Cell Therapeutics Inks Deal With FDA On SPA For PERSIST-2 Trial Of Pacritinib

(RTTNews) - Cell Therapeutics Inc. or CTI (CTIC) has entered into an agreement with the U.S. Food and Drug Administration or FDA on a Special Protocol Assessment or SPA for the planned pivotal Phase 3 study, known as the PERSIST-2 trial, evaluating pacritinib compared with best available therapy, including approved JAK2 inhibitors such as ruxolitinib, in patients with myelofibrosis whose platelet counts are <100,000/uL. The SPA is a written deal between CTI and the FDA concerning the design, endpoints and planned statistical analysis approach of the trial to be used in support of a potential New Drug Application or NDA submission.

The PERSIST-2 trial is the second of two planned Phase 3 trials in myelofibrosis patients. CTI may begin the PERSIST-2 trial in the fourth quarter of 2013. Patients would be randomized to receive 200 mg pacritinib twice daily, 400 mg pacritinib once daily or best available therapy. 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 at 24 weeks of treatment and the percentage of patients achieving a Total Symptom Score reduction of 50 percent or greater using six key symptoms as measured by the modified Myelofibrosis Symptom Assessment Form diary.

The study is likely to enroll patients at clinical sites in the U.S., Europe and Australia.