What are the Benefits of Elritercept for Myelofibrosis? Results from Phase 2 RESTORE Trial

Myelofibrosis is a blood cancer that is characterized by ineffective blood cell production, an enlarged spleen, fatigue, and low blood counts. 

Many myelofibrosis patients also have anemia, which not only lowers quality of life but is also linked to shorter survival. While current treatments, such as JAK inhibitors, can reduce spleen size and ease symptoms, they often worsen anemia and other blood-related complications. That’s why new treatments like elritercept are being studied to help manage these issues more effectively.

At the 66th annual American Society of Hematology, Dr. Claire Harrison presented updates from the ongoing phase 2 RESTORE trial NCT05037760 that evaluates elritercept alone and in combination with the JAK inhibitor ruxolitinib in patients with myelofibrosis who have anemia.

What is Elritercept?

Elritercept is designed to restore blood cell production by blocking the activin A protein, which is associated with ineffective blood cell production and progression. Researchers expect this medication to improve hemoglobin levels, reduce the need for blood transfusions, and help control other symptoms of myelofibrosis. You can read more details about this new medication here. 

Key Findings from the Trial

As of November 2024, 73 patients had received elritercept: 29 as monotherapy and 44 in combination with ruxolitinib.

• Anemia and hemoglobin improvements: 55.6% of all patients had an increase in hemoglobin. One patient's hemoglobin had a rapid increase from 10.2 to 12.1 g/dL, requiring a dose reduction as part of the trial’s safety protocol.
• Reduced need for blood transfusions: was seen in 72% of patients with elritercept therapy and in 68% of patients taking combination therapy. Notably, 24% of all patients achieved transfusion independence 
• Platelet levels: were generally maintained or improved in all participants including those with low platelet counts at baseline. However, only 6 patients from the elritercept group required platelet transfusions, and the platelet levels were higher for the combination group. 
• Spleen size reduction: 39% of patients enrolled had a spleen reduction equal or greater than 50% the spleen size within 24 weeks 
• Symptom relief: 73% of the total patients enrolled had a significant reduction in their symptoms. 

Safety and Side Effects

Elritercept was generally well tolerated. The most common side effects were:

• Low platelet counts (20%)
• Diarrhea (19%)
• Only 6 patients of each group discontinued treatment due to side effects.

Accelerating Elritercept’s FDA Approval

The U.S. Food and Drug Administration (FDA) granted Fast Track designation for the development of elritercept for very low-, low- and intermediate-risk MDS, and myelofibrosis.

To accelerate the distribution of elritercept, Takeda will be partnering with Keros Therapeutics through a licencing agreement to further develop, manufacture and commercialize elritercept.

“Elritercept has the potential to make a meaningful difference for patients with blood cancers, one of our key areas of strategic focus,” said Teresa Bitetti, President of the Global Oncology Business Unit at Takeda.

HealthTree will follow up on elritercept news as soon as there are new updates. 

What This Means for Patients

The results from the RESTORE trial suggest that elritercept may help address the anemia associated with myelofibrosis, whether caused by the disease itself or as a side effect of JAK inhibitors. Elritercept’s ability to maintain or improve platelet levels, reduce spleen size, and alleviate symptoms makes it a promising potential option as single-agent therapy or in combination. Further research is ongoing, and additional data will be presented as more patients are followed over a longer period.

For myelofibrosis patients and caregivers, staying informed about new treatments like elritercept is crucial for self-advocacy. Clinical trials provide opportunities to access innovative therapies before they become widely available, and understanding the latest research can help patients have more informed discussions with their healthcare providers about their treatment options.

Stay Tuned for More Clinical Trial Updates with HealthTree! 

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MYELOFIBROSIS NEWS

Sources: 

• Hematological Improvement and Other Clinical Benefits of Elritercept As Monotherapy and in Combination with Ruxolitinib in Participants with Myelofibrosis from the Ongoing Phase 2 Restore Trial