|View printer-friendly version|
|Cell Therapeutics Begins Enrollment in Phase 3 PERSIST-1 Trial of Pacritinib for the Treatment of Myelofibrosis|
Principal Investigators for the trial are
"Current treatment of myelofibrosis by targeting JAK2 inhibition has been shown to be effective in managing the debilitating symptoms that are associated with this disease, although thrombocytopenia and anemia continue to be a challenge in managing this disease," said
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
PERSIST-1 is a multicenter, randomized, controlled Phase 3 trial comparing the efficacy and safety of pacritinib with that of best available therapy in patients with primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis. A total of 270 eligible patients will be randomized 2:1 to receive either pacritinib 400 mg taken orally, once daily or the best available therapy. Best available therapy includes any physician-selected treatment with the exclusion of JAK inhibitors. There will be no exclusion by patient platelet count. The primary endpoint will be the percentage of patients achieving a ≥ 35% reduction in spleen volume measured by MRI or CT at 24 weeks of treatment. The trial is expected to enroll patients at clinical sites in
Additional PERSIST-1 (A Randomized Controlled Phase 3 Study of Oral Pacritinib vERsus BeSt Available Therapy in PatIentS with Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post Essential Thrombocythemia Myelofibrosis) study details will be available at www.clinicaltrials.gov.
PERSIST-1 is the first of two planned Phase 3 clinical trials in patients with myelofibrosis. The second Phase 3 clinical trial is planned to evaluate pacritinib compared to best available therapy, including JAK inhibitors, in patients with myelofibrosis whose platelet counts are <100,000 / µL.
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. Pactrinib has demonstrated encouraging results in Phase 1 and 2 studies for patients with myelofibrosis. Pacritinib has orphan drug designation in the U.S. and Europe.
This press release includes forward-looking statements that involve a number of risks and uncertainties the outcome of which could materially and/or adversely affect actual future results and the market price of CTI's securities. Specifically, the risks and uncertainties that could affect the development of pacritinib include risks associated with preclinical and clinical developments in the biopharmaceutical industry in general, and with pacritinib in particular, including, without limitation, the potential failure of pacritinib to prove safe and effective for the treatment of patients with myelofibrosis, either alone or in combination regimens, as determined by the