HealthTree Logo
search more_vert
person Sign In / Create Account
ASH 2023: Phase 1/2 Study Of Zilurgisertib Monotherapy And In Combination With Ruxolitinib For Anemic Myelofibrosis Patients
Posted: Jan 05, 2024
ASH 2023: Phase 1/2 Study Of Zilurgisertib Monotherapy And In Combination With Ruxolitinib For Anemic Myelofibrosis Patients image

Myelofibrosis disrupts the body’s normal production of blood cells. This can cause scarring in the bone marrow that leads to anemia, which can worsen over time. Hepcidin is a hormone produced in the liver that acts as an iron regulator which can cause anemia and chronic inflammation due to elevated levels in patients with myelofibrosis. 

A New Study

An abstract presented by Dr. Sanjay Mohan from Vanderbilt-Ingram Cancer Center at the 2023 American Society of Hematology conference discussed results from a Phase 1/2 study looking at the use of a medication called zilurgisertib administered both by itself and in combination with ruxolitinib in patients with anemia due to myelofibrosis. Zilurgisertib is an activin receptor-like kinase-2 inhibitor. “Activin receptor-like kinase-2 (ALK-2) contributes to myelofibrosis-associated anemia via hepcidin upregulation.” This study is currently ongoing and recruiting.

Criteria to Participate in the Study

  • Patients must be 18 years or older. (Median age is currently 73)
  • Patients must have intermediate or high-risk with primary or secondary myelofibrosis
  • Patients must have anemia whether they are transfusion or non-transfusion dependent

The Study Specifics

Group 1: The study started patients at 50mg of zilurgisertib and increased dosage until the maximum tolerated dose was reached.

Group 2: The second group of the study started patients on ruxolitinib for the first 12 weeks, then introduced zilurgisertib at 100mg.

The Study Results

Treatment with zilurgisertib monotherapy or in combination with ruxolitinib in this patient population was generally well tolerated,”

  • 55% of Group 1 and 25% of Group 2 were transfusion-dependent when starting the study
  • Hepcidin was high in both groups to begin with
  • Thrombocytopenia (low platelet levels) was the most common side effect found among participants 
  • The higher doses of both medications lowered the anemia risk in patients


Anemia can be a serious condition for myelofibrosis patients. Fatigue, weakness, shortness of breath and dizziness can result from myelofibrosis and its effect on the bone marrow. This ongoing study has shown that, “reduced hepcidin levels were observed at all dose levels with both monotherapy and in combination with ruxolitinib, with greater control of hepcidin over time observed at higher zilurgisertib doses. Preliminary improvements in anemia were observed in non–transfusion-dependent patients during dose escalation, suggesting potential for therapeutic activity.”

The author Lisa Foster

about the author
Lisa Foster

Lisa Foster is a mom of 3 daughters, a puzzle lover, writer and HealthTree advocate. She believes in the mission of the foundation and the team that builds it forward. She calls Houston, Texas home. 

Get the latest thought leadership on Acute Myeloid Leukemia delivered straight to your inbox.

Subscribe to the weekly "HealthTree Community for Acute Myeloid Leukemia Newsletter" for Acute Myeloid Leukemia news, life with Acute Myeloid Leukemia stories, Acute Myeloid Leukemia clinical trials, Acute Myeloid Leukemia 101 articles and events with Acute Myeloid Leukemia experts.

Thanks to our HealthTree Community for Acute Myeloid Leukemia Sponsors:

Astellas Pharma

Follow Us

facebook twitter