On July 3, 2013 Cell Therapeutics reported that a comprehensive article summarizing preclinical and clinical data for pacritinib, an oral JAK2/FLT3 inhibitor, authored by Srdan Verstovsek, M.D., Ph.D., et al., was published in the journal Drugs of the Future 2013 (Press release CTI BioPharma, JUL 2, 2013, View Source;p=RssLanding&cat=news&id=1835062 [SID:1234500579]). The article reviews the preclinical pharmacology and pharmacokinetics in addition to the safety and efficacy results from the Phase 1 and 2 clinical studies of pacritinib in patients with myelofibrosis and lymphoma.
The authors conclude that the clinical trials to date support the safety, efficacy and predictable pharmacokinetic profile of pacritinib. The hematological adverse events have been uncommon and no dose reductions for thrombocytopenia were necessary in the Phase 2 studies. In the two studies that enrolled patients with myelofibrosis, pacritinib led to disease response, improvement in splenomegaly (enlargement of the spleen) and lowered white blood cell count, and symptom reduction in patients with and without thrombocytopenia, or low blood cell count.
Pacritinib is currently being evaluated in a randomized Phase 3 clinical trial, known as PERSIST-1, in patients with myelofibrosis. Because of pacritinib’s relative lack of bone marrow suppression, there are no study entry restrictions due to thrombocytopenia or anemia and patients with platelet and red blood cell transfusion dependence are allowed to enroll in the ongoing PERSIST-1 trial. More details on this study can be found at www.clinicaltrials.gov. Pacritinib has orphan drug designation in the United States and Europe.