CTI BioPharma Corp. announced that it has completed recruitment in the PERSIST-1 pivotal Phase 3 clinical trial of pacritinib, a novel oral JAK2/FLT3 inhibitor that is being evaluated for the treatment of myelofibrosis. Under the development and commercialization agreement for pacritinib with Baxter International Inc., CTI expects to receive a $20 million development milestone payment in connection with the first treatment dosing of the last patient enrolled in PERSIST-1. CTI expects to achieve this milestone and receive payment by mid-third quarter 2014.
“The PERSIST-1 clinical trial evaluating pacritinib, a novel JAK2/FLT3 inhibitor, is the most inclusive study of myelofibrosis patients seen in routine clinical practice to date,” said Claire Harrison, consultant hematologist, Guy’s and St. Thomas’ NHS Foundation Trust, Guy’s Hospital, London, United Kingdom and one of the principal investigators for PERSIST-1. “Previous randomized clinical trials have excluded patients with low platelet counts (below 50,000-100,000 per microliter (uL)) despite almost 30% of all myelofibrosis patients having disease-related thrombocytopenia. Currently available JAK1/JAK2 inhibitors are associated with treatment-emergent myelosuppression, including thrombocytopenia as a side effect of their therapy requiring reduced doses and sometimes early cessation of treatment when used in patients with disease-related thrombocytopenia. These patients represent an unmet medical need; a non-myelosuppressive JAK2 inhibitor would represent a significant advancement in the treatment of this chronic disease.”
The PERSIST-1 trial is the first of two Phase 3 trials in the pacritinib development program in myelofibrosis. The second Phase 3 trial, PERSIST-2, is currently evaluating pacritinib for the treatment of patients with low platelet counts compared to best available therapy, including approved JAK2 inhibitors at their recommended dose and schedule for myelofibrosis patients with thrombocytopenia. The two clinical trials are intended to support an anticipated New Drug Application (NDA) regulatory submission in the U.S. in late 2015, followed by an anticipated Marketing Authorization Application (MAA) in Europe in 2016.
“Completing recruitment in the PERSIST-1 trial is a significant milestone in our development program for pacritinib, and we look forward to reporting top-line results in early 2015,” said James Bianco, president and CEO of CTI BioPharma. “The high physician and patient interest in participating in this trial underscores the need for new, effective and less toxic treatment options for patients with myelofibrosis.”
The PERSIST-1 trial was designed to enroll approximately 320 patients and is a randomized, open-label, multicenter trial comparing the efficacy and safety of pacritinib with that of best available therapy, other than JAK inhibitors, in patients with primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis, without exclusion for low platelet counts. The primary endpoint is the percentage of patients achieving a greater than or equal to 35% reduction in spleen volume measured by MRI or CT at 24 weeks of treatment.
Date: July 1, 2014
Source: CTI BioPharma
Filed Under: Drug Discovery