CTI BioPharma announced that following a planned second interim data review of the PAC203 study by the Independent Data Monitoring Committee (IDMC), the study will continue to the full enrollment of 150 patients. The IDMC did not identify significant drug- or dose-related safety concerns and specifically did not identify any concerns around hemorrhagic or cardiac toxicity. Following consultation with the U.S. Food and Drug Administration (FDA) and the IDMC, the company restricted this interim analysis to a safety review thereby allowing the study to continue to full enrollment. Continuing to full enrollment allows collection of a complete dataset (including efficacy, safety, pharmacokinetic and pharmacodynamic data) that will be used to determine the optimal dose for further development. Prior to the second interim analysis and with the agreement of the IDMC, the Company had submitted a protocol amendment to the FDA, establishing safety reviews as the basis for the second interim analysis and for subsequent interim analyses.
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"Continuing the PAC203 study with all three treatment arms allows us to obtain the maximum amount of data to determine the optimal dose for our planned Phase 3 registrational study, which is expected to address a patient population with severe thrombocytopenia," said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI BioPharma.
"The PAC203 study is on track to complete its objective and we continue to expect to complete enrollment by the end of 2018. We plan to present clinical data at an upcoming medical conference, potentially at the ASCO conference in 2019."
The PAC203 study is evaluating the safety and efficacy of three dosing schedules (100 mg once daily, 100 mg twice daily and 200 mg twice daily) over 24 weeks in patients with myelofibrosis previously treated with ruxolitinib.
Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. The JAK family of enzymes is 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. In addition to myelofibrosis, the kinase profile of pacritinib suggests its potential therapeutic utility in conditions such as acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), and chronic lymphocytic leukemia (CLL), due to its inhibition of c-fms, IRAK1, JAK2 and FLT3.