Peter Langmuir, MD, Group Vice President, Oncology Targeted Therapeutics at Incyte, discusses interim data of a phase 2 study assessing the combination of ruxolitinib and parsaclisib in patients with myelofibrosis. 

Myelofibrosis is a rare cancer characterized by extensive scarring in your bone marrow and the disruption of normal blood cells production. This leads to severe anemia that can cause weakness and fatigue. Bone marrow scarring can also lead to low platelet levels, which increases the risk of bleeding. Additionally, myelofibrosis often causes an enlarged spleen.

As Dr. Langmuir explains, the drug ruxolitinib has been approved for the treatment of myelofibrosis for a number of years. While many patients experience a significant reduction in spleen size and improvements in their symptoms, some patients do not. The current trial evaluates the efficacy and safety of combining parsaclisib, a PI3Kδ inhibitor, with ruxolitinib in patients with myelofibrosis who have been previously treated with ruxolitinib. The primary endpoints are spleen size reduction, improvement in symptoms, and whether the combination is well tolerated. Interim data, being presented at the 2021 EHA Virtual Congress, suggest the addition of parsaclisib to ruxolitinib leads to greater improvements in symptoms and reduction of spleen size compared to the placebo arm. The combination also appears to be well tolerated. 

Two phase 3 clinical trials are underway. One study is looking further into the safety and efficacy of the combination of ruxolitinib and parsaclisib in patients with myelofibrosis who have been previously treated with ruxolitinib. The other is looking at this drug combination as a treatment for myelofibrosis patients who have not received any previous treatment.  

To learn more about this and other rare cancers, visit https://checkrare.com/diseases/cancers