July 3, 2013
/PRNewswire/ -- Cell Therapeutics, Inc. (CTI) (NASDAQ and MTA: CTIC) today announced 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
. 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.
The link to the publication is available at
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
. Pacritinib has orphan drug designation in
the United States
Myelofibrosis is classified as a myeloproliferative neoplasm and is a chronic bone marrow disorder. Myelofibrosis is caused by the accumulation of malignant bone marrow cells that triggers an inflammatory response, scarring the bone marrow and limiting its ability to produce red blood cells prompting the spleen and liver to take over this function. Symptoms that arise from this disease include enlargement of the spleen, anemia, extreme fatigue and pain. It is estimated that the prevalence of myelofibrosis is approximately 30,000 in
the United States
Pacritinib is an oral, once-a-day, tyrosine kinase inhibitor (TKI) with dual activity against JAK2 and FLT3. The JAK family of enzymes are a central component in signal transduction pathways, which are critical to normal blood cell growth and development as well as inflammatory cytokine expression and immune responses. Mutations in these kinases have been shown to be directly related to the development of a variety of blood related cancers including myeloproliferative neoplasms, leukemia and lymphoma. Pacritinib may offer an advantage over other JAK inhibitors through effective treatment of symptoms while having less treatment-emergent thrombocytopenia and anemia than has been seen in currently approved and in-development JAK inhibitors.