Findings showed 50% of these patients (n=30/60) achieved an overall erythroid response, meeting either modified International Working Group 2006 Hematological Improvement-Erythroid response or transfusion independence for at least 8 weeks in transfusion-dependent patients who required at least 2 RBC units transfused at baseline.

The most common treatment-emergent adverse events reported were diarrhea, dyspnea, fatigue, nausea and headache. None of these patients had progressed to acute myeloid leukemia.

“Receiving Fast Track designation for KER-050 underscores the need for novel treatment options to address the serious unmet medical needs of people living with lower-risk MDS,” said Jasbir S. Seehra, PhD, President and CEO. “We look forward to working closely with the FDA as we engage on the design of a phase 3 clinical trial evaluating KER-050 in lower-risk MDS in the first half of this year.”

The FDA’s Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to therapies that are expected to have an impact on factors such as survival and daily functioning.