The full clinical hold (February 2016) implemented by the U.S. Food and Drug Administration (FDA) on all clinical trials conducted under the Investigational New Drug (IND) application for pacritinib has now been removed and the first patients have enrolled in the trial. The Company’s new trial, PAC203 plans to enroll up to approximately 105 patients with primary myelofibrosis who have failed prior ruxolitinib therapy to evaluate the safety and the effectiveness of pacritinib. The Company expects to start the trial in the second quarter of 2017.
Myelofibrosis is a type of blood cancer known as a myeloproliferative neoplasm. It involves the abnormal development and function of bone marrow cells that produce blood cells which leads to the formation of scar tissue in the bone marrow. This can cause anemia, enlargement of the spleen and liver, fatigue, and other problems. In some patients with myelofibrosis, the condition progresses to acute myeloid leukemia.
Proteins known as JAK1 and JAK2 may play a role in the development of MPNs, including myelofibrosis, by causing the body to make the wrong number of blood cells. Drugs that suppress JAK1 and JAK2 are used to treat different forms of MPN by reducing the number of abnormal blood cells associated with these disorders. JAK1/JAK2 inhibitors can, however, result in too much suppression of the bone marrow’s production of blood cells. Known as myelosuppression, this condition can negatively impact patient health and quality of life.
Pacritinib is designed to fight myelofibrosis by interfering with the kinases JAK2 and FLT3 to potentially keep tumors from developing. Unlike other JAK inhibitors, pacritinib does not cause myelosuppression, which, if safe and effective, could offer an advantage to patients. Information about the clinical trial is available on the NCI website: https://clinicaltrials.gov/ct2/show/NCT03165734?term=cti+biopharma&recrs=a&rank=1