Cell Therapeutics Begins Enrollment in Phase 3 PERSIST-1 Trial of Pacritinib for the Treatment of Myelofibrosis
SEATTLE, Jan. 9, 2013 /PRNewswire/ -- Cell Therapeutics, Inc. (CTI) (Nasdaq and MTA: CTIC) today announced that the Company has initiated clinical trial sites and began enrolling patients in a Phase 3 clinical trial, known as PERSIST-1 or PAC325, for pacritinib, CTI's investigational JAK2 inhibitor, which is being evaluated for the treatment of patients with myelofibrosis. The randomized trial is expected to enroll 270 patients and will evaluate the safety and efficacy of pacritinib compared to best available therapy, excluding JAK inhibitors, in patients with myelofibrosis. Pacritinib is a selective oral JAK2 inhibitor that demonstrated meaningful clinical benefits and good tolerability in myelofibrosis patients in Phase 2 clinical trials, without apparent drug-related thrombocytopenia or anemia. Myelofibrosis patients will be enrolled in the PERSIST-1 trial without exclusion for low platelet counts.
Principal Investigators for the trial are Ruben A. Mesa, M.D., Deputy Director, Mayo Clinic Cancer Center and Chair, Hematology and Medical Oncology, Mayo Clinic in Arizona, and Claire Harrison, M.D., Consultant Hematologist, Guy's and St. Thomas' NHS Foundation Trust, Guy's Hospital, London, United Kingdom.
"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 Steven E. Benner, M.D., Chief Medical Officer of CTI. "Data from earlier studies of pacritinib showed a clinically meaningful improvement in symptoms without suppression of platelets or red blood cells. We believe that pacritinib has the potential to offer an effective and well-tolerated treatment option for myelofibrosis patients, and are pleased to be initiating the Phase 3 PERSIST-1 clinical trial."
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.1
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 Europe, Australia and the United States.
Additional PERSIST-1 (A Randomized Controlled Phase 3 Study of Oral P acritinib v ER sus Be S t Available Therapy in Pat I ent S with Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post Essential T hrombocythemia 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.
About Cell Therapeutics, Inc.
Cell Therapeutics (Nasdaq and MTA: CTIC) is a biopharmaceutical company committed to the development and commercialization of an integrated portfolio of oncology products aimed at making cancer more treatable. CTI is headquartered in Seattle, WA. For additional information and to sign up for email alerts and get RSS feeds, please visit www.CellTherapeutics.com.
Source: Cell Therapeutics, Inc.
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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 U.S. Food and Drug Administration and/or the European Medicines Agency; that pacritinib may not satisfy a medical need not currently addressed with existing non-selective JAK1/JAK2 inhibitors; that the PERSIST-1 clinical trial of pacritinib may not occur as planned; that 270 patients may not enroll in the PERSIST-1 clinical trial; that the second Phase 3 clinical trial of pacritinib may not occur as planned; that CTI may not be able to successfully implement its plans, strategies and objectives related to the development of pacritinib; CTI's ability to continue to raise capital as needed to fund its operations, competitive factors, technological developments, costs of developing, producing and selling CTI's product candidates; and the risk factors listed or described from time to time in CTI's filings with the Securities and Exchange Commission including, without limitation, CTI's most recent filings on Forms 10-K, 10-Q and 8-K. Except as may be required by law, CTI does not intend to update or alter its forward-looking statements whether as a result of new information, future events, or otherwise.
SOURCE Cell Therapeutics, Inc.
Web Site: http://www.celltherapeutics.com
Posted: January 2013