Cell Therapeutics, Inc. (CTI), a Seattle-based biopharmaceutical company developing cancer treatments, announced that they initiated the PERSIST-2 Phase-3 clinical trial for the evaluation of pacritinib, which is a JAK2/FLT3 inhibitor used in patients with myelofibrosis. Study leaders expect to enroll up to 300 patients in North America, Europe, Australia and New Zealand by May 2015.
Myelofibrosis is a chronic bone marrow disorder that limits a patient’s ability to produce red blood cells. The disease also causes over-functioning of the spleen and liver, resulting in an enlargement of the spleen, anemia, extreme fatigue, and pain.
Pacritinib is taken by patients orally and inhibits tyrosine kinases JAK2 and FLT3. It is known that mutations in the JAK family of enzymes cause a variety of blood cancers, such as myeloproliferative neoplasms, leukemia and lymphoma. As a result pacritinib could have better efficacy as therapeutic inhibitors against them.
“JAK2 inhibitors have revolutionized the treatment of myelofibrosis by providing patients with an effective way to manage their disease,” said Srdan Verstovsek, MD, PhD, principal investigator of PERSIST-2, and Director, Clinical Research Center for MPNs, at The University of Texas MD Anderson Cancer Center.
PERSIST-2 is designed as a randomized, open-label, multi-center clinical trial to compare the therapeutic effect of pacritinib to other approved JAK2 inhibitors. Patients with platelet counts up to 100,000/uL will be randomized evenly into 3 groups, receiving 200 mg pacritinib twice daily, 400 mg pacritinib once daily, or the best currently-available therapy. Researchers will then evaluate pacritinib based on the percentage of patients achieving a 35% or greater reduction in spleen volume measured by MRI or CT scan in 24 weeks of treatment, and the percentage of patients achieving a Total Symptom Score (TSS) reduction by 50% or greater based on the modified Myeloproliferative Neoplasm Symptom Assessment (MPN-SAF TSS 2.0) diary in 24 weeks.
“With the initiation of the PERSIST-2 trial, we believe that the registration program for pacritinib is on track for a potential NDA submission in the latter part of 2015,” said James A. Bianco, MD, President and CEO of CTI. “We have seen meaningful clinical benefits and good tolerability with pacritinib in myelofibrosis patients in Phase 2 trials without apparent drug-related thrombocytopenia or anemia. As such, we have had strong interest in site participation for this trial and will work diligently to activate these sites over the next several months.”