Cell Therapeutics Begins Enrollment For PERSIST-2 Phase 3 Trial Of Pacritinib

(RTTNews) - Cell Therapeutics Inc. (CTIC) announced that it has initiated a Phase 3 clinical trial, known as PERSIST-2, which will evaluate pacritinib, a novel, investigational JAK2/FLT3 inhibitor, in patients with myelofibrosis whose platelet counts are less than or equal to 100,000 per microliter (uL).

The company expects to enroll up to 300 patients for the trial in North America, Europe, Australia and New Zealand within 12 to 14 months.

In October 2013, the company reached agreement with the U.S. Food and Drug Administration or FDA on a Special Protocol Assessment or SPA for the PERSIST-2 trial, which is a written agreement between the company and the FDA regarding the planned design, endpoints and statistical analysis approach of the trial to be used in support of a potential New Drug Application, or NDA, submission.

PERSIST-2 is the second of two planned Phase 3 trials in the pacritinib development program for myelofibrosis.

The trial will evaluate pacritinib as compared to best available therapy, including approved JAK2 inhibitors that are dosed according to the product label for myelofibrosis patients with thrombocytopenia. Patients will be randomized (1:1:1) to receive 200 mg pacritinib twice daily or BID, 400 mg pacritinib once daily or QD or best available therapy.

Under the SPA, the agreed upon co-primary endpoints are the percentage of patients achieving a 35 percent or greater reduction in spleen volume measured by MRI or CT scan from baseline to 24 weeks of treatment and the percentage of patients achieving a Total Symptom Score or TSS reduction of 50 percent or greater using six key symptoms as measured by the modified Myeloproliferative Neoplasm Symptom Assessment (MPN-SAF TSS 2.0) diary from baseline to 24 weeks.

The company stated that, based on pacritinib's efficacy and tolerability profile demonstrated to date, it is pursuing a broad approach to advancing this therapy for myelofibrosis patients by conducting two Phase 3 clinical trials: one in a broad set of patients without limitations on blood platelet counts, the ongoing PERSIST-1 trial, and the other in patients with low platelet counts, the PERSIST-2 trial.

Pacritinib is an oral tyrosine kinase inhibitor with dual activity against JAK2 and FLT3. 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.