Researchers reported a number of important findings in the treatment of myeloproliferative neoplasms (MPNs) at the 2019 Annual Meeting of the American Society of Clinical Oncology:

FDA approves fedratinib for treatment of certain types of myelofibrosis

In August 2019, the Food and Drug Administration (FDA) approved fedratinib for the treatment of patients with intermediate-2 or high-risk primary or secondary myelofibrosis (MF), including post-polycythemia vera and post-essential thrombocythemia MF. The approval was based on the results of the JAKARTA trial.

What Patients Need to Know

Fedratinib is a Janus kinase (JAK) inhibitor, a type of drug that works by inhibiting the activity of one or more of the Janus kinase family of enzymes. Ruxolitinib, another JAK inhibitor, was approved for post-polycythemia vera and post-essential thrombocythemia MF in 2011.

Investigational drug shows clinical benefit in treatment of refractory myelofibrosis

Interim results from the phase II MANIFEST trial indicated that the investigational drug CPI-0610 showed signs of beneficial clinical activity in the treatment of refractory (resistant to treatment) myelofibrosis, both as a monotherapy (a drug used alone) and in combination with the JAK inhibitor ruxolitinib.

What Patients Need to Know

CPI-0610 prevents bromodomain and extraterminal (BET) proteins from attaching to certain cancer-causing genes, which may “turn off” the genes and stop them from making new cancer cells.

Targeted therapy tagraxofusp being evaluated as treatment for relapsed/refractory myelofibrosis

A multicenter, phase I/II trial is evaluating the effectiveness and safety of tagraxofusp in the treatment of people with intermediate or high-risk myelofibrosis (MF) that has relapsed or is refractory (resistant) to treatment with a JAK inhibitor. Trial participants also include people who, because of side effects, could not tolerate the continued use of a JAK inhibitor as a treatment for MF.

What Patients Need to Know

Tagraxofusp, also called SL-401, is a targeted therapy directed at the interleukin-3 receptor CD123, which is expressed (present) on a variety of malignancies, including myelofibrosis.