According to a story from BioSpace, CTI BioPharma Corp. has recently concluded an End of Phase 2A meeting with representatives from the US Food and Drug Administration (FDA). This meeting is in regards to the development of pacritinib as a treatment for myelofibrosis, a form of cancer that affects the bone marrow. During the meeting, the company and the agency agreed that the next phase 3 trial would include 180 patients and 200mg doses of pacritinib, to be administered twice per day.
Myelofibrosis is considered a rare type of bone marrow cancer. The disease is characterized by the excessive accumulation of abnormal stem cells in the bone marrow which trigger a process called fibrosis, or scarring. Over time, the bone marrow is replaced with scar tissue. While the exact cause of myelofibrosis is not known, genetic mutations affecting the MPL, JAK2, and CALR genes are known risk factors. Symptoms of myelofibrosis include enlarged spleen, anemia, shortness of breath, easy bruising and bleeding, greater risk of infection, bone pain, gout, fatigue, weight and appetite loss, and increased blood cell volume. As a cancer that affects stem cells, stem cell transplant can cure the disease. However, this process carries many significant risks. Other forms of treatment are symptomatic and supportive and do not alter the course of myelofibrosis. There is a dire need for safer and more effective therapies for the disease. To learn more about myelofibrosis, click here.
The company plans to compare pacritinib to the current standard of care for myelofibrosis and is hoping to initiate trial operations in the third quarter of this year. With this time table, CTI hopes to have data from this study available by mid 2021.
Pacritinib is classified as an orally available kinase inhibitor which will specifically act on the JAK2, CSF1R, IRAK1, and FLT3 kinases. These kinases are frequently mutated in patients with blood-related cancers and other malignancies such as myelofibrosis and are likely to play a critical role in the development of these illnesses. This mechanism of action means that pacritinib has the potential to be effective in a variety of related cancers, such as myelodysplastic syndromes and acute myeloid leukemia.